PDF(Pubmed)
Abstract：
Background: Cystic fibrosis transmembrane conductance regulator modulators are the only available treatment for cystic fibrosis. Although elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) is well-tolerated, rash has been reported as very frequent. In severe rashes, ELX/TEZ/IVA withdrawal is necessary, leading to clinical deterioration. The objective of the study is to increment the experience of ELX/TEZ/IVA desensitization. Methods: Adult patients who developed a delayed hypersensitivity rash to ELX/TEZ/IVA between December 2021 and February 2023 and required withdrawal due to ineffective rescue medication were included. Skins test for ELX/TEZ/IVA and IVA were conducted to establish hypersensitivity mechanism. Balijepally ELX/TEZ/IVA desensitization protocol was selected. In cases where desensitization had to be discontinued due to rash, an extended desensitization was proposed. Clinical and health-related quality of life parameters were collected before ELX/TEZ/IVA and after desensitization. Results: 162 patients (81 women, 31.2 [23.8-42.5] years) started ELX/TEZ/IVA, developing rash 12 of them (7.4%, six women). Six patients (five women) required stopping ELX/TEZ/IVA and were selected for desensitization. Skin tests indicated delayed type-IV hypersensitivity in one patient. Two patients presented adequate tolerance to desensitization; while, four patients developed rash. Three of these patients, successfully concluded extended desensitization (one patient declined participation). No significant clinical deterioration or quality of life worsening was observed during desensitization; in fact, there was an improvement in practically all mesured parameters. All five patients who resumed ELX/TEZ/IVA are currently receiving therapy with good tolerance. Conclusion: Desensitization to ELX/TEZ/IVA could be a successful and safe strategy for reintroducing this essential treatment in cases of a delayed hypersensitivity rash.
摘要：
背景：囊性纤维化跨膜传导调节因子是囊性纤维化的唯一可用治疗方法。尽管elexacaftor/tezacaftor/ivacaftor（ELX/TEZ/IVA）耐受性良好，据报道，皮疹非常频繁。在严重的皮疹中，ELX/TEZ/IVA退出是必要的，导致临床恶化。该研究的目的是增加ELX/TEZ/IVA脱敏的经验。方法：纳入2021年12月至2023年2月期间出现ELX/TEZ/IVA迟发性过敏性皮疹并因无效抢救药物而需要停药的成年患者。进行ELX/TEZ/IVA和IVA皮肤测试以建立超敏机制。选择BalijepallyELX/TEZ/IVA脱敏方案。在因皮疹而不得不停止脱敏的情况下，提出了扩展脱敏。在ELX/TEZ/IVA脱敏前和脱敏后收集临床和健康相关生活质量参数。结果：162例患者（81例女性，31.2[23.8-42.5]年)开始ELX/TEZ/IVA，出现皮疹的其中12人(7.4%，六个女人）。6名患者（5名女性）需要停止ELX/TEZ/IVA，并选择进行脱敏。皮肤测试表明一名患者有IV型迟发型超敏反应。两名患者对脱敏表现出足够的耐受性；而，四名患者出现皮疹。其中三个病人,成功结束延长脱敏(1例患者拒绝参与).在脱敏过程中没有观察到明显的临床恶化或生活质量恶化；事实上，几乎所有测量参数都有改善。所有5名恢复ELX/TEZ/IVA的患者目前正在接受耐受性良好的治疗。结论：ELX/TEZ/IVA脱敏可能是一种成功且安全的策略，可以在迟发型超敏反应皮疹的情况下重新引入这种基本治疗方法。
