The role of thrombopoietin receptor agonists in the management of adult primary immune thrombocytopenia - single centre experience
Background / Aim. The availability of thrombopoietin receptor agonists (TPO-RA) for the treatment of primary immune thrombocytopenia (ITP) over the last decade has transformed its management. The aim of this study was to assess the efficacy of TPO-RA in adults with chronic ITP treated in the University Clinical Centre of Serbia. Methods. A total of 28 adult ITP patients (10 males; 18 females) given eltrombopag and/or romiplostim were enrolled in the study. Data on demographic characteristics, ITP duration, previous therapeutic modalities, comorbidities, concomitant therapy, indications for TPO-RA, bleeding episodes, TPO-RA doses, adverse events and response rates were collected from the patients? medical records. TPO-RAs were administered: (a) in patients with chronic refractory ITP; (b) when splenectomy was contraindicated/unfeasible; (c) as preparation for splenectomy. A favourable treatment response was defined as a stable platelet count ? 50x109/L. Results. Twenty two (78.57%) and 14 (50.0%) subjects were treated with eltrombopag and romiplostim, respectively. A good treatment response (GTR) was achieved in 81.8% of the patients receiving eltrombopag and in 71.4% of those treated with romiplostim. The non-responders to eltrombopag (4 patients) and those who had lost their response to eltrombopag (4 patients) were switched to romiplostim. Six of them achieved a GTR. At the time of TPO-RA initiation, 46.4% of the patients used concomitant ITP therapy which was ceased in all those with a GTR. The following adverse effects of TPO-RA were registered: transaminitis and transient ischemic attack for eltrombopag - one patient each, and pulmonary embolism in one romiplostim treated patient. Conclusion. Our study showed that TPO-RAs are an effective and safe treatment option, since the majority of patients achieved stable remission without bleeding episodes.