Phase II Study of Nonmetastatic Desmoplastic Medulloblastoma in Children Younger Than 4 Years of Age: A Report of the Children’s Oncology Group (ACNS1221)

Lafay-Cousin L, Bouffet E, Strother D, Rudneva V, Hawkins C, Eberhart C, Horbinski C, Heier L, Souweidane M, Williams-Hughes C, Onar-Thomas A, Billups CA, Fouladi M, Northcott P, Robinson G, Gajjar A. Phase II Study of Nonmetastatic Desmoplastic Medulloblastoma in Children Younger Than 4 Years of Age: A Report of the Children’s Oncology Group (ACNS1221). J Clin Oncol. 2020 Jan 20;38(3):223-231. doi: 10.1200/JCO.19.00845. Epub 2019 Nov 27. PubMed PMID: 31774708; PubMed Central PMCID: PMC6968798.

Nodular desmoplastic medulloblastoma (ND) and medulloblastoma with extensive nodularity (MBEN) have been associated with a more favorable outcome in younger children. However, treatment-related neurotoxicity remains a significant concern in this vulnerable group of patients. ACNS1221 was a prospective single-arm trial of conventional chemotherapy for nonmetastatic ND and MBEN based on a modified HIT SKK 2000 regimen excluding intraventricular methotrexate, aiming to achieve similar outcome (2-year progression-free survival [PFS] ≥ 90%) with reduced treatment-related neurotoxicity. Secondary objectives included feasibility of timely central pathology review and evaluation of tumor molecular profile.