Table 1.

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.

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