Evaluating the impact of thrombopoietin receptor agonist medications on patient outcomes and quality of life in paediatric immune thrombocytopenia through semi‐structured interviews

Abstract

Summary Over the last decade, treatment of immune thrombocytopenia (ITP) in children has advanced to include thrombopoietin receptor agonist (TPO‐RA) medications. Concurrently, there has been an increased emphasis on patient‐reported outcomes—especially quality of life—to guide treatment. Assessing the impact of TPO‐RAs on quality of life in paediatric ITP is therefore a priority. In this single‐centre integrative mixed‐methods study, a cohort of children with ITP prescribed a TPO‐RA was identified. These children and/or their caregivers were invited to participate in semi‐structured interviews focussed on quality‐of‐life measures. Independently, a retrospective chart review collected ITP‐related data (platelet count, bleeding events) and TPO‐RA data (dosing, side effects). Among the 23 eligible patients, 20 were represented in interviews. On chart review, 11/20 patients responded to TPO‐RA by meeting platelet count criteria of ≥50 × 109/L for six or more weeks in the absence of rescue therapy. In interviews with these children and/or their parents, 19/20 expressed the TPO‐RA had ‘worked’, with 11/20 reporting benefit to mood and 11/20 reporting increased participation in activities/sports. Concerns were raised in interviews about TPO‐RA medication cost (17/20), medication administration (10/20) and potential side effects (10/20). In conclusion, this study suggests that TPO‐RA use in children with ITP improves quality of life.

Burden of immune thrombocytopenia (ITP): Special considerations for refractory ITP

“…Negative concerns were raised related to the costs of the medication, potential long-term side effects and challenges related to taking the TPO-RA on an empty stomach. 32 In conclusion, with the second-line treatment of patients with ITP with TPO-RAs, Rituximab and/or splenectomy, there was no published evidence that Rituximab or splenectomy led to an improvement in HRQoL. With regard to TPO-RAs, there were mixed results on their influence on HRQoL, but a clear and reproducible effect leading to an improvement of HRQoL was not easily recognizable based on the published evidence.…”

“…Positive themes were that their child's mental health had improved since taking the TPO‐RA, their child was able to resume sports participation and overall reduced caregiver anxiety. Negative concerns were raised related to the costs of the medication, potential long‐term side effects and challenges related to taking the TPO‐RA on an empty stomach 32 …”

Face validity of the Kids’ ITP Tools (KIT) in the era of thrombopoietin receptor agonists

“…[6][7][8][9] However, cost and treatment duration may be a stressor for patients and their families. 6 Additionally, ASH guidelines place reliance on patient/family preference when selecting a second-line therapy, which further supports the need to update the KIT. 5 The purpose of this study was to review and update the internationally validated KIT to reflect current clinical practice to improve its face validity when evaluating HRQoL, both for pediatric ITP patients who have been prescribed TPO-Ras and those who have not.…”

A cost–utility analysis of thrombopoietin receptor agonists for treating pediatric immune thrombocytopenia purpura after failure of first‐line therapies

“…Up to now, the impact of TPO‐RAs on healthcare utilization for pediatric patients with ITP is complex and not yet fully understood. TPO‐RAs may improve health‐related quality‐of‐life (HRQoL) for both adult patients and children 14,15 . Individuals on TPO‐RA medications may require less hospital rescue care for ITP management, including emergency assessments and treatments.…”

“…TPO-RAs may improve health-related quality-of-life (HRQoL) for both adult patients and children. 14,15 Individuals on TPO-RA medications may require less hospital rescue care for ITP management, including emergency assessments and treatments. As such, a European qualitative observational study recommended starting TPO-RAs therapy earlier to reduce both hospitalization and therapy costs.…”