How I Determined a Bone Marrow Transplant was the Best Choice for My PNH Treatment
By Selena Burland
When I was diagnosed with paroxysmal nocturnal hemoglobinuria (PNH) and aplastic anemia (AA), I was given several options on how I wanted to manage, treat, and proceed with my next steps from my hematologist. The process was overwhelming, especially when faced with multiple treatment paths, each with potential side effects, long-term management considerations, and possible lifetime medications. The primary options I was offered included medication management, anti-thymocyte globulin (ATG), or bone marrow transplant (BMT), and I was at a crossroads with all three.
Now that I had the information on how to proceed, I had a big decision to make. Below, I’ll break down the options I was given and how I weighed the pros and cons of each.
Common Medication Management Options:
Medications for PNH may include:
Complement inhibitors: These drugs stop hemolysis which include eculizumab (Soliris), ravulizumab (Ultomiris), pegcetacoplan, crovalimab, iptacopan, and danicopan. Eculizumab and ravulizumab are considered the first-line treatments for PNH.
Recombinant Erythropoietin or androgens: These medications stimulate the production of red blood cells, a process known as erythropoiesis.
Anticoagulants: These drugs are used to manage thrombotic complications.
Immunosuppressive agents: These medications promote hematopoiesis during the aplastic phase.
Deferasirox: This iron chelator removes excess iron from the body.
Medications for AA include:
Immunosuppressants: These drugs, such as cyclosporine, can help restore blood cell production by suppressing the immune system. They are often used together with corticosteroids.
Eltrombopag: When combined with an immunosuppressant, this drug can enhance blood cell production, although it has a low response rate, and relapses may occur.
Growth factors: These drugs can help the body produce more blood cells, which can reduce the need for blood transfusions.
Antibiotics: These can be used to treat infections caused by bacteria, fungi, or viruses.
Pros and Cons of Medication Only Treatment:
While some individuals have successfully managed their symptoms solely through medication, this approach typically requires patients to remain on a significant daily medication regimen for life. While this may seem like a simpler way to manage symptoms, I personally did not want to rely on medication for the rest of my life.
Anti-Thymocyte Globulin (ATG) Treatment:
ATG is an FDA-approved drug used to treat moderate and severe aplastic anemia. This antibody preparation, derived from rabbits or horses, targets T-lymphocytes—immune cells that attack bone marrow stem cells in AA. By doing so, it allows the bone marrow to recover and produce new blood cells. It is administered as an IV infusion over four days and typically paired with cyclosporine. Many patients become independent of blood transfusions within a few months and may achieve remission around the nine-month mark.
Pros and Cons of ATG:
Many patients experience positive outcomes with ATG treatment and can remain in remission for years. Most still need one or two daily medications to remain in remission, but it is possible to live a very normal life after. The reason I chose not to pursue ATG treatment alone was due to the risk of relapse and my desire to avoid long-term medication. ATG is not guaranteed to be successful, and some patients initially try this option before transitioning to a BMT if they do not respond. Due to my severity with both conditions, I chose not to risk the possible failure or future relapse associated with ATG treatment.
Bone Marrow Transplant (BMT):
BMT, also known as stem cell transplant, replaces unhealthy bone marrow with healthy stem cells from a donor. Most BMT protocols involve preparatory medications, followed by ATG, chemotherapy, radiation, and finally, the stem cell transfusion. The healthy cells are administered intravenously, where they migrate to the bone marrow and begin producing new blood cells. The transplant process can take weeks to months, and recovery may extend from several months to over a year. During this time, patients are closely monitored for side effects and complications, such as infections and immune issues. Bone marrow transplants are often successful and can cure PNH and AA, but they come with risks, and not everyone is a candidate.
Pros and Cons of a BMT:
Learning that a transplant offers the only potential cure for PNH and AA made this option appealing to me. However, the BMT process entails numerous side effects and possible complications, making it a challenging treatment. Chemotherapy and radiation often guarantee hair loss, which was a significant “con” for me. There is also a common risk of graft-versus-host disease (GVHD). While many patients live without any complications from GVHD, some individuals may experience lifelong effects due to their transplant. I decided to pursue the BMT route because of the severity of my conditions and my hope to achieve a cure and live a normal life without the need for ongoing medications. Although the BMT process was not easy, I do not regret my decision, as I am now living in remission and no longer require medication for PNH or AA.
In conclusion
If you’re trying to assess how you should proceed with your treatment, just as I was, here’s a worksheet you can download with the framework I used to come to my decision. Everyone’s circumstances are unique, so there’s no singular way to proceed with PNH treatment and management, but I hope this helps! If you have other ways you’ve come to your decision, share in the comments - you never know who it could help one day!
