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Identification and Characterization of Hematopoietic Stem Cell Transplant Candidates in a Sickle Cell Disease Cohort.
Biol Blood Marrow Transplant 2019; 25(10):2103-2109BB

Abstract

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.

Authors+Show Affiliations

Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo, Instituto da Criança, São Paulo, Brazil. Electronic address: miriam.park@hc.fm.usp.br.Vitalant Research Institute, Epidemiology, San Francisco, CA, USA; University of California San Francisco Benioff Children's Hospital Oakland, CA, USA.Division of Bioestatistics and Epidemiology, Research Triangle Institute, Rockville, MD, USA.Vitalant Research Institute, Epidemiology, San Francisco, CA, USA; Department of Laboratory Medicine, University of California San Francisco, San Francisco, CA, USA.Research Division, Fundação HEMOMINAS, Belo Horizonte, Brazil.Department of Hematology, Fundação de Hematologia e Hemoterapia de Pernambuco, HEMOPE, Pernambuco, Brazil.Research Department, Fundação de Hematologia e Hemoterapia de Pernambuco, HEMOPE, Pernambuco, Brazil; Research Department, Universidade de Pernambuco, Pernambuco, Brazil.Department of Hematology, Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti-HEMORIO, Rio de Janeiro, Brazil.Fundação HEMOMINAS, Department of Hematology, Juiz de Fora, Brazil.Department of Hematology, Hemocentro Regional de Montes Claros, Fundação HEMOMINAS, Montes Claros, Brazil.Instituto de Medicina Tropical and Departamento de Moléstias Infecciosas e Parasitárias da Faculdade de Medicina da Universidade de São Paulo.Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo, Serviço de Hematologia, Hemoterapia e Terapia Celular, São Paulo, Brazil; Department of Haematology, University of Oxford, Churchill Hospital, NHS-BT, Oxford, United Kingdom.No affiliation info available

Pub Type(s)

Journal Article

Language

eng

PubMed ID

31229639

Citation

Flor-Park, Miriam V., et al. "Identification and Characterization of Hematopoietic Stem Cell Transplant Candidates in a Sickle Cell Disease Cohort." Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation, vol. 25, no. 10, 2019, pp. 2103-2109.
Flor-Park MV, Kelly S, Preiss L, et al. Identification and Characterization of Hematopoietic Stem Cell Transplant Candidates in a Sickle Cell Disease Cohort. Biol Blood Marrow Transplant. 2019;25(10):2103-2109.
Flor-Park, M. V., Kelly, S., Preiss, L., Custer, B., Carneiro-Proietti, A. B. F., Araujo, A. S., ... Rocha, V. (2019). Identification and Characterization of Hematopoietic Stem Cell Transplant Candidates in a Sickle Cell Disease Cohort. Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation, 25(10), pp. 2103-2109. doi:10.1016/j.bbmt.2019.06.013.
Flor-Park MV, et al. Identification and Characterization of Hematopoietic Stem Cell Transplant Candidates in a Sickle Cell Disease Cohort. Biol Blood Marrow Transplant. 2019;25(10):2103-2109. PubMed PMID: 31229639.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Identification and Characterization of Hematopoietic Stem Cell Transplant Candidates in a Sickle Cell Disease Cohort. AU - Flor-Park,Miriam V, AU - Kelly,Shannon, AU - Preiss,Liliana, AU - Custer,Brian, AU - Carneiro-Proietti,Anna B F, AU - Araujo,Aderson S, AU - Loureiro,Paula, AU - Maximo,Claudia, AU - Rodrigues,Daniela O W, AU - Mota,Rosimere A, AU - Sabino,Ester C, AU - Rocha,Vanderson, AU - ,, Y1 - 2019/06/20/ PY - 2019/03/17/received PY - 2019/06/12/revised PY - 2019/06/14/accepted PY - 2019/6/24/pubmed PY - 2019/6/24/medline PY - 2019/6/24/entrez KW - Bone marrow transplantation KW - Candidates for HSCT KW - Hematopoietic stem cell transplantation KW - Sickle cell disease SP - 2103 EP - 2109 JF - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JO - Biol. Blood Marrow Transplant. VL - 25 IS - 10 N2 - 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. SN - 1523-6536 UR - https://www.unboundmedicine.com/medline/citation/31229639/Identification_and_characterization_of_hematopoietic_stem_cell_transplant_candidates_in_a_sickle_cell_disease_cohort L2 - https://linkinghub.elsevier.com/retrieve/pii/S1083-8791(19)30377-5 DB - PRIME DP - Unbound Medicine ER -