Updating the Brazilian clinical practice guidelines for sickle cell disease: Recommendations and development process
Background: Sickle cell disease is a hereditary blood disorder that significantly impacts morbidity and mortality, requiring comprehensive care. In Brazil, its management in the National Health Service follows the Brazilian Clinical Practice Guidelines, based on evidence and expert consensus. Period...
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| Format: | Article |
| Language: | English |
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Elsevier
2025-10-01
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| Series: | Hematology, Transfusion and Cell Therapy |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2531137925002329 |
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| author | Ludmila Peres Gargano Mariana Millan Fachi Layssa Andrade Oliveira Clarisse Lobo Katharina Nelly Tobos Melnikoff Selma Soriano Marta da Cunha Lobo Souto Maior Meline Rossetto Kron-Rodrigues Dalila Fernandes Gomes Haliton Alves Oliveira Junior Rosa Camila Lucchetta |
| author_facet | Ludmila Peres Gargano Mariana Millan Fachi Layssa Andrade Oliveira Clarisse Lobo Katharina Nelly Tobos Melnikoff Selma Soriano Marta da Cunha Lobo Souto Maior Meline Rossetto Kron-Rodrigues Dalila Fernandes Gomes Haliton Alves Oliveira Junior Rosa Camila Lucchetta |
| author_sort | Ludmila Peres Gargano |
| collection | DOAJ |
| description | Background: Sickle cell disease is a hereditary blood disorder that significantly impacts morbidity and mortality, requiring comprehensive care. In Brazil, its management in the National Health Service follows the Brazilian Clinical Practice Guidelines, based on evidence and expert consensus. Periodic updates ensure alignment with new scientific findings. Objectives: This study describes the methodology for updating the clinical guidelines for sickle cell disease and provides an overview of recommendations for diagnosis, treatment and monitoring, emphasizing the evidence and health technology assessments for prioritized technologies. Methods: The update followed the technical guide of the Brazilian Ministry of Health, and the Gradings of Recommendation, Assessment, Development and Evaluation (GRADE) approach. All the recommendations were assessed by the National Committee for Health Technology Incorporation (Conitec). The clinical guidelines panel included health technology assessment researchers, clinical experts, and policymakers. Systematic reviews assessed new evidence with stakeholder contributions being incorporated through public consultation. Cost-effectiveness analysis was applied to support new technology coverage or changes. Results: The updated clinical guidelines provide structured recommendations for screening, diagnosis, prophylaxis, vaccination, and treatment, covering pharmacological and non-pharmacological approaches. It emphasizes patient and caregiver education to promote early recognition of complications. Expected benefits include fewer pain crises, fewer hospitalizations and transfusions, and improved fetal hemoglobin level, quality of life and survival rates. Key updates include listing epoetin alfa and 100 mg hydroxyurea tablets, expanding hydroxyurea eligibility criteria and revising monitoring protocols. Conclusion: The updated clinical practice guidelines standardize sickle cell disease care in the Brazilian NHS aligned with current evidence. Dissemination and integration aim to enhance healthcare delivery, while future assessments should optimize real-world implementation. |
| format | Article |
| id | doaj-art-f432c7efd98f4c47be4c4b3dbecf7f9b |
| institution | Kabale University |
| issn | 2531-1379 |
| language | English |
| publishDate | 2025-10-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Hematology, Transfusion and Cell Therapy |
