Table 2.

Comparison of existing therapies for anemia in SCD

Blood transfusionsHydroxyureaErythropoiesis-stimulating agentsVoxelotor
Indications 
  • Indicated for acute or severe episodes of anemia

  • No indication for chronic anemia

 
  • No indication for acute or chronic anemia

  • Potential adjunctive therapy during shortages of blood products

 
  • Indicated for chronic anemia related to kidney disease

  • No indication for SCD

 
  • No specific indication for anemia, but approval for SCD was based on increasing Hb level

 
Limitations 
  • Requires parenteral access

  • Requires blood banking support to find safe and compatible blood

  • Risk of alloimmunization, hemolytic transfusion reactions, and iron overload

 
  • Requires monitoring for myelosuppression

  • Possible risk of teratogenesis and leukemogenesis (evidence limited)

  • Not tolerated by or ineffective in some patients

 
  • Requires subcutaneous injection

  • Evidence in SCD is limited

  • Possible risk of viscosity-related and vaso-occlusive complications (evidence limited)

  • Poor compliance with injections

 
  • Requires access to new SCD therapies and insurance coverage (availability limited in low-resource settings)

  • Possible risk of increasing Hb oxygen affinity causing tissue hypoxia

  • Limited postmarketing data and clinical experience, especially when used in combination with other available therapies

 
Blood transfusionsHydroxyureaErythropoiesis-stimulating agentsVoxelotor
Indications 
  • Indicated for acute or severe episodes of anemia

  • No indication for chronic anemia

 
  • No indication for acute or chronic anemia

  • Potential adjunctive therapy during shortages of blood products

 
  • Indicated for chronic anemia related to kidney disease

  • No indication for SCD

 
  • No specific indication for anemia, but approval for SCD was based on increasing Hb level

 
Limitations 
  • Requires parenteral access

  • Requires blood banking support to find safe and compatible blood

  • Risk of alloimmunization, hemolytic transfusion reactions, and iron overload

 
  • Requires monitoring for myelosuppression

  • Possible risk of teratogenesis and leukemogenesis (evidence limited)

  • Not tolerated by or ineffective in some patients

 
  • Requires subcutaneous injection

  • Evidence in SCD is limited

  • Possible risk of viscosity-related and vaso-occlusive complications (evidence limited)

  • Poor compliance with injections

 
  • Requires access to new SCD therapies and insurance coverage (availability limited in low-resource settings)

  • Possible risk of increasing Hb oxygen affinity causing tissue hypoxia

  • Limited postmarketing data and clinical experience, especially when used in combination with other available therapies

 

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