Cochrane · systematic review · CD007476

Deferasirox for managing iron overload in people with thalassaemia

Bollig, C, Schell, LK, Rücker, G, et al. · Cochrane Database of Systematic Reviews · 2017 · Cochrane Library

Cystic Fibrosis and Genetic Disorders2 versionsAMSTAR 2: not assessed
SoF tableOnly the outcomes this review put in its Summary of Findings table are listed (the Cochrane Handbook caps them at about seven per comparison). Its other analyses are not shown here.
Questions
49
current version
GRADE rated
25
No studies
10
Included studies
NR
No studies 10 · 20.4%Ungraded 14 · 28.6%Very low 10 · 20.4%Low 8 · 16.3%Moderate 7 · 14.3%High 0 · 0%

Ungraded means an effect was reported without a GRADE rating; it is not a gap. No studies means the review found nothing to answer the question. The six categories are a display order, not a score.

Questions and results

Current version only

Deferasirox compared to deferoxamine in people with transfusion‐dependent thalassemia

Population: people with transfusion‐dependent thalassemia
CD007476.PUB3 (current) · 7 questions
OutcomeEffectEvidenceStatus / GRADE
Mortality at any time point
Deferasirox vs deferoxamine
RR 0.48 [0.09, 2.63]8 studies · 1170 participantsLow
Downgraded: risk of bias, imprecision
Responder analysis II (responder: LIC 1 to less than 7 mg Fe/g dw)
Deferasirox vs deferoxamine
RR 0.8 [0.69, 0.92]1 study · 553 participantsModerate
Downgraded: risk of bias, imprecision, inconsistency
Serum ferritin (ng/mL): mean change from baseline and at end of study
Deferasirox vs deferoxamine
no estimate extracted6 studies · 1002 participantsModerate
Downgraded: risk of bias, imprecision, inconsistency
LIC (mg Fe/g dw) evaluated by biopsy or SQUID: mean change from baseline
Deferasirox vs deferoxamine
no estimate extracted1 study · 541 participantsModerate
Downgraded: risk of bias, imprecision, inconsistency
Satisfaction with treatment (very satisfied or satisfied): participants previously treated with DFO
Deferasirox vs deferoxamine
RR 2.2 [1.89, 2.57]1 study · 571 participantsModerate
Downgraded: risk of bias
Adherence: discontinuations
Deferasirox vs deferoxamine
RR 0.95 [0.6, 1.5]8 studies · 1211 participantsLow
Downgraded: risk of bias, imprecision
AE: investigations ‐ isolated serum creatinine increase above ULN
Deferasirox vs deferoxamine
RR 2.57 [1.88, 3.51]2 studies · 657 participantsLow
Downgraded: risk of bias, indirectness

Deferasirox compared to deferiprone in people with transfusion‐dependent thalassemia

Population: people with transfusion‐dependent thalassemia
CD007476.PUB3 (current) · 7 questions
OutcomeEffectEvidenceStatus / GRADE
Mortality at any time point
Deferasirox vs deferiprone
no estimate extracted3 studies · 146 participantsVery low
Downgraded: risk of bias, imprecision
Responder analysis
Deferasirox vs deferiprone
no estimate extractedNRNo studies
Serum ferritin (ng/mL): mean change from baseline and at end of study
Deferasirox vs deferiprone
no estimate extracted2 studies · 83 participantsVery low
Downgraded: risk of bias, imprecision
LIC (mg/g) evaluated by MRI R2*: mean change from baseline
Deferasirox vs deferiprone
no estimate extracted1 study · 45 participantsVery low
Downgraded: risk of bias, imprecision
Satisfaction
Deferasirox vs deferiprone
no estimate extractedNRNo studies
Adherence: discontinuations
Deferasirox vs deferiprone
RR 0.16 [0.01, 2.99]3 studies · 179 participantsLow
Downgraded: risk of bias, imprecision
Renal failure
Deferasirox vs deferiprone
no estimate extracted1 study · 38 participantsVery low
Downgraded: risk of bias, imprecision

Deferasirox alone compared to combined deferasirox and deferiprone in people with transfusion‐dependent thalassemia

