Disease-specific Measures Research Articles

The effectiveness of social prescribing in the management of long-term conditions in community-based adults: A systematic review and meta-analysis

Objective The objective of this systematic review and meta-analysis was to evaluate the effectiveness of social prescribing interventions in the management of long-term conditions in adults. Data sources Eleven electronic databases were searched for randomised and quasi-randomised controlled trials. Review Methods Outcomes of interest were quality of life, physical activity, psychological well-being and disease-specific measures. Bias was assessed with the Cochrane Risk of Bias 2 tool. A narrative synthesis and meta-analysis were performed. Results Twelve studies (n = 3566) were included in this review. Social prescribing interventions were heterogeneous and the most common risks of bias were poor blinding and high attrition. Social prescribing interventions designed to target specific long-term conditions i.e., cancer and diabetes demonstrated significant improvements in quality of life (n = 2 studies) and disease-specific psychological outcomes respectively (n = 3 studies). There was some evidence for improvement in physical activity (n = 2 studies) but most changes were within group only (n = 4 studies). Social prescribing interventions did not demonstrate any significant changes in general psychological well-being. Conclusion Social prescribing interventions demonstrated some improvements across a range of outcomes although the quality of evidence remains poor.

Friedreich Ataxia Caregiver-Reported Health Index: Development of a Novel, Disease-Specific Caregiver-Reported Outcome Measure.

The Friedreich ataxia (FRDA) scientific community needs access to patient-centered outcome measures that satisfy regulatory guidelines and are capable of tracking clinically meaningful changes in FRDA disease burden. The objective of this research was to develop a novel, disease-specific caregiver-reported outcome measure for use in FRDA research and clinical care. In prior work, we conducted qualitative interviews and a cross-sectional study of FRDA caregivers and patients to determine the symptoms of greatest importance to individuals with FRDA. We designed the Friedreich Ataxia Caregiver-Reported Health Index (FACR-HI) to serially measure the symptoms of greatest importance to patients and utilized factor analysis, beta testing, reliability testing, and cross-sectional subgroup analysis to further evaluate and optimize this disease-specific outcome measure. The FACR-HI was designed to measure total disease burden and disease burden in 18 symptomatic domains. The FACR-HI total score demonstrated high internal consistency (Cronbach's α = 0.98) and test-retest reliability (intraclass correlation coefficient = 0.96). Beta interview participants found the FACR-HI to be highly relevant, comprehensive, and easy to use. FACR-HI total and subscale scores were associated with functional staging for ataxia scores and speech impairment. Initial evaluation of the FACR-HI supports its content validity, test-retest reliability, and construct validity as a caregiver-reported outcome measure for assessing how pediatric individuals with FRDA feel and function. The FACR-HI provides a potential mechanism to quantify changes in multifactorial FRDA disease burden during future clinical trials.

Health-Related Quality of Life Burden Among Adults with Vitiligo: Relationship to Disease Severity and Disease Location.

Vitiligo was historically regarded as a cosmetic disorder; however, it is an autoimmune disease. As a visible condition, it affects patient well-being. We assessed the impact of disease severity, lesion location, and body surface area (BSA) affected on patient health-related quality of life (HRQoL). Retrospective data were from the Adelphi Real World Vitiligo Disease Specific Programme: a cross-sectional survey of physicians and their patients with vitiligo (10/2021-07/2022). Patient-reported outcomes were assessed by the Vitiligo-Specific Quality of Life Instrument (VitiQoL), Hospital Anxiety and Depression Scale (HADS), and EQ-5D-5L. The Work Productivity and Impairment Questionnaire (WPAI) questionnaire was used to assess disease-related impairment of daily activities. Data were stratified by physician-reported disease severity, presence/absence of vitiligo on the face, and BSA percentage affected. In total, 1388 patients were included. Mean (SD) VitiQoL, HADS depression, and anxiety scores were higher for those with severe disease [40.5(26.1), 5.2(4.4), and 6.8(4.7)] than those with mild [24.8(18.8), 3.6(3.8), 4.2(3.8)] or moderate [27.1(22.6), 3.8(4.5), 4.3(4.4)] disease. Patients with face affected reported higher VitiQoL [30.0(22.3) versus 23.2(19.3)], and HADS scores [depression, 4.3(4.3) versus 3.2(3.9); anxiety, 5.0(4.3) versus 3.8 (3.9)] than those without. Patients with ≥ 5% BSA affected had higher VitiQoL, depression and anxiety scores [27.9(21.8), 4.0(4.4), and 4.5(4.2)] than those with 0-5% [24.6(19.7), 3.4(3.7), and 4.3(4.1)]. Patients with severe vitiligo, facial lesions, or ≥ 5% BSA reported higher activity impairment. Mean EQ-5D-5L-utility score was approximately 0.9 regardless of disease severity or total BSA affected. These data demonstrate the impact disease severity can have on HRQoL and daily activities for patients with vitiligo. Lesions that are more severe, on the face, or covering a greater BSA are more often associated with poorer outcomes and activity impairment. These data also highlight the potential insensitivity of commonly used HRQoL measures and a need for more sensitive disease-specific measures.

