A Multi-Center Study on the Efficacy of Eltrombopag in Management of Refractory Chronic Immune Thrombocytopenia: A Real-Life Experience
Journal Title: Turkish Journal of Hematology - Year 2019, Vol 36, Issue 4
Abstract
Objective: The aim of the present study was to evaluate the efficacy and safety of eltrombopag, an oral thrombopoietin receptor agonist, in patients with chronic immune thrombocytopenia (ITP). Materials and Methods: A total of 285 chronic ITP patients (187 women, 65.6%; 98 men, 34.4%) followed in 55 centers were enrolled in this retrospective cohort. Response to treatment was assessed according to platelet count (/mm3) and defined as complete (platelet count of >100,000/mm3), partial (30,000-100,000/mm3 or doubling of platelet count after treatment), or unresponsive (<30,000/mm3). Clinical findings, descriptive features, response to treatment, and side effects were recorded. Correlations between descriptive, clinical, and hematological parameters were analyzed. Results: The median age at diagnosis was 43.9±20.6 (range: 3-95) years and the duration of follow-up was 18.0±6.4 (range: 6-28.2) months. Overall response rate was 86.7% (n=247). Complete and partial responses were observed in 182 (63.8%) and 65 (22.8%) patients, respectively. Thirty-eight patients (13.4%) did not respond to eltrombopag treatment. For patients above 60 years old (n=68), overall response rate was 89.7% (n=61), and for those above 80 years old (n=12), overall response rate was 83% (n=10). Considering thrombocyte count before treatment, eltrombopag significantly increased platelet count at the 1st, 2nd, 3rd, 4th, and 8th weeks of treatment. As the time required for partial or complete response increased, response to treatment was significantly reduced. The time to reach the maximum platelet levels after treatment was quite variable (1-202 weeks). Notably, the higher the maximum platelet count after eltrombopag treatment, the more likely that side effects would occur. The most common side effects were headache (21.6%), weakness (13.7%), hepatotoxicity (11.8%), and thrombosis (5.9%). Conclusion: Results of the current study imply that eltrombopag is an effective therapeutic option even in elderly patients with chronic ITP. However, patients must be closely monitored for response and side effects during treatment. Since both response and side effects may be variable throughout the follow-up period, patients should be evaluated dynamically, especially in terms of thrombotic risk factors.
Authors and Affiliations
Demet Çekdemir, Serkan Güvenç, Füsun Özdemirkıra, Ali Eser, Tayfur Toptaş, Vildan Özkocaman, Handan Haydaroğlu Şahin, Esra Ermiş Turak, Ramazan Esen, Melda Cömert, Sevil Sadri, Müzeyyen Aslaner, Bahar Uncu Ulu, Abdullah Karakuş, Derya Selim Batut, İnci Alacacıoğlu, Atakan Tekinalp, Sinem Namdaroğlu, Funda Ceran, Pınar Tarkun, Mustafa Çetiner, Ahmet Muzaffer Demir, Güven Yılmaz, Hatice Terzi, Erden Atilla, Ümit Yavuz Malkan, Kadir Acar, Erman Öztürk, Anıl Tombak, Cenk Sunu, Ozan Salim, Nevin Alayvaz, Özkan Sayan, Ülkü Ozan, Mesut Ayer, Zafer Gökgöz, Neslihan Andıç, Ebru Kızılkılıç, Figen Noyan, Mehmet Özen, Güçhan Alanoğlu, Hasan Atilla Özkan, Vahap Aslan, Alev Akyol Erikçi, Burak Deveci, Fadime Ersoy Dursun, Hasan Dermenci, Pelin Aytan, Volkan Karakuş, Can Özlü, Sinan Demircioğlu, Olga Meltem Akay Yanar, Düzgün Özatlı, Levent Ündar, Eyüp Naci Tiftik, Ayhan Gülsan Türköz Sucak, İbrahim Haznedaroğlu, Muhit Özcan, Murat Tombuloğlu, Gülsüm Özet, Oktay Bilgir, Burhan Turgut, Mehmet Ali Özcan, Kadriye Bahriye Payzın, Orhan Ayyıldız, Mehmet Sinan Dal, Şehmus Ertop, Teoman Soysal, Emin Kaya, Işık Atagündüz, Tülin Tuğlular Fıratlı, Güray Saydam, Reyhan Diz Küçükkaya
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