Last updated March 15, 2022

Use of Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy in Patients With Cystic Fibrosis

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. (, )
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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)
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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)
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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)
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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)
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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)
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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)
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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)
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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)
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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)
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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)
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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)
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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)
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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)
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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)
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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)
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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)
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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)
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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)
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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)
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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. (, )
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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)
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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)
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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)
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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)
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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)
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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)
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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)
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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)
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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)
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Recommendation Grading

Overview

Title

Use of Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy in Patients With Cystic Fibrosis

Authoring Organization

Endorsing Organization

Publication Month/Year

November 18, 2017

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