{"doi":"10.1093/neuonc/noaa102","title":"Excellent outcome of young children with nodular desmoplastic medulloblastoma treated on “Head Start” III: a multi-institutional, prospective clinical trial","abstract":"BACKGROUND: \"Head Start\" III, was a prospective clinical trial using intensive induction followed by myeloablative chemotherapy and autologous hematopoietic cell rescue (AuHCR) to either avoid or reduce the dose/volume of irradiation in young children with medulloblastoma. METHODS: Following surgery, patients received 5 cycles of induction followed by myeloablative chemotherapy using carboplatin, thiotepa, and etoposide with AuHCR. Irradiation was reserved for children >6 years old at diagnosis or with residual tumor post-induction. RESULTS: Between 2003 and 2009, 92 children <10 years old with medulloblastoma were enrolled. Five-year event-free survival (EFS) and overall survival (OS) rates (±SE) were 46 ± 5% and 62 ± 5% for all patients, 61 ± 8% and 77 ± 7% for localized medulloblastoma, and 35 ± 7% and 52 ± 7% for disseminated patients. Nodular/desmoplastic (ND) medulloblastoma patients had 5-year EFS and OS (±SE) rates of 89 ± 6% and 89 ± 6% compared with 26 ± 6% and 53 ± 7% for classic and 38 ± 13% and 46 ± 14% for large-cell/anaplastic (LCA) medulloblastoma, respectively. In multivariate Cox regression analysis, histology was the only significant independent predictor of EFS after adjusting for stage, extent of resection, regimen, age, and sex (P <0.0001). Five-year irradiation-free EFS was 78 ± 8% for ND and 21 ± 5% for classic/LCA medulloblastoma patients. Myelosuppression was the most common toxicity, with 2 toxic deaths. Twenty-four survivors completed neurocognitive evaluation at a mean of 4.9 years post-diagnosis. IQ and memory scores were within average range overall, whereas processing speed and adaptive functioning were low-average. CONCLUSION: We report excellent survival and preservation of mean IQ and memory for young children with ND medulloblastoma using high-dose chemotherapy, with most patients surviving without irradiation.","journal":"Neuro-Oncology","year":2020,"id":54835,"datarank":0.692268077526189,"base_score":4.61512051684126,"endowment":4.61512051684126,"self_citation_contribution":0.692268077526189,"citation_network_contribution":0.0,"self_endowment_contribution":0.692268077526189,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":100,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9561,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2020-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":282345,"name":"Sharon O’Neil","orcid":"0000-0001-5043-4796","position":1,"is_corresponding":false},{"id":282346,"name":"Lingyun Ji","orcid":"0000-0003-2500-1993","position":2,"is_corresponding":false},{"id":283666,"name":"Kelley Haley","orcid":null,"position":3,"is_corresponding":false},{"id":282347,"name":"Ashley M. Whitaker","orcid":"0000-0002-4362-6295","position":4,"is_corresponding":false},{"id":282348,"name":"Marvin D. Nelson","orcid":"0000-0002-8038-8046","position":5,"is_corresponding":false},{"id":282349,"name":"Floyd H. Gilles","orcid":"0000-0002-5426-3549","position":6,"is_corresponding":false},{"id":282350,"name":"Sharon L. Gardner","orcid":"0000-0002-8857-5487","position":7,"is_corresponding":false},{"id":282351,"name":"Jeffrey C. Allen","orcid":"0000-0001-9957-6664","position":8,"is_corresponding":false},{"id":283667,"name":"Albert Cornelius","orcid":null,"position":9,"is_corresponding":false},{"id":282352,"name":"Kamnesh R. Pradhan","orcid":"0000-0002-1488-5992","position":10,"is_corresponding":false},{"id":282353,"name":"James H. Garvin","orcid":"0000-0003-4709-1088","position":11,"is_corresponding":false},{"id":282354,"name":"Randal Olshefski","orcid":"0000-0002-6035-2695","position":12,"is_corresponding":false},{"id":240509,"name":"Juliette Hukin","orcid":"0000-0002-8727-8456","position":13,"is_corresponding":false},{"id":283668,"name":"Melanie Comito","orcid":null,"position":14,"is_corresponding":false},{"id":260745,"name":"Stewart Goldman","orcid":"0000-0003-0134-1352","position":15,"is_corresponding":false},{"id":283669,"name":"Mark Atlas","orcid":null,"position":16,"is_corresponding":false},{"id":282355,"name":"Andrew W. Walter","orcid":"0000-0001-5653-5686","position":17,"is_corresponding":false},{"id":282356,"name":"Stephen A. Sands","orcid":"0000-0003-4550-263X","position":18,"is_corresponding":false},{"id":282357,"name":"Richard Sposto","orcid":"0000-0002-2729-2360","position":19,"is_corresponding":false},{"id":282358,"name":"Jonathan L. Finlay","orcid":"0000-0002-8312-6586","position":20,"is_corresponding":false},{"id":282344,"name":"Girish Dhall","orcid":"0000-0002-4918-0167","position":0,"is_corresponding":true}],"reference_count":48,"raw_metadata":{"citation_network_status":"fetched"},"created_at":"2026-07-18T21:04:14.743014Z","pmid":"32304218","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}