Stem Cell Transplant Curative Treatment For Thalassemia  

KUALA LUMPUR, Dec 30 (Bernama) -- A stem cell transplant involves replacing a patient’s bone marrow with stem cells from a matched donor could cure the patient of thalassaemia, eliminating the need for regular blood transfusions, if successful.

Consultant Paediatrician, Paediatric Haematologist & Oncologist, and Paediatric Transplant Haematologist at Sunway Medical Centre, Sunway City, Dr Mohamed Najib Mohamed Unni in a statement said thalassaemia is a blood disorder characterised by abnormal haemoglobin production.

He said that for those suffering from thalassemia, stem cell transplants could be more cost-effective than lifelong blood transfusions and iron chelation therapy in the long run.

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The transplant process begins with a human leukocyte antigen (HLA) typing blood test to determine donor-recipient compatibility for transfusion-dependent thalassaemia patients with siblings.

"If a complete match is confirmed, the donor must undergo additional blood tests. The recipient also undergoes various tests, including cardiac echocardiography, dental examinations, and other evaluations before transplantation.

"A central line is inserted to facilitate blood collection and medication administration. The patient will receive conditioning chemotherapy before the donor stem cells are infused, and after the stem cell infusion, we will have to wait for the donor stem cells to take their place in the marrow niche and start to grow," he said.

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According to Dr Mohamed Najib, typically, patients remain in the hospital for around six weeks post-transplant, followed by close monitoring in outpatient settings, and if the transplant is successful, patients will not need further blood transfusions anymore.

Commenting on the long-term advantages of stem cell transplants over other treatment options, he said matched sibling bone marrow transplants are a well-established curative treatment for thalassaemia, supported by long-term follow-up data demonstrating their efficacy.

As for the challenges and complications in stem cell transplants for thalassemia, he said patients with a matched sibling donor must adhere strictly to their iron chelation therapy to ensure the best possible outcome following transplantation.

"Potential complications include infections, bleeding, veno-occlusive disease, and graft-versus-host disease," he said.

Dr Mohamed Najib added that enhancing healthcare professionals' awareness of stem cell transplants as a viable treatment option for thalassaemia is a crucial step, and it can be achieved by including discussions on stem cell transplants in thalassaemia workshops, and education days.

“Additionally, it has been as well as incorporated into our National Clinical Practice Guidelines (CPG) on the management of thalassaemia,” he said.

Elaborating further, Dr Mohamed Najib said that patients with major thalassaemia often face iron overload from repeated blood transfusions, requiring iron chelation medications to reduce excess iron, fortunately, oral forms of these medications have now become available, making treatment more convenient and improving patient compliance.

On thalassaemia and its prevalence in Malaysia, he said it arose from reduced synthesis or irregularities in one or more of the globin subunits, and it is estimated that approximately five per cent of the population in this country are carriers of the thalassaemia gene.

According to him, a prevalent misconception about thalassaemia is that families believe having one child with the disease reduces the likelihood of having another affected child in subsequent pregnancies.

“The condition is inherited in an autosomal recessive manner, meaning that if both parents are carriers, there is a 25 per cent chance for each pregnancy that they will have a child with thalassaemia major, a 25 per cent chance of having a normal child, and a 50 per cent chance of having a child who is a carrier,” he said.

He explained that parents who are both thalassaemia carriers may consider In Vitro Fertilization (IVF) with Pre-Implantation Genetic Diagnosis (PGD), in which the father's sperm and the mother's eggs are extracted and fertilised in a laboratory setting, and the resulting embryos are tested for thalassaemia.

"Only carrier or normal embryos are transferred back to the mother, after which the parents will wait to see if a successful pregnancy occurs," he said.

According to Dr Mohamed Najib, children with a family history of thalassaemia or whose parents are carriers of the thalassaemia gene should undergo screening as the method is offered among Form 4 students whose parents have signed the consent form.

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-- BERNAMA