E-ISSN: 1308-5263
A Multi-Center Study on the Efficacy of Eltrombopag in Management of Refractory Chronic Immune Thrombocytopenia: A Real-Life Experience [Turk J Hematol]
Turk J Hematol. 2019; 36(4): 230-237 | DOI: 10.4274/tjh.galenos.2019.2018.0307  

A Multi-Center Study on the Efficacy of Eltrombopag in Management of Refractory Chronic Immune Thrombocytopenia: A Real-Life Experience

Demet Çekdemir1, Serkan Güvenç2, Füsun Özdemirkıran3, Ali Eser4, Tayfur Toptaş4, Vildan Özkocaman5, Handan Haydaroğlu Şahin6, Esra Ermiş Turak7, Ramazan Esen8, Melda Cömert9, Sevil Sadri10, Müzeyyen Aslaner11, Bahar Uncu Ulu12, Abdullah Karakuş13, Derya Selim Batut14, İnci Alacacıoğlu15, Demet Aydın, Atakan Tekinalp, Sinem Namdaroğlu, Funda Ceran
1Anadolu Medical Center, Bone Marrow Transplantation Center, Department of Hematology, Kocaeli, Turkey
2Yeni Yüzyıl University Gaziosmanpaşa Hospital, Department of Hematology, İstanbul, Turkey
3İzmir Atatürk Training and Research Hospital, Clinic of Hematology, İzmir, Turkey
4Marmara University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, İstanbul, Turkey
5Uludağ University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Bursa, Turkey
6Gaziantep University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Gaziantep, Turkey
7Erciyes University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Kayseri, Turkey
8Van Yüzüncü Yıl University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Van, Turkey
9İnönü University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Malatya, Turkey
10İstanbul University-Cerrahpaşa Cerrahpaşa Faculty of Medicine, Department of Internal Medicine, Division of Hematology, İstanbul, Turkey
11Bülent Ecevit University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Zonguldak, Turkey
12Dr. Abdurrahman Yurtaslan Oncology Training and Research Hospital, Clinic of Hematology, Ankara, Turkey
13Dicle University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Diyarbakır, Turkey
14Karadeniz Teknik University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Trabzon, Turkey
15Dokuz Eylül University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, İzmir, Turkey

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.

Keywords: Thrombocytopenia, Immune thrombocytopenic, Eltrombopag


Refrakter Kronik İmmün Trombositopeni Tedavisinde Eltrombopagın Etkinliğine İlişkin Çok Merkezli Bir Çalışma: Gerçek Yaşam Deneyimi

Demet Çekdemir1, Serkan Güvenç2, Füsun Özdemirkıran3, Ali Eser4, Tayfur Toptaş4, Vildan Özkocaman5, Handan Haydaroğlu Şahin6, Esra Ermiş Turak7, Ramazan Esen8, Melda Cömert9, Sevil Sadri10, Müzeyyen Aslaner11, Bahar Uncu Ulu12, Abdullah Karakuş13, Derya Selim Batut14, İnci Alacacıoğlu15, Demet Aydın, Atakan Tekinalp, Sinem Namdaroğlu, Funda Ceran
1Anadolu Medical Center, Bone Marrow Transplantation Center, Department of Hematology, Kocaeli, Turkey
2Yeni Yüzyıl University Gaziosmanpaşa Hospital, Department of Hematology, İstanbul, Turkey
3İzmir Atatürk Training and Research Hospital, Clinic of Hematology, İzmir, Turkey
4Marmara University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, İstanbul, Turkey
5Uludağ University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Bursa, Turkey
6Gaziantep University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Gaziantep, Turkey
7Erciyes University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Kayseri, Turkey
8Van Yüzüncü Yıl University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Van, Turkey
9İnönü University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Malatya, Turkey
10İstanbul University-Cerrahpaşa Cerrahpaşa Faculty of Medicine, Department of Internal Medicine, Division of Hematology, İstanbul, Turkey
11Bülent Ecevit University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Zonguldak, Turkey
12Dr. Abdurrahman Yurtaslan Oncology Training and Research Hospital, Clinic of Hematology, Ankara, Turkey
13Dicle University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Diyarbakır, Turkey
14Karadeniz Teknik University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Trabzon, Turkey
15Dokuz Eylül University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, İzmir, Turkey

