Salvage Therapy for Childhood Medulloblastoma: A Single Center Experience

• Published in: Canadian journal of neurological sciences Vol. 46; no. 4; p. 403
• Main Authors: Kameda-Smith, Michelle M, Wang, Alick, Abdulhadi, Noora, Voth, Rebecca, Sergeant, Anjali, Maharaj, Arjuna, Bakhshinyan, David, Adile, Ashley A, Pai, Akshat M, Ajani, Olufemi, Yarascavitch, Blake, Alyman, M Cheryl, Duckworth, JoAnn, Samaan, M Constantine, Farrokhyar, Forough, Singh, Sheila K, Fleming, Adam
• Format: Journal Article
• Language: English
• Published: England 01-07-2019
• Subjects: Adolescent; Cerebellar Neoplasms - mortality; Cerebellar Neoplasms - therapy; Child; Child, Preschool; Female; Humans; Infant; Male; Medulloblastoma - mortality; Medulloblastoma - therapy; Neoplasm Recurrence, Local - mortality; Neoplasm Recurrence, Local - therapy; Retrospective Studies; Salvage Therapy - methods; Salvage Therapy - mortality; Treatment Outcome; Recurrent or treatment-refractory pediatric medulloblastoma; Salvage therapy
• ISSN: 0317-1671
• DOI: 10.1017/cjn.2019.39

Children diagnosed with medulloblastoma (MB) who are refractory to upfront therapy or experience recurrence have very poor prognoses. Although phase I and phase II trials exist, these treatments bear significant treatment-related morbidity and mortality.
A retrospective review of children diagnosed with a recurrence of MB from 2002 to 2015 at McMaster University was undertaken.
Recurrent disease in 10 patients involved leptomeningeal dissemination, with 3 experiencing local recurrence. In three recurrent patients the disease significantly progressed, and the children were palliated. The remaining 10 children underwent some form of salvage therapy, including surgical re-resection, radiation, and chemotherapy, either in isolation or in varying combinations. Of the 13 children experiencing treatment-refractory or recurrent disease, 4 are currently alive with a median follow-up of 38.5 months (75.5 months). Of the eight patients with molecular subgrouping data, none of the Wnt MB experienced recurrence.
Recurrent MB carried a poor prognosis with a 5-year overall survival (OS) of 18.2% despite the administration of salvage therapy. The upfront therapy received, available treatment, and tolerability of the proposed salvage therapy resulted in significant heterogeneity in the treatment of our recurrent cohort.