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

@article{junior2026closing,
  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}},
  author = {Luiz Guilherme DARRIGO JUNIOR and Roseane GOUVEIA and Leonardo Javier ARCURI and Thalita COSTA and Cilmara KUWAHARA and Roberto LUIZ DA SILVA and Maria Cristina MACEDO and Nelson HAMERSCHLAK and Victor Gottardello ZECCHIN and João FELICIANO and Vivian ZANAO and Simone FRANCO and Antonella ZANETTE and Lorena Bedotti RIBEIRO and Afonso Celso VIGORITO and George Mauricio BARROS and Neysimélia Costa VILLELA and Alessandra GOMES and Vanderson ROCHA and Gisele LOTH and Eliane MIRANDA and Carmem BONFIM and Mary FLOWERS and Fabiola TRAINA and Bellinda SIMÕES and Adriana SEBER},
  journal = {Hematology, Transfusion and Cell Therapy},
  year = {2026},
  month = oct,
  doi = {10.1016/j.htct.2026.106840},
  url = {https://doi.org/10.1016/j.htct.2026.106840},
  note = {CC BY},
}

TY  - JOUR
TI  - 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
AU  - Luiz Guilherme DARRIGO JUNIOR
AU  - Roseane GOUVEIA
AU  - Leonardo Javier ARCURI
AU  - Thalita COSTA
AU  - Cilmara KUWAHARA
AU  - Roberto LUIZ DA SILVA
AU  - Maria Cristina MACEDO
AU  - Nelson HAMERSCHLAK
AU  - Victor Gottardello ZECCHIN
AU  - João FELICIANO
AU  - Vivian ZANAO
AU  - Simone FRANCO
AU  - Antonella ZANETTE
AU  - Lorena Bedotti RIBEIRO
AU  - Afonso Celso VIGORITO
AU  - George Mauricio BARROS
AU  - Neysimélia Costa VILLELA
AU  - Alessandra GOMES
AU  - Vanderson ROCHA
AU  - Gisele LOTH
AU  - Eliane MIRANDA
AU  - Carmem BONFIM
AU  - Mary FLOWERS
AU  - Fabiola TRAINA
AU  - Bellinda SIMÕES
AU  - Adriana SEBER
PY  - 2026
DA  - 2026/10/23
JO  - Hematology, Transfusion and Cell Therapy
DO  - 10.1016/j.htct.2026.106840
UR  - https://doi.org/10.1016/j.htct.2026.106840
AB  - 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.
SN  - 2531-1379
SN  - 2531-1387
ER  -