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 ‐ 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 ‐ 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 ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)

• 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 · 258 participants
  Moderate

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 ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor vs control

• 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
  4 studies · 634 participants
  Moderate

Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control

• 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
  3 studies · 603 participants
  High

Summary of findings: inhaled TOBI® (IV preparation) vs 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: aztreonam lysine for inhalation vs tobramycin

• Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
  no estimate extracted
  1 study · 268 participants
  Low

Summary of findings: amikacin liposome inhalation suspension (ALIS) vs 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: levofloxacin for inhalation solution vs 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: 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

Intervention not reported

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

Summary of findings: colistimethate dry powder for inhalation (Colobreathe®) vs tobramycin

Summary of findings: colistimethate dry powder for inhalation… showed no clear difference in survival: number of deaths compared with tobramycin (low certainty).

• Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
  RR 0.21 [0.01, 4.32]
  1 study · 374 participants
  Low

Summary of findings: tobramycin for inhalation powder vs tobramycin

• Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
  4.76 [0.25, 91.62]
  1 study · 517 participants
  Low

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