Concordance between patient-centered and adaptive behavior outcome measures after applied behavior analysis for autism

Our ability to evaluate outcomes which genuinely reflect patients’ unmet needs, hopes and concerns is of pivotal importance. However, much current clinical research and practice falls short of this objective by selecting outcome measures which do not capture patient value to the fullest. In this Opinion, we discuss Patient-Centered Outcomes Measures (PCOMs), which have the potential to systematically incorporate patient perspectives to measure those outcomes that matter most to patients. We argue for greater multi-stakeholder collaboration to develop PCOMs, with rare disease patients and families at the center. Beyond advancing the science of patient input, PCOMs are powerful tools to translate care or observed treatment benefit into an ‘interpretable’ measure of patient benefit, and thereby help demonstrate clinical effectiveness. We propose mixed methods psychometric research as the best route to deliver fit-for-purpose PCOMs in rare diseases, as this methodology brings together qualitative and quantitative research methods in tandem with the explicit aim to efficiently utilise data from small samples. And, whether one opts to develop a brand-new PCOM or to select or adapt an existing outcome measure for use in a rare disease, the anchors remain the same: patients, their daily experience of the rare disease, their preferences, core concepts and values. Ultimately, existing value frameworks, registries, and outcomes-based contracts largely fall short of consistently measuring the full range of outcomes that matter to patients. We argue that greater use of PCOMs in rare diseases would enable a fast track to Patient-Centered Care.

BackgroundSubstantial literature has highlighted the importance of patient-reported outcome and experience measures (PROMs and PREMs, respectively) to collect clinically relevant information to better understand and address what matters to patients. The purpose of this systematic review is to synthesize the evidence about how healthcare providers implement individual-level PROMs and PREMs data into daily practice.MethodsThis mixed methods systematic review protocol describes the design of our synthesis of the peer-reviewed research evidence (i.e., qualitative, quantitative, and mixed methods), systematic reviews, organizational implementation projects, expert opinion, and grey literature. Keyword synonyms for “PROMs,” PREMs,” and “implementation” will be used to search eight databases (i.e., MEDLINE, CINAHL, PsycINFO, Web of Science, Embase, SPORTDiscus, Evidence-based Medicine Reviews, and ProQuest (Dissertation and Theses)) with limiters of English from 2009 onwards. Study selection criteria include implementation at the point-of-care by healthcare providers in any practice setting. Eligible studies will be critically appraised using validated tools (e.g., Joanna Briggs Institute). Guided by the review questions, data extraction and synthesis will occur simultaneously to identify biographical information and methodological characteristics as well as classify study findings related to implementation processes and strategies. As part of the narrative synthesis approach, two frameworks will be utilized: (a) Consolidated Framework for Implementation Research (CFIR) to identify influential factors of PROMs and PREMs implementation and (b) Expert Recommendations for Implementing Change (ERIC) to illicit strategies. Data management will be undertaken using NVivo 12TM.DiscussionData from PROMs and PREMs are critical to adopt a person-centered approach to healthcare. Findings from this review will guide subsequent phases of a larger project that includes interviews and a consensus-building forum with end users to create guidelines for implementing PROMs and PREMs at the point of care.Systematic review registrationPROSPERO CRD42020182904.

<b>Purpose:</b> The purpose of this study was to use qualitative methods to describe the key lessons learned during the stakeholder engagement stage of planning a randomized clinical trial comparing outcomes of silver diamine fluoride (SDF) as an alternative to pit-and-fissure sealants in a school-based delivery system. <b>Methods:</b> Eighteen caregivers and community-based stakeholders with involvement in the school-based sealant program Sealants for Smiles from the state of Montana, were recruited for this qualitative study. United States (U.S.) Patient-Centered Outcomes Research Institute (PCORI) methodology standards were used to develop two semi-structured interview guides consisting of 6 questions. One interview guide was used for telephone interviews with caregivers and the second was used for a stakeholder focus group. Content analytic methods were used to analyze the data. <b>Results:</b> All participants believed that a study comparing SDF and sealants was clinically relevant. Non-caregiver stakeholders agreed with the proposed primary outcome of the study (caries prevention) whereas caregivers also emphasized the importance of child-centered outcomes such as minimizing dental anxiety associated with dental care. Stakeholders described potential concerns associated with SDF such as staining and perceptions of safety and discussed ways to address these concerns through community engagement, appropriate framing of the study, proper consent procedures, and ongoing safety monitoring during the trial. Finally, stakeholders suggested dissemination strategies such as direct communication of findings through professional organizations and encouraging insurance plans to incentivize SDF use by reimbursing dental providers. <b>Conclusions:</b> Involving key stakeholders in early planning is essential in developing patient-centered research questions, outcomes measures, study protocols, and dissemination plans for oral health research involving a school-based delivery system.

