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

Abstract

Summary 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.

Updated international consensus report on the investigation and management of primary immune thrombocytopenia

“…The impact of eltrombopag on HRQoL in children with persistent or chronic ITP was evaluated in the PETIT trial (evidence level Ib). 294 There was a wide variability in KIT scores at baseline. Small improvements in HRQoL were noted in responders to eltrombopag aged 1 to 5 years and 12 to 17 years but not those in who were 6 to 11 years old.…”

Use of Eltrombopag in Children With Chronic Immune Thrombocytopenia (ITP): A Real Life Retrospective Multicenter Experience of the Italian Association of Pediatric Hematology and Oncology (AIEOP)

“…Recently, Giordano et al explored with a narrative approach the real emotional impact of chronic ITP revealing that parent's anxiety and restrictions on children activities and sports participation have a prominent impact on their daily life with significant social, scholastic, and relational implications (13). Eltrombopag for easy administration and limited side effects could play a role in improving HRQoL but further studies are needed (35).…”

Efficacy and safety of eltrombopag in the treatment of Chinese children with chronic immune thrombocytopenia

“…Although ITP is a self-limiting process for the majority of children, the disease may become chronic in up to 20% of patients [3]. Pediatric chronic ITP (cITP) (lasting ≥12 months) is commonly associated with limited activities and fear of bleeding that can obviously affect the quality of life [4]. It is important that children with cITP receive appropriate treatment due to the bleeding risk associated with reduced platelet count.…”