Identification and Characterization of Hematopoietic Stem Cell Transplant Candidates in a Sickle Cell Disease Cohort

Carregando...
Imagem de Miniatura
Citações na Scopus
4
Tipo de produção
article
Data de publicação
2019
Editora
ELSEVIER SCIENCE INC
Indexadores
Título da Revista
ISSN da Revista
Título do Volume
Autores
KELLY, Shannon
PREISS, Liliana
CUSTER, Brian
CARNEIRO-PROIETTI, Anna B. F.
ARAUJO, Aderson S.
LOUREIRO, Paula
MAXIMO, Claudia
RODRIGUES, Daniela O. W.
MOTA, Rosimere A.
Autor de Grupo de pesquisa
NHLBI Recipient Epidemiology Donor
Editores
Coordenadores
Organizadores
Citação
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, v.25, n.10, p.2103-2109, 2019
Projetos de Pesquisa
Unidades Organizacionais
Fascículo
Resumo
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. (C) 2019 American Society for Transplantation and Cellular Therapy.
Palavras-chave
Sickle cell disease, Hematopoietic stem cell transplantation, Bone marrow transplantation, Candidates for HSCT
Referências
  1. Adams RJ, 1998, NEW ENGL J MED, V339, P5, DOI 10.1056/NEJM199807023390102
  2. Angelucci E, 2014, HAEMATOLOGICA, V99, P811, DOI 10.3324/haematol.2013.099747
  3. Bernaudin F, 2007, BLOOD, V110, P2749, DOI 10.1182/blood-2007-03-079665
  4. Bolanos-Meade J, 2019, LANCET HAEMATOL, V6, pE183, DOI 10.1016/S2352-3026(19)30031-6
  5. Brasil. Ministerio da Saude, EST LONG MULT DOENC
  6. Cancado RD, 2007, REV BRAS HEMATOL HEM, V29, P203, DOI 10.1590/S1516-84842007000300002
  7. Carneiro-Proietti ABF, 2018, BRIT J HAEMATOL, V182, P895, DOI 10.1111/bjh.15462
  8. Chaturvedi S, 2016, AM J HEMATOL, V91, P5, DOI 10.1002/ajh.24235
  9. Gluckman E, 2017, BLOOD, V129, P1548, DOI 10.1182/blood-2016-10-745711
  10. Gluckman E, 2013, HEMATOL-AM SOC HEMAT, P370, DOI 10.1182/asheducation-2013.1.370
  11. Hamerschlak N, DIRETRIZES SOC BRASI
  12. KELLEHER JF, 1984, HEMOGLOBIN, V8, P203, DOI 10.3109/03630268408996969
  13. Meinerz Carine, 2008, Rev. Bras. Hematol. Hemoter., V30, P359, DOI 10.1590/S1516-84842008000500006
  14. MENTZER WC, 1994, AM J PEDIAT HEMATOL, V16, P27
  15. PLATT OS, 1994, NEW ENGL J MED, V330, P1639, DOI 10.1056/NEJM199406093302303
  16. SCHNEIDER RG, 1968, BLOOD, V32, P250, DOI 10.1182/blood.V32.2.250.250
  17. Shenoy S, 2013, THER ADV HEMATOL, V4, P335, DOI 10.1177/2040620713483063
  18. Simoes B, 2011, 37 ANN M EUR GROUP B
  19. Simões Belinda P., 2010, Rev. Bras. Hematol. Hemoter., V32, P46
  20. STURGEON P, 1955, BLOOD, V10, P389, DOI 10.1182/blood.V10.5.389.389
  21. Tubman VN, 2007, PEDIATR BLOOD CANCER, V49, P207, DOI 10.1002/pbc.21269
  22. Walters MC, 1996, NEW ENGL J MED, V335, P369, DOI 10.1056/NEJM199608083350601
  23. WITKOWSKA HE, 1991, NEW ENGL J MED, V325, P1150, DOI 10.1056/NEJM199110173251607