Global perspectives on cellular therapy for children with sickle cell disease.
Curr Opin Hematol
; 29(6): 275-280, 2022 11 01.
Article
de En
| MEDLINE
| ID: mdl-36206076
ABSTRACT
PURPOSE OF REVIEW Low-income and middle-income countries (LMICs), primarily in sub-Saharan Africa (SSA), predominantly experience the burden of sickle cell disease (SCD). High frequency of acute and chronic complications leads to increased utilization of healthcare, which burdens fragile health systems. Mortality for children with limited healthcare access remains alarmingly high. Cellular based therapies such as allogeneic hematopoietic stem cell transplant (HSCT) are increasingly used in resource-rich settings as curative therapy for SCD. Broad access to curative therapies for SCD in SSA would dramatically alter the global impact of the disease. RECENT FINDINGS:
Currently, application of cellular based therapies in LMICs is limited by cost, personnel, and availability of HSCT-specific technologies and supportive care. Despite the challenges, HSCT for SCD is moving forward in LMICs. Highly anticipated gene modification therapies have recently proven well tolerated and feasible in clinical trials in resource-rich countries, but access remains extremely limited.SUMMARY:
Translation of curative cellular based therapies for SCD should be prioritized to LMICs where the disease burden and cost of noncurative treatments is high, and long-term quality of life is poor. Focus on thoughtful modifications of current and future therapies to meet the need in LMICs, especially in SSA, will be especially impactful.
Texte intégral:
1
Collection:
01-internacional
Base de données:
MEDLINE
Sujet principal:
Transplantation de cellules souches hématopoïétiques
/
Maladie du greffon contre l'hôte
/
Drépanocytose
Aspects:
Patient_preference
Limites:
Child
/
Humans
Pays/Région comme sujet:
Africa
Langue:
En
Journal:
Curr Opin Hematol
Sujet du journal:
HEMATOLOGIA
Année:
2022
Type de document:
Article