A pilot study to evaluate the safety and efficacy of treprostinil in the treatment of calcinosis in systemic sclerosis. (29th October 2021)

• Record Type: Journal Article
• Authors: Chung, Melody P; Valenzuela, Antonia; Li, Shufeng; Catanese, Benjamin; Stevens, Kate; Fiorentino, David; Strand, Vibeke; Chung, Lorinda
• Is Part Of: Rheumatology. Volume 61:Number 6(2022)
• Page Start: 2441
• Page End: 2449
• Publication Date: 2021-10-29
• Subjects: SSc -- scleroderma -- calcinosis -- cutis -- treprostinil -- prostacyclin -- clinical trial; Rheumatism -- Periodicals; Rheumatology -- Periodicals; 616.723005
• DOI: 10.1093/rheumatology/keab810
• Languages: English
• ISSNs: 1462-0324

Abstract

Abstract: Objectives: We evaluated the safety and efficacy of oral treprostinil in preventing progression of SSc-associated calcinosis. Methods: This prospective open-label study enrolled 12 SSc patients meeting 2013 ACR/EULAR classification criteria with confirmed clinical and radiographic evidence of one or more 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. Results: Twelve female patients were enrolled, half with diffuse cutaneous disease; median age was 55 years (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 five who completed treatment, calcinosis was stable in four (80%) with progression in one. Based on SF-36 Physical and Mental 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. Conclusion: Oral treprostinil was poorly tolerated in SSc patients with calcinosis. Of five patients who completed treatment, most (80%) had documented stability of calcinosis on hand radiographs at 1 year. ClinicalTrials.gov identifier: NCT02663895.