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Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload
Published on: March 14, 2017
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Treatment Options for Sickle Cell Disease.
1Sickle Cell Research, Indiana Hemophilia and Thrombosis Center, 8326 Naab Road, Indianapolis, IN 46260, USA.
Pediatric Clinics of North America
|May 28, 2018
Summary
Sickle cell disease (SCD) management focuses on preventing sickle hemoglobin polymerization. Current treatments include hydroxyurea, supportive care, and limited transfusions, with stem cell transplant as a curative but inaccessible option for most.
Area of Science:
- Hematology
- Genetic Blood Disorders
- Internal Medicine
Background:
- Sickle cell disease (SCD) complications arise from sickle hemoglobin (HbS) polymerization.
- Therapeutic strategies aim to inhibit HbS production or decrease its circulation.
Purpose of the Study:
- To review advancements in supportive and preventive care for SCD.
- To highlight the benefits of hydroxyurea therapy.
- To summarize indications for erythrocyte transfusion, stem cell transplant, and gene therapy.
Main Methods:
- Literature review of SCD management strategies.
- Analysis of current therapeutic approaches including hydroxyurea, transfusions, and stem cell transplantation.
- Summary of emerging gene therapy trials.
Main Results:
- Hydroxyurea is increasingly utilized for SCD management.
- Evidence-based indications for erythrocyte transfusion remain limited.
- Hematopoietic stem cell transplant offers a cure but is limited by donor availability (<25% of patients).
Conclusions:
- Improvements in supportive and preventive care are crucial for SCD patients.
- Hydroxyurea offers significant benefits in managing SCD.
- While stem cell transplant and gene therapy show promise, broader accessibility remains a challenge.
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