Efficacy of tailored treatment for high- and low-risk medulloblastoma in adults: A large prospective phase II trial.

Brandes, A.A.; Franceschi, Enrico; Tosoni, Alicia; Frezza, G.; Agati, R.; Maestri, A; Ghimenton, Claudio; Mazzocchi, V.; Scopece, L.; Ermani, Mario

doi:10.1200/jco.2010.28.15_suppl.2003

Cited by 13 publications

(3 citation statements)

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“…Even though there is consensus regarding the use of first-line chemotherapy in adult MB patients, the timing of chemotherapy (neoadjuvant, concurrent, and adjuvant) and the optimal chemotherapy regimen are unclear. Only three prospective studies of chemotherapy in adult MB patients have been performed to date, 15 , 19 , 21 , 36 , 37 and they each used different chemotherapy regimens.…”

Section: Discussionmentioning

confidence: 99%

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

Majd

Mastall

Lin

et al. 2021

Neuro-Oncology Advances

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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 200 adults with MB seen at a single institution from January 1978 to April 2017 were identified and followed for a median of 8.4y (7.1, 10.3). Results Patient’s median age at diagnosis was 29y (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 8.8y (7.2, 12.2) and median progression-free survival (PFS) was 6.6y (4.9, 11.2). High-risk patients had inferior OS (Hazard ratio [HR]=2.5 [1.5, 4.2], p=0.0006) and PFS (HR=2.3 [1.3, 3.9], p=0.002) 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=0.0357]. 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 = 0.016]. 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.

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Section: Discussionmentioning

confidence: 99%

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

Majd

Mastall

Lin

et al. 2021

Neuro-Oncology Advances

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“…Before AHSCT, all patients had radiotherapy as the first-line therapy. As a second-line therapy, they received combination chemotherapies such as Vincristine plus Procarbazine plus Mustard, Cisplatin plus Etoposide plus Cyclophosphamide, Cisplatin plus Lomustine plus Vincristine or Ifosfamide plus Carboplatin plus Etoposide (10,11). Medulloblastoma is a high risk for most of our patients that received AHSCT as the third-line therapy.…”

Section: Discussionmentioning

confidence: 99%

Retrospective analysis of patients with relapsed/refractory medulloblastoma after autologous stem cell transplantation

Acar¹,

Ertürk²,

Kiziloz³

et al. 2020

GMJ

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Aims: Medulloblastoma is very rare and accounts for only 1% of intracranial tumors in adults. There are limited treatment options for adult medulloblastoma patients who have relapsed or refractory disease. We aimed to show real-life data on health outcomes in adult patients with recurrent or refractory medulloblastoma patients who received autologous hematopoietic stem cell transplantation (AHSCT). Methods: We analyzed the data of 15 patients who underwent AHSCT after ifosfamide, carboplatin, and etoposide (ICE) as high-dose chemotherapy (HDCT) regimen for relapsed or refractory medulloblastoma from 2010 to 2020. Overall response rate (ORR), overall survival (OS) and progression-free survival (PFS) of the patients were evaluated. Results: Fifteen patients were observed in this study. The mean age of the study group was 27.9±8.1 years and 53.3% of the patients were female (n=8). ORR was 100%. The median OS and PFS were 24 months [95% confidence interval (CI): 11.4-36.5] and 13 months (95% CI: 10.2-15.8), respectively. One-year OS and PFS rates were 77% and 55.2%, respectively. Five-year OS rate was 82.5%. Conclusions: AHSCT with ICE as HDCT regimen is a safe and effective treatment option for relapsed or refractory adult medulloblastoma patients with an acceptable ORR, OS and PFS time.

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“…Initial results, published in 2007 and comprising 36 patients showed a five-year PFS and OS of 72% and 75%, respectively, with no difference in outcomes between low and high-risk patients [ 33 ]. However, an updated abstract reported in 2010 included 95 total patients and reported a 10-year OS of 65% in low-risk patients vs 45% in high-risk patients ( p = 0.02) [ 57 ].…”

Section: Clinical Trialsmentioning

confidence: 99%

Pediatric versus Adult Medulloblastoma: Towards a Definition That Goes beyond Age

Wooley

Penas‐Prado

2021

Cancers

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Medulloblastoma is a rare malignant brain tumor that predominantly affects children but also occurs in adults. The incidence declines significantly after age 15, and distinct tumor molecular features are seen across the age spectrum. Standard of care treatment consists of maximal safe surgical resection followed by adjuvant radiation and/or chemotherapy. Adjuvant treatment decisions are based on individual patient risk factors and have been informed by decades of prospective clinical trials. These trials have historically relied on arbitrary age cutoffs for inclusion (age 16, 18, or 21, for example), while trials that include adult patients or stratify patients by molecular features of disease have been rare. The aim of this literature review is to review the history of clinical trials in medulloblastoma, with an emphasis on selection criteria, and argue in favor of rational and inclusive trials based on molecular features of disease as opposed to chronological age. We performed a scoping literature review for medulloblastoma and clinical trials and include a summary of those results. We also discuss some of the significant advances made in understanding the molecular biology of medulloblastoma within the past decade, most notably the identification of four distinct subgroups based on gene expression profiling. We will also cite the recent experiences of childhood leukemia and the emergence of tissue-agnostic therapies as examples of successes of rationally designed, inclusive trials translating to improved clinical outcomes for patients across the age spectrum. Despite the prior trial history and recent molecular advances outcomes remain poor for ~30% of medulloblastoma patients. We believe that defining patients by the specific molecular alterations their tumors harbor is the best way to ensure they can access potentially efficacious therapies on clinical trials.

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Efficacy of tailored treatment for high- and low-risk medulloblastoma in adults: A large prospective phase II trial.

Cited by 13 publications

References 0 publications

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

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

Retrospective analysis of patients with relapsed/refractory medulloblastoma after autologous stem cell transplantation

Pediatric versus Adult Medulloblastoma: Towards a Definition That Goes beyond Age

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