DGRT All. A 622_2008_OffLabel

Transcript

Allegato A – Farmaci oncologici off-label per uso pediatrico
NOME COMPOSTO
ACIDO
TRANSRETINOICO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI SULLA BASE
INDICAZIONI GIÀ
DELLA
DELIBERA AIFA (corsivo) E
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
Nessuna
-Neuroblastoma
-Nefroblastoma
-Leucemia promielocitica acuta
BIBLIOGRAFIA
a sostegno dell’estensione dell’indicazione
Protocolli
nazionali e
internazionali in
uso
Neuroblastoma e Nefroblastoma
- AIDA-Prot.
(lap0493)
Adamson PC et al. A phase 2 trial of all-trans-retinoic acid GIMEMA AIEOP
in combination with interferon-alpha2a in children with
recurrent neuroblastoma or Wilms tumor: a Pediatric
Oncology Branch, NCI and Children's Oncology Group
Study. Pediatr Blood Cancer 2007;49:661-665
Leucemia promielocitica acuta
Dvorak CC et al. Reinduction of relapsed acute
promyelocytic leukemia with ATRA and low dose
antimetabolite-based chemotherapy. Pediatr Blood Cancer
2007;48:582-585
Ortega JJ et al. Treatment with all-trans retinoic acid and
anthracycline monochemotherapy for children with acute
promyelocytic leukemia: a multicenter study by the
PETHEMA Group. J Clin Oncol 2005;23:7632-7640
ACIDO 13-CISRETINOICO
Nessuna
-Neuroblastoma
Neuroblastoma
- NB-AR-01
Veal G et al. Pharmacokinetics and pharmacogenetics of
13-cis-retinoic acid in children with high risk
neuroblastoma. A study of the UK Children’s Cancer
Study Group. Br J Cancer 2007;96:424-431
Reynolds CP et al. Retinoid therapy of high-risk
neuroblastoma.Cancer Lett 2003;197:185-192
1
NOME COMPOSTO
ACTINOMICINA-D
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
-Nefroblastoma
-Neuroblastoma
- Tumori solidi
(nel primo anno di vita)
- Rabdomiosarcoma
- Tumori della famiglia del sarcoma di
Ewing
- Emangiopericitoma
- Carcinoma del rene a cellule chiare
- Carcinoma nasofaringeo
- Tumori a cellule germinali
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
Tumori solidi (nel primo anno di vita)
- AIEOP T.Wilms,
Orbach D et al. Soft tissue sarcoma or malignant 2003
mesenchymal tumors in the first year of life: experience of - EpSSG-RMS &
the International Society of Pediatric Oncology (SIOP) NRSTS, 2005
Malignant Mesenchymal Tumor Committee. J Clin Oncol
- ISG/SSG III
2005;23:4363-4371
- ISG/SSG IV
Rabdomiosarcoma
Waiterhouse I and Watson A. Optimal management - AIEOP/ ISG 02
strategies for rhabdomyosarcoma in children. Paediatr (EW- REC)
Drugs 2007;9:391-400
- AIEOP/SICP
Tumori della famiglia del sarcoma di Ewing
TREP, 2005
Bernstein M et al. Ewing’s sarcoma family of tumors:
current management. Oncologist 2006;11:503-519
Emangiopericitoma
Gowans LK et al. Successful treatment of an infant with
constitutional
chromosomal
abnormality
and
hemangiopericytoma with chemotherapy alone. J Pediatr
Hematol Oncol 2007;29:409-411
Carcinoma del rene a cellule chiare
Varan A et al. Renal cell carcinoma in children:
experience of a single center. Nephron Clin Pract
2007;105:58-61
Carcinoma nasofaringeo
Küpeli S et al. Treatment results of 84 patients with
nasopharyngeal carcinoma in childhood. Pediatr Blood
Cancer 2006;46:454-458
Tumori a cellule germinali
Lo Curto M et al. Mature and immature teratomas: results of
the first paediatric Italian study. Pediatr Surg Int
2007;23:315-322
ALEMTUZUMAB
Nessuna
-Anemia aplastica grave
Anemia aplastica grave e Sindromi mielodisplastiche
2
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
-Sindromi mielodisplastiche
-Trapianto di midollo osseo
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
Rzepecki P et al. Alemtuzumab, fludarabine and
melphalan as a conditioning therapy in severe aplastic
anemia and hypoplastic myelodysplastic syndrome –single
center experience–. Jpn J Clin Oncol 2006;36:46-49
Trapianto di midollo osseo
Benesch M et al. Five-month marrow aplasia in a child
with refractory acute myeloid leukaemia: successful
management with continuous granulocyte support and
reduced intensity conditioning followed by matched
unrelated bone marrow transplantation. J Pediatr Hematol
Oncol 2005;27:236-238
3
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
ARSENICO TRIOSSIDO Nessuna
-Leucemia acuta promielocitica
BIBLIOGRAFIA
Leucemia acuta promielocitica
Protocolli
nazionali e
internazionali in
uso
- ICC APL 01
Mantadakis E et al. A comprehensive review of acute
promyelocytic leukemia in children. Acta Haematol
2008;119:73-82
Fox E et al. Phase 1 trial and pharmacokinetic study of
arsenic trioxide in children and adolescents with refractory
or relapsed acute leukemia, including acute promyelocytic
leukemia or lymphoma. Blood 2008;111:566-573
Clark JJ et al. Update in childhood acute myeloid
leukemia: recent developments in the molecular basis of
disease and novel therapies. Curr Opin Hematol
2003;10:31-39
BEVACIZUMAB
Nessuna
-Tumori solidi refrattari
Tumori solidi refrattari
-Carcinoma del rene a cellule chiare
Bender JL et al. Phase I trial and pharmacokinetic study of
bevacizumab in pediatric patients with refractory solid
tumors: a Children's Oncology Group Study. J Clin Oncol
2008;26:399-405
-Carcinoma del colon-retto
Benesch M et al. Compassionate use of bevacizumab
(Avastin) in children and young adults with refractory or
recurrent solid tumors. Ann Oncol 2008;19:807-813
Joshi DD and Banerjee T. Vascular endothelial growth
factor (VEGF) receptor antibody bevacizumab (avastin)
induces regression of renal cell carcinoma in an adolescent
resulting in residual tumorectomy. Pediatr Blood Cancer
2008;50:903-904
Benesch M et al. Compassionate use of bevacizumab
4
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
(Avastin) in children and young adults with refractory or
recurrent solid tumors. Ann Oncol 2008;19:807-813
Carcinoma del colon-retto
Saab R and Furman WL. Epidemiology and management
options for colorectal cancer in children. Paediatr Drugs
2008;10:177-192
5
NOME COMPOSTO
BLEOMICINA
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
Nessuna
-In associazione negli schemi PEB per i
tumori germinali e ABVD nel linfoma di
Hodgkin
-Tumori del SNC
BUSULFANO
-Policitemia vera
-Trombocitopenia
essenziale
-Condizionamento pretrapianto
CARBOPLATINO
-Carcinoma dell’ovaio
dell’adolescente/giovane
adulta
-Neuroblastoma in stadio 4, in
associazione a melphalan nei regimi
mieloablativi
BIBLIOGRAFIA
Tumori del SNC
Protocolli
nazionali e
internazionali in
uso
- AIEOP LH 2004
Hukin J et al. Intracystic bleomycin therapy for - AIEOP TCG 2004
craniopharyngioma in children: the Canadian experience.
- AIEOP/SICP
Cancer 2007;109:2124-2131
TREP 2005
Leucemia mielomonocitica giovanile
- AIEOP LAM
2002/01-02
- Leucemia mielomonocitica giovanile
Locatelli F et al. Hematopoietic stem cell transplantation
(HSCT) in children with juvenile myelomonocytic - NB-AR-01
leukemia (JMML): results of the EWOG-MDS/EBMT
- SIOP-AIEOP NB
trial. Blood 2005;105:410-419
Infant
Sindrome di Shwachman-Diamond
- AIEOP LAM
Sauer M et al. Substitution of cyclophosphamide and REC/01
busulfan by fludarabine, treosulfan and melphalan in a
preparative regimen for children and adolescents with - AIEOP LLA REC
Shwachman-Diamond
syndrome.
