RESUMEN
PURPOSE: For breast cancer survivors (BCS) living with breast cancer-related lymphedema (BCRL), what outcome domains (OD) should be measured to assess the burden of the disease and efficacy of interventions? A Core Outcome Set (COS) that promotes standardized measurement of outcomes within the constraints of time influenced by work environments is essential for patients and the multidisciplinary professionals that manage and research BCRL. METHODS: Using Delphi methodology, a multidisciplinary group of BCRL experts (physical and occupational therapists, physicians, researchers, physical therapist assistants, nurses, and massage therapist) completed two waves of online surveys. BCRL expert respondents that completed the first survey (n = 78) had an average of 26.5 years in practice, whereas, respondents who completed the second survey (n = 33) had an average of 24.9 years. ODs were included in the COS when consensus thresholds, ranging from 70% to 80%, were met. RESULTS: A total of 12 ODs made up the COS. Reaching a minimum consensus of 70%; volume, tissue consistency, pain, patient-reported upper quadrant function, patient-reported health-related quality of life, and upper extremity activity and motor control were recommended at different phases of the BCRL continuum in a time-constrained environment. Joint function, flexibility, strength, sensation, mobility and balance, and fatigue met an 80% consensus to be added when time and resources were not constrained. CONCLUSION: The COS developed in this study thoroughly captures the burden of BCRL. Using this COS may reduce selective reporting, inconsistency in clinical use, and variability of reporting across interdisciplinary healthcare fields, which manage or research BCRL.
Asunto(s)
Linfedema del Cáncer de Mama , Supervivientes de Cáncer , Técnica Delphi , Calidad de Vida , Humanos , Femenino , Linfedema del Cáncer de Mama/terapia , Linfedema del Cáncer de Mama/etiología , Neoplasias de la Mama/complicaciones , Encuestas y Cuestionarios , Medición de Resultados Informados por el Paciente , Evaluación de Resultado en la Atención de Salud/métodos , Persona de Mediana EdadRESUMEN
PURPOSE: For breast cancer survivors (BCS) living with breast cancer-related lymphedema (BCRL), what outcome measures (OMs) are recommended to be used to measure standardized outcome domains to fully assess the burden of the disease and efficacy of interventions? An integral component of a standardized core outcome set (COS) are the OMs used to measure the COS. METHODS: A supplemental online survey was linked to a Delphi study investigating a COS for BCRL. OMs were limited to a maximum of 10 options for each outcome domain (OD). There were 14 ODs corresponding to the International Classification of Functioning, Disability, and Health (ICF) framework and respondents rated the OMs with a Likert level of recommendation. The feasibility of the listed OMs was also investigated for most outpatient, inpatient, and research settings. RESULTS: This study identified 27 standardized OMs with a few ODs having 2-3 highly recommended OMs for proper measurement. A few of the recommended OMs have limitations with reliability due to being semi-quantitative measures requiring the interpretation of the rater. CONCLUSION: Narrowing the choices of OMs to 27 highly recommended by BCRL experts may reduce selective reporting, inconsistency in clinical use, and variability of reporting across interdisciplinary healthcare fields which manage or research BCRL. There is a need for valid, reliable, and feasible OMs that measure tissue consistency. Measures of upper extremity activity and motor control need further research in the BCS with BCRL population.
Asunto(s)
Linfedema del Cáncer de Mama , Supervivientes de Cáncer , Técnica Delphi , Evaluación de Resultado en la Atención de Salud , Humanos , Femenino , Linfedema del Cáncer de Mama/terapia , Linfedema del Cáncer de Mama/diagnóstico , Linfedema del Cáncer de Mama/etiología , Evaluación de Resultado en la Atención de Salud/métodos , Neoplasias de la Mama/complicaciones , Encuestas y Cuestionarios , Calidad de Vida , Persona de Mediana Edad , Reproducibilidad de los ResultadosRESUMEN
OBJECTIVE: To assess the degree of core outcome set alignment and identify issues with alignment to the 2019 COS among clinical trial registrations focused on knee and/or hip osteoarthritis (OA). METHODS: Our search was performed on registered knee and hip OA randomized controlled trials (RCTs) available on ClinicalTrials.gov and WHO International Clinical Trials Registry Platform. The screening process considered trials registered between 8/2014 and 6/2023. We extracted data on general trial characteristics and the five trial endpoints detailed in the COS (pain, physical function, quality of life, patient global assessment, and adverse events), in a masked and duplicate manner. The frequencies of COS alignment were assessed over time prior to and after COS publication. RESULTS: Of the 10,718 RCTs screened, 481 met inclusion criteria. Most were phase 3 (368/481, 76.51%) and heavily university-funded (184/481, 38.25%). Despite the 2019 COS, no marked enhancement in overall alignment was noted. The outcome 'Pain' exhibited the highest degree of COS alignment (455/481, 94.59%), whereas 'adverse events' lagged behind (89/481, 18.50%). Additionally, trial factors such as 'Continent', 'Funding Type', and 'Recruitment Status' displayed no significant influence on COS alignment. CONCLUSIONS: Despite the acknowledged advantages of using COS in RCTs and the availability of an updated COS, the alignment to these outcomes remains notably low. Significant efforts are needed to encourage broader adoption in future studies on knee and hip OA, with the aim of improving research quality and patient care.
