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Outcome for children <4 years of age with malignant central nervous system tumors treated with high‐dose chemotherapy and autologous stem cell rescue
Author(s) -
Thorarinsdottir Halldora K.,
Rood Brian,
Kamani Naynesh,
Lafond Debbie,
PerezAlbuerne Evelio,
Loechelt Brett,
Packer Roger J.,
MacDonald Tobey J.
Publication year - 2007
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.20781
Subject(s) - medicine , thiotepa , carboplatin , ependymoma , vincristine , etoposide , chemotherapy , surgery , cyclophosphamide , oncology , cisplatin
Background Children <4 years of age (yo) with malignant central nervous system (CNS) tumors have a dismal prognosis. In an attempt to delay or obviate radiation therapy (XRT) and improve outcome, our institution has treated children <4 yo with newly diagnosed malignant CNS tumors with high‐dose chemotherapy (HDC) and autologous stem cell rescue (ASCR) followed by selective XRT. Procedure Fifteen children (age 4–38 months) with malignant CNS tumors have completed treatment with HDC/ASCR. All patients received three cycles of induction chemotherapy (cisplatin 3.5 mg/kg‐ day 0, cyclophosphamide 60 mg/kg‐ day 1 and 2, etoposide 2.5 mg/kg‐ day 0–2, vincristine 0.05 mg/kg, day 0, 7, 14) followed by three cycles of HDC (carboplatin 17 mg/kg and thiotepa 6 mg/kg, day 0 and 1) with ASCR. Histology included five medulloblastomas, four primitive neuroectodermal tumors (PNET), five malignant gliomas, and one ependymoma. Outcome and treatment toxicities were evaluated by retrospective chart review. Results Median follow‐up time of the 15 patients is 22 months (range 8–82 months). The 1‐ and 2‐year progression‐free survival (PFS) is 86.1% and 52.2% and overall survival (OS) 91.6% and 72.1%, respectively. Ten patients are alive and disease free 3–77 months (median 18 months) after having completed HDC/ASCR, thereoff five received XRT. Toxicity was primarily myelosuppression. There was no treatment mortality. Conclusions We are encouraged by the outcome of 15 children <4 yo with malignant CNS tumors treated with tandem cycles of HDC and ASCR at our institution. The treatment regimen is relatively well tolerated. Pediatr Blood Cancer 2007;48:278–284. © 2006 Wiley‐Liss, Inc.