Baseline and follow-up features
| Features at diagnosis∗ . | Capla-treated (n = 75) . | Non-capla-treated (n = 38) . | P value . |
|---|---|---|---|
| Female, n/N (%) | 56/75 (74.6) | 28/38 (73.7) | .910 |
| Age, mean ± SD | 49.2 ± 15.8 | 43.3 ± 15.4 | .069 |
| First-ever iTTP episode, n/N (%) | 69/73 (94.5) | 32/35 (91.4) | .541 |
| Symptoms/signs, n/N (%) | |||
| Neurologic involvement | 52/75 (69.3) | 22/38 (57.9) | .227 |
| Laboratory parameters† | |||
| Platelets, ×109/L | 11.0 (8.0-18.9) | 12.0 (8.0-17.0) | .364 |
| Hemoglobin, g/dL | 8.0 (7.1-10.2) | 8.3 (7.4-10.0) | .679 |
| LDH, U/L | 3.0 (2.0-5.0) | 3.0 (1.0-5.0) | .795 |
| Creatinine, mg/dL | 0.98 (0.77-1.29) | 0.88 (0.74-1.27) | .320 |
| Creatinine above normal range‡, n/N (%) | 20/75 (26.7) | 10/38 (26.3) | .968 |
| Elevated troponin, n/N (%) | 29/56 (51.7) | 12/20 (60.0) | .527 |
| ADAMTS13, % | 0 (0-0.4) | 0 (0-0.4) | .859 |
| ADAMTS13 inhibitor positive, n/N (%) | 72/72 (100) | 35/35 (100) | 1.000 |
| Features at diagnosis∗ . | Capla-treated (n = 75) . | Non-capla-treated (n = 38) . | P value . |
|---|---|---|---|
| Female, n/N (%) | 56/75 (74.6) | 28/38 (73.7) | .910 |
| Age, mean ± SD | 49.2 ± 15.8 | 43.3 ± 15.4 | .069 |
| First-ever iTTP episode, n/N (%) | 69/73 (94.5) | 32/35 (91.4) | .541 |
| Symptoms/signs, n/N (%) | |||
| Neurologic involvement | 52/75 (69.3) | 22/38 (57.9) | .227 |
| Laboratory parameters† | |||
| Platelets, ×109/L | 11.0 (8.0-18.9) | 12.0 (8.0-17.0) | .364 |
| Hemoglobin, g/dL | 8.0 (7.1-10.2) | 8.3 (7.4-10.0) | .679 |
| LDH, U/L | 3.0 (2.0-5.0) | 3.0 (1.0-5.0) | .795 |
| Creatinine, mg/dL | 0.98 (0.77-1.29) | 0.88 (0.74-1.27) | .320 |
| Creatinine above normal range‡, n/N (%) | 20/75 (26.7) | 10/38 (26.3) | .968 |
| Elevated troponin, n/N (%) | 29/56 (51.7) | 12/20 (60.0) | .527 |
| ADAMTS13, % | 0 (0-0.4) | 0 (0-0.4) | .859 |
| ADAMTS13 inhibitor positive, n/N (%) | 72/72 (100) | 35/35 (100) | 1.000 |
| Features at follow-up†,§ . | Capla-treated (n = 75) . | Non-capla-treated (n = 38) . | P value . |
|---|---|---|---|
| Days of hospitalization | 16 (8-26) | 16 (12-27) | .265 |
| Days of ICU | 1 (0-3) | 0 (0-4) | .273 |
| Days of follow-up|| | 1041 (697-1308) | 881 (346-1080) | <.001 |
| Treatments | |||
| PEX + CS, n/N (%) | 75/75 (100) | 38/38 (100) | n.a. |
| Time on PEX (d) | |||
| If capla given ≤3 d from PEX + CS start | 5 (4-9) | 11 (6-26) | <.001 |
| If capla given >3 d from PEX + CS start | 15 (11-21) | 11 (6-26) | .429 |
| Time on capla (d) | |||
| If capla given ≤3 d from PEX + CS start | 57 (41-80) | 50 (25-83) | .404 |
| If capla given >3 d from PEX + CS start | 57 (48-91) | 50 (25-83) | .155 |
| Rituximab, n/N (%) | 62/75 (82.7) | 27/38 (71.0) | .223 |
| If capla given ≤3 d from PEX + CS start | 29/37 (77.8) | 27/38 (71.0) | .597 |
