The efficacy and toxicity of high-dose chemotherapy and autologous stem cell transplantation (HDCT/ASCT) were investigated for improving the outcomes of patients with relapsed medulloblastoma. A total of 15 patients with relapsed medulloblastoma were enrolled in the KSPNO-S-053 study from May 2005 to May 2007. All patients received approximately 4 cycles of salvage chemotherapy after relapse. Thirteen underwent HDCT/ASCT; CTE and CM regimen were employed for the first HDCT (HDCT1) and second HDCT (HDCT2), respectively, and 7 underwent HDCT2. One transplant related mortality (TRM) due to veno-occlusive disease (VOD) occurred during HDCT1 but HDCT2 was tolerable with no further TRM. The 3-yr overall survival probability and event-free survival rates +/-95% confidence intervals (CI) were 33.3+/-12.2% and 26.7% +/-11.4%, respectively. When analysis was confined to only patients who had a complete response (CR) or partial response (PR) prior to HDCT, the probability of 3-yr overall survival rates +/-95% CI was 40.0+/-15.5%. No patients with stable disease (SD) or progressive disease (PD) survived. Survival rates from protocol KSPNO-S-053 are encouraging and show that tumor status prior to HDCT/ASCT is an important factor to consider for improving survival rates of patients with relapsed medulloblastoma.
Adolescent ; Cerebellar Neoplasms/drug therapy/mortality/*therapy ; Child ; Child, Preschool ; Combined Modality Therapy ; Disease-Free Survival ; Female ; *Hematopoietic Stem Cell Transplantation ; Humans ; Male ; Medulloblastoma/drug therapy/mortality/*therapy ; Neoplasm Recurrence, Local/drug therapy/mortality/*therapy ; Republic of Korea ; Salvage Therapy ; Transplantation, Autologous ; Young Adult

Medulloblastoma, once a tumor with a dismal prognosis, is one of the most common primary brain tumors of childhood. As the methods of treatment have been continuously refined, the outcome has improved remarkably during the last few decades. The outcome of 78 medulloblastoma patients, which were managed from 1972 to 1992 at the Department of Neurosurgery of Seoul National University Hospital, were analyzed to calculate the 3-year and 5-year survival rates (3yS and 5yS). Of those, 52 cases which were treated after July 1982 were studied 1) to calculate the 3yS and 5yS, 2) to figure out the prognostic factors of survival, and 3) to investigate the role of adjuvant chemotherapy ('8-drugs-in-a-day' protocol: CCNU, cisplatin, vincristine, hydroxyurea, procarbazine, cytosine arabinoside, methylprednisolone and cyclophosphamide). The 3yS and 5yS of the 78 patients were 57.4% and 47.3%, respectively. Of the 52 patients treated after July 1982, the 3yS and 5yS were 67.8% and 64.1%, respectively. The latest recurrence was at 56 months after surgery. All the recurrences were within the risk period of Collins' rule. Of the prognostic factors studied by univariate analysis (age, sex, Chang's classification T- and M-stages, extent of surgical removal, and chemotherapy), Chang's classification M-stage and sex were the statistically significant factors (p = 0.028 and 0.024 respectively). On multivariate analysis, only the M-stage was statistically significant (p = 0.004). Adjuvant chemotherapy had different influences in different patient groups. Only in the 'poor risk' group, did adjuvant chemotherapy have a strong tendency to better outcome (p = 0.069). Further data collection and analysis will lead to better treatment modalities and better outcome for this most common primary malignant brain tumor in childhood.
Adolescent ; Adult ; Antineoplastic Combined Chemotherapy Protocols/therapeutic use ; Cerebellar Neoplasms/*drug therapy/mortality/radiotherapy ; Chemotherapy, Adjuvant ; Child ; Child, Preschool ; Female ; Humans ; Infant ; Male ; Medulloblastoma/*drug therapy/mortality/radiotherapy ; Middle Aged ; Neoplasm Recurrence, Local ; Prognosis ; Survival Rate ; Treatment Outcome