Can bone marrow transplants help thalassaemia patients improve their quality of life?

As India observes World Thalassaemia Day today, health experts are sounding the alarm over the increasing number of children affected by thalassemia, a life-threatening genetic blood disorder. The theme this year is, 'Together for Thalassaemia: Uniting Communities, Prioritising Patients.' 

Late detection and lack of access to regular treatment are the two main problem areas in addressing Thalassaemia cases among children in India. This inherited blood disorder caused by mutations affecting hemoglobin production, leads to severe anemia and related complications.

Children born with thalassaemia major often require lifelong blood transfusions every 2–4 weeks and iron chelation therapy to prevent organ damage. This number is preventable through early carrier screening and genetic counselling, yet awareness remains low, especially in rural areas.

While prevention is the only sustainable solution, a simple blood test before marriage or childbirth is all it takes could save thousands of lives.

Bone marrow transplants- leading the way forward

Unlike regular blood transfusions and iron chelation therapy—which only manage symptoms—Bone marrow transplants (BMT) replaces the defective blood-forming stem cells with healthy ones, allowing the body to produce normal haemoglobin.

A successful transplant can free a patient from lifelong dependence on blood transfusions, iron overload complications such as damage to the heart and the liver and repeated hospital visits and treatments. The main challenge involved with a BMT transplant is to find a fully matched donor in the first place, which for many takes years. Add to that the high cost associated with the process and the risks of complications like graft-versus-host disease.

Interestingly, newer developments in science are making BMTs accessible - where a fully matched donor was crucial to get a transplant, a few hospitals in India are now offering Haploidentical transplants,  that is the transplant of stem cells from a half-matched (haplo) donor. 

Haploidentical transplant has been a life-saving solution for children suffering from critical conditions that require bone marrow transplants. This breakthrough significantly expands donor possibilities, reduces dependency on fully matched donors and helps to overcome the challenges posed by donor registry shortages and high costs. 

Narayana Health SRCC Children's Hospital is one such in Mumbai that offers the possibility of undergoing BMT with a half-matched donor.

As per a study published in Transplantation and Cellular Therapy, a bone marrow transplant with a half-matched donor led to a 79.4% survival rate one year after transplant. In India, though, this is a new area and requires to be researched further, for accurate data collection. 

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