Cochrane · systematic review · CD012349
Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia
Geneen, LJ, Dorée, C, Estcourt, LJ · Cochrane Database of Systematic Reviews · 2023 · Cochrane Library
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
42
current version
GRADE rated
25
No studies
16
Included studies
NR
No studies 16 · 38.1%Ungraded 1 · 2.4%Very low 18 · 42.9%Low 7 · 16.7%Moderate 0 · 0%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 onlySummary of findings: Comparison 1 ‐ deferiprone (DFP) versus deferoxamine (DFO)
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Adherence to iron chelation therapy (%, SD) Summary of findings: Comparison 1 ‐ deferiprone (DFP) vs deferoxamine (DFO) | no estimate extracted | 7 studies · 612 participants | Very low Downgraded: risk of bias, imprecision, inconsistency, indirectness |
Total reported SAEs (from therapy, disease, non‐adherence) Summary of findings: Comparison 1 ‐ deferiprone (DFP) vs deferoxamine (DFO) | RR 1.43 [0.83, 2.46] | 1 study · 228 participants | Very low Downgraded: risk of bias, imprecision, inconsistency, indirectness |
All‐cause mortality Summary of findings: Comparison 1 ‐ deferiprone (DFP) vs deferoxamine (DFO) | RR 0.47 [0.18, 1.21] | 3 studies · 376 participants | Very low Downgraded: risk of bias, imprecision, inconsistency, indirectness |
Sustained adherence Summary of findings: Comparison 1 ‐ deferiprone (DFP) vs deferoxamine (DFO) | no estimate extracted | NR | No studies |
QoL ( Summary of findings: Comparison 1 ‐ deferiprone (DFP) vs deferoxamine (DFO) | no estimate extracted | 1 study · 1 participants | Very low Downgraded: risk of bias, imprecision, inconsistency, indirectness |
Summary of findings: Comparison 2 ‐ deferasirox (DFX) versus deferiprone (DFO)
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Adherence to iron chelation therapy (%, SD) Summary of findings: Comparison 2 ‐ deferasirox (DFX) vs deferiprone (DFO) | no estimate extracted | 3 studies · 452 participants | Very low Downgraded: risk of bias, imprecision |
SAEs Thalassaemia‐related SAEs Summary of findings: Comparison 2 ‐ deferasirox (DFX) vs deferiprone (DFO) | RR 0.95 [0.41, 2.17] | 2 studies · 247 participants | Very low Downgraded: risk of bias, imprecision |
SAEs SCD‐related SAEs Summary of findings: Comparison 2 ‐ deferasirox (DFX) vs deferiprone (DFO) | no estimate extracted | 1 study · 195 participants | Very low Downgraded: risk of bias, imprecision |
All‐cause mortality Summary of findings: Comparison 2 ‐ deferasirox (DFX) vs deferiprone (DFO) | 0.96 [0.06, 15.42] | 2 studies · 240 participants | Very low Downgraded: risk of bias, imprecision |
Sustained adherence Summary of findings: Comparison 2 ‐ deferasirox (DFX) vs deferiprone (DFO) | no estimate extracted | NR | No studies |
QoL Summary of findings: Comparison 2 ‐ deferasirox (DFX) vs deferiprone (DFO) | no estimate extracted | NR | No studies |
Summary of findings: Comparison 3 ‐ deferiprone (DFP) versus deferasirox (DFX)
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Adherence to iron chelation (%, SD) Summary of findings: Comparison 3 ‐ deferiprone (DFP) vs deferasirox (DFX) | no estimate extracted | 1 study · 390 participants | Low Downgraded: risk of bias, imprecision |
SAE (chelation‐related) (n/N) Summary of findings: Comparison 3 ‐ deferiprone (DFP) vs deferasirox (DFX) | 1.54 [0.44, 5.39] | 1 study · 390 participants | Very low Downgraded: risk of bias, imprecision |
Total SAEs Summary of findings: Comparison 3 ‐ deferiprone (DFP) vs deferasirox (DFX) | RR 0.95 [0.46, 1.96] | 1 study · 390 participants | Very low Downgraded: risk of bias, imprecision |
All‐cause mortality (n/N) Summary of findings: Comparison 3 ‐ deferiprone (DFP) vs deferasirox (DFX) | RD 0 | 1 study · 390 participants | Low Downgraded: risk of bias, imprecision |
Sustained adherence Summary of findings: Comparison 3 ‐ deferiprone (DFP) vs deferasirox (DFX) | no estimate extracted | NR | No studies |
QoL Summary of findings: Comparison 3 ‐ deferiprone (DFP) vs deferasirox (DFX) | no estimate extracted | NR | No studies |
Summary of findings: Comparison 4 ‐ deferasirox (DFX) film‐coated tablets versus DFX dispersible tablets
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Adherence to iron chelation therapy (%, SD) Summary of findings: Comparison 4 ‐ deferasirox (DFX) film‐coated tablets vs DFX dispersible tablets | no estimate extracted | 1 study · 91 participants | Very low Downgraded: risk of bias, imprecision |
Sustained adherence to iron chelation therapy (%, SD) Summary of findings: Comparison 4 ‐ deferasirox (DFX) film‐coated tablets vs DFX dispersible tablets | no estimate extracted | 1 study · 54 participants | Very low Downgraded: risk of bias, imprecision |
Incidence of SAEs Summary of findings: Comparison 4 ‐ deferasirox (DFX) film‐coated tablets vs DFX dispersible tablets | RR 1.22 [0.62, 2.37] | 1 study · 173 participants | Very low Downgraded: risk of bias, imprecision |
All‐cause mortality Summary of findings: Comparison 4 ‐ deferasirox (DFX) film‐coated tablets vs DFX dispersible tablets | 7.3 [0.14, 368.15] | 1 study · 173 participants | Very low Downgraded: risk of bias, imprecision |
