In a new article, a multidisciplinary team of international researchers summarized key points and deliberations regarding basic, clinical, and novel therapeutic research made at a World Scleroderma Foundation three-day meeting that gathered senior and junior investigators across research areas, as well as a patient spokesperson.
The report’s main goals were to review current progress and to reach a consensus regarding the overall priorities for clinical and translational research in scleroderma (SSc) over the next 10 years. The target audience for the report, titled “Progress and priorities in systemic sclerosis: the next 10 years – report from the World Scleroderma Foundation” and published in the Journal of Scleroderma and Related Disorders, included researchers, policy makers, private and public funders, and people living with the disease.
Prior to the meeting, participants discussed their opinions regarding key pathways, organ systems, and their respective importance in a two-round Delphi procedure. This discussion on clinical research priorities was followed by a face-to-face meeting, in which the main objective was to reach a consensus on the prioritization of goals.
Overall, participants agreed that prioritizing a single aspect of SSc pathogenesis was not appropriate, and that all pathways — immunological, vascular or fibrotic — were likely to be interdependent. It was agreed that a focus on an integrative approach to SSc pathogenic pathways was the better way to improve understanding of the disease.
Participants also agreed on the need to correlate laboratory research to clinical features and disease classification, namely through a focus on biomarker research, as currently there is a failure in integrating what it is seen in research to the clinical heterogeneity of SSc.
They also suggested that future clinical trial designs and the establishment of outcomes should include patient concerns regarding topics such as fatigue, pain, and quality of life, as those concerns are important, in the patient’s perspective, to improved survival. The inclusion in all studies of serial sampling of tissues, cells, plasma and/or serum, as biomarkers that are reliable and predictive, was also considered of importance.
Consensus further supported an aversion to placebo, expressed both by physicians and patients, and recognized this constitutes a significant barrier to study recruitment. As such, the report advises more creativity be applied to clinical trial design, avoiding placebo as much as possible.
The EULAR (European League Against Rheumatism) standard recommendations were also endorsed in the meeting.
“This document can hopefully aid in prioritizing research in SSc. There is the optimistic realization that novel discoveries may change or expand this list of priorities,” the authors concluded.