Eltrombopag Added to Immunosuppression for Children with Treatment-naïve Severe Aplastic Anaemia

Overview

Journal Br J Haematol

Specialty Hematology

Date 2021 Jan 7

PMID 33410523

Citations 22

Authors

Emma M Groarke

Bhavisha A Patel

Fernanda Gutierrez-Rodrigues

Olga Rios

Jennifer Lotter

Daniela Baldoni

Annie St Pierre

Ruba Shalhoub

Colin O Wu

Danielle M Townsley

Neal S Young

Affiliations

Soon will be listed here.

Abstract

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-naïve 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 = 0·78). Adults (≥18 years) had a significantly improved ORR of 82% with EPAG compared to 58% historically (P < 0·001). 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-naïve SAA. EPAG in the paediatric population should not automatically be considered standard of care. Registration: clinicaltrials.gov (NCT01623167).

Citing Articles

Adjunctive effects of eltrombopag on immunosuppressive therapy for childhood aplastic anemia.

Eguchi K, Ishimura M, Ohga S, Endo S, Saito S, Kamimura S Int J Hematol. 2024; .

PMID: 39730862 DOI: 10.1007/s12185-024-03903-z.

Adding eltrombopag to intensive immunosuppressive therapy for severe aplastic anaemia may help adult patients achieve outcomes similar to paediatric patients.

Yang B, Wang L, Fu L, Chen M, Ma J, Han B Leukemia. 2024; 39(1):261-264.

PMID: 39572710 DOI: 10.1038/s41375-024-02450-0.

A Systematic Review and Meta-Analysis of Eltrombopag Efficacy Combined With Immunosuppressive Drugs in Treatment of Severe Aplastic Anemia.

Illango J, Seffah K, Naveen N, Paudel Y, Patel A, Pachchipulusu V Cureus. 2024; 16(8):e65970.

PMID: 39221321 PMC: 11365712. DOI: 10.7759/cureus.65970.

Eltrombopag combined with immunosuppressive therapy for pediatric severe aplastic anemia.

Yang B, Fu L, Li H, Chen H, Zhang R, Yao J Pediatr Res. 2024; .

PMID: 38822136 DOI: 10.1038/s41390-024-03253-w.

Resolvin E1 improves efferocytosis and rescues severe aplastic anemia in mice.

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