Taylor Alice, Westwood John Paul, Laskou Faidra, McGuckin Siobhan, Scully Marie
University College London Hospitals NHS Foundation Trust and University College London, London, UK.
Br J Haematol. 2017 May;177(3):475-480. doi: 10.1111/bjh.14564. Epub 2017 Mar 14.
Eltrombopag is well established in treatment of severe immune thrombocytopenia (ITP) and is increasingly commonplace in second-line management. A role is also suggested for both bridging therapy for surgery, as well as treating thrombocytopenia due to non-immune aetiologies. We present the largest single-centre experience with eltrombopag, with our cohort of 62 patients. Patients with severe ITP (n = 34) had 91·2% response, which was sustained over a median of 18·5 months. In 41·4% of ITP cases (n = 14), complete response (CR- platelet count >100 × 10 /l) was achieved and in 2 cases, therapy was stopped and CR maintained. In our bridging group (n = 15) with a higher baseline platelet count, 93·3% achieved a CR. In the non-ITP group (n = 13), a response was achieved in 76·9%. In all groups, side effects were transient, with the drug discontinued in 2 patients due to minor complications (rash, nausea, diarrhoea). We conclude that eltrombopag is both effective and well tolerated as therapy in severe ITP. It is also advantageous in ITP patients who do not normally require therapy, but need a temporary platelet count boost pre-procedure. Furthermore, there are potentially far wider implications for the use of eltrombopag in counteracting thrombocytopenia beyond ITP, which merit further investigation.
艾曲波帕在治疗重度免疫性血小板减少症（ITP）方面已得到充分确立，在二线治疗中也越来越普遍。它还被认为可用于手术的桥接治疗以及治疗非免疫性病因导致的血小板减少症。我们展示了关于艾曲波帕的最大单中心经验，涉及我们的62例患者队列。重度ITP患者（n = 34）的缓解率为91.2%，在中位时间18.5个月内得以维持。在41.4%的ITP病例（n = 14）中实现了完全缓解（CR - 血小板计数>100×10⁹/l），有2例患者停止治疗并维持了CR状态。在我们基线血小板计数较高的桥接治疗组（n = 15）中，93.3%的患者实现了CR。在非ITP组（n = 13）中，缓解率为76.9%。在所有组中，副作用都是短暂的，有2例患者因轻微并发症（皮疹、恶心、腹泻）而停药。我们得出结论，艾曲波帕作为重度ITP的治疗药物既有效又耐受性良好。对于通常不需要治疗但在术前需要临时提高血小板计数的ITP患者，它也具有优势。此外，艾曲波帕在抵消ITP以外的血小板减少症方面的潜在应用范围可能更广，值得进一步研究。
