Patient related Outcome Measures

PURPOSE: The objective of this study was to develop a cross-cultural adaptation and translation into French of the original Harris Hip Score (HHS) and the modified Harris Hip Score (mHHS). PATIENTS AND METHODS: The translation and cultural-adaptation of the questionnaire were performed following a 10-step process as recommended by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). During the cognitive debriefing, each item of the questionnaire has been rated between 1 and 10 according to the comprehension level of 8 patients who underwent total hip arthroplasty...

BACKGROUND: A time-motion study is a scientific method for recording time spent on various tasks in a narrow range of specialized work settings, beginning with initial enrollment in ART provision. Therefore, the study aimed to assess the time motion of patient satisfaction with antiretroviral therapy services in Central Ethiopia. METHODS: A facility-based cross-sectional study was conducted on a sample of 422 patients from June 14 to July 30, 2021. We used a simple random sampling technique to select the participants...

BACKGROUND: The provision of intensive care services is advancing globally. However, in resource-limited settings, it is lagging far behind and intensive care unit mortality is still higher due to various reasons. This study aimed to assess determinants of mortality among medical patients admitted to the intensive care unit. METHODS: A five-year facility-based retrospective Cohort Study was conducted. A total of 546 medical patients admitted to the intensive care unit from March 2017 to February 2022 were included...

INTRODUCTION: Epilepsy is a chronic non-communicable disease of the brain that affects millions of people worldwide. A significant number of children are affected globally, and most live in developing countries, often with physical and cognitive disabilities. Regardless of these factors, epilepsy is poorly controlled, particularly in the developing countries. Thus, this study aimed to assess the magnitude of treatment outcomes and its predictors among pediatrics patients with epilepsy who were followed-up at the Dessie Comprehensive Specialized Hospital, Northeast Ethiopia...

BACKGROUND: Familial chylomicronemia syndrome (FCS) is a rare metabolic disorder that impacts physical, emotional, social, and cognitive functioning. The FCS-Symptom and Impact Scale (FCS-SIS) patient-reported outcome (PRO) measure assesses common symptoms and impacts of FCS. This study was conducted to evaluate cross-sectional psychometric properties of the FCS-SIS and its scoring method. METHODS: This multisite, cross-sectional, observational study of individuals with FCS was conducted in the United States and Canada...

BACKGROUND: Cardiac arrest (CA) is a common public health problem. Worldwide, cardiac arrest ranks highly among hospitalised patients' public health concerns, particularly in low-income nations. Data on cardiac arrest in intensive care units in low-income countries are relatively scarce. Determining the incidence and predictors of cardiac arrest among ICU patients will be a very crucial and fruitful clinical practice in resource-limited areas like Ethiopia. METHODS: A retrospective cohort study was conducted by reviewing charts of 422 systematically selected patients admitted to the ICU from 2018 to 2022 in Wachemo University Comprehensive Specialized Hospital...

PURPOSE: Erythropoietic protoporphyria (EPP), a rare inherited disorder, presents in early childhood with severe, painful phototoxicity, with significant impacts on health-related quality of life (HRQoL). Previous studies have not captured all concepts important to patients. Therefore, this study sought to develop a novel, comprehensive, and content valid patient-reported outcome (PRO) measure to assess the efficacy of new therapies. PATIENTS AND METHODS: Qualitative interviews were conducted with EPP participants and clinical experts to obtain views on concepts relevant to patients...

INTRODUCTION: Home hemodialysis (HHD) offers patients with end-stage kidney disease (ESKD) greater flexibility and advantages of health outcomes over center hemodialysis (CHD). This study aims to investigate the differences between home and center hemodialysis with a focus on racial/ethnic minorities. METHODS: The US Renal Disease System (USRDS) 2019 patient core data containing mortality and hospitalization which are cumulative since 2010 were merged with 2016-2019 Medicare clinical claims...

INTRODUCTION: Delivering Bad News (DBN) presents a highly challenging situation in physician-patient communication. This study aims to gain insight into the various communication strategies employed by physicians when DBN. METHODS: This qualitative study conducted thematic analysis of in-depth interviews. Physicians from two comprehensive hospitals with large patient populations were selected purposively based on their engagement in delivering bad news to patients...

BACKGROUND: There is increasing number of patients undergoing hemodialysis globally. Patients on hemodialysis experience physical and emotional stress due to the changes brought by chronic kidney disease. AIM: The study aimed at exploring the lived experiences of patients on hemodialysis treatment in Kiruddu National Referral Hospital. METHODS: The study employed a phenomenological design. Data was collected using audio tape recording of the interview from 9 participants selected through purposive sampling at Kiruddu National Referral Hospital...

