Oct 10th 2018 – New Promising studies from the World of Research
Our colleagues at Benioff Children’s Hospital Oakland California, The UCSF Fetal Treatment Center and the Northern California Comprehensive Thalassemia Center are dedicated to high quality important work to improve the lives of children and families affected with thalassemia. Learn more about their work in clinical trials of in utero stem cell transplantation for fetuses with alpha thalassemia major.
Alpha thalassemia major (ATM), also called Hemoglobin Bart’s hydrops fetalis, is an extremely severe type of thalassemia that presents with anemia in fetuses before birth.
It is common in individuals of Chinese, Filipino, Southeast Asian, and Pacific Islander descent. Without intervention, ATM is almost always fatal for the fetus. However, in utero transfusion (IUT) of red blood cells has been life-saving even in severely ill (hydropic) fetuses with ATM and many of these patients have promising long-term developmental outcomes.
Individuals born with ATM require lifelong monthly blood transfusions and daily iron chelation therapy. These individuals may pursue a cure with a post-natal stem cell transplant, although this approach requires a matching donor and potentially severe conditioning to prepare the patient for the transplant. The UCSF Fetal Treatment Center and the Northern California Comprehensive Thalassemia Center have initiated a clinical trial of in utero stem cell transplantation for fetuses with ATM, using maternal stem cells.
This procedure takes advantage of existing maternal-fetal tolerance during pregnancy and eliminates the need for conditioning. Since the stem cell transplant is done at the same time as an IUT, this approach minimizes any additional procedural risk to the fetus.
Please visit their website at https://fetus.ucsf.edu/alpha-thalassemia for more information about fetal therapies. This site also contains links to translated patient and provider brochures as well as a patient education video.