Rheumatology Division, Hospital Universitario de La Princesa, Madrid, Spain.
Hospital Pharmacy Division, Hospital Universitario de La Princesa, Madrid, Spain.
Expert Opin Drug Saf. 2022 Mar;21(3):311-333. doi: 10.1080/14740338.2021.1973428. Epub 2021 Sep 1.
Interstitial lung disease (ILD) is one of the most important manifestations of connective tissue diseases (CTD) due to its association with high morbidity and mortality.
Literature review focused on the evidence on efficacy and safety of biological therapy.
Rituximab (RTX) is the most studied drug, though tocilizumab (TCZ) has methodologically more robust evidence, whereas abatacept (ABA) has only anecdotal reports. RTX studies suggest a clinically relevant effect on lung function and fibrosis in refractory to conventional treatment patients, with a good safety profile. Its multi-level efficacy in systemic sclerosis and the potentially more favorable response of anti-synthetase syndrome, especially when administered early in acute-onset or exacerbated ILD stand out over current standard of care, pending the availability of randomized controlled clinical trials. The significant and clinically meaningful benefit found in lung function and fibrosis with TCZ in faSScinate and focuSSced trials represents a change with respect to the usual practice, reinforcing the importance of treatment in early subclinical or clinical SSc-ILD patients with risk factors for ILD progression. This evidence has led to the inclusion of both RTX and TCZ in the expert-based therapeutic algorithms or recommendations for CTD-ILD management.
间质性肺疾病（ILD）是结缔组织疾病（CTD）的最重要表现之一，因为它与高发病率和死亡率有关。
文献综述侧重于生物治疗疗效和安全性的证据。
利妥昔单抗（RTX）是研究最多的药物，尽管托珠单抗（TCZ）具有更稳健的方法学证据，而阿巴西普（ABA）只有轶事报道。RTX 研究表明，在对常规治疗有抗药性的患者中，对肺功能和纤维化具有临床相关的疗效，且安全性良好。在系统性硬化症中，其多层次的疗效，以及在急性发作或加重的间质性肺病患者中，抗合成酶综合征的潜在更有利的反应，在等待随机对照临床试验的情况下，优于当前的标准治疗，尤其是在早期治疗。在 faSScinate 和 focuSSced 试验中，TCZ 在肺功能和纤维化方面的显著和具有临床意义的益处代表了对常规实践的改变，这强化了在有间质性肺病进展风险因素的早期亚临床或临床 SSc-ILD 患者中进行治疗的重要性。这一证据促使 RTX 和 TCZ 都被纳入了基于专家的 CTD-ILD 管理治疗算法或建议中。