Bone
Marrow 2003
Transplant 2007;9:143-147
- AIEOP ALCL 99
- Sindrome di Shwachman-Diamond
Medulloblastoma
- Medulloblastoma
Ridola V et al. High-dose chemotherapy with autologous - INTERINFAN 06
stem cell rescue followed by posterior fossa irradiation for
local medulloblastoma recurrence or progression after - ISG/SSG III
ISG/SSG IV
conventional chemotherapy. Cancer 2007;110:156-163
- AIEOP/ISG 02
(EW-REC)
-Neuroblastoma (stadio 3 e 4) in
combinazione con altri citotossici
Sarcomi dei tessuti molli
-Sarcomi della famiglia Ewing a cattiva
prognosi in associazione a melphalan
nei regimi mieloablativi
-Tumore di Wilms ad istologia
sfavorevole o metastatico in
combinazione con altri farmaci
- HLH 2004
- EICNHL ( ALCL
relapse )
Bernbeck B et al. Serial intense chemotherapy combining
topotecan, etoposide, carboplatin and cyclophosphamide - SIOPEL 3
(TECC) followed by autologous hematopoietic stem cell
support in patients with high risk soft tissue sarcoma - SIOPEL 4
(STS). Klin Pediatr 2007;219:318-322
- AIEOP T.Wilms
6
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
-Rabdomiosarcoma localizzato e
avanzato
-Gliomi a basso ed alto grado
-Retinoblastoma localmente avanzato e
metastatico
-Epatoblastoma a prognosi sfavorevole
-Medulloblastoma localizzato in
alternativa a cisplatino
-Sarcomi dei tessuti molli
-Osteosarcoma
-Tumori a cellule germinali
-Sarcoma di Ewing ad alto rischio
BIBLIOGRAFIA
Osteosarcoma
Protocolli
nazionali e
internazionali in
uso
2003
del Prever AB et al. Long-term survival in high-grade - NB- AR-01
axial osteosarcoma with bone and lung metastases treated
with chemotherapy only. J Pediatr Hematol Oncol - SIOP-AIEOP NB
Infant
2005;27:42-45
- SIOP EUROPE
UnresectNB 00
Daw NC et al. Metastatic osteosarcoma. Cancer
- AIEOP/ISG
2006;106:403-412
OSTEO REC VHR
- AIEOP/ISG 02
Lo Curto M et al. Malignant germ cell tumors in (EW -REC)
childhood: results of the first Italian cooperative study
- AIEOP RB 05
"TCG 91". Med Pediatr Oncol 2003;41:417-425
- AIEOP SNC
Sarcoma di Ewing ad alto rischio
95-01 <3a
Burdach S et al. Allogeneic and autologous stem-cell
transplantation in advanced Ewing tumors. An update AIEOP /SICP
after long-term follow-up from two centers of the TREP 2005
European Intergroup study EICESS. Stem-Cell Transplant - AIEOP TCG 2004
Programs at Düsseldorf University Medical Center,
Germany and St. Anna Kinderspital, Vienna, Austria. Ann - HIT-SIOP
PNET 4
Oncol 2000;11:1451-1462
Van Winkle P et al. Ifosfamide, carboplatin and etoposide - SIOP LGG 2004
(ICE) reinduction chemotherapy in a large cohort of
children and adolescents with recurrent/refractory
sarcoma: the Children's Cancer Group (CCG) experience.
Pediatr Blood Cancer 2005;44:338-347
Milano GM et al. High histologic and overall response to
dose intensification of ifosfamide, carboplatin and
etoposide with cyclophosphamide, doxorubicin and
vincristine in patients with high-risk Ewing sarcoma
family tumors: the Bambino Gesù Children's Hospital
experience. Cancer 2006;106:1838-1845
CARMUSTINA
Nessuna
-Gliomi ad alto grado di malignità
Gliomi ad alto grado di malignità
7
- AIEOP SNC 99
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
Chastagner P et al. Outcome of children treated with - HIT – SIOP
preradiation chemotherapy for a high-grade glioma: PNET 4
results of a French Society of Pediatric Oncology (SFOP)
Pilot Study. Pediatr Blood Cancer 2007;49:803-807
CICLOFOSFAMIDE
Nessuna
-Neuroblastoma
Neuroblastoma
-Tumori della famiglia del sarcoma di
Ewing
Donfrancesco A et al. Prolonged response to oral gefitinib,
cyclophosphamide and topotecan in heavily pretreated - AIDA-Prot.
relapsed stage 4 neuroblastoma: a case report. J Pediatr (lap0493)
GIMEMA AIEOP
-Gliomi a basso grado di malignità
-Leucemia linfoblastica acuta
-Linfoma di Hodgkin
-Linfoma non-Hodgkin
-Medulloblastoma
-Osteosarcoma
-Epatoblastoma
-Condizionamento pre-trapianto
-Rabdomiosarcoma
- AIEOP LAM
2002/01-02
Simon T et al. Topotecan, cyclophosphamide and - AIEOP EsPhALL
etoposide (TCE) in the treatment of high-risk
neuroblastoma. Results of a phase-II trial. J Cancer Res - AIEOP LH 2004
Clin Oncol 2007;133:653-661
- EURO LB-02
- AIEOP ALCL 99
Miser JS et al. Treatment of metastatic Ewing sarcoma/ - AIEOP/ISG
primitive neuroectodermal tumor of bone: evaluation of OSTEO REC VHR
increasing the dose intensity of chemotherapy-a report
from the Children's Oncology Group. Pediatric Blood - ISG/SSG III
ISG/SSG IV
Cancer 2007;49:894-900
Bernstein ML et al. Intensive therapy with growth factor - AIEOP/ISG 02
support for patients with Ewing tumor metastatic at (EW -REC)
diagnosis: Pediatric Oncology Group/Children's Cancer - ISG-AIEOP/ VHRGroup Phase II Study 9457--a report from the Children's EW-2
Oncology Group. J Clin Oncol 2006;24:152-159
- EpSSG RMS &
Gliomi a basso grado di malignità
NRSTS 2005
Lee MJ et al. Effectiveness of novel combination - SIOPEL 3
chemotherapy, consisting of 5-fluorouracil, vincristine,
cyclophosphamide and etoposide, in the treatment of low- - AIEOP
2° Ependi 03
grade gliomas in children. J Neurooncol 2006;80:277-284
Leucemia linfoblastica acuta
- SIOP LGG 2004
Shah AJ et al. Busulfan and cyclophosphamide as a - NB-AR-01
8
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
conditioning regimen for pediatric acute lymphoblastic - SIOP-AIEOP NB
leukemia
patients
undergoing
bone
marrow infant
transplantation. J Pediatr Hematol Oncol 2004;26:91-97
-AIEOP
Interinfant 99
Aricò M et al. Long-term results of the AIEOP-ALL-95 - INTERINFAN 06
Trial for Childhood Acute Lymphoblastic Leukemia:
insight on the prognostic value of DNA index in the - HLH 2004
framework
of
Berlin-Frankfurt-Muenster
based - AIEOP LLA
chemotherapy. J Clin Oncol 2008;26:283-289
REC 2003
Linfoma di Hodgkin
- AIEOP LAM
Krasin MJ et al. Patterns of treatment failure in pediatric REC/01
and young adult patients with Hodgkin's disease: local
disease control with combined-modality therapy. J Clin
Oncol 2005;23:8406-8413
Linfoma non-Hodgkin
Patte C et al. Results of the randomized international
FAB/LMB96 trial for intermediate risk B-cell nonHodgkin lymphoma in children and adolescents: it is
possible to reduce treatment for the early responding
patients. Blood 2007;109:2773-2780
Medulloblastoma
Dhall G et al. Outcome of children less than three years
old at diagnosis with non-metastatic medulloblastoma
treated with chemotherapy on the "Head Start" I and II
protocols. Pediatr Blood Cancer 2008;50:1169-1175
Osteosarcoma
Tunn PU and Reichardt P. Chemotherapy for
osteosarcoma without high-dose methotrexate: a 12-year
follow-up on 53 patients. Onkologie 2007;30:228-232
Epatoblastoma
Cacciavillano WD et al. Phase II study of high-dose
cyclophosphamide in relapsing and/or resistant
9
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
hepatoblastoma in children: a study from the SIOPEL
group. Eur J Cancer 2004;40:2274-2279
Condizionamento pre-trapianto
Shah AJ et al. Busulfan and cyclophosphamide as a
conditioning regimen for pediatric acute lymphoblastic
leukemia
patients
undergoing
bone
marrow
transplantation. J Pediatr Hematol Oncol 2004;26:91-97
Rabdomiosarcoma
Meza JL et al. Analysis of prognostic factors in patients
with nonmetastatic rhabdomyosarcoma treated on
intergroup rhabdomyosarcoma studies III and IV: the
Children's Oncology Group. J Clin Oncol 2006;20:38443851
CISPLATINO
Nessuna
-Neuroblastoma
Tumori desmoplastici a piccole cellule rotonde
-Epatoblastoma
Hayes-Jordan A et al. Continuous hyperthermic peritoneal - AIEOP/ISG/SSG
perfusion for desmoplastic small round cell tumor. J 02 (EW REC)
Pediatr Surg 2007;42:29-32
- ISG -AIEOP/VHRCarcinoma del rene a cellule chiare
EW-2
-Medulloblastoma
-Osteosarcoma
-Tumori germinali
-Linfomi Hodgkin e non-Hodgkin
recidivanti
- Tumori desmoplastici a piccole
cellule rotonde
- ISG/OSI
Varan A et al. Renal cell carcinoma in children: - AIEOP/SICP
experience of a single center. Nephron Clin Pract TREP 2005
2007;105:58-61
- SIOPEL 3
Carcinoma corticosurrenalico
- SIOPEL 4
Zancanella P et al. Mitotane associated with cisplatin,
etoposide and doxorubicin in advanced childhood - AIEOP TCG 2004
adrenocortical carcinoma: mitotane monitoring and tumor - SIOP LGG 2004
regression. J Pediatr Hematol Oncol 2006;28:513-524
- HIT-SIOP PNET 4
Retinoblastoma
- AIEOP
de Aguirre Neto JC et al. Retinoblastoma in children older SNC 95-01 <3a
than 5 years of age. Pediatr Blood Cancer 2007;48:292- NB-AR-01
295
10
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
-Carcinoma corticosurrenalico
Tumore del seno endodermico della vagina
-Retinoblastoma
-Tumore del seno endodermico della
vagina
-Carcinoma nasofaringeo
-Tumori
ovarici
dello
gonadico (sex cord-stromal)
stroma
Protocolli
nazionali e
internazionali in
uso
- SIOP-AIEOP NB
Infant
Terenziani M et al. Endodermal sinus tumor of the vagina.
Küpeli S et al. Treatment results of 84 patients with
nasopharyngeal carcinoma in childhood. Pediatr Blood
Cancer 2006;46:454-458
Tumori ovarici dello stroma gonadico (sex cordstromal)
Schneider DT et al. Ovarian sex cord-stromal tumors in
children and adolescents. J Reprod Med 2005;50:439-446
CITARABINA
-Leucemia mieloide acuta -Leucemia linfoblastica acuta
e cronica
Silverman LB. Acute lymphoblastic leukemia in infancy.
