Characteristics of the included studies and patients enrolled
| Study name (y) . | Setting . | Study design . | Enrollment period . | Drug assign-ment . | No. of partici-pants . | Age (y), mean (range) . | No. (%) female . | No. (%) non-White . | Cardiac troponin I (μg/L), median (range) . | No. (%) with neuro involvement on presentation‡ . | Median follow-up (mo) . | No. (%) presenting with recurrent TTP . | No. (%) with confirmed ADAMTS13 <10% . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| HERCULES (2019)9 | International (Australia, Europe, Canada, US): 92 sites worldwide | RCT | November 2015 - April 2017 | Caplacizumab + SOC | 72 | 45 (18-77) | 49 (68) | 22 (31) | 0.09 (0.01-75.96) | 6 (8) | 28 d after discontinuation of treatment | 24 (33) | 58 (81) |
| SOC | 73 | 47 (21-79) | 51 (70) | 14 (19) | 0.07 (0.01-7.28) | 5 (7) | 39 (53) | 65 (89) | |||||
| TITAN (2016)8 | International (Europe, US, Australia): 56 sites | RCT | October 2010 - January 2014 | Caplacizumab + SOC | 36 | 41 (19-72) | 24 (67) | 4 (11) | Not reported | Not reported | 1 (mortality); 12 (relapse) | 12 (33) | 28 (78) |
| SOC | 39 | 42 (21-67) | 20 (51) | 5 (13) | Not reported | Not reported | 12 (31) | 30 (77) | |||||
| France (2021)12 | France (multiple centers) | Observational study with historical control | September 2018 - December 2019 (intervention group); June 2015 - September 2018 (historical control) | Caplacizumab + SOC | 90 | 45 (34-57)† | 63 (70) | 16 (18) | Not reported; 51 (56%) with cardiac involvement | 55 (61) | 127 | 12 (13) | 90 (100) |
| SOC | 180 | 43 (30-57)† | 127 (70) | 31 (17) | Not reported; 86 (47%) with cardiac involvement | 111 (62) | 21 (12) | 180 (100) | |||||
| United Kingdom (2021)13 | United Kingdom (England, Scotland, and Wales): 22 sites | Observational study with historical control | May 2018 - January 2020 (intervention arm); 2014 - 2018 (historical control) | Caplacizumab + SOC | 85 | 45 (15-93) | 56 (66) | 28 (33) | Not reported | 56 (66) | 80 | Not reported | 84 (99) |
| SOC | 39 | 46 (3-82) | 31 (80) | Not reported | Not reported | 29 (74) | Not reported | Not reported | Not reported | ||||
| Barcelona∗ (2020)14 | Single center | Observational study with concurrent control | May 2014 - May 2020 | Caplacizumab + SOC | 9 | 43 (39-55)† | 8 (89) | Not reported | Not reported | 3 (33) | 6.8 | Not reported | Not reported |
| SOC | 9 | 41 (33-52)† | 6 (67) | Not reported | Not reported | 6 (66) | 51.8 | Not reported | Not reported |
| Study name (y) . | Setting . | Study design . | Enrollment period . | Drug assign-ment . | No. of partici-pants . | Age (y), mean (range) . | No. (%) female . | No. (%) non-White . | Cardiac troponin I (μg/L), median (range) . | No. (%) with neuro involvement on presentation‡ . | Median follow-up (mo) . | No. (%) presenting with recurrent TTP . | No. (%) with confirmed ADAMTS13 <10% . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| HERCULES (2019)9 | International (Australia, Europe, Canada, US): 92 sites worldwide | RCT | November 2015 - April 2017 | Caplacizumab + SOC | 72 | 45 (18-77) | 49 (68) | 22 (31) | 0.09 (0.01-75.96) | 6 (8) | 28 d after discontinuation of treatment | 24 (33) | 58 (81) |
| SOC | 73 | 47 (21-79) | 51 (70) | 14 (19) | 0.07 (0.01-7.28) | 5 (7) | 39 (53) | 65 (89) | |||||
| TITAN (2016)8 | International (Europe, US, Australia): 56 sites | RCT | October 2010 - January 2014 | Caplacizumab + SOC | 36 | 41 (19-72) | 24 (67) | 4 (11) | Not reported | Not reported | 1 (mortality); 12 (relapse) | 12 (33) | 28 (78) |
| SOC | 39 | 42 (21-67) | 20 (51) | 5 (13) | Not reported | Not reported | 12 (31) | 30 (77) | |||||
| France (2021)12 | France (multiple centers) | Observational study with historical control | September 2018 - December 2019 (intervention group); June 2015 - September 2018 (historical control) | Caplacizumab + SOC | 90 | 45 (34-57)† | 63 (70) | 16 (18) | Not reported; 51 (56%) with cardiac involvement | 55 (61) | 127 | 12 (13) | 90 (100) |
| SOC | 180 | 43 (30-57)† | 127 (70) | 31 (17) | Not reported; 86 (47%) with cardiac involvement | 111 (62) | 21 (12) | 180 (100) | |||||
| United Kingdom (2021)13 | United Kingdom (England, Scotland, and Wales): 22 sites | Observational study with historical control | May 2018 - January 2020 (intervention arm); 2014 - 2018 (historical control) | Caplacizumab + SOC | 85 | 45 (15-93) | 56 (66) | 28 (33) | Not reported | 56 (66) | 80 | Not reported | 84 (99) |
| SOC | 39 | 46 (3-82) | 31 (80) | Not reported | Not reported | 29 (74) | Not reported | Not reported | Not reported | ||||
| Barcelona∗ (2020)14 | Single center | Observational study with concurrent control | May 2014 - May 2020 | Caplacizumab + SOC | 9 | 43 (39-55)† | 8 (89) | Not reported | Not reported | 3 (33) | 6.8 | Not reported | Not reported |
| SOC | 9 | 41 (33-52)† | 6 (67) | Not reported | Not reported | 6 (66) | 51.8 | Not reported | Not reported |
We report the inclusion and exclusion criteria, additional differences in baseline characteristics, and differences in treatments received across studies in the supplemental Data 2.
We performed data extraction and quality appraisal based on abstract proceedings only. This study did not have corresponding full-text publications in peer-reviewed journals.
Median (interquartile range).
Defined in HERCULES as Glasgow Coma Scale ≤1; defined in the French study as headache, confusion, seizure, coma, or focal deficit; defined in the United Kingdom study as any neurologic symptom (including headaches, forgetfulness, limb weakness, paresthesia, agitation, encephalopathy, depressed mood, and anxiety); and defined in the Barcelona study as focal deficits, convulsion, headache, or dizziness.