Study Design and Patients
medulloblastoma
MEDULLOBLASTOMA
MEMMAT was a prospective, international, multicenter, single-arm, phase 2 study that included sites in Europe and the US. This academic trial depended on local institutions to generate funding to perform the study. The trial was conducted in accordance with the Declaration of HelsinkiPatients were enrolled from April 1, 2014, to March 31, 2021. Eligible patients were younger than 20 years at original diagnosis, with a histologically confirmed medulloblastoma diagnosis and a documented relapse on magnetic resonance imaging (MRI), biopsy, or cerebrospinal fluid (CSF) cytologic testing. There was no limit to the number of previous relapses or any restrictions to the types of previous therapies administered. Acceptable organ function and bone marrow recovery and a Karnofsky Performance Scale Index score or Lansky Play-Performance Scale score of 50% or higher were required (lower Karnofsky and Lansky scores indicate functional impairment). MEMMAT did not collect data on race or ethnicity. At the time of enrollment, patients were permitted to undergo additional surgical resection. Patients dependent on ventriculoperitoneal shunts were excluded because of the anticipated difficulties in providing adequate intraventricular therapy. The medulloblastoma molecular group was determined by DNA methylation profiling according to previously described methods.