Quality of life is an important indication for second-line treatment in children with immune thrombocytopenia

Kristin A. Shimano*, Cindy Neunert, James B. Bussel, Robert J. Klaassen, Rukhmi Bhat, Yves D. Pastore, Michele P. Lambert, Carolyn M. Bennett, Jenny M. Despotovic, Peter Forbes, Rachael F. Grace

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

1 Scopus citations

Abstract

Background: The decision to initiate second-line treatment in children with immune thrombocytopenia (ITP) is complex and involves many different factors. Methods: In this prospective, observational, longitudinal cohort study of 120 children from 21 centers, the factors contributing to the decision to start second-line treatments for ITP were captured. At study entry, clinicians were given a curated list of 12 potential reasons the patient required a second-line treatment. Clinicians selected all that applied and ranked the top three reasons. Results: Quality of life (QOL) was the most frequently cited reason for starting a second-line therapy. Clinicians chose it as a reason to treat in 88/120 (73%) patients, as among the top three reasons in 68/120 (57%), and as the top reason in 32/120 (27%). Additional factors ranked as the top reason to start second-line treatment included severity of bleeding (22/120, 18%), frequency of bleeding (19/120, 16%), and severity of thrombocytopenia (18/120, 15%). Patients for whom QOL (p =.006) or sports participation (p =.02) were ranked reasons were more likely to have chronic ITP, whereas those for whom severity (p =.003) or frequency (p =.005) of bleeding were ranked reasons were more likely to have newly diagnosed or persistent ITP. Parental anxiety, though rarely the primary impetus for treatment, was frequently cited (70/120, 58%) as a contributing factor. Conclusion: Perceived QOL is the most frequently selected reason pediatric patients start second-line therapies for ITP. It is critical that studies of treatments for childhood ITP include assessments of their effects on QOL.

Original languageEnglish (US)
Article numbere29023
JournalPediatric Blood and Cancer
Volume68
Issue number6
DOIs
StatePublished - Jun 2021

Keywords

  • bleeding
  • children
  • immune thrombocytopenia
  • quality of life
  • treatment

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

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