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Study protocol, rationale and recruitment in a European multi-centre randomized controlled trial to determine the efficacy and safety of azithromycin maintenance therapy for 6 months in primary ciliary dyskinesia

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Kobbernagel, Helene Elgaard ; Buchvald, Frederik F ; Haarman, Eric G ; Casaulta, Carmen ; Collins, Samuel A ; Hogg, Claire ; Kuehni, Claudia E ; Lucas, Jane S ; Omran, Heymut ; Quittner, Alexandra L ; Werner, Claudius ; Nielsen, Kim G. / Study protocol, rationale and recruitment in a European multi-centre randomized controlled trial to determine the efficacy and safety of azithromycin maintenance therapy for 6 months in primary ciliary dyskinesia. In: BMC Pulmonary Medicine. 2016 ; Vol. 16, No. 1. pp. 104.

Bibtex

@article{4341ca8413694493a88d820c7b609e9a,
title = "Study protocol, rationale and recruitment in a European multi-centre randomized controlled trial to determine the efficacy and safety of azithromycin maintenance therapy for 6 months in primary ciliary dyskinesia",
abstract = "BACKGROUND: Clinical management of primary ciliary dyskinesia (PCD) respiratory disease is currently based on improving mucociliary clearance and controlling respiratory infections, through the administration of antibiotics. Treatment practices in PCD are largely extrapolated from more common chronic respiratory disorders, particularly cystic fibrosis, but no randomized controlled trials (RCT) have ever evaluated efficacy and safety of any pharmacotherapeutics used in the treatment of PCD. Maintenance therapy, with the macrolide antibiotic azithromycin, is currently widely used in chronic respiratory diseases including PCD. In addition to its antibacterial properties, azithromycin is considered to have beneficial anti-inflammatory and anti-quorum-sensing properties. The aim of this study is to determine the efficacy of azithromycin maintenance therapy for 6 months on respiratory exacerbations in PCD. The secondary objectives are to evaluate the efficacy of azithromycin on lung function, ventilation inhomogeneity, hearing impairment, and symptoms (respiratory, sinus, ears and hearing) measured on a PCD-specific health-related quality of life instrument, and to assess the safety of azithromycin maintenance therapy in PCD.METHODS: The BESTCILIA trial is a European multi-centre, double-blind, randomized, placebo-controlled, parallel group study. The intervention is tablets of azithromycin 250/500 mg according to body weight or placebo administered three times a week for 6 months. Subjects with a confirmed diagnosis of PCD, age 7-50 years, are eligible for inclusion. Chronic pulmonary infections with Gram-negative bacteria or any recent occurrence of non-tuberculous mycobacteria are exclusion criteria. The planned number of subjects to be included is 125. The trial has been approved by the Research Ethics Committees of the participating institutions.DISCUSSION: We present a study protocol of an ongoing RCT, evaluating for the first time, the efficacy and safety of a pharmacotherapeutic treatment for patients with PCD. The RCT evaluates azithromycin maintenance therapy, a drug already commonly prescribed in other chronic respiratory disorders. Furthermore, the trial will utilize the Lung clearance index and new, PCD-specific quality of life instruments as outcome measures for PCD. Recruitment is hampered by frequent occurrence of Pseudomonas aeruginosa infection, exacerbations at enrolment, and the patients' perception of disease severity and necessity of additional management and treatment during trial participation.TRIAL REGISTRATION: EudraCT 2013-004664-58 (date of registration: 2014-04-08).",
keywords = "Adolescent, Adult, Anti-Bacterial Agents, Azithromycin, Child, Disease Progression, Double-Blind Method, Europe, Female, Humans, Kartagener Syndrome, Male, Middle Aged, Quality of Life, Regression Analysis, Research Design, Respiratory Tract Infections, Spirometry, Treatment Outcome, Vital Capacity, Young Adult, Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't",
author = "Kobbernagel, {Helene Elgaard} and Buchvald, {Frederik F} and Haarman, {Eric G} and Carmen Casaulta and Collins, {Samuel A} and Claire Hogg and Kuehni, {Claudia E} and Lucas, {Jane S} and Heymut Omran and Quittner, {Alexandra L} and Claudius Werner and Nielsen, {Kim G}",
year = "2016",
month = "7",
day = "22",
doi = "10.1186/s12890-016-0261-x",
language = "English",
volume = "16",
pages = "104",
journal = "BMC Pulmonary Medicine",
issn = "1471-2466",
publisher = "BioMed Central Ltd",
number = "1",

}

RIS

TY - JOUR

T1 - Study protocol, rationale and recruitment in a European multi-centre randomized controlled trial to determine the efficacy and safety of azithromycin maintenance therapy for 6 months in primary ciliary dyskinesia

AU - Kobbernagel, Helene Elgaard

AU - Buchvald, Frederik F

AU - Haarman, Eric G

AU - Casaulta, Carmen

AU - Collins, Samuel A

AU - Hogg, Claire

AU - Kuehni, Claudia E

AU - Lucas, Jane S

AU - Omran, Heymut

AU - Quittner, Alexandra L

AU - Werner, Claudius

AU - Nielsen, Kim G

PY - 2016/7/22

Y1 - 2016/7/22

N2 - BACKGROUND: Clinical management of primary ciliary dyskinesia (PCD) respiratory disease is currently based on improving mucociliary clearance and controlling respiratory infections, through the administration of antibiotics. Treatment practices in PCD are largely extrapolated from more common chronic respiratory disorders, particularly cystic fibrosis, but no randomized controlled trials (RCT) have ever evaluated efficacy and safety of any pharmacotherapeutics used in the treatment of PCD. Maintenance therapy, with the macrolide antibiotic azithromycin, is currently widely used in chronic respiratory diseases including PCD. In addition to its antibacterial properties, azithromycin is considered to have beneficial anti-inflammatory and anti-quorum-sensing properties. The aim of this study is to determine the efficacy of azithromycin maintenance therapy for 6 months on respiratory exacerbations in PCD. The secondary objectives are to evaluate the efficacy of azithromycin on lung function, ventilation inhomogeneity, hearing impairment, and symptoms (respiratory, sinus, ears and hearing) measured on a PCD-specific health-related quality of life instrument, and to assess the safety of azithromycin maintenance therapy in PCD.METHODS: The BESTCILIA trial is a European multi-centre, double-blind, randomized, placebo-controlled, parallel group study. The intervention is tablets of azithromycin 250/500 mg according to body weight or placebo administered three times a week for 6 months. Subjects with a confirmed diagnosis of PCD, age 7-50 years, are eligible for inclusion. Chronic pulmonary infections with Gram-negative bacteria or any recent occurrence of non-tuberculous mycobacteria are exclusion criteria. The planned number of subjects to be included is 125. The trial has been approved by the Research Ethics Committees of the participating institutions.DISCUSSION: We present a study protocol of an ongoing RCT, evaluating for the first time, the efficacy and safety of a pharmacotherapeutic treatment for patients with PCD. The RCT evaluates azithromycin maintenance therapy, a drug already commonly prescribed in other chronic respiratory disorders. Furthermore, the trial will utilize the Lung clearance index and new, PCD-specific quality of life instruments as outcome measures for PCD. Recruitment is hampered by frequent occurrence of Pseudomonas aeruginosa infection, exacerbations at enrolment, and the patients' perception of disease severity and necessity of additional management and treatment during trial participation.TRIAL REGISTRATION: EudraCT 2013-004664-58 (date of registration: 2014-04-08).

AB - BACKGROUND: Clinical management of primary ciliary dyskinesia (PCD) respiratory disease is currently based on improving mucociliary clearance and controlling respiratory infections, through the administration of antibiotics. Treatment practices in PCD are largely extrapolated from more common chronic respiratory disorders, particularly cystic fibrosis, but no randomized controlled trials (RCT) have ever evaluated efficacy and safety of any pharmacotherapeutics used in the treatment of PCD. Maintenance therapy, with the macrolide antibiotic azithromycin, is currently widely used in chronic respiratory diseases including PCD. In addition to its antibacterial properties, azithromycin is considered to have beneficial anti-inflammatory and anti-quorum-sensing properties. The aim of this study is to determine the efficacy of azithromycin maintenance therapy for 6 months on respiratory exacerbations in PCD. The secondary objectives are to evaluate the efficacy of azithromycin on lung function, ventilation inhomogeneity, hearing impairment, and symptoms (respiratory, sinus, ears and hearing) measured on a PCD-specific health-related quality of life instrument, and to assess the safety of azithromycin maintenance therapy in PCD.METHODS: The BESTCILIA trial is a European multi-centre, double-blind, randomized, placebo-controlled, parallel group study. The intervention is tablets of azithromycin 250/500 mg according to body weight or placebo administered three times a week for 6 months. Subjects with a confirmed diagnosis of PCD, age 7-50 years, are eligible for inclusion. Chronic pulmonary infections with Gram-negative bacteria or any recent occurrence of non-tuberculous mycobacteria are exclusion criteria. The planned number of subjects to be included is 125. The trial has been approved by the Research Ethics Committees of the participating institutions.DISCUSSION: We present a study protocol of an ongoing RCT, evaluating for the first time, the efficacy and safety of a pharmacotherapeutic treatment for patients with PCD. The RCT evaluates azithromycin maintenance therapy, a drug already commonly prescribed in other chronic respiratory disorders. Furthermore, the trial will utilize the Lung clearance index and new, PCD-specific quality of life instruments as outcome measures for PCD. Recruitment is hampered by frequent occurrence of Pseudomonas aeruginosa infection, exacerbations at enrolment, and the patients' perception of disease severity and necessity of additional management and treatment during trial participation.TRIAL REGISTRATION: EudraCT 2013-004664-58 (date of registration: 2014-04-08).

KW - Adolescent

KW - Adult

KW - Anti-Bacterial Agents

KW - Azithromycin

KW - Child

KW - Disease Progression

KW - Double-Blind Method

KW - Europe

KW - Female

KW - Humans

KW - Kartagener Syndrome

KW - Male

KW - Middle Aged

KW - Quality of Life

KW - Regression Analysis

KW - Research Design

KW - Respiratory Tract Infections

KW - Spirometry

KW - Treatment Outcome

KW - Vital Capacity

KW - Young Adult

KW - Journal Article

KW - Multicenter Study

KW - Randomized Controlled Trial

KW - Research Support, Non-U.S. Gov't

U2 - 10.1186/s12890-016-0261-x

DO - 10.1186/s12890-016-0261-x

M3 - Journal article

VL - 16

SP - 104

JO - BMC Pulmonary Medicine

JF - BMC Pulmonary Medicine

SN - 1471-2466

IS - 1

ER -

ID: 49873786