Treatment modalities
| . | Germany (n = 60) . | HERCULES cohort (N = 145) . | Comparison of Germany vs HERCULES (Fisher’s exact test), P . |
|---|---|---|---|
| Rituximab treatment, % (n/total) | 80 (48/60) | 43 (63/145) | <.0001 |
| Rituximab, % (n/total) | |||
| Frontline (within 72 h) | 18.3 (11/60) | 17 (25/145) | .8398 |
| Delayed | 60.0 (36/60) | ||
| Data missing | 1.7 (1/60) | ||
| Day of first rituximab after disease onset, median (range), d | 7 (0-35) | Not reported | |
| Cumulative rituximab dose, median (range), mg | 2000 (562.5-5000) | Not reported | |
| PEX treatment, % (n/total) | 96.7 (58/60) | 100 (145/145) | .0846 |
| Days of PEX treatment (n = 58) | |||
| Median, no. (range) | 9 (2-41) | ||
| Mean, no. (95% CI) | 11.8 (9.5-14.2) | ||
| Days of PEX under caplacizumab treatment | |||
| Median, no. (range) | 4 (0-22) | Verum arm (n = 72), 5 (1-35) | |
| Mean, no. (95% CI) | 5.3 (4.2-6.4) | Verum arm (n = 72), 5.8 (4.8-6.8) | |
| Caplacizumab doses, median (range), no. | 34.0 (2-89) | ||
| Caplacizumab treatment duration, median (range), d | 34 (2-211) | 35 (1-65)* | |
| Caplacizumab as frontline (within 72 h), % (n/total) | 58.3 (35/60) | Not applicable | |
| Caplacizumab start after disease onset, median (range), d | 3 (0-27; 27) | Not applicable | |
| Treatment according to HERCULES protocol, % (n/total)† | 13.3 (8/60) | Not applicable | |
| Steroid treatment, % (n/total) | |||
| Any dose | 98.3 (59/60) | 97 (140/145) | .6734 |
| High dose‡ | 76.6 (46/60) | ||
| Non-high dose | 8.3 (5/60) | ||
| Data missing | 13.3 (8/60) | ||
| Steroid initial dose (n = 51), median (range), mg/kg BW§ | 1.40 (0.64-15.11) | Not reported | |
| Other treatments, % (n/total) | |||
| Mycophenolate | 5.9 (3/60) | 4 (6/145) | |
| Hydroxychloroquine | 1.7 (1/60) | 2 (3/145) | |
| Vincristine | 1.7 (1/60) | Not reported | |
| Ciclosporin | 3.3 (2/60) | 1 (2/145) | |
| Azathioprine | 5.0 (3/60) | Not reported | |
| Splenectomy | 0.0 (0/60) | 2 (3/145) | |
| IVIG | 0.0 (0/60) | 3 (4/145) | |
| Bortezomib | 0.0 (0/60) | 1 (2/145) | |
| Immunoadsorption | 0.0 (0/60) | 0.7 (1/145) |
| . | Germany (n = 60) . | HERCULES cohort (N = 145) . | Comparison of Germany vs HERCULES (Fisher’s exact test), P . |
|---|---|---|---|
| Rituximab treatment, % (n/total) | 80 (48/60) | 43 (63/145) | <.0001 |
| Rituximab, % (n/total) | |||
| Frontline (within 72 h) | 18.3 (11/60) | 17 (25/145) | .8398 |
| Delayed | 60.0 (36/60) | ||
| Data missing | 1.7 (1/60) | ||
| Day of first rituximab after disease onset, median (range), d | 7 (0-35) | Not reported | |
| Cumulative rituximab dose, median (range), mg | 2000 (562.5-5000) | Not reported | |
| PEX treatment, % (n/total) | 96.7 (58/60) | 100 (145/145) | .0846 |
| Days of PEX treatment (n = 58) | |||
| Median, no. (range) | 9 (2-41) | ||
| Mean, no. (95% CI) | 11.8 (9.5-14.2) | ||
| Days of PEX under caplacizumab treatment | |||
| Median, no. (range) | 4 (0-22) | Verum arm (n = 72), 5 (1-35) | |
| Mean, no. (95% CI) | 5.3 (4.2-6.4) | Verum arm (n = 72), 5.8 (4.8-6.8) | |
| Caplacizumab doses, median (range), no. | 34.0 (2-89) | ||
| Caplacizumab treatment duration, median (range), d | 34 (2-211) | 35 (1-65)* | |
| Caplacizumab as frontline (within 72 h), % (n/total) | 58.3 (35/60) | Not applicable | |
| Caplacizumab start after disease onset, median (range), d | 3 (0-27; 27) | Not applicable | |
| Treatment according to HERCULES protocol, % (n/total)† | 13.3 (8/60) | Not applicable | |
| Steroid treatment, % (n/total) | |||
| Any dose | 98.3 (59/60) | 97 (140/145) | .6734 |
| High dose‡ | 76.6 (46/60) | ||
| Non-high dose | 8.3 (5/60) | ||
| Data missing | 13.3 (8/60) | ||
| Steroid initial dose (n = 51), median (range), mg/kg BW§ | 1.40 (0.64-15.11) | Not reported | |
| Other treatments, % (n/total) | |||
| Mycophenolate | 5.9 (3/60) | 4 (6/145) | |
| Hydroxychloroquine | 1.7 (1/60) | 2 (3/145) | |
| Vincristine | 1.7 (1/60) | Not reported | |
| Ciclosporin | 3.3 (2/60) | 1 (2/145) | |
| Azathioprine | 5.0 (3/60) | Not reported | |
| Splenectomy | 0.0 (0/60) | 2 (3/145) | |
| IVIG | 0.0 (0/60) | 3 (4/145) | |
| Bortezomib | 0.0 (0/60) | 1 (2/145) | |
| Immunoadsorption | 0.0 (0/60) | 0.7 (1/145) |
N = 60 unless indicated otherwise.
BW, body weight; IVIG, IV immunoglobulin.
Data source is the Orphan Maintenance Assessment Report (EMA/541816/2018). Differences from the HERCULES cohort were tested using the Fisher’s exact test if sufficient data were available.
Defined as caplacizumab start before or after first PEX and caplacizumab continued for a minimum of 30 d after last PEX treatment.
High doses are defined as ≥1 mg/kg body weight of prednisone equivalent.
Milligram of prednisone or prednisone equivalence dose if another glucocorticoid has been used.