Author: Dr. Nita Radhakrishnan, Head of Department, Pediatric Hematology Oncology, Post Graduate Institute of Child Health, Noida; Dr Sandeep Bhalla, AVP, Projects, ECHO India

A genetic blood disorder that affects the production of hemoglobin, Beta Thalassemia is a chronic condition that requires lifelong treatment. Even today, it is a major public health concern in India, with an estimated 10,000 to 15,000 children being born with Thalassemia Major every year [1].

The economic burden of Thalassemia treatment in India is also substantial for both individuals and the healthcare system as a whole. The treatment cost is quite high, and the financial strain can be rather overwhelming for families, particularly those from weaker socio-economic backgrounds.

At present, Hematopoietic Stem Cell Transplant (HSCT), more commonly known as Bone Marrow Transplantation (BMT), is the only cure for children born with the disease [2]. However, it is out of reach for many, largely owing to the following three factors:

  • The high costs associated with a Bone Marrow Transplantation, which can range anywhere from 10,00,000 to 60,00,000 depending on the type of donor and complications faced.
  • Lack of Bone Marrow Transplant (BMT) facilities and centres, especially in remote and underserved areas.
  • Non-availability of an HLA (Human Leukocyte Antigen) matched donor.

As standard of care, Thalassemia patients are prescribed regular blood transfusions, followed by chelation therapy to manage iron overload. While the Government of India ensures the free provision of blood & blood components and chelation therapy to those fighting Thalassemia, Sickle Cell Anemia and related disorders [3], families still find is difficult to access standard treatment due to non-uniformity of care provided in tier 2 and 3 cities as well as the out of pocket expenses that are incurred despite all the support. Many families still travel more than 300-400 kms to reach a center that is capable of providing transfusion and chelation services to these children, which add to additional expenses and loss of wages for the caretaker.

Thus, individuals end up paying anywhere between INR 1,050 and 4,500 [4] for a single blood transfusion, adding up to significant expenses over time. Similarly, iron chelation therapy, which is administered to remove excess iron caused by blood transfusions, is also expensive and often not accessible even in metros. Moreover, the cost of medications, laboratory tests, and equipment can be prohibitively high for most individuals, particularly those from economically disadvantaged backgrounds, and this financial burden may lead to incomplete or irregular treatments, significantly compromising patient outcomes. To add to this, there are many families, who have more than one child with the same disease, thus pushing them further down the poverty spiral.

Another critical challenge is the shortage of well-trained health professionals, including pediatric/adult hematologists and pediatricians as there is a need for life long commitment towards these patients. They are to be monitored regularly for complications related to both disease and iron overload, hence brining in the need for dedicated treatment centers with medical team that is familiar to the issues faced by these children.

The comprehensive care team typically is important for -Evaluating the growth and development of children with thalassemia -Monitoring for iron overload, initiation of early chelation therapy and regular screening for the status of iron loading in different organs -Screening for transfusion transmitted infections such as Hepatitis B, C and HIV -Need for joint clinics with cardiology, gastroenterology, dental, endocrinology and other supporting departments to ensure early diagnosis of complications and appropriate management -Providing psychosocial support and counselling to patients and their families -Genetic counseling of extended family to detect carriers and prevent the birth of another child with same disease -Ensuring continuity of care into adulthood

The treatment cost for genetic diseases like thalassemia and hemoglobinopathies falls outside the scope of medical insurance programs currently. Often, supplementary expenses such as consultations, medications other than chelation, diagnostic tests and hospitalization also add to already overbearing expenditure for the care of a child with this disease. As a consequence, patients’ families are forced to choose between providing necessary medical care and meeting basic needs, such as food, housing and schooling.

The economic burden of beta thalassemia treatment also poses significant implications for the Indian healthcare system as a whole. This is because high treatment costs result in delayed medical care or even inaccessibility in some cases, leading to severe complications and increased hospitalizations, further adding to the health system’s burden.

Today, inadequate funding, shortage of trained health workers, and lack of infrastructure are only a few challenges facing the Indian healthcare system. Despite provision for funding thalassemia care, there is a need to provide training of manpower, to ensure uniformity in healthcare delivery across the country. The need of the hour is to increase awareness and education around Thalassemia, both within the medical fraternity and among the general public. This can encompass information on the common signs and symptoms of the disease as well as the significance of early diagnosis, including genetic counseling [5] and treatment. Furthermore, there is an urgent need to increase access to affordable healthcare, primarily in remote regions of the country.

The Prevention & Control of Thalassemia and Hemoglobinopathies: A Collaborative Effort

As a complex genetic disorder and global health concern, Thalassemia prevention requires a multi-faceted approach, including unified effort. To address some of the challenges surrounding Hemoglobinopathies in India, ECHO India, PGICH Noida, Kolkata Municipal Corporation, Brihanmumbai Municipal Corporation, and the Indian Academy of Pediatrics joined hands to launch a capacity-building initiative.

The Post Graduate Institute of Child Health (PGICH) Noida, serving as the national hub, initiated the Training of Trainers (ToT) on 11 April 2023, with the primary objective of establishing a uniform approach for Thalassemia treatment for children and providing a strong impetus to the prevention of blood disorders.

Designed to upskill over 50 trainers and 350 Medical Officers (MOs) in a span of 3 years, the formal training for the state of West Bengal, Maharashtra, and Madhya Pradesh, commenced in May 2023. These will be conducted in partnership with the Kolkata Municipal Corporation (KMC), Brihanmumbai Municipal Corporation (BMC), and NHM Madhya Pradesh, with the Indian Academy of Pediatrics as the academic partner.

Today, collaboration is crucial to create a unified front against life-threatening genetic disorders, including Beta Thalassemia. By working together, we can maximize the impact of prevention-focused initiatives, improve access to healthcare services, and ultimately reduce the disease burden on patients, their families, and society at large.