title: Collecting clinical data in primary ciliary dyskinesia- challenges and opportunities.
abstract: Rationale:Primary ciliary dyskinesia (PCD) is under diagnosed and underestimated. Most clinical research has used some form of questionnaires to capture data but none has been critically evaluated particularly with respect to its end-user feasibility and utility.Objective:To critically appraise a clinical data collection questionnaire for PCD used in a large national PCD consortium in order to apply conclusions in future PCD research.Methods:We describe the development, validation and revision process of a clinical questionnaire for PCD and its evaluation during a national clinical PCD study with respect to data collection and analysis, initial completion rates and user feedback.Results:14 centers participating in the consortium successfully completed the revised version of the questionnaire for 173 patients with various completion rates for various items. While content and internal consistency analysis demonstrated validity, there were methodological deficiencies impacting completion rates and end-user utility. These deficiencies were addressed resulting in a more valid questionnaire.Conclusions:Our experience may be useful for future clinical research in PCD. Based on the feedback collected on the questionnaire through analysis of completion rates, judgmental analysis of the content, and feedback from experts and end users, we suggest a practicable framework for development of similar tools for various future PCD research.
authors: Department of Pediatrics, University of Alberta, Edmonton, Canada; Department of Pediatrics, Ziv Medical Center, Faculty of Medicine, Bar Ilan University, Safed, Israel.
authors: Israel
authors: I
authors: Amirav
authors: Department of Occupational Therapy, University of Alberta, Edmonton, Canada.
authors: Mary
authors: M
authors: Roduta Roberts
authors: Schneider Children's Medical Center of Israel, Tel Aviv, Israel.
authors: Huda
authors: H
authors: Mussaffi
authors: The Edith Wolfson Medical Center, Tel Aviv University, Holon, Israel.
authors: Avigdor
authors: A
authors: Mandelberg
authors: The Edith Wolfson Medical Center, Tel Aviv University, Holon, Israel.
authors: Yehudah
authors: Y
authors: Roth
authors: Department of Pediatrics, Ziv Medical Center, Faculty of Medicine, Bar Ilan University, Safed, Israel.
authors: Revital
authors: R
authors: Abitbul
authors: Department of Pediatrics, Ziv Medical Center, Faculty of Medicine, Bar Ilan University, Safed, Israel.
authors: Anthony
authors: A
authors: Luder
authors: Schneider Children's Medical Center of Israel, Tel Aviv, Israel.
authors: Hannah
authors: H
authors: Blau
authors: Soroka Medical Center, Beer Shiva, Israel.
authors: Soliman
authors: S
authors: Alkrinawi
authors: Soroka Medical Center, Beer Shiva, Israel.
authors: Micha
authors: M
authors: Aviram
authors: Department of Pediatrics, Ziv Medical Center, Faculty of Medicine, Bar Ilan University, Safed, Israel.
authors: Marta
authors: M
authors: Ben-Ami
authors: Rambam Medical Center, Haifa, Israel.
authors: Moshe
authors: M
authors: Rotschild
authors: Rambam Medical Center, Haifa, Israel.
authors: Lea
authors: L
authors: Bentur
authors: Hadassah-Hebrew University Medical Centers, Jerusalem, Israel.
authors: David
authors: D
authors: Shoseyov
authors: Hadassah-Hebrew University Medical Centers, Jerusalem, Israel.
authors: Malena
authors: M
authors: Cohen-Cymberknoh
authors: Hadassah-Hebrew University Medical Centers, Jerusalem, Israel.
authors: Eitan
authors: E
authors: Kerem
authors: Hadassah-Hebrew University Medical Centers, Jerusalem, Israel.
authors: Avraham
authors: A
authors: Avital
authors: Hadassah-Hebrew University Medical Centers, Jerusalem, Israel.
authors: Chaim
authors: C
authors: Springer
authors: Hadassah-Hebrew University Medical Centers, Jerusalem, Israel.
authors: Avigdor
authors: A
authors: Hevroni
authors: Galilee Medical Center, Naharia, Bar Ilan Faculty of Medicine, Safed, Israel.
authors: Husein
authors: H
authors: Dabbah
authors: Assaf Harofeh Medical Center, Zerifin, Israel.
authors: Arnon
authors: A
authors: Elizur
authors: Shaare Zedek Medical Center, Jerusalem, Israel.
authors: Elie
authors: E
authors: Picard
authors: Shaare Zedek Medical Center, Jerusalem, Israel.
authors: Shmuel
authors: S
authors: Goldberg
authors: Carmel Medical Center, Haifa, Israel.
authors: Joseph
authors: J
authors: Rivlin
authors: Carmel Medical Center, Haifa, Israel.
authors: Galit
authors: G
authors: Livnat
authors: Sheba Medical Center , Tel Aviv University, Tel Aviv, Israel.
authors: Moran
authors: M
authors: Lavie
authors: St Vincent Hospital, Nazaret, Israel.
authors: Nael
authors: N
authors: Alias
authors: Dana Children's Hospital, Tel Aviv, Israel.
authors: Ruth
authors: R
authors: Soferman
authors: Department of General Pediatrics, University Children's Hospital Muenster, Muenster, Germany.
authors: Heike
authors: H
authors: Olbrich
authors: Department of General Pediatrics, University Children's Hospital Muenster, Muenster, Germany.
authors: Johanna
authors: J
authors: Raidt
authors: Department of General Pediatrics, University Children's Hospital Muenster, Muenster, Germany.
authors: Julia
authors: J
authors: Wallmeier
authors: Department of General Pediatrics, University Children's Hospital Muenster, Muenster, Germany.
authors: Claudius
authors: C
authors: Werner
authors: Department of General Pediatrics, University Children's Hospital Muenster, Muenster, Germany.
authors: Niki T
authors: NT
authors: Loges
authors: Department of General Pediatrics, University Children's Hospital Muenster, Muenster, Germany.
authors: Heymut
authors: H
authors: Omran
keywords: clinical trial; cohorts; questionnaire; response rate; symptoms