Abatacept in early diffuse cutaneous systemic sclerosis – Results of a phase 2 investigator-initiated, multicenter, double-blind randomized placebo-controlled trial
Arthritis & Rheumatology Jul 31, 2019
Khanna D, Spino C, Johnson S, et al. - Via a 12-month, randomized, double-blind, placebo-controlled trial with 88 individuals randomized in a 1:1 ratio to either abatacept 125 mg subcutaneous or matching placebo, stratified by duration of diffuse cutaneous systemic sclerosis (dcSSc), the researchers evaluated the safety and effectiveness of abatacept in patients with dcSSc. At 12 months, the adjusted mean change in modified Rodnan skin score (mRSS) was -6.24 and -4.49 units in the abatacept and in the placebo, respectively, with an adjusted mean treatment difference of -1.75 units. Two secondary outcome measure was clinically and statistically important supporting abatacept. In relation to abatacept, a greater proportion of placebo subjects needed escape therapy. For the Inflammatory and Normal-like skin gene expression subsets, the drop in mRSS over 12 months was clinically and significantly higher in abatacept than the placebo. Including two and one deaths, respectively, 35 people in the abatacept and 40 in the placebo had adverse events. Hence, abatacept was well tolerated, however, the variation in mRSS was not statistically important, in this phase 2 trial. Secondary outcome measures, including gene expression subsets, exhibited some evidence in favor of abatacept. Moreover, this should be validated in a phase 3 trial.
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