Group 4 Medulloblastomas of Early Childhood Treated With High-Dose Chemotherapy- and Craniospinal Irradiation-Sparing Approach.
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Orduña Patricia, Cansanay Rose Daynielle, Erker Craig, Ratterman Erin, Schlosser Mary Pat, Ronsley Rebecca, Cacciotti Chantel, Cheng Sylvia, Hukin Juliette, Aguilera Dolly, Mazewski Claire, Magimairajan Vanan, Pillay-Smiley Natasha, Sait Sameer Farouk, Cluster Andrew, Abdelbaki Mohamed S, Margol Ashley S, Michaiel George, Chi Susan, Salloum Ralph, Harrod Virginia, Hoffman Lindsey, Bracher Annalise, Senapati Akanksha, Ramaswamy Vijay, Zapotocky Michal, Lopez Vicente Santa-Maria, Doris Kathleen, Mynarek Martin, Rutkowski Stefan, Lassaletta Alvaro, Bouffet Eric, Lafay-Cousin Lucie
Аннотация
METHODS: This multicenter study reported outcomes for patients younger than 7 years old with Group 4 MB treated with high-dose chemotherapy (HDC) without adjuvant CSI.
RESULTS: Thirty-eight patients were included (26 M/12 F). Median age at diagnosis was 46.4 months (25.9-78), with 24% ≤36 months. Twenty-four patients (63.2%) had localized disease. Fourteen (36.8%) presented with metastatic disease, and 26 (68.4%) underwent gross total resection (GTR). The most used HDC regimens were carboplatin/thiotepa (76.3%) and carboplatin/thiotepa/etoposide (21.1%). Twenty (52.6%) relapses occurred at a median 21.9 months (5-99.8) from diagnosis. Patients with upfront GTR and/or who received three cycles of HDC had better PFS (p = 0.02 and p = 0.002, respectively). Local relapse accounted for 45%. Eighteen patients received salvage therapy with curative intent, all with radiotherapy (16 = CSI, 2 = focal). The CSI dose ranged from 18 to 36 Gy, and 43.7% received ≤23.4 Gy. Patients who underwent salvage surgery and/or chemotherapy were more likely to receive CSI ≤23.4 Gy (p = 0.008 and p = 0.03). The 5-year post-relapse survival and overall survival (OS) were, respectively, 60.3% (95% confidence interval [CI]: 26.9-82) and 72.7% (95% CI: 51.9-85.7). The 5-year CSI-free OS was 69.7% (95% CI: 39.1-87.1).
CONCLUSION: High dose chemotherapy for Group 4 MB was associated with a relapse rate of 52.6%, which favorably compares to data reported with conventional chemotherapy. Salvage radiotherapy retrieved more than two-thirds of the patients. Half of the survivors never received radiotherapy. Such an approach may represent an alternative to upfront CSI for young children with Group 4 MB.