Centre Hospitalier Universitaire La Pitié-Salpétrière, Service de Médecine Interne, Assistance Publique-Hôpitaux de Paris, Paris, France; Université Pierre et Marie Curie Sorbonne Universités, Université Paris 06, Paris, France;
Université Pierre et Marie Curie Sorbonne Universités, Université Paris 06, Paris, France; Centre Hospitalier Régional Saint-Antoine, Service d'Hématologie, AP-HP, Paris, France;
Blood. 2014 Jul 10;124(2):204-10. doi: 10.1182/blood-2014-01-550244. Epub 2014 May 28.
In acquired thrombotic thrombocytopenic purpura (TTP), the persistence of severe ADAMTS13 deficiency (<10%) during remission is associated with more relapse. Preemptive (ie, after remission) administration of rituximab in these patients to prevent relapses remains controversial. We performed a cross-sectional analysis of 12-year follow-up data to compare the relapse incidence with or without preemptive rituximab infusion. Among 48 patients who experienced at least one episode of acquired TTP followed by severe ADAMTS13 deficiency during remission, 30 received preemptive rituximab (group 1); the other 18 did not (group 2). After a median of 17 months (interquartile range [IQR], 11-29) following rituximab, the relapse incidence decreased from 0.57 episodes/year (IQR, 0.46-0.7) to 0 episodes/year (IQR, 0-0.81) (P < .01) in group 1. ADAMTS13 activity 3 months after the first rituximab infusion increased to 46% (IQR, 30%-68%). Nine patients required additional courses of rituximab. In 5 patients, ADAMTS13 activity failed to increase durably. Four patients experienced manageable adverse effects. In group 2, the relapse incidence was higher (0.5 relapses/year; IQR, 0.12-0.5; P < .01). Relapse-free survival was longer in group 1 (P = .049). A persistent severe ADAMTS13 deficiency during TTP remission should prompt consideration of preemptive rituximab to prevent relapses.
在获得性血栓性血小板减少性紫癜（TTP）中，缓解期持续存在严重的 ADAMTS13 缺乏症（<10%）与更多的复发相关。在这些患者中，为了预防复发，在缓解期后（即预先）给予利妥昔单抗仍然存在争议。我们对 12 年的随访数据进行了横断面分析，以比较有无预先给予利妥昔单抗输注的复发率。在 48 例至少经历过一次获得性 TTP 发作且缓解期存在严重 ADAMTS13 缺乏症的患者中，30 例接受了预先给予利妥昔单抗（第 1 组）；另外 18 例未接受（第 2 组）。在利妥昔单抗治疗后中位数为 17 个月（四分位距 [IQR]，11-29）后，第 1 组的复发率从 0.57 次/年（IQR，0.46-0.7）降至 0 次/年（IQR，0-0.81）（P <.01）。首次利妥昔单抗输注后 3 个月 ADAMTS13 活性增加至 46%（IQR，30%-68%）。9 例患者需要额外的利妥昔单抗疗程。在 5 例患者中，ADAMTS13 活性未能持久增加。4 例患者出现可管理的不良反应。在第 2 组中，复发率较高（0.5 次复发/年；IQR，0.12-0.5；P <.01）。第 1 组的无复发生存时间更长（P =.049）。TTP 缓解期持续存在严重的 ADAMTS13 缺乏症时，应考虑预先给予利妥昔单抗以预防复发。