To assess to what extent individual physical therapy treatment goals for children with cerebral palsy are reflected in 2 standardized measures: the GMFM-88 and the PEDI. A total of 36 paediatric physical therapists working in primary healthcare with children with cerebral palsy in their caseload. The International Classification of Functioning, Disability and Health was used as a measure to link individual treatment goals to the GMFM-88 and the PEDI. Sixty percent of the treatment goals were covered by the 2 measures. Twenty-seven percent and 21% of the goals were reflected in the GMFM-88 and the PEDI, respectively; 12% of the goals were reflected in both instruments. Another 26% of the treatment goals appeared to be partly covered (21% in PEDI and 5% in GMFM-88). Fourteen percent of the goals were not covered by the 2 measures. Individual goals set in a physical therapy practice for children with cerebral palsy can be linked, to a large extent, to items and activities of 2 standardized measures.

Development of National Research and Clinical Agendas for Patient-Reported Outcomes in IR: Proceedings from a Multidisciplinary Consensus Panel

BackgroundStandardised use of patient-centred outcome measures (PCOMs) improves aspects of quality of care. Normalization Process Theory (NPT) considers the social (inter-)actions of implementation processes operationalised through four constructs: coherence-building, cognitive participation, collective action and reflexive monitoring. The aim of the study was to identify barriers and enablers for the successful use of PCOMs in specialist palliative home care (SPHC) using NPT, to collect clinically meaningful and reliable data to improve patient outcomes.MethodsQualitative study using semi-structured interviews with palliative care professionals from German SPHC teams who participated in a study using PCOMs. Data were analysed using Framework analysis, and contextualised within NPT.ResultsSeventeen interviews across five teams were conducted. Some teams already had an understanding of what PCOMs are and how to use them, based on previous experience. In other teams, this understanding developed through the perception of the benefits (coherence). Participation and engagement depended on individuals and was decisive for coherence-building. The attitude of the management level also played a major role (cognitive participation). Integration of PCOMs into everyday clinical practice varied and depended on the manifestation of the first two constructs and other already established routines (collective action). In the context of appraisal, both positive (e.g. focus on patient) and negative aspects (e.g. additional work) of using PCOMs were mentioned (reflexive monitoring).ConclusionsAlthough benefits of using PCOMs were partly recognised, not all teams continued standardised use. Here, not only the social (inter-)actions, but also the influence of the context (working environment) were decisive. Future implementation strategies should consider integrating PCOMs in existing electronic patient records, education sessions supporting coherence-building, internal facilitators/local champions, and ensuring frequent data analyses as it is beneficial and increases the readiness of using PCOMs.

271 Background: The prospective collection of standardized, patient-centered outcomes for men with prostate cancer will allow intra- and inter-institutional comparisons, patient education, self-assessment and dissemination of best practices. We lack recommended standardized sets of such outcomes. Methods: Through the International Consortium for Health Outcomes Measurement, a working group of urologists, radiation oncologists, registry representatives and patient advocates convened to develop a recommended minimum set of measures which institutions would collect on all prostate patients. Using a modified Delphi method over a series of in-person meetings and conference calls, a final set of recommendations was developed. Results: Approximately 30 experts in prostate cancer care from North America, Europe, Australia, and the Middle East participated in the process, representing academic centers, registries and patients. We defined the scope of this initial effort as outcomes related to the management of localized prostate cancer. The group recommended cross-disciplinary measures applicable to a variety of treatment approaches from surveillance to radiation and prostatectomy. Standards were identified for disease control definitions as well as baseline patient and disease-specific risk stratification factors. Domains of patient reported outcomes to be tracked in follow up were identified including urinary incontinence, urinary irritation, bowel irritation, sexual function, and symptoms related to hormonal therapy. Finally, specific tools to assess these domains and gaps in our current tools were identified. The final recommended set will be presented. Conclusions: Standardized outcome reporting is a necessary component in the movement towards high-value health care. Patient-centered outcomes related to toxicity and disease control can be identified and systemized by a multidisciplinary group of experts. The result of this project is an initial step in what will be an iterative process with the goal of improving the care of all men with prostate cancer. An international pilot project has been proposed based on implementing the final set of recommendations.

