Evaluation of Patients with Chronic Immune Thrombocytopenic Purpura Treated with Eltrombopag: A Single Center Experience

INTRODUCTION: The goal of treatment in chronic immune thrombocytopenia is to keep the platelet count within a safe range that can prevent bleeding. Eltrombopag is an oral thrombopoietin receptor agonist for the treatment of thrombocytopenia. We evaluated the demographic characteristics, eltrombopag treatment response and side-effect profile of a total of 79 patients using eltrombopag with follow-up at our center. By comparing our findings with the literature data, we aimed to reveal the differences in response rates and side-effect profiles.
METHODS: A total of 79 patients who were diagnosed with chronic immune thrombocytopenia and used eltrombopag, who applied to the hematology outpatient clinic of our center between January 1, 2010 and May 1, 2021, were evaluated in a randomized retrospective manner. Demographic characteristics of the patients, eltrombopag treatment response, and side-effect profile were recorded. Pearson Chi-square test was used to analyze categorical data.
RESULTS: In our study, the data of 79 patients who received eltrombopag treatment were analyzed retrospectively (45 females - 34 males, mean age 49±19.1 years). When the basal platelet count of the patients was evaluated before the treatment, the mean value was calculated as 17,000/mm3. When all platelet values ​​after eltrombopag treatment were examined, the mean maximum platelet value was 127,000/mm3, and the mean time to reach the maximum platelet value was 30 days. Complete remission was achieved in 46 (58%) patients, partial remission was achieved in 14 (17%) patients, and 19 (25%) didn't respond. Sixteen (20%) patients had a prolonged response.
DISCUSSION AND CONCLUSION: While basal platelet count and treatment responses for eltrombopag treatment were similar to those in the literature, prolonged response rates were higher in our study. Although the rate of elevation in liver enzyme follow-ups in our study was higher than the data in the literature, the rates of having to discontinue the drug for this reason were similar.

Eltrombopag Tedavisi Alan Kronik Ýmmün Trombositopenik Purpura Tanýlý Hastalarýn Deðerlendirilmesi: Tek Merkez Deneyimi

GÝRÝÞ ve AMAÇ: Kronik immün trombositopenide tedavi hedefi trombosit sayýsýný kanamayý önleyebilecek güvenli bir aralýkta tutmaktýr. Eltrombopag, trombositopeni tedavisinde oral bir trombopoietin reseptör agonistidir. Merkezimizde takipli eltrombopag kullanan toplam 79 hastanýn demografik özelliklerini, eltrombopag tedavi yanýtýný ve yan etki profilini deðerlendirdik. Bulgularýmýzý literatür verileri ile kýyaslayarak özellikle yanýt oranlarýndaki ve yan etki profilindeki farklýlýklarý ortaya koymayý amaçladýk.
YÖNTEM ve GEREÇLER: Çalýþmamýza 1 Ocak 2010 – 1 Mayýs 2021 tarihleri arasýnda merkezimizin hematoloji polikliniðine baþvuran kronik immun trombositopeni tanýsý konulan ve eltrombopag kullanan toplam 79 hasta randomize olacak þekilde retrospektif olarak deðerlendirildi. Hastalarýn demografik özellikleri, eltrombopag tedavi yanýtý, yan etki profili kaydedildi. Kategorik verilerin incelenmesinde Pearson Ki-kare testi kullanýlmýþtýr.
BULGULAR: Çalýþmamýzda eltrombopag tedavisi alan 79 hastanýn verileri retrospektif olarak incelendi ( 45 kadýn - 34 erkek, yaþ ortalamasý 49±19,1 yýl). Hastalarýn tedavi öncesi bazal trombosit sayýsý deðerlendirildiðinde ortalama deðeri 17,000/mm3 olarak hesaplandý. Eltrombopag tedavisinden sonraki tüm trombosit deðerleri incelendiðinde maksimum trombosit deðeri ortalamasý 127,000/mm3, maksimum trombosit deðerine ulaþýlan ortalama süre ise 30 gün þeklindeydi. Hastalarýn 46’sýnda (%58) komplet remisyon, 14’ünde (%17) parsiyel remisyon saðlanýrken 19’unda (%25) yanýt alýnamadýðý izlendi. Hastalarýn 16’sý (%20) uzamýþ cevaba sahipti.
TARTIÞMA ve SONUÇ: Eltrombopag tedavi kararý verilen bazal trombosit sayýsý ve tedavi yanýtlarý literatür ile benzerken, uzamýþ cevap oranlarýnýn bizim çalýþmamýzda daha fazla olduðu görüldü. Çalýþmamýzdaki karaciðer enzim takiplerindeki yükselme oraný literatürdeki verilerden daha yüksek bulunmasýna raðmen, bu sebepten ilacýn kesilmek zorunda kalýnmasýnýn oranlarý benzerdir.