Centre for Rare Disorders, Oslo University Hospital, Oslo, Norway.
Section of Clinical Immunology and Infectious Diseases, Oslo University Hospital, Oslo, Norway.
Expert Rev Clin Immunol. 2023 Jun;19(6):627-638. doi: 10.1080/1744666X.2023.2198208. Epub 2023 Apr 6.
Patients with common variable immunodeficiency (CVID) have a high frequency of inflammatory complications like autoimmune cytopenias, interstitial lung disease and enteropathy. These patients have poor prognosis and effective, timely, and safe treatment of inflammatory complications in CVID is essential, but guidelines and consensus on therapy are often lacking.
This review will focus on current medical treatment of inflammatory complications in CVID and point out some future perspectives based on literature indexed in PubMed. There are a number of good observational studies and case reports on treatment of specific complications, but randomized controlled trials are scarce.
In clinical practice, the most urgent issues that need to be addressed are the preferred treatment of GLILD, enteropathy and liver disease. Treating the underlying immune dysregulation and immune exhaustion in CVID is an alternative approach that potentially could alleviate these and other organ-specific inflammatory complications. Therapies of potential interest and wider use in CVID include mTOR-inhibitors like sirolimus, JAK-inhibitors like tofacitinib, the monoclonal IL-12/23 antibody ustekinumab, the anti-BAFF antibody belimumab and abatacept. For all inflammatory complications, there is a need for prospective therapeutic trials, preferably randomized controlled trials, and multi-center collaborations with larger cohorts of patients will be essential.
普通变异性免疫缺陷（CVID）患者常伴有炎症并发症，如自身免疫性血细胞减少症、间质性肺病和肠病。这些患者预后较差，因此及时有效地治疗 CVID 炎症并发症至关重要，但目前常缺乏相关治疗指南和共识。
本文将重点关注 CVID 炎症并发症的当前医学治疗方法，并根据 PubMed 索引文献探讨一些未来展望。针对特定并发症的治疗已有大量良好的观察性研究和病例报告，但随机对照试验却相对较少。
在临床实践中，最迫切需要解决的问题是 GLILD、肠病和肝病的首选治疗方法。治疗 CVID 中潜在的免疫失调和免疫衰竭是一种潜在的治疗方法，可能有助于缓解这些和其他器官特异性炎症并发症。在 CVID 中有应用前景且更广泛使用的治疗方法包括 mTOR 抑制剂（如西罗莫司）、JAK 抑制剂（如托法替布）、单克隆 IL-12/23 抗体乌司奴单抗、抗 BAFF 抗体贝利尤单抗和阿巴西普。所有炎症并发症都需要进行前瞻性治疗试验，最好是随机对照试验，多中心合作和更大的患者队列将至关重要。
