Findings

What do systematic reviews say?

873 questions · 883 findings · 38 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 319 · 36.1%GRADE not reported 18 · 2%Very low 198 · 22.4%Low 186 · 21.1%Moderate 147 · 16.6%High 15 · 1.7%

Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
5 studies · 944 participants
Low

Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone

FEV1 % predicted: absolute change from baseline · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
5 studies · 944 participants
Low

Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone

Adverse events: most commonly occurring events (occurring in at least 10% of participants) · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
8 studies · 1057 participants
Moderate

Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone

Time to first pulmonary exacerbation · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 506 participants
Moderate

Relaxation vs no treatment

Mood or personal well‐being · adults and children with hemophilia

The review found no eligible studies for this question.

1 finding from 1 review

Psychological interventions for people with hemophilia 2020 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Relaxation vs no treatment

Coping strategies · adults and children with hemophilia

The review found no eligible studies for this question.

1 finding from 1 review

Psychological interventions for people with hemophilia 2020 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Relaxation vs no treatment

QoL · adults and children with hemophilia

The review found no eligible studies for this question.

1 finding from 1 review

Psychological interventions for people with hemophilia 2020 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Relaxation vs no treatment

Compliance or adherence to the trial intervention (including satisfaction for it) · adults and children with hemophilia

1 finding from 1 review

Psychological interventions for people with hemophilia 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 7 participants
Very low

Relaxation vs no treatment

Physical health: Reduction of bleedings, factor replacement, pain · adults and children with hemophilia

1 finding from 1 review

Psychological interventions for people with hemophilia 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 7 participants
Very low

Relaxation vs no treatment

Cost of psychological intervention · adults and children with hemophilia

The review found no eligible studies for this question.

1 finding from 1 review

Psychological interventions for people with hemophilia 2020 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Relaxation vs no treatment

Family adjustment · adults and children with hemophilia

The review found no eligible studies for this question.

1 finding from 1 review

Psychological interventions for people with hemophilia 2020 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Pregabalin vs placebo

Self‐reported pain relief ‐ NPSI: ranges from 0 to 100. Higher scores are indicative of neuropathic pain. · adults and children with sickle cell disease experiencing neuropathic pain

1 finding from 1 review

Interventions for treating neuropathic pain in people with sickle cell disease 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 16 participants
Very low

Self‐reported pain relief ‐ S‐LANSS: Possible total scores range from 0 to 24, and a score of 12 or higher is indicative of neuropathic pain. · adults and children with sickle cell disease experiencing neuropathic pain

1 finding from 1 review

Interventions for treating neuropathic pain in people with sickle cell disease 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 16 participants
Very low

Pregabalin vs placebo

Patient Global Impression of Change · adults and children with sickle cell disease experiencing neuropathic pain

The review found no eligible studies for this question.

1 finding from 1 review

Interventions for treating neuropathic pain in people with sickle cell disease 2019 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Pregabalin vs placebo

Time to improvement of symptoms · adults and children with sickle cell disease experiencing neuropathic pain

The review found no eligible studies for this question.

1 finding from 1 review

Interventions for treating neuropathic pain in people with sickle cell disease 2019 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Pregabalin vs placebo

QoL · adults and children with sickle cell disease experiencing neuropathic pain

1 finding from 1 review

Interventions for treating neuropathic pain in people with sickle cell disease 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 16 participants
Very low

Pregabalin vs placebo

Quality of sleep · adults and children with sickle cell disease experiencing neuropathic pain

The review found no eligible studies for this question.

1 finding from 1 review

Interventions for treating neuropathic pain in people with sickle cell disease 2019 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Pregabalin vs placebo

Treatment‐related adverse effects · adults and children with sickle cell disease experiencing neuropathic pain

It is very uncertain whether summary of findings ‐ Pharmacological intervention (pregabalin) changes treatment‐related adverse effects compared with placebo (very low certainty).

1 finding from 1 review

Interventions for treating neuropathic pain in people with sickle cell disease 2019 · Cochrane Headline outcomes only
RR 1.33
1 study · 22 participants
Very low

Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo

Survival · adults with cystic fibrosis and with the F508del/MF genotype

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 55 participants
Low

Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo

Quality of life: total score · adults with cystic fibrosis and with the F508del/MF genotype

The review found no eligible studies for this question.

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found