Journal Article Review Research Support, N.I.H., Extramural Ppublish 10 citations
PubMed: 36206076 DOI: 10.1097/MOH.0000000000000738
Global perspectives on cellular therapy for children with sickle cell disease.
Curr Opin Hematol 29(6):275-280
Authors: Tami D John, Ruth Namazzi, Lulu Chirande, Venée N Tubman
Affiliations
- Center for Cell and Gene Therapy, Baylor College of Medicine.
- Texas Children's Cancer and Hematology Center, Texas Children's Hospital.
- Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA.
- Department of Paediatrics and Child Health, Makerere University College of Health Sciences, Kampala, Uganda.
- School of Medicine, The Muhimbili University of Health and Allied Sciences, Dar es Salaam, Tanzania.
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.
MeSH
Anemia, Sickle Cell, Child, Income, Cells, Therapeutics, Microscopy, Electron, Scanning Transmission, Genes
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