Identification and Characterization of Hematopoietic Stem Cell Transplant Candidates in a Sickle Cell Disease Cohort.
Sickle cell disease (SCD) is associated with significant morbidity, and allogeneic hematopoietic stem cell transplantation (HSCT) remains the primary curative treatment. Recently, the Brazilian Ministry of Health released a regulation that required the publically funded healthcare system to pay for HSCT for SCD patients with defined indications. We used an existing 2794-member SCD cohort established during 2013 to 2015 to characterize candidates for HSCT and estimate the number of possible donors. Of 2064 patients with SC anemia (SCA), 152 of 974 children (16%) and 279 of 1090 adults (26%) had at least 1 HSCT indication. The most common indication for transplant was stroke (n = 239) followed by avascular necrosis (n = 96), priapism (n = 82), cerebrovascular disease (n = 55), >2 vaso-occlusive episodes (n = 38), alloantibodies and chronic transfusion therapy (n = 18), and >2 acute chest syndrome episodes (n = 11). Increasing age, number of transfusions, abnormal transcranial Doppler, retinopathy, dactylitis, and use of hydroxyurea were more frequent in the 152 children with an indication for HSCT compared with 822 without (P < .001). Of 152 children and 279 adults meeting the eligibility definition, 77 (50%) and 204 (73%), respectively, had at least 1 non-SCD full sibling who could potentially serve as a donor. In conclusion, in a large cohort of SCA patients, 16% of children and 26% of adults had at least 1 indication for HSCT; these indications were associated with the severity of the disease. This study provides clinical data necessary for estimating the costs and infrastructure that would be required to implement HSCT in a public healthcare system.
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo, Instituto da Criança, São Paulo, Brazil. Electronic address: email@example.com.,
Vitalant Research Institute, San Francisco, California; University of California San Francisco Benioff Children's Hospital Oakland, California.,
Research Triangle Institute, Rockville, Maryland.,
Vitalant Research Institute, San Francisco, California; Department of Laboratory Medicine, University of California San Francisco, San Francisco, California.,
Fundação HEMOMINAS, Belo Horizonte, Brazil.,
Fundação de Hematologia e Hemoterapia de Pernambuco, HEMOPE, Pernambuco, Brazil.,
Fundação de Hematologia e Hemoterapia de Pernambuco, HEMOPE, Pernambuco, Brazil; Universidade de Pernambuco, Pernambuco, Brazil.,
Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti-HEMORIO, Rio de Janeiro, Brazil.,
Fundação HEMOMINAS, Juiz de Fora, Brazil.,
Hemocentro Regional de Montes Claros, Fundação HEMOMINAS, Montes Claros, Brazil.,
Instituto de Medicina Tropical e Departamento de Moléstias Infecciosas e Parasitárias da Faculdade de Medicina da Universidade de São Paulo, São Paulo, Brazil.,
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo, Serviço de Hematologia, Hemoterapia e Terapia Celular, São Paulo, Brazil; Churchill Hospital Department of Haematology NHS-BT, University of Oxford, Oxford, United Kingdom.
Pub Type(s)Journal Article