E-ISSN: 1308-5263
Outcomes of Eltrombopag Treatment and Development of Iron Deficiency in Children with Immune Thrombocytopenia in Turkey [Turk J Hematol]
Turk J Hematol. 2020; 37(3): 139-144 | DOI: 10.4274/tjh.galenos.2020.2019.0380  

Outcomes of Eltrombopag Treatment and Development of Iron Deficiency in Children with Immune Thrombocytopenia in Turkey

Ayça Koca Yozgat1, Göksel Leblebisatan2, Sinan Akbayram3, Simge Çınar Özel4, Zeynep Karakaş5, Erol Erduran6, Şebnem Yılmaz7, Ülker Koçak8, Şule Ünal9, Gül Nihal Özdemir10, Meryem Albayrak11, Emine Zengin12, Yeşim Oymak13, Özcan Bör14, Hasan Fatih Çakmaklı15, Murat Söker16, Dilek Gürlek Gökçebay17, Hüseyin Tokgöz18, Barış Malbora19, Serap Karaman5, Tiraje Celkan4, İlgen Şaşmaz2, Neşe Yaralı1, Hale Ören7, Ayşegül Ünüvar5
1Ankara City Hospital, Clinic of Pediatric Hematology, Ankara, Turkey
2Çukurova University Faculty of Medicine, Department of Pediatric Hematology, Adana, Turkey
3Gaziantep University Faculty of Medicine, Department of Pediatric Hematology, Gaziantep, Turkey
4İstanbul University-Cerrahpaşa Cerrahpaşa Faculty of Medicine, Department of Pediatric Hematology, İstanbul, Turkey
5İstanbul University, İstanbul Faculty of Medicine, Department of Pediatric Hematology, İstanbul, Turkey
6Karadeniz Technical University Faculty of Medicine, Department of Pediatric Hematology, Trabzon, Turkey
7Dokuz Eylül University Faculty of Medicine, Department of Pediatric Hematology, İzmir, Turkey
8Gazi University Faculty of Medicine, Department of Pediatric Hematology, Ankara, Turkey
9Hacettepe University Faculty of Medicine, Department of Pediatric Hematology, Ankara, Turkey
10Kanuni Sultan Süleyman Training and Research Hospital, Clinic of Pediatric Hematology, İstanbul, Turkey
11Kırıkkale University Faculty of Medicine, Department of Pediatric Hematology, Kırıkkale, Turkey
12Kocaeli University Faculty of Medicine, Department of Pediatric Hematology, Kocaeli, Turkey
13Dr. Behçet Uz Children’s Training and Research Hospital, Clinic of Pediatric Hematology, İzmir, Turkey
14Eskişehir University Faculty of Medicine, Department of Pediatric Hematology, Eskişehir, Turkey
15Ankara University Faculty of Medicine, Department of Pediatric Hematology, Ankara, Turkey
16Dicle University Faculty of Medicine, Department of Pediatric Hematology, Diyarbakır, Turkey
17Ankara Keçiören Training and Research Hospital, Clinic of Pediatric Hematology, Ankara, Turkey
18Necmettin Erbakan University Meram Faculty of Medicine, Department of Pediatric Hematology, Konya, Turkey
19Yeni Yüzyıl University, Gaziosmanpaşa Hospital, Clinic of Pediatric Hematology, İstanbul, Turkey

Objective: Immune thrombocytopenia (ITP) is a rare autoimmune disease and hematologic disorder characterized by reduced platelet counts that can result in significant symptoms, such as bleeding, bruising, epistaxis, or petechiae. The thrombopoietin receptor agonist eltrombopag (EPAG) is a second-line agent used to treat chronic ITP purpura in adults and children.
Materials and Methods: The present retrospective study evaluated the efficacy, safety, and side effects of EPAG treatment in pediatric patients with acute refractory and chronic immune thrombocytopenia, particularly focusing on iron-deficiency anemia.
Results: The diagnosis was chronic ITP in 89 patients and acute refractory ITP in 16 patients. The mean age of patients was 9.5±4.5 years (minimum-maximum: 1.2-18 years) at the beginning of EPAG treatment. The overall response rate was 74.3% (n=78). The mean time for platelet count of ≥50x109/L was 11.6±8 weeks (range: 1-34 weeks). The treatment was stopped for 27 patients (25.7%) at an average of 6.8±9 months (range: 1-38 months). The reason for discontinuation was lack of response in 18 patients, nonadherence in 4 patients, and hepatotoxicity in 2 patients. Response to treatment continued for an average of 4 months after cessation of EPAG in 3 patients.
Conclusion: Results of the current study imply that EPAG is an effective therapeutic option in pediatric patients with acute refractory and chronic ITP. However, patients must be closely monitored for response and side effects during treatment, and especially for iron deficiency.

