Thalassemia major
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About Thalassemia major
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- Beta Thalassemia Major (Transfusion-dependent)
- E-beta Thalassemia
- Severe Alpha Thalassemia (HbH disease, hydrops)
- Iron Overload Complications
- Bone Marrow Transplantation (curative option)
A procedure where healthy stem cells from a matched donor (usually a sibling or unrelated donor) are transplanted into the patient to replace damaged or defective bone marrow—used to treat conditions like thalassemia major, leukemia, and aplastic anemia.
- Potential Cure – Offers a lifelong cure for diseases like thalassemia major.
- Donor Immunity – Donor immune cells may help destroy any residual diseased cells.
- No Lifelong Transfusions – Eliminates the need for regular blood transfusions and iron chelation therapy.
- Risk of Graft-vs-Host Disease (GVHD) – Can lead to serious immune complications.
- Strict Donor Match Required – HLA-matching is critical; lack of a donor can delay or prevent treatment.
- High Cost & Recovery Time – Expensive and requires prolonged hospital stays and follow-up care.
- USD: $28,000 – $40,000
- KES: KSh 3.6 million – 5.2 million
Cost includes donor screening, chemotherapy, transplant, hospital stay (4–6 weeks), and initial post-transplant care.
This is the standard ongoing treatment for Thalassemia Major. It involves giving patients regular packed red blood cell transfusions to maintain hemoglobin levels, combined with iron chelation therapy (like deferasirox or deferoxamine) to remove excess iron from the body caused by repeated transfusions.
- Maintains Hemoglobin Levels – Prevents severe anemia and supports growth and development.
- Non-Surgical – Less invasive than bone marrow transplant; easier access.
- Widely Available – Can be administered in most major hospitals and clinics.
- Lifelong Treatment – Patients require transfusions every 2–5 weeks indefinitely.
- Iron Overload – Leads to organ damage (heart, liver) without strict chelation.
- Reduced Quality of Life – Frequent hospital visits and side effects from chelation drugs.
- USD: $4,000 – $7,000/year
- KES: KSh 520,000 – 910,000/year
Cost includes blood transfusions (monthly), iron chelation medicines, routine labs, and doctor follow-ups.
Gene therapy aims to correct the faulty gene responsible for Thalassemia by inserting a functional copy into the patient’s stem cells. It’s a one-time treatment, offering the potential for a permanent cure. Currently available only in select trials or specialized centers in India.
- Potential Cure – May eliminate the need for lifelong transfusions and chelation.
- Improves Quality of Life – Frees patients from frequent hospital visits.
- No Donor Required – Uses the patient’s own modified stem cells (autologous), reducing rejection risk.
- Experimental – Still under clinical trials in India; not yet widely available or approved.
- Expensive – High upfront cost, often not covered by insurance.
- Unknown Long-Term Risks – Long-term effects still being studied.
- USD: $80,000 – $120,000
- KES: KSh 10.4 million – 15.6 million
Cost varies based on trial availability, hospital, and technology used. May reduce in future with scale and approval.
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Upload table as is from word document or anywhere
Upload table as is from word document or anywhere