INTRODUCTION: Despite the implementation of complex interventions, ICU mortality remains high and more so in developing countries. The demand for critical care in Sub-Saharan Africa is more than ever before as the region experiences a double burden of rising rates of non-communicable diseases (NCD) in the background battle of combating infectious diseases. Limited studies in Tanzania have reported varying factors associated with markedly high rates of ICU mortality. Investigating the burden of ICU care remains crucial in providing insights into the effectiveness and challenges of critical care delivery...

BACKGROUND: Children with sickle cell disease (SCD) experience a multiplex of disease-related symptoms and complications, including vaso-occlusive crises (VOCs), episodes characterized by extreme pain. METHODS: A cross-sectional observational survey examined the health-related quality of life (HRQoL) and school experiences of children with SCD 2 months-11 years, burden experienced by their caregivers, and associations between these outcomes and VOC frequency. Caregivers (N=167) of children with SCD in the US completed the Infant-Toddler Quality of Life-Short Form 47 (ITQoL-SF47) for children 2 months-4 years, the Child Health Questionnaire-Parent Form 50 (CHQ-PF50) and PROMIS Pain Interference and Sleep Disturbance Parent Proxy short forms for children 5-11 years, and a study-specific survey of school experiences...

PURPOSE: The hypoparathyroidism symptom diary (HypoPT-SD) is a disease-specific patient-reported outcome (PRO) tool comprising a 7-item symptom subscale, a 4-item impact subscale and 1-item anxiety, and sadness or depression components. This analysis assessed the psychometric properties of the HypoPT-SD symptom subscale scores using data from two open-label, single arm, Phase 4 studies (Study 402 and Study 404). PATIENTS AND METHODS: Eligible patients were aged 18 years or older with a confirmed diagnosis of hypoparathyroidism...

PURPOSE: The VinelandTM Adaptive Behavior Scale is often used in autism spectrum disorder (ASD) trials. The Adaptive Behavior Composite Score (VABS-ABC) is the standardized overall score (the average of the Socialization, Communication and Daily Living skills domains), and the standardized 2-Domain Composite Score (VABS-2DC) is a novel outcome measure (average of the Socialization and Communication domains). A within-person meaningful change threshold (MCT) has not been established for the VABS-2DC...

PURPOSE: To explore, from the perspective of Study Partners (SPs; eg, caregivers) of clinical trial participants with autism spectrum disorder (ASD), any changes experienced in socialization and communication over the clinical trial, how these changes manifested, and the impact these changes had on the autistic individual, the SP, and family. This helps interpret whether changes in trial outcomes were meaningful. PATIENTS AND METHODS: Interviews were conducted with the SPs of individuals with ASD, without intellectual disability, from 2 clinical trials: 86 children (aged 5-12 years) or adolescents (aged 13-17 years) who took part in the aV1ation trial (83...

Myasthenia gravis (MG) is an autoimmune ion channel disorder in which antibodies to different end-plate antigens impair neuromuscular transmission, ultimately leading to muscle weakness and fatigability. In about 85% of patients with MG, autoantibodies against the acetylcholine receptor (AChR) activate the complement cascade, causing damage to the neuromuscular junction. MG is a chronic disorder for which standard therapies with corticosteroids, immunosuppressive drugs, and immunomodulation with plasma exchange or intravenous immunoglobulins modify the course of the disease, but the residual burden of physical, psychological, and social disability highlights several unmet needs, among these the need for specific, targeted, and well tolerated therapies able to improve the patients' quality of life...

INTRODUCTION: Parkinson's disease (PD) is a chronic and progressive neurodegenerative condition due to the degeneration of nigral dopaminergic cells. Both motor and non-motor symptoms (NMS) of PD produce a marked impairment in PD patients' quality of life (QoL), but contrary to motor features, NMS do not improve with dopamine replacement. Novel therapeutic interventions for PD have successfully controlled most motor manifestations of PD, but the management of NMS is still challenging...

PURPOSE: To describe the processes of developing domains and items for the MultiMorbidity Questionnaire (MMQ), a multimorbidity-specific PROM for the assessment of Needs-based QoL. PATIENTS AND METHODS: We developed items and domains for the MMQ through 17 qualitative content validity questionnaire interviews with adults with multimorbidity by testing items from an item bank (covering items with content inspired by existing Needs-based QoL measures for single diseases)...

PURPOSE: The purpose of this secondary data analysis was to examine the association between hematologic response and health-related quality of life (HRQoL) among patients with light-chain (AL) amyloidosis. PATIENTS AND METHODS: Data for this secondary analysis were collected through a non-interventional, longitudinal, online self-report survey of patients with AL amyloidosis. Patients completed an initial online survey, with follow-up surveys administered 1, 6, 12, 18, and 24 months after completion of the initial survey...

[This corrects the article DOI: 10.2147/PROM.S151143.].