Efficacy of Carboplatin and Isotretinoin in Children With High-risk Medulloblastoma: A Randomized Clinical Trial From the Children’s Oncology Group

Leary, Sarah E. S.; Packer, Roger J.; Li, Yimei; Billups, Catherine A.; Smith, Kyle S.; Jaju, Alok; Heier, Linda; Burger, Peter; Walsh, Karin; Han, Yuanyuan; Embry, Leanne; Hadley, Jennifer; Kumar, Rahul; Michalski, Jeff; Hwang, Eugene; Gajjar, Amar; Pollack, Ian F.; Fouladi, Maryam; Northcott, Paul A.; Olson, James M.

doi:10.1001/jamaoncol.2021.2224

Cited by 167Glioma Diagnosis and TreatmentAcute Lymphoblastic Leukemia researchNeuroblastoma Research and Treatments

Efficacy of Carboplatin and Isotretinoin in Children With High-risk Medulloblastoma: A Randomized Clinical Trial From the Children’s Oncology Group

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Leary, Sarah E. S., Packer, Roger J., Li, Yimei, Billups, Catherine A., Smith, Kyle S., Jaju, Alok, Heier, Linda, Burger, Peter +12 more·2021·JAMA Oncology

DOI 10.1001/jamaoncol.2021.2224PMC PMC8299367

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Abstract

IMPORTANCE: Brain tumors are the leading cause of disease-related death in children. Medulloblastoma is the most common malignant embryonal brain tumor, and strategies to increase survival are needed. OBJECTIVE: To evaluate therapy intensification with carboplatin as a radiosensitizer and isotretinoin as a proapoptotic agent in children with high-risk medulloblastoma in a randomized clinical trial and, with a correlative biology study, facilitate planned subgroup analysis according to World Health Organization consensus molecular subgroups of medulloblastoma. DESIGN, SETTING, AND PARTICIPANTS: A randomized clinical phase 3 trial was conducted from March 2007 to September 2018. Analysis was completed in September 2020. Patients aged 3 to 21 years with newly diagnosed high-risk medulloblastoma from Children's Oncology Group institutions within the US, Canada, Australia, and New Zealand were included. High-risk features included metastasis, residual disease, or diffuse anaplasia. INTERVENTIONS: Patients were randomized to receive 36-Gy craniospinal radiation therapy and weekly vincristine with or without daily carboplatin followed by 6 cycles of maintenance chemotherapy with cisplatin, cyclophosphamide, and vincristine with or without 12 cycles of isotretinoin during and following maintenance. MAIN OUTCOMES AND MEASURES: The primary clinical trial end point was event-free survival, using the log-rank test to compare arms. The primary biology study end point was molecular subgroup classification by DNA methylation array. RESULTS: Of 294 patients with medulloblastoma, 261 were evaluable after central radiologic and pathologic review; median age, 8.6 years (range, 3.3-21.2); 183 (70%) male; 189 (72%) with metastatic disease; 58 (22%) with diffuse anaplasia; and 14 (5%) with greater than 1.5-cm2 residual disease. For all participants, the 5-year event-free survival was 62.9% (95% CI, 55.6%-70.2%) and overall survival was 73.4% (95% CI, 66.7%-80.1%). Isotretinoin randomization was closed early owing to futility. Five-year event-free survival was 66.4% (95% CI, 56.4%-76.4%) with carboplatin vs 59.2% (95% CI, 48.8%-69.6%) without carboplatin (P = .11), with the effect exclusively observed in group 3 subgroup patients: 73.2% (95% CI, 56.9%-89.5%) with carboplatin vs 53.7% (95% CI, 35.3%-72.1%) without (P = .047). Five-year overall survival differed by molecular subgroup (P = .006): WNT pathway activated, 100% (95% CI, 100%-100%); SHH pathway activated, 53.6% (95% CI, 33.0%-74.2%); group 3, 73.7% (95% CI, 61.9%-85.5%); and group 4, 76.9% (95% CI, 67.3%-86.5%). CONCLUSIONS AND RELEVANCE: In this randomized clinical trial, therapy intensification with carboplatin improved event-free survival by 19% at 5 years for children with high-risk group 3 medulloblastoma. These findings further support the value of an integrated clinical and molecular risk stratification for medulloblastoma. TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT00392327.

