Challenges Thalassemia Patients Face
Challenges faced by thalassemia patients:
- Thalassemia patients face numerous challenges in their daily lives, exacerbating the already complex nature of their condition. Approximately 200,000 individuals are estimated to be affected by Thalassemia, with an additional 5,000 new cases diagnosed annually. However, there is a pressing need for a central registry and the regulation of Thalassemia centers by IBTA or relevant health regulatory authorities.
- The absence or unaffordability of leucodepletion or bedside filters further compounds the difficulties faced by patients. Moreover, the lack of standardized screening for blood products increases the risk of transfusion-transmitted infections. Compounding these issues is the reliance on replacement donors rather than volunteer donors, resulting in a scarcity of blood products
- Additionally, inadequate collaboration between Thalassemia centers and transplant centers leads to a lack of awareness among parents about the option of curative treatment and HLA typing. Consequently, patients are not managed optimally, and even if a suitable family donor is available, their chances of a successful transplant diminish due to financial constraints
- Although iron chelating drugs are available, affordability remains a significant hurdle for patients. These challenges highlight the urgent need for concerted efforts to address the financial barriers and ensure accessibility to comprehensive care for Thalassemia patients.
Health Complications
- Chronic Anemia: Thalassemia major leads to severe and chronic anemia, where the body produces fewer red blood cells or abnormal hemoglobin, resulting in reduced oxygen-carrying capacity. This leads to fatigue, weakness, paleness, and shortness of breath.
- Enlarged Spleen: Due to the excessive destruction of abnormal red blood cells, the spleen enlarges, causing pain and discomfort in the left upper abdomen. An enlarged spleen may require medical intervention or even surgery in severe cases.
- Bone Deformities: Thalassemia major can cause bone deformities and abnormal growth, particularly in the face and skull. This condition, known as “chipmunk facies,” can lead to facial disfigurement and impact an individual’s self-esteem.
- Growth and Developmental Delay: Children with thalassemia major may experience delayed growth and development because of chronic anemia on their overall health and well-being.
- Iron Overload: Frequent blood transfusions, a common treatment for thalassemia major, lead to iron overload in the body. Excess iron accumulates in organs such as the heart, liver, and endocrine glands, causing organ damage and dysfunction.
- Organ Damage: Iron overload and other complications associated with thalassemia major can lead to organ damage, particularly in the heart, liver, and endocrine system. This can increase the risk of heart failure, liver disease, and diabetes.
- Infections: People with thalassemia major have a weakened immune system due to chronic anemia and organ damage. They are more susceptible to infections, which can further impact their health and require prompt medical attention.
- Transfusion-Related Complications: Regular blood transfusions, a key treatment for thalassemia major, may lead to complications such as transfusion reactions, infections, or iron overload, necessitating careful monitoring and management.
- Emotional and Psychological Impact: Living with a chronic and potentially life-threatening condition can have emotional and psychological effects on both patients and their families. Anxiety, depression, and stress are common challenges faced by individuals with thalassemia major.
- Treatment Burden and Cost: Thalassemia major requires lifelong medical management, including regular blood transfusions, iron chelation therapy, and potential bone marrow transplantation and gene therapy. The cost of treatment, along with the physical burden of managing the condition, can be overwhelming for affected individuals and their families.
It is essential to address these problems comprehensively to improve the quality of life for individuals with thalassemia major. Early diagnosis, proper medical care, and support from organizations like the Thalassemia Hope Society can significantly alleviate the challenges faced by patients and their families.