Research progress in the treatment of interstitial lung disease related to polymyositis/dermatomyositis
ZHENG Xiaofeng,, XU Ling,
Department of Pulmonary and Critical Care Medicine, Shanghai Sixth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200233, China
Polymyositis/dermatomyositis (PM/DM) is a heterogeneous autoimmune disease characterized by inflammatory cell infiltration in skeletal muscles with unknown etiology. The incidence of interstitial lung disease (ILD) in PM/DM is higher. ILD is the most important factor affecting its prognosis. Existing evidence suggests that there is significant heterogeneity in PM/DM-related ILD, which requires individualized treatment based on the onset form, progression rate, lesion involvement range, laboratory examination, presence of adverse prognostic factors, and response to treatment of ILD. This article summarizes the research progress in the treatment of PM/DM-related ILD in recent years, including the selection of traditional therapeutic drugs, timing of medication, and new drugs attempted in this field in clinical practice (such as biological agents and Janus kinase inhibitors), intravenous immunoglobulin, anti-fibrotic drugs, and potentially effective non-drug treatment methods, aiming at providing reference for clinical doctors to formulate PM/DM-ILD treatment plans and improve patient prognosis.
ZHENG Xiaofeng, XU Ling. Research progress in the treatment of interstitial lung disease related to polymyositis/dermatomyositis. Journal of Shanghai Jiao Tong University (Medical Science)[J], 2024, 44(4): 531-536 doi:10.3969/j.issn.1674-8115.2024.04.015
1987年静脉输注免疫球蛋白（intravenous immunoglobulin，IVIG）首次用于治疗PM，1993年首次用于DM。在这之后的30多年间，IVIG用于治疗IIM的依据多来自于小型试验。2022年发表的ProDERM研究［26］是第一个大型前瞻性、随机双盲安慰剂对照、Ⅲ期临床试验，证明了IVIG治疗DM的有效性和安全性。基于这项研究，IVIG获得了美国食品药品监督管理局（FDA）的批准，用于治疗DM。这标志着IVIG治疗DM的证据级别已从病例报道和专家共识上升到随机对照研究。但是，遗憾的是该研究并未关注IVIG对DM-ILD的疗效［26-27］。我国的一项回顾性研究［28］显示，一线使用包含IVIG的治疗与对照组相比，能显著降低新发抗MDA-5抗体阳性RP-ILD患者3个月（19.4% vs 52.9%）及6个月的死亡率（22.6% vs 52.9%），提高3个月的缓解率。IVIG通过调节淋巴细胞和网状内皮功能，抑制促炎因子的产生，修饰补体激活，从而发挥免疫抑制和抗炎作用。IVIG通常耐受性良好，安全性也很好。
3.2 抗纤维化药物
目前有2种药物获批用于治疗特发性肺间质纤维化，即吡非尼酮和尼达尼布。尼达尼布还先后获批用于治疗进行性纤维化性间质性肺病（progressive fibrosing interstitial lung diseases，PF-ILD）以及系统性硬化相关ILD。 因此，若PM/DM-ILD表现为PF-ILD时，可使用尼达尼布治疗。但对于其他类型的PM/DM-ILD，目前没有治疗推荐。吡非尼酮在这方面有3项前瞻性研究结果公布。LI等［29］的研究显示，对于亚急性CADM-ILD患者，吡非尼酮能显著改善生存率（90% vs 44.4%），但对急性CADM-ILD患者的生存无影响。CHEN等［7］对IIM-ILD患者的研究显示，与他克莫司单药治疗组相比，他克莫司联合吡非尼酮治疗组胸部HRCT评分及呼吸相关疾病复发率较低，但2组累积生存率无明显差异。WANG等［30］对111例CTD-ILD患者进行了研究，其中IIM-ILD共有51例。经过24周治疗后，与未服用吡非尼酮的患者相比，吡非尼酮组FVC显著升高，且在具有非普通型间质性肺炎倾向的IIM患者中有更好的治疗效果。尼达尼布在这方面仅有一项回顾性研究［31］，结果显示，尼达尼布能提高IIM-ILD患者的生存率，降低RP-ILD的发生率。因此，对于PM/DM-ILD患者，可在免疫抑制剂治疗基础上加用抗纤维化药物治疗。
ZHENG Xiaofeng was responsible for collecting literature and writing the review. XU Ling was responsible for proposing writing ideas, and checking and correcting the content of the review. Both authors have read the last version of paper and consented for submission.
