Patient Reported Outcomes in Cleft Lip and Palate

Sammanfattning: Aim: Patient reported outcome measures (PROMs) quantify various domains of health related quality of life (HRQOL) from the patient perspective. The overall aim of this thesis was to investigate patient reported outcomes (PROs) in patients with cleft lip and/or palate (CL/P). Method: PROs were investigated in the CL/P population with PROMs and interviews. Health care professional (HCP) experience of implementing PROs was investigated with focus group discussion. Patients who had been investigated for velopharyngeal dysfunction (VPD) completed a questionnaire on satisfaction with speech. Parents of children with CL/P and HCPs were interviewed regarding psychosocial and educational issues. The Cleft Hearing Appearance and Speech Questionnaire (CHASQ) was translated and implemented in 8 countries. CHASQ was tested in a control population and compared with results of the CL/P population. Results of CHASQ and CLEFT-Q were compared and patient opinion on the two PROMs were investigated.Results: Most patients with VPD who underwent evaluation and treatment felt that surgery and speech therapy had improved their speech, but ultimately only approximately 50% of them were satisfied with the quality of their speech. Parental experience and views were diverse, ranging from the opinion that there were no specific problems related to the cleft - to a clear expression of both emotional and educational issues. The views and level of knowledge of HCPs also varied. All HCPs, however, wished for more information and training regarding psychosocial and educational issues and treatment. The CHASQ was translated into eight languages: Bulgarian, Estonian, Greek, Latvian, Macedonian, Romanian, Serbian and Swedish. Different levels of satisfaction between countries were revealed. There were patients in all countries who were less satisfied than expected and therefore should be identified for further investigation or treatment. Clinicians in multiple countries expressed the usefulness of CHASQ in their clinical work. It was perceived as useful, short, and easy to implement. CHASQ could be an effective instrument for collection of PROs on satisfaction with hearing, appearance and speech. Children and young people with CL/P were as satisfied with their appearance, hearing and speech as children and young people without CL/P. Scores from CHASQ and CLEFT-Q correlated well. Patients thought that CHASQ was easier to complete than CLEFT-Q. They liked CLEFT-Q more and thought that it better informed HCPs about their thoughts, opinions and feelings than CHASQ.Conclusion: PROs in the CL/P population indicate a generally high level of satisfaction with outcome which is comparable to that of a control population in regard to satisfaction with hearing, appearance and speech. A PROM could be a useful instrument for improved communication between clinicians and patients and their families. Efforts for care and research in the CL/P population should include implementation of a CL/P specific and holistic PROM. Consensus on an international level of adaption of the same PROM and method for implementation is favourable.