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The MB-meta portal integrates clinical, genomic, and survival data from 898 patients across three major medulloblastoma clinical trials (ACNS0331, ACNS0332, SJMB03).
“we developed the Medulloblastoma Meta-Analysis (MB-meta) portal, which integrates clinical, genomic, and survival data from 898 patients across three major medulloblastoma clinical trials (16–18).”
From The Medulloblastoma Meta-Analysis Portal Enables Integrative Exploration of Clinical and Genomic Data from Patients (2025) · cites this paper as [16]

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Manfreda, L., Rampazzo, E., Persano, L., Viola, G., & Bortolozzi, R. (2023) · Biochemical Pharmacology · doi:10.1016/j.bcp.2023.115697 · Cited by 15 · see citing works
Current Strategies for Management of Medulloblastoma
Prados, M. D. (2023) · Diagnostics · doi:10.3390/diagnostics13162622 · Cited by 17 · see citing works
Smart Design of Targeted Drug Delivery System for Precise Drug Delivery and Visual Treatment of Brain Gliomas
Song, B., Wu, M., Qin, L., Liang, W., & Wang, X. (2024) · Advanced Healthcare Materials · doi:10.1002/adhm.202402967 · Cited by 15 · see citing works
Evolution of Systemic Therapy in Medulloblastoma Including Irradiation-Sparing Approaches
Mushtaq, N., Ain, R. U., Hamid, S. A., & Bouffet, É. (2023) · Diagnostics · doi:10.3390/diagnostics13243680 · Cited by 13 · see citing works
OLIG2 mediates a rare targetable stem cell fate transition in sonic hedgehog medulloblastoma
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Children's Oncology Group's 2023 blueprint for research: Central nervous system tumors
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Advancing medulloblastoma therapy: strategies and survival insights
Pan, Z., Bao, J., & Wei, S. (2025) · Clinical and Experimental Medicine · doi:10.1007/s10238-025-01648-5 · Cited by 12 · see citing works
Evolocumab in paediatric heterozygous familial hypercholesterolaemia: cognitive function during 80 weeks of open-label extension treatment
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Disease Evolution Monitored by Serial Cerebrospinal Fluid Liquid Biopsies in Two Cases of Recurrent Medulloblastoma
O’Halloran, K., Margol, A., Davidson, T. B., Estrine, D. B., Tamrazi, B., Cotter, J. A., Ji, J., & Biegel, J. A. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms25094882 · Cited by 11 · see citing works
Multiparametric MRI-based machine learning system of molecular subgroups and prognosis in medulloblastoma
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EYA2 tyrosine phosphatase inhibition reduces MYC and prevents medulloblastoma progression
Wolin, A. R., Vincent, M. Y., Hotz, T. J., Purdy, S. C., Rosenbaum, S. R., Hughes, C. J., Hsu, J. Y., Oliphant, M. U. J., Armstrong, B. A., Wessells, V. M., et al. (2023) · Neuro-Oncology · doi:10.1093/neuonc/noad128 · Cited by 11 · see citing works
Factors influencing outcomes of older children with medulloblastoma over 15 years in Peru, a resource‐limited setting
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Pediatric Central Nervous System Embryonal Tumors: Presentation, Diagnosis, Therapeutic Strategies, and Survivorship—A Review
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Radiotherapy for Primary Pediatric Central Nervous System Malignancies: Current Treatment Paradigms and Future Directions
Liu, K. X., Haas-Kogan, D. A., & Elhalawani, H. M. (2023) · Pediatric Neurosurgery · doi:10.1159/000533777 · Cited by 10 · see citing works
The Current Landscape of Targeted Clinical Trials in Non-WNT/Non-SHH Medulloblastoma
Ghasemi, D. R., Fleischhack, G., Milde, T., & Pajtler, K. W. (2022) · Cancers · doi:10.3390/cancers14030679 · Cited by 11 · see citing works
Changes to pediatric brain tumors in 2021 World Health Organization classification of tumors of the central nervous system
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Hypogonadism After Treatment for Medulloblastoma: Results From the SJMB03 Trial of Risk-Adapted Radiation Therapy
