Department of Medicine I, Dresden Friedrichstadt Hospital, University Clinical Center Carl Gustav Carus, Technische Universität Dresden, and Schwerpunktpraxis Rheumatologie, Dresden, Germany.
Rheumatology (Oxford). 2014 Sep;53(9):1630-8. doi: 10.1093/rheumatology/keu024. Epub 2014 Apr 4.
While a double-blind trial has not met its endpoint, rituximab (RTX) is still seen as useful in refractory DM and PM. In this study we analysed the charts of all patients receiving RTX for myositis in our institutions for objective outcome parameters.
In a retrospective way, the charts of all patients with PM or DM who received RTX were analysed for glucocorticoid dose, creatine phosphokinase (CPK) and lung function tests, as well as for serious adverse events.
A total of 19 patients were identified, 1 of whom died from aspiration pneumonia 3 weeks after the first RTX infusion. The charts of 18 patients (13 PM, 5 DM) could be further analysed. In addition to the fatal pneumonia, six more severe infections were seen. One patient developed hypogammaglobulinaemia. Two patients had mild infusion reactions. Under RTX, both CPK and daily prednisolone dose were reduced by week 18. Six of eight patients with alveolitis improved under RTX. Overall, 9 of 13 PM patients responded. Six of the responders and two patients without documented response, all anti-synthetase syndrome patients, were re-treated. In contrast, all five DM patients responded and none required re-treatment.
In a real-life population of patients with severe, refractory PM or DM, objective improvement was seen in the majority of patients with regard to CPK and lung function tests, and glucocorticoids could be reduced. In contrast to the subgroup with DM, where one cycle of RTX appeared sufficient, patients with anti-synthetase syndromes commonly experienced flares necessitating RTX re-treatment. Infections are of concern.
虽然一项双盲试验尚未达到终点，但利妥昔单抗（RTX）仍被认为对难治性皮肌炎和多发性肌炎有效。在本研究中，我们分析了在我们机构接受 RTX 治疗肌炎的所有患者的图表，以评估客观结局参数。
回顾性地分析了所有接受 RTX 治疗的皮肌炎或多发性肌炎患者的图表，以评估糖皮质激素剂量、肌酸磷酸激酶（CPK）和肺功能测试以及严重不良事件。
共确定了 19 名患者，其中 1 名患者在首次 RTX 输注后 3 周因吸入性肺炎死亡。18 名患者（13 名皮肌炎，5 名多发性肌炎）的图表可进一步分析。除致命性肺炎外，还发现了 6 例更严重的感染。1 名患者出现低丙种球蛋白血症。2 名患者出现轻度输注反应。在 RTX 治疗下，CPK 和每日泼尼松剂量在第 18 周时均降低。6 名间质性肺病患者在 RTX 治疗下得到改善。总体而言，13 名皮肌炎患者中有 9 名有反应。6 名有反应的患者和 2 名无记录反应的患者（均为抗合成酶综合征患者）再次接受治疗。相比之下，所有 5 名多发性肌炎患者均有反应，且无一例需要再次治疗。
在一组严重、难治性皮肌炎或多发性肌炎的真实患者中，大多数患者的 CPK 和肺功能测试均有改善，且糖皮质激素可减少。与多发性肌炎亚组相比，单次 RTX 治疗似乎足够，抗合成酶综合征患者常出现需要 RTX 再次治疗的疾病发作。感染是一个关注点。
