Why finding a stem cell match remains a major challenge for patients with aplastic anaemia

Aplastic anaemia is a life-threatening blood disorder in which the bone marrow is unable to produce enough red blood cells, white blood cells, and platelets. When these blood cells are low in count, the combination is called pancytopenia. While iron deficiency anaemia can often be managed with supplements and a proper diet, aplastic anaemia requires complex medical interventions.

The Indian scenario

Thousands of people are diagnosed with aplastic anaemia in India every year. Studies from India report that a noticeable proportion of patients presenting with pancytopenia are ultimately diagnosed with aplastic anaemia. While the exact cause of aplastic anaemia is often unknown, several factors can contribute to the disorder, including autoimmune diseases and exposure to certain toxins, chemicals, and radiation. A 2015 study reported that aplastic anaemia accounts for a substantial number of bone marrow failure cases in the country. Another Indian review notes that patients in India often present at a younger age than in Western populations. These findings indicate that aplastic anaemia is highly prevalent in India, requiring urgent attention to build awareness.

Treatment options

Treatments are available to manage aplastic anaemia. They vary depending on severity of the condition and the patient’s age. Some options include:

• Blood stem cell transplants, which have potential to give a second chance at life

• Routine blood transfusions

• Medicines that are prescribed to stop the immune system from attacking stem cells in the bone marrow

• Medicines to help the patient’s body make or produce new blood cells.

The catch though, is that regular or routine blood transfusions carry risks: first, the patient needs specially matched blood to manage the condition for a long period; also repeated transfusions need to be monitored to avoid complications such as iron overload and alloimmunisation (immune reactions that have the potential to make future transfusions difficult). Therefore, access to donor management and to blood stem cell registries become critical. However, one of the biggest hurdles patients with aplastic anaemia usually face is finding a matching blood stem cell donor.

Also Read: Kerala Health department to set up bone marrow registry

HLA barrier, registry gap

The success of a transplant depends on a key criterion—a near-perfect Human Leukocyte Antigen (HLA). Transplant physicians look for a ‘10 out of 10’ match to ensure the patient’s body accepts the transplanted cells. However, only about 30% of patients find such a match within their family, leaving nearly 70% dependent on unrelated donors registered in donor databases.

Unfortunately, there is a huge gap in India’s stem cell donor registry. Countries such as Germany and the United States have millions of registered donors, representing a significantly higher proportion of their eligible population compared to India. Currently, 0.09% of the eligible population in India is registered as blood stem cell donors. This means patients and their families have limited options to access the means for a transplant.

India is a diverse genetic pool, with multiple ethnicities and regional groups showing distinct HLA patterns. This further complicates match prospects. When the donor pool is limited, patients from different ethnicities and communities have a slim chance of finding a perfectly-matched donor. This translates to prolonged international donor searches, which can delay medical interventions and put lives at risk, involve high costs, and sometimes, when there is no match at all, the families go through the emotional burden and anxiety of losing their loved ones.

Also Read: Aplastic anaemia registry launched in RGGGH

Why the low registrations

One of the concerning reasons for such low donor registration is misinformation, which underscores the urgent need to raise awareness about the importance of being a potential lifesaver and on the outcomes of blood stem cell donation for the recipient. People also shy away from registering because of the persistent myths around stem cell donation, and different emotions fog their decision-making process, such as fear of pain, long-term health effects, or impact on fertility. In reality, blood stem cell donation is safe. It is voluntary and is similar to donating blood platelets.

India needs a strategic and large-scale effort to bridge this gap. Expanding the existing blood stem cell donor registry pool is of paramount importance and requires a multifaceted approach. Spreading awareness , online and offline, donor registration drives, partnerships with corporates and educational institutions, and participation in community outreach initiatives can all go a long way towards this. Multiple stakeholders have to come together to create a robust donor registry pool, help in leveraging policy through advocacy as well as in building technology and strategic partnerships that can find effective solutions to challenges.

(Patrick Paul is executive chairman at DKMS Foundation India, a non-profit organization dedicated to the fight against blood cancer and other blood disorders. Patrick.paul@dkms-asia.com)