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

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

Nazanin K Majd  1 , Maximilan Mastall  2 , Heather Lin  3 , Seyede Shiva Dibaj  3 , Kenneth R Hess  3 , Ying Yuan  3 , Manuela Martin-Bejarano Garcia  4 , Gregory N Fuller  5 , Kristin D Alfaro  1 , Maria K Gule-Monroe  6 , Jason T Huse  5 , Soumen Khatua  7 , Ganesh Rao  8 , David I Sandberg  9 , Jeffrey S Wefel  10 , Debra N Yeboa  11 , Arnold C Paulino  11 , Susan L McGovern  11 , Wafik Zaky  7 , Anita Mahajan  12 , Dima Suki  7 , Shiao-Pei Weathers  1 , Rebecca A Harriso  1 , John F De Groo  1 , Vinay K Puduvalli  1 , Marta Penas-Prado  13
Affiliations

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

Nazanin K Majd et al. Neurooncol Adv. .

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.

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Figures

Figure 1.
Figure 1.
Kaplan-Meier curve of OS and PFS by risk status.
Figure 2.
Figure 2.
Flow chart of risk stratifications and first-line therapies.
Figure 3.
Figure 3.
Kaplan-Meier curve of OS and PFS by first-line therapies in patients with known risk status.
Figure 4.
Figure 4.
Kaplan-Meier curve of OS and PFS by first-line therapies (the Full data subset).
See this image and copyright information in PMC

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