Comparison of key characteristics of the current cohort vs those of 5 recently published cohorts in the literature (data were extracted from the articles shown)
| Cohort characteristics . | Current study . | EACH27,13 . | German15,18 . | United Kingdom2,22 . | Spanish AHASR14 . | Chinese CARE19 . | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| No. of patients | 32 | 501 | 102 | 172 | 151 | 187 | ||||||||
| Age, median, y | 77 | 73.9 | 74 | 78 | 74 | 52 | ||||||||
| Age >80 y, % | 28.1 | 19.1 (non-peripartum) | NA | NA | 28.6 | 5.5 (non-peripartum) | ||||||||
| Age >85 y, % | 12.5 | 7.9 (non-peripartum) | NA | 22.5 (non-peripartum) | NA | 1.8 (non-peripartum) | ||||||||
| Percent female | 56.3 | 50.7 | 51.8 | 43 | 57.4 | 43.7 | 54.5 | |||||||
| Initial FVIII, median, IU/dL | 1.0 | 2 | 1.4 | 5 | 2 | 1.7 | 1.7 | |||||||
| Initial inhibitor, median, BU | 17 | 12.8 | 19 | 8 | 18 | 13 | 13 | |||||||
| High inhibitor (>20 BU) | 14/32 (43.8%) | NA | 40/102 (39.2%) | NA | NA | NA | ||||||||
| Very high inhibitor (>100 BU) | 10/32 (31.3%) | 62/501 (12.4%) | 14/102 (13.7%) | 16/172 (9.3%) | 15/145 (10.3%) | 18/187 (9.6%) | ||||||||
| Extreme inhibitor (>1000 BU) | 1/32 (3.1%) | 0/501 (0%) | 1/102 (1.0%) | 0/172 (0%) | 4/145 (2.8%) | 1/187 (0.5%) | ||||||||
| Idiopathic (%) | 59.4 | 61 | 68 | 67 | 63.3 | 44.1 | 54.4 | |||||||
| Autoimmune (%) | 25 | 16 | 17 | 20 | 16.7 | 31.7 | 12.5 | |||||||
| Malignancy (%) | 21.9 | 11 | 10 | 13 | 14.7 | 10.3 | 6 | |||||||
| Pregnancy (%) | 0 | 12 | 2 | 5 | 2 | 6.2 | 12.5 | |||||||
| Median follow-up, d | 779.5 | 262 | 262 | NA (survey for patients in 2 y) | 360 | 205 | ||||||||
| Treatment | CyDRi | P | P + by mouth Cy | P ± by mouth Cy ± R | Sequential P/Cy | Simultaneous P + Cy | P | P + by mouth Cy | P + CNI | R ± | P | P + Cy | R + | Other |
| Alive in CR∗ at last F/U | 29/32 (90.6%) | 90/142 (63.4%) | 43/83 (62.3%) | 49/102 (48%) | 78/172 (45.3%) | 100/151 (66.2%) | 115/155 (74.2%) | |||||||
| TTCR, median, d | 77 | 108 | 74 | 79 | 49 | 39 | 30 | 53 | 45 | 42 | 69 | 62 | 47 | 74 |
| Alive, no CR,∗ last F/U | 0/32 (0%) | 5/142 (3.5%) | 1/83 (1.2%) | 19/102 (18.6%) | 8/172 (4.7%) | 15/151 (9.9%) | 29/155 (18.7%) | |||||||
| Lost to F/U, not accounted for | 0 | 7/142 (4.9%) | 14/83 (16.9%) | 0 | 16/172 (9.3%) | 0 | 22 (11.7%) | |||||||
| Relapse rate (% from CR∗) | 2/31 (6.5%) | 15/83 (18.1%) | 8/66 (12.1%) | 15/62 (24.2%) | 18/90 (20%) | 8/112 (7.1%) | 13/155 (8.4%) | |||||||
| All-cause mortality last F/U | 3/32 (9.4%) | 40/142 (28.2%) | 25/83 (30.1%) | 34/102 (33.3%) | 55/127 (43.3%) | 18/48 (37.5%) | 36/151 (23.8%) | 11/165 (6.7%) | ||||||
