CLEFT-Q: Development of a Patient-Reported Outcome Measure to Provide Clinically Meaningful Outcomes in Patients with Cleft Lip and/or Palate

Abstract

Background: The management of cleft lip and/or palate (CL/P) includes multidisciplinary care beginning in infancy and continuing through to adulthood. Outcomes of cleft care have been difficult to measure because of the subjective nature of evaluating concepts such as appearance and speech. Including the patient perspective in outcome evaluation through the use of a patient-reported outcome (PRO) measure would provide a more accurate reflection of a patient’s status. The overall objective of this thesis is to show that through adherence to rigorous methods of development, a PRO measure can provide clinically meaningful outcome evaluation in cleft care. Methods: The first paper uses the qualitative method of interpretive description to define a conceptual framework to guide the development of a PRO measure for patients with CL/P, the CLEFT-Q. The second paper describes the protocol for the entire development of the CLEFT-Q. The third paper analyzes the results of the cross-sectional field-test of the CLEFT-Q scales to determine whether or not the CLEFT-Q is able to detect differences between specific cleft types. Results: The qualitative study included 138 patients with CL/P from six countries. The final conceptual framework contained thirteen concepts within the domains of appearance, facial function, and health-related quality of life. The second paper details the process of designing the CLEFT-Q scales. The field-test included 2,434 patients from thirty sites in twelve countries, and CLEFT-Q scores were found to vary with cleft type for all scales. Conclusions: PRO measures need to be rigorously designed in order to provide scientifically sound, clinically meaningful measurement. The CLEFT-Q is able to detect differences between patients with various cleft types, and will be a useful tool to provide the patient perspective in future outcome evaluation in cleft care.