Table 4.
EPP-QOL Questionnaire Scores.*
| Trial and Questionnaire Score | Afamelanotide | Placebo | P Value | ||
|---|---|---|---|---|---|
| Score | No. of Patients | Score | No. of Patients | ||
| European Union trial | |||||
| Baseline score at day 0, before dose 1 | 39.0±25.8 | 37 | 35.3±23.7 | 34 | 0.39 |
| Score at day 60, before dose 2 | 68.0±19.1 | 37 | 60.1±22.0 | 35 | 0.09 |
| Score at day 120, before dose 3 | 78.8±16.2 | 37 | 63.6±23.9 | 35 | 0.005 |
| Score at day 180, before dose 4 | 84.6±12.6 | 35 | 73.5±24.3 | 35 | 0.03 |
| Score at day 240, before dose 5 | 84.8±10.7 | 34 | 73.1±24.1 | 34 | 0.01 |
| Score at day 270, final visit | 79.7±16.1 | 32 | 67.2±25.7 | 34 | 0.06 |
| U.S. trial | |||||
| Baseline score at day 0, before dose 1 | 26.6±19.9 | 47 | 26.2±19.4 | 43 | |
| Change at day 60, before dose 2 | 44.0±25.8 | 47 | 23.4±24.6 | 43 | <0.001 |
| Change at day 120, before dose 3 | 49.8±26.4 | 46 | 30.4±25.4 | 42 | <0.001 |
| Change at day 180 | 51.1±29.1 | 46 | 36.8±25.7 | 43 | 0.02 |
| Scores at day 360, 240 days after last dose | 38.4±27.0 | 44 | 45.4±29.6 | 40 | |
*
Plus–minus values are means ±SD. Scores on the Erythropoietic Protoporphyria Quality-of-Life (EPP-QOL) questionnaire range from 0 to 100, with higher scores indicating a better quality of life. In the European Union trial, P values for the comparison of afamelanotide with placebo were determined by means of the paired Wilcoxon rank-sum test; in the U.S. trial, P values are determined by means of the Kruskal–Wallis test. Additional details about the EPP-QOL questionnaire are provided in Table S1 in the Supplementary Appendix.