Weighing the pros and cons of aplastic anemia and PNH treatment
Being diagnosed with two blood disorders made my care analysis challenging
Note: This column describes the author’s own experiences with anti-thymocyte globulin and Soliris. Not everyone will have the same response to treatment. Consult your doctor before starting or stopping a therapy.
When you were diagnosed with a chronic illness, what questions ran through your mind? Did you begin to picture your future differently? I did! When I found out I had aplastic anemia and paroxysmal nocturnal hemoglobinuria (PNH), so many questions ran through my mind. One of the most important ones was basic: What are my treatment options?
I wanted to know the best ways to fight and beat aplastic anemia and PNH. I sat down with my doctor, who presented all of my options. I came ready with a pen and paper to ask questions about each treatment option. I wanted to know their side effects, the time they’d take before my body reacted positively, and the chances my body would reject treatment.
I gathered all of the information I could and gave myself a few days to think through each option. In the back of my mind, I knew that whatever option I chose would send me down a path I couldn’t come back from. I knew I had to take it seriously and decide the best option for me.
My treatment options were complicated for two reasons. Because I was battling two blood disorders, I had to select a plan that treated both. Choosing an option that treated only one of my disorders would make the fight more difficult.
Second, I had been diagnosed earlier, in 2009, with only aplastic anemia. I hadn’t yet been diagnosed with PNH. At the time, I chose to be treated with the immunosuppressive therapy anti-thymocyte globulin (ATG) because I was unsuccessful in finding a matched bone marrow donor. (Bone marrow transplant has been found to be a good option for treating aplastic anemia, according to Johns Hopkins Medicine and other medical experts.)
Thankfully, I went into remission in 2010. This time around, because I was already familiar with ATG treatment, I was concerned about how my body would react to it a second time. As you can see, collecting and analyzing my treatment options made for a complicated process.
According to PNH News, treatments for PNH, with which I was diagnosed in 2016, “aim to reduce the destruction of blood cells that drive its symptoms.” The U.S. Food and Drug Administration (FDA) has approved three medications to treat PNH: Soliris (eculizumab), Ultomiris (ravulizumab-cwvz), and Empaveli (pegcetacoplan). According to the Aplastic Anemia and MDS International Foundation (AAMDS), ATG can also be used to treat PNH.
It’s important to keep in mind that I’m sharing my own personal experience and decision-making process. Every patient is different and reacts differently to treatment. If you’re a patient seeking treatment, be sure to discuss all options carefully with your doctor and other members of your care team so that you can make the best informed decision for you.
Another option is a procedure called hematopoietic stem cell transplantation, sometimes referred to as stem cell transplant or bone marrow transplant. This aims to “reset” the hematopoietic stem cells (HSCs) in a person’s bone marrow. This option was the first we considered.
When evaluating the pros and cons of transplant, the pros included that it could be successful in treating both of my blood disorders. The cons noted that I’d have to consider my body’s possible reaction to it, the timeline of finding a match on the donor registry (the procedure involves collecting healthy HSCs from a healthy donor), and the length of time it’d take for me to go into remission.
As I wrote out the pros and cons, transplant seemed like a great choice. But the drawbacks were at the forefront of my mind, so I spent many days considering whether this option would be the best for me.
According to AAMDS, ATG works to allow an “aplastic anemia patient’s bone marrow to rebuild its supply of bone marrow stem cells, causing blood counts to go up.” The foundation adds that if ATG works, it “usually stops the need for blood transfusions.”
Weighing the pros and cons of this treatment option wasn’t too difficult for me, because, as I mentioned, I’d already been treated with it. The pros were the relatively short length of time to possible remission (often three to nine months) and the treatment delivery time (8-12 hours a day for four days, via IV infusion).
The cons were how my body might react to receiving the treatment a second time, how long I’d remain in remission, and whether I’d still have to manage other symptoms.
The discussion about Soliris was a big one between my doctor and me. Soliris was approved by the FDA for PNH treatment in 2007. There was research about how PNH patients had reacted to Soliris, but I couldn’t find enough to answer all of my questions.
Another factor I had to consider was the dosing regimen, which, according to PNH News, is:
- 600 mg weekly for the first four weeks
- a fifth dose of 900 mg one week later
- 900 mg every two weeks thereafter.
I couldn’t skip a treatment, even if I was traveling. I would’ve had to set up a time and place to receive treatment if I was out of town.
My biggest concern, though, was how long I’d have to take it.
Due to all of these factors, I considered this my last option, particularly because it only came on the market about nine years before my diagnosis. I also wondered if it’d be effective at treating aplastic anemia in addition to PNH.
As I considered every option, my doctor helped to ease my mind by discussing them all with me. He always reassured me that if one option didn’t work, it didn’t mean I couldn’t consider others.
I ended up choosing ATG, which, thankfully, sent me into remission for both my disorders. This treatment was the best option for me, as I knew what to expect.
Were my experiences helpful? Please share in the comments below.
Note: PNH News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of PNH News or its parent company, BioNews, and are intended to spark discussion about issues pertaining to paroxysmal nocturnal hemoglobinuria.