A coordinated approach for managing polypharmacy among children with medical complexity: rationale and design of the Pediatric Medication Therapy Management (pMTM) randomized controlled trial

Abstract

Background: Children with medical complexity (CMC) often rely upon the use of multiple medications to sustain quality of life and control substantial symptom burden. Pediatric polypharmacy (≥ 5 concurrent medications) is prevalent and increases the risk of medication-related problems (MRPs). Although MRPs are associated with pediatric morbidity and healthcare utilization, polypharmacy is infrequently assessed during routine clinical care for CMC. The aim of this randomized controlled trial is to determine if a structured pharmacist-led Pediatric Medication Therapy Management (pMTM) intervention reduces MRP counts, as well as the secondary outcomes of symptom burden and acute healthcare utilization.

Methods: This is a hybrid type 2 randomized controlled trial assessing the effectiveness of pMTM compared to usual care in a large, patient-centered medical home for CMC. Eligible patients include all children ages 2-18 years old, with ≥ 1 complex chronic condition, and with ≥ 5 active medications, as well as their English-speaking primary caregivers. Child participants and their primary parental caregivers will be randomized to pMTM or usual care before a non-acute primary care visit and followed for 90 days. Using generalized linear models, the overall effectiveness of the intervention will be evaluated using total MRP counts at 90 days following pMTM intervention or usual care visit. Following attrition, a total of 296 CMC will contribute measurements at 90 days, which provides > 90% power to detect a clinically significant 1.0 reduction in total MRPs with an alpha level of 0.05. Secondary outcomes include Parent-Reported Outcomes of Symptoms (PRO-Sx) symptom burden scores and acute healthcare visit counts. Program replication costs will be assessed using time-driven activity-based scoring.

Discussion: This pMTM trial aims to test hypotheses that a patient-centered medication optimization intervention delivered by pediatric pharmacists will result in lower MRP counts, stable or improved symptom burdens, and fewer cumulative acute healthcare encounters at 90 days following pMTM compared to usual care. The results of this trial will be used to quantify medication-related outcomes, safety, and value for a high-utilization group of CMC, and outcomes may elucidate the role of integrated pharmacist services as a key component of outpatient complex care programs for this priority pediatric population.

Trial registration: This trial was prospectively registered at clinicaltrials.gov (NCT05761847) on Feb 25, 2023.

Keywords: Adverse drug events; Children with medical complexity; Deprescribing; Medication safety; Pediatrics; Polypharmacy.

Fig. 1

Study Flow Diagram. Protocolized study flow is depicted. Following enrollment and consent, participants will be randomized 1:1 to pMTM intervention or usual care, with the pMTM intervention occurring ≤ 3 days before a scheduled well child visit or routine follow-up medical encounter. Both groups will then be seen for their scheduled PCP visit as occurring within usual care. Applicable outcomes will be re-assessed at 90-day follow-up

Fig. 2

Conceptual Framework for pMTM Intervention. This protocol is conceptualized based on the SHED-MEDS model of deprescribing [60]. With permission, we modified this model with the broader core activity of pMTM, during which parents and providers review medication changes, continuation, proper use, monitoring, and follow-up. For children with polypharmacy, our model specifies that the pMTM intervention (which accounts for and prioritizes safety, quality of life, and parental considerations) will lead to patient-centered optimization of medications, resulting in improved health and symptoms manifest as fewer MRPs [4, 12, 30].