How are Aplastic Anemia, MDS and PNH Treated?

Research and development of new drugs is on-going for all three diseases. In addition, there are several new drugs and combinations of existing drugs currently in clinical trials.

In addition to watchful waiting for the milder forms of the diseases the following treatments may help:

AA: supportive care (e.g. transfusions), immunosuppression using anti-thymocyte globulin (ATG) and cyclosporine, enrolling in clinical trials 4,7

MDS: supportive care (e.g. transfusions of red cells and/or platelets, drugs which may increase red and/or white cell counts), immunosuppression using anti-thymocyte globulin (ATG) and cyclosporine for hypocellular (too few cells in the bone marrow) MDS, lenalidomide for those with MDS-del 5q (a deletion of a section of the q arm of chromosome 5), chemotherapy with azacytidine, enrolling in clinical trials 5,7

PNH: supportive care, anti-coagulants (“blood thinners”), immunosuppression, drug therapies such as eculizumab, enrolling in clinical trials 6,7

You Are Not Alone

Unique challenges come with having a rare or uncommon disease.
Our volunteers can help guide you:

  • through the maze of unfamiliar terminology
  • keep you abreast of the latest treatment options
  • and help you adapt to the chronic health concerns and lifestyle changes and challenges that can follow diagnosis.

If you or someone you know has aplastic anemia, MDS or PNH, contact us. Volunteers with personal experience with these diseases can provide valuable guidance and support.

Support Offered

As a non-profit organization, we do not charge for resources, support or meetings. The donations of our generous supporters allow AAMAC to provide all services at no charge.

We also rely on our Medical and Scientific Advisory Committee made up of Canadian experts and may be able to refer you to helpful resources through a network of organizations developed over more than 20 years.

We can help you with the following:

All volunteers are asked to respect your privacy. You can share as much or as little as you like.

One cure for these diseases is a bone marrow or peripheral blood stem cell transplant. In a transplant, the patient’s own defective marrow is destroyed and replaced with healthy cells from the bone marrow or peripheral blood of an HLA-matched related or unrelated donor. A transplant is a significant undertaking and is not an option for those without a matching donor. Also, in some cases a person’s health or age may preclude them from undergoing the transplant.

Inadequate blood production can be supplemented temporarily with blood transfusions or marrow stimulating drug therapies. For information, ask us for a free booklet about transfusions.

While transfusions may temporarily manage symptoms and improve quality of life, they are not without their own risks. Patients may develop antibodies or experience allergic reactions to the blood. Repeated transfusions can also cause a condition called iron overload because of the iron in the donor blood. This must be treated using iron chelation to ensure internal organs are not damaged from the excess iron build-up.

Infection may also be a great danger to those patients whose marrow is failing to produce adequate white blood cells. Broad-spectrum antibiotics are often used liberally in such patients.

Drugs that suppress the body’s immune system may be helpful for some patients.

The important thing for a patient diagnosed with bone marrow failure disease to know is that there is a great deal of research being conducted in this area and that there are promising new and improved treatment options being developed for the diseases.

Always consult your hematologist or oncologist and coordinate with them before taking any alternative therapies.

1. AAMAC. Understand Aplastic Anemia, Myelodysplasia & PNH.
Available at Accessed February 2018.
2. Statistics Canada. Quarterly Demographic Estimates July to September 2017.
Available at Accessed February 2018.
3. AAMDSIF. Diseases.
Available at Accessed February 2018.
4. Medscape. Aplastic Anemia.
Available at Accessed April 2018.
5. Gangat N, et al. Am J Hematol 2016;91:76-89.
6. Hill A, et al. Nat Rev Dis Primers 2017. doi:10.1038/nrdp.2017.28.
7. AAMDSIF. Frequently Asked Questions.
Available at Accessed February 2018.