Characteristics of patients at baseline (safety population)
| Characteristic . | Rilzabrutinib (n = 133) . | Placebo (n = 69) . | All patients (N = 202) . |
|---|---|---|---|
| Age, median (range), y | 47 (18-80) | 46 (19-79) | 47 (18-80) |
| Sex, n (%) | |||
| Male | 55 (41) | 20 (29) | 75 (37) |
| Female | 78 (59) | 49 (71) | 127 (63) |
| Duration of ITP, median (range), y | 8.1 (0.3-52.2) | 6.2 (0.3-35.8) | 7.7 (0.3-52.2) |
| Baseline platelet count, median (range),∗ ×109/L | 15 (1-32) | 15 (1-54) | 15 (1-54) |
| Baseline platelet count by category, n (%) | |||
| <15 × 109/L | 65 (49) | 32 (46) | 97 (48) |
| ≥15 × 109/L | 68 (51) | 37 (54) | 105 (52) |
| Prior unique therapies,† n (%) | |||
| 1-2 | 43 (32) | 15 (22) | 58 (29) |
| 3-4 | 33 (25) | 18 (26) | 51 (25) |
| ≥5 | 57 (43) | 36 (52) | 93 (46) |
| No. of unique prior ITP therapies,† median (range) | 4 (1-15) | 5 (1-12) | 4 (1-15) |
| Prior splenectomy,† n (%) | 37 (28) | 19 (28) | 56 (28) |
| Most common prior ITP therapies,† n (%) | |||
| CS | 127 (95) | 66 (96) | 193 (96) |
| TPO-RA | 88 (66) | 51 (74) | 139 (69) |
| IVIg or anti-D immunoglobulin | 72 (54) | 40 (58) | 118 (58) |
| Immunosuppressants | 67 (50) | 42 (61) | 109 (54) |
| Rituximab | 42 (32) | 29 (42) | 71 (35) |
| Fostamatinib | 17 (13) | 7 (10) | 24 (12) |
| Prior response to most common prior ITP therapies,† response (n)/received (n), (% response) | |||
| CS | 93/127 (73) | 47/66 (71) | 140/193 (73) |
| TPO-RA | 55 /88 (63) | 32/51 (63) | 87/139 (63) |
| IVIg or anti-D immunoglobulin | 55/72 (76) | 31/40 (78) | 86 /118 (73) |
| Immunosuppressants | NR/67 (NR) | NR/42 (NR) | NR/109 (NR) |
| Rituximab | 11/42 (26) | 12/29 (41) | 23/71 (32) |
| Fostamatinib | 4/17 (24) | 3/7 (43) | 7/24 (29) |
| Concomitant ITP therapy, n (%) | |||
| None | 53 (40) | 23 (33) | 76 (38) |
| CS | 34 (26) | 20 (29) | 54 (27) |
| TPO-RA | 25 (19) | 12 (17) | 37 (18) |
| CS + TPO-RA | 21 (16) | 14 (20) | 35 (17) |
| Geographic region, n (%) | |||
| Asia/Pacific | 44 (33) | 26 (38) | 70 (35) |
| Western Europe | 33 (25) | 16 (23) | 49 (24) |
| Eastern Europe | 19 (14) | 13 (4) | 32 (16) |
| South America | 24 (18) | 11 (16) | 35 (17) |
| North America | 13 (10) | 3 (4) | 16 (8) |
| Characteristic . | Rilzabrutinib (n = 133) . | Placebo (n = 69) . | All patients (N = 202) . |
|---|---|---|---|
| Age, median (range), y | 47 (18-80) | 46 (19-79) | 47 (18-80) |
| Sex, n (%) | |||
| Male | 55 (41) | 20 (29) | 75 (37) |
| Female | 78 (59) | 49 (71) | 127 (63) |
| Duration of ITP, median (range), y | 8.1 (0.3-52.2) | 6.2 (0.3-35.8) | 7.7 (0.3-52.2) |
| Baseline platelet count, median (range),∗ ×109/L | 15 (1-32) | 15 (1-54) | 15 (1-54) |
| Baseline platelet count by category, n (%) | |||
| <15 × 109/L | 65 (49) | 32 (46) | 97 (48) |
| ≥15 × 109/L | 68 (51) | 37 (54) | 105 (52) |
| Prior unique therapies,† n (%) | |||
| 1-2 | 43 (32) | 15 (22) | 58 (29) |
| 3-4 | 33 (25) | 18 (26) | 51 (25) |
| ≥5 | 57 (43) | 36 (52) | 93 (46) |
| No. of unique prior ITP therapies,† median (range) | 4 (1-15) | 5 (1-12) | 4 (1-15) |
| Prior splenectomy,† n (%) | 37 (28) | 19 (28) | 56 (28) |
| Most common prior ITP therapies,† n (%) | |||
| CS | 127 (95) | 66 (96) | 193 (96) |
| TPO-RA | 88 (66) | 51 (74) | 139 (69) |
| IVIg or anti-D immunoglobulin | 72 (54) | 40 (58) | 118 (58) |
| Immunosuppressants | 67 (50) | 42 (61) | 109 (54) |
| Rituximab | 42 (32) | 29 (42) | 71 (35) |
| Fostamatinib | 17 (13) | 7 (10) | 24 (12) |
| Prior response to most common prior ITP therapies,† response (n)/received (n), (% response) | |||
| CS | 93/127 (73) | 47/66 (71) | 140/193 (73) |
| TPO-RA | 55 /88 (63) | 32/51 (63) | 87/139 (63) |
| IVIg or anti-D immunoglobulin | 55/72 (76) | 31/40 (78) | 86 /118 (73) |
| Immunosuppressants | NR/67 (NR) | NR/42 (NR) | NR/109 (NR) |
| Rituximab | 11/42 (26) | 12/29 (41) | 23/71 (32) |
| Fostamatinib | 4/17 (24) | 3/7 (43) | 7/24 (29) |
| Concomitant ITP therapy, n (%) | |||
| None | 53 (40) | 23 (33) | 76 (38) |
| CS | 34 (26) | 20 (29) | 54 (27) |
| TPO-RA | 25 (19) | 12 (17) | 37 (18) |
| CS + TPO-RA | 21 (16) | 14 (20) | 35 (17) |
| Geographic region, n (%) | |||
| Asia/Pacific | 44 (33) | 26 (38) | 70 (35) |
| Western Europe | 33 (25) | 16 (23) | 49 (24) |
| Eastern Europe | 19 (14) | 13 (4) | 32 (16) |
| South America | 24 (18) | 11 (16) | 35 (17) |
| North America | 13 (10) | 3 (4) | 16 (8) |
NR, not reported.
Defined as average of first and second qualifying screening platelet counts and study day 1 platelet count.
Unique ITP therapies were identified using standard medication term, different CS were counted as 1 therapy, and splenectomy could be counted as 1 prior ITP therapy. Patients could receive >1 therapy. Prior response was recorded on the electronic case report form as platelet count ≥50 × 109/L at any time. The Immunosuppressants category included immunosuppressant and other immunomodulatory agents (including cyclophosphamide).