Population: people with transfusion‐dependent thalassaemia
CD007476.PUB3 (current) · 7 questions
OutcomeEffectEvidenceStatus / GRADE
Mortality at any time point
Deferasirox alone vs combined deferasirox and deferiprone
no estimate extracted1 participantsUngraded
Responder analysis
Deferasirox alone vs combined deferasirox and deferiprone
no estimate extracted1 participantsUngraded
Serum ferritin (ng/mL)
Deferasirox alone vs combined deferasirox and deferiprone
no estimate extracted1 participantsUngraded
LIC (mg Fe/g dry weight)
Deferasirox alone vs combined deferasirox and deferiprone
no estimate extracted1 participantsUngraded
Satisfaction with treatment
Deferasirox alone vs combined deferasirox and deferiprone
no estimate extracted1 participantsUngraded
Adherence
Deferasirox alone vs combined deferasirox and deferiprone
no estimate extracted1 participantsUngraded
AE: serum creatinine increase
Deferasirox alone vs combined deferasirox and deferiprone
no estimate extracted1 participantsUngraded

Combined deferasirox and deferiprone compared to deferiprone alone in people with transfusion‐dependent thalassemia

Population: people with transfusion‐dependent thalassemia
CD007476.PUB3 (current) · 7 questions
OutcomeEffectEvidenceStatus / GRADE
Mortality at any time point
Combined deferasirox and deferiprone vs deferiprone alone
no estimate extracted1 participantsUngraded
Responder analysis
Combined deferasirox and deferiprone vs deferiprone alone
no estimate extracted1 participantsUngraded
Serum ferritin (ng/mL)
Combined deferasirox and deferiprone vs deferiprone alone
no estimate extracted1 participantsUngraded
LIC (mg Fe/g dry weight)
Combined deferasirox and deferiprone vs deferiprone alone
no estimate extracted1 participantsUngraded
Satisfaction with treatment
Combined deferasirox and deferiprone vs deferiprone alone
no estimate extracted1 participantsUngraded
Adherence
Combined deferasirox and deferiprone vs deferiprone alone
no estimate extracted1 participantsUngraded
AE: Serum creatinine increase
Combined deferasirox and deferiprone vs deferiprone alone
no estimate extracted1 participantsUngraded

Deferasirox and deferoxamine compared to deferoxamine in people with transfusion‐dependent thalassemia

Population: people with transfusion‐dependent thalassemia
CD007476.PUB3 (current) · 7 questions
OutcomeEffectEvidenceStatus / GRADE
Mortality at any time point
Deferasirox and deferoxamine vs deferoxamine
no estimate extracted1 study · 94 participantsLow
Downgraded: imprecision
Responder analysis
Deferasirox and deferoxamine vs deferoxamine
no estimate extractedNRNo studies
Serum ferritin (ng/mL) ‐ mean at end of study
Deferasirox and deferoxamine vs deferoxamine
no estimate extracted1 study · 94 participantsVery low
Downgraded: risk of bias, imprecision
LIC
Deferasirox and deferoxamine vs deferoxamine
no estimate extractedNRNo studies
Satisfaction with treatment
Deferasirox and deferoxamine vs deferoxamine
no estimate extractedNRNo studies
Adherence: Discontinuations
Deferasirox and deferoxamine vs deferoxamine
no estimate extracted1 study · 94 participantsVery low
Downgraded: risk of bias, imprecision
AE: serum creatinine increased
Deferasirox and deferoxamine vs deferoxamine
no estimate extractedNRNo studies

Deferasirox and deferiprone compared to deferiprone and deferoxamine in people with transfusion‐dependent thalassemia

