REQUERIMIENTOS DEL CLIENTE Y RESTRICCIONES DEL SISTEMA

Exploratory analysis of participant self-reported outcomes

Descriptive statistics (mean and 95% CIs) of the participant self-reported outcome measures at each time point and at 6 weeks compared with baseline (difference) are presented inTable 18and summarised overall by time point. Owing to the small number of participants taking part, further outcome summaries by potential confounders such as disease state, age, sex, level of support and recruitment site, were not undertaken.

This study was not powered to detect any changes in outcome measure score, and there was no change in average pain scores; however, there was a slight reduction in interference from pain (–1.6, 95% CI –2.8 to–0.4) and a modest increase (0.7, 95% CI 0.3 to 1.2) in self-efficacy scores (seeTable 18). There was no overall change in the intensity of common end-of-life symptoms (ESAS), health-related quality of life [EuroQol-5 Dimensions (EQ-5D)] or satisfaction with information about medicines (SIMS).

Figures 10–12present histograms of the change in average pain, pain interference and self-efficacy scores, respectively. These histograms show that, for the majority of participants, average pain intensity worsened

TABLE 18 Descriptive analysis of participant self-reported outcomes

Participant- reported outcome

Study time point

Differencea

Baseline Week 2 Week 4 Week 6

BPI (scale 0–10) Average pain 4.3 (3.1 to 5.6) 3.3 (2 to 4.6) 4.1 (2.4 to 5.7) 3.5 (2.3 to 4.8) _–0.2 (_–1.5 to 1.1) Pain interference 4.3 (3.1 to 5.5) 3.5 (1.8 to 5.2) 2.7 (2.4 to 5) 2.5 (1.4 to 3.6) –1.6 (–2.8 to–0.4) Worst pain 6.1 (4.5 to 7.6) 5.3 (3.4 to 7.2) 5.9 (3.9 to 7.8) 5.4 (3.6 to 7.2) _–0.1 (_–1.5 to 1.4) Least pain 2.8 (1.4 to 4.2) 2 (0.7 to 3.3) 2.7 (1.2 to 4.2) 2.5 (1.1 to 3.8) 0.1 (–1.1 to 1.3) Present pain 2.9 (1.8 to 4.1) 2.7 (1.5 to 3.8) 3.5 (1.8 to 5.1) 3.7 (1.9 to 5.5) 0.8 (_–0.8 to 2.4) SES (scale 0–10) Total score 7.1 (6.3 to 7.9) 7 (6.2 to 7.8) 7.5 (6.5 to 8.5) 7.7 (6.7 to 8.6) 0.7 (0.3 to 1.1) ESAS (scale 0–10) Total score 2.3 (1.7 to 2.9) 2.6 (1.6 to 3.6) 2.9 (2.3 to 3.5) 2.7 (1.9 to 3.5) 0.1 (_–0.5 to 0.7) EQ-5D (scale 0–1) Health status 0.52 (0.4 to 0.63) 0.56 (0.44 to 0.68) 0.52 (0.4 to 0.63) 0.58 (0.44 to 0.7) 0.05 (_–0.11 to 0.21) SIMS (scale 0–17) Total score 11.7 (9.7 to 13.8) 13.8 (11.4 to 16.2) 13.4 (11.4 to 15.4) 13.7 (7.5 to 20) 1.7 (_–4.5 to 7.8)

a Differences were calculated as the mean difference between baseline and 6-week follow-up time points. Notes

Data are presented as mean (95% CIs).

An intention-to-treat approach was taken, therefore the denominator at baseline wasn₌19 and at all follow-up time points wasn=15.