Merchant, T. E., Wu, S., Onar‐Thomas, A., Gajjar, A. J., & Delaney, A. (2023) · International Journal of Radiation Oncology*Biology*Physics · doi:10.1016/j.ijrobp.2023.01.001 · Cited by 9 · see citing works
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Synergy of retinoic acid and BH3 mimetics in MYC(N)-driven embryonal nervous system tumours
Seiboldt, T., Zeiser, C., Nguyen, D., Çelikyürekli, S., Herter, S., Najafi, S., Stroh-Dege, A., Meulenbroeks, C., Mack, N. L., Salem-Altintas, R., et al. (2024) · British Journal of Cancer · doi:10.1038/s41416-024-02740-5 · Cited by 9 · see citing works
Advancing Medulloblastoma Therapy in Pediatrics: Integrative Molecular Classification and Emerging Treatments
Kim, D. T., Uloho-Okundaye, M., Frederico, S. C., Guru, S., Kim, M. J., & Chang, S. D. (2025) · Brain Sciences · doi:10.3390/brainsci15080896 · Cited by 10 · see citing works
Enhanced Survival of High-Risk Medulloblastoma-Bearing Mice after Multimodal Treatment with Radiotherapy, Decitabine, and Abacavir
Gringmuth, M., Walther, J., Greiser, S., Toussaint, M., Schwalm, B., Kool, M., Kortmann, R., Glasow, A., & Patties, I. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms23073815 · Cited by 9 · see citing works
Review of the impact of molecular analysis on the therapy of medulloblastoma
Sarvode, S., & Gajjar, A. J. (2023) · Pediatric Hematology Oncology Journal · doi:10.1016/j.phoj.2023.05.001 · Cited by 9 · see citing works
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Subtyping-Directed Precision Treatment Refines Traditional One-Size-Fits-All Therapy for HR+/HER2− Breast Cancer
Zhu, X.-Z., Zhang, H.-Y.-L., Zhou, Y.-F., Chen, Y.-Y., Fu, T., Jin, M.-L., Zhao, Y.-X., Xie, Y.-F., Zeng, X., Jin, X., et al. (2025) · Cancer Research · doi:10.1158/0008-5472.can-24-5002 · Cited by 8 · see citing works
An integrated analysis of three medulloblastoma clinical trials refines risk-stratification approaches for reducing toxicity and improving survival
Smith, K. S., Dhanda, S. K., Billups, C. A., Sioson, E., Lu, C., Peraza, A. Z., Gangwani, K., Li, Y., Li, Q., Lin, T., et al. (2025) · Neuro-Oncology · doi:10.1093/neuonc/noaf250 · Cited by 8 · see citing works
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How do we approach the management of medulloblastoma in young children?
Lafay‐Cousin, L., Baroni, L., Ramaswamy, V., & Bouffet, É. (2022) · Pediatric Blood & Cancer · doi:10.1002/pbc.29838 · Cited by 9 · see citing works
Identification and validation of a novel HOX-related classifier signature for predicting prognosis and immune microenvironment in pediatric gliomas
Zhang, J., Zhang, X., Su, J., Zhang, J., Liu, S., Li, H., Liu, M., & Sun, D. (2023) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2023.1203650 · Cited by 7 · see citing works
Temporal changes in childhood cancer incidence and survival by stage at diagnosis in Australia, 2000–2017
Youlden, D. R., Baade, P. D., Frazier, A. L., Gupta, S., Gottardo, N. G., Moore, A. S., & Aitken, J. F. (2023) · Acta Oncologica · doi:10.1080/0284186x.2023.2251668 · Cited by 7 · see citing works
Long-term outcome of the Milano-hyperfractionated accelerated radiotherapy strategy for high-risk medulloblastoma, including the impact of molecular subtype
Massimino, M., Barretta, F., Dossena, C., Minasi, S., Buttarelli, F. R., Biassoni, V., Oriani, M., Schiavello, E., Ficorilli, M., Nigro, O., et al. (2024) · Neuro-Oncology · doi:10.1093/neuonc/noae189 · Cited by 7 · see citing works
Revised clinical and molecular risk strata define the incidence and pattern of failure in medulloblastoma following risk-adapted radiotherapy and dose-intensive chemotherapy: results from a phase III multi-institutional study
Lucas, J. T., Tinkle, C. L., Huang, J., Onar‐Thomas, A., Srinivasan, S., Tumlin, P., Becksfort, J. B., Klimo, P. D., Boop, F. A., Robinson, G. W., et al. (2021) · Neuro-Oncology · doi:10.1093/neuonc/noab284 · Cited by 7 · see citing works
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Questions and answers in the management of children with medulloblastoma over the time. How did we get here? A systematic review
