MB-66PILOT STUDY OF A SURGERY AND CHEMOTHERAPY-ONLY APPROACH IN THE UPFRONT THERAPY OF CHLDREN WITH Wnt POSITIVE STANDARD RISK MEDULLOBLASTOMA
Year: 2016
Type: article
Abstract: Children with Wnt-positive medulloblastoma, which in historical cohorts represents between 10-15% of children with medulloblastoma, have an excellent prognosis when treated with radiotherapy followed by adjuvant chemotherapy. The purpose of this study is determine whether radiation therapy can be entirely omitted in newly diagnosed children with standard risk, Wnt positive medulloblastoma utilizing a chemotherapy-only approach. METHODS: Following initial tumor resection, eligible subjects must have a gross-total resection, no evidence of metastatic tumor, and non-anaplastic histology. Wnt medulloblastoma is defined as beta-catenin nuclear reactivity by immunohistochemistry, monosomy 6 as determined by array CGH, and the absence of MYCN or MYC amplification as determined by fluorescent in-situ hybridization. A total of thirteen subjects can be enrolled. RESULTS: Three children have been screened to-date who met initial standard-risk criteria. 0/3 children met the protocol definition for Wnt-positive medulloblastoma with one child diagnosed with sonic hedgehog (SHH) medulloblastoma and two children with non-SHH/non-Wnt medulloblastoma. An additional 4 tumor specimens did not demonstrate nuclear beta-catenin reactivity and were not further evaluated for possible inclusion. CONCLUSION: As anticipated, frequency of Wnt-positive medulloblastoma is rare, resulting in slow accrual to the planned protocol therapy. The study is currently open in five centers. Updated accrual, rationale for the study design, and early outcomes of subjects enrolled on the trial will be detailed.
Source: Neuro-Oncology
Language: English
Subfield: Genetics
Field: Medicine
Domain: Health Sciences
Open Access status: green
OpenAlex ID: W2429200560