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Updated: Jul 28, 2025

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
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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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Emerging Therapies and Advances in Sickle Cell Disease with a Focus on Renal Manifestations

Mofiyin Obadina1, Sam Wilson1,2, Vimal K Derebail3

  • 1Division of Hematology, Department of Medicine, University of North Carolina, Chapel Hill, North Carolina.

Kidney360
|May 31, 2023
PubMed

Insights

Sickle cell disease (SCD) causes kidney problems like albuminuria, a predictor of mortality. New and existing therapies targeting SCD and kidney disease are being studied to improve patient survival.

Area of Science:

  • Nephrology
  • Hematology
  • Pharmacology

Background:

  • Sickle cell disease (SCD) is linked to complex kidney damage (nephropathy), often presenting as albuminuria.
  • In severe SCD (Hemoglobin SS [HbSS]), albuminuria and chronic kidney disease (CKD) significantly predict mortality.
  • Renal function monitoring and management are crucial components of comprehensive SCD care.

Purpose of the Study:

  • To review current and emerging therapeutic strategies for SCD-related nephropathy.
  • To highlight the impact of novel SCD-directed and conventional CKD-directed therapies on renal outcomes.
  • To emphasize the need for further research into the efficacy of these treatments for SCD nephropathy.

Main Methods:

  • Literature review of recent advancements in SCD and CKD management.
  • Analysis of approved and investigational therapies for SCD, including hydroxyurea, l-glutamine, crizanlizumab, and voxelotor.
  • Examination of the role of renin-angiotensin-aldosterone system blockers in SCD nephropathy.

Main Results:

  • Novel therapies for SCD target anemia, inflammation, and vasculopathy, with ongoing investigation into their renal effects.
  • Established treatments like hydroxyurea and transfusions are standard care.
  • CKD-directed therapies, such as RAAS blockers, are used, but long-term benefit data require further study.

Conclusions:

  • Effective management of SCD nephropathy requires both SCD-specific and CKD-specific therapeutic approaches.
  • Further assessment of novel and existing therapeutic agents is essential to improve survival in SCD patients with renal disease.
  • Understanding the mechanisms and efficacy of treatments is critical for optimizing care and outcomes.

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