Table 6

Change in iron burden on DFO or DFX monotherapy

MeasurementDFO
Mean time, y (SD)PDFX
Mean time, y (SD)PP DFO vs DFX§
Historical*Baseline*Follow-up*Historical*Baseline*Follow-up*
Ferritin (ng/mL) n = 64 n = 78 n = 67   n = 168 n = 188 n = 180    
    Mean (SD) 1964 (2275) 1664 (2162) 1376 (1282) 4.1 (2.0) .37 2202 (2082) 1906 (2197) 1859 (2353) 3.7 (1.4) .001 .004 
LIC (mg/g dw) n = 35 n = 56 n = 52   n = 63 n = 147 n = 136    
    Mean (SD) 12.3 (8.4) 10.2 (9.0) 9.5 (8.8) 4.0 (1.5) .06 13.7 (13.4) 10.4 (8.7) 6.6 (7.0) 3.8 (1.4) < .001 .42 
Cardiac T2*, ms n = 17 n = 39 n = 37   n = 43 n = 92 n = 85    
    Mean (SD) 30.9 (14.7) 25.9 (13.8) 24.6 (12.7) 3.3 (1.2) .09 25.0 (14.1) 26.3 (13.3) 27.4 (13.6) 3.8 (1.4) .76 .46 
Adherence, n (%) 
    < 25% 1 (1.7) 1 (1.4) 1 (2.1)  .12 1 (1.1) 1 (0.6%) 2 (1.5)  .72 .004 
    25%-49% 6 (0.0) 2 (2.9) 1 (2.1)  3 (3.3) 2 (1.1%) 1 (0.8)     
    50%-74% 11 (18.3) 11 (15.7) 7 (14.6)  6 (6.6) 15 (8.3%) 13 (9.8)     
    ≥ 75% 42 (70.0) 56 (80.0) 39 (81.3)  81 (89.0) 162 (90.0%) 117 (88.0)     
MeasurementDFO
Mean time, y (SD)PDFX
Mean time, y (SD)PP DFO vs DFX§
Historical*Baseline*Follow-up*Historical*Baseline*Follow-up*
Ferritin (ng/mL) n = 64 n = 78 n = 67   n = 168 n = 188 n = 180    
    Mean (SD) 1964 (2275) 1664 (2162) 1376 (1282) 4.1 (2.0) .37 2202 (2082) 1906 (2197) 1859 (2353) 3.7 (1.4) .001 .004 
LIC (mg/g dw) n = 35 n = 56 n = 52   n = 63 n = 147 n = 136    
    Mean (SD) 12.3 (8.4) 10.2 (9.0) 9.5 (8.8) 4.0 (1.5) .06 13.7 (13.4) 10.4 (8.7) 6.6 (7.0) 3.8 (1.4) < .001 .42 
Cardiac T2*, ms n = 17 n = 39 n = 37   n = 43 n = 92 n = 85    
    Mean (SD) 30.9 (14.7) 25.9 (13.8) 24.6 (12.7) 3.3 (1.2) .09 25.0 (14.1) 26.3 (13.3) 27.4 (13.6) 3.8 (1.4) .76 .46 
Adherence, n (%) 
    < 25% 1 (1.7) 1 (1.4) 1 (2.1)  .12 1 (1.1) 1 (0.6%) 2 (1.5)  .72 .004 
    25%-49% 6 (0.0) 2 (2.9) 1 (2.1)  3 (3.3) 2 (1.1%) 1 (0.8)     
    50%-74% 11 (18.3) 11 (15.7) 7 (14.6)  6 (6.6) 15 (8.3%) 13 (9.8)     
    ≥ 75% 42 (70.0) 56 (80.0) 39 (81.3)  81 (89.0) 162 (90.0%) 117 (88.0)     
*

Historical values are retrospective values obtained at the time point closest to starting (or continuing) the chelator at the time of enrollment, baseline values are values at the time point closest to the baseline visit, and follow-up values are the last available follow-up values while still receiving that chelator.

Time refers to total time on chelator (historical to follow-up).

For comparison of historical and follow-up values within each chelator type.

§

For comparison of change in iron burden between DFO and DFX after adjusting for duration of time on the chelator.

For overall comparison of adherence rates after adjusting for time effect.

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