Asunto(s)
Osteoartritis de la Cadera , Osteoartritis de la Rodilla , Ensayos Clínicos Controlados Aleatorios como Asunto , Humanos , Estudios Transversales , Calidad de Vida , Evaluación de Resultado en la Atención de SaludRESUMEN
OBJECTIVES: There is growing interest in collecting outcome information directly from patients in clinical trials. This study evaluates what patients with rheumatic and musculoskeletal diseases (RMDs) consider important to know about symptomatic side effects they may experience from a new prescription drug. METHODS: Patients with inflammatory arthritis, who had one or more prescribed drugs for their disease for at least 12 months, participated in focus groups and individual interviews. Discussions were analysed using reflexive thematic analysis. RESULTS: We conducted seven focus groups with 34 participants across three continents. We found four overarching and two underpinning themes. The 'impact on life' was connected to participants 'daily life', 'family life', 'work life', and 'social life'. In 'psychological and physical aspects' participants described 'limitation to physical function', 'emotional dysregulation' and 'an overall mental state'. Extra tests, hospital visits and payment for medication were considered a 'time, energy and financial burden' of side effects. Participants explained important measurement issues to be 'severity', 'frequency', and 'duration'. Underpinning these issues, participants evaluated the 'benefit-harm-balance' which includes 'the cumulative burden' of having several side effects and the persistence of side effects over time. CONCLUSIONS: In treatment for RMDs, there seems to be an urgent need for feasible measures of patient-reported bother (impact on life and cumulative burden) from side effects and the benefit-harm-balance. These findings contribute new evidence in support of a target domain-an outcome that represents the patient voice evaluating the symptomatic treatment-related side effects for people with RMDs enrolled in clinical trials.
RESUMEN
BACKGROUND: IgE-mediated food allergy (FA) is a global health concern with substantial individual and societal implications. While diverse intervention strategies have been researched, inconsistencies in reported outcomes limit evaluations of FA treatments. To streamline evaluations and promote consistent reporting, the Core Outcome Measures for Food Allergy (COMFA) initiative aimed to establish a Core Outcome Set (COS) for FA clinical trials and observational studies of interventions. METHODS: The project involved a review of published clinical trials, trial protocols and qualitative literature. Outcomes found as a result of review were categorized and classified, informing a two-round online-modified Delphi process followed by hybrid consensus meeting to finalize the COS. RESULTS: The literature review, taxonomy mapping and iterative discussions with diverse COMFA group yielded an initial list of 39 outcomes. The iterative online and in-person meetings reduced the list to 13 outcomes for voting in the formal Delphi process. One more outcome was added based on participant suggestions after the first Delphi round. A total of 778 participants from 52 countries participated, with 442 participating in both Delphi rounds. No outcome met a priori criteria for inclusion, and one was excluded as a result of the Delphi. Thirteen outcomes were brought to the hybrid consensus meeting as a result of Delphi and two outcomes, 'allergic symptoms' and 'quality of life' achieved consensus for inclusion as 'core' outcomes. CONCLUSION: In addition to the mandatory reporting of adverse events for FA clinical trials or observational studies of interventions, allergic symptoms and quality of life should be measured as core outcomes. Future work by COMFA will define how best to measure these core outcomes.
