Newland Adrian, Godeau Bertrand, Priego Victor, Viallard Jean-Francois, López Fernández María F, Orejudos Amelia, Eisen Melissa
The Pathology Clinical Academic Group, Pathology Clinical Academic Unit, Pathology and Pharmacy Building, The Royal London Hospital, London, UK.
Department of Internal Medicine, Centre Hospitalier Universitaire (CHU) Henri-Mondor, INSERM U955, Créteil Cedex, France.
Br J Haematol. 2016 Jan;172(2):262-73. doi: 10.1111/bjh.13827. Epub 2015 Nov 5.
In anecdotal reports, some patients with immune thrombocytopenia (ITP) maintained platelet counts after discontinuing romiplostim. Here, we examined rates of platelet response (≥50 × 10(9) /l), remission, splenectomy and adverse events in patients with primary ITP duration ≤6 months who were treated with romiplostim for ≤12 months. The starting dose of romiplostim was 1 μg/kg; concomitant and rescue treatments were permitted to maintain platelet counts. Patients with platelet counts ≥50 × 10(9) /l at the end of 12 months entered a dose taper in which the romiplostim dose was decreased as long as platelet counts were maintained. Remission (platelet count ≥50 × 10(9) /l for 24 consecutive weeks with no ITP treatments) was evaluated in patients once romiplostim was discontinued. Over the 12 months, a high response rate (>90%) was observed. Platelet response occurred quickly (median, ~2 weeks) and was observed for a cumulative median of 11 months. Remission was observed in 24 patients (32%); there were no significantly predictors of remission. Most (20/24) patients had remission start before the forced taper. No new safety signals were identified. Thus, in patients with early-stage ITP, romiplostim was well tolerated and induced rapid responses, with remission occurring in approximately one-third of patients (NCT01143038, Amgen 20080435).
在一些轶事报告中，部分免疫性血小板减少症（ITP）患者在停用罗米司亭后血小板计数得以维持。在此，我们研究了原发性ITP病程≤6个月且接受罗米司亭治疗≤12个月的患者的血小板反应率（≥50×10⁹ /L）、缓解情况、脾切除术及不良事件。罗米司亭的起始剂量为1μg/kg；允许采用联合治疗及挽救治疗以维持血小板计数。在12个月末血小板计数≥50×10⁹ /L的患者进入剂量递减阶段，只要血小板计数得以维持，罗米司亭剂量就逐渐降低。一旦停用罗米司亭，即对患者评估缓解情况（血小板计数≥50×10⁹ /L持续24周且未接受ITP治疗）。在这12个月期间，观察到高反应率（>90%）。血小板反应出现迅速（中位时间，约2周），累计中位观察时间为11个月。24例患者（32%）出现缓解；无显著的缓解预测因素。大多数（20/24）患者在强制减量前开始缓解。未发现新的安全信号。因此，在早期ITP患者中，罗米司亭耐受性良好且诱导快速反应，约三分之一的患者出现缓解（NCT01143038，安进公司，20080435）。
