Health-related quality of life in children with chronic immune thrombocytopenia treated with eltrombopag in the PETIT study

John D Grainger; Victor S Blanchette; Kelly M Grotzinger; Anuja Roy; James B Bussel

doi:10.1111/bjh.15732

Health-related quality of life in children with chronic immune thrombocytopenia treated with eltrombopag in the PETIT study

Br J Haematol. 2019 Apr;185(1):102-106. doi: 10.1111/bjh.15732. Epub 2018 Dec 27.

Authors

John D Grainger¹, Victor S Blanchette², Kelly M Grotzinger³, Anuja Roy⁴, James B Bussel⁵

Affiliations

¹ Central Manchester University Hospitals NHS Foundation Trust, NIHR/Wellcome Trust Manchester CRF, Royal Manchester Children's Hospital, University of Manchester, Manchester, England.
² Division of Hematology/Oncology, The Hospital for Sick Children, Toronto, Ontario, Canada.
³ GlaxoSmithKline, Collegeville, PA, USA.
⁴ Novartis AG, Basel, Switzerland.
⁵ Department of Pediatrics, Division of Hematology/Oncology, Weill Cornell Medicine, New York, NY, USA.

PMID: 30592022
DOI: 10.1111/bjh.15732

Abstract

The PETIT (Eltrombopag in Pediatric Patients with Thrombocytopenia from Chronic ITP) trial showed that in children aged 1-17 years with chronic or persistent immune thrombocytopenia (ITP), eltrombopag improved platelet counts, decreased clinically significant bleeding and reduced rescue medication need. We report the health-related quality of life (HRQoL) results from the PETIT study using the Kids' ITP Tools (KIT). A limitation was that PETIT was not powered for the HRQoL analysis. Eltrombopag did not impact children's HRQoL assessed by the KIT. Although median KIT scores in children treated with eltrombopag with platelet responses were numerically higher compared with non-responders in some age groups, the interquartile ranges overlapped.

Trial registration: ClinicalTrials.gov NCT00908037.

Keywords: Kid's ITP tool; fatigue; platelets; thrombopoietin; thrombopoietin receptor agonist.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Adolescent
Benzoates / administration & dosage
Benzoates / adverse effects
Benzoates / therapeutic use*
Child
Child, Preschool
Female
Humans
Hydrazines / administration & dosage
Hydrazines / adverse effects
Hydrazines / therapeutic use*
Infant
Male
Purpura, Thrombocytopenic, Idiopathic / diagnosis
Purpura, Thrombocytopenic, Idiopathic / drug therapy*
Purpura, Thrombocytopenic, Idiopathic / epidemiology*
Pyrazoles / administration & dosage
Pyrazoles / adverse effects
Pyrazoles / therapeutic use*
Quality of Life*
Thrombopoietin / administration & dosage
Thrombopoietin / adverse effects
Thrombopoietin / therapeutic use*
Treatment Outcome

Substances

Benzoates
Hydrazines
Pyrazoles
Thrombopoietin
eltrombopag

Associated data

ClinicalTrials.gov/NCT00908037