Osuna‐Marco, M. P., Martín‐López, L. I., Tejera, Á. M., & López‐Ibor, B. (2023) · Frontiers in Oncology · doi:10.3389/fonc.2023.1229853 · Cited by 7 · see citing works
Targeting synthetic lethality between non-homologous end joining and radiation in very-high-risk medulloblastoma
DeCarlo, A., MacLeod, G. D. A. H., da Silva, C. F., Shen, L., Povilaikaite, J., Deane, M., Aragao, L., Sie, M., Termini, D., Magee, J., et al. (2025) · Cell Reports Medicine · doi:10.1016/j.xcrm.2025.102202 · Cited by 6 · see citing works
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Medulloblastoma: Current Standard of Care and Future Treatment Opportunities
Mayr, L., Azizi, A. A., Gojo, J., & Peyrl, A. (2025) · Pediatric Drugs · doi:10.1007/s40272-025-00726-1 · Cited by 6 · see citing works
Systematic transcriptomic analysis of childhood medulloblastoma identifies N6-methyladenosine-dependent lncRNA signatures associated with molecular subtype, immune cell infiltration, and prognosis
Joshi, K., Yuan, M., Katsushima, K., Saulnier, O., Ray, A., Amankwah, E. K., Stapleton, S. L., Jallo, G. I., Taylor, M. D., Eberhart, C. G., et al. (2024) · Acta Neuropathologica Communications · doi:10.1186/s40478-024-01848-2 · Cited by 6 · see citing works
Medulloblastoma Molecular Subgrouping and Outcomes Data of a Single Center From a Low‐ and Middle‐Income Country
Mushtaq, N., Bashir, F., Zahid, S., Riaz, Q., Javed, G., Tariq, M., Qureshi, B. M., Hilal, K., Ramaswamy, V., Hawkins, C. E., et al. (2025) · Pediatric Blood & Cancer · doi:10.1002/pbc.31555 · Cited by 6 · see citing works
Therapeutic radiation drives leptomeningeal dissemination of medulloblastoma through an innate immune process
Nör, C., Kharas, K., Rasnitsyn, A., Vladoiu, M. C., Cho, N. W., Young, J. S., Nör, F., Mankahla, N., Haapasalo, J. A., Nordfors, K., et al. (2025) · Developmental Cell · doi:10.1016/j.devcel.2025.06.016 · Cited by 7 · see citing works
Effects of different molecular subtypes and tumor biology on the prognosis of medulloblastoma
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Clinico-Radiological Outcomes in WNT-Subgroup Medulloblastoma
Mani, S., Chatterjee, A., Dasgupta, A., Shirsat, N. V., Pawar, A., Epari, S., Sahay, A., Sahu, A., Moiyadi, A. V., Prasad, M., et al. (2024) · Diagnostics · doi:10.3390/diagnostics14040358 · Cited by 5 · see citing works
Targeting processive transcription for Myc-driven circuitry in medulloblastoma
Sobral, L. M., Walker, F. M., Madhavan, K. P. C., Janko, E., Donthula, S., Danis, E. P., Bompada, P., Balakrishnan, I., Wang, D., Pierce, A. M., et al. (2025) · Neuro-Oncology · doi:10.1093/neuonc/noaf121 · Cited by 5 · see citing works
Current Open Trials and Molecular Update for Pediatric Embryonal Tumors
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Prognostic significance of molecular subgroups in survival outcome for children with medulloblastoma in Malaysia
Rajagopal, R., Teng, A. J., Jawin, V., Wong, O. L., Mahsin, H., Rani, N. H. A., Yap, T. Y., Gunasagaran, K., Thevarajah, A., Yeoh, S. L., et al. (2023) · Frontiers in Oncology · doi:10.3389/fonc.2023.1278611 · Cited by 5 · see citing works
Long-term Outcomes of Protocol-Based Treatment for Newly Diagnosed Medulloblastoma
Ahn, W. K., Hahn, S. M., Yoon, H. I., Lee, J., Park, E. K., Shim, K., Kim, D. S., Suh, C., Kim, S. H., Lyu, C. J., et al. (2023) · Cancer Research and Treatment · doi:10.4143/crt.2023.865 · Cited by 5 · see citing works
Risk-stratification for treatment de-intensification in WNT-pathway medulloblastoma: finding the optimal balance between survival and quality of survivorship
Gupta, T., Mani, S., Chatterjee, A., Dasgupta, A., Epari, S., & Chinnaswamy, G. (2024) · Expert Review of Anticancer Therapy · doi:10.1080/14737140.2024.2357807 · Cited by 5 · see citing works
Infant brain tumor trials: Beyond feasibility
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When Can We Retire 3,600 cGy Craniospinal Irradiation in Medulloblastoma?