ImportanceCare management programs are increasingly being utilized by health systems as a new foundational strategy to advance value-based care. These programs offer the promise of improving patient outcomes while decreasing health care utilization and costs. However, as these programs proliferate in number and specialization, the field of care management is increasingly at risk of fragmentation, inefficiency, and failure to meet the core needs of the patient.ObservationsThis review of the current state of care management identifies several key challenges for the field, including an unclear value proposition, a focus on system- vs patient-centered outcomes, increased specialization by private and public entrants that produces care fragmentation, and lack of coordination among health and social service entities. A framework is proposed for reorienting care management to truly address the needs of patients through acknowledging the dynamic nature of patient care needs, providing a continuum of need-targeted programming, coordinating care among all involved entities and staff, and performing regular evaluations of outcomes that include patient-centered and health equity measures. Guidance on how this framework can be implemented within a health system and an outline of recommendations is provided for how policymakers may incentivize the development of high value and more equitable care management programs.Conclusions and RelevanceWith increased focus on care management as a cornerstone of value-based care, value-based health leaders and policymakers can improve the effectiveness and value of care management programs, reduce patient financial burden for care management services, and promote stakeholder coordination.

Sanfilippo syndrome is a lysosomal storage disease marked by severe neurodegeneration along with neurobehavioral problems and functional impairments including sleeping disturbances and gastrointestinal symptoms. Due to the neuroinflammatory aspect of the disease, we conducted a phase I/II open-label study of anakinra, an IL-1 receptor antagonist, in Sanfilippo syndrome (N=23). This post-hoc pragmatic analysis focuses specifically on 1) changes in the Child Sleep Habits Questionnaire (CSHQ) and Sanfilippo Stool Habit Questionnaire (SSHQ) among participants with sleep disturbance or diarrhea present on Day 1; 2) the Sanfilippo Behavior Rating Scale (SBRS) mood/anger/aggression cluster scores among participants with diagnosed agitation/aggression/disruptive behavior disorder, taken from medical history at screening; and 3) whether disease severity measured by the Vineland Adaptive Behavior Scales-3 (VABS-3) adaptive behavior composite (ABC) score is associated with response to treatment.

The International Dermatology Outcome Measures (IDEOM) initiative is a nonprofit organization dedicated to enhancing clinical care and research in dermatology by developing evidence-based, patient-centered outcome measures. At the 2019 annual meeting of the Group for Research and Assessment of Psoriasis and Psoriatic Arthritis (GRAPPA), the IDEOM psoriasis working group presented an overview of its selected deliverables and discussed its efforts to agree on meaningful, valid, and feasible outcome measures for quality measurement in psoriasis. The psoriatic arthritis (PsA) workgroup focused on the measurement of PsA symptoms in psoriasis clinical trials, and the measurement of nonspecific musculoskeletal symptoms among patients with psoriasis in psoriasis longitudinal clinical trials and cohort studies.

