Successful Treatment with Thrombopoietin Receptor Agonist in Avoiding Splenectomy for Patients with Chronic Refractory Immune Thrombocytopenia

BACKGROUND: 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. METHODS: 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). RESULTS: 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. CONCLUSION: 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.