Population: people with transfusion‐dependent thalassemia
CD007476.PUB3 (current) · 7 questions
OutcomeEffectEvidenceStatus / GRADE
Mortality at any time point
Deferasirox and deferiprone vs deferiprone and deferoxamine
no estimate extracted1 study · 96 participantsLow
Downgraded: imprecision
Responder analysis
Deferasirox and deferiprone vs deferiprone and deferoxamine
no estimate extractedNRNo studies
Serum ferritin (ng/mL): mean change from baseline
Deferasirox and deferiprone vs deferiprone and deferoxamine
no estimate extracted1 study · 96 participantsVery low
Downgraded: risk of bias, imprecision
LIC evaluated by MRI R2*: mean change from baseline
Deferasirox and deferiprone vs deferiprone and deferoxamine
no estimate extracted1 study · 96 participantsLow
Downgraded: risk of bias, imprecision
Satisfaction
Deferasirox and deferiprone vs deferiprone and deferoxamine
no estimate extractedNRNo studies
Adherence: Discontinuations
Deferasirox and deferiprone vs deferiprone and deferoxamine
no estimate extracted1 study · 96 participantsVery low
Downgraded: risk of bias, imprecision
Drug‐related AE: serum creatinine increased (≥ 33%) above baseline in 2 consecutive occasions
Deferasirox and deferiprone vs deferiprone and deferoxamine
RR 3 [0.32, 27.83]1 study · 96 participantsVery low
Downgraded: risk of bias, imprecision, indirectness

Deferasirox compared to placebo in people with non‐transfusion‐dependent thalassemia

Population: people with non‐transfusion‐dependent thalassemia
CD007476.PUB3 (current) · 7 questions
OutcomeEffectEvidenceStatus / GRADE
Mortality at any time point
Deferasirox vs placebo
no estimate extracted1 study · 148 participantsModerate
Downgraded: imprecision
Responder analysis
Deferasirox vs placebo
no estimate extractedNRNo studies
Serum ferritin (ng/mL): mean change from baseline
Deferasirox vs placebo
no estimate extracted1 study · 154 participantsModerate
Downgraded: imprecision
LIC (mg Fe/g dry weight) evaluated by MRI R2: least squares mean change from baseline
Deferasirox vs placebo
no estimate extracted1 study · 159 participantsModerate
Downgraded: imprecision
Satisfaction with treatment
Deferasirox vs placebo
no estimate extractedNRNo studies
Adherence: Discontinuations
Deferasirox vs placebo
RR 1.32 [0.5, 3.52]1 study · 166 participantsLow
Downgraded: imprecision
AE: abnormal serum creatinine (post‐baseline)
Deferasirox vs placebo
RR 3.59 [0.19, 68.4]1 study · 166 participantsVery low
Downgraded: imprecision, indirectness

Deferasirox compared to Deferoxamine for People with thalassaemia and secondary iron overload

Population: People with thalassaemia and secondary iron overload
CD007476.PUB2 · 7 questions
OutcomeEffectEvidenceStatus / GRADE
Mortality at any point in time
Deferasirox vs Deferoxamine
RR 0.33 [0.03, 3.12]2 studies · 622 participantsLow
Downgraded: risk of bias, imprecision
Responder analysis (Responder: LIC: 1 to less than 7 mg Fe/g dw)
Deferasirox vs Deferoxamine
RR 0.8 [0.69, 0.92]1 study · 553 participantsLow
Downgraded: risk of bias, inconsistency, indirectness
Mean change in serum ferritin (µg/l)
Deferasirox vs Deferoxamine
no estimate extracted1 study · 563 participantsModerate
Downgraded: risk of bias, inconsistency
Change in LIC (mg Fe/g dw) evaluated by biopsy or SQUID
Deferasirox vs Deferoxamine
no estimate extracted1 study · 541 participantsModerate
Downgraded: risk of bias, indirectness
Satisfaction with treatment (very satisfied or satisfied): Patients previously treated with DFO questionnaire
Deferasirox vs Deferoxamine
RR 1.82 [1.61, 2.05]1 study · 571 participantsModerate
Downgraded: risk of bias
Discontinuations
Deferasirox vs Deferoxamine
RR 1.19 [0.62, 2.29]1 study · 657 participantsLow
Downgraded: risk of bias, imprecision
Adverse event: Isolated serum creatinine increase above upper limit of normal
Deferasirox vs Deferoxamine
RR 2.57 [1.88, 3.51]2 studies · 657 participantsLow
Downgraded: risk of bias, indirectness