PNH Treatment Journeys: Real Stories from Patients
Being diagnosed with a rare chronic illness like Paroxysmal Nocturnal Hemoglobinuria (PNH) can be overwhelming. It often comes with a mixture of emotions such as confusion, fear, grief, and sometimes even relief if the road to diagnosis was long. But as many people quickly learn, diagnosis is only the first step. Figuring out what treatment works best for you is deeply personal.
Over the past year and a half, we have spoken with many individuals living with PNH, and one question comes up again and again: “What is the right treatment for me?” As new medications become available, that question can feel even bigger and more intimidating.
The reality is that treatment journeys can look very different. Some people may receive infusion therapies like Soliris or Ultomiris, others take oral medications such as Fabhalta, and some may undergo treatments like ATG or even a bone marrow transplant. Treatment plans can also change over time, and that is okay. New options may become available, or your body and needs may shift.
To help bring real perspectives to that question, four advocates shared their experiences with the treatments that worked for them at different stages in their journey.'
Brandi’s treatment journey
Brandi Lewis was 27 when she was diagnosed with PNH in 2016. At the same time, doctors also diagnosed her with aplastic anemia, something she had previously gone into remission from.
Because of her history with aplastic anemia, “the treatment [she] had was called ATG.” ATG, or antithymocyte globulin. ATG is an immune therapy used to suppress parts of the immune system that may be attacking bone marrow. Now an expert in ATG, Brandi talk about “two types of serums you can have, rabbit or horse serum. The first time I was treated for aplastic anemia, I had the horse serum. When we treated both aplastic anemia and PNH in 2016, we switched and used the rabbit serum.”
The treatment is typically given through intravenous infusion in a hospital over several days. Brandi notes: “I was in the hospital for five days and they infused it intravenously for about 12 hours a day.”
Today she lives in Alabama and works in corporate America while managing life with PNH. “Working and living with PNH has its own battles, but I’m actually doing it,” she says.
Brandi says she is currently treatment-free, continuing to monitor her health while also being blood disorder educator, speaker, and writer on surviving chronic illnesses.
Gail’s treatment journey
Gail Mahalik, an RN of 38 years from Wisconsin, first noticed something unusual when her platelet counts began dropping. “My main symptom was my platelets were dropping,” she explains. At first, her hematologist monitored the change because everything else in her bloodwork looked normal. When her platelets continued to fall, additional testing followed, including a bone marrow biopsy. Around the same time, Gail began noticing new symptoms she hadn’t experienced before.
“I had dizziness, numbness and tingling sensations, cold sensations, and sometimes shortness of breath,” she says. “There were times I felt like I might pass out because I was getting so lightheaded.”
After further testing, including screening for PNH, doctors confirmed the diagnosis.
Gail’s treatment began with Ultomiris infusions every eight weeks, though she only received three doses before her hematologist transitioned her to Fabhalta, an oral medication taken twice daily, which she takes to this day.
Since starting treatment, she says the difference has been noticeable. “I still have mild symptoms, but they’re nothing like before.” Today, Gail continues working full time while managing PNH and monitoring her blood counts with her care team. When her platelet levels drop significantly, she occasionally receives IVIG infusions to help stabilize them.
Jerri’s treatment journey
Jeri Keiller has been living with PNH for more than four decades. She was diagnosed in 1984 after blood work during pregnancy revealed something unusual. Early in her journey, she was also treated for aplastic anemia. “I had ATG treatment and was in the hospital for 10 days, 24 hours a day,” she recalls, reflecting on a time when treatment options were far more limited.
Over the years, her care evolved as new therapies became available. For many years she managed symptoms with prednisone because, as she explains, “it helped keep my red cell counts up and my platelets stable.”
Eventually, targeted therapies specifically for PNH became available. Jeri spent more than a decade on Soliris before deciding to switch treatments.
“I was on Soliris for about 12 years, and last year I switched to Ultomiris mostly because I don’t have to go as often,” she says.
After living with PNH for more than 40 years, Jeri’s story reflects just how much treatment options have changed over time.
Selena’s treatment journey
Selena Burland was diagnosed with both aplastic anemia and PNH in 2022. At the time, her condition was severe and she was heavily dependent on blood transfusions.
Because of this, her care team recommended moving quickly to a bone marrow transplant.
“I went straight to a bone marrow transplant,” Selena explains. The treatment included “chemo, radiation, ATG, and then the bone marrow transplant itself.”
For Selena, the transplant made the most sense given where she was in her condition at the time. She approached the decision step by step, balancing long-term health goals with plans for the future. With hopes of having children one day, Selena felt unsure of what a BMT might mean for her ability to become a mother. She knew the first step was to focus on stabilizing her health. Selena underwent her bone marrow transplant in February 2023, and after recovery, her journey took a joyful turn. She got her groove back, got her workout routine back, and YES, got pregnant!
She welcomed her first baby in December 2024.
Today, Selena says she is doing well. “Now I’m completely medication free and doing really good. My blood levels and counts and everything are really good.”
Watch the full PNH Panel with Selena, Gail, Jerri, and Brandi here:
