Use of Eltrombopag in Patients with Chronic Idiopathic Thrombocytopenic Purpura: A Single Center Experience

Öz

This study aimed to evaluate the efficacy and safety of eltrombopag in the outpatient clinic of the Department of Hematology, Uludag University Faculty of Medicine. Treatment responses and demographic characteristics of 25 patients diagnosed with refractory chronic immune thrombocytopenia (ITP) and receiving eltrombopag between January 2011 and March 2014 were analyzed retrospectively. Both platelet ≥30,000/mm³ and ≥50,000/mm³ values were considered as responses. Prolonged response was defined as the maintenance of platelet 50,000/mm³ values for 12 weeks or longer without the need for any additional treatment, and sustained response was defined as the maintenance of platelet 50,000/mm³ and at least 2 times the baseline values without the need for additional treatment. Twenty-five patients were evaluated. Ten patients (40%) had an initial platelet value of <15,000/mm³, and 15 (60%) had ≥15,000/mm³. The baseline median platelet value increased from 15,700/mm³ to 30,000/mm³ at week 2 and to 51,000/mm³ at week 6, and this level was maintained throughout the 85-week observation period. Thirteen (52%) patients had a sustained response, with a median follow-up of 18 months. In the study, participants were grouped according to splenectomy status, platelet <15,000/mm³, and gender, and there was no statistically significant difference in response. Eltrombopag is an effective and well-tolerated therapeutic agent in patients with chronic ITP refractory to other therapies and at increased bleeding risk. Responsiveness is independent of splenectomy, concomitant therapy, or baseline platelet values.

Anahtar Kelimeler

• Chronic immune thrombocytopenia
• refractory disease
• treatment
• eltrombopag.

Kronik İdiyopatik Trombositopenik Purpura Tanılı Hastalarda Eltrombopag Kullanımı: Tek Merkez Deneyimi

Öz

Bu çalışmada, Bursa Uludağ Üniversitesi Tıp Fakültesi Hematoloji Bilim Dalı polikliniğine başvuran hastalarda eltrombopag tedavisinin etkinliği ve güvenilirliğini değerlendirmeyi amaçladık. Ocak 2011 ile Mart 2014 tarihleri arasında refrakter kronik immün trombositopeni (ITP) tanısı konan ve eltrombopag alan toplam 25 hastanın tedavi yanıtları ve demografik özellikleri retrospektif olarak analiz edildi. Hem trombosit ≥30.000/mm3 hem de ≥50.000/mm3 değerleri yanıt olarak kabul edilmiştir. Uzamış yanıt, trombosit 50.000/mm3 değerlerinin herhangi bir ek tedaviye ihtiyaç duyulmadan 12 hafta veya daha uzun süre korunması ve sürekli yanıt, trombosit 50.000/mm3 ve başlangıç değerlerinin en az 2 katının ek tedaviye ihtiyaç duyulmadan korunması olarak tanımlandı. Yirmi beş hasta değerlendirildi. Başlangıç median trombosit değerinin 15,700 /mm3’den 2. haftada 30,000/mm3’e, 6. haftada 51,000/mm3’e yükseldiği ve 85 haftalık gözlem periyodu süresince bu düzeyin korunduğu görüldü. Hastaların 13’ünün (%52) devam eden yanıta sahip olduğu; median izlem sürelerinin 18 ay, eltrombopag kullanım sürelerinin ise 11 ay olduğu görüldü. Çalışmada katılımcılar splenektomi durumuna, trombosit <15,000/mm3 olup olmamasına ve cinsiyete göre gruplandırıldı ve yanıt açısından istatistiksel olarak anlamlı fark olmadığı görüldü. İlaç genel olarak iyi tolere edildi. Eltrombopag, diğer tedavilere dirençli ve artmış kanama riski olan kronik ITP hastalarında etkili ve iyi tolere edilen bir terapötik ajandır. Çalışmamızın sonuçları, yanıt durumunun splenektomi, eşzamanlı tedavi alma veya başlangıç trombosit değerlerinden bağımsız olduğunu ortaya koymuştur.

Anahtar Kelimeler

• Kronik immun trombositopeni
• refrakter hastalık
• tedavi

Kaynakça

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