Scleroderma patients, who are sometimes affected by pulmonary hypertension, are becoming routinely treated with stem cell transplants. Dr. Dinesh Khanna, at the annual European Congress of Rheumatology, previewed the results of the Autologous Stem Cell Transplantation International Scerloderma (ASTIS) trial evaluating 156 severe scleroderma patients randomized to receive a stem cell transplant or cyclophosphamide, a conventional drug used to treat some auto-immune diseases because it decreases the immune system’s response.
“Before and after treatment, patients look completely different,” said Dr. Khanna in a news report. “Some patients [treated with stem cell transplants] you can’t tell that they had scleroderma. But you need to select the right patients.” Initially, of the 79 patients treated with stem cells, 8 died from treatment-related causes, while none of the 77 control patients died when receiving cyclophosphamide. But in another follow-up, 24 control patients died, compared to 16 stem cell patients. Evidently, there was an overall decrease in mortality due to stem cell transplantation.
Patients not only had better survival, but they also enjoyed “large improvements” in their skin score and “substantial improvements” in forced vital capacity. They were also more active, as determined by the Health Assessment Questionnaire Disability Index (HAQ-DI).
Stem cell transplants are an attractive option for scleroderma patients because fibrosis and collagen deposits regress over time following transplantation, and stem cells can help heal prior organ damage.
Yet not everyone is sold on the idea of autologous stem cells for scleroderma treatment. According to Dr. Daniel Furst, a professor in rheumatology at University of California, Los Angeles, “I’m more of a skeptic. I would like to see it reproduced.” Reproducible results may come from the ongoing North American-based Scleroderma: Cyclophosphamide or Transplantation (SCOT) trial. Positive results are necessary to convince both skeptics and insurance companies in order to reimburse patients for the costs of treatment.
In addition, stem cell transplantation poses the risk of substantially suppressing the immune system, leaving auto-immune patients even more vulnerable to developing infections and complications. Dr. Furst stated that “the 10-15% of patients with scleroderma who are the worst” would benefit from stem cell treatment. He believes, “The next step is to move this treatment from the worst patients to those who are less severe. In some centers in Europe, stem cell transplantation is becoming widely used, even for patients with only skin symptoms.”
Dr. Khanna’s results will soon appear in a medical journal. He is currently the director of the scleroderma program at the University of Michigan in Ann Arbor. Previous results from ASTIS were presented during the EULAR 2012 meeting.