The iPCD Cohort is an international cohort that assembles available retrospective datasets and prospectively newly collected clinical and diagnostic data from patients suffering from primary ciliary dyskinesia (PCD) worldwide, to answer pertinent questions on clinical phenotype, disease severity, prognosis and effect of treatments in patients with this rare multiorgan disease.

Population

Patients of all ages diagnosed with PCD, followed in a PCD centre or other specialised clinic or registered in a national registry

Brief Title

International Primary Ciliary Dyskinesia Cohort

Official Title

International Primary Ciliary Dyskinesia Cohort

Brief Summary

The iPCD Cohort is an international cohort that assembles available retrospective datasets <br /> and prospectively newly collected clinical and diagnostic data from patients suffering from <br /> primary ciliary dyskinesia (PCD) worldwide, to answer pertinent questions on clinical <br /> phenotype, disease severity, prognosis and effect of treatments in patients with this rare <br /> multiorgan disease.

Detailed Description

The iPCD Cohort was set up under the framework of the European Union (EU) funded 7th
Framework Programme (FP7) project Better Experimental Screening and Treatment for Primary
Ciliary Dyskinesia (BESTCILIA). The iPCD Cohort is hosted at the Institute of Social and
Preventive Medicine at the University of Bern, Switzerland. Research is performed in close
collaboration with all data contributors.

Aims:

This combined international dataset allows investigation of PCD epidemiology in a large
international study population in order to: 1) describe the spectrum of clinical phenotypes
and disease severity in PCD patients by age, sex and time period of diagnosis; 2) describe
short-term and long-term prognosis of PCD, looking at important outcomes such as growth, lung
function and respiratory failure, bacterial colonisation, hearing loss, fertility, and
mortality; and 3) identify predictors of long-term outcomes such as age at diagnosis,
clinical phenotype, ultrastructural defects, genotype and clinical care.

Study design:

The iPCD Cohort is an international cohort, combining available data on PCD from national or
local registries and clinical or diagnostic databases. All participating centres delivered
retrospectively collected data; new centres joining the iPCD Cohort in the future can also
participate with retrospectively and prospectively collected data.

What information is collected:

The iPCD Cohort includes retrospectively collected patient data on the following 11 thematic
categories: 1) general information, 2) results of diagnostic tests, 3) baseline
characteristics, 4) growth and lung function, 5) clinical manifestations, 6) therapy, 7)
microbiology, 8) imaging, 9) surgical interventions, 10) neonatal period, and 11) family
history.

Study database:

The iPCD Cohort database is web-based, using the Research Electronic Data Capture (REDCap)
platform developed at Vanderbilt University. REDCap is widely used in academic research and
allows data entry and extraction in various formats.

How to participate:

Centres that wish to participate to the project and contribute data can contact the iPCD
Cohort to sign a data delivery agreement. They then will receive a password to access the
online software REDCap and they will be able to enter their data directly. They can also
upload follow-up data or add additional patients at a later time point.

For further details, contact: pcd@ispm.unibe.ch

Funding:

The setting up of the iPCD Cohort (salaries, consumables and equipment) was funded by the EU
FP7 project BESTCILIA (http://bestcilia.eu) and several Swiss funding bodies, including the
Lung Leagues of Bern, St Gallen, Vaud, Ticino and Valais and the Milena Carvajal
Pro-Kartagener Foundation. Data collection and management at each site was funded according
to local arrangements. Most participating researchers and data contributors participate in
the European Cooperation in Science and Technology (COST) Action "Better Evidence to Advance
Therapeutic options for PCD" (BEAT-PCD) (BM 1407; www.beatpcd.org). Infrastructure is
provided for free by the University of Bern, where the data are pooled and stored. The study
is currently funded by the Swiss National Science Foundation (320030_173044 and
320030B_192804).

Outcome Measures

Outcome measures are the tests that investigators perform to prove whether or not a treatment being tested in a clinical trial is having any effect.

Inclusion Criteria:

Patients diagnosed with primary ciliary dyskinesia

Exclusion Criteria:

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