KUALA LUMPUR: A promising treatment involving stem cell transplants could potentially cure thalassemia, a severe blood disorder, according to medical experts. This innovative approach, which involves replacing a patient’s bone marrow with stem cells from a matched donor, may eliminate the need for regular blood transfusions if successful.
According to BERNAMA News Agency, Dr. Mohamed Najib Mohamed Unni, a Consultant Paediatrician, Paediatric Haematologist and Oncologist, and Paediatric Transplant Haematologist at Sunway Medical Centre, Sunway City, emphasized the potential benefits of stem cell transplants for those suffering from thalassemia. He noted that this treatment could prove more cost-effective in the long term compared to lifelong blood transfusions and iron chelation therapy.
The process begins with a human leukocyte antigen (HLA) typing blood test to ensure donor-recipient compatibility. If a complete match is confirmed, both donor and recipient undergo a series of evaluations before the transplantation. Conditioning chemotherapy is administered to the patient before the infusion of donor stem cells. Post-transplant, patients typically remain in the hospital for about six weeks, followed by outpatient monitoring.
Dr. Mohamed Najib highlighted the long-term advantages of stem cell transplants, stating that matched sibling bone marrow transplants are a well-established curative treatment for thalassemia, supported by substantial long-term data. He also acknowledged the challenges and potential complications, such as infections and graft-versus-host disease, while stressing the importance of adherence to iron chelation therapy to ensure successful outcomes.
The expert advocates for increased awareness among healthcare professionals regarding stem cell transplantation as a viable treatment option. This could be achieved through workshops and educational events, and it is incorporated into the National Clinical Practice Guidelines on thalassemia management.
Dr. Mohamed Najib also addressed the issue of iron overload from repeated blood transfusions in major thalassemia patients, noting the recent availability of more convenient oral iron chelation medications.
Discussing thalassemia’s prevalence in Malaysia, he stated that approximately five percent of the population are carriers of the thalassemia gene. He clarified misconceptions about inheritance, explaining that if both parents are carriers, each pregnancy carries a 25 percent chance of producing a child with thalassemia major.
For parents who are both carriers, he suggested In Vitro Fertilization (IVF) with Pre-Implantation Genetic Diagnosis (PGD) as an option to avoid passing on the disease. Moreover, he recommended screening for children with a family history of thalassemia, noting that this is offered to students with parental consent.
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