Asunto(s)
Hipersensibilidad a los Alimentos , Calidad de Vida , Humanos , Técnica Delphi , Hipersensibilidad a los Alimentos/diagnóstico , Hipersensibilidad a los Alimentos/terapia , Inmunoglobulina E , Evaluación de Resultado en la Atención de Salud , Proyectos de Investigación , Resultado del Tratamiento , Ensayos Clínicos como Asunto , Estudios Observacionales como AsuntoRESUMEN
BACKGROUND: Adolescents and young adults (AYAs) with cancer, defined as individuals aged 15-39 years at initial cancer diagnosis, form a unique population; they face age-specific issues as they transition to adulthood. This paper presents the protocol for the development of a core outcome set (COS) for AYAs with cancer. METHODS: The methodological standards from the Core Outcome Measures in Effectiveness Trials (COMET) and the International Consortium for Health Outcomes Measurement (ICHOM) for COS development will guide the development of the COS for AYAs with cancer. The project will consist of the following phases: (1) define the scope of the COS; (2) establish the need for a COS in this field (3) assemble an international, multi-stakeholder working group; (4) develop a detailed protocol; (5) determine "what to measure" (i.e., outcomes); (6) determine "how to measure" (i.e., measures); and (7) determine "case-mix" variables. CONCLUSIONS: The development of a COS for AYAs with cancer will facilitate the implementation of efficient and relevant standards for data collection, both for clinical trials and in routine healthcare, thereby increasing the usefulness of these data to improve the value of the care given to these underserved young cancer patients.
Asunto(s)
Instituciones de Salud , Neoplasias , Humanos , Adolescente , Adulto Joven , Recolección de Datos , Neoplasias/diagnóstico , Neoplasias/terapia , Poblaciones VulnerablesRESUMEN
INTRODUCTION: Given the lack of data, we aimed to explore which therapeutic endpoints pediatric patients with eosinophilic esophagitis (EoE) and their parents consider to be relevant. METHODS: We created an educational brochure on EoE and a questionnaire, both of which were content-validated by pediatric patients and parents. Validated documents were sent to 112 patients and parents. They ranked the importance (5 levels) of short (during next 3 months) and long-term (≥1 year) treatment effect on symptoms, quality of life, endoscopic inflammation, stricture formation, histological inflammation, and fibrosis. RESULTS: A total of 45 parents and 30 pediatric patients ≥11 years completed the questionnaires. Pediatric patients identified improvement in the following domains as most important in the short- and long-term, respectively: symptoms (73% vs. 77%), QoL (53% vs. 57%), histologic inflammation (47% vs. 50%), histologic fibrosis (40% vs. 33%), endoscopic inflammation (47% vs. 40%), and strictures (33% vs. 40%). Parents of children ≥11 years old classified improvement in the following domains as most important in the short- and long-term, respectively: symptoms (70% vs. 83%), QoL (63% vs. 80%), histologic inflammation (67% vs. 77%), histologic fibrosis (47% vs. 63%), endoscopic inflammation (77% vs. 80%), and strictures (40% vs. 53%). Agreement between caregiver and children on the short-term importance of treatment outcomes was as follows: symptoms (77%), QoL (40%), histologic inflammation and fibrosis (47% and 43%), endoscopic inflammation and strictures (50% and 40%). CONCLUSION: Pediatric patients and parents attributed most importance to improvement in symptoms and QoL. Agreement between parents and patients regarding therapy goals is limited.
Asunto(s)
Esofagitis Eosinofílica , Padres , Calidad de Vida , Humanos , Esofagitis Eosinofílica/terapia , Esofagitis Eosinofílica/diagnóstico , Padres/psicología , Niño , Encuestas y Cuestionarios , Masculino , Femenino , Resultado del Tratamiento , Adolescente , PreescolarRESUMEN
AIMS: Describe the outcomes reported in research on health systems interventions for type 1 diabetes management in comparison to the outcomes proposed by a core outcome set (COS) for this condition, an essential list of outcomes that studies should measure. METHODS: Systematic search of studies published between 2010 and 2021 reporting health systems interventions directed to improve the management of type 1 diabetes using PubMed, EMBASE and CENTRAL. Information on the outcomes was extracted and classified according to a COS: self-management, level of clinical engagement, perceived control over diabetes, diabetes-related quality of life, diabetes burden, diabetes ketoacidosis, severe hypoglycemia, and glycated hemoglobin (HbA1C). RESULTS: 187 studies were included. Most of the studies included either children (n = 82/187) or adults (n = 82/187) living with type 1 diabetes. The most common outcome measured was HbA1C (n = 149/187), followed by self-management (n = 105/187). While the least measured ones were diabetes ketoacidosis (n = 15/187), and clinical engagement (n = 0/187). None of the studies measured all the outcomes recommended in the COS. Additionally, different tools were found to be used in measuring the same outcome. CONCLUSIONS: This study provides a description of what researchers are measuring when assessing health systems interventions to improve type 1 diabetes management. In contrast to a COS, it was found that there is a predominance of clinical-based outcomes over patient-reported outcome measures.
