Dose allogeneic bone marrow transplant for thalassemia ( stem cell transplantation) cure thalassemia ?
The first curative allogeneic bone marrow transplant or stem cell transplantation for thalassemia was performed in 1982. Since then, thousands of successful transplantation have been reported. Success rates for cure have reported as high as 89%-97%
Before the bone marrow transplant for thalassemia procedure patient needs to be administered chemotherapy to eradicate the thalassemia clone, enhance immunosuppression which will ease the stem cell transplant .
Donor choice is of great importance because transplantation may fail or be lethal resulting from immunological complications. The best results have been obtained with genetically matched (HLA-matched) siblings.
Haploidentical stem cell transplantation for thalassemia – Is an alternative option for children with beta-thalassemia major, particularly when a genetically matched sibling donor is not available.This new technique considered to be superior to transplant between unrelated donors.
Umbilical Cord blood transplantation for thalassemia – Is a recent development and has benefits of the low risk of viral contamination from a graft, the decreased incidence of acute and chronic,graft versus host diseases and easier accessibility.
Because of small number of stem cells in the umbilical cord blood collection , this procedure is being used mainly in pediatric patients.However some patients have received umbilical cord blood transplantation for thalassemia in combination with bone marrow transplant.
Just to mention , Fetal Hb induces and Gene therapy are being investigated as potential alternative to stem cell transplantation for thalassemia
What are the risks of bone marrow transplant or stem cell transplantation for thalassemia ?
There are several risk factors, including hepatomegaly , portal fibrosis, and inadequate iron chelation therapy, that can influence the outcome of bone marrow transplant for thalassemia
Patients are typically classified into 3 risk groups:
- Class 1- Those with no risk factors;
- Class 2, those with 1 or 2 risk factors; and
- Class 3, those with all risk factors.
In general bone marrow transplant for thalassemia in Class 1 & 2 patients is considered to be safer than in Class 3 patients . However advances in conditioning regimens have considerably improved the outcomes of class 3 patients younger than 17 years.
Growth failure and/or hypogonadism and infertility can develop after the chemotherapeutic preparative conditioning for transplantation or secondary to iron excess.
How do i find out the latest treatment options for thalassemia?
As the research for treatment of thalassemia is done across the globe and progressing at a rapid pace , Patient-Help.com cunnects thalassemia patients with experts across the globe . Patients can compare the available latest treatment options free!
Summary on bone marrow transplant or stem cell transplantation for thalassemia –
On the basis of all the available data to date, every patient with a severe form of thalassemia should be offered the option for bone marrow transplant or stem cell transplantation. In addition, a check should be made for genetically matched donors among family members
Although stem cell transplantation for thalassemia the only cure available, its use is still limited because of the relatively high cost and the difficulty in identifying suitable donors and more so availability of this treatment only in select countries.
Patiet-Help is a free service for patients to compare the treatment options for thalassemia available globally so that patients not only find best treatment option but also at the best cost.