QoL Summary of findings: Comparison 4 ‐ deferasirox (DFX) film‐coated tablets vs DFX dispersible tablets | no estimate extracted | NR | No studies |
Summary of findings: Comparison 5 ‐ deferiprone (DFP) plus deferoxamine (DFO) versus DFP
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Adherence to iron chelation therapy (%, SD) Summary of findings: Comparison 5 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP | no estimate extracted | 4 studies · 369 participants | Low Downgraded: risk of bias, imprecision |
Incidence of SAEs Summary of findings: Comparison 5 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP | RR 0.15 [0.01, 2.81] | 1 study · 213 participants | Low Downgraded: risk of bias, imprecision |
All‐cause mortality Summary of findings: Comparison 5 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP | 0.77 [0.17, 3.42] | 2 studies · 237 participants | Very low Downgraded: risk of bias, imprecision |
Sustained adherence Summary of findings: Comparison 5 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP | no estimate extracted | NR | No studies |
QoL Summary of findings: Comparison 5 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP | no estimate extracted | NR | No studies |
Summary of findings: Comparison 6 ‐ deferiprone (DFP) plus deferoxamine (DFO) versus DFO
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Adherence to iron chelation therapy (%, SD) Summary of findings: Comparison 6 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFO | no estimate extracted | 5 studies · 281 participants | Low Downgraded: risk of bias |
Incidence of SAEs Summary of findings: Comparison 6 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFO | no estimate extracted | 4 studies · 180 participants | Low Downgraded: risk of bias |
All‐cause mortality Summary of findings: Comparison 6 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFO | no estimate extracted | NR | No studies |
Sustained adherence Summary of findings: Comparison 6 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFO | no estimate extracted | NR | No studies |
QoL Summary of findings: Comparison 6 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFO | no estimate extracted | NR | No studies |
Summary of findings: Comparison 7 ‐ deferiprone (DFP) plus deferoxamine (DFO) versus DFP plus deferasirox (DFX)
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Adherence to iron chelation therapy rates (n, N) Summary of findings: Comparison 7 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP plus deferasirox (DFX) | RR 0.84 [0.72, 0.99] | 1 study · 96 participants | Low Downgraded: risk of bias, imprecision, indirectness |
Incidence of SAEs Summary of findings: Comparison 7 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP plus deferasirox (DFX) | 1 [0.06, 16.22] | 1 study · 96 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
All‐cause mortality ‐ at 1 year ‐ trial end Summary of findings: Comparison 7 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP plus deferasirox (DFX) | RD 0 | 1 study · 96 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
Sustained adherence Summary of findings: Comparison 7 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP plus deferasirox (DFX) | no estimate extracted | NR | No studies |
QoL Summary of findings: Comparison 7 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP plus deferasirox (DFX) | no estimate extracted | 1 study · 96 participants | Ungraded |
Summary of findings: Comparison 8 ‐ medication management versus standard care
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Adherence to iron chelation Summary of findings: Comparison 8 ‐ medication management vs standard care | no estimate extracted | NR | No studies |
SAEs Summary of findings: Comparison 8 ‐ medication management vs standard care | no estimate extracted | NR | No studies |
Mortality Summary of findings: Comparison 8 ‐ medication management vs standard care | no estimate extracted | NR | No studies |
Sustained adherence Summary of findings: Comparison 8 ‐ medication management vs standard care | no estimate extracted | NR | No studies |
QoL PedsQLTM total score Summary of findings: Comparison 8 ‐ medication management vs standard care | no estimate extracted | 1 study · 48 participants | Very low Downgraded: risk of bias, indirectness |
DFP compared to DFO for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia
Population: improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Adherence to iron chelation therapy (per cent, SD) DFP vs DFO | no estimate extracted | 4 studies · 242 participants | Very low Downgraded: risk of bias, inconsistency |
SAEs (from therapy, disease, non‐adherence) Agranulocytosis** DFP vs DFO | RR 7.88 | 1 study · 88 participants | Very low Downgraded: imprecision, indirectness |
All‐cause mortality DFP vs DFO | RR 0.44 [0.12, 1.63] | 1 study · 88 participants | Very low Downgraded: imprecision, indirectness |
Sustained adherence ‐ not measured DFP vs DFO | no estimate extracted | NR | No studies |
Quality of life ‐ not reported DFP vs DFO | no estimate extracted | NR | No studies |
