Summary of findings 6. Deferasirox and deferiprone compared to deferiprone and deferoxamine in people with transfusion‐dependent thalassemia.
| Deferasirox and deferiprone compared to deferiprone and deferoxamine in people with transfusion‐dependent thalassemia | ||||||
| Patient or population: people with transfusion‐dependent thalassemia Setting: outpatient care Intervention: deferasirox and deferiprone Comparison: deferiprone and deferoxamine | ||||||
| Outcomes | Anticipated absolute effects* (95% CI) | Relative effect (95% CI) | № of participants (studies) | Quality of the evidence (GRADE) | Comments | |
| Risk with deferiprone and deferoxamine | Risk with deferasirox and deferiprone | |||||
| Mortality at any time point | Study population | not estimable | 96 (1 RCT) | ⊕⊕⊝⊝ LOW 1 | "All the included patients continued till the end of study with no patients were lost follow‐up." (Elalfy 2015b) | |
| 0 per 1.000 | 0 per 1.000 (0 to 0) | |||||
| Responder analysis | Not measured | NA | ||||
| Serum ferritin (ng/mL): mean change from baseline | MD 315.9 ng/mL lower (1046.26 lower to 414.46 higher) | ‐ | 96 (1 RCT) | ⊕⊝⊝⊝ VERY LOW 2 3 | ||
| LIC evaluated by MRI R2*: mean change from baseline | MD 0.62 mg/g lower (2.25 lower to 1.01 higher) | ‐ | 96 (1 RCT) | ⊕⊕⊝⊝ LOW 2 4 | ||
| Satisfaction | Not reported | NA | "Compared to baseline, patient‐reported satisfaction associated with ICT was significantly higher in group B [DFX and DFP] compared to group A [DFP and DFO] (p<0.01)" (Elalfy 2015b) | |||
| Adherence: Discontinuations | Study population | not estimable | 96 (1 RCT) | ⊕⊝⊝⊝ VERY LOW 1 2 | ||
| 0 per 1.000 | 0 per 1.000 (0 to 0) | |||||
| Drug‐related AE: serum creatinine increased (≥ 33%) above baseline in 2 consecutive occasions | Study population | RR 3.00 (0.32 to 27.83) | 96 (1 RCT) | ⊕⊝⊝⊝ VERY LOW 2 3 5 | ||
| 21 per 1.000 | 63 per 1.000 (7 to 580) | |||||
| *The risk in the intervention group (and its 95% CI) is based on the assumed risk in the comparison group and the relative effect of the intervention (and its 95% CI). AE: adverse events; CI: confidence interval; LIC: liver iron concentration; MD: mean difference; MRI: magnetic resonance imaging; NA: not applicable; RR: risk ratio. | ||||||
| GRADE Working Group grades of evidence High quality: we are very confident that the true effect lies close to that of the estimate of the effect Moderate quality: we are moderately confident in the effect estimate: The true effect is likely to be close to the estimate of the effect, but there is a possibility that it is substantially different Low quality: our confidence in the effect estimate is limited: The true effect may be substantially different from the estimate of the effect Very low quality: We have very little confidence in the effect estimate: The true effect is likely to be substantially different from the estimate of effect | ||||||
1 Very serious imprecision: very few participants included. 2 Serious risk of bias: no blinding, selective reporting: no data for 18 months follow‐up. 3 Very serious imprecision: very wide confidence interval including both benefit as well as harm. 4 Serious imprecision: wide confidence interval including both benefit as well as harm. 5 Serious indirectness: surrogate of creatinine used for patient‐important outcome of kidney failure.