Evaluation of patient-relevant outcomes of lymphedema and lipedema treatment: development and validation of a new benefit tool

Eur J Vasc Endovasc Surg. 2014 Jan;47(1):100-7. doi: 10.1016/j.ejvs.2013.10.009. Epub 2013 Oct 22.


Objectives: Patient-relevant treatment benefit is traditionally measured with health-related quality of life (HRQoL) instruments. The Patient Benefit Index (PBI) methodology allows for a more direct measurement, with the patients rating both importance and achievement of treatment goals. Here, we developed and validated a PBI version specific for the assessment of benefit in lymphedema and lipedema treatment (PBI-L).

Methods: The development included five steps: (1) open item collection; (2) consensus of items in a multidisciplinary expert panel; (3) application of the German PBI-L in a cross-sectional study (n = 301); (4) translation into English; (5) application of the English PBI-L in a randomized clinical trial (n = 82). Subscales were developed using factor analysis. Construct validity was analyzed by correlating PBI-L and convergent criteria such as HRQoL and quality of care. To test for responsiveness, the association to change in HRQoL measures was computed.

Results: Floor and ceiling effects were low. There were few missing values. Two well-interpretable subscales were found with Cronbach's alpha >0.8 each. Global and subscale scores correlated with convergent criteria and with change in disease-specific HRQoL, but not with change in generic HRQoL.

Conclusions: The PBI-L is an internally consistent, valid, and responsive instrument for the assessment of patient-relevant benefit of edema treatment.

Keywords: Lipedema; Lymphedema; Patient benefit; Patient-reported outcomes; Quality of life; Validation.

Publication types

  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't
  • Validation Study

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Cost of Illness
  • Cross-Sectional Studies
  • Europe
  • Factor Analysis, Statistical
  • Female
  • Humans
  • Lymphedema / diagnosis*
  • Lymphedema / psychology
  • Lymphedema / therapy*
  • Male
  • Middle Aged
  • Predictive Value of Tests
  • Quality of Life*
  • Reproducibility of Results
  • Severity of Illness Index
  • Surveys and Questionnaires*
  • Translating
  • Treatment Outcome
  • United States
  • Young Adult