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.