Opportunities and Challenges in the Development and Evaluation of a Clinical Questionnaire for Primary Ciliary Dyskinesia (PCD)

Israel Amirav ¹ Mary Roduta Roberts ² Revital Abitbul ³ Bentur Lea ⁴ Malena Cohen-Cymberknoh ⁵ Micha Aviram ⁶ Husein Dabbah ⁷ Nael Elias ⁸ Arnon Elizur ⁹ Avigdor Hevroni ¹⁰ Moran Lavie ¹¹ Galit Livnat ¹² Avigdor Mandelberg ¹³ Ellie Picard ¹⁴ Heymut Omran ¹⁵ Huda Mussaffi ¹⁶

¹Pediatric Department, Ziv Medical Center, Faculty of Medicine, Bar IIan University and Alberta university.Canada
²Occupational Therapy Department, University of Alberta, Edmonton, Canada
³Pediatric Department, Ziv Medical Center, Faculty of Medicine, Bar IIan University
⁴Pulmonary Unit, Rambam Medical center
⁵Pulmonary Institute, Hadassah-Hebrew University Medical Center
⁶Pulmonary Unit, Soroka Medical Center
⁷Pediatric Department, Western Galilee Hospital
⁸Pediatric Department, Saint Vincent De-Paul Hospital
⁹Pediatric Department, Assaf Harofeh Medical Center,, ISRAEL
¹¹Pulmonary Unit, Edmond & Lili Safra Children's Hospital, Sheba Medical Center,
¹²Pulmonary Unit, Carmel Medical Center
¹³Pediatric Department, Wolfson Medical Center
¹⁴Pulmonary Unit, Shaaree Zedek Medical center
¹⁵Pediatric Department, University Children’s Hospital Muenster
¹⁶Pulmonary Institute, Schneider Children's Medical Center of Israel and Tel-Aviv-University

Rational: PCD is under diagnosed and underestimated. Most clinical research has used some form of questionnaire to capture data but no standardized questionnaire has been developed nor systematically evaluated. Objective:To critically appraise a clinical questionnaire for PCD used in a large national PCD consortium. Methods: We describe the development, validation and revision process of a clinical questionnaire for PCD and how it was evaluated 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 completed the revised version of the questionnaire for 173 patients with various completion rates for various items. While content analysis demonstrated validity, there were methodological deficiencies in completion rates. These 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 have developed the current version. Clinical research in PCD should gain benefit from future use and evaluation of this tool with additional patient populations and in various contexts.