Drivers of caregiver impact in Duchenne muscular dystrophy: a cohort study.

Background: In our companion paper, we addressed the interplay between caregiver impact, out-of-pocket expenditures, and Duchenne Muscular Dystrophy (DMD) disability. We found that DMD caregiver impact could be characterized by four Latent Profile Analysis impact profiles: lowest, lower middle, upper middle, and highest impact. The impact on caregivers was often but not always worse with greater out-of-pocket expenditures.

Interplay of disability, caregiver impact, and out-of-pocket expenditures in Duchenne muscular dystrophy: a cohort study.

Background: Providing caregiving support to people with Duchenne muscular dystrophy (DMD) is challenging, beginning in early childhood, and continuing through the progression of multidimensional disability. This study addressed the interplay between caregiver impact, out-of-pocket expenditures, and DMD disability. To examine these interconnections, we investigated the association between caregiver impact domains and out-of-pocket expenditures; and the presence of clusters in caregivers on the basis of DMD-related disability domains in the patients for whom they provided caregiving support.

Expanding the purview of wellness indicators: validating a new measure that includes attitudes, behaviors, and perspectives.

Objective: The present study validated the DeltaQuest Wellness Measure (DQ Wellness), a new 15-item measure of wellness that spans relevant attitudes, behaviors, and perspectives.

Design: This cross-sectional web-based study recruited chronically-ill patients and/or caregivers ( = 3,961) and a nationally representative comparison group (n = 855).

Main Outcome Measures: The DQ Wellness assesses: a way of being in the world that involves seeing and embracing the good and expressing kindness toward others; engagement in one's activities and self-care; downplaying negative thoughts that reduce one's energy; and an ability to feel joy.

Measuring Duchenne muscular dystrophy impact: development of a proxy-reported measure derived from PROMIS item banks.

Background: Person-reported outcomes measurement development for rare diseases has lagged behind that of more common diseases. In studies of caregivers of patients with rare diseases, one relies on proxy report to characterize this disability. It is important to measure the child's disability accurately and comprehensively because it affects caregiver burden.

Characterizing the quality-of-life impact of Duchenne muscular dystrophy on caregivers: a case-control investigation.

Background: This study examined the impact of Duchenne muscular dystrophy (DMD) on family-member caregivers in terms of quality of life, life stress, and indirect costs, as compared to a stratified comparison group of parents of similar-age children without DMD.

Methods: A web-based survey included DMD caregivers and a nationally representative comparison group of parents of children without DMD stratified by Child Age Group. Outcomes included quality of life, resilience, caregiver impact, stressful life events, financial strain, out-of-pocket expenditures, work productivity and unrealized ambitions.

If it's information, it's not "bias": a scoping review and proposed nomenclature for future response-shift research.

Background: The growth in response-shift methods has enabled a stronger empirical foundation to investigate response-shift phenomena in quality-of-life (QOL) research; but many of these methods utilize certain language in framing the research question(s) and interpreting results that treats response-shift effects as "bias," "noise," "nuisance," or otherwise warranting removal from the results rather than as information that matters. The present project will describe the various ways in which researchers have framed the questions for investigating response-shift issues and interpreted the findings, and will develop a nomenclature for such that highlights the important information about resilience reflected by response-shift findings.

Methods: A scoping review was done of the QOL and response-shift literature (n = 1100 articles) from 1963 to 2020.

No impact of Asian ethnicity on EORTC QLQ-C30 scores: Group differences and differential item functioning in paroxysmal nocturnal hemoglobinuria.

Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening terminal-complement-mediated disease resulting in intravascular hemolysis and thrombosis with significant morbidity and premature mortality. There exists no disease-specific quality-of-life (QOL) measure for PNH. Its QOL effects resemble those of hematologic cancers, which supports the use of cancer-specific QOL measures in PNH clinical trials.

Norm-based comparison of the quality-of-life impact of ravulizumab and eculizumab in paroxysmal nocturnal hemoglobinuria.

Aims: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening intravascular hematologic disorder with significant morbidity and premature mortality. Clinical trials (NCT02946463 and NCT03056040) comparing ravulizumab with eculizumab for PNH have supported the non-inferiority of the former and similar safety and tolerability. This secondary analysis compared PNH trial participants after 26 weeks on either treatment (n = 438) to a general-population sample (GenPop) (n = 15,386) and investigated response-shift effects.

Moving toward better health: exercise practice is associated with improved outcomes after spine surgery in people with degenerative lumbar conditions.

Background: Recovery and rehabilitation following surgery can take many months. Understanding what patients can do to facilitate recovery would be beneficial for spinal surgeons. This study sought to evaluate the impact of exercise practice, before and after surgery, on long-term outcomes of spine surgery in a robust clinical sample.

Creating idiometric short-form measures of cognitive appraisal: balancing theory and pragmatics.

Background: The Rapkin and Schwartz appraisal theory and measure provided a path toward documenting response-shift effects and describing individual differences in ways of thinking about quality of life (QOL) that distinguished people in different circumstances. Recent work developed and validated the QOL Appraisal Profile (QOLAP), an 85-item measure that taps response-shift-detection domains of Frame of Reference, Standards of Comparison, Sampling of Experience, and Combinatory Algorithm. Recent theoretical work proposed that appraisal measurement constitutes a new class of measurement (idiometric), distinct from psychometric and clinimetric.

"Looking Under the Hood" of Anchor-Based Assessment of Clinically Important Change: A Machine Learning Approach.