Linfoma non-Hodgkin
Patte C et al. The Société Française d'Oncologie
Pédiatrique LMB89 protocol: highly effective multiagent
chemotherapy tailored to the tumor burden and initial
response in 561 unselected children with B-cell
lymphomas and L3 leukemia. Blood 2001;97:3370-3379
- AIEOP LAM
2002/01-02
- AIDAProt.(lap0493)
GIMEMA AIEOP
- AIEOP LLA REC
Ph+ 2002
- AIEOP LLA R2006
- AIEOP LAM REC/
01
- AIEOP LNH 97
- ICC APL 01
- AIEOP ALCL 99
- EURO LB-02
- LCH-S-2005
- AIEOP
Interinfant 99
- INTERFAN T06
11
NOME COMPOSTO
CITARABINA
LIPOSOMIALE
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
Nessuna
BIBLIOGRAFIA
-Tumori del SNC
Tumori del SNC
-Leucemia mieloide acuta
Lassaletta A et al. Successful treatment of intracranial
ependymoma with leptomeningeal spread with systemic
chemotherapy and intrathecal liposomal cytarabine in a
two-year-old child. J Neurooncol 2007;83:303-306
Protocolli
nazionali e
internazionali in
uso
- AIEOP EsPhALL
Leucemia mieloide acuta e Leucemia linfoblastica
acuta
Benesch M and Urban C. Liposomal cytarabine for
leukemic and lymphomatous meningitis: recent
developments. Expert Opin Pharmacother 2008;9:301-309
Benesch M et al. Feasibility and toxicity of intrathecal
liposomal cytarabine in 5 children and young adults with
refractory neoplastic meningitis. J Pediatr Hematol Oncol
2007;29:222-226
CLADRIBINA
Nessuna
-Istiocitosi a cellule di Langerhans
Istiocitosi a cellule di Langerhans
- LCH -S-2005
Mottl H et al. Treatment of recurrent Langerhans cell
histiocytosis in children with 2-chlorodeoxyadenosine.
Leuk Lymphoma 2006;47:1881-1884
CLORAMBUCILE
Nessuna
-Linfoma di Hodgkin
Linfoma di Hodgkin
Hall GW et al. Outcome of children with nodular
lymphocyte predominant Hodgkin lymphoma - a
Children's Cancer and Leukaemia Group report-. Br J
Haematol 2007;138:761-768
Capra M et al. Long-term outcome in children with
Hodgkin's lymphoma: the United Kingdom Children's
Cancer Study Group HD82 trial. Eur J Cancer
2007;43:1171-1179
DACARBAZINA
Nessuna
-Linfoma di Hodgkin
Linfoma di Hodgkin
Engert A et al. Two cycles of doxorubicin, bleomycin,
12
- AIEOP LH 2004
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
vinblastine and dacarbazine plus extended-field
radiotherapy is superior to radiotherapy alone in early
favorable Hodgkin's lymphoma: final results of the GHSG
HD7 trial. J Clin Oncol 2007;25:3495-3502
Hsu SC et al. Comparison of treatment outcomes of
childhood Hodgkin lymphoma in two US centers and a
center in Recife, Brazil. Pediatr Blood Cancer
2007;49:139-144
Kung FH et al. POG 8625: a randomized trial comparing
chemotherapy with chemoradiotherapy for children and
adolescents with stages I, IIA, IIIA1 Hodgkin disease: a
report from the Children's Oncology Group. J Pediatr
Pratt CB et al. Treatment of unresectable or metastatic
pediatric soft tissue sarcomas with surgery, irradiation and
chemotherapy: a Pediatric Oncology Group Study. Med
Pediatr Oncol 1998;30:201-209
DAUNORUBICINA
Nessuna
-Leucemie e linfomi del bambino
recidivanti
Linfoma non-Hodgkin
- AIEOP EsPhALL
Crosswell HE et al. Successful treatment with modified - AIEOP
CHOP-rituximab in pediatric AIDS-related advanced Interinfant 99
stage Burkitt lymphoma. Pediatr Blood Cancer
- INTERINFAN 06
2008;50:883-885
- EURO LB-02
Escherich G et al. Daunomicin–induced cell kill with 1- - AIEOP LNH 97
hour versus 24–hours infusion: a randomized comparison
in children with newly diagnosed acute lymphoblastic
leukaemia. Klin Padiatr 2007;219:134-138
Leucemia mieloide acuta
Creutzig U et al. Significance of age in acute myeloid
leukemia patients younger than 30 years: a common
13
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
analysis of the pediatric trials AML-BFM 93/98 and the
adult trials AMLCG 92/99 and AMLSG HD93/98A.
Cancer 2008;112:562-571
Lange BJ et al. Outcomes in CCG-2961, a children's
oncology group phase 3 trial for untreated pediatric acute
myeloid leukemia: a report from the Children's Oncology
Group. Blood 2008;111:1044-1053
DOXORUBICINA
-Osteosarcoma
-Sarcoma di Ewing in prima linea o
recidivante
-Rabdomiosarcoma in prima linea o
recidivante
-Neuroblastoma
-Nefroblastoma
-Leucemia linfoblastica
acuta
-Epatoblastoma
recidivante
in
prima
linea
o
- AIEOP Es Ph ALL
Castex MP et al. Extraosseous localized Ewing tumors: - AIEOP ALCL 99
improved outcome with anthracyclines-the French society
of pediatric oncology and international society of pediatric - EURO LB-02
oncology. J Clin Oncol 2007;25:1176-1182
- AIEOP LNH 97
Tumori del SNC
- AIEOP LH 2004
- Emangiopericitoma
Fiorillo A et al. Shunt-related abdominal metastases in an - ISG/OSI
infant with medulloblastoma: long-term remission by
systemic chemotherapy and surgery. J Neurooncol - ISG/SSG III
2001;52:273-276
- ISG/SSG IV
Emangiopericitoma
- AIEOP
Gowans LK et al. Successful treatment of an infant with T.Wilms 2003
constitutional
chromosomal
abnormality
and - SIOPEL 3
hemangiopericytoma with chemotherapy alone. J Pediatr
- SIOPEL 4
- Carcinoma surrenalico
Carcinoma surrenalico
- Tumori rabdoidi
etoposide and doxorubicin in advanced childhood - ISG-AIEOP/VHR
adrenocortical carcinoma: mitotane monitoring and tumor EW2
- AIEOP RB 05
Ewing
- Tumori del SNC
- Retinoblastoma
- Istiocitosi a cellule di Langerhans
Tumori rabdoidi
- SIOP-AIEOP NB
infant
- AIEOP/SICP
TREP 2005
Hosoi H et al. Continuous remission in an infant with - EpSSG RMS &
chest wall malignant rhabdoid tumor after relapse. J NRSTS 2005
14
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Pediatr Surg 2007;42:9-12
Retinoblastoma
Protocolli
nazionali e
internazionali in
uso
- AIEOP
Interinfant 99
- AIEOP/ISG 02
Mustafa MM et al. Adjuvant chemotherapy with (EW- REC)
vincristine, doxorubicin and cyclophosphamide in the
treatment of postenucleation high risk retinoblastoma. J - AIEOP NB
Pediatr Hematol Oncol 1999;21:364-369
rec/ res 00
Istiocitosi a cellule di Langerhans
Morimoto A et al. Improved outcome in the treatment of
pediatric multifocal Langerhans cell histiocytosis: Results
from the Japan Langerhans Cell Histiocytosis Study
Group-96 protocol study. Cancer 2006;107:613-619
DOXORUBICINA
LIPOSOMIALE
Nessuna
-Tumori del SNC ad alto grado di
malignità
-Leucemia linfatica acuta in recidiva
- SIOP/EUROPE
UnresectNB 00
AIEOP LLA Rec
Wagner S et al. Pegylate liposomial doxorubicin and oral 2003
topotecan in eight children with relapsed high-grade
malignant brain tumors. J Neurooncol 2008;86:175-181
Tumori del SNC ad alto grado di malignità
Leucemia linfatica acuta in recidiva
Hempel et al. Population pharmacokinetics of liposomal
daunorubicin in children.
Br J Clin Pharmacol
2003;56:370-377
Lowe E et al. Phase I trial and pharmacokinetic study of
liposomal doxorubicin in children with refractory solid
tumors. Proc Ann Meet ASCO 2002;21:1099
EPIRUBICINA
Nessuna
Stohr W et al. Comparison of epirubicin and doxorubicin
cardiotoxicity in children and adolescents treated within
the German Cooperative Soft Tissue Sarcoma Study. J
Cancer Res Clin Oncol 2006;132:35-40
-Epatoblastoma ed epatocarcinoma
-Tumori mesenchimali (nel primo
anno di vita)
Linfoma non-Hodgkin
Pocali B et al. Ifosfamide, epirubicin and etoposide (IEV)
regimen as salvage and mobilization therapy for refractory
or early relapsing patients with aggressive non-Hodgkin's
15
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
lymphoma. Leuk Lymphoma 2004;45:1605-1609
Bhutani M et al. Randomized study comparing 4'-epidoxorubicin (epirubicin) versus doxorubicin as a part of
induction treatment in adult acute lymphoblastic leukemia.
Am J Hematol 2002;71:241-247
Eksborg S et al. A comparative pharmacokinetic study of
doxorubicin and 4'-epi-doxorubicin in children with acute
lymphocytic leukemia using a limited sampling procedure.
Anticancer Drugs 2000;11:129-136
Epatoblastoma ed epatocarcinoma
Casanova M et al. Etoposide, cisplatin, epirubicin
chemotherapy in the treatment of pediatric liver tumors.