| If capla given >3 d from PEX + CS start | 33/38 (86.8) | 27/38 (71.0) | .158 |
| Capla | |||
| Time from iTTP diagnosis to start (d) | 4 (1-9) | n.a. | n.a. |
| Capla started ≤3 d from PEX + CS start, n/N (%) | 37/75 (49.3) | n.a. | n.a. |
| Time on capla (d) | 34 (27-39) | n.a. | n.a. |
| AEs during treatment | |||
| Hemorrhagic diathesis | |||
| Mucocutaneous bleeding, n/N (%) | 5/74 (6.8) | n.a. | n.a. |
| Metrorrhagia, n/N (%) | 3/55 (5.4) | n.a. | n.a. |
| Other, n/N (%) | 3/74 (4.0) | n.a. | n.a. |
| Thrombocytosis, n/N (%) | 5/74 (6.8) | n.a. | n.a. |
| Erythema, n/N (%) | 4/74 (5.4) | n.a. | n.a. |
| Liver toxicity grade 2, n/N (%) | 1/74 (1.3) | n.a. | n.a. |
| Time to normalization of platelet count (d)† | |||
| If capla given ≤3 d from PEX + CS start | 6 (4-11) | 12 (7-19) | .007 |
| If capla given >3 d from PEX + CS start | 14 (10-22) | 12 (7-19) | .244 |
| Refractoriness to first treatment, n/N (%) | |||
| If capla given ≤3 d from PEX + CS start | 1/36¶ (2.8) | 4/35# (11.4) | .077 |
| If capla given >3 d from PEX + CS start | 14/38∗∗ (2.6) | 4/35# (11.4) | .012 |
| Clinical response, n/N (%) | 74/75 (98.7) | 35/38 (92.1) | .110 |
| Clinical remission, n/N (%) | 74/75 (98.7) | 35/38 (92.1) | .110 |
| Exacerbations, n/N (%) | |||
| If capla given ≤3 d from PEX + CS start | 3/36¶ (8.3) | 9/36†† (25.0) | .029 |
| If capla given >3 d from PEX + CS start | 4/38‡‡ (10.5) | 9/36†† (25.0) | .051 |
| Relapses, n/N (%) | 3/74¶ (4.2) | 6/36†† (16.7) | .012 |
| Exitus, n/N (%) | 2/75 (2.7) | 3/38 (7.9) | .202 |
| Cause | Unknown (1) | iTTP evolution (3) | n.a. |
| iTTP evolution (1) |
| Features at follow-up†,§ . | Capla-treated (n = 75) . | Non-capla-treated (n = 38) . | P value . |
|---|---|---|---|
| Days of hospitalization | 16 (8-26) | 16 (12-27) | .265 |
| Days of ICU | 1 (0-3) | 0 (0-4) | .273 |
| Days of follow-up|| | 1041 (697-1308) | 881 (346-1080) | <.001 |
| Treatments | |||
| PEX + CS, n/N (%) | 75/75 (100) | 38/38 (100) | n.a. |
| Time on PEX (d) | |||
| If capla given ≤3 d from PEX + CS start | 5 (4-9) | 11 (6-26) | <.001 |
| If capla given >3 d from PEX + CS start | 15 (11-21) | 11 (6-26) | .429 |
| Time on capla (d) | |||
| If capla given ≤3 d from PEX + CS start | 57 (41-80) | 50 (25-83) | .404 |
| If capla given >3 d from PEX + CS start | 57 (48-91) | 50 (25-83) | .155 |
| Rituximab, n/N (%) | 62/75 (82.7) | 27/38 (71.0) | .223 |
| If capla given ≤3 d from PEX + CS start | 29/37 (77.8) | 27/38 (71.0) | .597 |
| If capla given >3 d from PEX + CS start | 33/38 (86.8) | 27/38 (71.0) | .158 |
| Capla | |||
| Time from iTTP diagnosis to start (d) | 4 (1-9) | n.a. | n.a. |
| Capla started ≤3 d from PEX + CS start, n/N (%) | 37/75 (49.3) | n.a. | n.a. |
| Time on capla (d) | 34 (27-39) | n.a. | n.a. |
| AEs during treatment | |||