Asunto(s)
Diabetes Mellitus Tipo 1 , Cetoacidosis Diabética , Hipoglucemia , Adulto , Niño , Humanos , Diabetes Mellitus Tipo 1/terapia , Calidad de Vida , Hemoglobina Glucada , Cetoacidosis Diabética/prevención & controlRESUMEN
BACKGROUND: People with severe mental illness (SMI), such as schizophrenia, have higher rates of type 2 diabetes and worse outcomes, compared to those without SMI and it is not known whether diabetes self-management interventions are effective for people who have both conditions. Research in this area has been impeded by a lack of consensus on which outcomes to prioritise in people with co-existing SMI and diabetes. AIMS: To develop a core outcome set (COS) for use in effectiveness trials of diabetes self-management interventions in adults with both type 2 diabetes and SMI. METHODS: The COS was developed in three stages: (i) identification of outcomes from systematic literature review of intervention studies, followed by multi-stakeholder and service user workshops; (ii) rating of outcomes in a two-round online Delphi survey; (iii) agreement of final 'core' outcomes through a stakeholder consensus workshop. RESULTS: Seven outcomes were selected: glucose control, blood pressure, body composition (body weight, BMI, body fat), health-related quality of life, diabetes self-management, diabetes-related distress and medication adherence. CONCLUSIONS: This COS is recommended for future trials of effectiveness of diabetes self-management interventions for people with SMI and type 2 diabetes. Its use will ensure trials capture important outcomes and reduce heterogeneity so findings can be readily synthesised to inform practice and policy.
Asunto(s)
Diabetes Mellitus Tipo 2 , Trastornos Mentales , Automanejo , Humanos , Diabetes Mellitus Tipo 2/complicaciones , Diabetes Mellitus Tipo 2/terapia , Automanejo/métodos , Trastornos Mentales/terapia , Trastornos Mentales/complicaciones , Calidad de Vida , Técnica Delphi , Cumplimiento de la Medicación , Evaluación de Resultado en la Atención de Salud , Esquizofrenia/complicaciones , Esquizofrenia/terapia , Control Glucémico/métodos , Autocuidado/métodos , MasculinoRESUMEN
BACKGROUND: Previous systematic reviews have revealed an inconsistency of outcome definitions as a major barrier in providing evidence-based guidance for the use of plasma transfusion to prevent or treat bleeding. We reviewed and analyzed outcomes in randomized controlled trials (RCTs) to provide a methodology for describing and classifying outcomes. STUDY DESIGN AND METHODS: RCTs involving transfusion of plasma published after 2000 were identified from a prior review (Yang 2012) and combined with an updated systematic literature search of multiple databases (July 1, 2011 to January 17, 2023). Inclusion of publications, data extraction, and risk of bias assessments were performed in duplicate. (PROSPERO registration number is: CRD42020158581). RESULTS: In total, 5579 citations were identified in the new systematic search and 22 were included. Six additional trials were identified from the previous review, resulting in a total of 28 trials: 23 therapeutic and five prophylactic studies. An increasing number of studies in the setting of major bleeding such as in cardiovascular surgery and trauma were identified. Eighty-seven outcomes were reported with a mean of 11 (min-max. 4-32) per study. There was substantial variation in outcomes used with a preponderance of surrogate measures for clinical effect such as laboratory parameters and blood usage. CONCLUSION: There is an expanding literature on plasma transfusion to inform guidelines. However, considerable heterogeneity of reported outcomes constrains comparisons. A core outcome set should be developed for plasma transfusion studies. Standardization of outcomes will motivate better study design, facilitate comparison, and improve clinical relevance for future trials of plasma transfusion.
