New Delhi: Several districts in Gujarat, particularly in the west-central region, have long been known as the global epicentre of thalassemia, a genetic blood disorder that causes severe anaemia due to a deficiency or absence of haemoglobin which is essential for carrying oxygen in the blood.
For patients, mainly children, with severe forms of the disease, a bone marrow transplant (BMT) is the only chance at a normal life. However, the costly life-saving procedure has remained out of reach for many all these years.
Now, there’s a ray of hope: A newly opened state-of-the-art centre in Ahmedabad that offers treatment free of charge to those in need.
Ahmedabad-based Health1 super-speciality hospital on 8 February opened India’s second centre offering crucial BMT services to paediatric thalassemia patients.
The 10-bed dedicated centre – set up by DKMS, one of the world’s largest international blood stem cell donor centres, and Sankalp India Foundation, a Bengaluru-based not-for-profit that works in thalassemia management – promises to meet growing demand for stem cell transplantations in the region.
The two organisations, along with not-for-profit Cure2Children, have been running a similar centre in Bengaluru since 2021. So far, 460 children with thalassemia have received treatment at the centre.
“For such patients, bone marrow transplantation offers the only curative option, but access remains limited due to financial and logistical barriers,” Sankalp India Foundation president Rajat Kumar Agarwal told ThePrint. “The idea is to develop the newly opened centre as a specialised thalassemia BMT centre and cater to the needs of children suffering from the life-threatening disease in the region.”
Apart from 10 specialised pediatric BMT beds, the new centre also has apheresis care facilities – for the removal of specific blood or plasma components – and an outpatient unit.
The facility has more than 26 trained healthcare professionals, including four doctors and 14 specialised nurses, to provide advanced care for thalassemia patients.
A key feature is that the centre will also gather clinical data to build a record of treatment histories and interventions for thalassemia patients in India.
An estimated 12,000 children are born with thalassemia major, a severe form of the disease, in India each year, accounting for about 10 percent of global cases. The majority are in states such as Gujarat, Maharashtra, Punjab and West Bengal. A higher prevalence of the disease is also seen in particular communities in these states.
Children with thalassemia often require lifelong blood transfusions. Without proper treatment, many do not survive beyond the age of 20.
Dr Elke Neujahr, global chief executive of DKMS, said the new facility was particularly significant given the disease’s prevalence in the region. “We dream of giving every child suffering from thalassemia in India a second chance at life – a chance to smile, grow, and embrace the future they deserve,” she said.
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Limited options, barriers to access
Thalassemia is one of the most common hereditary diseases in the world. It is classified into two main types—alpha and beta—depending on which globin chain, the building block of haemoglobin, is affected.
India doesn’t have a national registry of thalassemia, but experts estimate that nearly 42 million Indians are thalassemia carriers or have thalassemia minor. Carriers have one faulty gene that causes thalassemia but don’t have the disease itself.
Children born to parents who both have thalassemia minor or major, however, have the disease from birth. It can either be moderate or severe, also known as transfusion-dependent thalassemia (TDT), when they need regular transfusion of haemoglobin to stay alive.
Beta thalassemia is the most common form requiring regular blood transfusions. Patients with TDT require lifelong blood transfusions and iron chelation therapy to manage iron overload, which can cause serious complications such as impaired growth, delayed puberty, hormonal imbalance and damage to the heart, liver, kidneys and bones.
Experts say these complications can severely affect the quality of life and pose significant health risks. Without treatment, severe beta-thalassemia can be fatal, often before the age of three.
Allogeneic stem cell transplants, in which healthy blood stem cells from a donor are used to replace bone marrow that’s not producing enough healthy blood cells, remain the cure for beta-thalassemia major patients.
Scientific evidence suggests that BMT has an over 90 percent success rate in patients with beta-thalassemia. Moreover, it is less expensive than conventional life-long treatment.
“People who receive blood transfusions tend to accumulate extra iron that the body can’t easily get rid of and this iron can build up in tissues, which can be potentially fatal,” explained Dr Nitin Agarwal, head of department, donor request management, DKMS Foundation India.
“Such patients, therefore, require chelation therapy or removal of iron from the body.”
The most effective treatment though is a blood stem cell transplant which can give patients a second chance at life, he said.
In practice, however, only about 30 percent of patients find a compatible donor. The success rate of the procedure depends on their age and the quality of clinical management received throughout their lives.
BMT outcomes tend to be better in young children. However, its affordability and access remain a challenge. The procedure costs about Rs 10-50 lakh in private facilities and a few centres offer the treatment.
A 2019 analysis of stem cell transplants showed that between 2012 and 2016, centres reporting to the Indian Stem Cell Transplant Registry (ISCTR) performed only about 960 BMT procedures for thalassemia, mainly due to a lack of access to patients in need.
Coal India, a Union government subsidiary, through its corporate social responsibility initiative started several years ago, supports BMT for thalassemia patients. However, the service is restricted to patients based on strict financial and clinical criteria.
“As a result, many patients who can immensely benefit from the procedure cannot access it and are doomed to a life of suffering. Our BMT centre will not make any discrimination based on these specifications and will offer the service to all paediatric centers with matching donors,” Agarwal said.
DKMS CEO Patrick Paul stressed that prenatal screening and genetic counselling are crucial tools to prevent thalassemia because it is a genetic disease. “These measures should be widely utilised to prevent strain on available resources,” Paul said.
Disclaimer: The author attended the opening of the Paediatric Bone Marrow Transplant facility at Health1 Super Speciality Hospital in Ahmedabad at the invitation of DKMS
(Edited by Sugita Katyal)
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