How can a stem cell transplant help with the symptoms of Alpha-mannosidosis?
A hematopoietic stem cell transplant, or HSCT, replaces the missing alpha-mannosidase enzyme in people with Alpha-mannosidosis. This can improve some of the symptoms of the Alpha-mannosidosis like a large liver and spleen, frequency of infections, and hearing loss. A HSCT does not usually improve the problems with the bones, problems with the eyes, or the problems with mental development and the brain. Since Alpha-mannosidosis is a progressive condition, having a HSCT is considered to be most beneficial early on in life. HSCT is not done routinely and is a risky procedure. There are some reports of people with Alpha-mannosidosis having successful HCST and other reports of people with this condition not having a successful HCST. HCST has significant risks to the person undergoing the procedure. The risks and benefits of HSCT should be discussed with your doctor.
SOURCE: Emory University - Department of Human Genetics in collaboration with ThinkGenetic • https://www.thinkgenetic.com/diseases/mannosidosis-alpha-b-lysosomal/living-with/9955 • DATE UPDATED: 2016-06-14