Baseline demographics and clinical characteristics (safety analysis set)
| . | Leniolisib (n = 21) . | Placebo (n = 10) . | Total (N = 31) . |
|---|---|---|---|
| Age, y | |||
| Median (range) | 20.0 (12-54) | 19.5 (15-48) | 20.0 (12-54) |
| <18, No. (%) | 8 (38.1) | 4 (40.0) | 12 (38.7) |
| Male/female sex, % | 52.4/47.6 | 40.0/60.0 | 48.4/51.6 |
| Weight, median (range), kg | 67.1 (46.9-100.6) | 68.9 (50.0-88.0) | 67.1 (46.9-100.6) |
| PIK3CD/PIK3R1 variant, No. | 16/5 | 9/1 | 25/6 |
| Baseline glucocorticoids,∗ No. (%) | 12 (57.1) | 6 (60.0) | 18 (58.1) |
| Baseline IRT, No. (%) | 14 (66.7) | 7 (70.0) | 21 (67.7) |
| Baseline antibiotic prophylaxis, No. (%) | 9 (42.9) | 4 (40.0) | 13 (41.9) |
| Previous sirolimus treatment, No. (%) | 4 (19.0) | 3 (30.0) | 7 (22.6) |
| Lymphoproliferation,† No. (%) | 15 (71.4) | 7 (70.0) | 22 (71.0) |
| Chronic infections, No. (%) | 18 (85.7) | 7 (70.0) | 25 (80.6) |
| Pulmonary disease, No. (%) | 14 (66.7) | 8 (80.0) | 22 (71.0) |
| Bronchiectasis | 8 (38.1) | 8 (80.0) | 16 (51.6) |
| Asthma | 7 (33.3) | 4 (40.0) | 11 (35.5) |
| Cytopenias, No. (%) | 13 (61.9) | 5 (50.0) | 18 (58.1) |
| Gastrointestinal disease, No. (%) | 10 (47.6) | 7 (70.0) | 17 (54.8) |
| . | Leniolisib (n = 21) . | Placebo (n = 10) . | Total (N = 31) . |
|---|---|---|---|
| Age, y | |||
| Median (range) | 20.0 (12-54) | 19.5 (15-48) | 20.0 (12-54) |
| <18, No. (%) | 8 (38.1) | 4 (40.0) | 12 (38.7) |
| Male/female sex, % | 52.4/47.6 | 40.0/60.0 | 48.4/51.6 |
| Weight, median (range), kg | 67.1 (46.9-100.6) | 68.9 (50.0-88.0) | 67.1 (46.9-100.6) |
| PIK3CD/PIK3R1 variant, No. | 16/5 | 9/1 | 25/6 |
| Baseline glucocorticoids,∗ No. (%) | 12 (57.1) | 6 (60.0) | 18 (58.1) |
| Baseline IRT, No. (%) | 14 (66.7) | 7 (70.0) | 21 (67.7) |
| Baseline antibiotic prophylaxis, No. (%) | 9 (42.9) | 4 (40.0) | 13 (41.9) |
| Previous sirolimus treatment, No. (%) | 4 (19.0) | 3 (30.0) | 7 (22.6) |
| Lymphoproliferation,† No. (%) | 15 (71.4) | 7 (70.0) | 22 (71.0) |
| Chronic infections, No. (%) | 18 (85.7) | 7 (70.0) | 25 (80.6) |
| Pulmonary disease, No. (%) | 14 (66.7) | 8 (80.0) | 22 (71.0) |
| Bronchiectasis | 8 (38.1) | 8 (80.0) | 16 (51.6) |
| Asthma | 7 (33.3) | 4 (40.0) | 11 (35.5) |
| Cytopenias, No. (%) | 13 (61.9) | 5 (50.0) | 18 (58.1) |
| Gastrointestinal disease, No. (%) | 10 (47.6) | 7 (70.0) | 17 (54.8) |
Systemic glucocorticoids in a dose equivalent to ≤25 mg per day of prednisone within 2 weeks before first dosing of study medication were allowed.
Although all patients were required to have lymphadenopathy for trial inclusion, documented clinical history of lymphoproliferation (eg, lymphadenopathy, splenomegaly, hepatomegaly) varied.