利益冲突声明
所有作者声明不存在利益冲突。
COMPETING INTERESTS
Both authors disclose no relevant conflict of interests.
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... 1987年静脉输注免疫球蛋白（intravenous immunoglobulin，IVIG）首次用于治疗PM，1993年首次用于DM.在这之后的30多年间，IVIG用于治疗IIM的依据多来自于小型试验.2022年发表的ProDERM研究［26］是第一个大型前瞻性、随机双盲安慰剂对照、Ⅲ期临床试验，证明了IVIG治疗DM的有效性和安全性.基于这项研究，IVIG获得了美国食品药品监督管理局（FDA）的批准，用于治疗DM.这标志着IVIG治疗DM的证据级别已从病例报道和专家共识上升到随机对照研究.但是，遗憾的是该研究并未关注IVIG对DM-ILD的疗效［26-27］.我国的一项回顾性研究［28］显示，一线使用包含IVIG的治疗与对照组相比，能显著降低新发抗MDA-5抗体阳性RP-ILD患者3个月（19.4% vs 52.9%）及6个月的死亡率（22.6% vs 52.9%），提高3个月的缓解率.IVIG通过调节淋巴细胞和网状内皮功能，抑制促炎因子的产生，修饰补体激活，从而发挥免疫抑制和抗炎作用.IVIG通常耐受性良好，安全性也很好. ...
... ［26-27］.我国的一项回顾性研究［28］显示，一线使用包含IVIG的治疗与对照组相比，能显著降低新发抗MDA-5抗体阳性RP-ILD患者3个月（19.4% vs 52.9%）及6个月的死亡率（22.6% vs 52.9%），提高3个月的缓解率.IVIG通过调节淋巴细胞和网状内皮功能，抑制促炎因子的产生，修饰补体激活，从而发挥免疫抑制和抗炎作用.IVIG通常耐受性良好，安全性也很好. ...
1
... 1987年静脉输注免疫球蛋白（intravenous immunoglobulin，IVIG）首次用于治疗PM，1993年首次用于DM.在这之后的30多年间，IVIG用于治疗IIM的依据多来自于小型试验.2022年发表的ProDERM研究［26］是第一个大型前瞻性、随机双盲安慰剂对照、Ⅲ期临床试验，证明了IVIG治疗DM的有效性和安全性.基于这项研究，IVIG获得了美国食品药品监督管理局（FDA）的批准，用于治疗DM.这标志着IVIG治疗DM的证据级别已从病例报道和专家共识上升到随机对照研究.但是，遗憾的是该研究并未关注IVIG对DM-ILD的疗效［26-27］.我国的一项回顾性研究［28］显示，一线使用包含IVIG的治疗与对照组相比，能显著降低新发抗MDA-5抗体阳性RP-ILD患者3个月（19.4% vs 52.9%）及6个月的死亡率（22.6% vs 52.9%），提高3个月的缓解率.IVIG通过调节淋巴细胞和网状内皮功能，抑制促炎因子的产生，修饰补体激活，从而发挥免疫抑制和抗炎作用.IVIG通常耐受性良好，安全性也很好. ...
1
... 1987年静脉输注免疫球蛋白（intravenous immunoglobulin，IVIG）首次用于治疗PM，1993年首次用于DM.在这之后的30多年间，IVIG用于治疗IIM的依据多来自于小型试验.2022年发表的ProDERM研究［26］是第一个大型前瞻性、随机双盲安慰剂对照、Ⅲ期临床试验，证明了IVIG治疗DM的有效性和安全性.基于这项研究，IVIG获得了美国食品药品监督管理局（FDA）的批准，用于治疗DM.这标志着IVIG治疗DM的证据级别已从病例报道和专家共识上升到随机对照研究.但是，遗憾的是该研究并未关注IVIG对DM-ILD的疗效［26-27］.我国的一项回顾性研究［28］显示，一线使用包含IVIG的治疗与对照组相比，能显著降低新发抗MDA-5抗体阳性RP-ILD患者3个月（19.4% vs 52.9%）及6个月的死亡率（22.6% vs 52.9%），提高3个月的缓解率.IVIG通过调节淋巴细胞和网状内皮功能，抑制促炎因子的产生，修饰补体激活，从而发挥免疫抑制和抗炎作用.IVIG通常耐受性良好，安全性也很好. ...
