{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,17]],"date-time":"2026-10-17T06:50:55Z","timestamp":1792219855207,"version":"4.4.0"},"reference-count":40,"publisher":"American Society of Hematology","issue":"24","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2022,12,15]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>We performed a prospective multicenter study of T-cell receptor \u03b1\u03b2 (TCR-\u03b1\u03b2)\/CD19\u2013depleted 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 &amp;lt;10 years of age, those treated with reduced toxicity conditioning (RTC) rather than myeloablative conditioning, and children with minimal residual disease &amp;lt;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 &amp;gt;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-\u03b1\u03b2\/CD19\u2013depleted 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.<\/jats:p>","DOI":"10.1182\/blood.2022015959","type":"journal-article","created":{"date-parts":[[2022,7,1]],"date-time":"2022-07-01T20:01:10Z","timestamp":1656705670000},"page":"2556-2572","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":31,"title":["KIR-favorable TCR-\u03b1\u03b2\/CD19-depleted haploidentical HCT in children with ALL\/AML\/MDS: primary analysis of the PTCTC ONC1401 trial"],"prefix":"10.1182","volume":"140","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3030-8420","authenticated-orcid":false,"given":"Michael A.","family":"Pulsipher","sequence":"first","affiliation":[{"name":"1Division of Hematology and Oncology, Intermountain Primary Children\u2019s Hospital, Huntsman Cancer Institute at the University of Utah Spencer Fox Eccles School of Medicine, Salt Lake City, UT"},{"name":"2Section of Transplantation and Cellular Therapy, Cancer 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CA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3665-3102","authenticated-orcid":false,"given":"Eric","family":"Anderson","sequence":"additional","affiliation":[{"name":"7Rady Children\u2019s Hospital San Diego and UC San Diego School of Medicine, San Diego, CA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Allyson","family":"Flower","sequence":"additional","affiliation":[{"name":"8Medical College of New York, Valhalla, NY"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mitchell S.","family":"Cairo","sequence":"additional","affiliation":[{"name":"8Medical College of New York, Valhalla, NY"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julie-An","family":"Talano","sequence":"additional","affiliation":[{"name":"9Division of Pediatric Hematology-Oncology, Medical College of Wisconsin, Milwaukee, WI"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sonali","family":"Chaudhury","sequence":"additional","affiliation":[{"name":"10Lurie Children\u2019s Hospital of Chicago, Chicago, IL"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carrie L.","family":"Kitko","sequence":"additional","affiliation":[{"name":"11Department of Pediatrics, Vanderbilt University Medical Center, Nashville, TN"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4768-0846","authenticated-orcid":false,"given":"Jamie L.","family":"Duke","sequence":"additional","affiliation":[{"name":"12Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania and Children\u2019s Hospital of Philadelphia, Philadelphia, PA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dimitrios","family":"Monos","sequence":"additional","affiliation":[{"name":"12Department of Pathology and Laboratory 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