Fisher, P. G. (2023) · Journal of Clinical Oncology · doi:10.1200/jco.23.00008 · Cited by 5 · see citing works
Medulloblastoma Spatial Transcriptomics Reveals Tumor Microenvironment Heterogeneity with High-Density Progenitor Cell Regions Correlating with High-Risk DiseasePreprint
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Integration of immunohistochemistry, RNA sequencing, and multiplex ligation‐dependent probe amplification for molecular classification of pediatric medulloblastoma
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B7-H6 enhances F-actin rearrangement by targeting c-MYC activation to promote medulloblastoma migration and invasion
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Treatment of Medulloblastoma in the Adolescent and Young Adult Population: A Systematic Review
Otth, M., Weiser, A., Lee, S.-Y., von Rohr, L. R., Heesen, P., Stücklin, A. G., & Scheinemann, K. (2024) · Journal of Adolescent and Young Adult Oncology · doi:10.1089/jayao.2024.0044 · Cited by 4 · see citing works
The Current Landscape of Molecular Pathology for the Diagnosis and Treatment of Pediatric Medulloblastoma
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Carboplatin During Craniospinal Radiotherapy for Children With Group 3 Medulloblastoma—A New Standard of Care?—Reply
Leary, S. E. S., Li, Y., & Olson, J. M. (2021) · JAMA Oncology · doi:10.1001/jamaoncol.2021.6526 · Cited by 4 · see citing works
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Impact of radiation response on survival in pediatric medulloblastoma with residual or disseminated disease
Shi, X., Sun, X., Fan, W., Dai, X., & Jiang, M. (2025) · Radiation Oncology · doi:10.1186/s13014-025-02632-9 · Cited by 3 · see citing works
Prior therapy defines mutation profiles in childhood cancer at relapse
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Successful Implementation of Image-Guided Pencil-Beam Scanning Proton Therapy in Medulloblastomas
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Children's Oncology Group's 2023 blueprint for research: Radiation oncology
Kalapurakal, J. A., Wolden, S. L., Haas-Kogan, D. A., Laack, N. N., Hua, C., Paulino, A. C., Hill‐Kayser, C. E., Hoppe, B. S., & FitzGerald, T. J. (2023) · Pediatric Blood & Cancer · doi:10.1002/pbc.30593 · Cited by 3 · see citing works
Fatal brainstem injury following proton radiation in a patient with medulloblastoma and a germline variant in RNF213
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Outcomes following radiation therapy for embryonal tumor with multilayered rosettes (ETMR): results from the Pediatric Proton/Photon Consortium Registry (PPCR)
Krisch, J. M., Ermoian, R. P., Indelicato, D. J., Lee, J. Y., Perentesis, J. P., Perkins, S. M., Laack, N. N., Chang, J. H. C., MacEwan, I. J., Wolden, S. L., et al. (2025) · Journal of Neuro-Oncology · doi:10.1007/s11060-025-05065-2 · Cited by 3 · see citing works
A cycling, progenitor-like cell population at the base of atypical teratoid rhabdoid tumor subtype differentiation trajectories
Blanco-Carmona, E., Paassen, I., He, J., DeMartino, J., Büllesbach, A., Anderson, N., Buhl, J. L., Federico, A., Mauermann, M., Brok, M. O., et al. (2025) · Neuro-Oncology · doi:10.1093/neuonc/noaf179 · Cited by 3 · see citing works
Management of pediatric brain tumors in low- and middle-income countries
Amayiri, N. Y., Mushtaq, N., Ghalibafian, M., & Bouffet, É. (2025) · Advances in cancer research · doi:10.1016/bs.acr.2025.08.004 · Cited by 2 · see citing works
GPC2-CAR T cells have potent preclinical activity against orthotopic medulloblastoma xenografts
Okada, R., Fanuzzi, M., Rodríguez, C., Oh, J., Sreenivasan, A., Stack, H. G., Puebla, M., Phadke, I., Cole, A. P., Bradford, S. Y. C., et al. (2025) · Molecular Therapy Oncology · doi:10.1016/j.omton.2025.201067 · Cited by 3 · see citing works