Self-determination theory (SDT) guided behavioral interventions are effective in improving several patient-centered metrics, including glaucoma-related distress. However, whether improvement in patient-centered metrics can drive an improvement in medication-taking behavior remains to be seen. The 7-month Support, Educate, Empower (SEE) personalized glaucoma coaching program was previously shown to improve glaucoma medication adherence by 21 percent points. This study's goal was to assess the impact of the SEE program on self-determination theory (SDT) metrics and other patient-centered outcome measures. Glaucoma patients (≥40y old, taking ≥1 medication) self-reporting poor medication adherence were recruited at the University of Michigan. Eight surveys (with 10 subscales) were completed before and after the 7-month SEE program. Three surveys assessed changes in SDT (Treatment Self-regulation Questionnaire, Healthcare-Climate Questionnaire, Perceived Competence) while the others assessed participants' Glaucoma Knowledge, Glaucoma Medication Self-efficacy, Glaucoma-related distress, Perceived benefits, confidence asking and getting questions answered. Thirty-nine participants completed the SEE program. Significant improvements were in 7 subscales, including all three SDT tenets of competence (mean change =0.9, SD =±1.2, adjusted P =0.0002), autonomy (0.5, ±0.9, 0.044), and relatedness ( P =0.002). Glaucoma-related distress (-2.0, ±3.2, 0.004), confidence in asking questions (1.1, ±2.0, 0.008), and confidence in getting questions answered (1.0, ±2.0, 0.009) also improved. Glaucoma-related distress was correlated with perceived competence ( r =-0.56, adjusted P =0.005), and an increase in perceived competence was associated with a decrease in glaucoma-related distress (β=-0.43, 95% CI -0.67 - -0.20, adjusted P =0.007). The SEE program improved participants' autonomous motivation, perceived support, perceived competence, glaucoma-related distress, and competence. These results point to the promising potential of SDT-guided behavioral interventions in improving patient-centered metrics.

BackgroundPhelan-McDermid syndrome (PMS) is a genetic neurodevelopmental disorder caused by SHANK3 haploinsufficiency and is associated with an increased risk for seizures. Previous literature indicates that around one third of individuals with PMS also have epilepsy or seizures, with a wide range of types and ages of onset. Investigating the impact of seizures on intellectual and adaptive functioning for PMS is a primary concern for caregivers and is important to understanding the natural history of this syndrome.MethodsWe report on results from 98 individuals enrolled in a prospective, longitudinal study. We detailed seizure frequency, type, and age of onset, and we analyzed seizure occurrence with best estimate IQ, adaptive functioning, clinical features, and genotype. We conducted multiple linear regression analyses to assess the relationship between the presence of seizures and the Vineland Adaptive Behavior Scale, Second Edition (VABS-II) Adaptive Behavior Composite score and the best estimate full-scale IQ. We also performed Chi-square tests to explore associations between seizure prevalence and genetic groupings. Finally, we performed Chi-square tests and t-tests to explore the relationship between seizures and demographic features, features that manifest in infancy, and medical features.ResultsSeizures were present in 41% of the cohort, and age of onset was widely variable. The presence of seizures was associated with significantly lower adaptive and intellectual functioning. Genotype–phenotype analyses were discrepant, with no differences in seizure prevalence across genetic classes, but with more genes included in deletions of participants with 22q13 deletions and seizures compared to those with 22q13 deletions and no seizures. No clinical associations were found between the presence of seizures and sex, history of pre- or neonatal complications, early infancy, or medical features. In this cohort, generalized seizures were associated with developmental regression, which is a top concern for PMS caregivers.ConclusionsThese results begin to eludicate correlates of seizures in individuals with PMS and highlight the importance of early seizure management. Importantly, presence of seizures was associated with adaptive and cognitive functioning. A larger cohort might be able to identify additional associations with medical features. Genetic findings suggest an increased capability to realize genotype–phenotype relationships when deletion size is taken into account.

To compare community diagnoses of Autism Spectrum Disorder (ASD) reported by parents to consensus diagnoses made using standardized tools plus clinical observation. 87 participants (85% male, average age 7.4 years), with reported community diagnosis of ASD were evaluated using the Autism Diagnostic Observation Schedule) (ADOS-2), Differential Ability Scale (DAS-II), and Vineland Adaptive Behavior Scales (VABS-II). Detailed developmental and medical history was obtained from all participants. Diagnosis was based on clinical consensus of at least two expert clinicians, using test results, clinical observations, and parent report. 23% of participants with a reported community diagnosis of ASD were classified as non-spectrum based on our consensus diagnosis. ASD and non-spectrum participants did not differ on age at evaluation and age of first community diagnosis. Non-verbal IQ scores and Adaptive Behavior Composite scores were significantly higher in the non-spectrum group compared to the ASD group (104.5 ± 21.7 vs. 80.1 ± 21.6, p < .01; 71.1 ± 15 versus 79.5 ± 17.6, p < .05, respectively). Participants enrolled with community diagnosis of PDD-NOS were significantly more likely to be classified as non-spectrum on the study consensus diagnosis than Participants with Autism or Asperger (36% versus 9.5%, Odds Ratio = 5.4, p < .05). This study shows suboptimal agreement between community diagnoses of ASD and consensus diagnosis using standardized instruments. These findings are based on limited data, and should be further studied, taking into consideration the influence of DSM 5 diagnostic criteria on ASD prevalence.

