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Eltrombopag added to immunosuppression for children with treatment‐naïve severe aplastic anaemia
Author(s) -
Groarke Emma M.,
Patel Bhavisha A.,
GutierrezRodrigues Fernanda,
Rios Olga,
Lotter Jennifer,
Baldoni Daniela,
St. Pierre Annie,
Shalhoub Ruba,
Wu Colin O.,
Townsley Danielle M.,
Young Neal S.
Publication year - 2021
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.17232
Subject(s) - medicine , eltrombopag , cohort , immunosuppression , pediatrics , gastroenterology , immunology , immune thrombocytopenia , platelet
Summary 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).