Amaç: Bu çalışmanın amacı kronik immün trombositopeni (ITP) hastalarında bir oral trombopoietin reseptör agonisti olan eltrombopagın etkinlik ve güvenirliliğini değerlendirmektir.
Gereç ve Yöntem: Elli beş merkezde izlem altındaki toplam 285 kronik ITP hastası (187 kadın, %65,6) bu geriye dönük küme çalışmasına alınmıştır. Tedaviye yanıt trombosit sayısına göre değerlendirilmiş ve tam yanıt (>100.000/mm3), kısmi yanıt (30.000-100.000/mm3 veya tedaviden sonra trombosit sayısının bir kat artmış olması) ve yanıtsızlık (<30.000/mm3) olarak tanımlanmıştır. Hastaların klinik bulguları, tanımlayıcı özellikleri, tedaviye yanıt ve yan etki bilgileri toplanmış ve aralarındaki ilişki incelenmiştir.
Bulgular: Tanı anında yaş ortalaması 43,9±20,6 (3-95) yıl olan hastalar ortalama 18,0±6,4 (6-28,2) ay izlenmiştir. Tam ve kısmi yanıtı içeren toplam yanıt %86,7 (n=247) bulundu. Sırasıyla 182 (%63,8) ve 65 (%22,8) hastada tam ve parsiyel tedavi yanıtları gözlenmiştir. Otuz sekiz hasta (%13,4) eltrombopag tedavisine yanıt vermemiştir. Altmış yaş üzerindeki hastalarda (n=68) toplam yanıt %89,7 (n=61) bulunurken, bu oran 80 yaş üzerindeki (n=12) hastalarda %83 (n=10) olmuştur. Tedavi öncesi trombosit sayısı göz önüne alındığında, eltrombopag, tedavinin 1., 2., 3., 4. ve 8. haftalarında trombosit sayısını anlamlı şekilde artırmıştır. Kısmi veya tam cevap için gereken süre arttıkça, tedaviye cevap önemli ölçüde azaldığı saptanmıştır. Eltrombopag tedavisinden sonra maksimum trombosit sayısı ne kadar yüksekse, yan etkilerin oluşabilme ihtimalinin o kadar yüksek olabildiği dikkati çekmiştir. En sık görülen yan etkiler baş ağrısı (%21,6), güçsüzlük (%13,7) ve hepatotoksisite (%11,8) ve trombozdur (%5,9).

Sonuç: Mevcut çalışmanın sonuçları, eltrombopag tedavisinin kronik ITP’de, yaşlı hastalar dahil olmak üzere, etkili bir tedavi seçeneği olduğunu göstermektedir. Bununla birlikte, hastalar tedavi sırasında yanıt ve yan etkiler açısından yakından izlenmelidir. Hem cevap hem de yan etkiler, takip süresi boyunca değişken olabileceğinden, hastalar özellikle tromboz risk faktörleri açısından dinamik olarak değerlendirilmelidir.

Anahtar Kelimeler: Trombositopeni, İdiyopatik trombositopenik purpura, Eltrombopag


Demet Çekdemir, Serkan Güvenç, Füsun Özdemirkıran, 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, Demet Aydın, Atakan Tekinalp, Sinem Namdaroğlu, Funda Ceran. A Multi-Center Study on the Efficacy of Eltrombopag in Management of Refractory Chronic Immune Thrombocytopenia: A Real-Life Experience. Turk J Hematol. 2019; 36(4): 230-237

Corresponding Author: Demet Çekdemir, Türkiye


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