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A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
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Treatment of sickle cell disease - options and perspective

Loubna Abdel-Hadi1, Yendry Ventura Carmenate1, Yandy Marx Castillo-Aleman1

  • 1Abu Dhabi Stem Cells Center Abu Dhabi, UAE.

American Journal of Blood Research
|May 22, 2023
PubMed

Insights

This study evaluates the cost-effectiveness of treatments for Sickle Cell Disease (SCD), comparing drug therapies and hematopoietic stem cell transplantation (HSCT). It aims to guide decisions on HSCT for SCD patients amidst rising healthcare costs.

Area of Science:

  • Hematology
  • Genetics
  • Health Economics

Background:

  • Sickle Cell Disease (SCD) is an inherited blood disorder with severe complications and high mortality.
  • Prevalence is highest in West Africa, India, the Mediterranean, and the Middle East.
  • Current treatments like hydroxyurea, L-glutamine, Voxelotor, and Crizanlizumab manage symptoms but are not curative.

Purpose of the Study:

  • To conduct a cost-effectiveness evaluation of SCD treatments.
  • To guide decision-making regarding hematopoietic stem cell transplantation (HSCT) as a curative option.
  • To address questions about the value of available drug therapies in the context of rising healthcare costs.

Main Methods:

  • Cost-effectiveness analysis comparing various SCD treatment modalities.
  • Evaluation of drug therapies (Hydroxyurea, L-glutamine, Voxelotor, Crizanlizumab).
  • Assessment of cell-based therapies including red blood cell (RBC) exchange and hematopoietic stem cell transplantation (HSCT).
  • Consideration of emerging gene-editing therapies for SCD and β-thalassemias.

Main Results:

  • While drug therapies are available, they do not offer a cure for SCD.
  • Hematopoietic stem cell transplantation (HSCT) is the only curative therapy currently.
  • Gene-editing therapies show promise but are associated with high costs.
  • Rising healthcare costs, including hospitalizations, necessitate value-based treatment evaluations.

Conclusions:

  • A cost-effectiveness evaluation is crucial for guiding treatment decisions in SCD.
  • HSCT remains a key consideration for curative therapy despite its complexities.
  • Future efforts should focus on optimizing the value of SCD treatments in light of economic factors.

Keywords:
Sickle cell diseasefetal hemoglobinhematopoietic stem cell transplant

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