Table 1. Demographics, sickle cell disease characteristics and morbidities.
| All dose cohorts (n = 21) | Lower dose cohorts (n = 12) | Higher dose cohorts (n = 9) | P | |
|---|---|---|---|---|
| Demographics | ||||
| Age (years), median (IQR) | 26 (10) | 24 (14) | 30 (10) | NS |
| Gender, % female | 38 | 50 | 22 | NS |
| SCD characteristics | ||||
| Hemoglobin (g/dL), mean (SD) | 9.0 (1.5) | 9.3 (1.4) | 8.7 (1.6) | NS |
| Recticulocyte count (106/μL), mean (SD) | 0.26 (0.12) | 0.20 (0.07) | 0.33 (0.12) | 0.005 |
| Mean cellular volume (fL), mean (SD) | 97.9 (10.5) | 98.8 | 96.7 | NS |
| WBC (k/uL), mean (SD) | 8.4 (2.9) | 6.8 (2.3) | 10.5 (2.3) | 0.002 |
| LDH (U/L), mean (SD) | 533.4 (313.1) | 624 (376) | 413 (146) | NS |
| CRP (mg/L), mean (SD) | 5.3 (4.8) | 5.2 (5.2) | 5.4 (4.5) | NS |
| Hydroxyurea, % yes | 67 | 83 | 44 | NS |
| SCD morbidities | ||||
| Pain episode in past year, % yes | 62 | 33 | 89 | NS |
| # pain episodes in past year, mean (SD) | 1.1 (1.2) | 0.83 (1.4) | 1.56 (1.0) | NS |
| ACS, % yes | 52 | 42 | 67 | NS |
| # ACS lifetime, mean (SD) | 1.2 (1.6) | 1.17 (1.9) | 1.33 (1.2) | NS |
| Stroke, % yes | 5 | 8 | 0 | NS |
| AVN, % yes | 24 | 8 | 44 | NS |
| Gallbladder disease, % yes | 38 | 42 | 33 | NS |
| Splenic sequestration, % yes | 19 | 17 | 22 | NS |
| # cumulative morbidities*, mean (SD) | 0.9 (1.0) | 0.6 (0.9) | 1.2 (1.0) | NS |
Definitions: ACS = acute chest syndrome, AVN = avascular necrosis, CRP = c-reactive protein, IQR = interquartile range, LDH = lactate dehydrogenase, SD = standard deviation, WBC = white blood count.
*cumulative morbidities = ACS + stroke + AVN + gallbladder disease + splenic sequestration.