Surgical management and outcome of medulloblastoma patients at Addis Ababa between 2010 and 2018
Akililu, Y. B., Bouffet, É., & Abebe, M. (2025) · Frontiers in Oncology · doi:10.3389/fonc.2025.1672757 · Cited by 2 · see citing works
Medulloblastoma in Adolescents and Young Adults: Molecular Subgroups, Prognostic Biomarkers, and Age-Specific Therapeutic Challenges
Ruggiero, A., Gessi, M., d’Amati, A., Albanese, A., & Attinà, G. (2026) · Current Issues in Molecular Biology · doi:10.3390/cimb48030297 · Cited by 2 · see citing works
YAP1–MAML2 Fusion in Young Children With Pediatric High‐Grade Glioma: A Case Report
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Isotretinoin as a Multifunctional Anticancer Agent: Molecular Mechanisms, Pharmacological Insights and Therapeutic Potential
Sarkar, P., Sultana, N., Debnath, P. K., Rouf, R., Mubarak, M. S., Uddin, S. J., Popa, D., Călina, D. C., & Sharifi‐Rad, J. (2025) · Archiv der Pharmazie · doi:10.1002/ardp.70084 · Cited by 3 · see citing works
Molecular and clinical correlates of medulloblastoma subgroups
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Genetic alterations of TP53 and OTX2 indicate increased risk of relapse in WNT medulloblastomas: “it’s a numbers game”—implications for WNT medulloblastoma dose-reduction clinical trials
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International experience by using of the SJMB protocols in treatment of children with medulloblastoma in the age group over 3 years (literature review)
Levashov, A., Рустамовна, З. С., Valiev, T. T., Строганова, А. М., Khochenkov, D. A., Grigorenko, V. A., Михайлова, Е. В., Тюрина, E. В., Ryzhova, M. V., Horielyshev, S. K., et al. (2023) · Journal of Modern Oncology · doi:10.26442/18151434.2023.3.202437 · Cited by 2 · see citing works
The Medulloblastoma Meta-Analysis Portal Enables Integrative Exploration of Clinical and Genomic Data from Patients
Gangwani, K., Lu, C., Dhanda, S. K., Sioson, E., Zaldívar Peraza, A., Matt, G. Y., Paul, R., Wang, J., Reilly, C., Acić, A., et al. (2025) · Cancer Research · doi:10.1158/0008-5472.can-24-4976 · Cited by 1 · see citing works
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You cannot diagnose what you cannot see: The importance of optimized imaging protocols for accurate disease staging and assessment
Gottardo, N. G., Fouladi, M., & Shaw, D. (2023) · Pediatric Blood & Cancer · doi:10.1002/pbc.30552 · Cited by 1 · see citing works
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Isotretinoin has no effect on event-free survival across high-risk medulloblastoma molecular groups when added to maintenance: A secondary analysis of the Children's Oncology Group ACNS0332 data
Raskin, S. I., de Blank, P. M. K., Billups, C. A., Li, Y., Olson, J. M., & Leary, S. E. S. (2025) · Neuro-Oncology Advances · doi:10.1093/noajnl/vdaf054 · Cited by 1 · see citing works
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Feasibility and Outcomes of Craniospinal Irradiation and Concurrent Daily Carboplatin in Children and Adults With High-risk Central Nervous System Tumours
Singer, R., Lorimer, C., Welsh, L. C., Carceller, F., Vaidya, S. J., Marshall, L. V., Stone, J. R., Evans, R., O’Leary, B., Zacharoulis, S., et al. (2025) · Clinical Oncology · doi:10.1016/j.clon.2025.103866 · Cited by 1 · see citing works
Cerebrospinal fluid liquid biopsy guides differential diagnosis of relapsed medulloblastoma versus secondary glioma: A case report of a pediatric patient enrolled on a PDGFRA inhibitor trial
