Adaptation and Validation of an Individualised Paediatric Health-Related Quality of Life Measure (Paediatric Measure Yourself Medical Outcome Profile) and its Evaluation in a Clinical Setting

Abstract

There is evidence that the measurement of patient-reported outcomes (PROs) during or before clinical encounters can improve patient outcomes. Individualised PRO measures (PROMs) are known to empower patients by incorporating their values, goals and preferences in clinical encounters. There is no validated, individualised, generic, PRO assessment tool for paediatric populations available. The objectives of the PhD were to 1) assess evidence on the effectiveness of the use of PROMs in clinical practice; 2) adapt and validate an adult individualised health-related quality of life (HRQOL) measure, Measure Yourself Medical Outcome Profile (MYMOP), for clinical use for children 7-11 years old; and 3) conduct a pilot study to evaluate the administration methods and feasibility of application of the new tool in a clinical setting. A systematic search was performed using a controlled vocabulary relating to the terms, clinical care setting, and patient-reported outcomes. An English-language study was included if it was a randomised controlled trial (RCT) with a PROM as an intervention in a patient population. Included studies were synthesised and their methodologic quality appraised using the Cochrane Risk of Bias tool. Twenty-two RCTs reporting on 25 comparisons of efficacy were included in the systematic review. Overall, positive findings in favour of the PROM intervention were reported on 21 occasions, but these effects were robust in only five cases, (i.e. were statistically significant and adequately powered). While combined evidence supports the use of PROMS in clinical practice, standards of reporting remain inadequate, with many published RCTs failing to pre-specify primary and secondary outcomes or adequately power their comparisons for clinically meaningful differences. Adaptation of the MYMOP was achieved via the following four iterative steps: 1. an online survey of local paediatricians and paediatric trainees; 2. a focus group discussion with paediatricians; 3. an online survey with paediatric research experts and paediatricians across Australia; and 4. interviews with child-parent pairs in a clinical setting. Four paediatricians completed the first survey, five paediatricians participated in the focus group, and four paediatric HRQOL research experts completed the second survey. Twenty-five children (17 from general medicine, and eight from a diabetes/endocrine clinic) aged 7-11 years completed the draft paediatric MYMOP (P-MYMOP) with parental help in a few cases and were interviewed afterwards. Analysis of the interview data were performed according to Braun and Clarke’s (2006) guidelines on thematic analysis. An inductive, interpretative approach with realist epistemology was used. Data from the completed P-MYMOP and interviews demonstrated that the majority of participating children were able to identify their own problems and activity limitations, and all children in the study understood the 7-point faces-scale. Most parents and children perceived that the P-MYMOP would be useful to complete before clinic appointments and enthusiastically welcomed the opportunity for their children to have a voice in a setting where they are often passive recipients of care. Finally, a feasibility pilot study was performed in the Department of General Medicine Outpatient clinic, Women’s and Children’s Hospital in Adelaide. The participants of this study were clinic nurses and doctors working in the Department of General Medicine. The study ran for 19 days with 11 doctors and eight clinic nurses taking part. The newly content-validated Paediatric MYMOP (P-MYMOP) was distributed to children 7-11 years old, who were invited to take their completed form to their doctors to discuss it. Both the participating nurses and doctors found the P-MYMOP to be feasible to use in the clinic and distribution of the tool and its integration into the clinical consultation did not appear to compromise nurses’ or doctors’ time management. In addition, interviews with clinicians suggest this tool would be a welcome addition to care of their paediatric patients. The P-MYMOP is the first content-validated generic individualised HRQOL measure for children. The wording, layout, and scale of the P-MYMOP have been successfully adapted for children 7-11 years old. Given validation is an iterative process, further research to assess its reliability and construct validity is needed but results thus far suggest that this tool would be welcomed by children, carers and healthcare practitioners.