Asunto(s)
Transfusión de Componentes Sanguíneos , Hemorragia , Plasma , Ensayos Clínicos Controlados Aleatorios como Asunto , Humanos , Hemorragia/terapia , Hemorragia/prevención & control , Hemorragia/etiología , Resultado del TratamientoRESUMEN
BACKGROUND: There is an inconsistency in treatment outcomes used in clinical trials for provoked vestibulodynia (PVD), which makes it impossible to compare the effects of different interventions. AIM: In this study, we completed the first step in creating a core outcome set (COS), defining what outcomes should be measured in clinical trials for PVD. METHODS: Identification of outcomes used in studies was done by extracting data from clinical trials in a recently published systematic review and via review of clinical trials for PVD registered on ClinicalTrials.gov. The COS process consisted of 2 rounds of Delphi surveys and a consensus meeting, during which the final COS was decided through a modified nominal group technique. OUTCOMES: Consensus on what outcomes to include in a COS for PVD. RESULTS: Forty scientific articles and 92 study protocols were reviewed for outcomes. Of those, 36 articles and 25 protocols were eligible, resulting in 402 outcomes, which were then categorized into 63 unique outcomes. Participants consisted of patients, relatives/partners of patients, health care professionals, and researchers. Out of 463 who registered for participation, 319 and 213 responded to the first and second surveys, respectively. The consensus meeting consisted of 18 members and resulted in 6 outcomes for the COS to be measured in all treatment trials regardless of intervention: insertional pain (nonsexual), insertional pain (sexual), provoked vulvar pain by pressure/contact, pain-related interference on one's life, pain interference on sexual life, and sexual function. CLINICAL IMPLICATIONS: Critical outcomes to be measured in clinical trials will allow for accurate comparison of outcomes across treatment interventions and provide solid treatment recommendations. STRENGTHS AND LIMITATIONS: The major strengths of the study are the adherence to methodological recommendations and the intentional focus on aspects of diversity of participating stakeholders (eg, status such as patients with lived experience and researchers, inclusiveness with respect to sexual identity), the latter of which will allow for broader application and relevance of the COS. Among the limitations of the study are the low rate of participants outside North America and Europe and the lower response rate (about 50%) for the second Delphi survey. CONCLUSION: In this international project, patients, health care professionals, and researchers have decided what critical outcomes are to be used in future clinical trials for PVD. Before the COS can be fully implemented, there is also a need to decide on how and preferably when the outcomes should be measured.
Asunto(s)
Técnica Delphi , Vulvodinia , Humanos , Vulvodinia/terapia , Femenino , Evaluación de Resultado en la Atención de Salud , Consenso , Resultado del Tratamiento , Ensayos Clínicos como Asunto , Adulto , Proyectos de InvestigaciónRESUMEN
OBJECTIVE: Technology and advances in clinical care have changed the management of abdominal aortic aneurysms (AAAs) but the clinical effectiveness of continuing advances needs to be assessed. To facilitate rapid synthesis of new evidence and improve stakeholder representation, including patients, the concept of core outcome sets (COS) has been developed. COS, reflecting the needs of all stakeholders, have been established across several surgical specialties. This study aimed to develop an international core outcome set for intact AAA repair. METHODS: Following COMET methodology, potential outcomes were identified from a systematic review of published outcomes and focus groups involving patients, carers, and nurses. A 38 question Delphi consensus survey in lay language was developed (with translation to local languages); this included 35 themes identified from the findings of the systematic review and three themes from the focus groups. All three of the themes identified by the focus groups (cognitive, physical, and social functioning) can be evaluated from quality of life instruments, with overall quality of life being identified from the systematic review. The survey was completed by patients, carers or family members, vascular nurses, vascular surgeons, trainees, interventional radiologists, anaesthetists, and industry partners from six European countries. After two rounds of the survey, the top outcomes were discussed at a face to face multistakeholder consensus meeting. RESULTS: The 38 item questionnaire was amended after piloting among all stakeholder groups. After the first round of the Delphi survey (98 respondents) 15 questions were eliminated, and 11 further questions were eliminated after round 2 (90 respondents). This left two outcome questions for discussion at the consensus meeting, where the top six outcomes were unanimously endorsed: death at 30 days (or in hospital if longer), secondary AAA rupture, overall quality of life and retention of cognitive functioning after recovery, five year survival, and continued sac growth. CONCLUSION: Six core outcomes are recommended for use as a minimum framework in all future studies and registries of intact open and endovascular AAA repair. Further work to select instruments for quality of life and to define instruments for cognitive functioning is needed.
