Overview of Treatment Options
Treatment varies for each individual, but the main goal is to reduce symptoms, manage complications, and improve quality of life. Options include medications, blood transfusions, stem cell transplants, and gene therapy — alongside new therapies under research.
Medications
Medications reduce pain, minimize complications, and improve hemoglobin function.
Oral Medication for Hemoglobin Production
Daily oral drug that boosts healthy hemoglobin, reducing sickling and blockages.
Monoclonal Antibody Infusion
IV therapy (16+) targeting P-selectin to reduce severe pain crises (VOCs).
Hemoglobin S Polymerization Inhibitor
Oral drug (age 4+) improving oxygen affinity to prevent red blood cell sickling.
Antioxidant-Based Oral Therapy
For patients 5+, supporting metabolism and reducing pain episodes through antioxidant effects.
Note: This list does not cover all approved medications for sickle cell disease.
Blood Transfusions
Used to prevent complications (like stroke) and improve oxygen delivery.
Simple Transfusion
Administering donated blood to the patient.
Red Blood Cell Exchange
Removing sickled cells and replacing them with healthy ones.
Risks: immune reactions, infections, iron overload.
Hematopoietic Stem Cell Transplant (HSCT)
Also known as bone marrow transplant, HSCT is the only treatment with curative potential. It is usually recommended for patients under 16 with a matched-sibling donor. HSCT does not directly correct the genetic cause of SCD.
Gene Therapy
Gene therapy targets sickle cell at the DNA level:
- Gene Addition: Adding new genetic material to improve hemoglobin production.
- Gene Editing: Modifying existing genes to correct the sickle mutation.
Advances in Sickle Cell Research
As of May 2024, more than 500 studies are completed, with over 250 active trials exploring new solutions.
Emerging Therapies
- PKR Activator: Daily oral drug improving hemoglobin and reducing VOCs.
- Monoclonal Antibody Therapy: Targets P-selectin to lower VOC frequency.
- Small-Molecule Therapy: Activates pyruvate kinase-R, improving ATP and reducing sickling.
HSCT Improvements
Research is reducing HSCT risks and expanding donor options (35+ completed studies, 50+ active).
Gene Therapy Developments
Using a patient’s own cells to correct genetic defects, removing the need for a donor.
Screening & Training
CDC initiatives improve newborn screening, track long-term care data, and train providers (e.g., STAMP program launched in 2020).
Taking Action
Forming a multidisciplinary care team and advocating for awareness are essential steps to improve sickle cell care.
“Stay proactive and listen to your body. Monitoring your hemoglobin levels and understanding how they fluctuate can make a significant difference in managing your health.” — Cory, Living with Sickle Cell Disease
How Does Sickle Cell Affect You?
Reflect on how sickle cell impacts your daily life. Sharing your story can guide care discussions and contribute to the wider movement to improve sickle cell treatment and awareness. Click here to share your story