Patient Related Outcome Measures最新文献

Objective: The objective of this pilot study was to examine the agreement between child self- and parent proxy-assessment of health-related quality of life (HRQoL) in spinal muscular atrophy (SMA) in the era of disease-modifying therapy.

Methods: Children with SMA and one of their parents were recruited via the German national TREAT-NMD SMA patient registry. HRQoL was measured using the Pediatric Quality of Life Inventory 3.0 Neuromuscular Module (PedsQL 3.0 NMM), KIDSCREEN-27, and the Health Utilities Index (HUI). Agreement between child self- and parent proxy-ratings of ordinal measures was estimated using Cohen's κ, and for continuous measures using intraclass correlation coefficients (ICCs) from one-way random-effects models.

Results: The final sample comprised 17 children with SMA (mean age: 9.88 years, SD: 4.33 years, range: 5-16 years; 59% female) and one of their parents. All but two patients (88%) were receiving disease-modifying therapy (nusinersen or risdiplam). The ICC for the total PedsQL 3.0 NMM score was estimated at 0.85 (95% CI: 0.64-0.94, p < 0.001) (indicative of excellent agreement). The corresponding estimate for the KIDSCREEN total score was 0.27 (95% CI: 0.00-0.75, p = 0.197) (poor/fair agreement) and the global HUI utility 0.98 (95% CI: 0.93 to 0.9952, p < 0.001) (excellent agreement). The lowest levels of concordance were found for school and family life, as well as mental well-being, as opposed to physical functioning and disability.

Conclusion: We show that the agreement between child self- and parent proxy-reports of HRQoL in SMA varies markedly across HRQoL measures and examined domains, ranging from poor/fair to excellent. Compared with previous research, agreement for the PedsQL 3.0 NMM was markedly higher in our contemporary cohort of patients treated with novel therapies. These preliminary findings will be helpful in informing the design of future research of HRQoL in SMA.

Purpose: Patient-reported outcome measures (PROMs) are relevant for assessing the bond between a mother and her child, both before and after childbirth. Several questionnaires have been developed with the Mother-to-Infant Bonding Scale (MIBS) prominent among them, as it is a valid and easy-to-administer questionnaire owing to its length. Even though, this PROM has been adapted in Japanese, Indonesian and Swedish, it remains to be translated into French. The objective of this study was to translate and cross-culturally adapt the MIBS into French (MIBS-Fr).

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 was rated between 1 to 10 according to the comprehension level by 11 mothers.

Results: The French version was very well understood with a mean level of comprehension of 9.7 ± 1.4 out of 10. The one-word descriptor used to express feelings in the original version was replaced by phrases in the French version for a better language adaptation. Slight modifications were made by the original developer, and back translations were found to be very consistent.

Conclusion: This study reports the development of a French version of the MIBS (MIBS-Fr) following the ISPOR's recommendations for the translation and intercultural adaptation of a questionnaire. The MIBS-Fr provides French-speaking healthcare professionals with a practical and standardized tool to assess mother-to-infant bonding, facilitating early identification of bonding difficulties and supporting appropriate interventions in postpartum care.

Purpose: Good health status has traditionally been linked to greater levels of patient satisfaction. With the emergence and integration of various medical modalities, including Homeopathy, Ayurveda, and other complementary and alternative medical treatments, patients are likely to have different satisfaction levels during their interaction with different practitioners. Amidst this diversity, the efficacy, accessibility, and cost-effectiveness of these treatment approaches have garnered significant attention. Hence, a tool was developed to assess the patient's interaction with Complementary and Alternative Medicine treatments specifically Homeopathy. The objective of this study is to evaluate the validity and reliability of the Patient Satisfaction Assessment Tool (PSAT) developed to assess patient interaction with homeopathic outreach services.

Methods: A new structured questionnaire was developed using a standardized procedure. After obtaining clearance from the scientific and ethics committee of the Central Council for Research in Homoeopathy, data was collected from consenting participants above 18 years of age attending the Dr. D.P. Rastogi Central Research Institute for Homoeopathy, Noida, Uttar Pradesh, India. Data was then computerized and analyzed by principal component analysis as the extraction method and orthogonal varimax as the rotation method.

Results: A total of 285 participants were enrolled for psychometric validation, and 254 participants were included in the final analyses after exclusion. The mean (±Standard Deviation) age of participants was 37.63 (±12.9) years (range 18-79). The final 34-item questionnaire was arranged into nine domains as per rotated component matrix analysis. The overall internal consistency of the final questionnaire, as calculated by Cronbach's alpha, was 0.79, and the measure of sampling adequacy was 0.85 (Kaiser-Meyer-Olkin test).

