Engagement of children and parents in developing outcome measures and determining minimal important difference: focus on probiotics and antibiotic-associated diarrhea
Creators: Khanpour Ardestani, Samaneh
Publisher: name: University of Alberta Library
Year: 2022

<p>Introduction <br>
Patient engagement as a major component of patient-oriented research (POR) emphasizes that patients should be engaged not only as participants, but also as research partners.<br>
Having patient perspectives to inform the content and relevance of patient-reported outcome measures (PROMs) is now a requirement in many regulatory body guidelines. Increasing use of PROMs in pediatric populations has been documented frequently. As such, child and adolescent involvement to establish content validity of pediatric instruments has been recommended.<br>
Minimal important difference (MID) is defined as the smallest difference/change which patients perceive as important. Historically, healthcare providers have had the sole role in MID estimation. However, over time, calls to incorporate patient perspectives have emerged. <br>
Recent calls to determine MID from the patient’s perspective are especially relevant for therapies that are accessed by consumers without a prescription (e.g., probiotics). According to the latest Cochrane systematic review, probiotics have moderate protective effects in preventing antibiotic-associated diarrhea (AAD) in children. However, no studies reported seeking the perspective of children or parents about the MID of probiotics in preventing AAD and there is no specific validated instrument available to measure the incidence and severity of pediatric AAD.<br>
The objective of this thesis was to investigate whether and how children and/or parents have been engaged in developing PROMs and determining MID, and to use patient-centered approaches to 1) inform the MID of probiotics in prevention of pediatric AAD and 2) to validate an instrument for the measurement of incidence and severity of AAD in children.<br>
Methods<br>
Two review studies were conducted. In chapter 2, the method, stage, and level of engagement of the studies developing a new PROM in the pediatric population which engaged children and/or their parents in the process of development were described. In chapter 3, characteristics and findings of the studies reporting patient involvement in determining MID of any intervention were described.<br>
In the survey study described in chapter 4, parents of children presenting to the emergency department of a children’s hospital and pediatricians were approached to develop parent and pediatrician-derived MID.<br>
In chapter 5, with the help of a patient advisory group, an instrument was developed to measure the incidence and severity of pediatric AAD. The internal consistency and convergent validity of the instrument were examined in a prospective observational study in children (birth to 17 years old) newly prescribed antibiotics or on antibiotics for ≤ 7 days.<br>
Results<br>
Both review studies showed scarcity of evidence in engagement of children/parents either in developing pediatric PROMs or determining MID. <br>
The survey study showed good agreement between parents and clinicians regarding MID of probiotics in preventing pediatric AAD. Half of the participants in both groups reported they would use probiotics if it reduced the risk of AAD by 39%. The most important outcomes to parents and clinicians in measurement of pediatric AAD were identified. <br>
In the PAAD instrument development study, we found a broad range of incidence risk of AAD (27%-83%) by applying four different definitions of diarrhea. Cronbach’s α for the severity scale was less than 0.7. A high correlation was found between the PAAD severity score and numerical rating score of diarrhea severity reported by parents (r&gt;0.5).<br>
Conclusion<br>
This doctoral dissertation showed that existing evidence regarding child/family engagement in PROM development and MID determination is sparse and at a preliminary stage. The estimated MID of probiotics in preventing pediatric AAD will help in sample size calculation and interpretation of results of future randomized clinical trials. Lastly, the PAAD instrument developed, and validity tested in this thesis will enable accurate measurement of pediatric AAD in future studies.</p>