Use of eltrombopag for the treatment of poor graft function after hematopoietic stem cell transplantation in children

The main objective of this study was to determine whether Eltrombopag, a synthetic thrombopoietin receptor agonist, could improve peripheral blood counts in the three hematopoietic lineages and achieve transfusion independence in children with poor graft function (PGF) after allogenic hematopoietic...

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Bibliographic Details
Published in:Pediatric transplantation 2021-06, Vol.25 (4), p.e14010-n/a
Main Authors: Uria‐Oficialdegui, M. Luz, Alonso, Laura, Benitez‐Carabante, M. Isabel, Renedo, Berta, Oliveras, Maria, Diaz‐de‐Heredia, Cristina
Format: Article
Language:English
Subjects:
Adolescent
Benzoates - therapeutic use
Blood Cell Count
CD34 antigen
Child
Children
eltrombopag
Female
Hematologic Agents - therapeutic use
Hematologic Diseases - blood
Hematologic Diseases - diagnosis
Hematologic Diseases - drug therapy
Hematologic Diseases - etiology
hematopoietic stem cell transplantation (HSCT)
Hematopoietic Stem Cell Transplantation - adverse effects
Hematopoietic stem cells
Humans
Hydrazines - therapeutic use
Male
Patients
pediatric
Peripheral blood
poor graft function
Pyrazoles - therapeutic use
Receptors, Thrombopoietin - agonists
Retrospective Studies
Stem cell transplantation
Stem cells
Thrombopoietin
transfusions
Treatment Outcome
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Summary:The main objective of this study was to determine whether Eltrombopag, a synthetic thrombopoietin receptor agonist, could improve peripheral blood counts in the three hematopoietic lineages and achieve transfusion independence in children with poor graft function (PGF) after allogenic hematopoietic stem cell transplantation (HSCT). Retrospective study of patients under 18 years who developed PGF post‐HSCT in a large tertiary institution between January 2013 and March 2019. Out of 198 allogeneic HSCT, five patients met PGF criteria and were treated with eltrombopag. Median time from HSCT to eltrombopag initiation was 120 days. The median starting dose was 50 mg/day and the maximum dose reached was 75 mg/day. Median treatment duration was 9 months. Three patients achieved complete response and one partial response. The median dose among responders was 75 mg/day and the median time to response 8 weeks. Responses were sustained in three patients and two required a booster dose of CD34+‐selected cells from the original donor. None of the patients had to stop treatment due to adverse effects. The use of eltrombopag in children with PGF achieved responses in 80% of cases and demonstrated to be an effective and safe therapeutic option in pediatric patients with PGF.
ISSN:1397-3142
1399-3046
DOI:10.1111/petr.14010