{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,7]],"date-time":"2026-07-07T22:25:27Z","timestamp":1783463127342,"version":"3.55.0"},"reference-count":36,"publisher":"Wiley","issue":"8","license":[{"start":{"date-parts":[[2026,6,4]],"date-time":"2026-06-04T00:00:00Z","timestamp":1780531200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,6,4]],"date-time":"2026-06-04T00:00:00Z","timestamp":1780531200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2026,8]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Ataxia\u2010telangiectasia (A\u2010T) is a DNA repair disorder characterized by neurodegeneration, immunodeficiency, and cancer predisposition. Hematopoietic cell transplantation (HCT) is an established therapy in related disorders such as Fanconi anemia (FA) and Nijmegen breakage syndrome (NBS), but its role in A\u2010T is unclear. This comprehensive review describes the current literature exploring the indications for HCT in A\u2010T, focusing on conditioning regimens, outcomes, and toxicities.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>A literature review of published A\u2010T HCT cases and preclinical studies was performed, analyzing conditioning regimens, immune reconstitution, complications, overall survival, and follow\u2010up.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      To date, 15 patients with A\u2010T who underwent HCT have been reported. Reduced\u2010intensity or FA\u2010adapted regimens were generally well tolerated and led to durable immune reconstitution, whereas myeloablative conditioning was associated with significant treatment\u2010related toxicities, including veno\u2010occlusive disease (VOD), graft\u2010versus\u2010host disease (GvHD), and hepatic failure. These clinical observations are supported by preclinical\n                      <jats:italic>Atm<\/jats:italic>\n                      \u2010deficient mouse models demonstrating restoration of immune function and reduced lymphoma risk following HCT. Experience from related disorders, such as FA and NBS, further supports the use of radiation\u2010sparing, fludarabine\u2010based conditioning approaches in A\u2010T.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>The use of HCT for the treatment of immunodeficiency and relapsed\/refractory hematologic malignancies in patients with A\u2010T is not well described and understood. Current evidence is limited and heterogeneous, supporting its use only in select high\u2010risk patients at specialized centers, with multicenter collaboration needed to define best practices.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/1545-5017.70414","type":"journal-article","created":{"date-parts":[[2026,6,5]],"date-time":"2026-06-05T06:55:12Z","timestamp":1780642512000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["The Role of Hematopoietic Cell Transplantation in Ataxia\u2010Telangiectasia"],"prefix":"10.1002","volume":"73","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3531-3093","authenticated-orcid":false,"given":"Laila","family":"Alkhouli","sequence":"first","affiliation":[{"name":"Department of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, Pediatric Institute Cleveland Clinic Foundation  Cleveland Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2896-1113","authenticated-orcid":false,"given":"Seth","family":"Rotz","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, Pediatric Institute Cleveland Clinic Foundation  Cleveland Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7580-9184","authenticated-orcid":false,"given":"Masatoshi","family":"Takagi","sequence":"additional","affiliation":[{"name":"Department of Pediatric and Developmental Biology Institute of Science Tokyo  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richa","family":"Sharma","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, Pediatric Institute Cleveland Clinic Foundation  Cleveland Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,6,4]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra0804615"},{"key":"e_1_2_11_3_1","volume-title":"World Cancer Report: Cancer Research for Cancer Prevention","author":"Dogliotti E.","year":"2020"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.3389\/fped.2019.00557"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1097\/CM9.0000000000001471"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1038\/bmt.2009.207"},{"issue":"5","key":"e_1_2_11_7_1","first-page":"573","article-title":"The Incidence and Gene Frequency of Ataxia\u2010telangiectasia in the United States","volume":"39","author":"Swift M.","year":"1986","journal-title":"American Journal of Human Genetics"},{"key":"e_1_2_11_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ctrv.2024.102808"},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010016\u20100543\u20107"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2014.56.5101"},{"key":"e_1_2_11_11_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.21.01495"},{"key":"e_1_2_11_12_1","first-page":"89","article-title":"Mortality and Cancer Incidence in 263 Patients With Ataxia\u2010Telangiectasia","volume":"77","author":"Morrell D.","year":"1986","journal-title":"JNCI: Journal of the National Cancer Institute"},{"key":"e_1_2_11_13_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2024024283"},{"issue":"1","key":"e_1_2_11_14_1","first-page":"421","article-title":"Bone Marrow and thymus Transplantation in Ataxia\u2010Telangiectasia","volume":"11","author":"Buckley R. 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