Table 1. Demographic and Clinical Characteristics of Patients Treated With Edaravone.
| Characteristic | No. (%) of patients | P value for EFAS vs non-EFAS | ||
|---|---|---|---|---|
| Total (N = 194) | EFAS (n = 64) | Non-EFAS (n = 130) | ||
| Baseline characteristics | ||||
| Sex | ||||
| Female | 69 (36) | 23 (36) | 46 (35) | .94 |
| Male | 125 (64) | 41 (64) | 84 (65) | |
| Symptom onset | ||||
| Spinal | 139 (71) | 47 (73) | 92 (71) | .88 |
| Bulbar | 52 (27) | 17 (27) | 35 (27) | |
| Unknown | 3 (2) | 0 | 4 (2) | |
| Age, median (IQR), y | ||||
| At disease onset | 55.6 (48.9 to 61.9) | 55.8 (48.6 to 61.2) | 55.6 (48.9 to 51.1) | .88 |
| At therapy start | 57.5 (50.7 to 63.8) | 57.3 (49.4 to 62.7) | 57.5 (51.1 to 64.4) | .69 |
| Disease duration at therapy start, mo | 16.5 (10.6 to 24.4) | 12.8 (10.0 to 17.6) | 19.1 (11.8 to 28.2) | <.001 |
| Receipt of riluzole | 186 (97) | 63 (98) | 123 (96) | .38 |
| ALSFRS-R score at start of therapy, median, (IQR), points | 37 (32 to 42) | 39.5 (36 to 42) | 34 (30 to 41) | <.001 |
| No. | 183 | 64 | 119 | |
| ΔFRS, median (IQR), points/mo | −0.58 (−0.35 to −1.06) | −0.61 (−0.46 to −0.93) | −0.58 (−0.29 to −1.22) | .83 |
| No. | 183 | 64 | 119 | |
| EESP criteria met | ||||
| Age between 20 and 75 y | 185 (95) | 64 (100) | 121 (93) | .04 |
| Definite or probable ALS | 194 (100) | 64 (100) | 130 (100) | >.99 |
| Maximum disease duration of 2 y | 144 (74) | 63 (98) | 81 (62) | <.001 |
| ALSFRS-R slope of −0.33 to −1.33 points/mo | 111 (57) | 58 (91) | 53 (41) | <.001 |
| ≥2 Points for each item of the ALSFRS-R | 41 (21) | 29 (45) | 12 (9) | <.001 |
| Preserved respiratory function | 115 (59) | 58 (91) | 57 (44) | <.001 |
| Follow-up | ||||
| Treatment duration, median (IQR), wk | 50.1 (26.1 to 78.7) | 62.0 (37.9 to 84.6) | 43.3 (24.7 to 78.0) | .03 |
| No. | 175 | 115 | 60 | |
| ALSFRS-R treatment slope, median (IQR), points/moa | −0.85 (−0.37 to −1.45) | −0.97 (−0.56 to −1.60) | −0.77 (−0.30 to −1.38) | .14 |
| No. | 134 | 53 | 82 | |
| Slope change, median (IQR), points/moa,b | −0.22 (−0.88 to 0.20) | −0.31 (−1.00 to 0.15) | −0.19 (−0.87 to 0.22) | .43 |
| No. | 131 | 53 | 78 | |
Abbreviations: ALS, amyotrophic lateral sclerosis; ALSFRS-R, ALS Functional Rating Scale–Revised; EESP, efficacy-expected subpopulation; ΔFRS, change in ALSFRS-R score slope between disease onset and baseline; EFAS, potentially eligible for the MCI186-ALS19 study; non-EFAS, ineligible for the MCI186-ALS19 study.
a
If received 4 or more consecutive cycles of edaravone.
b
Slope change is the ALSFRS-R treatment slope minus ΔFRS; negative values signify an acceleration of the ALSFRS-R score decrease.