Patient and disease characteristics, for example, for acute complications, and laboratory parameters of adult and pediatric individuals with SCA
| . | Pediatric cohort . | Adult cohort . |
|---|---|---|
| Patient characteristics | n = 177 | n = 50 |
| Age, median (range), y | 9 (2-19) | 26 (16-57) |
| Sex, female, n (%) | 76 of 177 (43) | 22 of 50 (44) |
| Hydroxyurea therapy, n (%) | 157 of 177 (89) | 32 of 50 (44) |
| α-thalassemia: 1 deletion∗, n (%) | n/a | 8 of 45 (18) |
| 2 deletions∗, n (%) | n/a | 7 of 45 (16) |
| Splenectomy, n (%) | 35 of 177 (20) | 10 of 50 (20) |
| Acute complications | ||
| ACS, n (%) | 97 of 177 (55) | 14 of 50 (28) |
| VOE, n (%) | 84 of 171 (49) | 33 of 50 (66) |
| Cerebral infarction, n (%) | 14 of 177 (8) | 3 of 50 (6) |
| >1 type of acute complication†, n (%) | 63 of 177 (36) | 15 of 50 (30) |
| TCD parameters | ||
| TCD left MCA (cm/s), mean (SD)‡ | 132 (26) | n/a |
| TCD right MCA (cm/s), mean (SD)§ | 129 (26) | n/a |
| Laboratory parameters | ||
| HbF, %, mean (SD)ǁ | 16.0 (8.2) | 13.3 (10.7) |
| Hb, g/dL, mean (SD) | 8.8 (1.4) | 9.1 (1.3) |
| HCT, %, mean (SD) | 26.9 (3.8) | 26.3 (3.4) |
| ARC, 109 per L, mean (SD) | 398 (152) | 227 (102) |
| MCV, fl, mean (SD) | 95 (13) | 89 (17) |
| MCHC, g/dL, mean (SD) | 32.8 (1.3) | 34.8 (1.4) |
| DRBC, %, mean (SD) | 4.9 (3.3) | n/a |
| Blood viscosity (45 s−1), mean (SD)¶ | 4.9 (1.1) | n/a |
| Blood viscosity (225 s−1), mean (SD)¶ | 3.4 (0.7) | n/a |
| HVR, mean (SD) | 5.7 (0.9) | n/a |
| Oxygen gradient ektacytometry parameters | ||
| PoS (mmHg), mean (SD) | 38.6 (9.6) | 49.9 (10.5) |
| EImax (EI), mean (SD) | 0.48 (0.09) | 0.44 (0.08) |
| EImin (EI), mean (SD) | 0.14 (0.10) | 0.13 (0.08) |
| . | Pediatric cohort . | Adult cohort . |
|---|---|---|
| Patient characteristics | n = 177 | n = 50 |
| Age, median (range), y | 9 (2-19) | 26 (16-57) |
| Sex, female, n (%) | 76 of 177 (43) | 22 of 50 (44) |
| Hydroxyurea therapy, n (%) | 157 of 177 (89) | 32 of 50 (44) |
| α-thalassemia: 1 deletion∗, n (%) | n/a | 8 of 45 (18) |
| 2 deletions∗, n (%) | n/a | 7 of 45 (16) |
| Splenectomy, n (%) | 35 of 177 (20) | 10 of 50 (20) |
| Acute complications | ||
| ACS, n (%) | 97 of 177 (55) | 14 of 50 (28) |
| VOE, n (%) | 84 of 171 (49) | 33 of 50 (66) |
| Cerebral infarction, n (%) | 14 of 177 (8) | 3 of 50 (6) |
| >1 type of acute complication†, n (%) | 63 of 177 (36) | 15 of 50 (30) |
| TCD parameters | ||
| TCD left MCA (cm/s), mean (SD)‡ | 132 (26) | n/a |
| TCD right MCA (cm/s), mean (SD)§ | 129 (26) | n/a |
| Laboratory parameters | ||
| HbF, %, mean (SD)ǁ | 16.0 (8.2) | 13.3 (10.7) |
| Hb, g/dL, mean (SD) | 8.8 (1.4) | 9.1 (1.3) |
| HCT, %, mean (SD) | 26.9 (3.8) | 26.3 (3.4) |
| ARC, 109 per L, mean (SD) | 398 (152) | 227 (102) |
| MCV, fl, mean (SD) | 95 (13) | 89 (17) |
| MCHC, g/dL, mean (SD) | 32.8 (1.3) | 34.8 (1.4) |
| DRBC, %, mean (SD) | 4.9 (3.3) | n/a |
| Blood viscosity (45 s−1), mean (SD)¶ | 4.9 (1.1) | n/a |
| Blood viscosity (225 s−1), mean (SD)¶ | 3.4 (0.7) | n/a |
| HVR, mean (SD) | 5.7 (0.9) | n/a |
| Oxygen gradient ektacytometry parameters | ||
| PoS (mmHg), mean (SD) | 38.6 (9.6) | 49.9 (10.5) |
| EImax (EI), mean (SD) | 0.48 (0.09) | 0.44 (0.08) |
| EImin (EI), mean (SD) | 0.14 (0.10) | 0.13 (0.08) |
Mean values of patient characteristics, (%), laboratory parameters (mean ± standard deviation), TCD parameters (mean ± standard deviation), and acute complications (%) are shown for the adult SCA cohort and pediatric SCA cohort.
HCT, hematocrit; HVR, hematocrit to viscosity ratio; MCA, media cerebral artery; MCHC, mean corpuscular Hb concentration; MCH, mean corpuscular Hb; n/a, not addressed.
Data of 45 individuals available.
ACS, VOEs, or cerebral infarction.
Data of 113 individuals available.
Data of 115 individuals available.
In pediatric cohort data of 90 individuals available.
Data of 110 individuals available.