Division of Rheumatology, Johns Hopkins University, Baltimore, MD, USA.
Division of Clinical Immunology and Rheumatology, Pontificia Universidad Católica de Chile, Santiago, Chile.
Rheumatology (Oxford). 2022 May 30;61(6):2441-2449. doi: 10.1093/rheumatology/keab810.
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 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.
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.
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.
我们评估了口服曲前列尼尔预防硬皮病相关钙沉积进展的安全性和有效性。
本前瞻性开放标签研究纳入了 12 名符合 2013 年 ACR/EULAR 分类标准的硬皮病患者，这些患者有手部一个或多个钙沉积的临床和影像学证实。患者接受了 1 年的口服曲前列尼尔治疗。主要终点为安全性/耐受性和 1 年后无钙沉积影像学进展的患者比例（Scleroderma Clinical Trials Consortium 放射评分增加<25%）。次要终点包括 1 年时硬皮病 HAQ（SHAQ）、Cochin 手部功能量表、医疗结局研究短表 36（SF-36）、雷诺氏病状况评分和钙沉积严重程度的患者/医生评估的变化。
共纳入 12 名女性患者，其中一半为弥漫性皮肤疾病；中位年龄为 55 岁（范围 35-68 岁）。5 名患者完成了研究。由于无法耐受的不良反应（n=3）、并发无关疾病（n=2，肝硬化、癌症）、进行性硬皮病（n=1）和个人原因（n=1），7 名患者退出。大多数患者出现头痛和胃肠道不良反应。11 名有 1 年手部 X 线随访的患者中，有 4 名（36%）出现钙沉积进展。5 名完成治疗的患者中，4 名（80%）的钙沉积稳定，1 名进展。根据 SF-36 生理和心理成分及域评分、过渡问题和 SF-6D 效用评分，所有完成试验的患者报告与基线相比总体改善或无变化。
口服曲前列尼尔在硬皮病伴钙沉积的患者中耐受性差。在完成治疗的 5 名患者中，大多数（80%）在 1 年时手部 X 线片上的钙沉积有记录的稳定。
NCT02663895。
