Delvino Paolo, Quartuccio Luca, Robson Joanna C, Ferretti Virginia V, Klersy Catherine, Alberici Federico, Bagnasco Diego, Berti Alvise, Caminati Marco, Camoni Marta, Cid Maria C, Conticini Edoardo, Costanzo Giulia, de Moreuil Claire, Del Giacco Stefano, Espigol-Frigole Georgina, Franceschini Franco, Iorio Luca, Kernder Anna, Lo Gullo Alberto, Losappio Laura, Manna Elena, Maule Matteo, Milanesi Alessandra, Montecucco Carlomaurizio, Negrini Simone, Padoan Roberto, Regola Francesca, Ricciardi Luisa, Schroeder Jan, Terrier Benjamin, Toniati Paola, Torracca Francesca, Treppo Elena, Urban Maria Letizia, Vaglio Augusto, Emmi Giacomo, Monti Sara
School of Medicine and Surgery, University of Milano-Bicocca, Milan, Italy.
Rheumatology Unit, Fondazione IRCSS San Gerardo dei Tintori, Monza, Italy.
Rheumatology (Oxford). 2025 Sep 1;64(9):4937-4947. doi: 10.1093/rheumatology/keaf232.
To prospectively evaluate the impact and the rapidity of the effect of mepolizumab on the ANCA-associated vasculitis patient-reported outcomes (AAV-PRO) questionnaire and patient global assessment (PtGA) in an international, multicentre cohort of patients with eosinophilic granulomatosis with polyangiitis (EGPA).
Patients with active EGPA initiating treatment with mepolizumab were included. PtGA and the AAV-PRO score were assessed at baseline and after 7, 14, 30, 90 and 180 days. Predictors of response of the AAV-PRO questionnaire were investigated.
Seventy patients were included: female 54.3%, median age 56 years (48-65), 63 (90%) with a relapsing/refractory course. PtGA showed a statistically significant decrease within 7 days. At 14 days, all the AAV-PRO domains, except treatment side effects, showed a statistically significant decline. The improvement at 6 months was greatest in organ-specific symptoms (ratio 0.53), physical function (ratio 0.57) and PtGA (ratio 0.58). PtGA and higher disease activity positively correlated with the AAV-PRO scores throughout the study. Female patients reported a greater burden in terms of systemic symptoms, treatment side effects, social and emotional impact and concerns about the future. Conversely, age, educational level, damage accrual, mepolizumab dose and ANCA status had no effect on the AAV-PRO scores.
Mepolizumab was associated with a quick and remarkable improvement of health-related quality of life in patients with EGPA. These findings highlight its early and sustained benefits beyond disease control and support the integration of the AAV-PRO questionnaire into routine clinical practice.
在一项国际多中心嗜酸性肉芽肿性多血管炎（EGPA）患者队列中，前瞻性评估美泊利单抗对抗中性粒细胞胞浆抗体相关性血管炎患者报告结局（AAV-PRO）问卷及患者整体评估（PtGA）的影响及起效速度。
纳入开始接受美泊利单抗治疗的活动性EGPA患者。在基线以及第7、14、30、90和180天时评估PtGA和AAV-PRO评分。研究AAV-PRO问卷反应的预测因素。
纳入70例患者：女性占54.3%，中位年龄56岁（48 - 65岁），63例（90%）有复发/难治性病程。PtGA在7天内出现统计学显著下降。在14天时，除治疗副作用外，所有AAV-PRO领域均出现统计学显著下降。6个月时，器官特异性症状（比值0.53）、身体功能（比值0.57）和PtGA（比值0.58）改善最大。在整个研究过程中，PtGA和更高的疾病活动度与AAV-PRO评分呈正相关。女性患者在全身症状、治疗副作用、社会和情感影响以及对未来的担忧方面报告负担更重。相反，年龄、教育水平、损伤累积、美泊利单抗剂量和ANCA状态对AAV-PRO评分无影响。
美泊利单抗与EGPA患者健康相关生活质量的快速显著改善相关。这些发现突出了其在疾病控制之外的早期和持续益处，并支持将AAV-PRO问卷纳入常规临床实践。
