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

Purpose To determine the survival of infants and young children with non‐metastatic medulloblastoma using intensive myeloablative chemotherapy and autologous hematopoietic progenitor cell rescue (AuHCR). Methods Twenty‐one children less than 3 years old at diagnosis with non‐metastatic medulloblasto...

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Published in:Pediatric Blood & Cancer 2008-06, Vol.50 (6), p.1169-1175
Main Authors: Dhall, Girish, Grodman, Howard, Ji, Lingyun, Sands, Stephen, Gardner, Sharon, Dunkel, Ira J., McCowage, Geoffrey B., Diez, Blanca, Allen, Jeffrey C., Gopalan, Anjali, Cornelius, Albert S., Termuhlen, Amanda, Abromowitch, Minnie, Sposto, Richard, Finlay, Jonathan L.
Format: Article
Language:English
Subjects:
Antineoplastic Combined Chemotherapy Protocols - therapeutic use
Cerebellar Neoplasms - drug therapy
Cerebellar Neoplasms - mortality
Cerebellar Neoplasms - surgery
Child Behavior
Child, Preschool
Combined Modality Therapy
Disease-Free Survival
Female
Humans
Infant
infants
Intelligence Tests
Male
medulloblastoma
Medulloblastoma - drug therapy
Medulloblastoma - mortality
Medulloblastoma - surgery
myeloablative chemotherapy and late effects
Neuropsychological Tests
Quality of Life
Survival Rate
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Summary:Purpose To determine the survival of infants and young children with non‐metastatic medulloblastoma using intensive myeloablative chemotherapy and autologous hematopoietic progenitor cell rescue (AuHCR). Methods Twenty‐one children less than 3 years old at diagnosis with non‐metastatic medulloblastoma were enrolled on two identical serial studies, “Head Start” I and “Head Start” II. After surgery, patients received five cycles of induction chemotherapy consisting of vincristine, cisplatin, cyclophosphamide and etoposide. Following induction, all patients underwent myeloablative chemotherapy using carboplatin, thiotepa and etoposide with AuHCR. Irradiation was used only at relapse. Results The 5‐year event‐free (EFS) and overall survival (OS) rates (±SE) for all patients, patients with gross total resection, and patients with residual tumor were 52 ± 11% and 70 ± 10%, 64 ± 13% and 79 ± 11%, and 29 ± 17% and 57 ± 19%, respectively. The 5‐year EFS and OS ( ± SE) for patients with desmoplastic and classical medulloblastoma were 67 ± 16% and 78 ± 14%, and 42 ± 14 and 67 ± 14%, respectively. There were four treatment related deaths. The majority of survivors (71%) avoided irradiation completely. Mean intellectual functioning and quality of life (QoL) for children surviving without irradiation was within average range for a majority of survivors tested. Conclusion This strategy of brief intensive chemotherapy for young children with non‐metastatic medulloblastoma eliminated the need for craniospinal irradiation 52% of the patients, and may preserve QoL and intellectual functioning. The excellent survival rates are somewhat dampened by high toxic mortality. Pediatr Blood Cancer 2008;50:1169–1175. © 2008 Wiley‐Liss, Inc.
ISSN:1545-5009
1545-5017
1096-911X
DOI:10.1002/pbc.21525