| Bleeding-related mortality | 0/32 (0%) | 1/142 (0.7%) | 0/83 (0%) | 3/102 (2.9%) | 13/175 (7.4%) | 5/151 (3.3%) | 6/165 (0.6%) | |||||||
| Treatment-related mortality | 1/32 (3.1%) | 5/142 (3.5%) | 4/83 (4.8%) | 16/102 (15.7%) | 12/175 (6.9%) | 15/151 (9.9%) | 2/165 (1.2%) | |||||||
| Mortality related to underlying disease | 1/32 (3.1%) | 6/142 (4.2%) | 4/83 (4.8%) | 3/102 (2.9%) | NA | 16/151 (10.6%) | 2/165 (1.2%) | |||||||
| Toxicity (AE) | 5/32 (15.6%) | 36/142 (25%) | 34/83 (41%) | 67/102 (66%) | 57/112 (51%) | NA | 11/155 (7.1%) | |||||||
| Infection | 5/32 (15.6%) | 23/142 (16%) | 22/83 (27%) | 37/102 (36%) | 37/112 (33%) | NA | 4/155 (2.6%) | |||||||
| Symptomatic neutropenia | 0 | 2/142 (1%) | 12/83 (14%) | 1/102 (1%) | 13/112 (12%) | NA | 1/155 (0.6%) | |||||||
| Diabetes mellitus | 0 | 11/142 (8%) | 5/83 (6%) | 12/102 (12%) | 9/112 (8%) | NA | NA | |||||||
| Psychiatric | 0 | 6/142 (4%) | 3/83 (4%) | 3/102 (3%) | 2/112 (2%) | NA | NA | |||||||
| Thromboembolic/cardiovascular | 0 | 10/501 (2%) | 8/102 (7.8%) | 0 | NA | 3/155 (1.9%) | ||||||||
| Those with no AE | 27/32 (84.4%) | 106/142 (75%) | 49/83 (59%) | 35/102 (34%) | 55/112 (49%) | NA | 144/155 (92.9%) | |||||||
| Cohort characteristics . | Current study . | EACH27,13 . | German15,18 . | United Kingdom2,22 . | Spanish AHASR14 . | Chinese CARE19 . | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| No. of patients | 32 | 501 | 102 | 172 | 151 | 187 | ||||||||
| Age, median, y | 77 | 73.9 | 74 | 78 | 74 | 52 | ||||||||
| Age >80 y, % | 28.1 | 19.1 (non-peripartum) | NA | NA | 28.6 | 5.5 (non-peripartum) | ||||||||
| Age >85 y, % | 12.5 | 7.9 (non-peripartum) | NA | 22.5 (non-peripartum) | NA | 1.8 (non-peripartum) | ||||||||
| Percent female | 56.3 | 50.7 | 51.8 | 43 | 57.4 | 43.7 | 54.5 | |||||||
| Initial FVIII, median, IU/dL | 1.0 | 2 | 1.4 | 5 | 2 | 1.7 | 1.7 | |||||||
| Initial inhibitor, median, BU | 17 | 12.8 | 19 | 8 | 18 | 13 | 13 | |||||||
| High inhibitor (>20 BU) | 14/32 (43.8%) | NA | 40/102 (39.2%) | NA | NA | NA | ||||||||
| Very high inhibitor (>100 BU) | 10/32 (31.3%) | 62/501 (12.4%) | 14/102 (13.7%) | 16/172 (9.3%) | 15/145 (10.3%) | 18/187 (9.6%) | ||||||||
| Extreme inhibitor (>1000 BU) | 1/32 (3.1%) | 0/501 (0%) | 1/102 (1.0%) | 0/172 (0%) | 4/145 (2.8%) | 1/187 (0.5%) | ||||||||
| Idiopathic (%) | 59.4 | 61 | 68 | 67 | 63.3 | 44.1 | 54.4 | |||||||
| Autoimmune (%) | 25 | 16 | 17 | 20 | 16.7 | 31.7 | 12.5 | |||||||
| Malignancy (%) | 21.9 | 11 | 10 | 13 | 14.7 | 10.3 | 6 | |||||||
