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Updated: Feb 17, 2026
A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
Sickle cell disease: a malady beyond a hemoglobin defect in cerebrovascular disease
Junaid Ansari1, Youmna E Moufarrej2, Rafal Pawlinski3
1a Department of Molecular and Cellular Physiology , Louisiana State University Health Sciences Center - Shreveport , Shreveport , LA , USA.
Insights
Sickle cell disease (SCD) is a severe genetic blood disorder affecting many in the US. While treatments are evolving, there
Area of Science:
- Hematology
- Genetics
- Vascular Biology
Background:
- Sickle cell disease (SCD) is a debilitating monogenic disorder impacting around 100,000 people in the U.S.
- Current SCD management focuses on prevention, symptom relief, and targeting red blood cell sickling.
- Cerebrovascular and psychosocial challenges are significant, yet often under-recognized aspects of SCD.
Purpose of the Study:
- To review advancements in SCD understanding and management over recent decades.
- To highlight clinical (especially cerebrovascular) and psychosocial challenges in the U.S.
- To discuss the evolving science and therapeutic landscape for this inherited condition.
Main Methods:
- Review of scientific literature and clinical trial data.
- Analysis of progress in understanding SCD pathophysiology.
- Examination of current and emerging therapeutic strategies.
Main Results:
- Hydroxyurea (HU) was the sole FDA-approved therapy until recently.
- New trials target thrombo-inflammation, endothelial dysfunction, oxidant stress, and the sickle cell mutation.
- Despite progress, therapeutic options remain limited, and psychosocial impacts are substantial.
Conclusions:
- There is a critical need for novel and repurposed adjuvant therapies for SCD.
- Comprehensive clinics addressing both medical and psychosocial aspects are essential.
- Integrated care models are required to address the multifaceted nature of SCD.
Introduction:
Sickle cell disease (SCD) is a devastating monogenic disorder that presents as a multisystem illness and affects approximately 100,000 individuals in the United States alone. SCD management largely focuses on primary prevention, symptomatic treatment and targeting of hemoglobin polymerization and red blood cell sickling. Areas covered: This review will discuss the progress of SCD over the last few decades, highlighting some of the clinical (mainly cerebrovascular) and psychosocial challenges of SCD in the United States. In addition, focus will also be made on the evolving science and management of this inherited disease. Expert commentary: Until recently hydroxyurea (HU) has been the only FDA approved therapy for SCD. However, advancing understanding of SCD pathophysiology has led to multiple clinical trials targeting SCD related thrombo-inflammation, abnormal endothelial biology, increased oxidant stress and sickle cell mutation. Yet, despite advancing understanding, available therapies are limited. SCD also imposes great psychosocial challenges for the individual and the affected community, which has previously been under-recognized. This has created a pressing need for complementary adjuvant therapies with repurposed and novel drugs, in addition to the establishment of comprehensive clinics focusing on both the medical treatment and the psychosocial issues associated with SCD.
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