A Multi-Center Study on the Efficacy of Eltrombopag in Management of

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 (/mm(3)) and defined as complete (platelet count of >100,000/mm(3)), partial (30,000-100,000/mm(3) or doubling of platelet count after treatment), or unresponsive (<30,000/mm(3)). 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.

C1 [Cekdemir, Demet] Anadolu Med Ctr, Bone Marrow Transplantat Ctr, Dept Hematol, Kocaeli, Turkey.

[Guvenc, Serkan; Payzin, Kadriye Bahriye] Yeni Yuzyil Univ, Dept Hematol, Gaziosmanpasa Hosp, Istanbul, Turkey.

[Ozdemirkiran, Fusun] Ataturk Training & Res Hosp, Clin Hematol, Izmir, Turkey.

[Eser, Ali; Topts, Tayfur; Atagunduz, Isik; Firatli, Tulin Tuglular] Marmara Univ, Dept Internal Med, Div Hematol, Fac Med, Istanbul, Turkey.

[Ozkocaman, Vildan] Uludag Univ, Dept Internal Med, Div Hematol, Fac Med, Bursa, Turkey.

[Sahin, Handan Haydaroglu; Pehlivan, Mustafa] Gaziantep Univ, Dept Internal Med, Div Hematol, Fac Med, Gaziantep, Turkey.

[Turak, Esra Ermis; Unal, Ali] Erciyes Univ, Dept Internal Med, Div Hematol, Fac Med, Kayseri, Turkey.

[Esen, Ramazan; Demircioglu, Sinan] Van Yuzuncu Yil Univ, Dept Internal Med, Div Hematol, Fac Med, Van, Turkey.

[Comert, Melda; Kaya, Emin] Inonu Univ, Dept Internal Med, Div Hematol, Fac Med, Malatya, Turkey.

[Sadrilo, Sevil; Soysal, Teoman] Istanbul Univ Cerrahpasa, Dept Internal Med, Div Hematol, Cerrahpasa Fac Med, Istanbul, Turkey.

[Aslaner, Muzeyyen; Ertop, Sehmus] Bulent Ecevit Univ, Dept Internal Med, Div Hematol, Fac Med, Zonguldak, Turkey.

[Ulu, Bahar Uncu; Dal, Mehmet Sinan] Dr Abdurrahman Yurtaslan Oncol Training & Res Hos, Clin Hematol, Ankara, Turkey.

[Karakus, Abdullah; Ayyildiz, EOrhan] Dicle Univ, Dept Internal Med, Div Hematol, Fac Med, Diyarbakir, Turkey.

[Bapur, Derya Selim; Sonmez, Mehmet] Karadeniz Teknik Univ, Dept Internal Med, Div Hematol, Fac Med, Trabzon, Turkey.

[Alacacioglu, Inci; Ozcan, Mehmet Ali] Dokuz Eylul Univ, Dept Internal Med, Div Hematol, Fac Med, Izmir, Turkey.

[Aydin, Demet] Okmeydani Training & Res Hosp, Clin Hematol, Istanbul, Turkey.

[Tekinalp, Atakan; Turgut, Burhan] Namik Kemal Univ, Dept Internal Med, Div Hematol, Fac Med, Tekirdag, Turkey.

[Namdaroglu, Sinem; Bilgir, Oktay] Izmir Bozyaka Training & Res Hosp, Clin Hematol, Izmir, Turkey.

[Ceran, Funda; Ozet, Gulsum] Ankara Numune Training & Res Hosp, Clin Hematol, Ankara, Turkey.

[Tarkun, Pinar] Kocaeli Univ, Dept Internal Med, Div Hematol, Fac Med, Kocaeli, Turkey.

[Kiper, Demet; Tombuloglu, Murat; Saydam, Guray] Ege Univ, Dept Internal Med, Div Hematol, Fac Med, Izmir, Turkey.

[Cetiner, Mustafa; Ozturk, Erman] Koc Univ, Dept Internal Med, Div Hematol, Fac Med, Istanbul, Turkey.

[Yenerel, Mustafa] Istanbul Univ, Dept Internal Med, Div Hematol, Istanbul Fac Med, Istanbul, Turkey.

[Demir, Ahmet Muzaffer] Trakya Univ, Dept Internal Med, Div Hematol, Fac Med, Edirne, Turkey.

[Yilmaz, Guven] Dr Lutfi Kirdar Kartal Training & Res Hosp, Clin Hematol, Istanbul, Turkey.

