{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"institution":[{"name":"medRxiv"}],"indexed":{"date-parts":[[2026,1,16]],"date-time":"2026-01-16T14:07:55Z","timestamp":1768572475484,"version":"3.49.0"},"posted":{"date-parts":[[2025,9,2]]},"group-title":"Hematology","reference-count":55,"publisher":"openRxiv","license":[{"start":{"date-parts":[[2025,9,2]],"date-time":"2025-09-02T00:00:00Z","timestamp":1756771200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/publicdomain\/zero\/1.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"accepted":{"date-parts":[[2025,9,2]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                <jats:p>The morbidity and mortality of sickle cell disease (SCD) remain high. Novel nonmyeloablative haploidentical hematopoietic cell transplant (HCT) regimens are being proposed.<\/jats:p>\n                <jats:sec>\n                  <jats:title>Methods<\/jats:title>\n                  <jats:p>This report compared the effect of adding an oral cyclophosphamide and IV pentostatin (PC) preconditioning to the nonmyeloablative NIH HCT platform of alemtuzumab and total body irradiation (TBI).<\/jats:p>\n                <\/jats:sec>\n                <jats:sec>\n                  <jats:title>Results<\/jats:title>\n                  <jats:p>Thirty-nine adult SCD patients were included. The median age was 33 years, 61% were male, and 92% were HbSS genotype. The median follow-up was 6.5 years. Many patients had severe end-organ damage, including dialysis-dependent end-stage kidney disease (10%) and cirrhosis (8%).<\/jats:p>\n                  <jats:p>One-year overall survival was 95%. The PC regimen was associated with a reduction in acute rejection one-year post-HCT (5% vs. 44%; p=0.004) and lower graft failure rates throughout the follow-up period. After a median follow-up of 5.2 years, the disease-free survival was 71% for the PC regimen. The PC preconditioning was associated with higher rates of full donor chimerism at 2-years post-HCT (0% vs. 43%; p=0.02). Grade II-IV acute graft-versus-host disease (GVHD) rates were low; no patients developed moderate to severe chronic GVHD. There remain no cases of myeloid malignancy after PC. With the increased immunosuppression of PC, 23% of patients developed post-transplant lymphoproliferative disorder, 19% developed immune cytopenias, and 62% had viral reactivation.<\/jats:p>\n                <\/jats:sec>\n                <jats:sec>\n                  <jats:title>Summary<\/jats:title>\n                  <jats:p>Further study to determine an optimal nonmyeloablative haploidentical regimen for SCD patients with compromised organ function is imperative.<\/jats:p>\n                <\/jats:sec>","DOI":"10.1101\/2025.08.31.25334807","type":"posted-content","created":{"date-parts":[[2025,9,3]],"date-time":"2025-09-03T01:00:17Z","timestamp":1756861217000},"source":"Crossref","is-referenced-by-count":0,"title":["Nonmyeloablative Pentostatin-Cyclophosphamide Preconditioning Improves Rates of Engraftment in Adults Undergoing Haploidentical HCT for Sickle Cell Disease"],"prefix":"10.64898","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1591-2451","authenticated-orcid":false,"given":"Emily","family":"Limerick","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Matt","family":"Hsieh","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jackie","family":"Queen","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mary 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