P.057 Achievement of minimal symptom expression in acetylcholine receptor antibody-positive participants treated with efgartigimod in ADAPT/ADAPT+

Background: A key efficacy indicator in generalized myasthenia gravis (gMG) treatment is improvement in MG-ADL score. Minimal symptom expression (MSE, MG-ADL total score of 0 or 1) is explored as a novel proposed treatment target in gMG in the phase 3 study of intravenous efgartigimod, ADAPT, and its open-label extension, ADAPT+. Methods: Post hoc analyses of acetylcholine receptor antibody positive participants in ADAPT (n=129) and ADAPT+ (n=111) were performed. Results: In ADAPT, 44.6% receiving efgartigimod achieved MSE vs 10.9% of participants given placebo. Despite less frequent assessment during ADAPT+, 40.5% of participants achieved MSE. Eighty-one percent of participants treated with efgartigimod who achieved MSE in ADAPT also achieved MSE during ADAPT+; 23% who had not achieved MSE in ADAPT did in ADAPT+. Achieving MSE was associated with substantial improvements in QMG, MGC, MG-QoL15r, and EQ-5D-5L mean scores of 11.4, 16.0, 12.4, and 0.3 points, respectively, from baseline to best score (across all visits). These drastic improvements resulted in quality of life (QoL) comparable to that of healthy populations. MSE achievement also resulted in sustained improvements in these disease-specific and QoL measures. Conclusions: Participants who achieved MSE showed substantial and consistent improvements across multiple disease measures and experienced QoL comparable to that of healthy populations.

'Listen to women as if they were your most cherished person': Australian women's perspectives on living with the pain of endometriosis: A mixed-methods study.

This mixed-methods study used an online cross-sectional survey to explore perspectives of 533 adult Australian women living with endometriosis pain, and their relationship with biopsychosocial factors. Four themes were constructed: The primary theme, 'Stigma and change' reflected women's experience of dismissal, and the wish to reverse the narrative of pain as normal. Some women emphasised self-education and self-advocacy to affect change, reflecting the theme 'self-empowerment'. Participants described the 'debilitating impact' of endometriosis and the enduring difficulty of 'inadequate healthcare', reflecting themes three and four. Analysis indicated type of social support may impact perceived outcomes for endometriosis. Hierarchical regression analyses indicated too few significant relationships between biopsychosocial factors and themes to indicate meaningful patterns without risk of common method variance. Future research should explore the influence of social support and interventions which develop participant autonomy and practitioner competence and knowledge, using disease-specific measures over time.

Surgically-relevant quality of life thresholds for the Short Inflammatory Bowel Disease Questionnaire in Crohn’s disease