Fischer, T. T., Maaß, K. K., Schoof, M., Puranachot, P., Sievers, P., Montigel, S. H., Fluhr, T. L., Damaty, A. E., Seitz, A., Harrabi, S., et al. (2025) · Neuro-Oncology Advances · doi:10.1093/noajnl/vdaf110 · Cited by 1 · see citing works
Old Tools in a New Era: The Continued Relevance of Chemotherapy in Pediatric Neuro-Oncology
Felton, K. E. A., Lafay‐Cousin, L., & Cheng, S. S. (2025) · Current Oncology · doi:10.3390/curroncol32070410 · Cited by 1 · see citing works
Challenges and opportunities for treatment of medulloblastoma
Mehta, M., Somasundaram, D. B., Munshi, A., & Ramesh, R. (2025) · Advances in cancer research · doi:10.1016/bs.acr.2025.06.006 · Cited by 1 · see citing works
Severe Hearing Loss in Children With Central Nervous System Tumors: A Population‐Based Cancer in Young People in Canada (CYP‐C) Report
Coltin, H., Trudeau-Ferrin, O., Perreault, S., Lafay‐Cousin, L., Tsang, D. S., Renzi, S., Larouche, V., Erker, C. A., Hukin, J., Barber, R., et al. (2025) · Pediatric Blood & Cancer · doi:10.1002/pbc.32024 · Cited by 1 · see citing works
Exploring deep learning and hybrid approaches in molecular subgrouping and prognostic-related genetic signatures of medulloblastoma
Li, Y., Liu, H., Liu, Y., Li, J., Suzuki, H. H., Liu, Y., Jiang, T., & Qiu, X. (2025) · Chinese Neurosurgical Journal · doi:10.1186/s41016-025-00405-7 · Cited by 1 · see citing works
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The potential and risks of FLASH radiotherapy in pediatric patients
Schuemann, J., Rothwell, B., Mascia, A. E., Fan, Y., Loo, B. W., Yock, T. I., Liu, K. X., Perentesis, J. P., & Haas-Kogan, D. A. (2025) · Neuro-Oncology Pediatrics · doi:10.1093/neuped/wuaf014 · Cited by 1 · see citing works
High-dose chemotherapy and craniospinal irradiation-sparing approach for WNT medulloblastoma of early childhood
Nobre, L. F., Ramaswamy, V., Coleman, C., Dhall, G., Senapati, A., Toledano, H., Jabado, N., Bouffet, É., & Lafay‐Cousin, L. (2026) · Journal of Neuro-Oncology · doi:10.1007/s11060-026-05425-6 · Cited by 1 · see citing works

MBCL-16. EFFICACY OF CARBOPLATIN GIVEN CONCOMITANTLY WITH RADIATION AND ISOTRETINOIN AS A PRO-APOPTOTIC AGENT IN MAINTENANCE THERAPY IN HIGH-RISK MEDULLOBLASTOMA: A REPORT FROM THE CHILDREN’S ONCOLOGY GROUP
Leary, S. E. S., Packer, R. J., Jaju, A. I., Heier, L., Burger, P. C., Smith, K. S., Michalski, J. M., Li, Y., Billups, C. A., Hwang, E. I., et al. (2020) · Neuro-Oncology · doi:10.1093/neuonc/noaa222.492 · Cited by 2 · see citing works
Self-citation
Isotretinoin has no effect on event-free survival across high-risk medulloblastoma molecular groups when added to maintenance: A secondary analysis of the Children's Oncology Group ACNS0332 data
Raskin, S. I., de Blank, P. M. K., Billups, C. A., Li, Y., Olson, J. M., & Leary, S. E. S. (2025) · Neuro-Oncology Advances · doi:10.1093/noajnl/vdaf054 · Cited by 1 · see citing works
Self-citation
MDB-86. ISOTRETINOIN HAS NO EFFECT ON EVENT FREE SURVIVAL ACROSS HIGH-RISK MEDULLOBLASTOMA MOLECULAR SUBGROUPS WHEN ADDED TO MAINTENANCE CHEMOTHERAPY: A SECONDARY ANALYSIS OF THE CHILDREN’S ONCOLOGY GROUP ACNS0332 DATA
Raskin, S. I., de Blank, P. M. K., Billups, C. A., Li, Y., Olson, J. M., & Leary, S. E. S. (2024) · Neuro-Oncology · doi:10.1093/neuonc/noae064.534
Self-citation
Risk-adapted therapy for young children with medulloblastoma (SJYC07): therapeutic and molecular outcomes from a multicentre, phase 2 trial
Robinson, G. W., Rudneva, V. A., Buchhalter, I., Billups, C. A., Waszak, S. M., Smith, K. S., Bowers, D. C., Bendel, A. E., Fisher, P. G., Partap, S., et al. (2018) · The Lancet Oncology · doi:10.1016/s1470-2045(18)30204-3 · Cited by 238 · see citing works
Self-citation
Survival of patients with medulloblastoma treated with carboplatin and etoposide before and after radiotherapy.
