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My 4-year-old son has sickle cell disease. Is there a permanent cure, and how can it be achieved?
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My 4-year-old son has sickle cell disease. Is there a permanent cure, and how can it be achieved?
Asked by Female, 34 · 3 months ago
Yes, a permanent cure is possible through a bone marrow (stem cell) transplant from a matched donor. Additionally, new options for gene therapy are being developed. These options are not suitable for everyone, but they are the only treatments that can fully eliminate the disease.
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What is the success rate of stem cell transplants?Are there risks with stem cell transplants?How early should treatment be started?What are the chances gene therapy will be available?Can supportive care alone manage sickle cell?
What is Sickle Cell Disease (SCD)
- SCD is a genetic disorder affecting hemoglobin in red blood cells, causing them to become rigid and sickle-shaped, leading to various complications.
Permanent Cure Options
- Hematopoietic Stem Cell Transplant (HSCT) is the only established permanent cure, replacing defective marrow with healthy donor marrow.
Eligibility and Considerations for HSCT
- Requires a matched donor, often a sibling. Risks include graft-versus-host disease and infertility. Early intervention is preferred.
Emerging Treatments and Research
- Gene therapy is a promising future option, currently in clinical trials.
Supportive Care and Management
- Ongoing care is crucial, including medications like hydroxyurea and regular monitoring to manage symptoms and prevent complications.
Consulting Specialists
- Consult a hematologist experienced in SCD. Multidisciplinary care is essential.
Emotional and Family Support
- Recognize the emotional impact on families. Seek counseling and support groups for assistance.
Answered 3 months ago