Service de Médecine Interne, Centre National de Référence des Cytopénies Auto-Immunes de l'Adulte, Centre Hospitalier Universitaire Henri-Mondor, Assistance Publique-Hôpitaux de Paris, Université Paris Est Créteil, Créteil, France.
Service de Médecine Interne, Normandie Univ, UNICAEN, CHU de Caen Normandie, Caen, France.
Am J Hematol. 2019 Dec;94(12):1314-1324. doi: 10.1002/ajh.25632. Epub 2019 Oct 8.
Rituximab is a second-line option in adults with immune thrombocytopenia (ITP), but the estimated 5-year response rate, only based on pooled retrospective data, is about 20%, and no studies have focused on long-term safety. We conducted a prospective multicenter registry of 248 adults with ITP treated with rituximab with 5 years of follow-up to assess its long-term safety and efficacy. The median follow-up was 68.4 [53.7-78.5] months. The incidence of severe infections was only 2/100 patient-years. Profound hypogammaglobulinemia (<5 g/L) developed in five patients at 15 to 31 months after the last rituximab infusion. In total, 25 patients died at a median age of 80 [69.5-83.9] years, corresponding to a mortality rate of 2.3/100 patient-years. Only three deaths related to infection that occurred 12 to 14 months after rituximab infusions could be due in part to rituximab. At 60 months of follow-up, 73 (29.4%) patients had a sustained response. On univariate and multivariate analysis, the only factor significantly associated with sustained response was a previous transient response to corticosteroids (P = .022). Overall, 24 patients with an initial response and then relapse received retreatment with rituximab, which gave a response in 92%, with a higher duration of response in 54%. As a result of its safety profile and its sustained response rate, rituximab remains an important option in the current therapeutic armamentarium for adult ITP. Retreatment could be an effective and safe option.
利妥昔单抗是成人免疫性血小板减少症（ITP）的二线选择，但基于汇总的回顾性数据估计的 5 年缓解率仅为 20%左右，并且没有研究关注长期安全性。我们进行了一项前瞻性多中心登记研究，纳入了 248 例接受利妥昔单抗治疗的 ITP 成人患者，随访时间为 5 年，以评估其长期安全性和疗效。中位随访时间为 68.4[53.7-78.5]个月。严重感染的发生率仅为 2/100 患者年。在末次利妥昔单抗输注后 15 至 31 个月，有 5 例患者出现严重低丙种球蛋白血症（<5 g/L）。共有 25 例患者死亡，中位年龄为 80[69.5-83.9]岁，死亡率为 2.3/100 患者年。只有 3 例与感染相关的死亡可部分归因于利妥昔单抗，发生在利妥昔单抗输注后 12 至 14 个月。在 60 个月的随访中，73（29.4%）例患者有持续缓解。单因素和多因素分析显示，唯一与持续缓解显著相关的因素是既往对皮质类固醇的短暂缓解（P=0.022）。总体而言，24 例初始缓解后复发的患者接受了利妥昔单抗再治疗，92%的患者有缓解，缓解持续时间更长，为 54%。由于其安全性和持续缓解率，利妥昔单抗仍然是成人 ITP 当前治疗武器库中的重要选择。再次治疗可能是一种有效且安全的选择。
