Meaghan O'COnnor

Leukocyte adhesion deficiency, type I (LAD-I) is a rare inborn error of immunity characterized by recurrent, severe infections/inflammation and poor wound healing. Little is known about the patient/family experience of severe LAD-I. We conducted a multiple-case study exploring the burdens of illness, caregiving, and treatment in severe LAD-I. Nine caregivers (parents) representing five families with ≥1 child with severe LAD-I participated in individual, in-depth interviews. Memoing and inductive/deductive coding were used to analyze and compare each family’s experience. The caregivers described frequent and severe infections, physical and social restrictions, a complex journey to diagnosis, rearranging life around severe LAD-I, financial strain, isolation, and uncertainty. Treatment burden varied by treatment type. This multiple-case study highlights how severe LAD-I affects patients and families. As the first study to address the humanistic burden of LAD-I in patients and caregivers, it is an important contribution to the literature on LAD-I and qualitative methods for investigating the burden of rare disease on families.

Introduction For patients with autosomal dominant polycystic kidney disease (ADPKD), the only approved treatment is tolvaptan, a twice-daily medication that works to slow kidney function decline. Patients on tolvaptan report a considerable burden related to the drug’s aquaretic effects which can intensify immediately following a dose of the medication. Methods A targeted literature review and individual concept elicitation (CE) interviews with nephrologists and adult patients with ADPKD taking tolvaptan were conducted to inform the development of a hybrid diary, programmed as a smartphone application. A sample of adults with ADPKD taking tolvaptan tested the diary for 7 days; a subset also participated in cognitive debriefing (CD) interviews. The diary was then revised and finalized. Results CE interviews confirmed the importance of tracking urinary frequency and urgency and timing of tolvaptan doses in real time. Results from the usability/feasibility test and CD interviews confirmed the understandability and relevance of the diary and provided critical insights to improve its content and functionality. The resulting PKD Daily includes two daily entries to report dose timing, overnight urination, and daily impacts of frequent/urgent urination; real-time entries to capture frequency/urgency of daytime urination; and reminders to encourage regular data entry. Conclusion Originally developed for use in a clinical trial, the PKD Daily can also provide valuable information to clinicians and patients to support treatment decisions and inform dose selection in clinical practice, becoming a valuable part of a clinician’s toolbox. Further, the development of the PKD Daily can serve as a case study for the successful creation of a hybrid diary.

Purpose Mapping or matching the items in a clinical outcome assessment (COA) to concepts that define a condition is a common method for evaluating a COA’s concept coverage. The purpose of this research was to address the lack of formal guidance for conducting this task by developing a framework for best practices in COA concept mapping and applying it to a case study. Methods To develop the framework, we examined the literature and created a draft set of best practices which was then reviewed by experienced researchers through focus groups before being finalized. To conduct the case study, we extracted data from a systematic review of knee osteoarthritis (KO) symptoms and impacts and used the framework to map relevant concepts to items in the SF-36v2^® Health Survey (SF-36v2). Results The framework guides researchers in defining the purpose of and data sources for the mapping, establishing guiding principles and decision-making thresholds, and conducting the mapping exercise. The results of the case study demonstrate the usefulness of the framework in identifying 27/36 items (75%) in the SF-36v2 that addressed concepts that define KO. Conclusion This case study illustrates how the framework for best practices in COA concept mapping may be used, highlighting how establishing clear concept definitions and guiding principles and following a structured process throughout can help produce consistent, reliable, and reproducible results. The results from this rigorous approach can provide valuable evidence to support decisions about the appropriateness of a COA for the intended patient population.

This qualitative, cross-sectional study aimed to understand the barriers and facilitators related to the adherence and completion of the recombinant zoster vaccine (RZV) two-dose series in Canada, as perceived by healthcare providers (HCPs) and patients. Data collection occurred via 60-minute concept elicitation interviews with 12 HCPs (4 physicians, 2 nurse practitioners, 6 pharmacists) who had prescribed and/or administered RZV in Canada, and 21 patients aged ≥50 years who had received ≥1 dose of RZV. Patients were categorized as adherent (received both doses within the recommended 2-to-6-month timeframe; n = 11) or non-adherent (received only one dose or second dose outside the recommended timeframe; n = 10). Interview transcripts were coded and analyzed using a two-part thematic analysis approach. HCP-identified barriers to RZV adherence included high out-of-pocket cost, inconsistent/lack of health plan coverage, inconvenient processes for accessing RZV, and patient forgetfulness. HCP-identified facilitators included desire for shingles protection, HCP encouragement, and reminders. Barriers to RZV adherence identified by patients included lack of HCP knowledge/experience with RZV, receiving unreliable/confusing information, having unpleasant/severe side effects following the first dose, high out-of-pocket cost, lack of insurance coverage, and forgetfulness. Patient-identified facilitators included self-motivation, financial support, convenient processes for obtaining RZV, and reminders. In conclusion, many factors can influence RZV series completion and adherence among adults in Canada, including cost, insurance coverage, HCP knowledge and encouragement, and reminders. Awareness of these factors may inform HCPs in helping patients overcome barriers and identify opportunities for future consideration, facilitating protection against herpes zoster.

