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

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

Author information

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.

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.

KEYWORDS:

Kid's ITP tool; fatigue; platelets; thrombopoietin; thrombopoietin receptor agonist

PMID:
30592022
DOI:
10.1111/bjh.15732

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