Outcome measures for adult and pediatric hemophilia patients with inhibitors

Research output: Contribution to journalReview article

Abstract

Recent advancements in almost all aspects of hemophilia treatment have vastly improved patient care and management, and new and emerging treatments hold the promise of further progress. However, there remains a scarcity of data on long-term outcomes in hemophilia, particularly among those patients with inhibitors, for whom no validated outcome assessment tools are currently available. At the 15th Zürich Haemophilia Forum, an expert panel reviewed the most important outcome measures in inhibitor patients and considered the challenges associated with assessing outcomes in this population. A framework for outcome assessment in inhibitor patients incorporates traditional hemophilia outcome measures, such as bleed frequency and mortality, alongside measures of health, functioning, disability, social participation, quality of life, and economic considerations. It is important to remember that inhibitor patients differ in their clinical needs, perspectives, and priorities according to age, inhibitor status, degree of joint disease, and activity levels; as a result, the relative importance of different outcome measures will change throughout an inhibitor patient's life. Challenges inherent in measuring long-term outcomes in inhibitor patients include the small number of known patients, the subjective nature of many outcome assessment tools, and the risk of overburdening patients with repeated requests to complete questionnaires or participate in studies. Therefore, there is an urgent need to reach consensus on the most important and appropriate assessment tools for measuring outcomes in this population. These tools should ideally be standardized, easily applied, and internationally applicable in order to collect and generate quality outcome data.

Details

Authors
  • Cedric Hermans
  • Günter Auerswald
  • Gary Benson
  • Gerry Dolan
  • Anne Duffy
  • Victor Jiménez-Yuste
  • Rolf Ljung
  • Massimo Morfini
  • Thierry Lambert
  • Mehdi Osooli
  • Silva Zupančić Šalek
Organisations
External organisations
  • Saint-Luc University Hospital
  • Klinikum Bremen-Mitte
  • Queen's Medical Centre, Nottingham
  • Irish Haemophilia Society
  • University Hospital La Paz
  • Skåne University Hospital
  • University of Osijek
  • University Hospital Centre Zagreb
  • University of Zagreb
  • Autonomous University of Madrid
  • No affiliation available (private)
  • Bicêtre Hospital
  • University of Paris-Sud
Research areas and keywords

Subject classification (UKÄ) – MANDATORY

  • Hematology

Keywords

  • adults, children, hemophilia, inhibitors, outcome assessment, treatment outcome
Original languageEnglish
Pages (from-to)103-111
Number of pages9
JournalEuropean Journal of Haematology
Volume99
Issue number2
Publication statusPublished - 2017 Aug 1
Publication categoryResearch
Peer-reviewedYes