Is Severity Score Associated With Indication for Hematopoietic Stem Cell Transplantation in Individuals With Sickle Cell Anemia?

Miriam V Flor-Park; Mina Cintho Ozahata; Isabel Cristina Gomes Moura; Paula Blatyta; Shannon Kelly; Claudia di Lorenzo Oliveira; Ligia Capuani; André Rolim Belisário; Anna B F Carneiro-Proietti; Aderson S Araujo; Paula Loureiro; Claudia Maximo; Daniela O W Rodrigues; Rosimere A Mota; Ester Sabino; Brian Custer; Vanderson Rocha; Recipient Epidemiology and Donor Evaluation Study-III (REDS-III) International Component Brazil

doi:10.1016/j.jtct.2022.06.024

Is Severity Score Associated With Indication for Hematopoietic Stem Cell Transplantation in Individuals With Sickle Cell Anemia?

Transplant Cell Ther. 2022 Oct;28(10):708.e1-708.e8. doi: 10.1016/j.jtct.2022.06.024. Epub 2022 Jul 3.

Authors

Miriam V Flor-Park¹, Mina Cintho Ozahata², Isabel Cristina Gomes Moura³, Paula Blatyta⁴, Shannon Kelly⁵, Claudia di Lorenzo Oliveira⁶, Ligia Capuani⁷, André Rolim Belisário⁸, Anna B F Carneiro-Proietti⁸, Aderson S Araujo⁹, Paula Loureiro¹⁰, Claudia Maximo¹¹, Daniela O W Rodrigues¹², Rosimere A Mota¹³, Ester Sabino¹⁴, Brian Custer¹⁵, Vanderson Rocha¹⁶; Recipient Epidemiology and Donor Evaluation Study-III (REDS-III) International Component Brazil¹⁷

Affiliations

¹ 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.
² Departmentof Computer Science, Universidade de São Paulo, Brazil.
³ Programa de Pós-graduação em Ciências da Saúde, Faculdade de Ciências Médicas de Minas Gerais, Brazil.
⁴ Hospital Moyses Deutsch, São Paulo, Brazil.
⁵ Vitalant Research Institute, Epidemiology, San Francisco, California; University of California San Francisco Benioff Children's Hospital, Oakland, California.
⁶ Universidade de São João Del Rei, Minas Gerais, Brazil.
⁷ Departamento de Moléstias Infecciosas e Parasitárias da Faculdade de medicina da Universidade de São Paulo, Brazil.
⁸ Fundação Hemominas, Belo Horizonte, Brazil.
⁹ Department of Hematology, Fundação de Hematologia e Hemoterapia de Pernambuco, HEMOPE, Pernambuco, Brazil.
¹⁰ Research Department, Fundação de Hematologia e Hemoterapia de Pernambuco, HEMOPE, Pernambuco, Brazil; Research Department, Universidade de Pernambuco, Pernambuco, Brazil.
¹¹ Department of Hematology, Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti-HEMORIO, Rio de Janeiro, Brazil.
¹² Department of Hematology, Fundação HEMOMINAS, Juiz de Fora, Brazil.
¹³ Department of Hematology, Hemocentro Regional de Montes Claros, Fundação HEMOMINAS, Montes Claros, Brazil.
¹⁴ Instituto de Medicina Tropical and Departamento de Moléstias Infecciosas e Parasitárias da Faculdade de Medicina da Universidade de São Paulo, Brazil.
¹⁵ Vitalant Research Institute, Epidemiology, San Francisco, California; Department of Laboratory Medicine, University of California San Francisco, San Francisco, CA, USA.
¹⁶ Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo, Serviço de Hematologia, Hemoterapia e Terapia Celular, São Paulo, Brazil; Laboratory of Medical Investigation (LIM 31) in Pathogenesis and Directed Therapy in Onco-Immuno-Hematology, Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo, São Paulo, Brazil; Department of Haematology, University of Oxford, Churchill Hospital, Oxford, United Kingdom.
¹⁷ Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo, Instituto da Criança, São Paulo, Brazil.

Abstract

Manifestations of sickle cell disease (SCD) begin early in childhood and cause morbidity and decreased life expectancy. Hematopoietic stem cell transplantation (HSCT) is curative but associated with risk of mortality attributable to the transplant. This risk should be counterbalanced with SCD morbidity and mortality. A severity score using a Bayesian network model was previously validated to predict the risk of death in adult individuals with SCD. The objective of this study is to calculate the severity scores of participants in a multicenter cohort of Brazilians with SCD, using a previously published Bayesian network-derived score, associated with risk of death and then compare the severity scores between participants with and without an indication for HSCT as defined by the Brazilian Ministry of Health (MoH) criteria. This is an observational, retrospective study. We analyzed 2063 individuals with sickle cell anemia from the Recipient Epidemiology and Donor Evaluation Study-III Brazil SCD cohort and applied a Bayesian network-derived score to compare candidates and non-candidates for HSCT according to the Brazilian MoH transplant criteria. Classical statistical methods were used to analyze data and make comparisons. We compared severity scores between cohort members with (n = 431) and without (n = 1632) HSCT indications according to Brazilian MoH. Scores were not different in adult participants with ≥1 HSCT indication when compared to those with no indication (mean 0.342 versus 0.292; median 0.194 versus 0.183, P = .354) and receiver operating characteristic curves did not demonstrate an obvious threshold to differentiate participants with or without HSCT indications. Severity score may predict risk of death but does not differentiate HSCT candidates. Current indications should be evaluated to ensure that patients with more severe disease who might benefit from HSCT are appropriately identified.

Keywords: Bayesian model; Candidates; Cohort; HSCT; Hematopoietic stem cell transplantation; Severity score; Sickle cell anemia; Sickle cell disease.

Publication types

Multicenter Study
Observational Study
Research Support, Non-U.S. Gov't
Research Support, N.I.H., Extramural

MeSH terms

Adult
Anemia, Sickle Cell* / therapy
Bayes Theorem
Hematopoietic Stem Cell Transplantation* / adverse effects
Humans
Retrospective Studies
Tissue Donors

Abstract

Publication types

MeSH terms

Grants and funding