Table 1. Patient Demographic and Baseline Characteristics.
| Characteristic | Patients, No. (%) | ||
|---|---|---|---|
| Placebo group | Fenfluramine group | ||
| 0.2 mg/kg/d | 0.7 mg/kg/d | ||
| No. of patients | 87 | 89 | 87 |
| Age, mean (SD), y | 14 (8) | 13 (8) | 13 (7) |
| Median (range), y | 13 (2-35) | 13 (3-35) | 13 (2-35) |
| Age group, y | |||
| 2-<6 | 9 (10) | 17 (19) | 12 (14) |
| 6-<12 | 23 (26) | 24 (27) | 25 (29) |
| 12-<18 | 29 (33) | 23 (26) | 25 (29) |
| 18-35 | 26 (30) | 25 (28) | 25 (29) |
| Sex | |||
| Male | 46 (53) | 46 (52) | 54 (62) |
| Female | 41 (47) | 43 (48) | 33 (38) |
| Race and ethnicitya | |||
| Asian | 2 (2) | 3 (3) | 4 (5) |
| Black or African American | 4 (5) | 5 (6) | 3 (3) |
| White | 71 (82) | 67 (75) | 70 (80) |
| Otherb | 0 | 1 (1) | 0 |
| Unknown, not reportedc | 10 (11) | 13 (15) | 10 (11) |
| Region | |||
| North America | 44 (51) | 45 (51) | 43 (49) |
| Europe | 41 (47) | 43 (48) | 38 (44) |
| Australia | 2 (2) | 1 (1) | 6 (7) |
| BMI, mean (SD) | 20 (5) | 20 (5) | 20 (5) |
| Median (range) | 18 (11-36) | 19 (20-47) | 19 (10-37) |
| Baseline weight, kg | |||
| <37.5 | 42 (48) | 42 (47) | 40 (46) |
| ≥37.5 | 45 (52) | 47 (53) | 47 (54) |
| Baseline median seizure frequency per 28 d, No. (range) | |||
| Drop seizures | 53 (2-1761) | 85 (4-2943) | 83 (7-1803) |
| All countable seizures | |||
| Motord | 68 (14-1761) | 106 (4-2943) | 111 (10-1897) |
| Motor and nonmotor | 120 (14-1761) | 138 (14-2967) | 152 (10-5472) |
| Previous ASM usee | |||
| Mean (SD) | 7 (4) | 7 (4) | 8 (4) |
| Median (range) | 6 (1-19) | 7 (1-18) | 7 (1-20) |
| Concomitant ASM use | |||
| Mean (SD) | 3 (1) | 3 (1) | 3 (1) |
| Median (range) | 3 (1-4) | 3 (1-5) | 3 (1-4) |
| Concomitant ASMs in ≥20% of subgroupf | |||
| Valproate (all forms) | 49 (56) | 52 (58) | 46 (53) |
| Clobazam | 38 (44) | 36 (40) | 45 (52) |
| Lamotrigine | 29 (33) | 30 (34) | 29 (33) |
| Levetiracetam | 20 (23) | 17 (19) | 23 (26) |
| Rufinamide | 18 (21) | 17 (19) | 18 (21) |
| Causeg | |||
| Unknown | 22 (25) | 24 (27) | 17 (20) |
| Structural | 13 (15) | 11 (12) | 9 (10) |
| Genetic | 15 (17) | 19 (21) | 21 (24) |
| Metabolic | 1 (1) | 3 (3) | 2 (2) |
| Infectious | 3 (3) | 1 (1) | 2 (2) |
| Immune | 0 | 0 | 1 (1) |
Abbreviations: ASM, antiseizure medication; BMI, body mass index (calculated as weight in kilograms divided by height in meters squared).
Race and ethnicity were self-reported by patients or their caregivers.
Other included American Indian or Alaskan Native and Native Hawaiian or Other Pacific Islander.
Not reported or missing. Privacy laws in some regions and countries preclude disclosure of certain personal information.
Countable seizures included generalized tonic-clonic seizure, tonic seizure, clonic seizure, atonic seizure, tonic or atonic seizure, and clearly recognizable focal seizure.
Cannabidiol (72 [27%]); stiripentol (13 [5%]). Two patients (1%) had had a corpus callosotomy.
Additional concomitant therapies: vagus nerve stimulation (82 [31%]) and ketogenic diet (11 [4%]).
Causes were grouped into categories on the basis of investigator-designated primary International League Against Epilepsy classifications.