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The Indian Expressfil
06 Oct 2012

Blood transfusions, fortnightly or monthly, have been the only line of treatment for the survival of patients with thalassaemia major " a genetic blood disorder. But a new line is now emerging in the treatment of the dreaded disease in this part of the region and the results are encouraging.

For 10–year–old Uday Pawar, blood transfusions have been a thing of past since 2008. Jatin Sejpal, who had been on blood transfusion for the last 30 years, too, has not required this for 150 days now.

Approved by the Food and Drug Administration of the US several years ago, an anti–cancer drug that contains hydroxyurea along with wheatgrass is used in combination for thalassaemia major patients for raising the haemoglobin level and thereby avoiding blood transfusion.

Says Dr Vijay Ramanan, haemotologist at Ruby Hall Clinic: "Hydroxyurea (HU) enhances foetal hemoglobin (Hb) production. The disease burden is high. Approximately 8 per cent of the population has the thalassaemia trait and there are 13,000 live births of thalassaemia per year. We decided to study the effect of this treatment in patients with beta–thalassemia major. Around 200 patients were given the treatment and there has been a marked elevation of Hb levels among 35 such patients."

He adds: "Even among those who did not respond to the treatment, the immediate impact was that they did not have to frequently go for blood transfusions."

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For Jatin Sejpal (34) of Pune, the combination therapy has worked wonders. "Initially, I used to take 30 blood transfusions every year since I was four years old. To remove the excess iron in the blood caused due to the transfusion, highly expensive medicines had to be taken," he says. "Living with blood transfusions was not easy," says Sejpal, who has gone for a transfusion for five months now.

Uday Pawar’s mother Sangeeta, a social worker at Ahmednagar, says they were amazed at the result of the combination drug treatment. "My son required blood transfusions for a year when he was five years old. But since 2008, there has been no such requirement," she says.

Priyanka Kulkarni (23), who received blood transfusions for the last ten years, faced complications like an enlarged spleen for which she was even operated upon. "From April, I have not required blood transfusions," says the graduate from Pandharpur.

Dr Sameer Mellinkiri, haematologist at Deenanath Mangeshkar hospital, admits that the treatment works well with patients who responded to it. "So far, we too have had examples of patients who have not taken blood transfusion for more than five months," he says.

So why is the response restricted to a few patients only? According to Ramanan, foetal haemoglobin (HbF) has a higher affinity for oxygen than adult Hb and develops in the foetus during the last six months of gestation. As both mother and foetus share the same blood supply, the HbF essentially draws oxygen from the mother’s blood. Hence, the drug tries to stimulate or induce the HbF in thalassaemia major patients, helping them improve their condition.

The reverse switch from adult to foetal Hb is a complex process and the lucky among those thalassaemia major patients benefit to a great extent. If the child has XMN–1 polymorhphism, the chances are higher that this treatment will remove blood transfusion," says Ramanan.

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