Table 1.
Change from baseline to weeks 16 and 48 for PROs
| PRO | Baseline, mean (SD) [N1] (range) |
Week 16, LS mean ∆ (SE) [N1] | Week 48, LS mean ∆ (SE) [N1] | |||
| Tofacitinib 5 mg twice daily (N=133) |
Placebo (N=136) |
Tofacitinib 5 mg twice daily (N=133) |
Placebo (N=136) |
Tofacitinib 5 mg twice daily (N=133) |
Placebo→ tofacitinib 5 mg twice daily (N=136) |
|
| Pain | ||||||
| Total back pain (NRS 0–10)‡,§ | 6.9 (1.5) (1–10) | 6.9 (1.6) (2–10) | –2.57 (0.19)*** [129] | –0.96 (0.19) [131] | –3.57 (0.22)* [113] | –2.87 (0.22) [112] |
| Nocturnal spinal pain (NRS 0–10)§ | 6.8 (1.9) (0–10) | 6.8 (1.9) (1–10) | –2.67 (0.20)*** [129] | –0.84 (0.20) [131] | –3.52 (0.23) [112] | –3.01 (0.23) [112] |
|
BASDAI overall spinal pain (Question 2; NRS 0–10)§ |
7.3 (1.7) (2–10) | 7.3 (1.6) (2–10) | –2.85 (0.20)*** [129] | –1.34 (0.20) [131] | –3.79 (0.22)* [113] | –3.07 (0.22) [113] |
| Fatigue | ||||||
| FACIT-F§ | ||||||
| Total score (0–52)¶ | 27.2 (10.7) (4–52) | 27.4 (9.3) (1–46) | 6.54 (0.80)*** [129] | 3.12 (0.79) [131] | 9.54 (0.90) [112] | 7.35 (0.89) [111] |
| Experience domain score (0–20) | 8.9 (4.3) (0–20) | 8.7 (4.0) (0–17) | 2.85 (0.36)*** [129] | 1.29 (0.36) [131] | 4.22 (0.40) [112] | 3.40 (0.40) [111] |
| Impact domain score (0–32) | 18.3 (6.9) (4–32) | 18.8 (5.9) (1–30) | 3.68 (0.49)** [129] | 1.81 (0.49) [131] | 5.32 (0.54)* [112] | 3.95 (0.54) [111] |
| BASDAI fatigue (Question 1; NRS 0–10)§ | 6.8 (1.5) (2–10) | 6.8 (1.7) (2–10) | –2.36 (0.20)*** [129] | –1.08 (0.20) [131] | –3.21 (0.22)* [113] | –2.57 (0.22) [113] |
| HRQoL | ||||||
| ASQoL (0–18)¶ †† | 11.6 (4.7) (0–18) | 11.3 (4.2) (0–18) | –4.03 (0.40)*** [129] | –2.01 (0.41) [130] | –5.97 (0.45)* [112] | –4.70 (0.45) [112] |
| SF-36v2 PCS (norm-based)¶ †† | 33.5 (7.3) (17.9–57.3) | 33.1 (7.0) [135] (14.7–53.7) | 6.69 (0.59)*** [129] | 3.14 (0.59) [130] | 8.81 (0.72) [112] | 7.39 (0.71) [111] |
| SF-36v2 MCS (norm-based)†† | 39.4 (11.1) (14.1–65.3) | 39.8 (12.7) [135] (8.0–64.7) | 3.45 (0.91) [129] | 2.13 (0.92) [130] | 7.07 (0.93) [112] | 6.35 (0.92) [111] |
| SF-36v2 domain scores (norm-based)†† | ||||||
| Physical functioning | 36.2 (9.5) (16.2–57.1) | 36.6 (9.2) [135] (16.2–57.1) | 5.52 (0.67)** [129] | 3.29 (0.67) [130] | 7.80 (0.78) [112] | 6.94 (0.77) [111] |
| Role-physical | 34.1 (9.2) (18.4–56.6) | 34.0 (8.1) [135] (18.4–56.6) | 6.13 (0.74)** [129] | 3.13 (0.75) [130] | 8.66 (0.87) [112] | 7.29 (0.86) [111] |
| Bodily pain | 32.5 (6.4) (19.2–54.2) | 32.2 (6.0) [135] (19.2–50.1) | 7.93 (0.71)*** [129] | 3.47 (0.71) [130] | 11.67 (0.92) [112] | 9.55 (0.91) [111] |
| General health | 31.4 (8.4) (16.8–59.0) | 31.5 (8.2) [135] (16.8–52.9) | 5.00 (0.62)*** [129] | 1.76 (0.62) [130] | 6.31 (0.78) [112] | 5.10 (0.77) [111] |
| Vitality | 40.3 (9.3) (22.0–66.9) | 38.5 (9.2) [135] (22.0–60.9) | 5.34 (0.86) [129] | 3.56 (0.87) [130] | 9.83 (1.00) [112] | 9.28 (0.99) [111] |
| Social functioning | 36.7 (10.5) (13.4–56.4) | 36.9 (10.5) [135] (13.4–56.4) | 5.45 (0.84)** [129] | 2.49 (0.84) [130] | 8.16 (0.92) [112] | 6.77 (0.92) [111] |
| Role-emotional | 35.2 (12.3) (10.2–55.7) | 36.3 (13.1) [135] (10.2–55.7) | 4.13 (1.02) [129] | 2.05 (1.02) [130] | 7.17 (1.00) [112] | 6.32 (0.99) [111] |
| Mental health | 38.7 (11.2) (8.0–63.4) | 39.5 (12.3) [135] (8.0–63.4) | 3.57 (0.89) [129] | 2.49 (0.89) [130] | 7.10 (0.96) [112] | 6.45 (0.95) [111] |
| EQ-5D-3L dimension scores (1–3)†† | ||||||
