Table 1.
Baseline Characteristics by Treatment Arm and Overall—Intention-to-Treat Set
| Parameter | Statistics | Arm 1, PM/MF 14 Days (n = 171) |
Arm 2,a PM/MF 28 Days (n = 98) |
Arm 3, SSG/PM (n = 170) |
Overall (n = 439) |
|---|---|---|---|---|---|
| Age, years | Mean (SD) | 13.5 (7.9) | 13.9 (8.0) | 13.0 (7.1) | 13.4 (7.6) |
| Median (Q1–Q3) | 10.0 (8.0–19.0) | 12.0 (8.0–20.0) | 10.0 (8.0–18.0) | 11.0 (8.0–19.0) | |
| Age category | 4 to ≤12 years, n (%) | 103 (60.2) | 54 (55.1) | 105 (61.8) | 262 (59.7) |
| >12 to 50 years, n (%) | 68 (39.8) | 44 (44.9) | 65 (38.2) | 177 (40.3) | |
| Sex | Female, n (%) | 34 (19.9) | 17 (17.3) | 38 (22.4) | 89 (20.3) |
| Male, n (%) | 137 (80.1) | 81 (82.7) | 132 (77.6) | 350 (79.7) | |
| Weight, kg | Range (Min–Max) | 11.0–71.0 | 14.0–61.0 | 11.0–60.0 | 11.0–71.0 |
| Mean (SD) | 32.7 (14.4) | 33.4 (13.7) | 31.4 (13.4) | 32.4 (13.9) | |
| Median (Q1–Q3) | 28.0 (21.0–46.0) | 32.0 (20.5–47.0) | 27.3 (19.5–43.0) | 28.0 (20.0–45.0) | |
| Height, cm | Range (Min–Max) | 93.0–190.0 | 102.0–182.0 | 92.0–190.0 | 92.0–190.0 |
| Mean (SD) | 141.0 (23.8) | 143.9 (22.8) | 139.9 (23.3) | 141.2 (23.4) | |
| Median (Q1–Q3) | 137.0 (124.0–165.0) | 141.5 (123.0–166.0) | 138.0 (121.0–163.0) | 139.0 (122.0–164.0) | |
| Nutritional status according to World Health Organization reference curves and Body mass index | N = 145 | N = 79 | N = 145 | N = 369 | |
| Moderate underweight, n (%) | 43 (29.7) | 35 (44.3) | 49 (33.8) | 127 (34.4) | |
| Normal, n (%) | 101 (69.7) | 42 (53.2) | 95 (65.5) | 238 (64.5) | |
| Overweight, n (%) | 1 (0.7) | 2 (2.5) | 1 (0.7) | 4 (1.1) | |
| Nutritional status, mid-upper arm circumference (MUAC), mm | N = 26 | N = 19 | N = 25 | N = 70 | |
| Range (Min–Max) | 160.0–260.0 | 165.0–240.0 | 168.0–255.0 | 160.0–260.0 | |
| Mean (SD) | 217.0 (23.1) | 200.1 (18.8) | 208.2 (23.3) | 209.3 (22.8) | |
| Median (Q1–Q3) | 220.0 (200.0–230.0) | 200.0 (184.0–220.0) | 205.0 (190.0–225.0) | 210.0 (190.0–223.0) | |
| Hemoglobin, g/dL | Range (Min–Max) | 5.0–12.3 | 5.1–19.7 | 5.1–13.8 | 5.0–19.7 |
| Mean (SD) | 8.0 (1.4) | 8.5 (2.2) | 8.1 (1.7) | 8.2 (1.7) | |
| Median (Q1–Q3) | 8.0 (6.9–8.9) | 8.1 (7.0–9.6) | 8.0 (6.7–9.2) | 8.0 (6.9–9.1) | |
