Objective The primary objective of this study was to assess whether there are different patterns (classes) of joint health in young boys with severe haemophilia A (SHA) prescribed primary tailored prophylaxis. We also assessed whether age at first index joint bleed, blood group, FVIII gene abnormality variant, factor VIII trough level, first‐year bleeding rate and adherence to the prescribed prophylaxis regimen significantly predicted joint damage trajectory, and thus class membership. Methods Using data collected prospectively as part of the Canadian Hemophilia Primary Prophylaxis Study (CHPS), we implemented a latent class growth mixture model technique to determine how many joint damage classes existed within the cohort. We used a multinomial logistic regression to predict the odds of class membership based on the above predictors. We fitted a survival model to assess whether there were differences in the rate of dose escalation across the groups. Results We identified three distinct classes of trajectory: persistently low, moderately increasing and rapidly increasing joint scores. By multinomial regression, we found that only age at first index joint bleed predicted rapidly increasing joint scores. The rapidly increasing joint score class group moved through dose escalation significantly faster than the other two groups. Conclusions Using tailored prophylaxis, boys with SHA follow one of three joint health trajectories. By using knowledge of disease trajectories, clinicians may be able to adjust treatment according to a subject's predicted long‐term joint health and institute cost‐effective programmes of prophylaxis targeted at the individual subject level.

Background Patient engagement is an important tool for quality improvement (QI) and optimizing the uptake of research findings. The Plan-Do-Study-Act (PDSA) model is a QI tool that encourages ongoing evaluation of clinical care, thus improving various aspects of patient care. Ascertaining pediatric patient priorities for a pain questionnaire in the post-acute, or transitional pain, setting is important to guide clinical care since active engagement with the population of interest can optimize uptake. We used the PDSA model to adapt a chronic pain questionnaire for the pediatric transitional pain setting to reflect pediatric patient and parent/guardian preferences and to form an example of how the PDSA model can be used to improve clinical care through patient engagement. Methods This project employed the PDSA model to adapt the pediatric Ontario Chronic Pain Questionnaire for use in the pediatric Transitional Pain Service (pedTPS) setting. Plan: Following reviewing the Ontario Chronic Pain Questionnaire and literature on pain questionnaire development, goal-based questions, questions on pain location, relevant Patient-Reported Outcomes Measurement Information System (PROMIS®) measures and the Pain Catastrophizing Scale, child (PCS-C) and parent (PCS-P), informed the questionnaire. Do: The questionnaire and a satisfaction survey were sent to patients and families through Research Electronic Data Capture (REDCap™). Study: Results from the satisfaction survey were analyzed. Act: Using descriptive statistics employing ordinal mixed-models with random effects, ANOVA, and double-blinded qualitative thematic coding, questionnaire preferences were analyzed and the questionnaire was adapted accordingly before implementation into the (pedTPS). Results Eighty-eight questionnaires and satisfaction surveys were analyzed from 69 respondents (32 patients; 37 parents/guardians). Sixty-six (75.00%) surveys indicated satisfaction with the questionnaire. A combined 77 (87.50%) “strongly agreed” (25/88) or “agreed” (52/88) that the questionnaire language was clear. The application of suggested changes to the questionnaire resulted in four versions across the project timeline, which reflected patient and parent/guardian preferences for questions that reflect the themes, “Story”; “Time-Optimal”; and “Pertinent” (“STOP”). There were no statistically significant differences in satisfaction across the versions due to sample size. Conclusion Most respondents were satisfied with the questionnaire and prefer “STOP” questions. Future studies will focus on testing the questionnaire for validity and reliability across pedTPS populations.

Background There is limited evidence evaluating the impact of patient and public involvement in setting priorities for healthcare, specifically drug funding recommendation committees. We describe the development phases of the Patient and Public Involvement Questionnaire (PPIQ).Methods The development of the PPIQ was informed by previous work, which established nine criteria to evaluate patient and public involvement. The PPIQ was developed with a multi-method, multiphased approach: 1) item generation and refinement (item bank creation, user feedback sessions); 2) sensibility testing (using Feinstein's criteria and structured interviews); and 3) pilot testing with drug funding committee's in Canada. ResultsIn phase one of development, a bank of 846 items were derived from key informant interviews and a literature review. Guided by the nine evaluation criteria, an initial draft of the PPIQ was created using the item bank. Two user feedback sessions (n=7) resulted in further revisions of the PPIQ. In phase two, participants (n=21) completed a sensibility questionnaire with a median score 6 out of 7 on 80% of items. Interview participants (n=14) articulated the PPIQ was clear and appropriate. In phase three, response rate for pilot testing the PPIQ was 25% (n=14) and the average time for participants to complete the PPIQ was 00:19:00 minutes (SD ± 13:46). ConclusionsThe methods used for the development of the PPIQ including qualitative interviews, literature review, user feedback, sensibility and pilot testing have resulted in a questionnaire that may be used with committees making drug funding recommendations in Canada.