Bone marrow transplant for PNH

Overview
A bone marrow transplant is currently the only cure for paroxysmal nocturnal globunaria (PNH). When someone with PNH develops severe complications such as bone marrow failure, severe aplastic anemia, or repeated life-threatening blood clots, a bone marrow transplant may be recommended. Bone marrow transplants are also called stem cell transplants, or specifically hematopoietic (referring to blood-forming cells) stem cell transplants.

There are different types of bone marrow transplants. An allogeneic bone marrow transplant is used for people with PNH. In this type of transplant, cells come from a donor with closely matching genetic cell markers. An ideal donor for a bone marrow transplant is a sibling, but closely matched genetic markers can also be found in other relatives or unrelated individuals. It may take a long time to find a close enough match.

An allogeneic bone marrow transplant works by replacing a patient’s unhealthy bone marrow with healthy bone marrow from a genetically matched donor.

What does it involve?
Before a bone marrow transplant, the recipient’s bone marrow is destroyed with chemotherapy, radiation therapy, immunotherapy, or a combination of treatments. Bone marrow is harvested from the donor and infused into the recipient in a process similar to receiving a blood transfusion.

After receiving a bone marrow transplant, most people with PNH will receive lifelong immunosuppressant medications to prevent side effects.

Constraints
One of the most dangerous risks of allogeneic stem cell transplant is graft-versus-host disease (GVHD). In GVHD, the transplanted donor cells attack the host’s tissues. GVHD disease ranges from mild to potentially life-threatening.

A combination of allogeneic stem cell transplant, chemotherapy performed before the procedure, and maintenance medications can compromise the immune system. There is a risk of developing serious or even life-threatening infections.

For more details about this treatment, visit:

Bone Marrow and Stem Cell Transplantation — The Aplastic Anemia and MDS International Foundation
https://www.aamds.org/bone-marrow-and-stem-cell...

Current Status of Stem Cell Transplantation in Paroxysmal Nocturnal Hemoglobinuria — Journal of Applied Hematology
https://www.jahjournal.org/article.asp?issn=165...