Hematopoietic cell transplantation (HCT) is the only treatment that offers reasonable control for a cure for thalassemia at this time. HCT relies on high-dose chemotherapy to eliminate thalassemia-producing cells in the marrow. It replaces them with healthy donor cells from bone marrow or umbilical cord blood. It is usually taken from a human-leukocyte antigen (HLA) match: an identical sibling.

This therapy should be chosen by all the patients who have a suitable donor. Early referral to a transplant center is recommended, as HCT has a better outcome in younger patients.

What are the major risk factors involved?

There are some risk factors that are needed to pay attention to. This makes the treatment a necessary condition for some, these factors are:

• age of the patient
• adequacy of chelation
• the presence or absence of liver fibrosis
• the presence or absence of hepatomegaly

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. Alternative immunosuppressive preparations and therapy are being studied to decrease graft-versus-host disease and improve graft survival. Pregnant mothers of affected children are more frequently undergoing prenatal diagnosis for thalassemia and determining a fetal HLA typing on the prenatal sample.