Launceston General Hospital, University of Tasmania, Australia.
Mediterr J Hematol Infect Dis. 2012;4(1):e2012003. doi: 10.4084/MJHID.2012.003. Epub 2012 Jan 18.
Chronic immune thrombocytopenia (ITP) is a condition associated with significant morbidity; however the management options are often unsatisfactory with a portion of patients exhibiting a refractory-relapsing disease path despite various lines of treatment including splenectomy. As a thrombopoietin receptor agonist, eltrombopag (GlaxoSmithKline, Australia) provides a novel treatment option for patients with refractory disease. We describe the outcomes of four patients with chronic ITP, who were treated with eltrombopag as a single agent.
Four Caucasian patients with chronic refractory ITP (2 males; 2 females) were enrolled in this study with a mean age of 48 years (range, 39-59). All patients were non-splenectomised and were refractory to several lines of treatment including steroids, intravenous immunoglobulin, vincristine, and azathioprine, one patient has also received rituximab (a monoclonal antibody that binds the CD20 antigen expressed by B-lymphocytes). All patients were treated with oral eltrombopag (50-75 mg) for a median period of 12 months (range, 9-16).
After a median follow up of 20 months (range, 11-34), platelet counts recovered to normal levels in two patients. One recovered a normal platelet count after 13 months, the other 34 months of completion of treatment with eltrombopag. No additional immune suppressive therapy was required. The other two patients also discontinued eltrombopag at 27 and 11 months after achievement of satisfactory platelet counts above 30/nL without any bleeding complications. Other forms of immune therapy were also ceased in these two cases. None of the four patients required splenectomy.
The clinical outcomes in this small cohort of patients suggests that eltrombopag may have a role to play in the long term control of chronic ITP whilst avoiding splenectomy and long term immunosuppressive therapy. The beneficial outcomes in our patients led to a sustained elevation in platelets with no adverse effects noted when used for relatively longer periods than previously reported. It is worth noting that spontaneous remission does occur with ITP and is the most likely cause for the favourable outcome with eltrombopag therapy. However, if eltrombopag is able to reduce the need for splenectomy in patients with chronic ITP then a distinct quality of care outcome can be achieved by avoiding the recognised short- and long-term complications of splenectomy. Randomised controlled trials with long-term follow up are warranted.
慢性免疫性血小板减少症（ITP）是一种与显著发病率相关的疾病；然而，尽管许多患者接受了包括脾切除术在内的多种治疗方案，治疗效果仍往往不尽人意，其中一部分患者表现为难治性复发疾病过程。作为一种血小板生成素受体激动剂，艾曲波帕（葛兰素史克，澳大利亚）为难治性疾病患者提供了一种新的治疗选择。我们描述了四名接受艾曲波帕单药治疗的慢性 ITP 患者的结局。
这项研究纳入了四名慢性难治性 ITP 患者（2 名男性；2 名女性），平均年龄为 48 岁（范围，39-59 岁）。所有患者均未行脾切除术，且对几种治疗方案（包括类固醇、静脉注射免疫球蛋白、长春新碱和硫唑嘌呤）均耐药，其中一名患者还接受了利妥昔单抗（一种与 B 淋巴细胞表达的 CD20 抗原结合的单克隆抗体）治疗。所有患者均接受艾曲波帕（50-75mg）口服治疗，中位治疗时间为 12 个月（范围，9-16 个月）。
中位随访 20 个月（范围，11-34 个月）后，两名患者血小板计数恢复正常。一名患者在 13 个月后恢复正常血小板计数，另一名患者在完成 34 个月的艾曲波帕治疗后恢复正常。无需额外的免疫抑制治疗。另外两名患者在达到满意的血小板计数（>30/nL）后，分别在 27 个月和 11 个月后停用艾曲波帕，且无出血并发症。在这两例患者中，其他形式的免疫治疗也停止了。四名患者均未行脾切除术。
这项小队列患者的临床结局表明，艾曲波帕可能在慢性 ITP 的长期控制中发挥作用，同时避免脾切除术和长期免疫抑制治疗。我们的患者获得了良好的结局，导致血小板持续升高，且在之前报道的更长时间内使用时未观察到不良反应。值得注意的是，ITP 会自发缓解，这可能是艾曲波帕治疗疗效良好的最可能原因。然而，如果艾曲波帕能够减少慢性 ITP 患者脾切除术的需求，那么通过避免脾切除术的短期和长期并发症，可以实现显著的护理质量改善。需要进行长期随访的随机对照试验。