| White blood cells, ×103/μL | Range (Min–Max) | 0.9–9.1 | 1.0–8.5 | 0.5–6.4 | 0.5–9.1 |
| Mean (SD) | 2.8 (1.3) | 2.4 (1.0) | 2.8 (1.2) | 2.7 (1.2) | |
| Median (Q1–Q3) | 2.6 (2.0–3.4) | 2.3 (1.6–3.0) | 2.6 (1.9–3.5) | 2.5 (1.9–3.3) | |
| Platelets, ×103/μL | Range (Min–Max) | 19.0–338.0 | 45.0–342.0 | 26.0–446.0 | 19.0–446.0 |
| Mean (SD) | 118.8 (56.5) | 113.9 (50.8) | 120.8 (61.7) | 118.5 (57.3) | |
| Median (Q1–Q3) | 108.0 (73.0–154.0) | 108.5 (83.0–134.0) | 105.0 (80.0–147.0) | 107.0 (79.0–146.0) | |
| Aspartate aminotransferase, U/L | Range (Min–Max) | 6.0–592.0 | 13.0–118.0 | 10.0–621.0 | 6.0–621.0 |
| Mean (SD) | 79.9 (85.3) | 51.9 (26.0) | 89.8 (94.8) | 77.5 (81.6) | |
| Median (Q1–Q3) | 58.0 (40.0–83.0) | 45.5 (31.0–68.0) | 62.5 (38.0–99.0) | 56.0 (36.0–87.0) | |
| Alanine aminotransferase, U/L | Range (Min–Max) | 0.8–473.0 | 2.0–105.0 | 6.0–397.0 | 0.8–473.0 |
| Mean (SD) | 40.1 (50.0) | 27.0 (18.9) | 46.7 (58.9) | 39.7 (49.4) | |
| Median (Q1–Q3) | 27.0 (18.0–44.0) | 22.0 (14.0–40.0) | 30.0 (18.0–48.0) | 27.0 (16.0–45.0) | |
| Creatinine, mg/dL | Range (Min–Max) | 0.1–1.5 | 0.3–1.3 | 0.1–1.4 | 0.1–1.5 |
| Mean (SD) | 0.8 (0.3) | 0.9 (0.2) | 0.7 (0.3) | 0.8 (0.3) | |
| Median (Q1–Q3) | 0.8 (0.6–1.0) | 0.8 (0.7–1.0) | 0.7 (0.6–0.9) | 0.8 (0.6–1.0) | |
| Albumin, g/L | Range (Min–Max) | 1.5–54.61 | 4.0–53.2 | 9.0–48.6 | 1.5–54.6 |
| Mean (SD) | 27.4 (7.8) | 29.9 (9.5) | 26.8 (7.1) | 27.7 (8.0) | |
| Median (Q1–Q3) | 27.0 (23.0–31.0) | 28.4 (23.6–35.4) | 26.4 (22.2–31.0) | 27.0 (23.0–31.8) | |
| Spleen size, cm | Range (Min–Max) | 0.0–22.0 | 0.0–20.0 | 0.0–24.0 | 0.0–24.0 |
| Mean (SD) | 7.6 (4.1) | 8.8 (3.9) | 7.8 (4.5) | 7.9 (4.2) | |
| Median (Q1–Q3) | 7.0 (4.0–10.0) | 8.0 (6.0–11.5) | 7.0 (4.0–11.0) | 8.0 (5.0–11.0) | |
| Liver size, cm | Range (Min–Max) | 0.0–7.0 | 0.0–8.0 | 0.0–10.0 | 0.0–10.0 |
| Mean (SD) | 1.6 (2.0) | 1.0 (1.8) | 1.5 (2.1) | 1.4 (2.0) | |
| Median (Q1–Q3) | 0.0 (0.0–4.0) | 0.0 (0.0–2.0) | 0.0 (0.0–3.0) | 0.0 (0.0–2.0) |
Abbreviations: MF, miltefosine; PM, paromomycin; Q, quartile; SD, standard deviation; SSG, sodium stibogluconate.
Recruitment into arm 2 was discontinued.