{"doi":"10.1200/jco.20.02262","title":"Long-Term Follow-Up of CD19-CAR T-Cell Therapy in Children and Young Adults With B-ALL","abstract":"PURPOSE CD19 chimeric antigen receptor (CD19-CAR) T cells induce high response rates in children and young adults (CAYAs) with B-cell acute lymphoblastic leukemia (B-ALL), but relapse rates are high. The role for allogeneic hematopoietic stem-cell transplant (alloHSCT) following CD19-CAR T-cell therapy to improve long-term outcomes in CAYAs has not been examined. METHODS We conducted a phase I trial of autologous CD19.28ζ-CAR T cells in CAYAs with relapsed or refractory B-ALL. Response and long-term clinical outcomes were assessed in relation to disease and treatment variables. RESULTS Fifty CAYAs with B-ALL were treated (median age, 13.5 years; range, 4.3-30.4). Thirty-one (62.0%) patients achieved a complete remission (CR), 28 (90.3%) of whom were minimal residual disease−negative by flow cytometry. Utilization of fludarabine/cyclophosphamide–based lymphodepletion was associated with improved CR rates (29/42, 69%) compared with non–fludarabine/cyclophosphamide–based lymphodepletion (2/8, 25%; P = .041). With median follow-up of 4.8 years, median overall survival was 10.5 months (95% CI, 6.3 to 29.2 months). Twenty-one of 28 (75.0%) patients achieving a minimal residual disease−negative CR proceeded to alloHSCT. For those proceeding to alloHSCT, median overall survival was 70.2 months (95% CI, 10.4 months to not estimable). The cumulative incidence of relapse after alloHSCT was 9.5% (95% CI, 1.5 to 26.8) at 24 months; 5-year EFS following alloHSCT was 61.9% (95% CI, 38.1 to 78.8). CONCLUSION We provide the longest follow-up in CAYAs with B-ALL after CD19-CAR T-cell therapy reported to date and demonstrate that sequential therapy with CD19.28ζ-CAR T cells followed by alloHSCT can mediate durable disease control in a sizable fraction of CAYAs with relapsed or refractory B-ALL (ClinicalTrials.gov identifier: NCT01593696 ).","journal":"Journal of Clinical Oncology","year":2021,"id":145586,"datarank":6.909955423523361,"base_score":5.8944028342648505,"endowment":5.8944028342648505,"self_citation_contribution":0.8841604251397277,"citation_network_contribution":6.025794998383634,"self_endowment_contribution":0.8841604251397277,"citer_contribution":6.025794998383634,"corpus_percentile":null,"corpus_rank":null,"citation_count":362,"citer_count":100,"citers_with_citation_signal":100,"citers_with_endowment":100,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9576,"is_data_producer":true,"deposit_databanks":{"ClinicalTrials.gov":["NCT01593696"]},"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2021-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":439622,"name":"Daniel W. Lee","orcid":"0000-0002-3249-9796","position":1,"is_corresponding":false},{"id":283310,"name":"Bonnie Yates","orcid":null,"position":2,"is_corresponding":false},{"id":278259,"name":"Constance M. Yuan","orcid":"0000-0002-2601-3665","position":3,"is_corresponding":false},{"id":280937,"name":"Haneen Shalabi","orcid":"0000-0001-8692-8034","position":4,"is_corresponding":false},{"id":109189,"name":"Staci Martin","orcid":"0000-0002-9342-4173","position":5,"is_corresponding":false},{"id":109180,"name":"Pamela L. Wolters","orcid":"0000-0001-9319-6281","position":6,"is_corresponding":false},{"id":109203,"name":"Seth M. Steinberg","orcid":"0000-0002-8280-551X","position":7,"is_corresponding":false},{"id":308552,"name":"Eva H. Baker","orcid":"0000-0001-7347-2170","position":8,"is_corresponding":false},{"id":283313,"name":"Cindy Delbrook","orcid":null,"position":9,"is_corresponding":false},{"id":278258,"name":"Maryalice Stetler‐Stevenson","orcid":"0000-0003-2591-0353","position":10,"is_corresponding":false},{"id":309028,"name":"Terry J. Fry","orcid":"0000-0001-8044-5226","position":11,"is_corresponding":false},{"id":233604,"name":"David F. Stroncek","orcid":"0000-0001-5867-3265","position":12,"is_corresponding":false},{"id":225351,"name":"Crystal L. Mackall","orcid":"0000-0003-0359-9023","position":13,"is_corresponding":false},{"id":280941,"name":"Nirali N. Shah","orcid":"0000-0002-8474-9080","position":0,"is_corresponding":true}],"reference_count":32,"raw_metadata":null,"created_at":"2026-07-18T23:42:12.871665Z","pmid":"33764809","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":[]}