Conclusion: Initial results from the pilot tests suggest that Patient Satisfaction Assessment Tool (PSAT) is effective in capturing key aspects of patient satisfaction, from homeopathic clinical services which can guide future quality improvement initiatives in alternative medicine healthcare facilities.

Background: Long COVID (LC) is a clinical syndrome with persistent, fluctuating symptoms subsequent to COVID-19 infection. LC has significant detrimental effects on health-related quality of life (HRQoL), activities of daily living (ADL), and work productivity. Condition-specific patient-reported outcome measures (PROMs), such as the modified COVID-19 Yorkshire Rehabilitation Scale (C19-YRSm) do not provide the health utility data required for cost-utility analyses of LC interventions. The aim of this study was to derive a mapping algorithm for the C19-YRSm to enable health utilities to be generated from this PROM.

Methods: Data were collected from a large study evaluating LC services in the UK. A total of 1434 people with LC had completed both the C19-YRSm and the EQ-5D. Correlation and linear regression analyses were applied to determine items from the C19-YRSm and covariates for inclusion in the algorithm. Model fit, mean differences across the range of EQ-5D-3L utility scores, and Bland-Altman plots were evaluated. Responsiveness (standardised response mean; SRM) of the mapped utilities was investigated on a subset of participants with repeat assessments.

Results: There was a strong level of association between 8 items and one domain on the C19-YRSm with the EQ-5D single-item dimensions. Model fit was good (R² = 0.7). The mean difference between observed and mapped scores was <0.10 for the range from 0 to 1 indicating good targeting for positive values of the EQ-5D-3L. The SRM for the mapped EQ-5D-3L was 0.37 compared to 0.17 for the observed utility scores, suggesting the mapped EQ-5D-3L is more responsive to change.

Conclusion: A simple, responsive, and robust mapping algorithm was developed to generate enable EQ-5D-3L health utilities from the C19-YRSm. This will facilitate economic evaluations of LC interventions, treatment, and management, as well as further helping to describe and characterise patients with LC irrespective of any treatment and interventions.

Introduction: The COVID-19 Symptoms Daily Diary (CSDD) is a patient-reported outcome measure designed to assess the severity of core COVID-19 symptoms in clinical trials. The preliminary version of the CSDD was developed based on regulatory guidance and the hallmark COVID-19 symptoms identified by the CDC. This study aimed to evaluate CSDD content validity, to determine whether it is fit for the purpose of supporting efficacy endpoints in clinical trials of treatments for COVID-19. This research also sought to evaluate the appropriateness of a newly developed Pre-COVID-19 Symptoms Questionnaire.

Methods: A targeted literature review was completed to evaluate the relevance of concepts included in the diary and to identify any important symptoms that may have been missing. Hybrid (concept elicitation and cognitive debriefing) semistructured qualitative interviews were then conducted across 3 iterative rounds with 30 adults in the United States recently diagnosed with COVID-19.

Results: The CSDD included concepts that were most frequently reported by interview participants, including those identified as most bothersome and most important to treat. During cognitive debriefing, participants described the diary concepts as salient and reported the instructions, recall period, and response options as clear and appropriate. Only 2 of 15 CSDD items were modified across 3 interview rounds; specifically, definitions for the vomiting and diarrhea frequency items were clarified for consistent interpretation and response. Interview participants also reported general ease in understanding and responding to the Pre-COVID-19 questionnaire, with feedback resulting in only minor changes to the reference period and instructions.

Conclusion: The findings of the current study provide strong evidence for the content validity of the CSDD and the appropriateness of each of the items assessed. This rigorous evaluation (aligned with regulatory guidance) indicates that the CSDD is fit for the purpose of supporting efficacy endpoints in planned clinical trials evaluating medications for COVID-19 treatment.

Alpha-1 antitrypsin deficiency (AATD) is a rare cause of chronic lung and liver disease without its own patient reported-outcome measure (PROM). PROMs for Chronic Obstructive Pulmonary Disease (COPD) are commonly used instead, but AATD differs from COPD in several ways. We reviewed whether the PROMs used in the AATD literature adequately assess quality-of-life in these patients. 11 studies used PROMs as their primary outcomes; 21 included them as secondary outcomes. The St George's Respiratory Questionnaire (SGRQ) was the most commonly used PROM, used by 7 of the 11 primary outcome studies. Others included the COPD Assessment Tool, SF-36, LCOPD, EQ-5D, and the Chronic Respiratory Diseases Questionnaire. Several studies assessed SGRQ as being associated with respiratory disease severity as measured by FEV1% predicted, exacerbation rate, oxygen use and exercise tolerance. However, no studies used PROMs which included assessment of liver-related symptoms, other extra-pulmonary manifestations of AATD, or concerns related to genetics or finances. These factors are likely to have an impact on quality of life in AATD. A specific AATD-PROM is therefore required to holistically address the quality of life effects of an AATD diagnosis.

