BERLIN—Two studies presented here show promising new approaches in systemic sclerosis that could help with treatment and prediction of worsening symptoms.
Investigators out of the United Kingdom presented findings on a goat serum as a new treatment for established diffuse cutaneous systemic sclerosis (SSc) and a team from Belgium and Italy showed how a nailfold test might be helpful in predicting future organ involvement in several types of SSc. The work was presented in a session at the European League Against Rheumatism (EULAR) 2012 Annual European Congress of Rheumatology, held June 6–9.
Serum from Goats Could Combat SSc
Researchers from the University College London treated 20 subjects with diffuse cutaneous SSc—10 with goat serum and 10 with a placebo. Subjects must have had diffuse SSc for at least three years and no longer be on disease-modifying antirheumatic drugs, and they could not have had a previous lymphoma or any malignancy in the past five years. They also could not have active tuberculosis or hepatitis B or C, any opportunistic infections, or any progressive or active organ disease.
The hyperimmune caprine serum product, called Aimspro, is derived from serum obtained from a herd of specially vaccinated goats, certified as scrapie-free, in Tasmania. It has already been given Orphan Drug Approval for chronic inflammatory demyelinating polyradiculoneuropathy and motor neuron disease in Australia and for amyotrophic lateral sclerosis in the U.S.
The subcutaneous Aimspro injections were given twice weekly, the first d
Niamh Quillinan, MD, clinical research fellow at University College London, said the findings establish safety and feasibility, with a similar number of serious adverse events in both groups (six events in three patients in the placebo group and four in three patients in the goat serum group). Two subjects withdrew due to adverse events in each group.
Researchers found that the mean skin score fell by 1.4 in those on active treatment after 26 weeks but got worse in the patients on placebo, by an average of 2.1 points. The study wasn’t large enough for statistical significance on that point. However, analysis suggested a clinically meaningful improvement in half of the patients on active treatment, compared with one in the placebo group (p=.01).
There are logistic challenges with the drug, since it has to be stored at -20 degrees Celsius (-4 degrees Fahrenheit).
“These data confirm the safety and tolerability of a novel biological agent in a complex multisystem autoimmune rheumatic disease,” Dr. Quillinan said. “Improvement in 50% of subjects receiving active medication versus 10% on placebo warrants further investigation.”
Test to Predict Organ Involvement in SSc
Researchers at Ghent University Hospital in Belgium and University Sapienza and the University of Genova in Italy are proposing that nailfold video capillaroscopy (NVC) might be able to predict severe clinical organ involvement in systemic sclerosis patients.
NVC was performed to get baseline levels in patients with systemic sclerosis—including the limited cutaneous, and diffuse cutaneous systemic sclerosis, and limited systemic forms—with the capillary patterns categorized as early, active, late, or normal, and non-specific changes.
At baseline and at a future visit one-and-a-half or two years later, a clinical evaluation was performed for nine organ systems that were rated 0 to 4 on the Medsger scale, with a score of 2 or greater considered “severe.”
The baseline NVC analysis was done on 66 consecutive subjects, with 8% categorized early, 38% as active, 41% as late, and 14% as normal, said Vanessa Smith, MD, PhD, a rheumatologist and chief of clinics at Ghent University Hospital.
Fifty-eight of the subjects came in for a follow-up visit, and analysis of organ involvement was done for 55 of them (there was missing baseline data on disease severity for three patients).
Of the seven with normal NVC baseline readings, there was no new severe involvement of organs. Of the five in the early baseline severity category, two had new severe organ involvement, along with 10 of 20 in the active group and 13 of 23 in the late group. An association was seen between worsening baseline NVC patterns and novel severe organ involvement at 1.5 to two years (p = .01).
Of those with novel severe organ involvement, most instances occurred in the peripheral vascular system (12) and the lungs (6). Overall, 25 of the 55 subjects had new severe involvement of organs.
The data in this study were underpowered to actually predict where future problems are more likely to appear. Later, researchers reperformed the analysis in 82 consecutive Italian patients, yielding a similar odds ratio as the previous study. They then had enough events to analyze peripheral vascular disease separately, and concluded that worsening scleroderma patterns at baseline can predict new future digital trophic lesions.
Dr. Smith said she hopes the study spurs other work.
“These data,” she said, “may instigate us to multicentrally, prospectively collect data to detect baseline NVC predictors of well-defined, novel future organ involvement.”
Thomas Collins is a freelance medical writer based in Florida.
