Longitudinal evaluation of eltrombopag in paediatric acquired severe aplastic anaemia

Filippidou, Maria; Avgerinou, Georgia; Tsipou, Haroula; Tourkantoni, Natalia; Katsibardi, Κaterina; Vlachou, Antonia; Roka, Kleoniki; Solomou, Elena E.; Kattamis, Antonis

doi:10.1111/bjh.16766

Cited by 11 publications

(11 citation statements)

References 13 publications

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“…One series of 11 children showed longer-term administration of EPAG may be efficacious. 26 There was a trend towards longer survival in the paediatric EPAG group, but this is likely related to disparate time-periods (1989-2010 for the IST group, 2012-2019 for the EPAG group) as survival in SAA has increased over time due to supportive care. 27 Transplant has become more widely utilised due to decreased transplant-related morbidity and mortality, and wider availability of alternative donors; more patients went off study in the EPAG group to pursue HSCT.…”

Section: Discussionmentioning

confidence: 93%

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Eltrombopag added to immunosuppression for children with treatment‐naïve severe aplastic anaemia

et al. 2021

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Acquired severe aplastic anaemia (SAA) has an immune pathogenesis, and immunosuppressive therapy (IST) with anti-thymocyte globulin and cyclosporine is effective therapy. Eltrombopag (EPAG) added to standard IST was associated with higher overall and complete response rates in patients with treatment-na€ ıve SAA compared to a historical IST cohort. We performed a paediatric subgroup analysis of this trial including all patients aged <18 years who received EPAG plus standard IST (n = 40 patients) compared to a historical cohort (n = 87) who received IST alone. Response, relapse, clonal evolution, event-free survival (EFS), and overall survival were assessed. There was no significant difference in either the overall response rate (ORR) or complete response rate at 6 months (ORR 70% in EPAG group, 72% in historical group, P = 0Á78). Adults (≥18 years) had a significantly improved ORR of 82% with EPAG compared to 58% historically (P < 0Á001). Younger children had lower response rates than did adolescents. The trend towards relapse was higher and EFS significantly lower in children who received EPAG compared to IST alone. Addition of EPAG added to standard IST did not improve outcomes in children with treatment-na€ ıve SAA. EPAG in the paediatric population should not automatically be considered standard of care. Registration: clinicaltrials.gov (NCT01623167).

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Section: Discussionmentioning

confidence: 93%

“…Haematological responses may in some patients be dependent on either marrow stimulation or ongoing IST. One series of 11 children showed longer‐term administration of EPAG may be efficacious 26 …”

Section: Discussionmentioning

confidence: 99%

Eltrombopag added to immunosuppression for children with treatment‐naïve severe aplastic anaemia

et al. 2021

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“…This pointed out that addition of eltrombopag to standard IST did not improve outcomes in children with SAA, and eltrombopag in the pediatric population should not automatically be considered the standard of care [14] . On the other hand, a study performed in Greece showed OR and CR rates of 81.8% and 72.7% at 3 months, respectively, indicating addition of eltrombopag to IST is associated with increased hematological response [19] . St. Jude Children's Research Hospital reported in 2021 that addition of eltrombopag to standard IST was well tolerated and resulted in satisfactory hematological response at 6 and 12 months [15] .…”

Section: Discussionmentioning

confidence: 95%

A single-center historical control study of eltrombopag combined with immunosuppressive therapy for severe aplastic anemia in children

Yang

et al. 2022

Preprint

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Severe aplastic anemia (SAA) is caused by immune-mediated destruction. Standard immunosuppressive therapy (IST) is effective, but needs to be improved. A total of 115 patients (60 males; median age of 5.77 years and median follow-up time of 45 months) were enrolled in this historical control study. The complete response (CR) rates in the eltrombopag group were 30.3% at 3 months, 50.0% at 6 months, conpared to 8.2% at 3 months, 10.2% at 6 months in the control group. There were significant differences between the two groups at 3 months and 6 months after IST. The overall response rates in the two groups showed no significant differences during the study. There were significant differences in the times separated from granulocyte colony stimulating factor (G-CSF) G-CSF, red blood cell transfusion and Platelet transfusion between the two groups. Overall survival rates were 97.0% in the eltrombopag group and 96.0% in the control group (P=0.998). In the eltrombopag group 10.2% cases relapsed compared to 4.1% in the control group (P=0.703). No case progressed to myelodysplastic syndrome or myeloid leukemia in the eltrombopag group; one patient (2.0%) progressed to myelodysplastic syndrome in the control group. Totally 11 patients (16.7%) showed myeloid gene mutations in the eltrombopag group. Event-free survival (EFS) was 66.6% in the eltrombopag group and 57.1% in the control group. There were no significant differences in EFS between the two groups (P=0.967). In the eltrombopag group the common adverse reactions were transient and reversible hyperbilirubinemia, elevated liver enzymesand hyperuricemia.

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“…72.7% for those receiving concomitant IST with eltrombopag, which was not inferior from their historical result. 2 Owing to the limited data number and different result of IST in each pediatric group, whether frontline concomitant IST with eltrombopag is superior to historical IST is still controversial, thus more data is necessary.…”

Section: + Modelmentioning

confidence: 99%

“…1 However, data of eltrombopag is limited in pediatric with low prevalence AA, showing equivocal result. 2,3 Here we report eight pediatric with AA cases treated with eltrombopag.…”

Section: Introductionmentioning

confidence: 99%

Role of eltrombopag in severe aplastic anemia treatment in children

Chang

Chou

et al. 2021

Pediatrics & Neonatology

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Longitudinal evaluation of eltrombopag in paediatric acquired severe aplastic anaemia

Cited by 11 publications

References 13 publications

Eltrombopag added to immunosuppression for children with treatment‐naïve severe aplastic anaemia

Eltrombopag added to immunosuppression for children with treatment‐naïve severe aplastic anaemia

A single-center historical control study of eltrombopag combined with immunosuppressive therapy for severe aplastic anemia in children

Role of eltrombopag in severe aplastic anemia treatment in children

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