Bone Marrow Transplant for Severe Thalassemia: A Guide for Families

Q: Bone Marrow Transplant for Severe Thalassemia: A Guide for Families

Hello Ms. <br>IRIS： Severe thalassemia (formerly known as Mediterranean anemia) is indeed a troublesome disease. <br>Children usually require lifelong blood transfusions to grow, but later on, they may need chelation therapy due to iron overload damaging various organs. <br>The c

Severe Mediterranean anemia (also known as severe beta-thalassemia)?

I have a 9-year-old niece from Hong Kong who is suffering from severe thalassemia and requires blood transfusions every month.
The doctors in Hong Kong have suggested a bone marrow transplant, but the regulations there state that only blood relatives can donate, which poses practical difficulties for my cousin's family.
Therefore, I would like to inquire about the preparations and application process for undergoing a bone marrow transplant in Taiwan.
Approximately how much would it cost? I have already consulted several major hospitals, but no one could provide answers.
I have the child's medical records; how can I get a doctor to review them? Once a doctor reviews them, can they provide guidance on how to obtain answers to the above questions? Please provide me with some channels for inquiry.
Thank you.

IRIS LIU, 20~29 year old female. Ask Date: 2002/05/10

Dr. Tang Jieru reply Cardiology

Hello Ms.
IRIS: Severe thalassemia (formerly known as Mediterranean anemia) is indeed a troublesome disease.
Children usually require lifelong blood transfusions to grow, but later on, they may need chelation therapy due to iron overload damaging various organs.
The cost of chelation therapy is estimated to exceed 400,000 New Taiwan Dollars annually.
Bone marrow transplantation is currently the best option; however, the donor's bone marrow must match the patient, making it easier to find suitable donors among blood relatives.
For those without familial ties, the chances of finding a suitable match are only a few ten-thousandths.
But don't be discouraged; Hualien Tzu Chi Hospital in Taiwan has the most comprehensive bone marrow database in the world.
I recommend contacting them.
A press release from Tzu Chi Hospital in February of this year indicated that they had just completed a bone marrow transplant for a patient with severe thalassemia, so you can reach out to them.

Reply Date: 2002/05/10

More Info

Bone marrow transplantation (BMT) is a critical treatment option for severe thalassemia, especially for patients who do not respond adequately to regular blood transfusions and iron chelation therapy. In your case, your 9-year-old niece from Hong Kong is facing significant challenges due to her condition, and it is understandable that you are seeking information on how to proceed with a potential transplant in Taiwan.

Understanding Thalassemia and Bone Marrow Transplantation
Thalassemia is a genetic blood disorder characterized by reduced hemoglobin production, leading to anemia and requiring regular blood transfusions. Severe forms of thalassemia can lead to complications such as iron overload, which can damage organs over time. Bone marrow transplantation offers a potential cure by replacing the patient's defective bone marrow with healthy stem cells from a compatible donor, ideally a sibling or another family member.

Preparation and Application for Bone Marrow Transplant in Taiwan
1. HLA Typing: The first step is to perform Human Leukocyte Antigen (HLA) typing for your niece and her family members. This test determines compatibility for transplantation. If a suitable donor is found within the family, the process can move forward more smoothly.

2. Consultation with a Specialist: It is crucial to consult with a hematologist or an oncologist specializing in bone marrow transplants. They can provide detailed information about the procedure, risks, and benefits, as well as the specific requirements for your niece's case.

3. Hospital Selection: In Taiwan, several hospitals specialize in bone marrow transplants. You may want to consider hospitals with established hematology and transplant programs, such as National Taiwan University Hospital, Chang Gung Memorial Hospital, or Taipei Veterans General Hospital. Contact these hospitals directly to inquire about their transplant programs and the necessary steps for international patients.

4. Financial Considerations: The cost of a bone marrow transplant can vary significantly depending on the hospital, the complexity of the case, and whether the patient has health insurance. In Taiwan, the cost can range from NT$1 million to NT$3 million (approximately USD 35,000 to USD 100,000), depending on the specifics of the treatment and post-operative care. It is advisable to discuss financial options with the hospital's billing department and inquire about any available financial assistance programs.

5. Medical Records: To facilitate the consultation process, gather all relevant medical records, including your niece's diagnosis, treatment history, and any previous test results. This information will help the doctors assess her condition and determine the best course of action.

6. Communication with Physicians: When reaching out to hospitals or specialists, you can either call their international patient services or email them directly. Provide a brief overview of your niece's condition and express your interest in a consultation. Many hospitals have dedicated teams to assist international patients, making the process smoother.

7. Follow-Up Care: After the transplant, your niece will require close monitoring and follow-up care to manage potential complications and ensure the success of the transplant. This includes regular check-ups, blood tests, and possibly additional treatments.

Additional Resources
- Patient Support Groups: Consider connecting with organizations that support families dealing with thalassemia and bone marrow transplants. They can provide valuable resources, emotional support, and guidance through the process.

- Online Forums and Communities: Websites and forums dedicated to thalassemia can offer insights from other families who have gone through similar experiences, providing practical advice and emotional support.

In conclusion, while the journey towards a bone marrow transplant can be complex and challenging, with the right preparation and support, it is possible to navigate this process effectively. I recommend starting with HLA typing and consulting with a specialist in Taiwan to explore the best options for your niece. Best wishes to your family during this difficult time.

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