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Abstract OBJECTIVE To describe real-world outcomes in children with medulloblastoma treated with radiation-sparing chemotherapy in the USA. METHODS Clinical and molecular data were obtained from the Children’s Brain Tumor Network (CBTN), a multi-institutional collaboration to collect and freely share biological samples and longitudinal clinical data from consented patients. Patients with medulloblastoma treated with radiation-sparing regimens were included. Clinical data included patient demographics, stage, extent of resection, treatment regimen, and survival. Three-year overall survival (OS3) was calculated from diagnosis to death from any cause or last follow-up with log-rank p-value. RESULTS Eighty-one patients (56% male, median age 2.51 range 0-7.44 years) with medulloblastoma (32% metastatic) were included. Molecular group was available for 36 (44%). Patients were treated according to protocols CCG99703 (n=26), ACNS0334 Arm A (n=13), ACNS0334 Arm B (n=30), PBTC026 (n=10), and ACNS1221 (n=2). Only 29 (36%) were enrolled on a therapeutic study. Seventy-two patients had median follow-up of 2.7 years (range 0.3-13 years). OS3 was 79+6; OS3 by molecular group was 90+9 in SHH (n=14), 55+14 in Group 3 (n=16), 56+25 in Group 4 (n=6). OS3 was 92+5 for 55 patients with localized disease versus 53+12 for 26 with metastasis (p=0.001); 86+6 for 55 patients with gross total resection versus 62+11 in 26 with partial resection or biopsy (p=0.03). OS3 was not associated with upfront radiation, 80+7 for 54 patients who received no radiation before relapse versus 78+9 for 27 who received radiation before relapse (21 focal, 6 craniospinal). OS3 was 87+8 for 30 patients on a methotrexate-containing regimen versus 74+7 for 51 without methotrexate (p=0.27). Five patients developed second malignancy. CONCLUSIONS CBTN data report real-world survival for young children treated with radiation-sparing approaches, most not enrolled on studies. Ongoing molecular characterization of the CBTN cohort will facilitate additional analysis to improve risk stratification and therapy selection for young children.
Ronsley et al. (Tue,) studied this question.