[Terzi, Hatice; Sencan, Mehmet] Cumhuriyet Univ, Dept Internal Med, Div Hematol, Fac Med, Sivas, Turkey.

[Atilla, Erden; Ozcan, Muhit] Ankara Univ, Dept Internal Med, Div Hematol, Fac Med, Ankara, Turkey.

[Malkan, Umit Yavuz; Haznedaroglu, Ibrahim] Hacettepe Univ, Dept Internal Med, Div Hematol, Fac Med, Ankara, Turkey.

[Acar, Kadir; Sucak, EAyhan Gulsan Turkoz] Gazi Univ, Dept Internal Med, Div Hematol, Fac Med, Ankara, Turkey.

[Tombak, Anil; Tiftik, Eyup Naci] Mersin Univ, Dept Internal Med, Div Hematol, Fac Med, Mersin, Turkey.

[Sunu, Cenk] Sakarya Univ, Dept Internal Med, Div Hematol, Fac Med, Sakarya, Turkey.

[Salim, Ozan; Undar, Levent] Akdeniz Univ, Dept Internal Med, Div Hematol, Fac Med, Antalya, Turkey.

[Alayvaz, Nevin; Ozatli, Duzgun; Turgut, Mehmet] Ondokuz Mayis Univ, Dept Internal Med, Div Hematol, Fac Med, Samsun, Turkey.

[Sayan, Ozkan] Medicana Camlica Hosp, Clin Hematol, Istanbul, Turkey.

[Ozan, Ulku] Med Pk Hosp, Clin Hematol, Bursa, Turkey.

[Ayer, Mesut] Haseki Training & Res Hosp, Clin Hematol, Istanbul, Turkey.

[Gokgoz, Zafer] Medicana Int Ankara Hosp, Clin Hematol, Ankara, Turkey.

[Andic, Neslihan; Yanar, Olga Meltem Akay] Eskisehir Osmangazi Univ, Dept Internal Med, Div Hematol, Fac Med, Eskisehir, Turkey.

[Kizilkilic, Ebru] Acibadem Kozyatagi Hosp, Clin Hematol, Istanbul, Turkey.

[Noyan, Figen] Baskent Univ, Dept Internal Med, Div Hematol, Istanbul Hosp, Istanbul, Turkey.

[Ozen, Mehmet] Tokat Gaziosmanpasa Univ, Dept Internal Med, Div Hematol, Fac Med, Tokat, Turkey.

[Tanrikulu, Funda Pepedil] Dr Ersin Arslan Training & Res Hosp, Clin Hematol, Gaziantep, Turkey.

[Alanoglu, Guchan] Suleyman Demirel Univ, Dept Internal Med, Div Hematol, Fac Med, Isparta, Turkey.

[Ozkan, Hasan Atilla] Yeditepe Univ, Dept Internal Med, Div Hematol, Fac Med, Istanbul, Turkey.

[Aslan, Vahap] Umit Hosp, Clin Hematol, Eskisehir, Turkey.

[Cetin, Guven] Bezmialem Vakif Univ, Dept Internal Med, Div Hematol, Fac Med, Istanbul, Turkey.

[Erikci, Alev Akyol] Istanbul Yeni Yuzyil Univ, Dept Internal Med, Div Hematol, Gaziosmanpasa Hosp,Fac Med, Istanbul, Turkey.

[Deveci, Burak] Medstar Antalya Hosp, Clin Hematol, Antalya, Turkey.

[Dursun, Fadime Ersoy] Goztepe Training & Res Hosp, Clin Hematol, Istanbul, Turkey.

[Dermenci, Hasan] Haydarpasa Numune Training & Res Hosp, Clin Hematol, Istanbul, Turkey.

[Aytan, Pelin] Baskent Univ Training & Res Hosp, Bone Marrow & Stem Cell Transplantat Ctr, Dept Hematol, Adana, Turkey.

[Gunduz, Mehmet] Ataturk Training & Res Hosp, Clin Hematol, Ankara, Turkey.

[Karakus, Volkan] Mugla Sitki Kocman Univ Training & Res Hosp, Dept Hematol, Mugla, Turkey.

[Ozlu, Can] Minist Hlth, Clin Hematol, Erzurum Reg Training & Res Hosp, Erzurum, Turkey.

[Kucukkaya, Reyhan Diz] Istanbul Univ, Dept Mol Biol & Genet, Sci Fac, Istanbul, Turkey.