Presentation Details
| Child Hemo-TEM Observer-Reported Outcome Measure: Understanding and Assessing Treatment Burden in Children With Hemophilia Meryl Brod1, Donald M.Bushnell2, Jesper Skov Neergaard3, Anne Kirstine Busk3, Vlady Ostrow4. 1The Brod Group, Mill Valley, CA, USA.2Evidera|PPD, Bethesda, MD, USA.3Novo Nordisk A/S, Søborg, Denmark.4Novo Nordisk Inc., Plainsboro, NJ, USA |
Abstract
Background: Treatment advancements have improved quality of life for patients with hemophilia. However, children with hemophilia (CwH) may still experience treatment burden, leading to adherence problems, due to factors related to administration of intravenous infusions. Objectives: This study aimed to develop and validate the Child Hemo-TEM observer-reported outcome (ObsRO) measure to assess treatment experiences of CwH based on caregiver-reported observations. Methods: Concept elicitation (CE) was performed using data gathered from a review of the current hemophilia literature and interviews with 4 clinical experts (hematologists or nurses with a 50% hemophilia patient load and ≥5 years’ experience providing care to CwH), 25 CwH (males aged 8 to 12 years with hemophilia A or B with or without inhibitors who were currently self-administering on-demand or prophylactic treatment for hemophilia), and 25 caregivers of CwH (adult parents or guardians of a male CwH aged 2 to <12 years with hemophilia A or B with or without inhibitors). Interview transcripts were analyzed for themes using Dedoose software, and emerging concepts were coded and grouped into domains. Validation was performed by collecting data from caregivers of CwH aged 2 to <12 years in the US via a web-based survey. Recruitment quotas for this phase were designed to capture input from a diverse population with a variety of prophylactic treatment frequencies and treatment administration types. Results: In the CE phase, child participants were, on average, aged 9.6 years with 8.9 years of factor treatment experience. Most participants were White (n=16; 64%) and had hemophilia A (n=23, 92%). Caregivers’ CwH were, on average, aged 6.3 years with 6.0 years of factor treatment experience. Most were White (n=11; 44%) and had hemophilia A (n=22; 88%). All CwH and caregivers’ CwH had severe hemophilia. The most frequently reported treatment experience concepts reported by CwH were self-treatment; child adherence; emotional impacts and treatment concerns; treatment-related physical impacts; interference with daily life; treatment satisfaction; and treatment experience differences based on age, regimen, and disease characteristics. The most frequently reported treatment experience concepts reported by caregivers of CwH were self-treatment, caregiver ease of use, caregiver adherence, caregiver emotional impacts and treatment concerns, interference with caregiver’s daily life, and treatment satisfaction. At the end of the CE phase, a 19-item, validation-ready version of the Child Hemo-TEM was finalized. During the validation phase, in which 187 caregivers of CwH were surveyed, 12 items were dropped from the measure due to factors such as high ceiling effects at baseline and high correlation with retained items. The final 7-item measure covered domains of treatment concerns (3 items), adherence (1 item), physical impact (1 item), and interference with daily life (2 items). The Child Hemo-TEM was found to be internally consistent (Cronbach’s α=0.855). A priori hypothesized associations were predominantly confirmed. Conclusions: This study identified key treatment-related experiences encountered by CwH, supporting development of the Child Hemo-TEM ObsRO measure. Validation found adequate evidence supporting the measure’s reliability and validity. The Child Hemo-TEM ObsRO measure may be useful to clinicians and researchers assessing treatment experience in CwH.
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No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.