A Pilot Study to Evaluate the Safety and Efficacy of Treprostinil in the Treatment of Calcinosis in Systemic Sclerosis. Rheumatology (Oxford, England) Chung, M. P., Valenzuela, A., Li, S., Catanese, B., Stevens, K., Fiorentino, D., Strand, V., Chung, L. 2021


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