Recognizing the need for personalization of haemophilia patient-reported outcomes in the prophylaxis era

Michael Recht, B. A. Konkle, S. Jackson, E. J. Neufeld, K. Rockwood, S. Pipe

Research output: Contribution to journalArticle

15 Citations (Scopus)

Abstract

The safety and efficacy of treatment options for patients with haemophilia have significantly improved over the last two decades, particularly with greater utilization of prophylactic approaches. Consequently, it is becoming increasingly difficult to differentiate the treatment benefits of available choices based on standard endpoints such as annualized bleeding rates and joint health scores. Patient-reported outcomes (PROs) have shown limited ability to discriminate between treatment outcomes, in part because of their comprehensive nature; i.e. differences in specific outcomes meaningful to individual patients are masked by a global scoring system based on a fixed set of items, many of which may be unimportant for any given patient. There is a clear need for new outcome measures. Initiatives to develop patient-centric outcomes that capture clinically meaningful change are ongoing. One such approach, goal attainment scaling (GAS), allows patients, in collaboration with a trained clinician, to select goals from a medical condition-specific menu of options and subsequently facilitates quantitative assessment of goal realization. Thus, it is fully personalized and sensitive to small, often idiosyncratic, treatment benefits, such as improvements in functional capacity. In this paper, we present the underlying rationale for GAS and one other novel approach to PRO personalization, and discuss their potential to augment current outcome measures by reliably detecting and quantifying treatment effects in individuals with haemophilia on prophylaxis.

Original languageEnglish (US)
JournalHaemophilia
DOIs
StateAccepted/In press - 2016

Fingerprint

Hemophilia A
Outcome Assessment (Health Care)
Therapeutics
Joints
Patient Reported Outcome Measures
Hemorrhage
Safety
Health

Keywords

  • Function
  • Haemophilia
  • Outcomes
  • Patient-reported outcomes
  • Personalized medicine
  • Quality of life

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

Cite this

Recognizing the need for personalization of haemophilia patient-reported outcomes in the prophylaxis era. / Recht, Michael; Konkle, B. A.; Jackson, S.; Neufeld, E. J.; Rockwood, K.; Pipe, S.

In: Haemophilia, 2016.

Research output: Contribution to journalArticle

Recht, Michael ; Konkle, B. A. ; Jackson, S. ; Neufeld, E. J. ; Rockwood, K. ; Pipe, S. / Recognizing the need for personalization of haemophilia patient-reported outcomes in the prophylaxis era. In: Haemophilia. 2016.
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