ARE WE FAILING OUR CHILDREN WITH THALASSEMIA?

What is Thalassemia?

Thalassemia is a genetic disorder of the blood found in children. In India over 10,000 children are born each year with the major form of this often a fatal condition. Thalassemia in the severe form is called Thalassemia Major.

Children with Thalassemia inherit the genes from both parents. The treatment needs regular blood transfusions, and the frequency of transfusions affect the normal life of a child with Thalassemia.

The child becomes listless and irritable a few months after birth, as an indication of Thalassemia.  By 6-9 months, most children need regular blood transfusions for survival.

Challenges in life for a child with Thalassemia:

  • Blood transfusion is needed once every 4 to 6 weeks to maintain haemoglobin levels above 12 gm%, and not letting it sink below 9 gm%. Such a transfusion schedule if maintained diligently results in normal growth and development of the child. However, this comes at a cost.
  • But regular blood transfusions that save child can also result in death of the child! Frequent blood transfusions result in excess iron in the blood, and the excess iron gets deposited in critical organs such as the heart and the liver. This can ultimately lead to death.
  • So removing the excess iron from the thalassemic child’s body is a process called chelation. Desferrioxamine was the first drug used for chelation. But Desferrioxamine can only be injected through the veins or under the skin. The children with Thalassemia need small pumps to continuously inject this drug for 8-10 hours usually at night.
  • Children disliked the unpleasant process and often ignore the chelation schedule. This can lead to death. The average survival age for children with Thalassemia in developing countries barely exceeded 20 years.

An oral drug:

Researchers worked for two decades to find an oral drug for chelation (removing excess iron in the blood), to replace the cumbersome injections. Deferiprone was the first oral drug that proved effective, but as is the unfortunate case with Thalassemia, this solution too was double-edged.  Deferiprone had lethal toxicities. It was not approved for use in the western countries but was strangely introduced in India ostensibly due to resource constraints.

A new drug called Deferasirox then produced similar results to the injectable desferrioxamine. Fortunately, this was found to be without side-effects.

Is Deferasirox the solution to save thousands of children suffering from thalassemia in India?

The cart is there, but not the horse – meaning we have an effective oral chelation tablet to remove the fatal excess iron in the blood, but we have a shortage of donated blood!

Voluntary and dedicated blood donation centres in India are not enough to meet the needs of millions of children. India suffers from having poor blood bank infrastructure. More dangerously and tragically, over 50% of children receiving blood transfusions received infected blood. This led to infections such as hepatitis B or C and HIV.

So the children and parents struggle to get the life-saving blood needed for regular transfusions, but improperly screened blood causes other fatal complications for these suffering children. Shortage of safe donated blood prevents Indian children with Thalassemia from leading a normal life like Thalassemia-affected children in western countries.

Bone Marrow Transplantation:

The only effective long-term treatment for Thalassemia Major is the Allogeneic Blood and Marrow Transplantation (BMT) from an HLA-matched family donor. From genetic inheritance, 25% of children should be able to find such a donor within the family.

Yet such a donor is available for a very few patients of Thalassemia since parents often do not have more children once a child with thalassemia major is born. HLA-matched unrelated donors from registries are available to very few patients. The treatment process is there to save the children but the genetic material for the treatment process is not sufficiently available.

In recent years, BMT from half-matched or HLA-haploidentical family donor has been tried in patients with Thalassemia – with poor results. Attempts at haploidentical BMT in children should be carried out only within the premises of a clinical trial.

Narayana Health is one of the few health service providers in the world to have developed innovative approaches for haploidentical BMT, in both adults and children. Narayana Health is undertaking clinical trials for haploidentical BMT in selected patients with Thalassemia.

Thehaploidentical BMT is possible in children with Thalassemia is a process still undergoing trial, and therefore such procedures should not be performed outside approved clinical studies. Early results are promising, but it will take more time before this innovate approach becomes safe enough to be made available worldwide.

Developing a Thalassemia-treatment infrastructure in India

Much needs to done to cure the majority of children suffering from Thalassemia. Frequent blood transfusions and chelation treatment play havoc with their school life and normal childhood.

At the same time, every child born with Thalassemia can live a near-normal life. For that, India must enhance its Thalassemia-infrastructure to provide safe blood transfusions and inexpensive medicines for chelation. Governmental and non-governmental organisations should be involved to ensure such a life-saving infrastructure for children that becomes a reality at the earliest.

There might be hopes of a cure for every child with Thalassemia in future, but the brutal reality at present is of India’s healthcare system failing the children.

Let us resolve today to take the necessary measures to save our children suffering from Thalassemia major.

Dr. Sarita Jaiswal & Dr. Suparno Chakrabarti, Dept of Hematology and BMT, DharamshilaNarayana Hospital and Research Centre

Narayana Health

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