Contemporary Management of Thalassemia: A Perspective on Current Standards and the Emergence of FDA‐Approved Oral Therapy
Fnu Zainab, Abdul Haseeb Hasan, Muhammad Hafi Abid, Muhammad Ali Abid · 2026
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Description
Zainab and co-authors published this 2026 perspective on the modern management of thalassemia, an inherited blood disorder that cripples red cell production. Lifelong care rests on regular transfusions, drugs that strip out the iron those transfusions leave behind, and wide supportive care; stem cell transplant can cure a lucky few. Newer gene therapies remain fenced in by cost, access, and unproven long-term safety.
The review walks through standards for both alpha and beta forms, then spotlights a newcomer. Mitapivat, an oral pyruvate kinase activator recently approved by the FDA, coaxes red cells to work better. Drawing on guidelines, trials, and regulatory sources, the authors weigh where the pill fits beside luspatercept, hydroxyurea, and gene therapy. Their message is cautious optimism.
Catalog record
| CREATOR | Fnu Zainab, Abdul Haseeb Hasan, Muhammad Hafi Abid, Muhammad Ali Abid |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 30 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |