Baseline patient characteristics and disease parameters of the cohort reported in this manuscript and the HERCULES trial
| . | Germany* (n = 60) . | HERCULES cohort (N = 145)4 . | Comparison of Germany vs HERCULES (Fisher’s exact test), P . |
|---|---|---|---|
| Age, mean (range), y | 45.7 (22-83) | 46 (18-79) | |
| Female sex, % (n/total) | 70 (42/60) | 69 (100/145) | >.9999 |
| White, % (n/total) | 98.3 (59/60) | 71.0 (97/137) | <.0001 |
| BMI, median (range), kg/m2 (n = 59) | 27 (16.0-42.6) | 30 (18-59) | |
| First occurrence of TTP, % (n/total) | 76.7 (46/60) | 57 (82/145) | .0072 |
| Hemoglobin, initial, median (range), g/dL | 8.2 (5.0-13.6) | Placebo arm (n = 63), 8.7 (6.4-15.1); verum arm (n = 62), 8.6 (5.9-15.9)† | |
| Hemoglobin, minimum, median (range), g/dL | 6.3 (4.5-10.9) | Not reported | |
| Platelets, initial, median (range), ×109/L | 13.0 (3-85) | 24 (3 – 133) | |
| Platelets, minimum, median (range), ×109/L | 10 (2-62) | Not reported | |
| LDH, initial, median (range), U/L | 985 (316-2588) | 422 (120-3343) | |
| LDH, maximum, median (range), U/L | 1088 (316-2918) | Not reported | |
| Troponin above ULN at any point, % (n/total) (n = 32) | 84.4 (27/32) | cTnI above ULN at baseline, 49.0 (71/145)‡ | |
| Creatinine, initial, median (range), µmol/L | 96.5 (52.2-470) | 80 (35-717) | |
| Creatinine, maximum, median (range), µmol/L | 120.4 (55.8-470) | Not reported | |
| ADAMTS13 activity below 10%, % (n/total) | 100 (60/60) | 85 (123/145) | .0004 |
| ADAMTS13 antibody above 12 IU/ml (ULN) at any point, % (n/total) | 91.7 (55/60) | Not reported | |
| ADAMTS13 antibody concentration, maximum, median (range), IU/ml | 75.5 (0.0-131.6) | Not reported | |
| Glasgow Coma Scale, % (n/total) | .1909 | ||
| <13 | 13.3 (8/60) | 8 (11/145) | |
| 13-15 | 83.3 (50/60) | 91 (132/145) | |
| Data missing | 3.3 (2/60) | 1 (2/145) | |
| PLASMIC score, % (n/total)5-7 | |||
| 0-4 | 1.7 (1/60) | Not reported | |
| 5-6 | 36.7 (22/60) | Not reported | |
| 7 | 60.0 (36/60) | Not reported | |
| Data missing | 1.7 (1/60) | Not reported |
| . | Germany* (n = 60) . | HERCULES cohort (N = 145)4 . | Comparison of Germany vs HERCULES (Fisher’s exact test), P . |
|---|---|---|---|
| Age, mean (range), y | 45.7 (22-83) | 46 (18-79) | |
| Female sex, % (n/total) | 70 (42/60) | 69 (100/145) | >.9999 |
| White, % (n/total) | 98.3 (59/60) | 71.0 (97/137) | <.0001 |
| BMI, median (range), kg/m2 (n = 59) | 27 (16.0-42.6) | 30 (18-59) | |
| First occurrence of TTP, % (n/total) | 76.7 (46/60) | 57 (82/145) | .0072 |
| Hemoglobin, initial, median (range), g/dL | 8.2 (5.0-13.6) | Placebo arm (n = 63), 8.7 (6.4-15.1); verum arm (n = 62), 8.6 (5.9-15.9)† | |
| Hemoglobin, minimum, median (range), g/dL | 6.3 (4.5-10.9) | Not reported | |
| Platelets, initial, median (range), ×109/L | 13.0 (3-85) | 24 (3 – 133) | |
| Platelets, minimum, median (range), ×109/L | 10 (2-62) | Not reported | |
| LDH, initial, median (range), U/L | 985 (316-2588) | 422 (120-3343) | |
| LDH, maximum, median (range), U/L | 1088 (316-2918) | Not reported | |
| Troponin above ULN at any point, % (n/total) (n = 32) | 84.4 (27/32) | cTnI above ULN at baseline, 49.0 (71/145)‡ | |
| Creatinine, initial, median (range), µmol/L | 96.5 (52.2-470) | 80 (35-717) | |
| Creatinine, maximum, median (range), µmol/L | 120.4 (55.8-470) | Not reported | |
| ADAMTS13 activity below 10%, % (n/total) | 100 (60/60) | 85 (123/145) | .0004 |
| ADAMTS13 antibody above 12 IU/ml (ULN) at any point, % (n/total) | 91.7 (55/60) | Not reported | |
| ADAMTS13 antibody concentration, maximum, median (range), IU/ml | 75.5 (0.0-131.6) | Not reported | |
| Glasgow Coma Scale, % (n/total) | .1909 | ||
| <13 | 13.3 (8/60) | 8 (11/145) | |
| 13-15 | 83.3 (50/60) | 91 (132/145) | |
| Data missing | 3.3 (2/60) | 1 (2/145) | |
| PLASMIC score, % (n/total)5-7 | |||
| 0-4 | 1.7 (1/60) | Not reported | |
| 5-6 | 36.7 (22/60) | Not reported | |
| 7 | 60.0 (36/60) | Not reported | |
| Data missing | 1.7 (1/60) | Not reported |
Bold P values indicate statistical significance (P < .05).
cTnl, cardiac troponin; IULN, upper limit of normal.
n = 60 unless indicated otherwise due to missing data. Differences in the HERCULES cohort were tested using the Fisher’s exact test if sufficient data were available.
Source data from Sanofi are on file shared by the company.
Data source is the Orphan Maintenance Assessment Report (EMA/541816/2018). Please note the difference in the time point of troponin assessment (at any time point in the German cohort and at baseline in HERCULES).