Objectives: The Global Assessment of Change (GAC) item has facilitated the interpretation of change in patient-reported outcomes, providing an anchor for computing minimally important differences. Construct validity has been documented via disease-specific patient-reported outcomes change. We examined what domains, sociodemographic characteristics, attributions of change, and cognitive-appraisal processes are reflected in GAC ratings.

Patient factors that matter in predicting spine surgery outcomes: a machine learning approach.

Objective: There is an increasing recognition of the importance of predictive analytics in spine surgery. This, along with the addition of personalized treatment, can optimize treatment outcomes. The goal of this study was to examine the value of clinical, demographic, expectation, and cognitive appraisal variables in predicting outcomes after surgery.

Patient Factors That Matter in Predicting Hip Arthroplasty Outcomes: A Machine-Learning Approach.

Background: Despite the success of total hip arthroplasty (THA), approximately 10%-15% of patients will be dissatisfied with their outcome. Identifying patients at risk of not achieving meaningful gains postoperatively is critical to pre-surgical counseling and clinical decision support. Machine learning has shown promise in creating predictive models.

Response-shift effects in neuromyelitis optica spectrum disorder: estimating response-shift-adjusted scores using equating.

Background: In our companion paper, random intercept models (RIMs) investigated response-shift effects in a clinical trial comparing Eculizumab to Placebo for people with neuromyelitis optica spectrum disorder (NMOSD). RIMs predicted Global Health using the EQ-5D Visual Analogue Scale item (VAS) to encompass broad criteria that people might consider. The SF36™v2 mental and physical component scores (MCS and PCS) helped us detect response shift in VAS.

Response-shift effects in neuromyelitis optica spectrum disorder: a secondary analysis of clinical trial data.

Background: Researchers have long posited that response-shift effects may obfuscate treatment effects. The present work investigated possible response-shift effects in a recent clinical trial testing a new treatment for Neuromyelitis Optica Spectrum Disorder (NMOSD). This pivotal trial provided impressive support for the drug Eculizumab in preventing relapse, but less strong or null results as the indicators became more subjective or evaluative.

Capturing patient experience: does quality-of-life appraisal entail a new class of measurement?

Background: Two decades of research on quality-of-life (QOL) appraisal have demonstrated links between patient experience and health outcomes and have accounted for both intra-individual change and inter-individual differences in a wide range of research contexts. The present work investigates patterns across diagnostic and demographic groupings to demonstrate how population-specific circumstances drive the structure of QOL appraisal.

Methods: This secondary analysis (N = 6448) utilized data from six patient groups: spine surgery, multiple sclerosis, heterogeneous chronically ill, heterogeneous cancer, bladder cancer, and human immunodeficiency virus (HIV).

Understanding haemophilia caregiver burden: does appraisal buffer the impact of haemophilia on caregivers over time?

The present work investigated haemophilia caregiver and patient characteristics associated with change in negative and positive aspects of caregiving. Web-based survey data were collected from haemophilia A or B caregivers at baseline and two years later. Outcomes included Burden Summary and Positive Emotions change scores from the Haemophilia Caregiver Impact measure.

Lung transplant referral practice patterns: a survey of cystic fibrosis physicians and general pulmonologists.

Background: Many individuals with cystic fibrosis (CF) die from respiratory failure without referral for lung transplant. Physician practices that may expedite, delay, or preclude referral, are poorly understood.

Methods: Two parallel, web-based surveys focusing on lung transplant referral triggers and barriers, as well as pre-referral evaluation, were emailed to pulmonologists practicing in the New England region.

Responsiveness of standard spine outcome tools: do they measure up?

Objective: Over the past 2 decades, spine outcome research has become more standardized in response to recommendations from Deyo and others. By using the same generic and condition-specific patient-reported outcome (PRO) measures across studies, results are more easily compared. Given the challenges of maintaining high-quality data in clinical research studies, it would be important to evaluate the contribution of each PRO to confirm that it merits the respondent burden.

Positive emotions despite substantial burden: The alchemy of haemophilia caregiving.

Introduction: Qualitative interviews when developing the haemophilia caregiver impact measure (HCI) documented the importance of capturing the positive aspects of caregiving, not just the negative.

Aim: The present study thus investigates the construct underlying the positive emotions HCI subscale and tests models proposing a more comprehensive way of thinking about this construct.

Methods: Secondary analysis was implemented on longitudinal web-based survey data (n = 323) from haemophilia A or haemophilia B caregivers.

Salvage proton beam therapy in local recurrent uveal melanoma.

Purpose: To evaluate survival and ocular outcome in recurrent uveal melanoma treated with proton beam therapy as salvage therapy.

Design: Retrospective, interventional case series.

Methods: We evaluated 48 patients with local recurrence of uveal melanoma after primary treatment with brachytherapy, transpupillary thermotherapy, proton beam therapy, laser photocoagulation, CyberKnife radiation, or photodynamic therapy.

A new, four-item instrument to measure workplace bullying.

Studies on workplace bullying either in the U.S. or internationally rarely include nurses.

Proton or stereotactic photon irradiation for posterior uveal melanoma? A planning intercomparison.

Background And Purpose: Proton and stereotactic radiotherapy with photons (SRT) are both used to treat choroidal melanomas in proximity to optic disk and fovea centralis, a situation where plaque therapy is prone to complications. A comparative treatment- planning study was done to assess the capability of both modalities to preserve vision.

Patients And Methods: In ten patients treated with 68-MeV protons, SRT with 6-MV photons was planned.