Turkish journal of haematology : official journal of Turkish Society of Haematology · 2025
A Multicenter Retrospective Study Comparing Immunosuppressive Therapy Combined with Eltrombopag to Immunosuppressive Therapy Alone as Frontline Treatment for Pediatric Severe Aplastic Anemia.
СтатьяМногоцентровое исследованиеСравнительное исследованиеНазвание и аннотация: Перевод в очереди
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Atay Didem, Yılmaz Karapınar Deniz, Kaçar Gonca, Karadaş Nihal, Apak Hilmi, Çakmaklı Hasan Fatih, Gadashova Aysha, Aksu Tekin, Celkan Tiraje, Yaralı Neşe, Orhan Mehmet Fatih, Özcan Alper, Ertem Mehmet, Aytaç Selin, Ören Hale, Akbayram Sinan, Işık Melek, Yılmaz Şebnem, İleri Talia, Büyükavcı Mustafa, Karakükcü Musa, Karapınar Tuba Hilkay, Erbey Fatih, Eker İbrahim, Albayrak Davut, Ünal Şule, Turkish Society of Pediatric Hematology’s Bone Marrow Failure Scientific Working Group
Аннотация
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.
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.