Baseline characteristics and primary outcome by type of therapy (n = 419)
| Variable . | No disease-modifying treatment (n = 98) . | Hydroxyurea (n = 243) . | Transfusion (n = 56) . | HSCT (n = 22) . | P∗ . |
|---|---|---|---|---|---|
| Age at start of follow up (y), median (IQR) | 11.5 (1.1-27.6) | 9.0 (0.5-21.4) | 8.8 (5.0-21.5) | 20.5 (13.2-27.3) | .032 |
| Sex (male), n (%) | 43 (43.9) | 119 (49.0) | 32 (57.1) | 15 (68.2) | .132 |
| Follow-up time (y), median (IQR) | 2.5 (1.1-5.7) | 9.1 (4.9-14.0) | 11.7 (7.2-16.5) | 4.2 (2.8-11.4) | <.001 |
| Largest gap in care (y), median (IQR) | 0.6 (0.3-1.0) | 0.8 (0.5-1.2) | 0.6 (0.3-1.0) | 0.6 (0.5-1.0) | .002 |
| Use of Medicaid, n (%) | <.001 | ||||
| 0% | 41 (41.8) | 50 (20.6) | 8 (14.3) | 9 (40.9) | |
| 1%-99% | 22 (22.4) | 99 (40.7) | 27 (48.2) | 9 (40.9) | |
| 100% | 35 (35.7) | 94 (38.7) | 21 (37.5) | 4 (18.2) | |
| Death, n (%) | 12 (12.2) | 23 (9.5) | 12 (21.4) | 0 (0.0) | .027† |
| Rate of death/100 patient-years (95% CI) | 3.01 (1.56-5.26) | 1.00 (0.64-1.51) | 1.82 (0.94-3.18) | 0.0 (not applicable) | <.001‡ |
| Variable . | No disease-modifying treatment (n = 98) . | Hydroxyurea (n = 243) . | Transfusion (n = 56) . | HSCT (n = 22) . | P∗ . |
|---|---|---|---|---|---|
| Age at start of follow up (y), median (IQR) | 11.5 (1.1-27.6) | 9.0 (0.5-21.4) | 8.8 (5.0-21.5) | 20.5 (13.2-27.3) | .032 |
| Sex (male), n (%) | 43 (43.9) | 119 (49.0) | 32 (57.1) | 15 (68.2) | .132 |
| Follow-up time (y), median (IQR) | 2.5 (1.1-5.7) | 9.1 (4.9-14.0) | 11.7 (7.2-16.5) | 4.2 (2.8-11.4) | <.001 |
| Largest gap in care (y), median (IQR) | 0.6 (0.3-1.0) | 0.8 (0.5-1.2) | 0.6 (0.3-1.0) | 0.6 (0.5-1.0) | .002 |
| Use of Medicaid, n (%) | <.001 | ||||
| 0% | 41 (41.8) | 50 (20.6) | 8 (14.3) | 9 (40.9) | |
| 1%-99% | 22 (22.4) | 99 (40.7) | 27 (48.2) | 9 (40.9) | |
| 100% | 35 (35.7) | 94 (38.7) | 21 (37.5) | 4 (18.2) | |
| Death, n (%) | 12 (12.2) | 23 (9.5) | 12 (21.4) | 0 (0.0) | .027† |
| Rate of death/100 patient-years (95% CI) | 3.01 (1.56-5.26) | 1.00 (0.64-1.51) | 1.82 (0.94-3.18) | 0.0 (not applicable) | <.001‡ |