Lung (2014) 192:823–824 DOI 10.1007/s00408-014-9629-y
CT Imaging of Pediatric Patients with Cystic Fibrosis on Ivacaftor Therapy Don Hayes Jr. • Frederick R. Long • Karen S. McCoy • Shahid I. Sheikh
Received: 6 May 2014 / Accepted: 16 July 2014 / Published online: 7 August 2014 Ó Springer Science+Business Media New York 2014
Correction of the gating defect of the cystic fibrosis transmembrane conductance regulator (CFTR) channel through the new drug ivacaftor has significantly improved the clinical course of patients with cystic fibrosis (CF) with G551D mutation. The majority of the studies investigating the impact of ivacaftor have focused on improvement related to pulmonary function and growth. We present two pediatric CF patients with DF508/G551D genotype, who had significant improvement on computed tomography (CT) imaging of the chest. Two 14-year-old children started ivacaftor within a week of each other (1 female, 1 male). There was no other alteration in their medical care. Figure 1 illustrates the improvement of airway mucosal thickening in the female patient, while Figure 2 shows the improvement in mucus plugging in the male patient 16 months after starting ivacaftor in both cases. The sweat chloride levels decreased to
D. Hayes Jr. K. S. McCoy S. I. Sheikh Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, USA D. Hayes Jr. Department of Internal Medicine, The Ohio State University College of Medicine, Columbus, OH, USA
34 from 101 mmol/L and to 27 from 107 mmol/L, respectively. Body mass index increased from 16.2 to 21.2 kg/m2 and from 17.7 to 19.6 after 2 years of therapy, respectively. For the female patient, spirometry showed improvement in forced vital capacity (FVC) from 2.19 L (105 % predicted) to 3.89 L (118 % predicted) and forced expiratory volume in one second (FEV1) from 1.84 L (98 % predicted) to 3.06 L (105 % predicted). For the male patient, spirometry demonstrated improvement in FVC from 2.47 L (59 % predicted) to 3.32 L (64 % predicted) and FEV1 from 1.36 L (38 % predicted) to 1.76 L (41 % predicted). Respiratory cultures continued to isolate methicillin-susceptible Staphylococcus aureus for the female patient and Achromobacter xylosoxidans and methicillin-susceptible S. aureus for the male patient. Based on our experience with these two patients, ivacaftor is effective in the treatment of CF patients with CFTR-G551D mutation. Pulmonary function significantly improved in one patient while not in the other. Despite no alteration in pulmonary function in the male patient, CT scan of the chest clearly showed an improvement with ivacaftor therapy. CT imaging of the chest is a useful tool to evaluate response to ivacaftor in children with CF having CFTR-G551D mutation.
D. Hayes Jr. (&) K. S. McCoy S. I. Sheikh Sections of Pulmonary Medicine, Nationwide Children’s Hospital, 700 Children’s Drive, Columbus, OH 43205, USA e-mail: [email protected] F. R. Long Department of Radiology, The Ohio State University College of Medicine, Columbus, OH, USA F. R. Long Department of Radiology, Nationwide Children’s Hospital, Columbus, OH, USA
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824 Fig. 1 Computed tomography of the chest without contrast demonstrating alterations in airway mucosal thickening in the female patient one month before a compared to 16 months after b starting ivacaftor therapy
Fig. 2 Computed tomography of the chest without contrast demonstrating alterations in mucus plugging in the male patient 6 months before a compared to 16 months after b starting ivacaftor therapy
Conflict of interest The authors report no conflict of interest and have no relevant financial disclosures.
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Lung (2014) 192:823–824
CT Imaging of Pediatric Patients with Cystic Fibrosis on Ivacaftor Therapy Don Hayes Jr. • Frederick R. Long • Karen S. McCoy • Shahid I. Sheikh
Received: 6 May 2014 / Accepted: 16 July 2014 / Published online: 7 August 2014 Ó Springer Science+Business Media New York 2014
Correction of the gating defect of the cystic fibrosis transmembrane conductance regulator (CFTR) channel through the new drug ivacaftor has significantly improved the clinical course of patients with cystic fibrosis (CF) with G551D mutation. The majority of the studies investigating the impact of ivacaftor have focused on improvement related to pulmonary function and growth. We present two pediatric CF patients with DF508/G551D genotype, who had significant improvement on computed tomography (CT) imaging of the chest. Two 14-year-old children started ivacaftor within a week of each other (1 female, 1 male). There was no other alteration in their medical care. Figure 1 illustrates the improvement of airway mucosal thickening in the female patient, while Figure 2 shows the improvement in mucus plugging in the male patient 16 months after starting ivacaftor in both cases. The sweat chloride levels decreased to
D. Hayes Jr. K. S. McCoy S. I. Sheikh Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, USA D. Hayes Jr. Department of Internal Medicine, The Ohio State University College of Medicine, Columbus, OH, USA
34 from 101 mmol/L and to 27 from 107 mmol/L, respectively. Body mass index increased from 16.2 to 21.2 kg/m2 and from 17.7 to 19.6 after 2 years of therapy, respectively. For the female patient, spirometry showed improvement in forced vital capacity (FVC) from 2.19 L (105 % predicted) to 3.89 L (118 % predicted) and forced expiratory volume in one second (FEV1) from 1.84 L (98 % predicted) to 3.06 L (105 % predicted). For the male patient, spirometry demonstrated improvement in FVC from 2.47 L (59 % predicted) to 3.32 L (64 % predicted) and FEV1 from 1.36 L (38 % predicted) to 1.76 L (41 % predicted). Respiratory cultures continued to isolate methicillin-susceptible Staphylococcus aureus for the female patient and Achromobacter xylosoxidans and methicillin-susceptible S. aureus for the male patient. Based on our experience with these two patients, ivacaftor is effective in the treatment of CF patients with CFTR-G551D mutation. Pulmonary function significantly improved in one patient while not in the other. Despite no alteration in pulmonary function in the male patient, CT scan of the chest clearly showed an improvement with ivacaftor therapy. CT imaging of the chest is a useful tool to evaluate response to ivacaftor in children with CF having CFTR-G551D mutation.
D. Hayes Jr. (&) K. S. McCoy S. I. Sheikh Sections of Pulmonary Medicine, Nationwide Children’s Hospital, 700 Children’s Drive, Columbus, OH 43205, USA e-mail: [email protected] F. R. Long Department of Radiology, The Ohio State University College of Medicine, Columbus, OH, USA F. R. Long Department of Radiology, Nationwide Children’s Hospital, Columbus, OH, USA
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824 Fig. 1 Computed tomography of the chest without contrast demonstrating alterations in airway mucosal thickening in the female patient one month before a compared to 16 months after b starting ivacaftor therapy
Fig. 2 Computed tomography of the chest without contrast demonstrating alterations in mucus plugging in the male patient 6 months before a compared to 16 months after b starting ivacaftor therapy
Conflict of interest The authors report no conflict of interest and have no relevant financial disclosures.
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Lung (2014) 192:823–824