Keywords: Immune thrombocytopenia, Eltrombopag, Iron deficiency


İmmün Trombositopenili Çocuk Hastalarda Eltrombopag Tedavisi ve Tedaviye Bağlı Demir Eksikliği

Ayça Koca Yozgat1, Göksel Leblebisatan2, Sinan Akbayram3, Simge Çınar Özel4, Zeynep Karakaş5, Erol Erduran6, Şebnem Yılmaz7, Ülker Koçak8, Şule Ünal9, Gül Nihal Özdemir10, Meryem Albayrak11, Emine Zengin12, Yeşim Oymak13, Özcan Bör14, Hasan Fatih Çakmaklı15, Murat Söker16, Dilek Gürlek Gökçebay17, Hüseyin Tokgöz18, Barış Malbora19, Serap Karaman5, Tiraje Celkan4, İlgen Şaşmaz2, Neşe Yaralı1, Hale Ören7, Ayşegül Ünüvar5
1Ankara City Hospital, Clinic of Pediatric Hematology, Ankara, Turkey
2Çukurova University Faculty of Medicine, Department of Pediatric Hematology, Adana, Turkey
3Gaziantep University Faculty of Medicine, Department of Pediatric Hematology, Gaziantep, Turkey
4İstanbul University-Cerrahpaşa Cerrahpaşa Faculty of Medicine, Department of Pediatric Hematology, İstanbul, Turkey
5İstanbul University, İstanbul Faculty of Medicine, Department of Pediatric Hematology, İstanbul, Turkey
6Karadeniz Technical University Faculty of Medicine, Department of Pediatric Hematology, Trabzon, Turkey
7Dokuz Eylül University Faculty of Medicine, Department of Pediatric Hematology, İzmir, Turkey
8Gazi University Faculty of Medicine, Department of Pediatric Hematology, Ankara, Turkey
9Hacettepe University Faculty of Medicine, Department of Pediatric Hematology, Ankara, Turkey
10Kanuni Sultan Süleyman Training and Research Hospital, Clinic of Pediatric Hematology, İstanbul, Turkey
11Kırıkkale University Faculty of Medicine, Department of Pediatric Hematology, Kırıkkale, Turkey
12Kocaeli University Faculty of Medicine, Department of Pediatric Hematology, Kocaeli, Turkey
13Dr. Behçet Uz Children’s Training and Research Hospital, Clinic of Pediatric Hematology, İzmir, Turkey
14Eskişehir University Faculty of Medicine, Department of Pediatric Hematology, Eskişehir, Turkey
15Ankara University Faculty of Medicine, Department of Pediatric Hematology, Ankara, Turkey
16Dicle University Faculty of Medicine, Department of Pediatric Hematology, Diyarbakır, Turkey
17Ankara Keçiören Training and Research Hospital, Clinic of Pediatric Hematology, Ankara, Turkey
18Necmettin Erbakan University Meram Faculty of Medicine, Department of Pediatric Hematology, Konya, Turkey
19Yeni Yüzyıl University, Gaziosmanpaşa Hospital, Clinic of Pediatric Hematology, İstanbul, Turkey

Amaç: İmmün trombositopeni (İTP) nadir otoimmün bir hastalık olup, platelet sayısındaki azalmaya bağlı olarak peteşi, ekimoz, epistaksis gibi kanama semptomları ile karakterizedir. Trombopoietin reseptör agonisti eltrombopag (EPAG), yetişkinlerde ve çocuklarda kronik immün trombositopeni tedavisinde kullanılan ikinci basamak bir ajandır.
Gereç ve Yöntem: Bu retrospektif çalışma, akut refrakter ve kronik immün trombositopenili çocuk hastalarda EPAG tedavisinin etkinliğini, güvenilirliğini, yan etkilerini özellikle de demir eksikliği anemisi gelişimini değerlendirmiştir.
Bulgular: Hastalarımızın 89’u kronik İTP, 16’sı ise akut refrakter İTP tanısı ile takip edilmekte idi. Tedavi başlangıcı sırasında hastalarımızın yaş ortalamaları 9,5±4,5 yıl (1,2-18 yıl) idi. Toplam yanıt oranımız %74,3 (n=78) idi. Platelet sayısının ≥50x109/L olması için geçen ortalama süre 11,6±8 hafta (1-34 hafta) idi. Tedavi 27 hastamızda (%25,7) ortalama 6,8±9 ayda (1-38 ay) kesildi. Tedavi kesim nedenlerimiz; 18 hastada yanıtsızlık, 4 hastada tedaviye uyumsuzluk, 2 hastamızda ise hepatotoksisite idi. Üç hastada ise EPAG tedavisi kesilmesinden sonra yanıt ortalama 4 ay devam etti.
Sonuç: Çalışmamız, EPAG tedavisinin akut refrakter ve kronik İTP’li pediyatrik hastalarda etkili bir terapötik seçenek olduğunu göstermektedir. Bununla birlikte, hastalar EPAG tedavisi sırasında yanıt ve yan etkiler ve özellikle demir eksikliği açısından yakından izlenmelidir.

Anahtar Kelimeler: İmmün trombositopeni, Eltrombopag, Demir eksikliği


Ayça Koca Yozgat, Göksel Leblebisatan, Sinan Akbayram, Simge Çınar Özel, Zeynep Karakaş, Erol Erduran, Şebnem Yılmaz, Ülker Koçak, Şule Ünal, Gül Nihal Özdemir, Meryem Albayrak, Emine Zengin, Yeşim Oymak, Özcan Bör, Hasan Fatih Çakmaklı, Murat Söker, Dilek Gürlek Gökçebay, Hüseyin Tokgöz, Barış Malbora, Serap Karaman, Tiraje Celkan, İlgen Şaşmaz, Neşe Yaralı, Hale Ören, Ayşegül Ünüvar. Outcomes of Eltrombopag Treatment and Development of Iron Deficiency in Children with Immune Thrombocytopenia in Turkey. Turk J Hematol. 2020; 37(3): 139-144

Corresponding Author: Ayça Koca Yozgat, Türkiye


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