Tumori mesenchimali (nel primo anno di vita)
Orbach D et al. Soft tissue sarcoma or malignant
mesenchymal tumors in the first year of life: experience of
the International Society of Pediatric Oncology (SIOP)
2005;23:4363-4371
ETOPOSIDE
Nessuna
-Tumore di Wilms a
sfavorevole e metastatico
prognosi
-Neuroblastoma di grado 3 e 4
-Sarcomi dei tessuti molli alla ricaduta
-Famiglia dei sarcomi di Ewing
-Rabdomiosarcoma recidivante
-Osteosarcoma
- AIEOP LLA REC
Ph+ 2002
Gassas A et al. Comparative outcome of hematopoietic
stem cell transplant for pediatric acute lymphoblastic - HLH 2004
leukaemia following cyclophosphamide and total body
irradiation or VP16 and total body irradiation conditioning - AIEOP
Interinfant 99
regimens. Bone Marrow Transplant 2006;38:739-743
- INTERINFAN 06
Styczynski J et al. Fludarabine, treosulfan and etoposide - AIEOP ALCL 99
sensitivity and the outcome of hematopoietic stem cell - AIEOP LNH 97
transplantation in childhood acute myeloid leukemia.
16
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
Anticancer Res 2007;27:1547-1551
-Ependimomi
Ayas M et al. The outcome of children with acute myeloid - AIEOP LAM
leukemia (AML) post-allogeneic stem cell transplantation 2002/01-02
(SCT) is not improved by the addition of etoposide to the
conditioning regimen. Pediatr Blood Cancer 2006;47:926- - ISG/SSG III
930
- ISG/SSG IV
-Tumori neuroectodermici
cerebrali
primitivi
-Retinoblastoma localmente avanzato o
metastatico
- AIEOP LH 2004
Epatoblastoma
- AIEOP
Casanova M et al. Etoposide, cisplatin, epirubicin T Wilms 2003
chemotherapy in the treatment of pediatric liver tumors. - AIEOP SNC 99
- AIEOP RB 05
Häberle B et al. Differentiated treatment protocols for
high- and standard-risk hepatoblastoma- an interim report - AIDA –
of the German Liver Tumor Study HB99-. Klin Padiatr Prot.(lap0493
GIMEMA AIEOP)
2003;215:159-165
-Epatoblastoma
-Tumori a cellule germinali
-Carcinoma surrenalico
-Linfoma di Hodgkin
- SIOP LGG 2004
Shinkoda Y et al. Successful treatment of an endodermal - AIEOP/ISG 02
sinus tumor of the vagina by chemotherapy alone: a rare (EW-REC)
case of an infant diagnosed by pathological examination - SIOP-AIEOP NB
of discharged tumor fragment. Pediatr Hematol Oncol infant
2006;23:563-569
- AIEOP SNC
Cushing B et al. Randomized comparison of combination 95-01<3a
chemotherapy with etoposide, bleomycin and either highdose or standard-dose cisplatin in children and adolescents - AIEOP TCG 2004
with high-risk malignant germ cell tumors: a pediatric
- AIEOP
intergroup study -Pediatric Oncology Group 9049 and
2° Ependi 03
Children's Cancer Group 8882-. J Clin Oncol
2004;22:2691-2700
17
etoposide and doxorubicin in advanced childhood
adrenocortical carcinoma: mitotane monitoring and tumor
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
Linfoma di Hodgkin
Landman-Parker J et al. Localized childhood Hodgkin's
disease: response-adapted chemotherapy with etoposide,
bleomycin, vinblastine and prednisone before low-dose
radiation therapy-results of the French Society of Pediatric
Oncology Study MDH90. J Clin Oncol 2000;18:15001507
Linfoma non-Hodgkin
Patte C et al. The Société Française d'Oncologie
Pédiatrique LMB89 protocol: highly effective multiagent
chemotherapy tailored to the tumor burden and initial
response in 561 unselected children with B-cell
lymphomas and L3 leukemia. Blood 2001;97:3370-3379
FLUDARABINA
Nessuna
-Leucemia mieloide acuta in recidiva
pre-trapianto
-Leucemia
recidiva
linfoblastica
acuta
-Anemia di Fanconi
in
Leucemia mieloide acuta in recidiva pre-trapianto
- AIEOP/SICP
TREP 2005
Styczynski J et al. Fludarabine, treosulfan and etoposide
sensitivity and the outcome of hematopoietic stem cell - ISG/OSI
transplantation in childhood acute myeloid leukemia.
- AIEOP/ISG
Anticancer Res 2007;27:1547-1551
OSTEO REC VHR
Leucemia linfoblastica acuta in recidiva
- ISG-AIEOP/VHRGutierrez-Aguirre CH et al. Non-myeloablative stem cell EW-2
transplantation in patients with relapsed acute
lymphoblastic leukemia: results of a multicenter study. - NB-AR-01
Bone Marrow Transplant 2007;40:535-539
- AIEOP LAM
REC/01
Anemia di Fanconi
Wagner JE et al. Unrelated donor bone marrow - EICNHL
transplantation for the treatment of Fanconi anemia. Blood (ALCL-relapse)
2007;109:2256-2262
- SIOP EUROPE
Unresect NB 00
18
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
Kornguth DG et al. Fludarabine allows dose reduction for - AIEOP LLA Rec
total body irradiation in pediatric hematopoietic stem cell 2003
transplantation. Int J Radiat Oncol Biol Phys
2007;68:1140-1144
Sauer M et al. Substitution of cyclophosfamide and
preparation regimen for children and adolescents with
Shwachman-Diamond
syndrome.
Bone
Marrow
Transplant 2007;39:143-147
Sauer M et al. Complete substitution of cyclophosphamide
by fludarabine and ATG in a busulfan based preparative
regimen for children and adolescents with betaThalassemia. Bone Marrow Transplant 2005;36:383-387
FLUOROURACILE
Nessuna
-Tumori maligni del fegato
Haimi M et al. Nasopharyngeal carcinoma in the pediatric
age group: the northern Israel (Rambam) medical center
experience, 1989-2004. J Pediatr Hematol Oncol
2005;27:510-516
- AIEOP/SICP
TREP 2005
Rodriguez-Galindo C et al. Preradiation chemotherapy
with methotrexate, cisplatin, 5-fluorouracil and leucovorin
for pediatric nasopharingeal carcinoma. Cancer
2005;103:850-857
Tumori maligni del fegato
Malogolowkin MH et al. Intensified platinum therapy is
an ineffective strategy for improving outcome in pediatric
patients with advanced hepatoblastoma. J Clin Oncol
2006;24:2879-2884
Katzenstein HM et al. Fibrolamellar hepatocellular
carcinoma in children and adolescents. Cancer
2003;97:2006-2012
Varan A et al. Renal cell carcinoma in children:
19
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
Protocolli
nazionali e
internazionali in
uso
BIBLIOGRAFIA
2007;105:58-61
GEMCITABINA
Nessuna
-Tumori pediatrici refrattari
Tumori pediatrici refrattari
-Linfoma di Hodgkin
Fulfaro F et al. Antiblastic drug combinations with
ifosfamide: an update. Oncology 2003;65:21-30
-Carcinoma midollare del rene
Linfoma di Hodgkin
- Ep SSG RMS &
NRSTS 2005
Ozkaynak MF and Jayabose S. Gemcitabine and
vinorelbine as a salvage regimen for relapse in Hodgkin
lymphoma after autologous hematopoietic stem cell
transplantation. Pediatr Hematol Oncol 2004;21:107-113
Carcinoma midollare del rene
Strouse JJ et al. Significant responses to platinum-based
chemotherapy in renal medullary carcinoma. Pediatr
Blood Cancer 2005;44:407-411
Bell MD. Response to paclitaxel, gemcitabine and
cisplatin in renal medullary carcinoma. Pediatr Blood
Cancer 2006;47:228
GEMTUZUMAB
Nessuna
refrattaria o recidivante
Leucemia acuta
recidivante
promielocitica
refrattaria
o - ICC APL 01
Aplenc R et al. Safety and efficacy of gemtuzumab
ozogamicin in combination with chemotherapy for
pediatric acute myeloid leukemia: a report from the
Children's Oncology Group. J Clin Oncol 2008;26:23903295
Aribi A et al. Combination therapy with arsenic trioxide,
all-trans retinoic acid and gemtuzumab ozogamicin in
recurrent acute promyelocytic leukemia. Cancer
2007;109:1355-1359
Corey SI. New agents in the treatment of childhood
leukemias and myelodysplastic syndromes. Curr Oncol
20
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
Rep 2005;7:399-405
IDARUBICINA
-Leucemia linfocitica
acuta (II linea)
-Leucemia promielocitica acuta
Leucemia promielocitica acuta
Testi AM et al. GIMEMA-AIEOPAIDA protocol for the
treatment of newly diagnosed acute promyelocitic - AIDA-Prot.
leukaemia (APL) in children. Blood 2005;106:447-453
(lap0493)
GIMEMA AIEOP
- ICC APL 01
Liang DCI et al. Improved treatment results for childhood
acute myeloid leukemia in Taiwan. Leukemia - AIEOP ALCL 99
2006;20:136-141
-AIEOP LLA Rec
Lange BJ et al. Outcomes in CCG-2961, a children's 2003
oncology group phase 3 trial for untreated pediatric acute
myeloid leukemia: a report from the children's oncology
group. Blood 2008;111:1044-1053
-Tumori del SNC
- AIEOP
LAM2002/01-02
Tumori SNC
Dreyer ZE et al. Phase 2 study of idarubicin in pediatric
brain tumors: Pediatric Oncology Group study POG 9237.
