Thrombopoietin Receptor Agonists Are Often Ineffective in Immune Thrombocytopenia and/or Cause Adverse Reactions: Results from One Hand

Abstract

Background: Eltrombopag and romiplostim are thrombopoietin receptor agonists (TPOs) that have been increasingly used for the treatment of immune thrombocytopenia (ITP). Based on our experience, the incidence of abortive treatment with these drugs and the occurrence of adverse reactions that lead to therapy break-off despite response are higher than has been previously suggested. Methods: During the last 8 years, a total of 65 patients were treated with eltrombopag and/or romiplostim at our institute. Results: 36 of a total of 58 patients responded well to eltrombopag. In 12 patients that responded, treatment with eltrombopag was discontinued due to the development of adverse reactions. Eltrombopag was replaced by romiplostim in 23 cases (14 non-responders, 9 patients with adverse reactions). Of these patients, 83% responded to romiplostim. Among all patients treated with romiplostim (n = 32), 75% initially responded; however, 8 of these patients developed adverse reactions. Romiplostim was replaced by eltrombopag in 5 cases (4 due to adverse reactions, 1 non-responsive patient), and only 3 (60%) of these patients were observed to respond to eltrombopag. Conclusion: TPOs often remain ineffective in ITP or result in adverse reactions, which lead to treatment stop or to drug switch. Therefore, alternative treatment options are required.

Efficacy and tolerability of old and new drugs used in the treatment of immune thrombocytopenia: Results from a long-term observation in clinical practice

“…Of the remaining 45 patients, 35 patients (78%) responded to the drug. Treatment was discontinued in 8 (23%) of the responding patients due to adverse reactions [ 33 , 34 ]. None of the patients treated with romiplostim entered sustained remission.…”

“…Four of these patients (9%) entered sustained remission. Fourteen (31%) of the 45 responding patients discontinued treatment due to intolerable adverse reactions [ 33 , 34 ]. Eighteen patients (29%) did not rspond to eltrombopag.…”

A critical appraisal of the evidence for the role of splenectomy in adults and children with ITP

“…Notably, in a prospective multicentre observational study 42% (142 out of 340 evaluable patients) discontinued romiplostim for various reasons before the end of the 2‐year observation period (Steurer et al , ). Less exciting results in terms of efficacy and safety were also reported in a monocentric cohort (Depre et al , ). Furthermore, there are no convincing data that fatigue is abrogated with TPO‐RA and data on the improvement of quality of life are weak and inconsistent.…”

New aspects on the efficacy of high‐dose intravenous immunoglobulins in patients with autoimmune thrombocytopenia

“…It is worth mentioning that the majority of our therapy refractory patients refused splenectomy. In addition, treatment with TPOs was intolerable and/or ineffective in much more patients than has yet been suggested . The limitations of this study are reflected by the relatively small number and heterogeneity of patients.…”