Purpose: Growth hormone deficiency (GHD) causes decreased growth rate in children, resulting in short stature in childhood and adulthood. Daily subcutaneous injections with growth hormone (GH) have been standard treatment. Newer weekly GH formulations now exist. This study estimates utilities associated with GHD treatment for both people with the disease and caregivers by employing time trade-off (TTO) methodology.

Methods: Three online surveys were conducted amongst the general population in the UK and Canada. Based on a pilot, data collection was conducted in two surveys only (Survey A and Survey B). In Survey A, adults aged ≥18 years evaluated health states as if they were receiving injections themselves. In Survey B, adults with a child <15 years evaluated health states as if they were administering injections to a child. The surveys assessed device complexity, injection frequency, injection pain, needle visibility and storage possibilities.

Results: 2026 and 2028 respondents completed Survey A and Survey B, respectively. Of these, 1782 respondents and 1678 respondents were valid for inclusion. Avoiding weekly injection pain was associated with a significant utility gain of 0.030 (95% CI 0.026-0.035, p<0.001) in Survey A and 0.044 (95% CI 0.038-0.051, p<0.001) in Survey B. Additionally, less complex injection devices and lower injection frequencies had a significant impact in both Survey A (0.020, 95% CI 0.016-0.025, p<0.001; 0.009, 95% CI 0.005-0.014, p<0.001) and Survey B (0.008, 95% CI 0.002-0.014, p=0.006; 0.009, 95% CI 0.003-0.014, p=0.003).

Conclusion: Several aspects are associated with a significant impact on utilities for people with GHD and potential caregivers. Treatment options without injection pain, a time-consuming and complex injection process and daily injections are expected to result in higher health-related quality of life. These results may inform future economic evaluations and treatment choices.

Purpose: Being a young carer of a parent with dementia can be challenging, with many carers undertaking various practical and caring tasks. The weekend course Time to be young? gathers young carers, aiming to support them to cope with their challenges in everyday life. The aim of this study was to explore their role as a caregiver and the experienced impact of the course on their strategies of coping in their everyday life.

Participants and methods: The study had a qualitative descriptive design inspired by Lindseth and Norberg's phenomenological hermeneutical method, using individual semi-structured interviews for data collection. The participants were recruited from former participants of the course Time to be young?, and the final sample included eight participants.

Results: Through the analysis, four main themes were identified: 1) Help to accept the situation, 2) A sense of community, 3) The need for information and 4) The need to live one's own life. The study found that attendance at Time to be young? for young adults having a parent with dementia affected their coping strategies in their situation as a young care.

Conclusion: The study demonstrated the importance of courses like Time to be young?, and need for a meeting place, tailored information about dementia, and an opportunity to share and reflect upon their experiences as a young carer.

Introduction: Polytrauma remains a major global health challenge, with rapid intervention being critical for survival, especially during the "Golden Hour". This study examines the impact of Helicopter Emergency Medical Services (HEMS) on procedural care during the transfer of polytraumatized patients to urban hospitals in Romania.

Methods: A retrospective cohort study was conducted at the County Emergency Hospital "St. Ap. Andrei" in Galați, covering January 2020 to October 2021. The study analyzed data from 89 patients transported by the Romania's Mobile Emergency Service for Resuscitation and Extrication (SMURD) Galați air unit. Key parameters included demographics, injury mechanism, vital statistics, and prehospital interventions. Statistical analyses were performed using SPSS, with significance set at p < 0.05.

Results: Out of 89 patients (mean age 21.6 years, 80.3% male), trauma causes were primarily traffic accidents (34.8%) and falls (33.7%). A Glasgow Coma Scale (GCS) score ≤8 was noted in 28.1% of cases, with head trauma observed in 51.6% of patients. HEMS interventions frequently involved oxygen therapy (89.5%) and patient stabilization maneuvers, leading to a mortality rate of 6.7%. Notably, helicopter transport enabled efficient transfer and improved survival outcomes in this cohort.

Discussion: HEMS demonstrated benefits in reducing intervention times and enhancing prehospital care quality for polytrauma patients, especially in hard-to-reach areas. The study aligns with global data on HEMS's role in trauma systems, underlining the importance of multidisciplinary collaboration and rapid transport.

Conclusion: HEMS plays a crucial role in improving survival rates for severely injured patients through timely interventions and specialized care. Further research comparing HEMS and ground services could refine trauma management protocols in similar settings.