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Indian medical experts are concerned over curative options to battle sickle cell disease. There is paucity of research for new drugs although options for early stem cell transplant are seen help patient with severe sickle cell disease from organ damage, even as affordability is the biggest issue.
Sickle cell disease, an inherited blood disorder, is a major health burden in India. Chhattisgarh, Bihar, Uttar Pradesh, Madhya Pradesh, Jharkhand, Assam, Meghalaya, Arunachal Pradesh and Rajasthan are reporting the highest prevalence of this disease.
On the occasion of the World Sickle Cell Day observed annually on June 19, India is estimated to have the second highest national burden of sickle cell disease, after Nigeria. The disease is reportedly most prevalent in central part of India which is referred to as the ‘sickle cell belt’.
There are as many as 2.5 million carrier of the gene (haemoglobin AS) in India that can lead to sickle cell disease, with more than 1,25,000 actual patients spread across the country, with a much higher incidence in the tribal belt of the country. Sickle cell disease is associated with a significant risk of morbidity and premature mortality, especially among children. While in the west, a child receiving comprehensive care in high-resource settings has an estimated 99% survival into adulthood. However, in India, according to an ICMR study, about 20 per cent of children with sickle disease died by the age of two, and 30 per cent children with sickle cell disease die before they reach adulthood.
Dr. Biju George, Professor & Head, Department of Haematology, CMC Vellore, said “People with Sickle Cell Disease, may beat a risk of progressive organ damage, impaired quality of life, considerable morbidity in childhood, and risk of premature mortality in adulthood . Patients undergoing regular life-long blood transfusions, have the best chance of survival and cure with a blood stem cell transplant. This transplant can come from a sibling or a family member. However, there is only a 30% chance of finding a "matched" sibling donor in the same family. The remaining 70% patients look for a matching donor through a stem cell registry or donor center- a database of voluntary donors between the age group of 18 to 50 years.”
Dr. Govind Eriat Nair, consultant hematology hemato-oncology and bone marrow transplant, Gleneagles Global BGS Hospital, Bengaluru, noted that if there is a fully HLA matched donor in the family, there is a 90-95% chance of cure with higher cure rates in younger children of below 12 years of age. However, due to underrepresentation of Indians in the global donor data pool, patients are unable to find a match on time.
There is low awareness about the disease. Genetic counseling and newborn screening are the way forward. Effects of endogamy, consanguinity and prenatal counseling needs to be addressed in primary screening, he added.
According to Patrick Paul, CEO, DKMS BMST Foundation India said, “Sickle cell has variance and only the severe form needs a stem cell transplant. But due to the misconceptions and lack of awareness about blood stem cell donation, Indians are highly underrepresented in the global donor pool. This situation can only be changed by recruiting many more potential blood stem cell donors from the Indian ethnicity. So, far we have registered over 60,000 potential donors and helped 60 patients with second chance at life.
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