“I see this sort of work as seeking to understand biology and generating new hypotheses that we can go forward with,” says Dr. Gordon.
Limitations & Future Outlook
The study’s small sample size was one of its limitations, and it was underpowered to detect modest differences between cohorts, the authors write. Use of the comparator minimized ability to detect whether belimumab affected clinical outcomes.
In summarizing the study, Dr. Gordon says that, overall, the safety findings were as expected; the skin score results were interesting, even though statistical significance was not achieved; and secondary measures were negative for the most part. This line of study is important to pursue because systemic sclerosis has both the highest mortality and the highest rate of disability of all the rheumatic diseases, she says.
In the past 10 to 20 years, Dr. Gordon says, rheumatologists have “seen dramatic improvements in the lives of many of our patients. Some examples are those with rheumatoid arthritis or psoriatic arthritis who have multiple treatment options. Patients’ experience of those illnesses today vs. 20 years ago is dramatically different.
“Although we’ve made really important strides in the understanding of systemic sclerosis, this condition continues to be a particularly difficult one to treat,” she continues. “The condition is rare and heterogeneous, so it can be challenging to study. Patients with systemic sclerosis urgently need improved therapies. That is a major motivation to look at novel medicines.”
Catherine Kolonko is a medical writer based in Oregon.
Reference
- Gordon JK, Martyanov V, Franks JM, et al. Belimumab for the treatment of early diffuse systemic sclerosis. Results of a randomized, double-blind, placebo-controlled, pilot trial. Arthritis Rheumatol. 2018 Feb;70(2):308–316.