Neuro Oncol 2003;5:261-267
IDROSSIUREA
-Leucemia mieloide
cronica
-Drepanocitosi
Drepanocitosi
-Talassemia intermedia
Zimmerman SA et al. Hydroxyurea therapy lowers
transcranial Doppler flow velocities in children with sickle
cell anemia. Blood 2007;110:1043-1047
De Baun MR et al. Hydroxyurea as secondary prevention for
stroke in children with sickle cell anemia. J Pediatr
2005;147:560-561
Ware RE et al. Prevention of secondary stroke and resolution
of transfusional iron overload in children with sickle cell
anemia using hydroxyurea and phlebotomy. J Pediatr
2004;145:346-352
Talassemia intermedia
Dixit A et al. Hydroxyurea in thalassemia intermedia – A
promising therapy. Ann Hematol 2005;84:441-446
21
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
Koren A et al. Response to hydroxyurea therapy in betathalassemia. Am J Hematol 2008;83:366-370
IFOSFAMIDE
Nessuna
-Tumore di Wilms a prognosi
sfavorevole o metastatico
-Epatoblastoma a prognosi sfavorevole
o metastatico
-Sarcoma di Ewing
-Osteosarcoma
-Rabdomiosarcoma
-Neuroblastoma
-Tumori del SNC
-Linfoma di Hodgkin
-Retinoblastoma
- AIEOP EsPhALL
Ferrari A et al. Adult-type soft tissue sarcomas in pediatric - AIEOP ALCL 99
age patients: experience at the Istituto Nazionale Tumori
- AIEOP LNH 97
in Milan. J Clin Oncol 2005;23:4021-4030
- AIEOP LH 2004
Tumori del SNC
Burzynski SR. Treatments for astrocytic tumors in - ISG/OSI
children: current and emerging strategies. Paediatr Drugs - ISG/SSG III
2006;8:167-168
Tekautz TM et al. Atypical teratoid/rhabdoid tumors
(ATRT): improved survival in children 3 years age and
older with radiation therapy and high-dose alchilator - ISG/SSG IV
based chemotherapy J Clin Oncol 2005;23:1491-1499
- AIEOP
Linfoma di Hodgkin
T. Wilms 2003
Schellong G et al. Salvage therapy of progressive and
- AIEOP RB 05
recurrent Hodgkin's disease: results from a multicenter study
of the pediatric DAL/GPOH-HD study group. J Clin Oncol - AIEOP/ISG 02
2005;23:6181-6189
(EW-REC)
Linfoma non-Hodgkin
- ISG AIEOP/VHR-
Spreafico F et al. Intensive, very short-term chemotherapy for EW-2
advanced Burkitt's lymphoma in children. J Clin Oncol - Ep SSG RMS &
2002;20:2783-2788
NRSTS 2005
-Blastoma pleuropolmonare
Voûte PA et al. Ifosfamide in the treatment of pediatric
- AIEOP TCG 2004
malignancies. Semin Oncol 1996;23:8-11
Seidemann K et al. Short-pulse B-non-Hodgkin lymphomatype chemotherapy is efficacious treatment for pediatric
anaplastic large cell lymphoma: a report of the BerlinFrankfurt-Münster Group Trial NHL-BFM 90. Blood
2001;97:3699-3706
Retinoblastoma
22
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
De Aguirre Neto JC et al. Retinoblastoma in children older
than 5 years of age. Pediatr Blood Cancer 2007;48:292-295
Imataki O et al. Intensive multimodality therapy including
paclitaxel and reduced-intensity allogeneic hematopoietic
stem cell transplantation in the treatment of adrenal cancer
with multiple metastases. Int J Clin Oncol 2006;11:156-158
Blastoma pleuropolmonare
Büyükavci M et al. An aggressive childhood tumor
mimicking pleural empyema: pleuropulmonary blastoma. J
IMATINIB
-Leucemia mieloide
-Leucemia linfoblastica acuta Ph+
cronica Ph+ alla diagnosi o
in crisi blastica
-Tumori stromali gastrointestinali
(GIST)
Leucemia linfoblastica acuta Ph+
- AIEOP EsPhALL
Stubbs MC and Armstrong SA. FLT3 as a therapeutic - AIEOP LLA
target in childhood acute leukaemia. Curr Drug Targets REC 2003
2007;8:703-714
Fuster JL et al. Imatinib mesylate in combination with
chemotherapy in four children with de novo and advanced
stage
Philadelphia
chromosome-positive
acute
lymphoblastic leukemia. Haematologica 2007;92:17231724
Goemans BF et al. Mutations in KIT and RAS are
frequent events in pediatric core-binding factor acute
myeloid leukemia. Leukemia 2005;19:1536-1542
Corey SI. New agents in the treatment of childhood
leukemias and myelodysplastic syndromes. Curr Oncol
Rep 2005;7:399-405
Skolnik JM. Tyrosine kinase inhibitors in pediatric
malignancies. Cancer Invest 2007;18:1-7
23
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
Sindromi mielodisplastiche
Bierings IM et al. Stem cell transplantation in pediatric
leukemia and mielodisplasia: state of art and current
challeges. Curr Stem Cell Res Ther 2007;2:53-63
Tumori stromali gastrointestinali (GIST)
Sauseng W et al. Clinical, radiological and pathological
findings in four children with gastrointestinal stromal
tumors of the stomach. Pediatr Hematol Oncol
2007;24:209-219
Skolnik JM and Adamson PC. Tyrosine kinase inhibitors
in pediatric malignancies. Cancer Invest 2007;18:1-7
INTERFERONE ALFA- Nessuna
2B
-Anemia diseritropoietica
-Leucemia mieloide cronica
-Neuroblastoma
-Nefroblastoma
- Ep SSG RMS &
Varan A et al. Renal cell carcinoma in children: NRSTS 2005
2007;105:58-61
Anemia diseritropoietica
Marwaha RK et al. Interferon therapy in congenital
dyserythropoietic anemia type I/II. Pediatr Hematol Oncol
2005;22:133-138
Yarali N et al. Successful management of congenital
dyserythropoietic anemia type I with interferon alpha in a
child. Pediatr Hematol Oncol 2005;22:265-270
Leucemia mieloide cronica
Millot F et al. Results of a phase II trial testing interferonalfa 2B and cytarabine in children and adolescents with
chronic myelogenous leukaemia. Pediatric Blood Cancer
2006;47:555-559
Neuroblastoma e Nefroblastoma
Adamson PC et al. A phase 2 trial of all-trans-retinoic acid
in combination with interferon-alpha 2a in children with
24
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
recurrent neuroblastoma or Wilms tumor: a pediatric
oncology branch, NCI and children's oncology group
study. Pediatr Blood Cancer 2007;49:661-665
INTERLEUCHINA-2
Nessuna
-Neuroblastoma
-Sarcomi ossei
- AIEOP/SICP
Lange BJ et al. Outcomes in CCG-2961, a Children's TREP 2005
Oncology Group Phase 3 Trial for untreated pediatric - AIEOP/ISG
acute myeloid leukemia: a report from the Children's OSTEO REC VHR
Oncology Group. Blood 2008;111:1044-1053
-Nefroblastoma
Neuroblastoma
Osenga KL et al. A phase I clinical trial of the hu14.18IL2 (EMD 273063) as a treatment for children with
refractory or recurrent neuroblastoma and melanoma: a
study of the Children's Oncology Group. Clin Cancer Res
2006;12:1750-1759
Sarcomi ossei
-Melanoma
Luksch R et al. Immunomodulation in a treatment
program including pre- and post-operative interleukin-2
and chemotherapy for childhood osteosarcoma. Tumori
2003;89:263-268
Nefroblastoma
Schwinger W et al. Feasibility of high-dose interleukin-2
in heavily pretreated pediatric cancer patients. Ann Oncol
2005;16:1199-1206
Selle B et al. Population-based study of renal cell
carcinoma in children in Germany, 1980-2005: more
frequently localized tumors and underlying disorders
compared with adult counterparts. Cancer 2006;107:29062914
25
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
Melanoma
Osenga KL et al. A phase I clinical trial of the hu14.18IL2 (EMD 273063) as a treatment for children with
refractory or recurrent neutroblastoma and melanoma: a
study of the Children’ Oncology Group. Clin Cancer Res
2006;12:1750-1759
Schwinger W et al. Feasibility of high-dose interleukin 2
in heavily pre-treated pediatric cancer patients. Ann Oncol
2005;16:1199-1206
IRINOTECANO
Nessuna
-Rabdomiosarcoma recidivante
-Sarcomi della famiglia di Ewing
recidivanti
Bisogno G et al. Phase II study of a protracted irinotecan
schedule in children with refractory or recurrent soft tissue
sarcoma. Cancer 2006;106:703-707
Gupta AA and Pappo AS. New drugs for the treatment of
metastatic or refractory soft tissue sarcomas in children.
Future Oncol 2006;2:675-685
-Tumori del SNC
Tumori del SNC
-Tumori solidi recidivanti o refrattari
Mora J et al. Successful treatment of childhood
intramedullary spinal cord astrocytomas with irinotecan
and cisplatin. Neuro Oncol 2007;9:39-46
Tumori solidi recidivanti o refrattari
Vassal G et al. Phase II trial of irinotecan in children with
relapsed or refractory rhabdomyosarcoma: a joint study of
the French Society of Pediatric Oncology and the United
Kingdom Children's Cancer Study Group. J Clin Oncol
26
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
2007;25:356-361
Bomgaars L et al. A phase I study of irinotecan
administered on a weekly schedule in pediatric patients.
MELFALAN
-Neuroblastoma (stadi
avanzati)
-Sarcomi della famiglia di Ewing a
cattiva prognosi in associazione a
busulfano nei regimi mieloablativi
-Tumori del SNC
-Anemia aplastica
-Linfoistiocitosi emofagocitica
-Nefroblastoma
-Sindrome di Shwachman-Diamond
-Retinoblastoma
Tumori del SNC
- AIEOP LAM
2002/01-02
Pérez-Martínez A et al. High-dose chemotherapy with
autologous stem cell rescue as first line of treatment in - AIEOP LAM
young children with medulloblastoma and supratentorial REC/01
primitive neuroectodermal tumors. J Neurooncol
- AIEOP LLA
2004;67:101-106
REC 2003
- INTERINFAN 06
Lang P et al. Haploidentical stem cell transplantation in
patients with pediatric solid tumors: preliminary results of - NB-AR-01
a pilot study and analysis of graft versus tumor effects.
