Models of care for sickle cell disease in low-income and lower-middle-income countries: a scoping review

Drown, Laura; Osei, Miriam; Thapa, Ada; Boudreaux, Chantelle; Archer, Natasha; Bukhman, Gene; Adler, Alma J

doi:10.1016/s2352-3026(24)00007-3

The Lancet Haematology

2024

DOI: 10.1016/s2352-3026(24)00007-3

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Models of care for sickle cell disease in low-income and lower-middle-income countries: a scoping review

Laura Drown,

Miriam Osei,

Ada Thapa

et al.

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Gene Therapies for Sickle Cell Disease

Weaver,

Singh,

Wilson

2024

Journal of Pharmacy Technology

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Background: Sickle cell disease (SCD) is a prevalent autosomal recessive hemoglobinopathy affecting millions worldwide, particularly individuals of African ancestry. Sickle cell disease is a lifelong condition associated with a negative impact on quality of life and mortality, causing complications such as painful vaso-occlusive episodes, acute chest syndrome, stroke, long-term anemia, and end-organ damage. Currently, there are 4 U.S. Food and Drug Administration (FDA)-approved drugs, including hydroxyurea, l-glutamine, voxelotor, and crizanlizumab, which work to alleviate symptoms and prevent complications associated with SCD, albeit without addressing the underlying cause of SCD. Allogeneic hematopoietic stem cell transplant (HSCT) has shown promise as a curative approach to SCD but is limited by donor availability and associated complications. This paper aims to review the efficacy and safety of exagamglogene autotemcel and lovotibeglogene autotemcel for managing patients with SCD, including their place in therapy, cost, and accessibility in clinical care. Data Sources: The authors searched PubMed and Medline from 2017 to 2024, for primary literature on both exagamglogene autotemcel and lovotibeglogene autotemcel. Results: The authors identified relevant studies and summarized the data on the two gene therapies. Conclusion: Exagamglogene autotemcel and lovotibeglogene autotemcel are two management strategies that address the underlying cause of SCD and provide curative potential for patients with SCD.

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Gene Therapies for Sickle Cell Disease

Weaver,

Singh,

Wilson

2024

Journal of Pharmacy Technology

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show abstract

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Models of care for sickle cell disease in low-income and lower-middle-income countries: a scoping review

Cited by 1 publication

References 111 publications

Gene Therapies for Sickle Cell Disease

Gene Therapies for Sickle Cell Disease

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