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Comparative Study
. 2025 Aug 29;42(3):213-222.
doi: 10.4274/tjh.galenos.2025.2025.0245. Epub 2025 Aug 11.

A Multicenter Retrospective Study Comparing Immunosuppressive Therapy Combined with Eltrombopag to Immunosuppressive Therapy Alone as Frontline Treatment for Pediatric Severe Aplastic Anemia

Didem Atay  1 , Deniz Yılmaz Karapınar  2 , Gonca Kaçar  3 , Nihal Karadaş  2 , Hilmi Apak  3 , Hasan Fatih Çakmaklı  4 , Aysha Gadashova  2 , Tekin Aksu  5 , Tiraje Celkan  3 , Neşe Yaralı  6 , Mehmet Fatih Orhan  7 , Alper Özcan  8 , Mehmet Ertem  4 , Selin Aytaç  5 , Hale Ören  9 , Sinan Akbayram  10 , Melek Işık  11 , Şebnem Yılmaz  9 , Talia İleri  4 , Mustafa Büyükavcı  12 , Musa Karakükcü  8 , Tuba Hilkay Karapınar  13 , Fatih Erbey  14 , İbrahim Eker  15 , Davut Albayrak  16 , Şule Ünal  5 ; Turkish Society of Pediatric Hematology’s Bone Marrow Failure Scientific Working Group
Collaborators, Affiliations
Comparative Study

A Multicenter Retrospective Study Comparing Immunosuppressive Therapy Combined with Eltrombopag to Immunosuppressive Therapy Alone as Frontline Treatment for Pediatric Severe Aplastic Anemia

Didem Atay et al. Turk J Haematol. .

Abstract
in English, Turkish

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.

Amaç: Standart immünosüpresif tedaviye (IST) eklenen eltrombopag (EPAG), yetişkinlerde tedavi görmemiş ağır aplastik anemisi (SAA) olan hastalarda daha yüksek genel (OR) ve tam yanıt (CR) oranları ile ilişkilendirilmiştir, ancak edinsel aplastik anemisi olan çocuklarda EPAG’nin etkinliğine ilişkin klinik kanıtlar sınırlı ve tartışmalıdır.

Gereç ve yöntemler: Çalışmada SAA’lı pediatrik hastalarda ilk basamak tedavi olarak IST ile kombine EPAG’nin (n=38) etkinliği ve güvenliği tek başına IST (n=57) alan hasta grubu ile karşılaştırıldı.

Bulgular: EPAG+IST grubu 3 ve 6. aylarda daha yüksek CR ve OR oranlarına sahipti, ancak 1 yıllık OR, CR ve parsiyel yanıt oranları iki grup arasında anlamlı bir fark göstermedi. EPAG+IST grubunda tanı anında daha büyük yaşta olmak (>8,95 yaş), 6 ay ve 1 yılda daha yüksek OR oranları ile ilişkiliydi (sırasıyla p=0,007, p=0,005). Bu çalışmada EPAG’nin IST’ye eklenmesi, genel sağkalım (OS) ve olaysız sağkalım (EFS) açısından tek başına IST’ye göre üstünlük göstermedi (1 yıllık EFS EPAG+IST için %81,1 ve IST için %71,3 ve 1 yıllık OS %89,2’ye karşı %80,4).

Sonuç: EPAG toksik etkileri artırmadan tek başına IST alanlara kıyasla daha hızlı yanıt almayı sağladı. Ancak IST’ye EPAG eklenmesi çocuklarda OS veya relaps oranları açısından tek başına IST’ye göre ek fayda sağlamadı. Tanı sırasında daha büyük yaşta olan çocuklarda özellikle IST’ye EPAG eklenmesi daha iyi yanıt oranları ile önemli ölçüde ilişkiliydi.

Keywords: Eltrombopag; Immunosuppression; Treatment; Severe aplastic anemia.

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Conflict of interest statement

Conflict of Interest: No conflict of interest was declared by the authors.

Figures

Figure 1
Figure 1
Comparisons of overall survival and event-free survival between treatment groups.
Figure 2
Figure 2
Comparisons of overall survival and event-free survival between subgroups according to median age (subgroup 1: age of <8.95 years; subgroup 2: age of >8.95 years).
See this image and copyright information in PMC

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