{ "abstract": "Introduction: HLA-matched donor hematopoietic cell transplantation (HCT) is curative for sickle cell disease (SCD) but available to few patients; haploidentical HCT expands access, though Latin American data remain scarce. Objectives: To compare outcomes after haploidentical and HLA-matched donor HCT for SCD in a Brazilian multicenter cohort. Methods: This multicenter retrospective study included 223 patients with SCD who underwent haploidentical (n = 64) or HLA-matched donor (n = 159; 98% related) HCT between 2003 and 2026. Primary outcomes were OS and GFFS; secondary outcomes included GVHD. Survival and cumulative incidence were estimated using Kaplan-Meier and Gray methods. Univariable Cox regression adjusted for donor type identified factors associated with GFFS. Results: We included 64 patients (median follow-up, 20 months) in the haploidentical group and 159 patients (median follow-up, 33 months) in the HLA-matched donor group. Stroke was the most common indication; most patients had received hydroxyurea, and most donors carried the HbS trait. Conditioning consisted predominantly of FluCyTT plus TBI in the haploidentical group and BuFlu or BuCy in the HLA-matched donor group. One-year OS and GFFS were 90% (95% CI, 83–98) and 81% (95% CI, 72–92) in the haploidentical group, compared with 95% (95% CI, 92–98; p = 0.25) and 91% (95% CI, 86–96; p = 0.14) in the HLA-matched donor group. GFFS did not differ according to conditioning regimen (BuCy, 92%; BuFlu, 90%; FluCyTT, 87%; p = 0.80). Moyamoya syndrome was independently associated with inferior GFFS (HR, 2.18; 95% CI, 1.06–4.46; p = 0.034), after adjustment for donor type. Among haploidentical recipients, GFFS was higher in the 400 cGy than in the 200 cGy TBI group (90% vs. 66%) (p = 0.46). The 6-month cumulative incidences of grade II–IV and grade III–IV acute GVHD were 22% and 11% in the haploidentical group, compared with 14% (p = 0.20) and 4% (p = 0.034) in the HLA-matched donor group. The 1-year cumulative incidence of chronic GVHD was 10% versus 6% (p = 0.028), whereas the 2-year cumulative incidence of moderate-to-severe chronic GVHD was 7% versus 1% (p = 0.0012). At day 100, full donor chimerism was observed in 81% of haploidentical recipients and 38% of HLA-matched donor recipients, whereas no donor chimerism was observed in 7% and 1%, respectively (p < 0.001; n = 196). At the last assessment (median, 16 months), full donor chimerism was present in 83% versus 42%, and no donor chimerism in 10% versus 9% (p < 0.001; n = 199). Deaths were attributed to bacterial (62%), viral (14%), and fungal (10%) infections and graft failure (14%). Discussion and Conclusion: Outcomes after HLA-matched donor HCT were comparable to those reported in the largest published international series (Gluckman et al., Blood, 2017; n = 1,000; 5-year OS, 92.9%). In the haploidentical group, the 1-year OS (90%) and GFFS (81%) were comparable to those reported in the International Learning Collaborative phase II trial, which similarly included both pediatric and adult patients (Kassim et al., Blood, 2024; 2-year OS, 94.1%). Although the absolute incidences were low, both acute and chronic GVHD were more frequent after haploidentical HCT. Overall, haploidentical HCT represents an effective and safe alternative for patients with SCD who lack an HLA-matched related donor.", "abstract_shown": true, "affiliations": [ "Universidade de São Paulo (USP), Ribeirão Preto, SP, Brazil", "Grupo de Apoio ao Adolescente e à Criança com Câncer (GRAACC), São Paulo, SP, Brazil", "Einsten, São Paulo, SP, Brazil", "Hospital Pequeno Principe, Curitiba, PR, Brazil", "IBCC Oncologia, São Paulo, SP, Brazil", "Beneficiência