1
... 目前有2种药物获批用于治疗特发性肺间质纤维化，即吡非尼酮和尼达尼布.尼达尼布还先后获批用于治疗进行性纤维化性间质性肺病（progressive fibrosing interstitial lung diseases，PF-ILD）以及系统性硬化相关ILD. 因此，若PM/DM-ILD表现为PF-ILD时，可使用尼达尼布治疗.但对于其他类型的PM/DM-ILD，目前没有治疗推荐.吡非尼酮在这方面有3项前瞻性研究结果公布.LI等［29］的研究显示，对于亚急性CADM-ILD患者，吡非尼酮能显著改善生存率（90% vs 44.4%），但对急性CADM-ILD患者的生存无影响.CHEN等［7］对IIM-ILD患者的研究显示，与他克莫司单药治疗组相比，他克莫司联合吡非尼酮治疗组胸部HRCT评分及呼吸相关疾病复发率较低，但2组累积生存率无明显差异.WANG等［30］对111例CTD-ILD患者进行了研究，其中IIM-ILD共有51例.经过24周治疗后，与未服用吡非尼酮的患者相比，吡非尼酮组FVC显著升高，且在具有非普通型间质性肺炎倾向的IIM患者中有更好的治疗效果.尼达尼布在这方面仅有一项回顾性研究［31］，结果显示，尼达尼布能提高IIM-ILD患者的生存率，降低RP-ILD的发生率.因此，对于PM/DM-ILD患者，可在免疫抑制剂治疗基础上加用抗纤维化药物治疗. ...
1
... 目前有2种药物获批用于治疗特发性肺间质纤维化，即吡非尼酮和尼达尼布.尼达尼布还先后获批用于治疗进行性纤维化性间质性肺病（progressive fibrosing interstitial lung diseases，PF-ILD）以及系统性硬化相关ILD. 因此，若PM/DM-ILD表现为PF-ILD时，可使用尼达尼布治疗.但对于其他类型的PM/DM-ILD，目前没有治疗推荐.吡非尼酮在这方面有3项前瞻性研究结果公布.LI等［29］的研究显示，对于亚急性CADM-ILD患者，吡非尼酮能显著改善生存率（90% vs 44.4%），但对急性CADM-ILD患者的生存无影响.CHEN等［7］对IIM-ILD患者的研究显示，与他克莫司单药治疗组相比，他克莫司联合吡非尼酮治疗组胸部HRCT评分及呼吸相关疾病复发率较低，但2组累积生存率无明显差异.WANG等［30］对111例CTD-ILD患者进行了研究，其中IIM-ILD共有51例.经过24周治疗后，与未服用吡非尼酮的患者相比，吡非尼酮组FVC显著升高，且在具有非普通型间质性肺炎倾向的IIM患者中有更好的治疗效果.尼达尼布在这方面仅有一项回顾性研究［31］，结果显示，尼达尼布能提高IIM-ILD患者的生存率，降低RP-ILD的发生率.因此，对于PM/DM-ILD患者，可在免疫抑制剂治疗基础上加用抗纤维化药物治疗. ...
1
... 目前有2种药物获批用于治疗特发性肺间质纤维化，即吡非尼酮和尼达尼布.尼达尼布还先后获批用于治疗进行性纤维化性间质性肺病（progressive fibrosing interstitial lung diseases，PF-ILD）以及系统性硬化相关ILD. 因此，若PM/DM-ILD表现为PF-ILD时，可使用尼达尼布治疗.但对于其他类型的PM/DM-ILD，目前没有治疗推荐.吡非尼酮在这方面有3项前瞻性研究结果公布.LI等［29］的研究显示，对于亚急性CADM-ILD患者，吡非尼酮能显著改善生存率（90% vs 44.4%），但对急性CADM-ILD患者的生存无影响.CHEN等［7］对IIM-ILD患者的研究显示，与他克莫司单药治疗组相比，他克莫司联合吡非尼酮治疗组胸部HRCT评分及呼吸相关疾病复发率较低，但2组累积生存率无明显差异.WANG等［30］对111例CTD-ILD患者进行了研究，其中IIM-ILD共有51例.经过24周治疗后，与未服用吡非尼酮的患者相比，吡非尼酮组FVC显著升高，且在具有非普通型间质性肺炎倾向的IIM患者中有更好的治疗效果.尼达尼布在这方面仅有一项回顾性研究［31］，结果显示，尼达尼布能提高IIM-ILD患者的生存率，降低RP-ILD的发生率.因此，对于PM/DM-ILD患者，可在免疫抑制剂治疗基础上加用抗纤维化药物治疗. ...
