Variables associated with long-term survival in thalassemia major
. | . | CHF, arrhythmia, or both . | . | . | . | |
|---|---|---|---|---|---|---|
| Variable . | No. patients . | Yes . | No . | Dead . | Alive . | |
| LVEF decreased to absolute value below 45% or by more than 10% | 34 | 12 | 22 | 7 | 27 | |
| LVEF never decreased to absolute value below 45% or by more than 10% | 47 | 1 | 46 | 0 | 47 | |
| Total no. patients | 81 | 13 | 68 | 7 | 74 | |
| Subcutaneous DFO started after age 10 y | 30 | 11 | 19 | 7 | 23 | |
| Subcutaneous DFO started before age 10 y | 48 | 5 | 43 | 2 | 46 | |
| Total no. patients | 78 | 16 | 62 | 9 | 69 | |
| Serum ferritin greater than 2500 μg/L for at least one third of follow-up time* | 47 | 13 | 34 | 10 | 37 | |
| Serum ferritin greater than 2500 μg/L for less than one third of follow up-time | 38 | 4 | 34 | 0 | 38 | |
| Total no. patients | 85 | 17 | 68 | 10 | 85 | |
. | . | CHF, arrhythmia, or both . | . | . | . | |
|---|---|---|---|---|---|---|
| Variable . | No. patients . | Yes . | No . | Dead . | Alive . | |
| LVEF decreased to absolute value below 45% or by more than 10% | 34 | 12 | 22 | 7 | 27 | |
| LVEF never decreased to absolute value below 45% or by more than 10% | 47 | 1 | 46 | 0 | 47 | |
| Total no. patients | 81 | 13 | 68 | 7 | 74 | |
| Subcutaneous DFO started after age 10 y | 30 | 11 | 19 | 7 | 23 | |
| Subcutaneous DFO started before age 10 y | 48 | 5 | 43 | 2 | 46 | |
| Total no. patients | 78 | 16 | 62 | 9 | 69 | |
| Serum ferritin greater than 2500 μg/L for at least one third of follow-up time* | 47 | 13 | 34 | 10 | 37 | |
| Serum ferritin greater than 2500 μg/L for less than one third of follow up-time | 38 | 4 | 34 | 0 | 38 | |
| Total no. patients | 85 | 17 | 68 | 10 | 85 | |
Cardiac disease-free survival is shown in patients as a function of LVEF, age of DFO commencement, and serum ferritin. Subsequent development of heart disease (P < .001) and death (P = .001) was significantly more likely in patients who displayed LVEF less than 45% or decreases exceeding 10 percentage units. Symptomatic heart disease occurred more frequently in patients who were older than 10 when subcutaneous DFO therapy was started than in those in whom this treatment was started at an earlier age (P = .005). This was also true of death from cardiac causes (P = .01). Cardiac complications (P = .05) and death (P = .002) were significantly greater in patients who did not maintain ferritin levels lower than 2500 μg/L on at least two thirds of occasions.
Serum ferritin data were available for a median period of 8 years (interquartile range, 4-16 years).