奥马珠单抗治疗老年中重度大疱性类天疱疮5例:前瞻性病例系列研究

Omalizumab in the Treatment of Five Elderly Patients with Moderate-to-Severe Bullous Pemphigoid:A Prospective Case-Series Study

目的 通过前瞻性病例系列研究设计,观察奥马珠单抗治疗老年中重度大疱性类天疱疮(BP)的长期临床疗效及安全性。方法 纳入5例老年中重度BP患者,在常规外用强效糖皮质激素、或联合系统治疗基础上予奥马珠单抗300 mg,每4周1次皮下注射,16周后根据病情予维持或调整剂量,疗程不低于24周,此后继续治疗并随访。主要结局为首次给药后4周内达到疾病控制的患者比例,同时于治疗前和首次治疗后第2、4、8、16、24周观察患者BP面积指数(BPDAI)、皮肤病生活质量指数(DLQI)、瘙痒数字评价量表(NRS)、嗜酸性粒细胞绝对数(EO)及抗BP180抗体值,同时评估疾病复发率与安全性。结果 5例患者在首次使用奥马珠单抗后4周内均达到了疾病控制,第24周时患者BPDAI评分、DLQI评分、瘙痒NRS评分、EO及抗BP180抗体值均较治疗前明显降低(P<0.05)。研究期间所有患者均未报告明显不良反应,其中2例患者在24周内复发。结论 奥马珠单抗可显著改善BP病情,提高患者生存质量,长期使用安全性良好。

Objective To evaluate the long-term clinical efficacy and safety of omalizumab in the treatment of elderly patients with moderate-to-severe bullous pemphigoid(BP).Methods Five elderly patients with moderate-to-severe BP were treated with omalizumab 300 mg once every 4 weeks on the basis of potent topical glucocorticoids,or combined with systemic therapy.After 16 weeks,the dose was maintained or adjusted according to the condition,and the course of treatment was not less than 24 weeks.The primary outcome was the proportion of patients with control of disease activity within 4 weeks after the first dose.The bullous pemphigoid area index(BPDAI),dermatology life quality index(DLQI),itching numerical rating scale(NRS),eosinophil count(EO) and anti-BP180 antibodies were measured before and at 2,4,8,16,24 weeks after the treatment.The recurrence rate and safety were also evaluated.Results All patients achieved disease control within 4 weeks.At week 24,the scores of BPDAI,DLQI,NRS,EO and anti-BP180 antibodies were significantly lower than those before treatment(P<0.05).No significant adverse reactions(AEs) were reported,and two patients relapsed within 24 weeks.Conclusion Omalizumab can significantly improve the condition and quality of life of BP,and long-term use of omalizumab is safe.