Patients with immune thrombocytopenia (ITP) may experience durable long-term response to first-line therapy with eltrombopag plus pulsed dexamethasone, according to research published in the British Journal of Cancer.

As some patients with ITP develop resistant disease, splenectomy, pulsed dexamethasone, thrombopoietin receptor agonists (TPO-RAs), and rituximab are second-line therapies being used with increasing frequency. Furthermore, there is some evidence that eltrombopag, an oral TPO-RA, may work synergistically with pulsed dexamethasone.

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For this open-label pilot study, researchers evaluated this combination “to establish whether eltrombopag plus dexamethasone has potential as first-line treatment and whether it is worth proceeding to a subsequent phase 3 trial.” The study’s primary end point was durable response after treatment discontinuation, defined as platelet counts of at least 50 x 109/L for at least 6 weeks.

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From November 2014 through March 2019, 50 patients with ITP were enrolled to receive 25 mg to 75 mg eltrombopag daily, according to platelet response, for 12 weeks, plus 40 mg dexamethasone daily for 4 days, every 4 weeks, for 1 to 3 cycles. At the end of the study, 46 patients were evaluable for response, and 26 had achieved the study’s primary end point. Two of these patients experienced relapse within 9 months.

There were no grade 3 or worse adverse events reported, though there were 2 cases of grade 2 fatigue and 2 cases of grade 2 bleeding.

“Eltrombopag plus pulsed dexamethasone as a first-line therapy could result in durable response off therapy in a significant number of patients with ITP,” the researchers concluded.


1.     Zhang L, Zhang M, Du X, Cheng Y, Cheng G. Safety and efficacy of eltrombopag plus pulsed dexamethasone as first‐line therapy for immune thrombocytopenia [published online January 13, 2020]. Br J Haematol. doi:10.1111/bjh.16327