CONTEMPORARY SICKLE CELL DISEASE IN BRAZIL: FINDINGS FROM A MULTICENTER COHORT OF 3,049 PARTICIPANTS

@article{belisario2026contemporary,
  title = {{CONTEMPORARY SICKLE CELL DISEASE IN BRAZIL: FINDINGS FROM A MULTICENTER COHORT OF 3,049 PARTICIPANTS}},
  author = {André Rolim BELISÁRIO and Carla Luana DINARDO and Donald E. WARDEN and Sarah WILSON and Mina Cintho OZAHATA and Sheila De Oliveira Garcia MATEOS and Carolina Teixeira MIRANDA and Anna Bárbara CARNEIRO-PROIETTI and Rosimere Afonso MOTA and Daniela De Oliveira Werneck RODRIGUES and Ester Cerdeira SABINO and Ligia CAPUANI and Alessandra FERRAZ and Cláudia MÁXIMO and Miriam V. FLOR-PARK and Allyson Guimarães da COSTA and Vanderson ROCHA and Cassandra JOSEPHSON and Steve KLEINMAN and Brian CUSTER and Shannon KELLY},
  journal = {Hematology, Transfusion and Cell Therapy},
  year = {2026},
  month = oct,
  doi = {10.1016/j.htct.2026.106562},
  url = {https://doi.org/10.1016/j.htct.2026.106562},
  note = {CC BY},
}

TY  - JOUR
TI  - CONTEMPORARY SICKLE CELL DISEASE IN BRAZIL: FINDINGS FROM A MULTICENTER COHORT OF 3,049 PARTICIPANTS
AU  - André Rolim BELISÁRIO
AU  - Carla Luana DINARDO
AU  - Donald E. WARDEN
AU  - Sarah WILSON
AU  - Mina Cintho OZAHATA
AU  - Sheila De Oliveira Garcia MATEOS
AU  - Carolina Teixeira MIRANDA
AU  - Anna Bárbara CARNEIRO-PROIETTI
AU  - Rosimere Afonso MOTA
AU  - Daniela De Oliveira Werneck RODRIGUES
AU  - Ester Cerdeira SABINO
AU  - Ligia CAPUANI
AU  - Alessandra FERRAZ
AU  - Cláudia MÁXIMO
AU  - Miriam V. FLOR-PARK
AU  - Allyson Guimarães da COSTA
AU  - Vanderson ROCHA
AU  - Cassandra JOSEPHSON
AU  - Steve KLEINMAN
AU  - Brian CUSTER
AU  - Shannon KELLY
PY  - 2026
DA  - 2026/10/23
JO  - Hematology, Transfusion and Cell Therapy
DO  - 10.1016/j.htct.2026.106562
UR  - https://doi.org/10.1016/j.htct.2026.106562
AB  - Introduction: Sickle cell disease (SCD) remains associated with substantial morbidity and premature mortality despite major advances in disease-modifying therapies. However, contemporary multicenter data describing the clinical characteristics, disease burden, and treatment patterns of individuals with SCD in Brazil remain scarce. Objectives: We aimed to characterize the clinical and treatment profile of participants enrolled in the Recipient Epidemiology and Donor Evaluation Study-IV-Pediatric (REDS-IV-P) Brazil SCD cohort. Methods: REDS-IV-P is a prospective multicenter cohort conducted at eight Brazilian referral centers: HCFMUSP and ITACI (São Paulo), Fundação Hemominas (Belo Horizonte, Juiz de Fora, and Montes Claros), Hemope (Recife), Hemorio (Rio de Janeiro), and Hemoam (Manaus). Demographic characteristics, laboratory parameters, treatment utilization, SCD-related complications, and hospitalizations were collected using standardized procedures and uniform, protocol-defined outcome definitions across all participating centers. SCD-related outcomes were evaluated according to genotype (HbSS, HbSβ⁰, and HbSD-Punjab grouped as sickle cell anemia [SCA]) and age group (<18 vs. ≥18-years). Results: Of the 3,049 participants, there was a slight predominance of adults (55.6%) and females (54.4%). HbSS was the most common genotype (72.4%), followed by HbSC (21.6%), HbSβ⁰ (2.9%), and HbSβ+ (2.8%). Participants with SCA had significantly higher white blood cell and platelet counts, bilirubin and lactate dehydrogenase levels, and lower hemoglobin concentrations than other genotypes. Hydroxyurea was used by 57.5% of the overall cohort and by 70.0% of participants with SCA, whereas chronic transfusion therapy was used by approximately one-fifth of individuals with SCA. Among adults, avascular necrosis affected 24.9%, 22.7%, and 33.3% of participants with SCA, HbSC, and HbSβ+, respectively, while ischemic stroke was observed in 18.4%, 3.5%, and 9.5%, and leg ulcers in 17.6%, 3.5%, and 19.1%. Splenic sequestration affected 40.0% of children with SCA. During follow-up, SCA showed the highest incidence rates of ischemic stroke (0.18 vs. 0.07 and 0.00 events per 100 person-yrs), bacteremia/sepsis (0.45 vs. 0.00 and 0.00), and leg ulcers (0.85 vs. 0.14 and 0.00) compared with HbSC and HbSβ+, respectively. Hospitalization rates for acute chest syndrome and vaso-occlusive episodes were consistently highest among participants with SCA, whereas all-cause hospitalization rates among adults were highest in HbSβ+ disease. Hydroxyurea utilization varied substantially across participating centers, with the lowest use observed in Montes Claros and the highest in HEMOAM. Discussion: Although access to hydroxyurea and chronic transfusion therapy has expanded substantially in Brazil over the past decade, the persistence of frequent acute complications, chronic organ damage, and hospitalizations, together with persistent regional disparities in treatment implementation, demonstrates that current therapeutic strategies remain insufficient to fully prevent disease progression. Conclusions: The REDS-IV-P SCD cohort demonstrates that despite broad use of disease-modifying therapies, individuals with SCD in Brazil continue to experience a considerable burden of acute and chronic complications. These findings provide an updated national overview of SCD and support continued efforts to optimize treatment strategies and expand access to effective and curative therapies.
SN  - 2531-1379
SN  - 2531-1387
ER  -