| Pregnancy (%) | 0 | 12 | 2 | 5 | 2 | 6.2 | 12.5 | |||||||
| Median follow-up, d | 779.5 | 262 | 262 | NA (survey for patients in 2 y) | 360 | 205 | ||||||||
| Treatment | CyDRi | P | P + by mouth Cy | P ± by mouth Cy ± R | Sequential P/Cy | Simultaneous P + Cy | P | P + by mouth Cy | P + CNI | R ± | P | P + Cy | R + | Other |
| Alive in CR∗ at last F/U | 29/32 (90.6%) | 90/142 (63.4%) | 43/83 (62.3%) | 49/102 (48%) | 78/172 (45.3%) | 100/151 (66.2%) | 115/155 (74.2%) | |||||||
| TTCR, median, d | 77 | 108 | 74 | 79 | 49 | 39 | 30 | 53 | 45 | 42 | 69 | 62 | 47 | 74 |
| Alive, no CR,∗ last F/U | 0/32 (0%) | 5/142 (3.5%) | 1/83 (1.2%) | 19/102 (18.6%) | 8/172 (4.7%) | 15/151 (9.9%) | 29/155 (18.7%) | |||||||
| Lost to F/U, not accounted for | 0 | 7/142 (4.9%) | 14/83 (16.9%) | 0 | 16/172 (9.3%) | 0 | 22 (11.7%) | |||||||
| Relapse rate (% from CR∗) | 2/31 (6.5%) | 15/83 (18.1%) | 8/66 (12.1%) | 15/62 (24.2%) | 18/90 (20%) | 8/112 (7.1%) | 13/155 (8.4%) | |||||||
| All-cause mortality last F/U | 3/32 (9.4%) | 40/142 (28.2%) | 25/83 (30.1%) | 34/102 (33.3%) | 55/127 (43.3%) | 18/48 (37.5%) | 36/151 (23.8%) | 11/165 (6.7%) | ||||||
| Bleeding-related mortality | 0/32 (0%) | 1/142 (0.7%) | 0/83 (0%) | 3/102 (2.9%) | 13/175 (7.4%) | 5/151 (3.3%) | 6/165 (0.6%) | |||||||
| Treatment-related mortality | 1/32 (3.1%) | 5/142 (3.5%) | 4/83 (4.8%) | 16/102 (15.7%) | 12/175 (6.9%) | 15/151 (9.9%) | 2/165 (1.2%) | |||||||
| Mortality related to underlying disease | 1/32 (3.1%) | 6/142 (4.2%) | 4/83 (4.8%) | 3/102 (2.9%) | NA | 16/151 (10.6%) | 2/165 (1.2%) | |||||||
| Toxicity (AE) | 5/32 (15.6%) | 36/142 (25%) | 34/83 (41%) | 67/102 (66%) | 57/112 (51%) | NA | 11/155 (7.1%) | |||||||
| Infection | 5/32 (15.6%) | 23/142 (16%) | 22/83 (27%) | 37/102 (36%) | 37/112 (33%) | NA | 4/155 (2.6%) | |||||||
| Symptomatic neutropenia | 0 | 2/142 (1%) | 12/83 (14%) | 1/102 (1%) | 13/112 (12%) | NA | 1/155 (0.6%) | |||||||
| Diabetes mellitus | 0 | 11/142 (8%) | 5/83 (6%) | 12/102 (12%) | 9/112 (8%) | NA | NA | |||||||
| Psychiatric | 0 | 6/142 (4%) | 3/83 (4%) | 3/102 (3%) | 2/112 (2%) | NA | NA | |||||||
| Thromboembolic/cardiovascular | 0 | 10/501 (2%) | 8/102 (7.8%) | 0 | NA | 3/155 (1.9%) | ||||||||
| Those with no AE | 27/32 (84.4%) | 106/142 (75%) | 49/83 (59%) | 35/102 (34%) | 55/112 (49%) | NA | 144/155 (92.9%) | |||||||
AE, adverse event; CNI, calcineurin inhibitors; Cy, cyclophosphamide; F/U, follow-up; NA, not available; P, prednisone; R, rituximab.
CR considered as no bleed, no inhibitor, FVIII >50 IU/dL, and immunosuppression stopped.