A Multicenter, Retrospective Study Comparing Immunosuppressive Therapy Combined with Eltrombopag to Immunosuppressive Therapy Alone as Front-Line Treatment for Pediatric Severe Aplastic Anemia
Year: 2025
Type: article
Study design: Observational Study
Abstract: Objective: Eltrombopag (EPAG) added to standard immunosuppressive therapy (IST) has been associated with higher overall response (OR) and complete response (CR) rates in adult patients with treatment-naïve severe aplastic anemia (SAA), but clinical evidence on the efficacy of EPAG in children with acquired aplastic anemia is limited and controversial. This retrospective study aimed to determine the efficacy and safety of EPAG combined with IST in pediatric patients with SAA compared to a standard IST group. Materials and Methods: We compared the efficacy and safety of EPAG combined with IST (n=38) versus IST alone (n=57) as frontline treatment for pediatric patients with SAA. Results: The EPAG+IST group had higher CR and OR rates at 3 and 6 months, although the 1-year OR, CR, and partial response rates showed no significant difference between the two groups. Older age at diagnosis (>8.95 years) was associated with a higher OR rate at 6 months and 1 year in the EPAG+IST group (p=0.007 and p=0.005, respectively). The addition of EPAG to IST did not achieve superiority over IST alone in terms of overall survival (OS) and event-free survival (EFS) in this study, with 1-year EFS of 81.1% for EPAG+IST and 71.3% for IST, and 1-year OS of 89.2% versus 80.4%, respectively. Conclusion: EPAG+IST induced a faster response compared to IST alone without increasing toxic effects, but EPAG did not confer additional benefits regarding OS or relapse rates in children. Notably, older age at diagnosis was significantly associated with improved response rates in the EPAG+IST group.
Source: Turkish Journal of Hematology
Authors: Didem Atay, Deniz Yılmaz Karapınar, Gonca Kaçar, Nihal Karadas, Hilmi Apak, Hasan Fatih Çakmaklı, Aysha Gadashova, Tekin Aksu, Tiraje Celkan, Neşe Yaralı, Mehmet Fatih Orhan, Alper Özcan, Mehmet Ertem, Selin Aytaç, Hale Ören, Sinan Akbayram, Melek Işık, Şebnem Yılmaz, Talia İleri, Mustafa Büyükavcı, Musa Karakükcü, Tuba Hilkay Karapınar, Fatih Erbey, İbrahim Eker, Davut Albayrak, Şule Ünal
Institutions: Acıbadem University, Ege University, Istanbul University-Cerrahpaşa, Istanbul University, Ankara University, Hacettepe University, Ankara Yıldırım Beyazıt University, Ankara Bilkent City Hospital, Sakarya University, Erciyes University, Dokuz Eylül University, Gaziantep University, Necmettin Erbakan University, Dr. Behçet Uz Çocuk Hastalıkları Hastanesi, Sağlık Bilimleri Üniversitesi, Koç University, Afyonkarahisar Sağlık Bilimleri Üniversitesi
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: diamond
OpenAlex ID: W4413099994