RESUMEN
OBJECTIVE: Inconsistencies in outcome data of therapeutic strategies for acute lower limb ischaemia (ALI) have hindered the synthesis of findings. A core outcome set (COS) may offer a solution to this problem by defining a minimum set of outcomes that are considered essential to all stakeholders involved. The first step in developing a COS is to review the previously reported outcomes on various treatment strategies for ALI. DATA SOURCES: PubMed, Embase, Cochrane Central Register of Controlled Trials (CENTRAL), and Web of Science databases were searched from inception to August 2023. REVIEW METHODS: This systematic review was conducted in accordance with the Core Outcome Measures in Effectiveness Trials (COMET) initiative framework, adhering to Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, and was pre-registered with PROSPERO (CRD42022320073). Abstracts were independently screened by two authors for full text review. All outcomes and their definitions were extracted from selected papers. Outcomes with different terminologies were then categorised into an "agreed outcome term". The list of agreed outcomes was given a standardised outcome domain and core area using a 38 item standardised taxonomy. RESULTS: Of 6 184 articles identified, 176 relevant studies were included, yielding 1 325 verbatim outcomes. After deduplication, 72 unique verbatim outcomes were categorised into five broad outcome domains. Outcomes considered key to the evaluation of treatment of ALI were further categorised as delivery of care (19.4%), vascular outcomes (13.8%), and adverse events (12.5%). The three most frequently reported agreed outcomes were amputation (14.1%), mortality (12.3%), and general bleeding (11.6%). CONCLUSION: This systematic review provides an overview of currently reported outcomes in the literature of interventions for ALI. After categorisation into agreed outcome terms, 72 outcomes were identified that can be used in the development of a COS.
RESUMEN
BACKGROUND: The Core Outcome Measures in Effectiveness Trials (COMET) working group proposed core outcome sets (COS) to address the heterogeneity in outcome measures in clinical studies. According to the recommendations of COMET, performing systematic reviews (SRs) usually was the first step for COS development. However, the SRs that serve as a basis for COS are not specifically appraised by organizations such as COMET regarding their quality. Here, we investigated the status of SRs related to development of COS and evaluated their methodological quality. METHODS: We conducted a search on PubMed to identify SRs related to COS development published from inception to May 2022. We qualitatively summarized the disease included in SR topics, and the studies included in the SRs. We evaluated the methodological quality of the SRs using AMSTAR 2.0 and compared the overall quality of SRs with and without protocols using the Mann-Whitney U test. RESULTS: We included 175 SRs from 23 different countries or regions, and they mainly focused on five diseases: musculoskeletal system or connective tissue disease (n = 19, 10.86%), injury, poisoning, or certain other consequences of external causes (n = 18, 10.29%), digestive system disease (n = 16, 9.14%), nervous system disease (n = 15, 8.57%), and genitourinary system disease (n = 15, 8.57%). Although 88.00% of SRs included randomized controlled trials (RCTs), only a few SRs (23.38%) employed appropriate tools to assess the risk of bias in RCTs. The assessment results on the basis of AMSTAR 2.0 indicated that most SRs (93.71%) were rated as ''critically low'' to ''low'' in terms of overall confidence. The overall confidence of SRs with protocols was significantly higher than that without protocols (P <.001). Compared to the SRs with protocols on Core Outcome Measures in Effectiveness Trials (COMET), SRs with protocols on PROSPERO were of better overall confidence (P = .017). CONCLUSION: The overall quality of published SRs regarding COS development was poor. Our findings emphasize the need for researchers to carefully select the disease topic and strictly adhere to the requirements of optimal methodology when conducting a SR for the establishment of a COS.
Asunto(s)
Evaluación de Resultado en la Atención de Salud , Proyectos de Investigación , Humanos , Revisiones Sistemáticas como Asunto , SesgoRESUMEN
Prehabilitation aims to optimise patients' physical and psychological status before treatment. The types of outcomes measured to assess the impact of prehabilitation interventions vary across clinical research and service evaluation, limiting the ability to compare between studies and services and to pool data. An international workshop involving academic and clinical experts in cancer prehabilitation was convened in May 2022 at Sheffield Hallam University's Advanced Wellbeing Research Centre, England. The workshop substantiated calls for a core outcome set to advance knowledge and understanding of best practice in cancer prehabilitation and to develop national and international databases to assess outcomes at a population level.
