The evolving treatment landscape for children with sickle cell disease.
Lancet Child Adolesc Health
; 7(11): 797-808, 2023 11.
Article
en En
| MEDLINE
| ID: mdl-37858508
ABSTRACT
Sickle cell disease is the most common inherited pathological haemoglobinopathy. Over the past 30 years, disease-related morbidity and mortality have improved in high-income countries due to advances in preventive care and treatments. Established disease-modifying therapies, such as hydroxyurea (hydrocarbamide), are continuing to have an important role in the treatment of sickle cell disease, and newer agents also show promise. In the past 5 years, the US Food and Drug Administration approved three additional sickle cell disease-modifying medications, and new gene therapies have been developed as an alternative curative treatment to haematopoietic stem-cell transplantation. In this Review, we discuss the current treatment landscape for paediatric sickle cell disease and emerging innovations in care. We also review the need for close, long-term management for children receiving newer therapies and the importance of ongoing investment in people with sickle cell disease in low-income and middle-income countries.
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Trasplante de Células Madre Hematopoyéticas
/
Anemia de Células Falciformes
Límite:
Child
/
Humans
País/Región como asunto:
America do norte
Idioma:
En
Revista:
Lancet Child Adolesc Health
Año:
2023
Tipo del documento:
Article
País de afiliación:
Estados Unidos