López‐Aguilar, E., Ac, S.-V., Rivera-Márquez, H., Cerecedo-Dı́az, F., Santacruz-Castillo, E., Valdéz-Sánchez, M., Arias-Gómez, J., & Quintana-Roldán, G. (1998) · PubMed · Cited by 8 · see citing works

Authors: Leary, S. E. S., Packer, R. J., Li, Y., Billups, C. A., Smith, K. S., Jaju, A., Heier, L., Burger, P., Walsh, K., Han, Y., Embry, L., Hadley, J., Kumar, R., Michalski, J., Hwang, E., Gajjar, A., Pollack, I. F., Fouladi, M., Northcott, P. A., & Olson, J. M.
Affiliations: Seattle Children's Hospital (US)
Children's National (US)
St. Jude Children's Research Hospital (US)
Lurie Children's Hospital (US)
Weill Cornell Medicine (US)
Johns Hopkins University (US)
The University of Texas at San Antonio Health Science Center (US)
Washington University in St. Louis (US)
Children's Hospital of Pittsburgh (US)
Nationwide Children's Hospital (US)
Corresponding author: Leary, Sarah E. S.
ORCID: Leary, Sarah E. S.
Packer, Roger J.
Li, Yimei
Billups, Catherine A.
Smith, Kyle S.
Jaju, Alok
Heier, Linda
Burger, Peter
Walsh, Karin
Han, Yuanyuan
Embry, Leanne
Hadley, Jennifer
Kumar, Rahul
Michalski, Jeff
Hwang, Eugene
Gajjar, Amar
Pollack, Ian F.
Fouladi, Maryam
Northcott, Paul A.
Olson, James M.
Venue: JAMA Oncology
Publisher: American Medical Association (AMA)
Published: 2021-09-01
Volume: 7
Issue: 9
Pages: 1313
ISSN: 2374-2437 (ISSN-L)
DOI: 10.1001/jamaoncol.2021.2224
PMC: PMC8299367
OpenAlex: W3184618654
Cited by: 164
References: 34
Citations by year: 2026: 25 · 2025: 50 · 2024: 27 · 2023: 34 · 2022: 17 · 2021: 11
FWCI: 12.90
Type: journal article
Language: en
Open access: bronze
doi.org
Locations: JAMA Oncology (publishedVersion)
PubMed Central (publishedVersion)
PubMed (publishedVersion)
Funders: Brain Tumour Charity (GN-000575)
National Cancer Institute (R01CA114567, U10CA180899, U10CA180886)
Topics: Health Sciences › Medicine › Genetics › Glioma Diagnosis and Treatment
Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Acute Lymphoblastic Leukemia research
Health Sciences › Medicine › Neurology › Neuroblastoma Research and Treatments
Keywords: high-risk medulloblastoma, carboplatin, isotretinoin, radiosensitization, event-free survival, overall survival, DNA methylation, pediatric brain tumors, maintenance chemotherapy, futility, metastatic disease, residual disease
MeSH terms: Adolescent, Adult, Antineoplastic Combined Chemotherapy Protocols, Cerebellar Neoplasms, Child, Child, Preschool, Humans, Male, Medulloblastoma, Isotretinoin, Carboplatin, Disease-Free Survival, Young Adult

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Efficacy of Carboplatin and Isotretinoin in Children With High-risk Medulloblastoma: A Randomized Clinical Trial From the Children’s Oncology Group

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