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

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

Ay�a Koca Yozgat¹, G�ksel Leblebisatan², Sinan Akbayram³, Simge ��nar �zel⁴, Zeynep Karaka�⁵, Erol Erduran⁶, �ebnem Y�lmaz⁷, �lker Ko�ak⁸, �ule �nal⁹, G�l Nihal �zdemir¹⁰, Meryem Albayrak¹¹, Emine Zengin¹², Ye�im Oymak¹³, �zcan B�r¹⁴, Hasan Fatih �akmakl�¹⁵, Murat S�ker¹⁶, Dilek G�rlek G�k�ebay¹⁷, H�seyin Tokg�z¹⁸, Bar�� Malbora¹⁹, Serap Karaman⁵, Tiraje Celkan⁴, �lgen �a�maz², Ne�e Yaral�¹, Hale �ren⁷, Ay�eg�l �n�var⁵
¹Ankara City Hospital, Clinic of Pediatric Hematology, Ankara, Turkey
²�ukurova University Faculty of Medicine, Department of Pediatric Hematology, Adana, Turkey
³Gaziantep University Faculty of Medicine, Department of Pediatric Hematology, Gaziantep, Turkey
⁴�stanbul University-Cerrahpa�a Cerrahpa�a Faculty of Medicine, Department of Pediatric Hematology, �stanbul, Turkey
⁵�stanbul University, �stanbul Faculty of Medicine, Department of Pediatric Hematology, �stanbul, Turkey
⁶Karadeniz Technical University Faculty of Medicine, Department of Pediatric Hematology, Trabzon, Turkey
⁷Dokuz Eyl�l University Faculty of Medicine, Department of Pediatric Hematology, �zmir, 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 S�leyman Training and Research Hospital, Clinic of Pediatric Hematology, �stanbul, Turkey
¹¹K�r�kkale University Faculty of Medicine, Department of Pediatric Hematology, K�r�kkale, Turkey
¹²Kocaeli University Faculty of Medicine, Department of Pediatric Hematology, Kocaeli, Turkey
¹³Dr. Beh�et Uz Children�s Training and Research Hospital, Clinic of Pediatric Hematology, �zmir, Turkey
¹⁴Eski�ehir University Faculty of Medicine, Department of Pediatric Hematology, Eski�ehir, Turkey
¹⁵Ankara University Faculty of Medicine, Department of Pediatric Hematology, Ankara, Turkey
¹⁶Dicle University Faculty of Medicine, Department of Pediatric Hematology, Diyarbak�r, Turkey
¹⁷Ankara Ke�i�ren Training and Research Hospital, Clinic of Pediatric Hematology, Ankara, Turkey
¹⁸Necmettin Erbakan University Meram Faculty of Medicine, Department of Pediatric Hematology, Konya, Turkey
¹⁹Yeni Y�zy�l University, Gaziosmanpa�a Hospital, Clinic of Pediatric Hematology, �stanbul, 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

�mm�n Trombositopenili �ocuk Hastalarda Eltrombopag Tedavisi ve Tedaviye Ba�l� Demir Eksikli�i

Ama�: �mm�n trombositopeni (�TP) nadir otoimm�n bir hastal�k olup, platelet say�s�ndaki azalmaya ba�l� olarak pete�i, ekimoz, epistaksis gibi kanama semptomlar� ile karakterizedir. Trombopoietin resept�r agonisti eltrombopag (EPAG), yeti�kinlerde ve �ocuklarda kronik imm�n trombositopeni tedavisinde kullan�lan ikinci basamak bir ajand�r.
Gere� ve Y�ntem: Bu retrospektif �al��ma, akut refrakter ve kronik imm�n trombositopenili �ocuk hastalarda EPAG tedavisinin etkinli�ini, g�venilirli�ini, yan etkilerini �zellikle de demir eksikli�i anemisi geli�imini de�erlendirmi�tir.
Bulgular: Hastalar�m�z�n 89�u kronik �TP, 16�s� ise akut refrakter �TP tan�s� ile takip edilmekte idi. Tedavi ba�lang�c� s�ras�nda hastalar�m�z�n ya� ortalamalar� 9,5�4,5 y�l (1,2-18 y�l) idi. Toplam yan�t oran�m�z %74,3 (n=78) idi. Platelet say�s�n�n ≥50x109/L olmas� i�in ge�en ortalama s�re 11,6�8 hafta (1-34 hafta) idi. Tedavi 27 hastam�zda (%25,7) ortalama 6,8�9 ayda (1-38 ay) kesildi. Tedavi kesim nedenlerimiz; 18 hastada yan�ts�zl�k, 4 hastada tedaviye uyumsuzluk, 2 hastam�zda ise hepatotoksisite idi. �� hastada ise EPAG tedavisi kesilmesinden sonra yan�t ortalama 4 ay devam etti.
Sonu�: �al��mam�z, EPAG tedavisinin akut refrakter ve kronik �TP�li pediyatrik hastalarda etkili bir terap�tik se�enek oldu�unu g�stermektedir. Bununla birlikte, hastalar EPAG tedavisi s�ras�nda yan�t ve yan etkiler ve �zellikle demir eksikli�i a��s�ndan yak�ndan izlenmelidir.

Anahtar Kelimeler: �mm�n trombositopeni, Eltrombopag, Demir eksikli�i

Ay�a Koca Yozgat, G�ksel Leblebisatan, Sinan Akbayram, Simge ��nar �zel, Zeynep Karaka�, Erol Erduran, �ebnem Y�lmaz, �lker Ko�ak, �ule �nal, G�l Nihal �zdemir, Meryem Albayrak, Emine Zengin, Ye�im Oymak, �zcan B�r, Hasan Fatih �akmakl�, Murat S�ker, Dilek G�rlek G�k�ebay, H�seyin Tokg�z, Bar�� Malbora, Serap Karaman, Tiraje Celkan, �lgen �a�maz, Ne�e Yaral�, Hale �ren, Ay�eg�l �n�var. 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: Ay�a Koca Yozgat, T�rkiye

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