Asunto(s)
Neoplasias , Ejercicio Preoperatorio , Humanos , Consenso , Neoplasias/cirugía , Terapia por Ejercicio , Evaluación de Resultado en la Atención de SaludRESUMEN
BACKGROUND: Postsurgical outcome measures are crucial to define the efficacy of perioperative pain management; however, it is unclear which are most appropriate. We conducted a prospective study aiming to assess sensitivity-to-change of patient-reported outcome measures assessing the core outcome set of domains pain intensity (at rest/during activity), physical function, adverse events, and self-efficacy. METHODS: Patient-reported outcome measures were assessed preoperatively, on day 1 (d1), d3, and d7 after four surgical procedures (total knee replacement, breast surgery, endometriosis-related surgery, and sternotomy). Primary outcomes were sensitivity-to-change of patient-reported outcome measures analysed by correlating their changes (d1-d3) with patients' global impression of change and patients' specific impression of change items as anchor criteria. Secondary outcomes included identification of baseline and patient characteristic variables explaining variance in change for each of the scales and descriptive analysis of various patient-reported outcome measures from different domains and after different surgeries. RESULTS: Of 3322 patients included (18 hospitals, 10 countries), data from 2661 patients were analysed. All patient-reported outcome measures improved on average over time; the median calculated sensitivity-to-change for all patient-reported outcome measures (overall surgeries) was 0.22 (range: 0.07-0.31, scale: 0-10); all changes were independent of baseline data or patient characteristics and similar between different procedures. CONCLUSIONS: Pain-related patient-reported outcome measures have low to moderate sensitivity-to-change; those showing higher sensitivity-to-change from the same domain should be considered for inclusion in a core outcome set of patient-reported outcome measures to assess the effectiveness and efficacy of perioperative pain management.
Asunto(s)
Dolor Agudo , Femenino , Humanos , Estudios Prospectivos , Evaluación de Resultado en la Atención de Salud , Dolor Postoperatorio/diagnóstico , Medición de Resultados Informados por el PacienteRESUMEN
AIM: Faecal incontinence (FI) is a prevalent issue which can have a detrimental impact on the patient's quality of life. Current international guidelines lack strong treatment recommendations due to few studies in the field, in combination with the heterogeneity in outcome reporting. To address this, a core outcome set (COS) is proposed to standardize outcome reporting in FI studies, facilitating meta-analyses and enhancing therapeutic recommendations. Through several steps outlined by COMET 'what' to measure will be determined prior to determining 'how' to measure these outcomes. This systematic review aims to identify 'what' outcomes have been used in FI intervention studies so far in adult patients as a starting phase for the development of a future COS for FI. METHOD: Medline, Embase and the Cochrane library were searched to identify all outcomes reported in comparative effectiveness trials assessing one or more treatment option in adult patients suffering from FI. The outcomes were categorized according to the Core Outcome Measurement in Effectiveness Trials (COMET) taxonomy to standardize outcome terminology, assess completeness, and inform subsequent steps in COS development. RESULTS: A total of 109 studies were included, which revealed 51 unique outcomes classified into 38 domains within four core areas. On average four outcomes were reported per study (range 1-11). The most commonly reported outcomes were "severity of FI" (83%), "quality of life" (64%), "number of FI episodes" (40%), "anorectal motor function" (39%), and "frequency of bowel movements" (16%). CONCLUSION: This systematic review offers an overview of outcomes reported in FI studies, highlighting the heterogeneity between studies. This heterogeneity emphasizes the need for standardizing outcome reporting which can be established through the creation of a COS.
Asunto(s)
Incontinencia Fecal , Calidad de Vida , Incontinencia Fecal/terapia , Humanos , Evaluación de Resultado en la Atención de Salud/métodos , Resultado del Tratamiento , Femenino , Adulto , MasculinoRESUMEN
BACKGROUND: Generating rigorous evidence to inform care for rare diseases requires reliable, sustainable, and longitudinal measurement of priority outcomes. Having developed a core outcome set for pediatric medium-chain acyl-CoA dehydrogenase (MCAD) deficiency, we aimed to assess the feasibility of prospective measurement of these core outcomes during routine metabolic clinic visits. METHODS: We used existing cohort data abstracted from charts of 124 children diagnosed with MCAD deficiency who participated in a Canadian study which collected data from birth to a maximum of 11 years of age to investigate the frequency of clinic visits and quality of metabolic chart data for selected outcomes. We recorded all opportunities to collect outcomes from the medical chart as a function of visit rate to the metabolic clinic, by treatment centre and by child age. We applied a data quality framework to evaluate data based on completeness, conformance, and plausibility for four core MCAD outcomes: emergency department use, fasting time, metabolic decompensation, and death. RESULTS: The frequency of metabolic clinic visits decreased with increasing age, from a rate of 2.8 visits per child per year (95% confidence interval, 2.3-3.3) among infants 2 to 6 months, to 1.0 visit per child per year (95% confidence interval, 0.9-1.2) among those ≥ 5 years of age. Rates of emergency department visits followed anticipated trends by child age. Supplemental findings suggested that some emergency visits occur outside of the metabolic care treatment centre but are not captured. Recommended fasting times were updated relatively infrequently in patients' metabolic charts. Episodes of metabolic decompensation were identifiable but required an operational definition based on acute manifestations most commonly recorded in the metabolic chart. Deaths occurred rarely in these patients and quality of mortality data was not evaluated. CONCLUSIONS: Opportunities to record core outcomes at the metabolic clinic occur at least annually for children with MCAD deficiency. Methods to comprehensively capture emergency care received at outside institutions are needed. To reduce substantial heterogeneous recording of core outcome across treatment centres, improved documentation standards are required for recording of recommended fasting times and a consensus definition for metabolic decompensations needs to be developed and implemented.