– 8 – 7 – 6 – 5 – 4 – 3 – 2 – 1 0 1 2 3 Change in average BPI at 6 weeks from baseline

(< 0 indicates improvement) 0 1 2 3 4 5 Count

FIGURE 10 Histogram of change in BPI average pain intensity scores. N.B. change scores calculated week 6 score minus baseline score.

board on BPI pain interference (i.e. scores of≥0) and, largely, there were improvements for all participants on the self-efficacy scale (i.e. scores of>0).Table 19presents the variability (SD) with 95% CI for

candidate primary outcome measures, along with the estimated effect size for the change in average pain, pain intensity and self-efficacy at 6 weeks compared with baseline.

The number, proportion and 95% CI around the proportion of participants with clinically meaningful reduction in average pain and pain interference are summarised inTable 20. These data show that at follow-up weeks 2 and 6 there were more responders based on pain interference than average pain intensity.

– 8 – 7 – 6 – 5 – 4 – 3 – 2 – 1 0 1 2 3 Change in BPI interference at 6 weeks from baseline

(< 0 indicates improvement) 0 1 2 3 4 Count

FIGURE 11 Histogram of change in BPI pain interference score. N.B. change scores calculated week 6 score minus baseline score.

– 1.5 – 1.0 – 0.5 0.0 0.5 1.0 1.5 2.0 2.5 Change in self-efficacy at 6 weeks from baseline

(> 0 indicates improvement) 0 2 4 6 Count

FIGURE 12 Histogram of change in SES score. N.B. change scores calculated week 6 score minus baseline score;

Participant acceptability of self-reported outcomes

Generally, participants’experience of completing the self-reported outcome measures was acceptable; however, there were some limitations related to the wording of some questions given the end-of-life context (e.g.‘normal work’and‘enjoyment of life’on the interference subscale of the BPI). A small number of participants criticised the‘duplicity’(H4Pt001) of some questions, given the combination of five different measures. Overall, there was dislike for the SIMS as many participants experienced difficulty remembering specific information that they had received about their medicines over the preceding 2 weeks.

Overall, completion rates for the questionnaire packs were high: all questionnaires were completed by participants with a researcher at each time point. The proportion of missing data at individual item level was very low (Table 21) and did not prevent any summary scores from being calculated at any time point. The proportion of missing data was<3% for all participant self-reported outcome measures at all time points, except for BPI at the week 4 follow-up (3.9%). The most commonly missing item on the BPI was the final item about the extent to which pain interferes with enjoyment of life (missing in three cases from the same participant); a number of participants responded that they felt that this question was inappropriate for people approaching the end of life. Similarly, for the EQ-5D, one response to the final item asking respondents to rate their overall health from best to worst was missing at all time points (from the same participant in each case). The field notes kept by the researchers identified a general lack of acceptability of this question by participants. One participant responded during the end-of-study interview, ‘it’s a stupid question to ask people in palliative care’(H1Pt011-C), when asked specifically about this response item. The SIMS was least liked by the participants; nevertheless the proportion of missing data was extremely low.

TABLE 20 Responders based on reductions in average pain intensity and pain interference at each follow-up

Time point

Responders

BPI average pain BPI pain interference

Number of responders % 95% CI Number of responders % 95% CI

Follow-up week 2 4 26.7 7.8 to 55.1 6 40.0 16.3 to 67.7

Follow-up week 4 3 20.0 4.3 to 48.1 3 20.0 4.3 to 48.1

Follow-up week 6 3 20.0 4.3 to 48.1 7 46.7 4.3 to 48.1

Notes

The denominator at all follow-ups wasn=15.

Responders were classified as participants reporting a≥2-point or≥30% reduction in average pain score or a≥1-point reduction in pain interference at each follow-up time point compared with baseline.

TABLE 19 The SDs and 95% CIs for candidate primary outcome variables for a definitive trial

Time point

Candidate primary outcome variable, SD (95% CI)

BPI average pain BPI pain interference SES

Follow-up week 2 2.6 (1.9 to 3.8) 2.5 (1.9 to 3.7) 1.6 (1.2 to 2.4)

Follow-up week 4 2.4 (1.7 to 3.7) 3.1 (2.2 to 4.8) 1.4 (1.0 to 2.2)