Findings

What do systematic reviews say?

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Evidence found and GRADE certainty

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

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%

Psychological interventions vs usual care

Treatment‐related adverse events Depression PHQ‐8 (Kroenke 2009) · children, adolescents or adults with CF

Summary of findings: psychological interventions showed no clear difference in treatment‐related adverse events Depression PHQ‐8 (Kroenke 2009) compared with usual care (low certainty).

1 finding from 1 review

Summary of findings ‐ Hydroxyurea and phlebotomy vs transfusion and chelation

All‐cause mortality · adults and children with sickle cell disease and an increased risk of stroke

Summary of findings ‐ Hydroxyurea and phlebotomy showed no clear difference in death during the study (all deaths) compared with transfusion and chelation (low certainty).

1 finding from 1 review

Summary of findings ‐ Hydroxyurea and phlebotomy vs transfusion and chelation

Pain alteration: proportion experiencing serious VOC or sickle‐related pain events · adults and children with sickle cell disease and an increased risk of stroke

Summary of findings ‐ Hydroxyurea and phlebotomy may leads to higher pain alteration: proportion experiencing serious VOC or… compared with transfusion and chelation (low certainty).

1 finding from 1 review

Recombinant human growth hormone vs placebo or no intervention

Measures of mineral metabolism: change in serum phosphate mmol/L · children with X‐linked hypophosphataemia

It is very uncertain whether summary of findings ‐ recombinant growth hormone changes measures of mineral metabolism: change in serum phosphate mmol/L compared with placebo or no intervention (very low certainty).

1 finding from 1 review

Recombinant human growth hormone vs placebo or no intervention

Measures of renal function: change in TmP/GFR · children with X‐linked hypophosphataemia

It is very uncertain whether summary of findings ‐ recombinant growth hormone changes measures of renal function: change in TmP/GFR compared with placebo or no intervention (very low certainty).

1 finding from 1 review

Recombinant human growth hormone vs placebo or no intervention

Body proportions: sitting height SDS · children with X‐linked hypophosphataemia

Summary of findings ‐ recombinant growth hormone showed no clear difference in body proportions: sitting height SDS compared with placebo or no intervention (moderate certainty).

1 finding from 1 review