Use of Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy in Patients With Cystic Fibrosis
Publication Date: November 18, 2017
Last Updated: March 14, 2022
Recommendations
Ivacaftor for gating mutations other than G551D or R117H
The committee recommends IVA for individuals aged 2–5 years with a diagnosis of CF and gating mutations other than G551D or R117H. For individuals under 2 years of age, the committee makes no recommendation. (, )
322286
The committee suggests IVA for individuals aged 6–11 years with a diagnosis of CF with PPFEV1 less than 40% and a gating mutation other than G551D or R117H. (C, VL)
322286
The committee suggests IVA treatment for individuals aged 6–11 years with a diagnosis of CF with PPFEV1 40%–90% and a gating mutation other than G551D or R117H. (C, L)
322286
The committee suggests IVA be used for individuals aged 6–11 years with a diagnosis of CF with PPFEV1 greater than 90% and a gating mutation other than G551D or R117H. (C, L)
322286
The committee suggests IVA for individuals aged 12–17 years with a diagnosis of CF with PPFEV1 40%–90% and a gating mutation other than G551D or R117H. (C, L)
322286
The committee suggests IVA for individuals aged 12–17 years with a diagnosis of CF with PPFEV1 greater than 90% and a gating mutation other than G551D or R117H. (C, M)
322286
The committee suggests IVA for individuals aged 18 years or older with a diagnosis of CF with PPFEV1 less than 40% and a gating mutation other than G551D or R117H. (C, M)
322286
The committee suggests IVA for individuals with a diagnosis of CF aged 18 years or older with PPFEV1 40%–90% and a gating mutation G551D or R117H. (C, L)
322286
The committee suggests IVA for individuals aged 18 years or older with a diagnosis of CF with PPFEV1 greater than 90% and a gating mutation G551D or R117H. (C, M)
322286
The committee suggests IVA for individuals aged 12–17 years with a diagnosis of CF with PPFEV1 less than 40% and a gating mutation other than G551D or R117H. (C, M)
322286
Ivacaftor for the R117H mutation
The committee suggests against IVA therapy for individuals aged 0–5 years and with a CF diagnosis due to the R117H mutation. (C, VL)
(against)
322286
The committee suggests IVA for individuals aged 6–11 years with PPFEV1 less than 40% with a diagnosis of CF due to the R117H mutation. (C, VL)
322286
The committee suggests IVA treatment for individuals aged 6–11 years with PPFEV1 40%–90% with a diagnosis of CF due to the R117H mutation. (C, VL)
322286
The committee suggests that IVA not be used for individuals aged 6–11 years with PPFEV1 greater than 90% with a diagnosis of CF due to the R117H mutation. (C, L)
(against)
322286
The committee suggests IVA for individuals aged 12–17 years with PPFEV1 less than 40% with a diagnosis of CF due to the R117H mutation. (C, VL)
322286
The committee suggests IVA for individuals aged 12–17 years with PPFEV1 40%–90% with a diagnosis of CF due to the R117H mutation. (C, VL)
322286
The committee suggests against IVA for individuals aged 12–17 years with PPFEV1 greater than 90% with a diagnosis of CF due to the R117H mutation. (C, VL)
(against)
322286
The committee suggests IVA for individuals aged 18 years or older with PPFEV1 less than 40% with a diagnosis of CF due to the R117H mutation. (C, VL)
322286
The committee suggests IVA for individuals aged 18 years or older with PPFEV1 40%–90% with a diagnosis of CF due to the R117H mutation. (C, M)
322286
The committee suggests IVA for individuals aged 18 years or older with PPFEV1 greater than 90% with a diagnosis of CF due to the R117H mutation. (C, L)
322286
Ivacaftor/lumacaftor for two copies of F508del
The committee makes no recommendation for or against IVA/LUM combination therapy for individuals with a diagnosis of CF and two copies of the F508del mutation who are aged 0–5 years. (, )
322286
The committee suggests IVA/LUM combination therapy for individuals with a diagnosis of CF and two copies of the F508del mutation who are aged 6–11 years with PPFEV1 less than 40%. (C, VL)
322286
The committee suggests IVA/LUM combination therapy for individuals aged 6–11 years with a diagnosis of CF and two copies of the F508del mutation with PPFEV1 40%–90%. (C, VL)
322286
The committee suggests IVA/LUM combination therapy for individuals aged 6–11 years with a diagnosis of CF and two copies of the F508del mutation with PPFEV1 greater than 90%. (C, VL)
322286
The committee suggests IVA/LUM combination therapy for individuals aged 12–17 years with a diagnosis of CF and two copies of the F508del mutation with PPFEV1 less than 40%. (S, M)
322286
The committee suggests IVA/LUM combination therapy for individuals aged 12–17 years with a diagnosis of CF and two copies of the F508del mutation with PPFEV1 40%–90%. (S, M)
322286
The committee suggests IVA/LUM combination therapy for individuals with a diagnosis of CF and two copies of the F508del mutation who are aged 12–17 years with PPFEV1 greater than 90%. (C, L)
322286
The committee suggests IVA/LUM combination therapy for individuals aged 18 years or older with a diagnosis of CF and two copies of the F508del mutation with PPFEV1 less than 40%. (S, M)
322286
The committee suggests IVA/LUM combination therapy for individuals aged 18 years or older with a diagnosis of CF and two copies of the F508del mutation with PPFEV1 40%–90%. (S, M)
322286
The committee suggests IVA/LUM combination therapy for individuals aged 18 years or older with a diagnosis of CF and two copies of the F508del mutation with PPFEV1 greater than 90%. (C, L)
322286
Recommendation Grading
Overview
Title
Use of Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy in Patients With Cystic Fibrosis
Authoring Organization
Cystic Fibrosis Foundation
Endorsing Organization
American Thoracic Society
Publication Month/Year
November 18, 2017
Last Updated Month/Year
August 2, 2022
Document Type
Guideline
External Publication Status
Published
Country of Publication
US
Document Objectives
Develop evidence-based guidelines for CFTR modulator therapy in patients with CF
Inclusion Criteria
Female, Male, Adolescent, Adult, Child, Older adult
Health Care Settings
Ambulatory, Hospital, Outpatient
Intended Users
Respiratory therapist, nurse, nurse practitioner, physician, physician assistant
Scope
Management, Treatment
Diseases/Conditions (MeSH)
D003550 - Cystic Fibrosis
Keywords
cystic fibrosis, CFTR, Modulator therapy