A bone marrow transplant (BMT) is the only cure for PNH, however it involves significant risk and it is often difficult for a patient to obtain a well-matched donor. A BMT is considered a “last resort” for a PNH patient and is usually only performed where a patient has an excellent match (twins and siblings are best) or their PNH is immediately life-threatening and/or not responding to other treatments.

A basic explanation of a bone marrow transplant is that a patient’s own bone marrow is replaced by a healthy matching donor’s bone marrow. The patient is usually treated with high dose chemotherapy to destroy the bone marrow tissue and often also anti-thymocyte globulin (ATG) to prevent rejection of the donor cells. Donor cells are collected directly from the bone marrow or blood stream.

There are many complications associated with bone marrow transplants, the most evident being Graft vs Host Disease.

There are many factors to take into account when considering a bone marrow transplant.  We will shortly upload a Resources page with more information.

Disclaimer: This information has been prepared by members of the PNHSAA Inc and has been checked for accuracy by practitioners experienced with PNH. The PNHSAA Inc does not guarantee its accuracy, it is intended as general information only and not to be substituted for medical advice. Please consult your medical practitioner for further information regarding your personal circumstances.