| Mobility | 1.8 (0.4) (1–2) | 1.7 (0.5) (1–2) | –0.23 (0.04)** [129] | –0.06 (0.04) [131] | –0.32 (0.05) [112] | –0.26 (0.05) [112] |
| Self-care | 1.6 (0.5) (1–2) | 1.6 (0.5) (1–2) | –0.21 (0.04) [129] | –0.20 (0.04) [131] | –0.33 (0.05) [112] | –0.33 (0.05) [112] |
| Usual activities | 1.9 (0.4) (1–3) | 1.9 (0.4) (1–3) | –0.18 (0.05) [129] | –0.09 (0.05) [131] | –0.32 (0.05) [112] | –0.34 (0.05) [112] |
| Pain/discomfort | 2.3 (0.5) (2–3) | 2.3 (0.5) (1–3) | –0.30 (0.04)*** [129] | –0.12 (0.04) [131] | –0.37 (0.05) [112] | –0.36 (0.05) [112] |
| Anxiety/depression | 1.7 (0.6) (1–3) | 1.6 (0.6) (1–3) | –0.11 (0.05) [129] | –0.10 (0.05) [131] | –0.17 (0.05) [112] | –0.21 (0.05) [112] |
| EQ-VAS (0–100 mm)†† | 46.9 (18.6) (10.0–95.0) | 47.4 (21.9) [135] (5.0–95.0) | 13.0 (1.84)*** [128] | 2.89 (1.84) [130] | 20.64 (1.88) [112] | 18.00 (1.86) [111] |
| Work productivity | ||||||
| WPAI†† | ||||||
| Activity impairment, % | 56.5 (23.4) (0–90) | 56.0 (21.4) (0–100) | –19.03 (1.97)*** [129] | –5.63 (1.97) [131] | –27.37 (2.34)** [112] | –19.77 (2.31) [112] |
| Absenteeism (work time missed), % | 9.9 (22.4) [81] (0–100) | 11.5 (24.6) [88] (0–100) | –3.65 (2.66) [74] | 0.88 (2.62) [81] | –8.10 (2.14) [61] | –5.79 (2.05) [70] |
| Presenteeism (impairment while working), % | 48.4 (26.3) [79] (0–100) | 49.6 (22.2) [85] (0–90) | –19.83 (2.27)*** [71] | –6.94 (2.30) [77] | –25.35 (2.77) [58] | –23.00 (2.66) [70] |
| Overall work impairment, % | 50.8 (27.4) [79] (0–100) | 53.5 (23.1) [85] (0–100) | –21.49 (2.51)*** [71] | –7.64 (2.56) [76] | –27.63 (3.01) [58] | –23.22 (2.90) [69] |
Mean baseline values and LS mean changes from baseline to weeks 16 and 48 for PROs in patients with AS receiving tofacitinib 5 mg twice daily or placebo→tofacitinib 5 mg twice daily.†
*p≤0.05, **p<0.01, ***p<0.001 versus placebo (week 16) or placebo→tofacitinib 5 mg twice daily (week 48). For endpoints not prespecified for type I error control, p values are reported without multiple comparison adjustment.
†Patients receiving placebo advanced to tofacitinib 5 mg twice daily at week 16.
‡Change from baseline at week 16 was a type I error-controlled secondary endpoint.
§Week 16 results are based on MMRM including all postbaseline data to week 16 (data cut-off 19 December 2019; data snapshot 29 January 2020); week 48 results based on another MMRM including all postbaseline data to week 48.
¶Change from baseline at week 16 was a global type I error-controlled endpoint.
††Week 16 results based on ANCOVA including all postbaseline data at week 16 (data cut-off 19 December 2019; data snapshot 29 January 2020); week 48 results based on MMRM including all postbaseline data to week 48.
N, number of patients in full analysis set; N1, number of patients with observation at visit, if different from the full analysis set.
∆, change from baseline; ANCOVA, analysis of covariance; AS, ankylosing spondylitis; ASQoL, Ankylosing Spondylitis Quality of Life; BASDAI, Bath Ankylosing Spondylitis Disease Activity Index; BID, twice daily; EQ-5D-3L, EuroQol-Five Dimension-Three Level Health Questionnaire; EQ-VAS, EuroQol Visual Analogue Scale; FACIT-F, Functional Assessment of Chronic Illness Therapy-Fatigue; HRQoL, health-related quality of life; LS, least squares; MCS, Mental Component Summary; MMRM, mixed model for repeated measures; NRS, numerical rating scale; PCS, Physical Component Summary; PRO, patient-reported outcome; SF-36v2, Short Form-36 Health Survey Version 2; WPAI, Work Productivity and Activity Impairment questionnaire.