Asunto(s)
Errores Innatos del Metabolismo Lipídico , Evaluación de Resultado en la Atención de Salud , Niño , Humanos , Acil-CoA Deshidrogenasa , Canadá , Estudios Prospectivos , PreescolarRESUMEN
BACKGROUND: Establishing the most important outcomes for school-based speech-language therapy is essential to guide future research and program evaluation for these services. Many health disciplines have developed core outcomes sets (COS) for this purpose. A COS encompasses the most important outcomes for particular health services as identified by appropriate interested parties. These interested parties usually represent health care providers and those with the health condition. In this paper, we report the development of a guiding framework for a COS for speech-language therapy services in schools in a Canadian context. METHODS: Using a group concept mapping method, we identified the outcomes for inclusion in the COS guiding framework through the elicited opinions of key interested parties: speech-language therapists, teachers, and family members of children with speech, language, and communication needs. We extracted 103 statements (potential outcomes) from a previous data set of interview transcripts. We then asked participants to sort the statements into conceptually similar groups, which were aggregated and transformed into a cluster map using multidimensional scaling followed by hierarchical cluster analysis. Participants also rated each statement on 5-point scales for importance and feasibility. We calculated mean ratings for individual statements and for all statements in a cluster, for all participants and for participant groups separately. RESULTS: We identified seven core outcomes for school-based speech-language services in Ontario, Canada. These included: classroom-based services, a holistic approach, support for teachers, care coordination, accessible services, family supports, and student success. All outcomes were rated highly for importance. Feasibility ratings were consistently below importance ratings. All participant groups concurred that a holistic approach was the most important outcome and accessible services was the least feasible outcome to achieve. CONCLUSIONS: The seven outcomes identified in this study are recommended to guide the development of a full COS to direct future research and program evaluation for school-based speech-language services. These outcomes have not been widely included in previous research and should be incorporated into future research alongside specific intervention outcomes. Data for some outcomes may be available from non-traditional sources such as administrative data sets. Consequently, their use for program evaluations should be accompanied by appropriate institutional support to allow speech-language therapists to make meaningful use of appropriate outcomes data.
Asunto(s)
Logopedia , Habla , Niño , Humanos , Ontario , Instituciones Académicas , Evaluación de Resultado en la Atención de SaludRESUMEN
This scoping review was conducted to identify the outcomes and measurement tools used in IC intervention studies, as first step towards the development of a core outcome set (COS) for IC trials. PRISMA-ScR and COS-STAD were followed. The review considered randomized controlled trials targeting IC published in Medline, Scopus, Embase, Cochrane Central Register of Controlled Trials, and clinicaltrials.gov, until June 2023. Of 699 references, 534 studies were screened once duplicates were removed, 15 were assessed for eligibility, and 7 (4 articles and 3 protocols) met eligibility criteria. Twenty-eight outcomes were identified (19 related to IC and its domains and 9 unrelated). The most reported primary outcome was the change in IC levels postintervention (5 over 7 studies) and the most reported outcomes (either as primary and/or secondary) were the changes in physical performance and in depressive symptoms (6 over 7 studies). Fifty-five tools used to construct the domains' z-scores and/or assess the effect of interventions were identified (47 related to IC and its domains and 8 unrelated). The most reported tool was an IC Z-score, calculated by 4 domains' z-scores: locomotor, vitality, cognitive, and psychological (5 over 7 studies). The tools differed among studies (10 locomotor related, 6 vitality related, 16 cognitive related, 8 psychological related, 6 sensorial related, 8 unrelated tools). The vast heterogeneity (28 outcomes and 55 tools within 7 studies) highlighted the need of a COS. These outcomes and tools will be presented to experts in a future step, to select the ones that should be taken into consideration in IC trials.