Efficacy of Eltrombopag in the Treatment of Steroid Resistant Immune Thrombocytopenia: A Retrospective Single Center Experience

INTRODUCTION: Immune thrombocytopenia (ITP), which is also used as idiopathic thrombocytopenic purpura, is a clinical condition that can progress with severe thrombocytopenia and life-threatening bleeding. Thrombopoetin receptor analogues such as rompilostim and eltrombopag, which have been used in the last 10 years, are new agents with proven efficacy. Our aim in this study is to evaluate the efficacy and toxicity of eltrombopag in steroid resistant ITP patients with real life data.
METHODS: This study was conducted with a retrospective evaluation of patients treated with a diagnosis of ITP between January 2012 and September 2020. Patients under 18 years of age and using eltrombopag for non-ITP indications were excluded from the study.
RESULTS: The median age of 31 patients included in our study was 60 (25-87). Eighteen (58.0%) of these patients were women. The number of patients with at least one comorbidity was 19 (61.2%). All of the patients had received glucocorticosteroid treatment in the first line for ITP treatment. Splenectomy was applied as a second line treatment to 16 (51.6%) of these patients who were resistant to steroid treatment. Before eltrombopag, the median platelet value was 11x109/L (1x109/L - 46x109/L), while 24 (77.4%) patients responded to the treatment. In patients with a response, the median day 17 (day 3-57) thrombocyte count increased over 50x109/L. Sixteen of these patients (51.6%) had a response of 50 mg/day, seven (22.5%) 25 mg/day, and one (3.2%) patient 75 mg/day eltrombopag treatment. Two (6.4%) patients had an increase in grade 1 liver function tests. There was no patient who could not continue or interrupted the treatment due to side effects.
DISCUSSION AND CONCLUSION: It has been observed that eltrombopag treatment is an effective and safe option even in the group of patients who have previously received multiple-line therapy in ITP treatment.

Steroide Dirençli Ýmmun Trombositopeni Tedavisinde Eltrombopag Etkinliði: Retrospektif Tek Merkez Deneyimi

GÝRÝÞ ve AMAÇ: Ýdiopatik trombositopenik purpura olarak da adlandýrýlan immün trombositopeni (ITP), derin trombositopeni ve hayatý tehdit edici kanamalarla seyredebilen bir klinik tablodur. Birinci basamakta kullanýlan tedavilere yanýtsýz hastalarda son 10 yýldýr kullanýlmaya baþlanan trombopoetin reseptör analoglarý rompilostim ve eltrombopag etkinliði kanýtlanmýþ yeni ajanlardýr. Bu çalýþmadaki amacýmýz steroide dirençli ITP hastalarýnda eltrombopag etkinliðini ve yan etkilerini gerçek yaþam verileriyle deðerlendirmektir.
YÖNTEM ve GEREÇLER: Bu çalýþma Ocak 2012 – Eylül 2020 tarihleri arasýnda ITP tanýsý ile tedavi edilen hastalarýn retrospektif olarak deðerlendirilmesi ile yapýlmýþtýr. 18 yaþ altý ve ITP dýþý endikasyonla eltrombopag kullanan hastalar çalýþma dýþý býrakýlmýþtýr.
BULGULAR: Çalýþmamýza dahil edilen 31 hastanýn ortanca yaþý 60’dý (25-87). Bu hastalarýn 18’i (%58.0) kadýndý. En az bir komorbiditesi olan hasta sayýsý 19’du (%61.2). Hastalarýn tamamý ITP tedavisi için birinci sýrada glukokortikosteroid tedavisi almýþtý. Steorid tedavisine dirençli olan bu hastalarýn 16’sýna (%51.6) ikinci sýra tedavi olarak splenektomi uygulanmýþtý. Eltrombopag öncesinde ortanca trombosit deðeri 11x109/L (1x109/L - 46x109/L) iken 24 (%77.4) hastada tedaviye yanýt görülmüþtü. Yanýt elde edilen hastalarda ortanca 17.günde (3-57.gün) trombosit sayýsý 50x109 üzerine çýkmýþtý. Bu hastalarýn 16’sýnda (%51.6) 50 mg/gün, yedisinde (%22.5) 25 mg/gün, ve bir (%3.2) hastada ise 75 mg/gün eltrombopag tedavisi ile yanýt elde edilmiþti. Ýki (%6.4) hastada grade 1 karaciðer fonksiyon testlerinde yükselme görülmüþtü. Yan etki nedeniyle tedaviye devam edemeyen ya da tedaviye ara veren hastamýz ise yoktu.
TARTIÞMA ve SONUÇ: Eltrombopagin ITP tedavisi için daha önce çoklu basamak tedavi almýþ olan hasta grubunda bile etkin ve güvenli bir seçenek olduðu görülmüþtür.