Table 1 Baseline Patient Demographics and Disease Characteristics
Cohort A (N = 32) | Cohort B (N = 6) | Total (N = 38) | |
|---|---|---|---|
Demographics | |||
Age (years) | |||
Mean (SD) | 45.9 (24.50) | 23.2 (13.72) | 42.3 (24.48) |
Q1; Q3 | 22.5; 66.5 | 14.0; 37.0 | 19.0; 65.0 |
Range | 3; 78 | 9; 43 | 3; 78 |
Age, n (%) | |||
0–18 years | 6 (18.75) | 3 (50.00) | 9 (23.68) |
19–35 years | 7 (21.88) | 1 (16.67) | 8 (21.05) |
36–55 years | 3 (9.38) | 2 (33.33) | 5 (13.16) |
56–70 years | 11 (34.38) | 0 | 11 (28.95) |
over 70 years | 5 (15.63) | 0 | 5 (13.16) |
Sex, n (%) | |||
Female | 16 (50.00) | 3 (50.00) | 19 (50.00) |
Male | 16 (50.00) | 3 (50.00) | 19 (50.00) |
Disease Characteristics | |||
Diagnosis, n (%) | |||
MPS I disease | |||
Hurler – Scheie | 0 | 3 (50.00) | 3 (7.89) |
Scheie for MPS I | 0 | 3 (50.00) | 3 (7.89) |
Pompe disease | |||
Infantile-onset Pompe Disease | 5 (15.63) | 0 | 5 (13.16) |
Late-onset Pompe Disease | 27 (84.38) | 0 | 27 (71.05) |
Cognitive delay, n (%) | |||
No | 32 (100.00) | 4 (66.67) | 36 (94.74) |
Yes | 0 | 2 (33.33) | 2 (5.26) |
Age at diagnosis (years) | |||
Mean (SD) | 31.3 (23.53) | 10.0 (9.06) | 28.0 (23.18) |
Q1; Q3 | 4.5; 52.0 | 5.0; 10.0 | 5.0; 50.0 |
Range | 0; 64 | 4; 28 | 0; 64 |
Age at start of ERT administration in hospital setting (years) | |||
Mean (SD) | 35.4 (25.16) | 12.7 (9.79) | 31.8 (24.77) |
Q1; Q3 | 9.5; 58.5 | 6.0; 20.0 | 7.0; 54.0 |
Range | 0; 66 | 4; 29 | 0; 66 |
Time from diagnosis to start of ERT administration in hospital setting (months) | |||
Mean (SD) | 50.5 (61.44) | 31.0 (60.29) | 47.4 (60.88) |
Q1; Q3 | 3.5; 100.5 | 5.0; 10.0 | 4.0; 99.0 |
Range | 0; 211 | 4; 154 | 0; 211 |
Patients Starting The ERT Administration In A Homecare Setting, N (%) * | 30 (93.75) | 6 (100.00) | 36 (94.74) |
Reasons for switching to homecare setting, n (%) * | |||
COVID-19 related | 19 (63.33) | 1 (16.67) | 20 (55.56) |
Patient request | 8 (26.67) | 3 (50.00) | 11 (30.56) |
Center distant | 3 (10.00) | 0 | 3 (8.33) |
Other | 0 | 2 (33.33) | 2 (5.56) |
Age at start of ERT administration in a homecare setting (years)* | |||
Mean (SD) | 47.9 (22.87) | 17.7 (15.64) | 42.8 (24.46) |
Q1; Q3 | 26.0; 67.0 | 7.0; 34.0 | 19.5; 64.5 |
Range | 8; 78 | 5; 41 | 5; 78 |
Time from diagnosis to start of ERT administration in homecare setting (months)* | |||
Mean (SD) | 173.2 (85.79) | 93.7 (126.51) | 159.9 (96.37) |
Q1; Q3 | 102.0; 248.0 | 15.0; 157.0 | 83.0; 238.5 |
Range | 12; 315 | 15; 326 | 12; 326 |
Time to switch to homecare setting (months)* | |||
Mean (SD) | 119.2 (52.76) | 62.5 (75.78) | 109.8 (59.89) |
Q1; Q3 | 82.0; 168.0 | 11.0; 147.0 | 58.5; 163.5 |
Range | 10; 200 | 8; 172 | 8; 200 |
Time from start of ERT administration in homecare setting to screening (months)* | |||
Mean (SD) | 14.6 (7.51) | 69.5 (40.41) | 23.8 (26.66) |
Q1; Q3 | 9.0; 19.0 | 27.0; 106.0 | 10.0; 23.0 |
Range | 0; 26 | 26; 116 | 0; 116 |
Patients who started the ERT administration in homecare setting prior to enrollment, n (%)* | |||
No | 3 (9.38) | 0 | 3 (7.89) |
Yes | 29 (90.63) | 6 (100.00) | 35 (92.11) |
Other Medical Conditions | |||
Patients with prior medical conditions, n (%) | 16 (50.00) | 6 (100.00) | 22 (57.89) |
Patients with ongoing medical conditions, n (%) | 26 (81.25) | 5 (83.33) | 31 (81.58) |
Patients reporting the presence of any significant respiratory disease, n (%) | 18 (56.25) | 1 (16.67) | 19 (50.00) |
Patients with evidence of serious obstructive airway disease, n (%) $ | |||
Other | 10 (55.56) | 1 (100.00) | 11 (57.89) |
Respiratory Failure | 8 (44.44) | 0 | 8 (42.11) |
Predicted forced vital capacity, (%) $ | |||
n | 18 | 1 | 19 |
Mean (SD) | 50.4 (20.07) | 38.0 | 49.8 (19.71) |
Q1; Q3 | 36.0; 68.0 | 38.0; 38.0 | 36.0; 68.0 |
Range | 10; 82 | 38; 38 | 10; 82 |