| Hemorrhagic diathesis | |||
| Mucocutaneous bleeding, n/N (%) | 5/74 (6.8) | n.a. | n.a. |
| Metrorrhagia, n/N (%) | 3/55 (5.4) | n.a. | n.a. |
| Other, n/N (%) | 3/74 (4.0) | n.a. | n.a. |
| Thrombocytosis, n/N (%) | 5/74 (6.8) | n.a. | n.a. |
| Erythema, n/N (%) | 4/74 (5.4) | n.a. | n.a. |
| Liver toxicity grade 2, n/N (%) | 1/74 (1.3) | n.a. | n.a. |
| Time to normalization of platelet count (d)† | |||
| If capla given ≤3 d from PEX + CS start | 6 (4-11) | 12 (7-19) | .007 |
| If capla given >3 d from PEX + CS start | 14 (10-22) | 12 (7-19) | .244 |
| Refractoriness to first treatment, n/N (%) | |||
| If capla given ≤3 d from PEX + CS start | 1/36¶ (2.8) | 4/35# (11.4) | .077 |
| If capla given >3 d from PEX + CS start | 14/38∗∗ (2.6) | 4/35# (11.4) | .012 |
| Clinical response, n/N (%) | 74/75 (98.7) | 35/38 (92.1) | .110 |
| Clinical remission, n/N (%) | 74/75 (98.7) | 35/38 (92.1) | .110 |
| Exacerbations, n/N (%) | |||
| If capla given ≤3 d from PEX + CS start | 3/36¶ (8.3) | 9/36†† (25.0) | .029 |
| If capla given >3 d from PEX + CS start | 4/38‡‡ (10.5) | 9/36†† (25.0) | .051 |
| Relapses, n/N (%) | 3/74¶ (4.2) | 6/36†† (16.7) | .012 |
| Exitus, n/N (%) | 2/75 (2.7) | 3/38 (7.9) | .202 |
| Cause | Unknown (1) | iTTP evolution (3) | n.a. |
| iTTP evolution (1) |
Capla, caplacizumab; CS, corticosteroids; d, days; ICU, intensive care unit; IQR, interquartile range; LDH, lactate dehydrogenase; n.a., not applicable; n/N, number of patients positive/number of patients with information regarding the variable; SD, standard deviation.
These are the baseline features of those iTTP episodes reported during the study period whose information regarding the time required to recover ADAMTS13 levels >20% was available.
Median (IQR) except otherwise indicated.
1.1 mg/dL for women and 1.3 mg/dL for men.
This is the information corresponding to the follow-up of all episodes reported during the study, either first episodes or relapses, whose evolution of ADAMTS13 levels was available. Three patients who died shortly after diagnosis, 1 in the capla group and 2 in the noncapla group, whose ADAMTS13 levels beyond baseline were not reported, have also been included, although they were only considered for calculations regarding choice of treatment and rates of response, exacerbation, remission and exitus.
The median (IQR) follow-up period of the whole cohort was 914 (418-1224) days.
Data of 1 episode missing.
Data of 3 episodes missing.
Capla was added to PEX + CS due to refractoriness to the initial treatment in 13 cases.
Data of 2 episodes missing.
These data correspond to the period of time once capla treatment had already started. It must be mentioned that there were 11 episodes whose exacerbations prompted the initiation of capla.