Standardization of outcome measurement using a patient-centered approach in pediatric facial palsy may help aid the advancement of clinical care in this population. To develop a standardized outcome measurement set for pediatric patients with facial palsy through an international multidisciplinary group of health care professionals, researchers, and patients and patient representatives. A working group of health care experts and patient representatives (n = 21), along with external reviewers, participated in the study. Seven teleconferences were conducted over a 9-month period between December 3, 2016, and September 23, 2017, under the guidance of the International Consortium for Health Outcomes Measurement, each followed with a 2-round Delphi process to develop consensus. This process defined the scope, outcome domains, measurement tools, time points for measurements, and case-mix variables deemed essential to a standardized outcome measurement set. Each teleconference was informed by a comprehensive review of literature and through communication with patient advisory groups. Literature review of PubMed was conducted for research published between January 1, 1981, and November 30, 2016. The study aim was to develop the outcomes and measures relevant to children with facial palsy as opposed to studying the effect of a particular intervention. The 21 members of the working group included pediatric facial palsy experts from 9 countries. The literature review identified 1628 papers, of which 395 (24.3%) were screened and 83 (5.1%) were included for qualitative evaluation. A standard set of outcome measurements was designed by the working group to allow the recording of outcomes after all forms of surgical and nonsurgical facial palsy treatments among pediatric patients of all ages. Unilateral or bilateral, congenital or acquired, permanent or temporary, and single-territory or multiterritory facial palsy can be evaluated using this standard set. Functional, appearance, psychosocial, and administrative outcomes were selected for inclusion. Clinimetric and psychometric outcome measurement tools (clinician-, patient-, and patient proxy-reported) and time points for measuring patient outcomes were established. Eighty-six independent reviews of the standard set were completed, and 34 (85%) of the 40 patients and patient representatives and 44 (96%) of the 46 health care professionals who participated in the reviews agreed that the standard set would capture the outcomes that matter most to children with facial palsy. This international collaborative study produced a free standardized set of outcome measures for evaluating the quality of care provided to pediatric patients with facial palsy, allowing benchmarking of clinicians, comparison of treatment pathways, and introduction of value-based reimbursement strategies in the field of pediatric facial palsy. NA.

To compare the effectiveness of occupational therapist-led assessments of older people on dependency and service costs with that of social worker-led assessments. Pragmatic community-based randomised controlled trial over 2 years 4 months. Cambridgeshire, UK. 321 older people aged 65 and over living in their own homes and 113 carers. participants were randomised to two groups, to receive either occupational therapist-led or social worker-led assessment. Primary outcome was dependency (Community Dependency Index). Secondary outcomes included quality of life scores (EQ-5D) and psychological outlook (Perceived Stress Scale (PSS)). Outcome measures for carers included Carer Assessment of Difficulty Index (CADI), PSS and EQ-5D, collected at baseline, 4 and 8 months. Resource use data were collected from professional practice records, participants and carers at final follow-up. 264 (82%) of the randomised participants completed the study. No between-group statistically significant differences were found, except that carers in the occupational therapist arm had significantly better EQ-5D scores at the 8 month follow-up (thermometer P = 0.03) and in the social worker arm better CADI scores on stress (P = 0.047) and amount of caring (P = 0.049). There was no clear difference in patient-centred effectiveness measures between occupational therapists and social workers in assessing frail older people and their carers in the community. More extensive use of primary care health services by occupational therapists may have contributed to the differences in EQ-5D scores for carers. Delays in making occupational therapy assessments and in completing recommended housing adaptations may have contributed to these negative findings.