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

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 �ekdemir¹, Serkan G�ven�², F�sun �zdemirk�ran³, Ali Eser⁴, Tayfur Topta�⁴, Vildan �zkocaman⁵, Handan Haydaro�lu �ahin⁶, Esra Ermi� Turak⁷, Ramazan Esen⁸, Melda C�mert⁹, Sevil Sadri¹⁰, M�zeyyen Aslaner¹¹, Bahar Uncu Ulu¹², Abdullah Karaku�¹³, Derya Selim Batut¹⁴, �nci Alacac�o�lu¹⁵, Demet Ayd�n, Atakan Tekinalp, Sinem Namdaro�lu, Funda Ceran
¹Anadolu Medical Center, Bone Marrow Transplantation Center, Department of Hematology, Kocaeli, Turkey
²Yeni Y�zy�l University Gaziosmanpa�a Hospital, Department of Hematology, �stanbul, Turkey
³�zmir Atat�rk Training and Research Hospital, Clinic of Hematology, �zmir, Turkey
⁴Marmara University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, �stanbul, Turkey
⁵Uluda� 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 Y�z�nc� Y�l University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Van, Turkey
⁹�n�n� University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Malatya, Turkey
¹⁰�stanbul University-Cerrahpa�a Cerrahpa�a Faculty of Medicine, Department of Internal Medicine, Division of Hematology, �stanbul, Turkey
¹¹B�lent 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, Diyarbak�r, Turkey
¹⁴Karadeniz Teknik University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Trabzon, Turkey
¹⁵Dokuz Eyl�l University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, �zmir, 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 �mm�n Trombositopeni Tedavisinde Eltrombopag�n Etkinli�ine �li�kin �ok Merkezli Bir �al��ma: Ger�ek Ya�am Deneyimi

Ama�: Bu �al��man�n amac� kronik imm�n trombositopeni (ITP) hastalar�nda bir oral trombopoietin resept�r agonisti olan eltrombopag�n etkinlik ve g�venirlili�ini de�erlendirmektir.
Gere� ve Y�ntem: Elli be� merkezde izlem alt�ndaki toplam 285 kronik ITP hastas� (187 kad�n, %65,6) bu geriye d�n�k k�me �al��mas�na al�nm��t�r. Tedaviye yan�t trombosit say�s�na g�re de�erlendirilmi� ve tam yan�t (>100.000/mm3), k�smi yan�t (30.000-100.000/mm3 veya tedaviden sonra trombosit say�s�n�n bir kat artm�� olmas�) ve yan�ts�zl�k (<30.000/mm3) olarak tan�mlanm��t�r. Hastalar�n klinik bulgular�, tan�mlay�c� �zellikleri, tedaviye yan�t ve yan etki bilgileri toplanm�� ve aralar�ndaki ili�ki incelenmi�tir.
Bulgular: Tan� an�nda ya� ortalamas� 43,9�20,6 (3-95) y�l olan hastalar ortalama 18,0�6,4 (6-28,2) ay izlenmi�tir. Tam ve k�smi yan�t� i�eren toplam yan�t %86,7 (n=247) bulundu. S�ras�yla 182 (%63,8) ve 65 (%22,8) hastada tam ve parsiyel tedavi yan�tlar� g�zlenmi�tir. Otuz sekiz hasta (%13,4) eltrombopag tedavisine yan�t vermemi�tir. Altm�� ya� �zerindeki hastalarda (n=68) toplam yan�t %89,7 (n=61) bulunurken, bu oran 80 ya� �zerindeki (n=12) hastalarda %83 (n=10) olmu�tur. Tedavi �ncesi trombosit say�s� g�z �n�ne al�nd��nda, eltrombopag, tedavinin 1., 2., 3., 4. ve 8. haftalar�nda trombosit say�s�n� anlaml� �ekilde art�rm��t�r. K�smi veya tam cevap i�in gereken s�re artt�k�a, tedaviye cevap �nemli �l��de azald�� saptanm��t�r. Eltrombopag tedavisinden sonra maksimum trombosit say�s� ne kadar y�ksekse, yan etkilerin olu�abilme ihtimalinin o kadar y�ksek olabildi�i dikkati �ekmi�tir. En s�k g�r�len yan etkiler ba� a�r�s� (%21,6), g��s�zl�k (%13,7) ve hepatotoksisite (%11,8) ve trombozdur (%5,9).

Sonu�: Mevcut �al��man�n sonu�lar�, eltrombopag tedavisinin kronik ITP�de, ya�l� hastalar dahil olmak �zere, etkili bir tedavi se�ene�i oldu�unu g�stermektedir. Bununla birlikte, hastalar tedavi s�ras�nda yan�t ve yan etkiler a��s�ndan yak�ndan izlenmelidir. Hem cevap hem de yan etkiler, takip s�resi boyunca de�i�ken olabilece�inden, hastalar �zellikle tromboz risk fakt�rleri a��s�ndan dinamik olarak de�erlendirilmelidir.

Anahtar Kelimeler: Trombositopeni, �diyopatik trombositopenik purpura, Eltrombopag

Demet �ekdemir, Serkan G�ven�, F�sun �zdemirk�ran, Ali Eser, Tayfur Topta�, Vildan �zkocaman, Handan Haydaro�lu �ahin, Esra Ermi� Turak, Ramazan Esen, Melda C�mert, Sevil Sadri, M�zeyyen Aslaner, Bahar Uncu Ulu, Abdullah Karaku�, Derya Selim Batut, �nci Alacac�o�lu, Demet Ayd�n, Atakan Tekinalp, Sinem Namdaro�lu, 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 �ekdemir, T�rkiye

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