What is Therapy for Congenital Dyserythropoietic Anaemia and Its Cost?

Therapy for CDA – Gene Therapy, Transplants and Cost Breakdown

Did you know that this rare inherent disorder can lead to liver cirrhosis, heart problems and diabetes? Do not leave Congenital Dyserythropoietic Anaemia (CDA) untreated. CDA requires long-term monitoring and follow-up treatments. Scroll down to the next sections for information on several definitive treatment options for this disease, starting from Rs. 1,000 in India.

What is the Therapy for Congenital Dyserythropoietic Anaemia?

1. Blood Transfusion

Blood transfusion is a necessary treatment option to balance an adequate haemoglobin level and severe anaemia. This option contributes to the immediate amelioration of anaemia. However, frequent blood transfusions can increase the risk of iron overload and necessitate meticulous management and monitoring.

2. Iron Chelation Therapy

This therapy is necessary for individual patients who are undergoing regular blood transfusions.  Iron chelation therapy prevents subsequent organ damage, iron overload, and related complications, like cardiac and hepatic dysfunction.

Iron chelation therapy helps prevent organ damage, including liver disease, by reducing iron overload; however, exact percentages of risk reduction are not clinically established.

3. Bone Marrow Transplantation

Bone marrow transplantation is considered only for severe or refractory cases of CDA; most patients are managed with supportive treatments such as blood transfusion and iron chelation. After measuring the patient's overall health condition, age and assessing the availability of a compatible donor, doctors decide to pursue bone marrow transplantation.

Advanced technique successfully improves patient outcomes. Post-transplantation care can reduce the severity of graft-versus-host disease and the survival rate of patients.

4. Gene Therapy

Presently, gene therapy is an increasingly promising option for treating CDA. The goal of this therapy is to measure and correct underlying gene mutations that cause the disease. Gene therapy for CDA is currently experimental and under investigation; it is not yet an established clinical treatment.

What is the Cost of Congenital Dyserythropoietic Anaemia?

In India, the costs of CDA treatment depend on the complexity of cases and the hospital where you are admitted.

• Blood transfusion ranges from Rs. 10,000 to Rs. 25,000 at a time.
• At the same time, iron chelation therapy costs more than Rs. 90,000 each month.
• In India, the costs of bone marrow transplantation range from Rs. 15 lakhs to a maximum of Rs. 40 lakhs.
• Gene therapy in India starts at 25 lakhs, with an average cost around 50 lakhs, and can go up to Rs. 1 cro

Note: To get the exact pricing of individual treatments, patients must consult a specialist.

Pro Tip: You can buy medical health insurance from Star Health. With a minimum sum insured of Rs. 5,00,000, Super Star insurance plan ensures coverage for expensive treatments, including stem cell therapy for bone marrow transplantation.

What Type of Specialised Care is Needed for Children with Congenital Dyserythropoietic Anaemia?

Doctors focus on enhancing the health and quality of life for children, since CDA treatment options are limited. Adolescents and children with chronic disease anaemia (CDA) receive treatment through iron disorder and rare anaemia programs, ensuring comprehensive ongoing medical management and exploring new treatment approaches.

Is Congenital Dyserythropoietic Anaemia Curable?

Several treatment approaches for congenital dyserythropoietic anaemia focus on managing disease complications and symptoms, since there is no particular curative option. The treatment options focus on eliminating anaemia-related symptoms, such as weakness, fatigue and health complications associated with the production of ineffective red blood cells.

CDA treatment has a goal to manage complications and symptoms. Blood transfusion helps to alleviate anaemia symptoms, whereas medications can reduce the overload of iron. Additionally, stem cell transplantation is the only definite treatment option for patients with severe CDA.