Çekdemir Demet, Güvenç Serkan, Özdemirkıran Füsun, Eser Ali, Toptaş Tayfur, Özkocaman Vildan, Haydaroğlu Şahin Handan, Ermiş Turak Esra, Esen Ramazan, Cömert Melda, Sadri Sevil, Aslaner Müzeyyen, Uncu Ulu Bahar, Karakuş Abdullah, Selim Bapur Derya, Alacacıoğlu İnci, Aydın Demet, Tekinalp Atakan, Namdaroğlu Sinem, Ceran Funda, Tarkun Pınar, Kiper Demet, Çetiner Mustafa, Yenerel Mustafa, Demir Ahmet Muzaffer, Yılmaz Güven, Terzi Hatice, Atilla Erden, Malkan Ümit Yavuz, Acar Kadir, Öztürk Erman, Tombak Anıl, Sunu Cenk, Salim Ozan, Alayvaz Nevin, Sayan Özkan, Ozan Ülkü, Ayer Mesut, Gökgöz Zafer, Andıç Neslihan, Kızılkılıç Ebru, Noyan Figen, Özen Mehmet, Pepedil Tanrıkulu Funda, Alanoğlu Güçhan, Özkan Hasan Atilla, Aslan Vahap, Çetin Güven, Akyol Erikçi Alev, Deveci Burak, Ersoy Dursun Fadime, Dermenci Hasan, Aytan Pelin, Gündüz Mehmet, Karakuş Volkan, Özlü Can, Demircioğlu Sinan, Akay Yanar Olga Meltem, Özatlı Düzgün, Ündar Levent, Tiftik Eyüp Naci, Türköz Sucak Ayhan Gülsan, Haznedaroğlu İbrahim, Özcan Muhit, Şencan Mehmet, Tombuloğlu Murat, Özet Gülsüm, Bilgir Oktay, Turgut Burhan, Özcan Mehmet Ali, Payzın Kadriye Bahriye, Sönmez Mehmet, Ayyıldız Orhan, Dal Mehmet Sinan, Ertop Şehmus, Turgut Mehmet, Soysal Teoman, Kaya Emin, Ünal Ali, Pehlivan Mustafa, Atagündüz Işık, Tuğlular Fıratlı Tülin, Saydam Güray, Diz Küçükkaya Reyhan
Anadolu Medical Center, Bone Marrow Transplantation Center, Department of Hematology, Kocaeli, Turkey
Yeni Yüzyıl University Gaziosmanpaşa Hospital, Department of Hematology, İstanbul, Turkey
Turk J Haematol. 2019 Nov 18;36(4):230-237. doi: 10.4274/tjh.galenos.2019.2018.0307. Epub 2019 Jul 22.
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).
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) and defined as complete (platelet count of >100,000/mm), partial (30,000-100,000/mm or doubling of platelet count after treatment), or unresponsive (<30,000/mm). Clinical findings, descriptive features, response to treatment, and side effects were recorded. Correlations between descriptive, clinical, and hematological parameters were analyzed.
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 1, 2, 3, 4, and 8 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%).
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.
本研究旨在评估口服血小板生成素受体激动剂艾曲泊帕对慢性免疫性血小板减少症（ITP）患者的疗效和安全性。
本回顾性队列研究纳入了55个中心随访的285例慢性ITP患者（187例女性，占65.6%；98例男性，占34.4%）。根据血小板计数（/mm）评估治疗反应，定义为完全缓解（血小板计数>100,000/mm）、部分缓解（30,000 - 100,000/mm或治疗后血小板计数翻倍）或无反应（<30,000/mm）。记录临床发现、描述性特征、治疗反应和副作用。分析描述性、临床和血液学参数之间的相关性。
诊断时的中位年龄为43.9±20.6（范围：3 - 95）岁，随访时间为18.0±6.4（范围：6 - 28.2）个月。总体缓解率为86.7%（n = 247）。分别有182例（63.8%）和65例（22.8%）患者达到完全缓解和部分缓解。38例患者（13.4%）对艾曲泊帕治疗无反应。60岁以上患者（n = 68）的总体缓解率为89.7%（n = 61），80岁以上患者（n = 12）的总体缓解率为83%（n = 10）。考虑治疗前的血小板计数，艾曲泊帕在治疗第1、2、3、4和8周时显著增加血小板计数。随着达到部分或完全缓解所需时间的增加，治疗反应显著降低。治疗后达到最大血小板水平的时间差异很大（1 - 202周）。值得注意的是，艾曲泊帕治疗后最大血小板计数越高，发生副作用的可能性越大。最常见的副作用是头痛（21.6%）、虚弱（13.7%）、肝毒性（11.8%）和血栓形成（5.9%）。
本研究结果表明，即使对于老年慢性ITP患者，艾曲泊帕也是一种有效的治疗选择。然而，治疗期间必须密切监测患者的反应和副作用。由于在整个随访期间反应和副作用可能会有所变化，应动态评估患者，尤其是在血栓形成风险因素方面。
