What do systematic reviews say?

Summary of findings ‐ monotherapy: lumacaftor vs placebo

• 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
  2 studies · 147 participants
  Low

Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo

• 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
  2 studies · 1108 participants
  High

Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo

• 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 · 204 participants
  Moderate

Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo

• 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 · 62 participants
  Moderate

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

• 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
  9 studies · 1132 participants
  Low

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

• 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‐152‐tezacaftor‐ivacaftor vs placebo

• 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 · 76 participants
  Low

Summary of findings ‐ monotherapy: cavosonstat vs placebo

• 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 · 26 participants
  Very low

Summary of findings: continuous cycles alternating aztreonam lysine for inhalation with tobramycin for inhalation solution vs continuous cycles alternating placebo with tobramycin

The review found no eligible studies for this question.

• Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
  no estimate extracted
  Studies not reported
  No studies found

Summary of findings ‐ ivacaftor vs placebo

• Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
  no estimate extracted
  1 study · 140 participants
  GRADE not reported

Summary of findings ‐ ivacaftor vs placebo

• Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
  no estimate extracted
  1 study · 69 participants
  GRADE not reported

Summary of findings ‐ ivacaftor vs placebo

• Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
  no estimate extracted
  3 studies · 238 participants
  GRADE not reported

Summary of findings: anti‐pseudomonal antibiotics vs placebo

Summary of findings: anti‐pseudomonal antibiotics showed no clear difference in survival: number of deaths compared with placebo (low certainty).

• Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
  RR 0.17 [0.03, 1.09]
  2 studies · 767 participants
  Low

Propionyl‐L‐carnitine vs placebo

The review found no eligible studies for this question.

• Interventions for treating leg ulcers in people with sickle cell disease 2021 · Cochrane Headline outcomes only
  no estimate extracted
  Studies not reported
  No studies found

RGD peptide matrix vs placebo

The review found no eligible studies for this question.

• Interventions for treating leg ulcers in people with sickle cell disease 2021 · Cochrane Headline outcomes only
  no estimate extracted
  Studies not reported
  No studies found

Isoxsuprine vs placebo

The review found no eligible studies for this question.

• Interventions for treating leg ulcers in people with sickle cell disease 2021 · Cochrane Headline outcomes only
  no estimate extracted
  Studies not reported
  No studies found