Defining the clinically important difference in pain outcome measures

Pain. 2000 Dec 1;88(3):287-294. doi: 10.1016/S0304-3959(00)00339-0.

Abstract

The purpose of this study was to determine the levels of change on standard pain scales that represent clinically important differences to patients. Data from analgesic studies are often difficult to interpret because the clinical importance of the results is not obvious. Differences between groups, as summarized by a change in mean values over time, can be difficult to apply to clinical care. Baseline scores vary widely and group mean differences could reflect large changes in a few patients, small changes in many patients, or any combination of these outcomes. Determination of the proportion of patients who have a clinically important improvement in their pain would provide a more interpretable result with direct clinical implications. However, determining a clinically important outcome requires information about the degree of change over time that is clinically important. Data from the titration phase of a multiple cross-over randomized clinical trial of oral transmucosal fentanyl citrate (OTFC) for the treatment of cancer-related breakthrough pain were re-analyzed to examine the differences in pain scores between treatment episodes that did and did not yield adequate pain relief. The scales evaluated were absolute pain intensity difference (PID, 0-10 scale), percentage pain intensity difference (PID%, 0-100% scale), pain relief (PR, 0 (none), 1 (slight), 2 (moderate), 3 (lots), 4 (complete)), sum of the pain intensity difference (SPID over 60 min), percentage of maximum total pain relief (% Max TOTPAR over 60 min), and global medication performance (0 (poor), 1 (fair), 2 (good), 3 (very good), 4 (excellent)). Adequate relief was defined by the patient's decision not to use another dose of opioid medication as a rescue, in addition to the study medication, to treat each painful episode. One hundred thirty OTFC naive patients contributed data on 1268 episodes of breakthrough pain. The scales that were converted to a percentage change yielded the best accuracy in predicting adequate relief, with balanced sensitivity and specificity. The best cut-off point for both the % Max TOTPAR and the PID% was 33%. The best cut-off points for the absolute scales were absolute pain intensity difference of 2, pain relief of 2 (moderate), and SPID of 2. The global medication performance of 2 (good) had excellent values as well. This study presents data-derived cut-off points for the changes in several pain scales, each reflecting the clinically important improvement for patients treating breakthrough cancer pain episodes with OTFC. Confirmation in other patient populations and different pain syndromes will be needed. The use of consistent clinically important cut-off points as the primary outcome in future pain therapy clinical trials will enhance their validity, comparability, and clinical applicability.

Publication types

  • Clinical Trial
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Analgesics, Opioid / therapeutic use
  • Clinical Trials, Phase I as Topic
  • Clinical Trials, Phase II as Topic
  • Confidence Intervals
  • Cross-Over Studies
  • Double-Blind Method
  • Fentanyl / therapeutic use
  • Humans
  • Logistic Models
  • Pain Measurement / methods*
  • Pain Measurement / psychology
  • Pain, Intractable / drug therapy
  • Randomized Controlled Trials as Topic
  • Treatment Outcome*

Substances

  • Analgesics, Opioid
  • Fentanyl