Klin Padiatr 2006;218:321-326
- SIOP- AIEOP NB
Anemia aplastica e Sindromi mielodisplastiche
Infant
Stary J et al. Stem cell transplant for aplastic anemia and - AIEOP/ISG 02
myelodysplastic syndrome. Bone Marrow Transplant (EW-REC)
2005;35:813-816
- ISG/SSG III
Linfoistiocitosi emofagocitica
ISG/SSG IV
Cesaro S et al. Successful engraftment and stable full - ISG- AIEOP/VHRdonor chimerism after myeloablation with thiotepa, EW-2
fludarabine and melphalan and CD34-selected peripheral
allogeneic stem cell transplantation in hemophagocytic - AIEOP SNC
95-01 < 3 a
lymphohistiocytosis. Am J Hematol 2003;72:143-146
Nefroblastoma
Campbell AD et al. Treatment of relapsed Wilms' tumor
with high-dose therapy and autologous hematopoietic
stem-cell rescue: the experience at Children's Memorial
Hospital. J Clin Oncol 2004;22:2885-2890
Sindrome di Shwachman-Diamond
27
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
Sauer M et al. Substitution of cyclophosphamide and
preparative regimen for children and adolescents with
Shwachman-Diamond
syndrome.
Bone
Marrow
Transplant 2007;39:143-147
Retinoblastoma
Matsubara H et al. A multidisciplinary treatment strategy
that includes high-dose chemotherapy for metastatic
retinoblastoma without CNS involvement. Bone Marrow
Transplant 2005;35:763-766
Izaki S et al. Long-term follow-up of busulfan, etoposide
and nimustine hydrochloride (ACNU) or melphalan as
conditioning regimens for childhood acute leukemia and
lymphoma. Int J Hematol 2007;86:253-260
Puig N et al. Morbidity and transplant-related mortality of
CBV and BEAM preparative regimens for patients with
lymphoid malignancies undergoing autologous stem-cell
transplantation. Leuk Lymphoma 2006;47:1488-1494
MERCAPTOPURINA
Nessuna
-Linfoma linfoblastico dell’infanzia
Chauvenet AR et al. Antimetabolite therapy for lesser-risk - AIEOP LLA2002
B-lineage acute lymphoblastic leukemia of childhood: a
report from Children's Oncology Group Study P9201. - AIEOP LLA
REC 2003
Blood 2007;110:1105-1111
Winter SS et al. Antimetabolite-based therapy in - AIDA-Prot.
childhood T-cell acute lymphoblastic leukemia: a report of (lap0493)
GIMEMA AIEOP
POG study 9296. Pediatr Blood Cancer 2006;46:179-186
- AIEOP EsPhALL
-Malattia di Rosai-Dorfman
Perel Y et al. Treatment of childhood acute myeloblastic - AIEOP
leukemia: dose intensification improves outcome and Interinfant 99
maintenance therapy is of no benefit--multicenter studies
of the French LAME (Leucémie Aiguë Myéloblastique - EURO LB-02
Enfant) Cooperative Group. Leukemia 2005;19:2082-2089 - AIEOP LNH 97
- INTERINFAN 06
28
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
- LCH-S-2005
Dvorak CC et al. Reinduction of relapsed acute - LCH III 2001
promyelocytic leukemia with ATRA and low dose
antimetabolite-based chemotherapy. Pediatr Blood Cancer
2007;48:582-585
Malattia di Rosai-Dorfman
Tasso M et al. Sinus histiocytosis with massive
lymphadenopathy (Rosai-Dorfman disease) treated with 2chlorodeoxyadenosine.
Pediatr
Blood
Cancer
2006;47:612-615
METOTRESSATO
acuta
-Gliomi ad alto grado
Osteosarcoma
-Tumori neuroectodermici primitivi
cerebrali
Longhi A et al. Primary bone osteosarcoma in the - AIEOP LLA 2002
pediatric age: state of the art. Cancer Treat Rev
- AIEOP ALCL 99
2006;32:423-436
- AIEOP LLA REC
Medulloblastoma
2003
Yoshimura J et al. Intrathecal chemotherapy for refractory
disseminated medulloblastoma. Childs Nerv Syst - INTERINFAN 06
2008;24:581-585
- AIEOP
Grill J et al. Treatment of medulloblastoma with Interinfant 99
-Sarcomi dei tessuti molli -Fibromatosi aggressiva
-Osteosarcoma
-Medulloblastoma
- AIEOP EsPhALL
postoperative chemotherapy alone: an SFOP prospective- AIEOP LNH 97
trial in young children. Lancet Oncol 2005;6:573-580
- LCH-S-2005
- LCH III 2001
Rodriguez-Galindo C et al. Preradiation chemotherapy
with methotrexate, cisplatin, 5-fluorouracil and leucovorin - AIDA-Prot.
for pediatric nasopharyngeal carcinoma. Cancer (lap0493)
GIMEMA AIEOP
2005;103:850-857
- AIEOP SNC 99
- AIEOP SNC
29
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
95-01 <3a
- EURO LB-02
- ISG/OSI
- Ep SSG RMS &
NRSTS 2005
MITOTANO
Nessuna
etoposide and doxorubicin in advanced childhood
adrenocortical carcinoma: mitotane monitoring and tumor
- AIEOP/SICP
TREP 2005
Michalkiewicz E et al. Clinical and outcome
characteristics of children with adrenocortical tumors: a
report from the International Pediatric Adrenocortical
Tumor Registry. J Clin Oncol 2004;22:838-845
MITOXANTRONE
Nessuna
Zwaan CM et al. Different drug sensitivity profiles of
acute myeloid and lymphoblastic leukaemia and normal - INTERINFAN 06
peripheral blood mononuclear cells in children with and
- AIDA-Prot.
without Down syndrome. Blood 2002;99:245-251
(lap0493)
GIMEMA AIEOP
- AIEOP LAM
2002/01-02
Creutzig U et al. Improved treatment results in high-risk - ICC APL 01
pediatric acute myeloid leukemia patients after
intensification
with
high-dose
cytarabine
and - AIEOP ALCL 99
mitoxantrone: results of Study Acute Myeloid Leukemia-
30
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
Berlin-Frankfurt-Münster 93. J Clin Oncol 2001;19:27052713
Dahl GV et al. Mitoxantrone, etoposide and cyclosporine
therapy in pediatric patients with recurrent or refractory
acute myeloid leukemia. J Clin Oncol 2000;18:1867-1875
Testi AM et al. GIMEMA-AIEOPAIDA protocol for the
treatment of newly diagnosed acute promyelocytic
leukemia (APL) in children. Blood 2005;106:447-453
OXALIPLATINO
Nessuna
Spunt SL et al. Phase I clinical trial of oxaliplatin in
children and adolescents with refractory solid tumors. J
Clin Oncol 2007;25:2274-2280
Fouladi M et al. Phase II study of oxaliplatin in children
with
recurrent
or
refractory
medulloblastoma,
supratentorial primitive neuroectodermal tumors and
atypical teratoid rhabdoid tumors: a pediatric brain tumor
consortium study. Cancer 2006;107:2291-2297
PACLITAXEL
Nessuna
-Leucemia refrattaria
-Neuroblastoma disseminato
- Ep SSG RMS &
NRSTS 2005
Horton TM et al. A Phase 1 and pharmacokinetic clinical trial
Leucemia refrattaria
of paclitaxel for the treatment of refractory leukemia in
children: a Children's Oncology Group study. Pediatr Blood
Cancer 2008;50:788-792
-Carcinoma midollare del rene
Neuroblastoma disseminato
Kretschmar CS et al. Response to paclitaxel, topotecan and
topotecan-cyclophosphamide in children with untreated
disseminated neuroblastoma treated in an upfront phase II
investigational window: a pediatric oncology group study. J
Clin Oncol 2004;22:4119-4126
Surico G et al. New paclitaxel-cisplatin based chemotherapy
31
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
regimen for advanced stage, recurrent or refractory
neuroblastoma-preliminary report. Med Pediatr Oncol
2003;40:130-132
Linfoma non-Hodgkin
Berrak SG et al. Partial intestinal obstruction due to childhood
refractory non-Hodgkin's lymphoma, successfully treated
with taxol. Eur J Pediatr Surg 2003;13:276-279
Carcinoma midollare del rene
Strouse JJ et al. Significant responses to platinum-based
chemotherapy in renal medullary carcinoma. Pediatr Blood
Cancer 2005;44:407-411
PROCARBAZINA
Nessuna
-Linfoma di Hodgkin
Linfoma di Hodgkin
-Tumori del SNC
Fermé C et al. Chemotherapy plus involved-field radiation
in early-stage Hodgkin's disease. N Engl J Med
2007;357:1916-1927
-AIEOP LH 2004
Eich HT et al. A Contribution to solve the problem of the
need for consolidative radiotherapy after intensive
chemotherapy in advanced stages of Hodgkin's lymphoma
-analysis of a quality control program initiated by the
radiotherapy reference center of the German Hodgkin
Study Group (GHSG). Int J Radiat Oncol Biol Phys
2007;69:1187-1192
Dann EJ et al. Risk-adapted BEACOPP regimen can
reduce the cumulative dose of chemotherapy for standard
and high-risk Hodgkin lymphoma with no impairment of
outcome. Blood 2007;109:905-909
Tumori del SNC
Bruggers CS and Greene D. A phase 2 feasibility study of
sequential, dose intensive chemotherapy to treat
progressive low-grade gliomas in children. J Pediatr
RITUXIMAB
Nessuna
-Disordini linfoproliferativi CD20+
Disordini linfoproliferativi CD20+
32
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
-Porpora trombocitopenica idiopatica
(III linea)
Attias D and Weitzman S. The efficacy of rituximab in highgrade pediatric B-cell lymphoma/leukemia: a review of
available evidence. Curr Opin Pediatr 2008;20:17-22
-Citopenie autoimmuni
Trappe RU et al. Salvage therapy for relapsed post-transplant
lymphoproliferative disorders (PTLD) with a second
progression of PTLD after upfront chemotherapy: the role of
single-agent rituximab. Transplantation 2007;84:1708-1712
-Neuroblastoma e opsoclonomioclono
Protocolli
nazionali e
internazionali in
uso
Comoli P et al. Preemptive therapy of EBV-related
lymphoproliferative disease after pediatric haploidentical
stem cell transplantation. Am J Transplant 2007;7:1648-1655
Porpora trombocitopenica idiopatica (III linea)
Arnold DM and Kelton JG. Current options for the
treatment of idiopathic thrombocytopenic purpura. Semin
Hematol 2007;44:12-23
Rüfer A et al. Rituximab for acute plasma-refractory
thrombotic thrombocytopenic purpura. A case report and
concise review of the literature. Swiss Med Wkly
2007;137:518-524
Citopenie autoimmuni
Kim JJ et al. Rituximab for the treatment of autoimmune
cytopenias in children with immune deficiency. Br J
Haematol 2007;138:94-96
Bader-Meunier B et al. Rituximab therapy for childhood
Evans syndrome. Haematologica 2007;92:1691-1694
Zecca M et al. Anti-CD20 monoclonal antibody for the
treatment of severe, immune-mediated, pure red cell
aplasia and hemolytic anemia. Blood 2001;97:3995-3997
Neuroblastoma e opsoclono-mioclono
Burke MJ and Cohn SL. Rituximab for treatment of
opsoclonus-myoclonus syndrome in neuroblastoma.
