Clinical characterization of adult medulloblastoma and the effect of first-line therapies on outcome; The MD Anderson Cancer Center experience

Nazanin K Majd; Maximilan Mastall; Heather Lin; Seyede Shiva Dibaj; Kenneth R Hess; Ying Yuan; Manuela Martin-Bejarano Garcia; Gregory N Fuller; Kristin D Alfaro; Maria K Gule-Monroe; Jason T Huse; Soumen Khatua; Ganesh Rao; David I Sandberg; Jeffrey S Wefel; Debra N Yeboa; Arnold C Paulino; Susan L McGovern; Wafik Zaky; Anita Mahajan; Dima Suki; Shiao-Pei Weathers; Rebecca A Harriso; John F De Groo; Vinay K Puduvalli; Marta Penas-Prado

doi:10.1093/noajnl/vdab079

Clinical characterization of adult medulloblastoma and the effect of first-line therapies on outcome; The MD Anderson Cancer Center experience

Neurooncol Adv. 2021 Jun 22;3(1):vdab079. doi: 10.1093/noajnl/vdab079. eCollection 2021 Jan-Dec.

Authors

Nazanin K Majd¹, Maximilan Mastall², Heather Lin³, Seyede Shiva Dibaj³, Kenneth R Hess³, Ying Yuan³, Manuela Martin-Bejarano Garcia⁴, Gregory N Fuller⁵, Kristin D Alfaro¹, Maria K Gule-Monroe⁶, Jason T Huse⁵, Soumen Khatua⁷, Ganesh Rao⁸, David I Sandberg⁹, Jeffrey S Wefel¹⁰, Debra N Yeboa¹¹, Arnold C Paulino¹¹, Susan L McGovern¹¹, Wafik Zaky⁷, Anita Mahajan¹², Dima Suki⁷, Shiao-Pei Weathers¹, Rebecca A Harriso¹, John F De Groo¹, Vinay K Puduvalli¹, Marta Penas-Prado¹³

Affiliations

¹ Department of Neuro-Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
² Department of Neurology, Clinical Neuroscience Center and Brain Tumor Center, University Hospital Zurich, Zurich, Switzerland.
³ Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
⁴ Health Research Institute of the Hospital Clínico San Carlos, Madrid, Spain.
⁵ Department of Pathology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
⁶ Department of Neuroradiology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
⁷ Department of Pediatric Neuro-Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
⁸ Department of Neurosurgery, Baylor College of Medicine, Houston, TX, USA.
⁹ Department of Pediatric Neurosurgery, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
¹⁰ Department of Neuropsychology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
¹¹ Department of Radiation Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
¹² Department of Radiation-Oncology, Mayo Clinic, Rochester, Minnesota, USA.
¹³ Neuro-Oncology Branch, Center for Cancer Research, National Cancer Institute, Bethesda, Maryland, USA.

Abstract

Background: Adult medulloblastoma (MB) is rare, and management guidelines are largely based on pediatric clinical trials and retrospective series. Limited data exist with respect to clinical characteristics, prognostic factors, and outcomes based on first-line treatments.

Methods: Two hundred adults with MB seen at a single institution from January 1978 to April 2017 were identified and followed for a median of 8.4 y (7.1, 10.3).

Results: Patient's median age at diagnosis was 29 y (18, 63). One hundred eleven (55.5%) were standard-risk, 59 (29.5%) were high-risk, and 30 (15.0%) were indeterminate. Most received post-operative radiation (RT) (184 [92.0%]), and 105 (52.5%) received first-line chemotherapy. Median overall survival (OS) was 8.8 y (7.2, 12.2) and median progression-free survival (PFS) was 6.6 y (4.9, 11.2). High-risk patients had inferior OS (Hazard ratio [HR] = 2.5 [1.5, 4.2], P = .0006) and PFS (HR = 2.3 [1.3, 3.9], P = .002) compared to standard-risk patients. Age, sex, and metastatic disease were not associated with survival. After adjusting for risk status, those who received RT plus adjuvant chemotherapy had superior PFS compared to RT plus neoadjuvant chemotherapy [HR = 0.46 (0.22, 0.95), P = .0357]. Within a subgroup for whom detailed clinical data were available, those who received RT plus adjuvant chemotherapy had improved PFS compared to RT only [HR = 0.24 (0.074-0.76), P = .016]. The substitution of cisplatin for carboplatin and the elimination of vincristine did not negatively affect outcomes.

Conclusion: This is the largest single-institution retrospective study of adult MB to our knowledge and identifies standard-risk status, first-line RT and adjuvant chemotherapy as factors associated with improved outcomes.

Keywords: adult medulloblastoma; chemotherapy; clinical characterization; first-line therapy; radiation therapy.