For a rare, disabling disease like SPS, HSCT may provide an important option for patients, moving forward. “A lot of people hear about it from friends instead of their doctors,” adds Ms. Blake. “It is definitely a good thing for doctors to have HSCT on their radar as a potential therapy.”
HSCT & Systemic Sclerosis: Julie Gould is a 46-year-old woman, a nurse and a mother of three. After several years of misdiagnosis, she was eventually diagnosed with systemic sclerosis in 2015. She experienced extreme fatigue and swelling of her hands and feet. Her lung function was beginning to deteriorate, and she also was diagnosed with gastric antral vascular ectasia (GAVE).
“All of the information that I had seen on the Internet and that my local rheumatologist had given me was pretty dismal. It pretty much told me that if I took good care of myself and watched my blood pressure that I would be lucky if I lived five years,” Ms. Gould notes. “My mom and I were obsessed about finding information about this disease. We just happened to come across an article on stem cell transplant, and we started researching it.”
Eventually, she learned about the systemic sclerosis trial at Northwestern. During an open enrollment period, she switched to an insurance plan that would cover a clinical trial for stem cell treatment. She underwent HSCT in summer 2016.
Ms. Gould had the transplant before her lung function had deteriorated significantly. Because of her borderline symptoms, her medical team offered her the option of waiting to have the transplant. Ms. Gould opted to proceed. She explains, “I wanted to ahead and have it because I didn’t want to get any worse. Because the transplant only stops the progression—it doesn’t necessarily improve any of the conditions that you have that have already gotten bad.”
Ms. Gould notes, “I didn’t have a lot of the issues that some of the people had. I pretty much went through it without a lot of complications.” She believes part of that is because she had the procedure before her symptoms became too severe.
Ms. Gould was pleased with the outcome of her HSCT, and she recommends it to others in a similar situation. Her pulmonary function tests have stabilized, and she notes, “My feet are the size that they were before I got sick. I do feel so much better.”
Ms. Gould believes it is important for doctors to take a balanced approach when discussing HSCT with their patients. “I have taken care of oncology patients in the past as a nurse, so I knew how sick those patients can be. So I can see why they would not be an advocate for stem cell transplant from that point of view.” However, Ms. Gould believes it is important for doctors not to discourage patients about the procedure prematurely. Ms. Gould sought treatment even after both of her local doctors discouraged her from pursuing HSCT because they saw it as an experimental procedure. “I was willing to try something somewhat experimental,” she says.
SSc HSCT Research Update
To date, systemic sclerosis is the rheumatic autoimmune disease with the most empirical support. Many studies have provided evidence that HSCT can potentially provide effective treatment for systemic sclerosis.15-20 The ASTIS trial was a Phase 3 randomized trial of 156 patients with diffuse cutaneous systemic sclerosis. HSCT in these patients gave a significant long-term survival benefit, although it was associated with increased treatment-related mortality in the first year after treatment.21
