Dermatomyositis is an idiopathic inflammatory myopathy characterised by rashes and progressive muscle weakness. The recent ProDERM (Progress in DERMatomyositis) study is the first large randomised, placebo-controlled trial to establish the efficacy and safety of intravenous immunoglobulin (IVIg) in adult patients with dermatomyositis. Objectives of this analysis were to closely examine the safety and tolerability of IVIg in patients from the ProDERM study. ProDERM was a double-blind, randomised, placebo-controlled, multicentre, phase 3 study. In the first period (weeks 0–16), adults with active dermatomyositis received 2.0 g/kg IVIg (Octagam 10%; Octapharma AG) or placebo every 4 weeks. In the open-label extension period (weeks 16–40), all patients received IVIg for 6 additional cycles; dose reduction (1.0 g/kg) was permitted if patients were stable. Treatment-emergent adverse events (TEAEs) were documented. The 95 patients enrolled were randomised to receive IVIg (N = 47) or placebo (N = 48) in the first period, with 5 switching from placebo to IVIg. Overall, 664 IVIg infusion cycles were administered. During the first period, 113 TEAEs were possibly/probably related to treatment in 30/52 patients (57.7%) receiving IVIg and 38 in 11 patients (22.9%) on placebo. Eight patients discontinued therapy due to IVIg-related TEAEs. Eight thromboembolic events (TEEs) occurred in six patients on IVIg; six in five patients were deemed possibly/probably related to IVIg. Patients with TEEs exhibited more baseline TEE risk factors than those without TEEs (2.4–15.2-fold higher). Lowering infusion rate reduced the rate of TEEs, and none occurred at the lower IVIg dose. No haemolytic transfusion reactions or deaths occurred. Results from this study demonstrate that IVIg has a favourable safety profile for treatment of adult dermatomyositis patients and provides evidence that will help to inform treatment choice for these patients. Dermatomyositis patients receiving high-dose IVIg should be monitored for TEEs, and a low rate of infusion should be used to minimise TEE risk, particularly in those with pre-existing risk factors. ProDERM study (NCT02728752).

中文翻译：

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活动性皮肌炎患者静脉注射免疫球蛋白的安全性和耐受性：随机、安慰剂对照 ProDERM 研究的结果

皮肌炎是一种特发性炎症性肌病，其特征是皮疹和进行性肌无力。最近的 ProDERM（皮肌炎进展）研究是第一个大型随机、安慰剂对照试验，旨在确定静脉注射免疫球蛋白 (IVIg) 对成年皮肌炎患者的有效性和安全性。该分析的目的是仔细检查 ProDERM 研究患者中 IVIg 的安全性和耐受性。ProDERM 是一项双盲、随机、安慰剂对照、多中心、3 期研究。在第一阶段（第 0-16 周），患有活动性皮肌炎的成人每 4 周接受 2.0 g/kg IVIg（Octagam 10%；Octapharma AG）或安慰剂。在开放标签延长期（第16-40周），所有患者均接受额外6个周期的IVIg治疗；如果患者病情稳定，则允许减少剂量（1.0 g/kg）。记录治疗中出现的不良事件（TEAE）。入组的 95 名患者在第一阶段被随机分配接受 IVIg (N = 47) 或安慰剂 (N = 48)，其中 5 名患者从安慰剂转为 IVIg。总共进行了 664 个 IVIg 输注周期。在第一阶段，接受 IVIg 治疗的 30/52 名患者 (57.7%) 中，有 113 例 TEAE 可能/很可能与治疗相关；接受安慰剂的 11 名患者 (22.9%) 中，有 38 例 TEAE 可能/可能与治疗相关。8 名患者因 IVIg 相关 TEAE 停止治疗。6 名接受 IVIg 治疗的患者发生了 8 例血栓栓塞事件 (TEE)；五分之六的患者被认为可能/很可能与 IVIg 有关。患有 TEE 的患者比没有 TEE 的患者表现出更多的基线 TEE 危险因素（高 2.4-15.2 倍）。降低输注速度会降低 TEE 发生率，而较低 IVIg 剂量时则不会发生 TEE。未发生溶血性输血反应或死亡。这项研究的结果表明，IVIg 对于治疗成人皮肌炎患者具有良好的安全性，并提供了有助于为这些患者选择治疗选择的证据。接受高剂量 IVIg 的皮肌炎患者应监测 TEE 情况，并应采用低输注速度以尽量减少 TEE 风险，特别是对于那些已有危险因素的患者。ProDERM 研究 (NCT02728752)。