33
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
Bell J et al. Response to rituximab in a child with
neuroblastoma and opsoclonus-myoclonus. Pediatr Blood
Cancer 2008;50:370-371
Yao G et al. A systematic review and economic model of
the clinical and cost-effectiveness of immunosuppressive
therapy for renal transplantation in children. Health
Technol Assess 2006;10:1-157
SORAFENIB
Nessuna
Grandinetti CA and Goldspiel BR. Sorafenib and
sunitinib: novel targeted therapies for renal cell cancer.
Pharmacotherapy 2007;27:1125-1144
SUNITINIB
Nessuna
Grandinetti CA and Goldspiel BR. Sorafenib and sunitinib:
novel targeted therapies for renal cell cancer.
Pharmacotherapy 2007;27:1125-1144
TALIDOMIDE
Nessuna
-Glioblastoma multiforme
Glioblastoma multiforme
-Malattia cronica da reazione del
trapianto contro l’ospite (Chronic
Graft versus Host Disease)
Turner CD et al. Phase II study of thalidomide and
radiation in children with newly diagnosed brain stem
gliomas and glioblastoma multiforme. J Neurooncol
2007;82:95-101
-Melanoma oculare
Malattia cronica da reazione del trapianto contro
l’ospite (Chronic Graft versus Host Disease)
-Tumori refrattari
Kulkarni S et al. Thalidomide after allogeneic
haematopoietic stem cell transplantation: activity in
chronic but not in acute graft-versus-host disease. Bone
Marrow Transplant 2003;32:165-170
Cole CH et al. Thalidomide in the management of chronic
graft-versus-host disease in children following bone
marrow transplantation. Bone Marrow Transplant
1994;14:937-942
34
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
Melanoma oculare
Soni S et al. Treatment of pediatric ocular melanoma with
high-dose interleukin-2 and thalidomide. J Pediatr
Tumori refrattari
Kieran MW et al. A feasibility trial of antiangiogenic
(metronomic) chemotherapy in pediatric patients with
recurrent or progressive cancer. J Pediatr Hematol Oncol
2005;27:573-581
Bessmertny O and Pham T. Thalidomide use in pediatric
patients. Ann Pharmacother 2002;36:521-525
TAMOXIFENE
Nessuna
-Fibromatosi aggressiva
-Tumori ricorrenti del SNC
TEMOZOLOMIDE
Nessuna
Tumori ricorrenti del SNC
Ben Arush MW et al. Clinical and radiographic response
in three children with recurrent malignant cerebral tumors
with high-dose tamoxifen. Pediatr Hematol Oncol
1999;16:245-250
-Tumori desmoidi resistenti o
inoperabili
Tumori desmoidi resistenti o inoperabili
-Tumori del SNC (III linea)
Tumori del SNC (III linea)
- Ep SSG RMS &
NRSTS 2005
Lackner H et al. Multimodal treatment of children with
unresectable or recurrent desmoid tumors: an 11-year
longitudinal observational study. J Pediatr Hematol Oncol
2004;26:518-522
Nicholson HS et al. Phase 2 study of temozolomide in
children and adolescents with recurrent central nervous
system tumors: a report from the Children's Oncology
Group. Cancer 2007;110:1542-1550
De Sio L et al. Temozolomide in resistant or relapsed
pediatric solid tumors. Pediatr Blood Cancer 2006;47:30-
35
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
36
Korones DN et al. Temozolomide and oral VP-16 for
children and young adults with recurrent or treatmentinduced malignant gliomas. Pediatr Blood Cancer
2006;47:37-41
TENIPOSIDE
Nessuna
-Retinoblastoma
- AIEOP LLA
Winter SS et al. Antimetabolite-based therapy in REC 2003
childhood T-cell acute lymphoblastic leukemia: a report of
POG study 9296. Pediatr Blood Cancer 2006;46:179-186
Linfoma non-Hodgkin
Millot F et al. Value of high-dose cytarabine during
interval therapy of a Berlin-Frankfurt-Munster-based
protocol in increased-risk children with acute
lymphoblastic leukemia and lymphoblastic lymphoma:
results of the European Organization for Research and
Treatment of Cancer 58881 randomized phase III trial. J
Clin Oncol 2001;19:1935-1942
Amylon MD et al. Intensive high-dose asparaginase
consolidation improves survival for pediatric patients with
T cell acute lymphoblastic leukemia and advanced stage
lymphoblastic lymphoma: a Pediatric Oncology Group
study. Leukemia 1999;13:335-342
Orbach D et al. Soft tissue sarcoma or malignant
mesenchymal tumors in the first year of life: experience of
the International Society of Pediatric Oncology (SIOP)
2005;23:4363-4371
Retinoblastoma
Antoneli CB et al. The addition of ifosfamide/etoposide to
cisplatin/teniposide improves the survival of children with
36
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
retinoblastoma and orbital involvement. J Pediatr Hematol
Oncol 2007;29:700-704
TIOGUANINA
acuta
Linfoma non-Hodgkin
-Gliomi a basso grado di malignità
Chantada GL et al. Results of a BFM-based protocol for - AIDAthe treatment of childhood B-non-Hodgkin's lymphoma Prot.(lap0493)
and B-acute lymphoblastic leukemia in Argentina. Med GIMEMA AIEOP
Pediatr Oncol 1997;28:333-341
- AIEOP LNH 97
Gliomi a basso grado di malignità
- EURO LB-02
Heath JA et al. Chemotherapy for progressive low-grade
gliomas in children older than ten years: the Dana-Farber - AIEOP
Interinfant 99
experience. Pediatr Hematol Oncol 2003;20:497-504
Lancaster DL et al. Tolerance of nitrosurea-based - INTERINFAN 06
- AIEOP LLA 2002
multiagent chemotherapy regime for low-grade pediatric - AIEOP LAM
gliomas. J Neurooncol 2003;63:289-294
REC/01
TIOTEPA
Nessuna
-Regimi mieloablativi dei tumori
cerebrali
-Sindrome di Evans
Ewing
-Neuroblastoma
-Anemia aplastica grave
Sindrome di Evans
- AIEOP/ISG
OSTEO REC VHR
Urban C et al. Successful unrelated cord blood
transplantation in a 7-year-old boy with Evans syndrome - ISG-AIEOP/VHRrefractory to immunosuppression and double autologous EW 1e 2
stem cell transplantation. Eur J Haematol 2006;76:526- AIEOP TCG 2004
530
- AIEOP SNC
95-01<3a
Burdach S and Jürgens H. High-dose chemoradiotherapy
(HDC) in the Ewing family of tumors (EFT). Crit Rev - AIEOP LLA
REC 2003
Oncol Hematol 2002;41:169-189
Neuroblastoma
Lang P et al. Retransplantation with stem cells from
mismatched related donors after graft rejection in pediatric
37
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
patients. Blood Cells Mol Dis 2008;40:33-39
Kamani N et al. A study of thiotepa, etoposide and
fractionated total body irradiation as a preparative regimen
prior to bone marrow transplantation for poor prognosis
patients with neuroblastoma. Bone Marrow Transplant
1996;17:911-916
Anemia aplastica grave
Urban C et al. Non-radiotherapy conditioning with stem
cell transplantation from alternative donors in children
with refractory severe aplastic anemia. Bone Marrow
Transplant 2005;35:591-594
Sindromi mielodisplastiche
Strahm B et al. Reduced intensity conditioning in
unrelated donor transplantation for refractory cytopenia in
childhood. Bone Marrow Transplant 2007;40:329-333
Cheuk DK et al. Autologous hematopoietic stem cell
transplantation for high-risk brain tumors in children. J
Neurooncol 2008;86:337-347
Urban C et al. Non-radiotherapy conditioning with stem
cell transplantation from alternative donors in children
with refractory severe aplastic anemia. Bone Marrow
Transplant 2005;35:591-594
TOPOTECANO
Nessuna
-Neuroblastoma resistente alla terapia
di prima linea
-Sarcoma di Ewing resistente alla
terapia di prima linea
Bernbeck B et al. Serial intense chemotherapy combining
topotecan, etoposide, carboplatin and cyclophosphamide
(TECC) followed by autologous hematopoietic stem cell
support in patients with high risk soft tissue sarcoma
38
- NB-AR- 01
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
-Rabdomiosarcoma resistente alla
terapia di prima linea
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
(STS). Klin Padiatr 2007;219:318-322
-Osteosarcoma
Ewing (recidivanti o resistenti alla
