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Updated: May 26, 2025
A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
A road map for uniform, comprehensive long-term follow-up after curative therapy for sickle cell disease
Shalini Shenoy1, Julie Kanter2, Adetola A Kassim3
1Department of Pediatrics, Washington University School of Medicine, St Louis, MO.
Insights
Gene therapies and stem cell transplants aim to cure sickle cell disease by eliminating sickle hemoglobin. Standardized assessments are crucial for comparing treatment effectiveness and guiding patient care.
Area of Science:
- Hematology
- Transplant Medicine
- Gene Therapy
Background:
- Sickle cell disease treatments, including gene modification and stem cell transplants, are advancing to eradicate sickle hemoglobin and its complications.
- These transformative therapies offer hope for reducing hemolysis, vasculopathy, and mortality associated with sickle cell disease.
Purpose of the Study:
- To establish uniform assessments for evaluating the efficacy and long-term effects of sickle cell disease therapies.
- To provide a roadmap for functional evaluations and management guidelines for clinicians and researchers.
Main Methods:
- The study summarizes current expert recommendations for assessing treatment outcomes.
- It outlines a pathway for functional evaluations and suggests timing for follow-up assessments.
Main Results:
- Standardized functional evaluations are necessary to compare different sickle cell disease treatment modalities.
- A structured approach to long-term follow-up will facilitate informed decision-making for patients and providers.
Conclusions:
- Consistent assessment strategies are vital for comparing allogeneic transplant and autologous gene modification therapies.
- This expert summary provides a framework for managing and monitoring patients undergoing innovative sickle cell disease treatments.
Abstract:
An increasing number of allogeneic transplant and autologous gene modification transplant therapies seek to eradicate sickle hemoglobin and the consequent hemolysis, vasculopathy, functional compromise, morbidity, and mortality. Because these modalities are used in parallel, it is important to be able to define the spectrum and stability of correction, long-term effects, and the pros and cons of each modality. A comparison between interventions that will be sought by providers and patients undergoing intervention requires uniform assessments that evaluate disease- and intervention-related effects for informed decision-making. This expert summary outlines a pathway to functional evaluations with timing recommendations, provides broad management guidelines, and touches upon ongoing research efforts in the field. The road map for long-term follow-up can help clinicians and researchers choose assessments and time them in comparable fashion between the various transformative therapy efforts.
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