Dataset Information

Characterization of recurrence patterns and outcomes of medulloblastoma in adults: The University of Texas MD Anderson Cancer Center experience.

ABSTRACT:

Background

Medulloblastoma in adults is rare and treatment decisions are largely driven from pediatric literature. We sought to characterize recurrent medulloblastoma in adults.

Methods

From a single-institution dataset of 200 adult patients diagnosed with medulloblastoma during 1978-2017, those with recurrence were analyzed for clinical features, treatment, and outcome.

Results

Of the 200 patients, 82 (41%) with median age of 29 years (18-59) had recurrence after a median follow-up time of 8.4 years (95% CI = 7.1, 10.3). Of these, 30 (37%) were standard-risk, 31 (38%) were high-risk, and 21 (26%) had unknown-risk diseases at the time of initial diagnosis. Forty-eight (58%) presented with recurrence outside the posterior fossa, of whom 35 (43%) had distant recurrence only. Median Progression-free survival (PFS) and OS from initial surgery were 33.5 and 62.4 months, respectively. Neither PFS nor OS from initial diagnosis differed between the standard-risk and high-risk groups in those who experience recurrence (P = .505 and .463, respectively). Median OS from first recurrence was 20.3 months, also with no difference between the standard-risk and high-risk groups (P = .518). Recurrences were treated with combinations of re-resection (20 patients; 25%), systemic chemotherapy (61 patients; 76%), radiation (29 patients; 36%), stem cell transplant (6 patients; 8%), and intrathecal chemotherapy (4 patients; 5%). Patients who received radiation at recurrence had better OS (32.9 months) than those who did not (19.2 months) (P = .034).

Conclusions

Recurrent medulloblastoma in adults has a poor prognosis irrespective of initial risk stratification. Recurrence commonly arises outside the posterior fossa years after initial diagnosis.

SUBMITTER: Gregory TA

PROVIDER: S-EPMC10129387 | biostudies-literature | 2023 Jan-Dec

REPOSITORIES: biostudies-literature

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Publications

Characterization of recurrence patterns and outcomes of medulloblastoma in adults: The University of Texas MD Anderson Cancer Center experience.

Gregory Timothy A TA Mastall Maximilian M Lin Heather H Hess Kenneth R KR Yuan Ying Y Martin-Bejarano Garcia Manuela M Fuller Gregory N GN Alfaro Kristin D KD Gule-Monroe Maria K MK Huse Jason T JT Khatua Soumen S Rao Ganesh G Sandberg David I DI Wefel Jeffrey S JS Yeboa Debra N DN Paulino Arnold C AC McGovern Susan L SL Zaky Wafik W Mahajan Anita A Suki Dima D Weathers Shiao-Pei SP Harrison Rebecca A RA de Groot John F JF Puduvalli Vinay K VK Penas-Prado Marta M Majd Nazanin K NK

Neuro-oncology advances 20230101 1

<h4>Background</h4>Medulloblastoma in adults is rare and treatment decisions are largely driven from pediatric literature. We sought to characterize recurrent medulloblastoma in adults.<h4>Methods</h4>From a single-institution dataset of 200 adult patients diagnosed with medulloblastoma during 1978-2017, those with recurrence were analyzed for clinical features, treatment, and outcome.<h4>Results</h4>Of the 200 patients, 82 (41%) with median age of 29 years (18-59) had recurrence after a median ...[more]

PMID: 37114244

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Project description:Background/aimsIn our 2009 article, we showed that Bayesian methods had established a foothold in developing therapies in our institutional oncology trials. In this article, we will document what has happened since that time. In addition, we will describe barriers to implementing Bayesian clinical trials, as well as our experience overcoming them.MethodsWe reviewed MD Anderson Cancer Center clinical trials submitted to the institutional protocol office for scientific and ethical review between January 2009 and December 2013, the same length time period as the previous article. We tabulated Bayesian methods implemented for design or analyses for each trial and then compared these to our previous findings.ResultsOverall, we identified 1020 trials and found that 283 (28%) had Bayesian components so we designated them as Bayesian trials. Among MD Anderson-only and multicenter trials, 56% and 14%, respectively, were Bayesian, higher rates than our previous study. Bayesian trials were more common in phase I/II trials (34%) than in phase III/IV (6%) trials. Among Bayesian trials, the most commonly used features were for toxicity monitoring (65%), efficacy monitoring (36%), and dose finding (22%). The majority (86%) of Bayesian trials used non-informative priors. A total of 75 (27%) trials applied Bayesian methods for trial design and primary endpoint analysis. Among this latter group, the most commonly used methods were the Bayesian logistic regression model (N = 22), the continual reassessment method (N = 20), and adaptive randomization (N = 16). Median institutional review board approval time from protocol submission was the same 1.4 months for Bayesian and non-Bayesian trials. Since the previous publication, the Biomarker-Integrated Approaches of Targeted Therapy for Lung Cancer Elimination (BATTLE) trial was the first large-scale decision trial combining multiple treatments in a single trial. Since then, two regimens in breast cancer therapy have been identified and published from the cooperative Investigation of Serial Studies to Predict Your Therapeutic Response with Imaging and Molecular Analysis (I-SPY 2), enhancing cooperation among investigators and drug developers across the nation, as well as advancing information needed for personalized medicine. Many software programs and Shiny applications for Bayesian trial design and calculations are available from our website which has had more than 21,000 downloads worldwide since 2004.ConclusionBayesian trials have the increased flexibility in trial design needed for personalized medicine, resulting in more cooperation among researchers working to fight against cancer. Some disadvantages of Bayesian trials remain, but new methods and software are available to improve their function and incorporation into cancer clinical research.

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Dataset Information

Characterization of recurrence patterns and outcomes of medulloblastoma in adults: The University of Texas MD Anderson Cancer Center experience.

Background

Methods

Results

Conclusions

Publications

Characterization of recurrence patterns and outcomes of medulloblastoma in adults: The University of Texas MD Anderson Cancer Center experience.

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