terapia di I linea)
VINBLASTINA
-Istiocitosi a cellule di
Langerhans
Osteosarcoma e Tumori della famiglia del sarcoma di
Ewing (recidivanti o resistenti alla terapia di I linea)
Hawkins DS et al. Topotecan by 21-day continuous
infusion in children with relapsed or refractory solid
tumors: a Children's Oncology Group study. Pediatr Blood
Cancer 2006;47:790-794
Fibromatosi aggressiva
- AIEOP LH 2004
-Linfoma di Hodgkin
Imashuku S et al. Treatment of neurodegenerative CNS - AIEOP ALCL 99
disease in Langerhans cell histiocytosis with a
combination of intravenous immunoglobulin and - LCH III 2001
chemotherapy. Pediatr Blood Cancer 2008;50:308-311
- LCH-S-05
Skapek SX et al. Vinblastine and methotrexate for - Ep SSG RMS &
desmoid fibromatosis in children: results of a Pediatric NRSTS 2005
Oncology Group Phase II Trial. J Clin Oncol
2007;25:501-506
Linfoma di Hodgkin
Fermé C et al. Chemotherapy plus involved-field radiation
in early-stage Hodgkin's disease. N Engl J Med
2007;357:1916-1927
Chow LM et al. Survival and late effects in children with
Hodgkin's lymphoma treated with MOPP/ABV and lowdose, extended-field irradiation. J Clin Oncol
2006;24:5735-5741
39
NOME COMPOSTO
VINCRISTINA
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
-Neuroblastoma
-Sarcoma di Ewing
-Nefroblastoma
Bernstein M et al. Ewing's sarcoma family of tumors: - AIEOP EsPhALL
current management. Oncologist 2006;11:503-519
- AIEOP LLA
Navid F et al. Concomitant administration of vincristine, REC Ph+ 2002
doxorubicin, cyclophosphamide, ifosfamide and etoposide
for high-risk sarcomas: the St. Jude Children's Research - AIEOP
Interinfant 99
Hospital experience. Cancer 2006;106:1846-1856
-Rabdomiosarcoma
acuta
Ewing
-Tumori del SNC
-Retinoblastoma
-Linfoma di Hodgkin e
non-Hodgkin
-Emangiopericitoma
-Sindrome di Evans
-Blastoma pleuropolmonare
-Sindrome PHACES
-Sindrome di Kasabach-Merritt
-Sarcomi (non rabdomiosarcomi)
- AIEOP LLA 2002
Miser JS et al. Treatment of metastatic Ewing - INTERINFAN 06
sarcoma/primitive neuroectodermal tumor of bone: - AIEOP LH 2004
evaluation of increasing the dose intensity of
chemotherapy- a report from the Children's Oncology - AIEOP LNH 97
Group. Pediatr Blood Cancer 2007;49:894-900
- EURO LB-02
Retinoblastoma
- NB-AR-01
Shields CL et al. Chemoreduction plus focal therapy for
retinoblastoma: factors predictive of need for treatment - SIOP-AIEOP NB
with external beam radiotherapy or enucleation. Am J Infant
Ophthalmol 2002;133:657-664
- AIEOP NB
Antoneli CB et al. Treatment of retinoblastoma patients rec/res 00
with chemoreduction plus local therapy: experience of the - Ep SSG RMS &
AC Camargo Hospital, Brazil. J Pediatr Hematol Oncol
NRSTS 2005
2006;28:342-345
- ISG/SSG III
Emangiopericitoma
ISG/SSG IV
Gowans LK et al. Successful treatment of an infant with
constitutional
chromosomal
abnormality
and - AIEOP/ISG 02
hemangiopericytoma with chemotherapy alone. J Pediatr (EW-REC)
- ISG-AIEOP/VHREW 1e 2
Sindrome di Evans
Urban C et al. Successful unrelated cord blood - AIEOP
transplantation in a 7-year-old boy with Evans syndrome T.Wilms 2003
refractory to immunosuppression and double autologous - AIEOP/SICP
stem cell transplantation. Eur J Haematol 2006;76:526-
40
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
530
Protocolli
nazionali e
internazionali in
uso
TREP 2005
- AIEOP SNC 99
Küpeli S et al. Treatment results of 84 patients with - AIEOP SNC
nasopharyngeal carcinoma in childhood. Pediatr Blood 95-01 < 3a
Cancer 2006;46:454-458
- AIEOP
Nakamura RA et al. High-dose-rate brachytherapy as part 2° Ependi 03
of a multidisciplinary treatment of nasopharyngeal
lymphoepithelioma in childhood. Cancer 2005;104:525- - HIT-SIOP PNET 4
531
- SIOP LGG 2004
Blastoma pleuropolmonare
Priest JR et al. Type I pleuropulmonary blastoma: a report
from the International Pleuropulmonary Blastoma
Registry. J Clin Oncol 2006;24:4492-4498
Fibromatosi aggressiva
Alebouyeh M et al. Aggressive intra-abdominal
fibromatosis in children and response to chemotherapy.
Buitendijk S et al. Pediatric aggressive fibromatosis: a
retrospective analysis of 13 patients and review of
literature. Cancer 2005;104:1090-1099
Sindrome PHACES
Herrero Hernández A et al. Successful treatment with
vincristine in PHACES syndrome. Oncol Clin Transl
2007;9:262-263
Syndrome di Kasabach-Merritt
Taki M et al. Successful treatment with vincristine of an
infant with intractable Kasabach-Merritt syndrome.
Pediatr Int 2006;48:82-84
Hauer J et al. Effective treatment of kaposiform
hemangioendotheliomas associated with Kasabach-Merritt
phenomenon using four-drug regimen. Pediatr Blood
Cancer 2007;49:852-854
41
NOME COMPOSTO
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
BIBLIOGRAFIA
Protocolli
nazionali e
internazionali in
uso
Sarcomi (non rabdomiosarcomi)
Pappo AS et al. Phase II trial of neoadjuvant vincristine,
ifosfamide and doxorubicin with granulocyte colonystimulating factor support in children and adolescents with
advanced-stage nonrhabdomyosarcomatous soft tissue
sarcomas: a Pediatric Oncology Group Study. J Clin
Oncol 2005;23:4031-4038
Nathan PC et al. Adjuvant chemotherapy for the treatment
of advanced pediatric nonrhabdomyosarcoma soft tissue
sarcoma: the National Cancer Institute experience. Pediatr
Blood Cancer 2005;44:449-454
VINDESINA
acuta resistente
- AIEOP LNH 97
-Linfomi
Reiter A et al. Chemotherapy in 998 unselected childhood - AIEOP EsPhALL
acute lymphoblastic leukemia patients. Results and
conclusions of the multicenter trial ALL-BFM 86. Blood - AIEOP ALCL 99
1994;84:3122-3133
-Osteosarcomi
Linfomi
Fermé C et al. A weekly regimen with dose escalation of
doxorubicin for patients with advanced Hodgkin's
lymphoma: results of a phase II study of the Groupe
d'Etudes des Lymphomes de l'Adulte (GELA). Leuk
Lymphoma 2007;48:691-698
Ueda T et al. Administration of vindesine sulfate for the
treatment of malignant hematological tumors. Gan To
Kagaku Ryoho 1982;9:306-315
Osteosarcoma
Philip T et al. High-dose methotrexate and HELP
[Holoxan (ifosfamide), eldesine (vindesine), platinum]doxorubicin in non-metastatic osteosarcoma of the
extremity: a French multicentre pilot study. Fédération
Nationale des Centres de Lutte contre le Cancer and
Société Française d'Oncologie Pédiatrique. Ann Oncol
1999;10:1065-1071
42
NOME COMPOSTO
VINORELBINA
ESTENSIONE DELLE
SINTESI DELLE
INDICAZIONI GIÀ
DELLA
AUTORIZZATE PER
DI
NUOVE EVIDENZE
L’IMPIEGO IN
SCIENTIFICHE
(grassetto)
PEDIATRIA
Nessuna
BIBLIOGRAFIA
Tumori del SNC ad alto grado di malignità
-Rabdomiosarcoma
Johansen M et al. Phase I evaluation of oral and
intravenous vinorelbine in pediatric cancer patients: a
report from the Children's Oncology Group. Clin Cancer
Res 2006;12:516-522
-Tumori del SNC ad alto grado di
malignità
-Linfoma di Hodgkin recidivante
-Tumore desmoplastico a piccole
cellule
Protocolli
nazionali e
internazionali in
uso
- Ep SSG RMS &
NRSTS 2005
Linfoma di Hodgkin recidivante
Ozkaynak MF and Jayabose S. Gemcitabine and
vinorelbine as a salvage regimen for relapse in Hodgkin
lymphoma after autologous hematopoietic stem cell
transplantation. Pediatr Hematol Oncol 2004;21:107-113
Tumore desmoplastico a piccole cellule
Ferrari A et al. Response to vinorelbine and low-dose
cyclophosphamide chemotherapy in two patients with
desmoplastic small round cell tumor. Pediatr Blood
Cancer 2007;49:864-866
Johansen M et al. Phase I evaluation of oral and
intravenous vinorelbine in pediatric cancer patients: a
report from the Children's Oncology Group. Clin Cancer
Res 2006;12:516-522
43

DGRT All. A 622_2008_OffLabel

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