What do systematic reviews say?

2,261 questions · 2,280 findings · 114 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 856 · 37.5%GRADE not reported 58 · 2.5%Very low 550 · 24.1%Low 502 · 22%Moderate 271 · 11.9%High 43 · 1.9%

Deferasirox vs deferoxamine

Adverse event: Isolated serum creatinine increase above upper limit of normal · people with transfusion‐dependent thalassemia

Deferasirox may leads to higher AE: investigations ‐ isolated serum creatinine increase above ULN compared with deferoxamine (low certainty).

1 finding from 1 review

Deferasirox for managing iron overload in people with thalassaemia 2017 · Cochrane Headline outcomes only
RR 2.57 [1.88, 3.51]
2 studies · 657 participants
Low

Deferasirox vs deferiprone

Mortality at any time point · people with transfusion‐dependent thalassemia

1 finding from 1 review

Deferasirox for managing iron overload in people with thalassaemia 2017 · Cochrane Headline outcomes only
no estimate extracted
3 studies · 146 participants
Very low

Deferasirox vs deferiprone

Responder analysis · people with transfusion‐dependent thalassemia

The review found no eligible studies for this question.

1 finding from 1 review

Deferasirox for managing iron overload in people with thalassaemia 2017 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Any prophylaxis vs on‐demand therapy with FEIBA

Overall bleeding rates: total number of bleeding events · adults and children with hemophilia A or B with inhibitors

1 finding from 1 review

Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors 2017 · Cochrane Headline outcomes only
no estimate extracted
1 study · 26 participants
Low

High‐dose vs low‐dose rFVIIa

Annualized joint Bleeding Rate (AjBR) · adults and children with hemophilia A or B with inhibitors

The review found no eligible studies for this question.

1 finding from 1 review

Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors 2017 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

High‐dose vs low‐dose rFVIIa

Quality of life · adults and children with hemophilia A or B with inhibitors

The review found no eligible studies for this question.

1 finding from 1 review

Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors 2017 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

High‐dose vs low‐dose rFVIIa

Safety of bypassing agents · adults and children with hemophilia A or B with inhibitors

1 finding from 1 review

Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors 2017 · Cochrane Headline outcomes only
no estimate extracted
2 studies · 46 participants
Moderate

Any prophylaxis vs on‐demand therapy with FEIBA

Annualised bleeding rate · adults and children with hemophilia A or B with inhibitors

1 finding from 1 review

Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors 2017 · Cochrane Headline outcomes only
no estimate extracted
1 study · 34 participants
Moderate

Any prophylaxis vs on‐demand therapy with FEIBA

Target joint bleeding rate · adults and children with hemophilia A or B with inhibitors

1 finding from 1 review

Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors 2017 · Cochrane Headline outcomes only
no estimate extracted
1 study · 26 participants
Low

Any prophylaxis vs on‐demand therapy with FEIBA

Annualized joint Bleeding Rate (AjBR) · adults and children with hemophilia A or B with inhibitors

1 finding from 1 review

Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors 2017 · Cochrane Headline outcomes only
no estimate extracted
1 study · 34 participants
Moderate

Any prophylaxis vs on‐demand therapy with FEIBA

Quality of life · adults and children with hemophilia A or B with inhibitors

1 finding from 1 review

Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors 2017 · Cochrane Headline outcomes only
no estimate extracted
2 studies · 58 participants
Low

Any prophylaxis vs on‐demand therapy with FEIBA

Safety of bypassing agents · adults and children with hemophilia A or B with inhibitors

1 finding from 1 review

Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors 2017 · Cochrane Headline outcomes only
no estimate extracted
2 studies · 67 participants
Low

High‐dose vs low‐dose rFVIIa

Number of total bleeds per year or bleeding frequency · adults and children with hemophilia A or B with inhibitors

1 finding from 1 review

Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors 2017 · Cochrane Headline outcomes only
no estimate extracted
1 study · 22 participants
Moderate

High‐dose vs low‐dose rFVIIa

Annualised bleeding rate · adults and children with hemophilia A or B with inhibitors

1 finding from 1 review

Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors 2017 · Cochrane Headline outcomes only
no estimate extracted
1 study · 24 participants
GRADE not reported

High‐dose vs low‐dose rFVIIa

Target joint bleeding rate · adults and children with hemophilia A or B with inhibitors

1 finding from 1 review

Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors 2017 · Cochrane Headline outcomes only
no estimate extracted
1 study · 22 participants
Moderate

Intervention not reported

Cardiac function · infantile‐onset Pompe disease

1 finding from 1 review

Enzyme replacement therapy for infantile‐onset Pompe disease 2017 · Cochrane Headline outcomes only
no estimate extracted
1 study · 18 participants
Low

Intervention not reported

Time to ventilation · infantile‐onset Pompe disease

1 finding from 1 review

Enzyme replacement therapy for infantile‐onset Pompe disease 2017 · Cochrane Headline outcomes only
no estimate extracted
1 study · 18 participants
Low

Intervention not reported

Survival · infantile‐onset Pompe disease

1 finding from 1 review

Enzyme replacement therapy for infantile‐onset Pompe disease 2017 · Cochrane Headline outcomes only
61
1 study · 18 participants
Low

Intervention not reported

Motor development · infantile‐onset Pompe disease

1 finding from 1 review

Enzyme replacement therapy for infantile‐onset Pompe disease 2017 · Cochrane Headline outcomes only
no estimate extracted
1 study · 18 participants
Low

Intervention not reported

Quality of life · infantile‐onset Pompe disease

1 finding from 1 review

Enzyme replacement therapy for infantile‐onset Pompe disease 2017 · Cochrane Headline outcomes only
no estimate extracted
1 study · 18 participants
GRADE not reported