Can thalassemia be treated with bone marrow transplant?

Can thalassemia be treated with bone marrow transplant?

Hematopoietic stem cell transplantation is the only well-established curative treatment of thalassemia major and shows excellent long-term results,4-6 but allogeneic bone marrow transplantation (BMT) is often limited by a lack of suitable donors.

What would a bone stem cell transplant do for a patient with thalassemia?

The purpose of stem cell transplantation is to cure life threatening or chronic diseases, such as thalassemia, using high doses of chemotherapy, followed by transplantation of donor marrow or stem cells. This treatment is an intense one, causing your child’s body to temporarily not be able to make blood cells.

Can thalassemia major Be Cured?

Bone marrow and stem cell transplant from a compatible related donor is the only treatment that can cure thalassemia. It is the most effective treatment.

Can thalassemia minor donate bone marrow?

Individuals with thalassemia trait, mild α-thalassemia, or β-thalassemia minor are suitable, whereas donations in case of intermediate/major forms are generally not recommended. The same holds true for sickle cell anemia. Subjects with sickle cell β-thalassemia minor may be suitable for BM donation.

What is HLA matching?

A process in which blood or tissue samples are tested for human leukocyte antigens (HLAs). HLA matching is done before a donor stem cell or organ transplant to find out if tissues match between the donor and the person receiving the transplant. Also called human leukocyte antigen matching.

Is there any permanent cure for thalassemia?

The most common treatment for severe cases is regular blood transfusions to relieve the symptoms, which continue for life and have side-effects. The only cure currently available for thalassemia is a bone marrow transplant, which is only possible for a small proportion of patients.

Why do bone marrow transplants fail?

Grafts fail when the body does not accept the new stem cells (the graft). The stem cells that were given do not go into the bone marrow and multiply like they should. Graft failure is more common when the patient and donor are not well matched and when patients get stem cells that have had the T-cells removed.

How long can a thalassemia patient live?

“Most thalassaemia patients would live up to the age of 25 to 30 years. Improved facilities will help them live up to the age of 60,” said Dr Mamata Manglani, head of pediatrics, Sion hospital.

What is the prognosis of thalassemia?

The overall thalassemia-free survival of low-risk, HLA-matched sibling stem cell transplantation patients is 85 to 90 percent, with a 95 percent overall survival. While not as effective, new approaches to Class 2 and 3 patients have significantly improved their overall survival.

What are the stem cell options for patients with thalassemia?

Since HLA-matched sibling transplantation in healthy thalassemia patients offers a very high cure rate, stem cell options for families without matched siblings are being studied. Most patients do not have an HLA-matched sibling. Experimental trials with unrelated, matched umbilical cord blood or stem cell transplantation are being conducted.

Do bone marrow transplants improve cancer survival?

In 2010, a team at Fred Hutchinson Cancer Research Center reported a striking improvement in survival for patients who had a bone marrow transplant from the 1990s through the early 2000s. The team’s latest analysis shows that trend has continued.

What is the survival rate for HLA transplantation?

HEMATOPOIETIC CELL TRANSPLANTATION. The overall thalassemia-free survival of low-risk, HLA-matched sibling stem cell transplantation patients is 85 to 90 percent, with a 95 percent overall survival. While not as effective, new approaches to Class 2 and 3 patients have significantly improved their overall survival.