Purpose: Patients with transthyretin amyloidosis (ATTR) experience a wide variety of symptoms and impacts on health-related quality of life (HRQoL). However, the lack of an ATTR-specific patient-reported outcome (PRO) measure has made consistent measurement of HRQoL in ATTR challenging. This paper describes the development of a conceptual model and subsequent content for the Transthyretin Amyloidosis – Quality of Life Questionnaire (ATTR-QOL), an ATTR-specific PRO measure. Methods: This was a cross-sectional, non-interventional, US-based study. The study design included three stages: 1) a targeted literature review followed by qualitative data collection with patients and experts; 2) development of a conceptual model and PRO measure; and 3) review of the PRO measure using a modified Delphi method, translatability assessment, and interviews with patients and experts. Revisions were made to the measure after each round of review. Results: Forty-four patients and 29 experts participated in this study. The conceptual model included two primary concepts of interest: symptoms (cardiac, neuropathic–peripheral, neuropathic–autonomic, and other) and impacts (eg, physical, role, and mental/emotional functioning). Seventy-two items were created (32 symptoms; 40 impacts) to align with the model. A recall period of one month was selected based on participant input. Conclusion: The ATTR-QOL was created with significant patient involvement and guidance from a multidisciplinary group of experts. The mix of patient and clinical perspectives helped to ensure a balanced representation of all relevant disease experiences and clinical specialties. With further refinement from psychometric testing, the ATTR-QOL will provide a standard, comprehensive measure for all ATTR-specific research including both clinical trials and clinical practice.

The increase in global health networks as mechanisms for improving health and affecting social change has been recognized as a key component of the global health landscape today. To successfully raise awareness of shared causes, global health networks need tools to help them plan successful campaigns and evaluate the impact of their work, as well as to coordinate and reinforce each other’s efforts. One global health network, the Respectful Maternity Care (RMC) Global Council, can be credited with raising the profile of the issues of disrespect and abuse (D&A) in childbirth and the need for RMC within global maternal health. We set out to learn from the work of the RMC Global Council and the RMC movement at large to develop a tool—a framework for planning and evaluating awareness-raising efforts—useful for networks focused on global health and human rights. We reviewed the literature for theoretical models on awareness raising and, finding a lack of appropriate tools, developed a new, draft framework using components of the Framework for Effective Campaigns, the SpitFire SmartChart 3.0, and Network Theory. We conducted semi-structured interviews with members of the RMC Global Council to validate the draft framework and identify any additional strategies or tactics that were used during their efforts to raise awareness of D&A and RMC. We also interviewed “influenced” individuals to validate inputs from the influencers and determine the key documents, events, individuals, and organizations that made the greatest contribution to the increased awareness of D&A/RMC. Data were analyzed using deductive and inductive qualitative research methods. The validated awareness-raising framework includes five strategies that characterize a successful awareness-raising effort. Each strategy has a set of tactics that can operationalize those strategies. Each tactic is classified as essential, helpful, or variable based on the number of key informants who utilized it. This case study offers an example of how global health networks can create a movement that effects change at global and local levels by providing an empirically-grounded framework to help plan, coordinate, and evaluate future campaigns designed to raise awareness and create momentum in global health, human rights, and quality of care.

In February 2015, the World Health Organization (WHO) released “Strategies toward ending preventable maternal mortality (EPMM)” (EPMM Strategies), a direction-setting report outlining global targets and strategies for reducing maternal mortality in the Sustainable Development Goal (SDG) period. In May 2015, the EPMM Working Group outlined a plan to develop a comprehensive monitoring framework to track progress toward the achievement of these targets and priorities. This monitoring framework was developed in two phases. Phase I, which focused on identifying indicators related to the proximal causes of maternal mortality, was completed in October 2015. This paper describes the process and results of Phase II, which was completed in November 2016 and aimed to build consensus on a set of indicators that capture information on the social, political, and economic determinants of maternal health and mortality. A total of 150 experts from more than 78 organizations worldwide participated in this second phase of the process to develop a comprehensive monitoring framework for EPMM. The experts considered a total of 118 indicators grouped into the 11 key themes outlined in the EPMM report, ultimately reaching consensus on a set of 25 indicators, five equity stratifiers, and one transparency stratifier. The indicators identified in Phase II will be used along with the Phase I indicators to monitor progress towards ending preventable maternal deaths. Together, they provide a means for monitoring not only the essential clinical interventions needed to save lives but also the equally important political, social, economic and health system determinants of maternal health and survival. These distal factors are essential to creating the enabling environment and high-performing health systems needed to ensure high-quality clinical care at the point of service for every woman, her fetus and newborn. They complement and support other monitoring efforts, in particular the “Survive, Thrive, and Transform” agenda laid out by the Global Strategy for Women’s, Children’s and Adolescents’ Health (2016-2030) and the SDG3 global target on maternal mortality.

Making high-quality health care available to all women during pregnancy is a critical strategy for improving perinatal outcomes for mothers and babies everywhere. Research from high-income countries suggests that antenatal care delivered in a group may be an effective way to improve the provision, experiences, and outcomes of care for pregnant women and newborns. A number of researchers and programmers are adapting group antenatal care (ANC) models for use in low- and middle-income countries (LMIC), but the evidence base from these settings is limited and no studies to date have assessed the feasibility and acceptability of group ANC in India. We adapted a “generic” model of group antenatal care developed through a systematic scoping review of the existing evidence on group ANC in LMICs for use in an urban setting in India, after looking at local, national and global guidelines to tailor the model content. We demonstrated one session of the model to physicians, auxiliary nurse midwives, administrators, pregnant women, and support persons from three different types of health facilities in Vadodara, India and used qualitative methods to gather and analyze feedback from participants on the perceived feasibility and acceptability of the model. Providers and recipients of care expressed support and enthusiasm for the model and offered specific feedback on its components: physical assessment, active learning, and social support. In general, after witnessing a demonstration of the model, both groups of participants—providers and beneficiaries—saw group ANC as a vehicle for delivering more comprehensive ANC services, improving experiences of care, empowering women to become more active partners and participants in their care, and potentially addressing some current health system challenges. This study suggests that introducing group ANC would be feasible and acceptable to stakeholders from various care delivery settings, including an urban primary health clinic, a community-based mother and child health center, and a private hospital, in urban India.