{"doi":"10.1182/blood.2022015959","title":"KIR-favorable TCR-αβ/CD19-depleted haploidentical HCT in children with ALL/AML/MDS: primary analysis of the PTCTC ONC1401 trial","abstract":"We performed a prospective multicenter study of T-cell receptor αβ (TCR-αβ)/CD19-depleted haploidentical hematopoietic cell transplantation (HCT) in children with acute leukemia and myelodysplastic syndrome (MDS), to determine 1-year disease-free survival (DFS) and compare 2-year outcomes with recipients of other donor cell sources. Fifty-one patients aged 0.7 to 21 years were enrolled; donors were killer immunoglobulin-like receptor (KIR) favorable based on ligand mismatch and/or high B content. The 1-year DFS was 78%. Superior 2-year DFS and overall survival (OS) were noted in patients <10 years of age, those treated with reduced toxicity conditioning (RTC) rather than myeloablative conditioning, and children with minimal residual disease <0.01% before HCT. Multivariate analysis comparing the KIR-favorable haploidentical cohort with controls showed similar DFS and OS compared with other donor cell sources. Multivariate analysis also showed a marked decrease in the risk of grades 2 to 4 and 3 to 4 acute graft versus host disease (aGVHD), chronic GVHD, and transplant-related mortality vs other donor cell sources. Ethnic and racial minorities accounted for 53% of enrolled patients, and data from a large cohort of recipients/donors screened for KIR showed that >80% of recipients had a KIR-favorable donor by our definition, demonstrating that this approach is broadly applicable to groups often unable to find donors. This prospective, multicenter study showed improved outcomes using TCR-αβ/CD19-depleted haploidentical donors using RTC for children with acute leukemia and MDS. Randomized trials comparing this approach with matched unrelated donors are warranted. This trial was registered at https://clinicaltrials.gov as #NCT02646839.","journal":"Blood","year":2022,"id":249407,"datarank":0.8647320845005134,"base_score":3.367295829986474,"endowment":3.367295829986474,"self_citation_contribution":0.5050943744979712,"citation_network_contribution":0.3596377100025422,"self_endowment_contribution":0.5050943744979712,"citer_contribution":0.3596377100025422,"corpus_percentile":null,"corpus_rank":null,"citation_count":28,"citer_count":16,"citers_with_citation_signal":10,"citers_with_endowment":10,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.905,"is_data_producer":true,"deposit_databanks":{"ClinicalTrials.gov":["NCT02646839"]},"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2022-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":284114,"name":"Kwang Woo Ahn","orcid":"0000-0003-4567-8037","position":1,"is_corresponding":false},{"id":323428,"name":"Nancy Bunin","orcid":"0000-0001-6210-2262","position":2,"is_corresponding":false},{"id":674174,"name":"Nahal Lalefar","orcid":"0000-0002-5651-7426","position":3,"is_corresponding":false},{"id":889672,"name":"Eric Anderson","orcid":"0000-0003-3665-3102","position":4,"is_corresponding":false},{"id":313189,"name":"Allyson Flower","orcid":null,"position":5,"is_corresponding":false},{"id":313711,"name":"Mitchell S. Cairo","orcid":"0000-0002-2075-434X","position":6,"is_corresponding":false},{"id":890062,"name":"Julie-An Talano","orcid":null,"position":7,"is_corresponding":false},{"id":263438,"name":"Sonali Chaudhury","orcid":"0000-0001-9816-5126","position":8,"is_corresponding":false},{"id":312099,"name":"Carrie L. Kitko","orcid":"0000-0001-9216-2901","position":9,"is_corresponding":false},{"id":312426,"name":"Jamie L. Duke","orcid":"0000-0002-4768-0846","position":10,"is_corresponding":false},{"id":454180,"name":"Dimitrios Monos","orcid":null,"position":11,"is_corresponding":false},{"id":490736,"name":"Wing Leung","orcid":"0000-0002-2996-7229","position":12,"is_corresponding":false},{"id":263437,"name":"Christopher C. Dvorak","orcid":"0000-0002-6146-3952","position":13,"is_corresponding":false},{"id":249842,"name":"Hisham Abdel‐Azim","orcid":"0000-0003-0880-8701","position":14,"is_corresponding":false},{"id":263463,"name":"Michael A. Pulsipher","orcid":"0000-0003-3030-8420","position":0,"is_corresponding":true}],"reference_count":43,"raw_metadata":null,"created_at":"2026-07-19T00:24:19.356023Z","pmid":"35776909","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":[]}