Fenaux Pierre, Muus Petra, Kantarjian Hagop, Lyons Roger M, Larson Richard A, Sekeres Mikkael A, Becker Pamela S, Orejudos Amelia, Franklin Janet
Service d'Hématologie Clinique, Hôpital St. Louis and Paris 7 University, Paris, France.
Department of Haematology, Radboud University Medical Centre, Nijmegen, The Netherlands.
Br J Haematol. 2017 Sep;178(6):906-913. doi: 10.1111/bjh.14792. Epub 2017 Jun 14.
Romiplostim can improve platelet counts in about 50% of patients with low- or intermediate 1-risk (lower risk) myelodysplastic syndromes (MDS) and thrombocytopenia, but its long-term toxicity and efficacy are not known. This open-label extension study evaluated the long-term safety and efficacy of romiplostim in 60 patients with lower risk MDS and platelet counts ≤50 × 10 /l. The primary endpoint was adverse event (AE) incidence. Secondary endpoints were efficacy parameters, including bleeding events and platelet response. Median (range) treatment time in the extension study and the median observation times thereafter were 25 (2-181) and 57 (11-209) weeks, respectively. Treatment-related AEs and serious AEs were reported in 14/60 (23%) and 4/60 (7%) patients, respectively. Progression to acute myeloid leukaemia (AML) occurred in two patients after 44 and 46 weeks. Patients (n = 34, 57%) with a platelet response were further evaluated for length of response. Median (range) response duration was 33 (7-174) weeks; 28/34 (82%) patients had a continuous response. Five of 34 patients (15%) had grade ≥3 bleeding events; three when the platelet count was >50 × 10 /l. There were no new safety concerns and the rate of progression to AML was low; response to romiplostim was maintained for most patients.
罗米司亭可使约50%的低危或中危1级（较低风险）骨髓增生异常综合征（MDS）伴血小板减少症患者的血小板计数升高，但其长期毒性和疗效尚不清楚。这项开放标签的扩展研究评估了罗米司亭对60例较低风险MDS且血小板计数≤50×10⁹/L患者的长期安全性和疗效。主要终点是不良事件（AE）发生率。次要终点是疗效参数，包括出血事件和血小板反应。扩展研究中的中位（范围）治疗时间以及之后的中位观察时间分别为25（2 - 181）周和57（11 - 209）周。分别有14/60（23%）和4/60（7%）的患者报告了治疗相关AE和严重AE。两名患者分别在44周和46周后进展为急性髓系白血病（AML）。对有血小板反应的患者（n = 34，57%）进一步评估反应持续时间。中位（范围）反应持续时间为33（7 - 174）周；34例患者中有28例（82%）有持续反应。34例患者中有5例（15%）发生≥3级出血事件；3例发生在血小板计数>50×10⁹/L时。未出现新的安全问题，进展为AML的发生率较低；大多数患者对罗米司亭的反应得以维持。