| spelling | doaj-art-f432c7efd98f4c47be4c4b3dbecf7f9b2025-08-24T05:13:59ZengElsevierHematology, Transfusion and Cell Therapy2531-13792025-10-0147410396410.1016/j.htct.2025.103964Updating the Brazilian clinical practice guidelines for sickle cell disease: Recommendations and development processLudmila Peres Gargano0Mariana Millan Fachi1Layssa Andrade Oliveira2Clarisse Lobo3Katharina Nelly Tobos Melnikoff4Selma Soriano5Marta da Cunha Lobo Souto Maior6Meline Rossetto Kron-Rodrigues7Dalila Fernandes Gomes8Haliton Alves Oliveira Junior9Rosa Camila Lucchetta10Hospital Alemão Oswaldo Cruz, São Paulo, BrazilHospital Alemão Oswaldo Cruz, São Paulo, BrazilHospital Alemão Oswaldo Cruz, São Paulo, BrazilInstituto Estadual de Hematologia Arthur de Siqueira Cavalcanti, SES, Rio de Janeiro, BrazilCoordenadoria de Saúde Sudeste do Município de São Paulo, São Paulo, BrazilHospital Alemão Oswaldo Cruz, São Paulo, BrazilBrazilian Ministry of Health's (MoH), Brasília, BrazilBrazilian Ministry of Health's (MoH), Brasília, BrazilBrazilian Ministry of Health's (MoH), Brasília, BrazilHospital Alemão Oswaldo Cruz, São Paulo, BrazilHospital Alemão Oswaldo Cruz, São Paulo, Brazil; Corresponding author at: Health Technology Assessment Unit, Hospital Alemão Oswaldo Cruz, Paulista Avenue, 500 (5th floor), Bela Vista, São Paulo, 01310-000, Brazil.Background: Sickle cell disease is a hereditary blood disorder that significantly impacts morbidity and mortality, requiring comprehensive care. In Brazil, its management in the National Health Service follows the Brazilian Clinical Practice Guidelines, based on evidence and expert consensus. Periodic updates ensure alignment with new scientific findings. Objectives: This study describes the methodology for updating the clinical guidelines for sickle cell disease and provides an overview of recommendations for diagnosis, treatment and monitoring, emphasizing the evidence and health technology assessments for prioritized technologies. Methods: The update followed the technical guide of the Brazilian Ministry of Health, and the Gradings of Recommendation, Assessment, Development and Evaluation (GRADE) approach. All the recommendations were assessed by the National Committee for Health Technology Incorporation (Conitec). The clinical guidelines panel included health technology assessment researchers, clinical experts, and policymakers. Systematic reviews assessed new evidence with stakeholder contributions being incorporated through public consultation. Cost-effectiveness analysis was applied to support new technology coverage or changes. Results: The updated clinical guidelines provide structured recommendations for screening, diagnosis, prophylaxis, vaccination, and treatment, covering pharmacological and non-pharmacological approaches. It emphasizes patient and caregiver education to promote early recognition of complications. Expected benefits include fewer pain crises, fewer hospitalizations and transfusions, and improved fetal hemoglobin level, quality of life and survival rates. Key updates include listing epoetin alfa and 100 mg hydroxyurea tablets, expanding hydroxyurea eligibility criteria and revising monitoring protocols. Conclusion: The updated clinical practice guidelines standardize sickle cell disease care in the Brazilian NHS aligned with current evidence. Dissemination and integration aim to enhance healthcare delivery, while future assessments should optimize real-world implementation.http://www.sciencedirect.com/science/article/pii/S2531137925002329Sickle cell anemialPractice guidelinesPublic healthHealth technology assessment |
| spellingShingle | Ludmila Peres Gargano Mariana Millan Fachi Layssa Andrade Oliveira Clarisse Lobo Katharina Nelly Tobos Melnikoff Selma Soriano Marta da Cunha Lobo Souto Maior Meline Rossetto Kron-Rodrigues Dalila Fernandes Gomes Haliton Alves Oliveira Junior Rosa Camila Lucchetta Updating the Brazilian clinical practice guidelines for sickle cell disease: Recommendations and development process Hematology, Transfusion and Cell Therapy Sickle cell anemial Practice guidelines Public health Health technology assessment |
| title | Updating the Brazilian clinical practice guidelines for sickle cell disease: Recommendations and development process |
| title_full | Updating the Brazilian clinical practice guidelines for sickle cell disease: Recommendations and development process |
| title_fullStr | Updating the Brazilian clinical practice guidelines for sickle cell disease: Recommendations and development process |
| title_full_unstemmed | Updating the Brazilian clinical practice guidelines for sickle cell disease: Recommendations and development process |
| title_short | Updating the Brazilian clinical practice guidelines for sickle cell disease: Recommendations and development process |
| title_sort | updating the brazilian clinical practice guidelines for sickle cell disease recommendations and development process |
| topic | Sickle cell anemial Practice guidelines Public health Health technology assessment |
| url | http://www.sciencedirect.com/science/article/pii/S2531137925002329 |
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