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CFTR modulator use and risk of nontuberculous mycobacteria positivity in cystic fibrosis, 2011–2018

Emily E. Ricotta, D. Rebecca Prevots, Kenneth N. Olivier
ERJ Open Research 2022 8: 00724-2021; DOI: 10.1183/23120541.00724-2021
Emily E. Ricotta
1Epidemiology and Population Studies Unit, Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health (NIH), Bethesda, MD, USA
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  • For correspondence: emily.ricotta@nih.gov
D. Rebecca Prevots
1Epidemiology and Population Studies Unit, Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health (NIH), Bethesda, MD, USA
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Kenneth N. Olivier
2Pulmonary Branch, National Heart, Lung, and Blood Institute, NIH, Bethesda, MD, USA
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  • FIGURE 1
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    FIGURE 1

    Flowchart depicting inclusion criteria for cystic fibrosis transmembrane conductance regulator (CFTR) modulator analysis from the Cystic Fibrosis Foundation Patient Registry (CFFPR), 2011–2018. These data included only individuals aged ≥12 years. NTM: nontuberculous mycobacteria; M. tuberculosis: Mycobacterium tuberculosis.

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    FIGURE 2

    Kaplan–Meier plots and 95% confidence intervals evaluating the event probability of patients having incident nontuberculous mycobacteria (NTM) cultures. a) Probability of incident NTM comparing receipt of any cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy to nonreceipt of CFTR modulator therapy. b) Probability of incident NTM comparing receipt of ivacaftor monotherapy, combination therapy and no modulator therapy.

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  • TABLE 1

    Individual and encounter-level characteristics at baseline and censoring time

    BaselineCensored#
    Gender
     Male9137 (52.5)
     Female8266 (47.5)
    Mutation category¶
     Class 14062 (23.3)
     Class 215043 (86.4)
     Class 31007 (5.8)
     Class 41068 (6.1)
     Class 51120 (6.4)
     Unknown668 (3.8)
     F508del only7827 (45.0)
    Least severe mutation
     Class 1/213614 (78.2)
     Class 3950 (5.5)
     Class 4/52171 (12.5)
     Unknown668 (3.8)
    Age
     12 to <18 years6554 (37.7)3694 (21.2)
     ≥18 years10849 (62.3)13709 (78.8)
    FEV1pp
     Normal3923 (22.5)4313 (24.8)
     Mild4840 (27.8)5125 (29.4)
     Moderate4959 (28.5)5597 (32.2)
     Severe1210 (6.9)2256 (13.0)
     Unknown2471 (14.2)112 (0.6)
    BMI
     Underweight1135 (6.5)1282 (7.4)
     Normal11517 (66.2)10940 (62.9)
     Overweight2345 (13.5)2852 (16.4)
     Obese839 (4.8)1045 (6.0)
     Unknown1567 (9.0)1284 (7.4)
    Chronic macrolides7620 (43.8)8192 (47.1)
    NTM positive4077 (23.4)
     Culture positive for MAB only1203 (6.9)
     Culture positive for MAC only2066 (11.9)
     Culture positive for other species only741 (4.3)
     Culture positive for multiple species67 (0.4)
    Any modulator therapy7384 (42.4)
     Ever received ivacaftor monotherapy1893 (10.9)
     Ever received combination therapy5869 (33.7)
     Neither10019 (57.6)

    Data are presented as n (%). FEV1pp: percentage predicted forced expiratory volume in 1 s; BMI: body mass index; NTM: nontuberculous mycobacteria; MAB: Mycobacterium abscessus; MAC: Mycobacterium avium complex. #: censor: NTM-positive culture or patient's last time point in dataset; ¶: ≥1.

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      Supplementary material 00724-2021.SUPPLEMENT

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    CFTR modulator use and risk of nontuberculous mycobacteria positivity in cystic fibrosis, 2011–2018
    Emily E. Ricotta, D. Rebecca Prevots, Kenneth N. Olivier
    ERJ Open Research Apr 2022, 8 (2) 00724-2021; DOI: 10.1183/23120541.00724-2021

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    CFTR modulator use and risk of nontuberculous mycobacteria positivity in cystic fibrosis, 2011–2018
    Emily E. Ricotta, D. Rebecca Prevots, Kenneth N. Olivier
    ERJ Open Research Apr 2022, 8 (2) 00724-2021; DOI: 10.1183/23120541.00724-2021
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