Table 1. Baseline Characteristics of Patients.
| Characteristic | Enrolled patients | Patients included in final analysis | ||||
|---|---|---|---|---|---|---|
| Total enrolled patients (N = 32) | Patients (duration >6 mo, n = 28)a | Patients included in final analysis (n = 26) | Splenectomized patients (n = 5) | Patients by age when starting ambroxolb | ||
| <18 y (n = 15) | ≥18 y (n = 6) | |||||
| Sex, No. (%) | ||||||
| Male | 15 (46.9) | 15 (53.6) | 14 (53.8) | 3 (60.0) | 8 (53.3) | 2 (33.3) |
| Female | 17 (53.1) | 13 (46.4) | 12 (46.2) | 2 (40.0) | 7 (46.7) | 4 (66.7) |
| Age at start of ambroxol, mean (SD), y | 17.2 (16.5) | 16.9 (15.3) | 18.1 (16.6) | 37.2 (11.9) | 6.3 (2.7) | 31.3 (8.3) |
| Spleen status, No. (%) | ||||||
| Intact | 27 (84.4) | 23 (82.1) | 21 (80.8) | 0 | 15 (100) | 6 (100) |
| Underwent splenectomy | 5 (15.6) | 5 (17.9) | 5 (19.2) | 5 (100) | 0 | 0 |
| Children (<18 y), No./total No. (%) | 20/32 (62.5) | 17/28 (60.7) | 15/26 (57.7) | 0 | 15/15 (100) | 0 |
| Hemoglobin, mean (SD), g/dL | 10.6 (1.8) | 10.7 (1.8) | 10.4 (1.7) | 12.7 (1.3) | 10.3 (1.5) | 10.6 (2.4) |
| Platelet count, mean (SD), ×103/µL | 99 (89) | 102 (92) | 104 (96) | 243 (156) | 75 (24) | 56 (24) |
| White blood count, mean (SD), /μL | 5870 (3170) | 6190 (3250) | 6130 (3340) | 11 600 (2400) | 5810 (1680) | 2840 (1050) |
| Chitotriosidase, median (range), nmol/mL/hc | 13 634 (3849-29 628)d | 13 634 (3849-29 628)e | 14 598 (3849-29 628)f | 22 568 (15 507-29 628)g | 15 710 (4092-28 422)h | 10 276 (3849-17 607)i |
| Glucosylsphingosine, median (range), ng/mLj | 282.8 (73.6-944.2) | 265.1 (73.6-944.2) | 251.3 (73.6-944.2) | 335.5 (73.6-944.2) | 248.5 (122.8-674.9) | 271.3 (179.8-549.7) |
SI conversion factors: To convert hemoglobin to grams per liter, multiply by 10.0; platelet count to ×109 per liter, multiply by 1.0; and white blood cell count to ×109 per liter, multiply by 0.001.
Excludes 4 patients lost to follow-up.
Excludes 5 splenectomized patients.
Normal range, 3 to 47 nmol/mL/h.
Twelve patients with no chitotriosidase activity were excluded.
Eleven patients with no chitotriosidase activity were excluded.
Ten patients with no chitotriosidase activity were excluded.
Two patients with no chitotriosidase activity were excluded.
Five patients with no chitotriosidase activity were excluded.
Two patients with no chitotriosidase activity were excluded.
Normal ranges, less than 1.1 ng/mL in plasma sample, less than 10 ng/mL in dried blood spot sample.