DFX compared to DFO for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia
Population: improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Adherence to iron chelation therapy (per cent, SD) DFX vs DFO | no estimate extracted | 1 study · 197 participants | Very low Downgraded: risk of bias, imprecision |
SAEs ‐ thalassaemia‐related SAEs DFX vs DFO | no estimate extracted | 2 studies · 247 participants | Very low Downgraded: risk of bias, imprecision |
SAEs ‐ SCD‐related SAEs DFX vs DFO | RR 1.08 [0.77, 1.51] | 1 study · 195 participants | Very low Downgraded: risk of bias, imprecision |
Incidence of SCD‐related SAEs ‐pain crisis DFX vs DFO | RR 1.05 [0.68, 1.62] | 1 study · 195 participants | Very low Downgraded: risk of bias, imprecision |
All‐cause mortality (thalassaemia) DFX vs DFO | RR 0.96 [0.06, 15.06] | 2 studies · 240 participants | Very low Downgraded: risk of bias, imprecision |
Sustained adherence ‐ not measured DFX vs DFO | no estimate extracted | NR | No studies |
Quality of life ‐ not reported DFX vs DFO | no estimate extracted | NR | No studies |
DFX film‐coated tablet compared to DFX dispersible tablet for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia
Population: improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Adherence to iron chelation therapy (n, N) DFX film‐coated tablet vs DFX dispersible tablet | RR 1.1 [0.99, 1.22] | 1 study · 173 participants | Low Downgraded: risk of bias |
Incidence of SAEs DFX film‐coated tablet vs DFX dispersible tablet | RR 1.22 [0.62, 2.37] | 1 study · 173 participants | Very low Downgraded: risk of bias, imprecision |
All‐cause mortality DFX film‐coated tablet vs DFX dispersible tablet | RR 2.97 [0.12, 71.81] | 1 study · 173 participants | Very low Downgraded: risk of bias, imprecision |
Sustained adherence ‐ not measured DFX film‐coated tablet vs DFX dispersible tablet | no estimate extracted | NR | No studies |
Quality of life ‐ not reported DFX film‐coated tablet vs DFX dispersible tablet | no estimate extracted | NR | No studies |
DFP and DFO compared to DFP for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia
Population: improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Adherence to iron chelation therapy (per cent, SD) DFP and DFO vs DFP | no estimate extracted | 3 studies · 289 participants | Very low Downgraded: risk of bias |
Incidence of SAEs DFP and DFO vs DFP | RR 0.15 [0.01, 2.81] | 1 study · 213 participants | Low Downgraded: risk of bias, imprecision |
All‐cause mortality DFP and DFO vs DFP | RR 0.77 [0.18, 3.35] | 2 studies · 237 participants | Very low Downgraded: risk of bias, imprecision |
Sustained adherence ‐ not measured DFP and DFO vs DFP | no estimate extracted | NR | No studies |
Quality of life ‐ not reported DFP and DFO vs DFP | no estimate extracted | NR | No studies |
DFP and DFO compared to DFO for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia
Population: improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Adherence to iron chelation therapy (per cent, SD) DFP and DFO vs DFO | no estimate extracted | 4 studies · 205 participants | Low Downgraded: risk of bias |
Incidence of SAEs DFP and DFO vs DFO | no estimate extracted | 4 studies · 205 participants | Low Downgraded: risk of bias |
All‐cause mortality DFP and DFO vs DFO | no estimate extracted | 4 studies · 205 participants | Low Downgraded: risk of bias |
Sustained adherence ‐ not measured DFP and DFO vs DFO | no estimate extracted | NR | No studies |
Quality of life ‐ not reported DFP and DFO vs DFO | no estimate extracted | NR | No studies |
DFP and DFO compared to DFP and DFX for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia
Population: improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Adherence to iron chelation therapy rates (n,N) ‐ 1 year DFP and DFO vs DFP and DFX | RR 0.84 [0.72, 0.99] | 1 study · 96 participants | Low Downgraded: risk of bias, indirectness |
Incidence of SAE DFP and DFO vs DFP and DFX | RR 1 [0.06, 15.53] | 1 study · 96 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
All‐cause mortality ‐ at 1 year ‐ trial end DFP and DFO vs DFP and DFX | no estimate extracted | 1 study · 96 participants | Low Downgraded: risk of bias, indirectness |
Sustained adherence ‐ not measured DFP and DFO vs DFP and DFX | no estimate extracted | NR | No studies |
Quality of life see comment DFP and DFO vs DFP and DFX | no estimate extracted | NR | No studies |
Medication management compared to standard care for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia
Population: improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Adherence to iron chelation therapy ‐ not reported Medication management vs standard care | no estimate extracted | NR | No studies |
SAEs ‐ not reported Medication management vs standard care | no estimate extracted | NR | No studies |
Mortality ‐ not reported Medication management vs standard care | no estimate extracted | NR | No studies |
Sustained adherence Medication management vs standard care | no estimate extracted | NR | No studies |
Quality of life Medication management vs standard care | no estimate extracted | 1 study · 48 participants | Very low Downgraded: risk of bias, indirectness |