Portuguesa – SP, São Paulo, SP, Brazil", "Hospital de Base, São José do Rio Preto, SP, Brazil", "Santa Marcelina, São Paulo, SP, Brazil", "Hospital da Criança, Brasília, DF, Brazil", "Hospital Erastinho, Curitiba, PR, Brazil", "Universidade Estadual de Campinas (UNICAMP), Campinas, SP, Brazil", "Hospital de Amor, Barretos, SP, Brazil", "Hospital Sírio-Libanês, São Paulo, SP, Brazil", "Universidade Federal do Paraná (UFPR), Curitiba, PR, Brazil", "Fred Hutchinson Cancer Research Center, United States", "Hospital Samaritano, São Paulo, SP, Brazil" ], "article_type": null, "arxiv": null, "author_total": 26, "authors": [ { "affs": [ 0 ], "name": "Luiz Guilherme DARRIGO JUNIOR" }, { "affs": [ 1 ], "name": "Roseane GOUVEIA" }, { "affs": [ 2 ], "name": "Leonardo Javier ARCURI" }, { "affs": [ 0 ], "name": "Thalita COSTA" }, { "affs": [ 3 ], "name": "Cilmara KUWAHARA" }, { "affs": [ 4 ], "name": "Roberto LUIZ DA SILVA" }, { "affs": [ 4 ], "name": "Maria Cristina MACEDO" }, { "affs": [ 2 ], "name": "Nelson HAMERSCHLAK" }, { "affs": [ 5 ], "name": "Victor Gottardello ZECCHIN" }, { "affs": [ 6 ], "name": "João FELICIANO" }, { "affs": [ 7 ], "name": "Vivian ZANAO" }, { "affs": [ 8 ], "name": "Simone FRANCO" }, { "affs": [ 9 ], "name": "Antonella ZANETTE" }, { "affs": [ 10 ], "name": "Lorena Bedotti RIBEIRO" }, { "affs": [ 10 ], "name": "Afonso Celso VIGORITO" }, { "affs": [ 11 ], "name": "George Mauricio BARROS" }, { "affs": [ 11 ], "name": "Neysimélia Costa VILLELA" }, { "affs": [ 12 ], "name": "Alessandra GOMES" }, { "affs": [ 12 ], "name": "Vanderson ROCHA" }, { "affs": [ 13 ], "name": "Gisele LOTH" }, { "affs": [ 10 ], "name": "Eliane MIRANDA" }, { "affs": [ 3 ], "name": "Carmem BONFIM" }, { "affs": [ 14 ], "name": "Mary FLOWERS" }, { "affs": [ 0 ], "name": "Fabiola TRAINA" }, { "affs": [ 0 ], "name": "Bellinda SIMÕES" }, { "affs": [ 15 ], "name": "Adriana SEBER" } ], "bibtex": "https://research.draconitic.com/paper/7f71xm9ewk8ks.bib", "cited_by": 0, "doi": "10.1016/j.htct.2026.106840", "fulltext_url": "http://www.sciencedirect.com/science/article/pii/S2531137926005870", "id": "7f71xm9ewk8ks", "issns": [ "2531-1379", "2531-1387" ], "landing_url": "https://doi.org/10.1016/j.htct.2026.106840", "language": "en", "licence": "CC-BY", "licence_label": "CC BY", "licence_source": "journal", "licence_url": "https://creativecommons.org/licenses/by/4.0/", "metadata_licence": null, "native_subjects": [ [ "lcc", "RC633-647.5" ], [ "openalex", "2720|Hematopoietic Stem Cell Transplantation" ] ], "openalex": "W7223121239", "pdf_url": "https://www.sciencedirect.com/science/article/pii/S2531137926005870/pdf", "pmcid": null, "pmid": null, "published": "2026-10-23", "publisher": "Elsevier", "ris": "https://research.draconitic.com/paper/7f71xm9ewk8ks.ris", "sources": [ { "first_seen": "2026-10-30T13:43:25Z", "native_id": "2eec14426be543278967d3638a6c353d", "source": "doaj", "url": "https://doi.org/10.1016/j.htct.2026.106840" } ], "subjects": [ { "field": "medicine", "origin": "lcc:RC633-647.5", "subfield": "endocrinology-metabolism" } ], "title": "CLOSING THE DONOR GAP: HAPLOIDENTICAL VS. HLA-MATCHED TRANSPLANTATION FOR SICKLE CELL DISEASE: DATA FROM THE BRAZILIAN REGISTRY OF HEMATOPOIETIC STEM CELL TRANSPLANTATION IN SICKLE CELL DISEASE, ON BEHALF OF THE SBTMO", "title_text": "CLOSING THE DONOR GAP: HAPLOIDENTICAL VS. HLA-MATCHED TRANSPLANTATION FOR SICKLE CELL DISEASE: DATA FROM THE BRAZILIAN REGISTRY OF HEMATOPOIETIC STEM CELL TRANSPLANTATION IN SICKLE CELL DISEASE, ON BEHALF OF THE SBTMO", "type": "peer-reviewed", "updated_at": "2026-10-30T13:43:25Z", "url": "https://research.draconitic.com/paper/7f71xm9ewk8ks", "venue": "Hematology, Transfusion and Cell Therapy", "versions": [] }