Table 1.
Summary of Patient Characteristics in Population Pharmacokinetic Analysis
| Patient Characteristics | Total (N = 285) |
|---|---|
| Sex, n (%) | |
| Female | 162 (56.8) |
| Male | 123 (43.2) |
| Race, n (%) | |
| White | 181 (63.5) |
| Asian | 82 (28.8) |
| Other (including uncollected or unreported) | 22 (7.7) |
| Renal impairment category, n (%) | |
| No (eGFR ≥90 mL/min/1.73 m2) | 245 (86.0) |
| Mild impairment (eGFR 60‐89 mL/min/1.73 m2) | 37 (13.0) |
| Moderate impairment (eGFR 30‐59 mL/min/1.73 m2) | 3 (1.1) |
| Splenectomy, n (%) | |
| Yes | 170 (59.6) |
| No | 115 (40.4) |
| Genotype, n (%) | |
| β0/β0 | 67 (23.5) |
| Non‐β0/β0 | 153 (53.7) |
| Missing (almost all from phase 2) | 65 (22.8) |
| Concurrent use of ICT, n (%) | |
| Yes | 242 (84.9) |
| No | 21 (7.4) |
| Missing | 22 (7.7) |
| Age (years), median (min‐max) | 32.0 (18.0‐66.0) |
| Weight (kg), median (min‐max) | 57.1 (34.1‐97.0) |
| Erythropoietin (U/L), median (min‐max) | 60.5 (2.4‐972.0) |
| RBCT burden (units/24 weeks), median (min‐max) | 14.1 (0‐34.0) |
| Total bilirubin (μmol/L), median (min‐max) | 32.8 (5.0‐246.0) |
| Albumin (g/L), median (min‐max) | 46.0 (30.0‐56.0) |
| Aspartate transaminase (U/L), median (min‐max) | 22.0 (10.0‐116.0) |
| eGFR (mL/min/1.73 m2), median (min‐max) | 120.0 (53.7‐314.0) |
eGFR, estimated glomerular filtration rate; ICT, iron chelation therapy; max, maximum; min, minimum; n, number of patients in each category; N, overall number of patients; RBCT, red blood cell transfusion.