We evaluated the safety and efficacy of oral treprostinil in preventing progression of SSc-associated calcinosis.This prospective open-label study enrolled 12 SSc patients meeting 2013 ACR/EULAR classification criteria with confirmed clinical and radiographic evidence of?=?1 calcinosis deposit in the hands. Patients received oral treprostinil for 1?year. Primary endpoints were safety/tolerability and percentage of patients without radiographic progression of calcinosis at 1?year (<25% increase in Scleroderma Clinical Trials Consortium radiographic score). Secondary endpoints included 1-year changes in Scleroderma HAQ (SHAQ), Cochin Hand Functional Scale, Medical Outcomes Survey Short Form 36 (SF-36), Raynaud Condition Score, and patient/physician assessment of calcinosis severity.Twelve female patients were enrolled, half with diffuse cutaneous disease; median age was 55 (range 35-68) years. Five patients completed the study. Seven patients withdrew due to intolerable adverse effects (n?=?3), intercurrent unrelated illness (n?=?2, cirrhosis, cancer), progressive SSc (n?=?1), and personal reasons (n?=?1). Most patients developed headaches and gastrointestinal adverse effects. Four of 11 (36%) patients with 1-year follow-up hand radiographs experienced progression of calcinosis. Of 5 who completed treatment, calcinosis was stable in 4 (80%) with progression in 1. Based on SF-36 Physical (PCS) and Mental (MCS) Component and Domain scores, transition question, and SF-6D utility score, all patients who finished the trial reported overall improvement or no change compared with baseline.Oral treprostinil was poorly tolerated in SSc patients with calcinosis. Of 5 patients who completed treatment, most (80%) had documented stability of calcinosis on hand radiographs at 1?year.NCT02663895.
View details for DOI 10.1093/rheumatology/keab810
View details for PubMedID 34718447