Patient-reported outcomes

For the EQ-5D utility index and the MacTSQ, an instrument designed to assess patients’ satisfaction with treatment for nAMD, higher summary scores represent better utility and a higher treatment satisfaction, respectively. For the MacDQol, an instrument designed to assess macular disease-specific quality of life, lower scores represent less impact of nAMD on quality of life. HUI3 data were also collected, but have been used only for the health economics analysis. Baseline median EQ-5D utilities were similar by drug and treatment frequency (Table 23). Median MacDQoL and MacTSQ scores and EQ-5D utilities were also very similar at 1 and 2 years (see Table 23). The distribution of responses to individual EQ-5D questions at baseline and 2 years by drug and treatment regimen is given in Appendix 3. EQ-5D was dichotomised as ‘perfect health’ (EQ-5D score of 1) compared with less than perfect health, and MacTSQ was dichotomised at the median (i.e. < median vs. ≥ median). There were no significant differences in the odds of a patient having perfect health by either drug or treatment regimen (p = 0.51 and 0.64, respectively), or of a patient having ‘good’ treatment satisfaction compared with ‘low’ treatment satisfaction (p = 0.23 and p = 0.47) (Figure 32). When analysing the MacDQoL score, this outcome was analysed on a reverse scale as outlined in Chapter 2 (see Statistical methods). As with EQ-5D score, there were no significant differences between groups (p = 0.74 for the drug comparison and p = 0.73 for the treatment regimen comparison) (see Figure 32).

TABLE 23

The EQ-5D utility, macular disease-specific quality of life and treatment satisfaction scores

FIGURE 32

The EQ-5D utility, macular disease-specific quality of life and treatment satisfaction scores (a) by drug and (b) by treatment regimen. Ratios of < 1 reflect better score in the ranibizumab or continuous groups. Circles = GMR (more...)

In addition to comparing scores across the cohort as a whole, we also investigated whether or not utility, macular disease-specific quality of life and treatment satisfaction differed between the subgroups of participants when vision in the study eye was > 5 letters better than in the non-study eye (i.e. vision in the study eye better than the fellow eye vs. the same or worse). The results of this post hoc subgroup analysis are shown in Figure 33. For all three patient-reported outcomes, no statistically significant difference between the subgroups was found.

FIGURE 33

The EQ-5D utility, macular disease-specific quality of life and treatment satisfaction scores for subgroups of participants with vision in the study eye > 5 letters better and ≤ 5 letters better (a) by drug and (b) by treatment (more...)

Blood pressure

Descriptive summaries of blood pressure measurements were regularly reviewed by the DMSC throughout the trial. Average diastolic and systolic blood pressure at baseline, 1 year and 2 years are given in Table 24. Serial measurements by group are shown in Figures 34 and 35; there did not appear to be any differences in blood pressure over time by drug or by treatment regimen. These data were not formally compared at 2 years.

TABLE 24

Blood pressure

FIGURE 34

Systolic blood pressure by visit (a) by drug and (b) by treatment regimen.

FIGURE 35

Diastolic blood pressure by visit (a) by drug and (b) by treatment regimen.

Changes in best corrected distance visual acuity between consecutive visits

Changes in BCVA between consecutive visits were monitored throughout the trial. In particular, a decrease of ≥ 15 letters in the number of letters read on a ETDRS chart (equivalent to the loss of three lines of letters) was considered to be a visually significant event. BCVA was measured at every visit but a refraction to check the optical correction was carried out at intermediate visits only if the VA dropped by ≥ 15 letters or a refractive change was suspected. Overall, 127 participants (21%) experienced a drop of ≥ 15 letters between visits on at least one occasion. A breakdown by group is shown in Table 25.

TABLE 25

Number and percentage of patients in each group with a decrease of ≥ 15 letters between consecutive visits during the trial

Worsening angina

Incident or worsening angina, defined as a change of two or more classes, or from class 3 to hospital admission for angina, using the Canadian Cardiovascular Society (CCS) classification, was also monitored. However, incident or worsening of angina was not defined as a SAE. (Hospital admission for angina was, by definition, considered to be a SAE.) Worsening angina was reported for just eight participants (Table 26).

TABLE 26

Number and percentage of patients in each group reporting worsening angina during the trial

Summary

Scores for generic and disease-/treatment-specific HRQoL were similar by drug and treatment regimen at both 1 and 2 years. No differences for subgroups of participants with better or worse vision in the study eye were found for any of the three HRQoL measures. No differences over time or between groups were observed for blood pressure. Overall, 127 participants (21%) experienced a drop of ≥ 15 letters between consecutive visits at some point during follow-up.