Background or PurposeDespite interest in patient-reported outcome measures to track progression in Crohn’s disease, frameworks to apply these questionnaires in the preoperative setting are lacking. Using the short inflammatory bowel disease questionnaire (sIBDQ), this study aimed to describe interpretable quality of life thresholds and examine potential associations with future bowel resection in Crohn’s disease. MethodsAdult Crohn’s disease patients completing an sIBDQ at a clinic visit between 2020-2022 were eligible. A stoplight framework was adopted for sIBDQ scores, including a ‘Resection Red zone’ suggesting poor quality of life that may benefit from discussions about surgery as well as a ‘Nonoperative Green’ zone. Thresholds were identified with both anchor- and distribution-based methods using receiver operating characteristic curve analysis and subgroup percentile scores, respectively. To quantify associations between sIBDQ scores and subsequent bowel resection, multivariable logistic regression models were fit with covariates of age, sex, body mass index, medications, disease pattern and location, resection history, and the Harvey Bradshaw Index. Incremental discriminatory value of the sIBDQ beyond clinical factors was assessed through area under the receiver operating characteristics curve (AUC) with an internal validation through bootstrap resampling. ResultsOf 2003 included patients, 102 underwent Crohn’s-related bowel resection. The sIBDQ Nonoperative Green zone threshold ranged from 61 to 64 and the Resection Red zone from 36 to 38. When adjusting for clinical covariates, a worse sIBDQ score was associated with greater odds of subsequent 90-day bowel resection when considered as a one-point (OR [95% CI], 1.05 [1.03-1.07]) or five-point change (OR [95% CI], 1.25 [1.13-1.40]). Inclusion of the sIBDQ modestly improved discriminative performance (AUC [95% CI] 0.85 [0.85 – 0.86]) relative to models that included only demographics (0.57 [0.57 – 0.58]) or demographics with clinical covariates (0.83 [0.83 – 0.84]). Discussion or ConclusionIn the decision-making process for bowel resection, disease-specific patient-reported outcome measures may be useful to identify Crohn’s disease patients with poor quality of life and promote shared understanding of personalized burden.

COMPARISON OF POSTOPERATIVE QUALITY OF LIFE AND UTILITY VALUES FOLLOWING TOTAL HIP ARTHROPLASTY AND BIPOLAR HEMIARTHROPLASTY FOR OSTEONECROSIS OF THE FEMORAL HEAD

Purpose: The appropriate surgical procedure for Stage 3 osteonecrosis of the femoral head (ONFH) remains controversial. This study aimed to evaluate and compare postoperative quality of life (QOL) in patients who underwent total hip arthroplasty (THA) and bipolar hemiarthroplasty (BHA) for ONFH based on comprehensive and disease-specific scales using patient-directed questionnaires. Methods: We included 54 of 66 patients who underwent artificial joint replacement for ONFH more than 1 year ago at our hospital between April 2013 and September 2020. THA was performed for ONFH Stage 4 and BHA for Stage 3 or below. The mean postoperative observation period in the THA and BHA groups was 3.9 and 3.7 years, respectively. The Short-Form 6-Dimension measure was used to calculate utility values. Results: No significant differences in questionnaire results regarding disease-specific or comprehensive measures were observed after arthroplasty for ONFH between the THA and BHA groups. The utility values were 0.60 and 0.58 in the THA and BHA groups, respectively. Conclusion: The postoperative QOL was similar between patients who underwent THA for Stage 4 ONFH and BHA for Stage 3 ONFH. Therefore, THA or BHA can be performed on patients with ONFH after considering age, stage classification, and previous medical conditions.

Surgical Intervention is Associated with Improvement in Health-Related Quality of Life Outcomes in Patients with Symptomatic Sacral Tarlov Cysts: Results from a Prospective Longitudinal Cohort Study

The treatment of symptomatic Tarlov cysts remains a controversial topic within neurosurgery. We describe our experience with patients who underwent surgical intervention for sacral Tarlov cysts at a single institution. General and disease-specific outcome measures were used to assess health-related quality of life. Patients who underwent surgical treatment for one or more sacral Tarlov cysts between 2018 and 2021 were included. The Tarlov Cyst Quality of Life (TCQoL), a validated disease-specific measure, was the primary outcome of the study. Secondary outcomes included general outcome measures: 36-Item Short Form Survey, the Oswestry Disability Index, and Visual Analog Scale. Patients were followed at 3, 6, and 12 months postoperative. Repeated measures analyses were used to assess change from preoperative to 12 months postoperative. Data were obtained from 144 patients who underwent surgery for sacral Tarlov cysts, average age 52.3 ± 11.3 years, 90.3% female. Patients reported significant mean improvement on the TCQoL over time (preoperative 3.2±0.1; 3-months postoperative 2.1±0.1; 6-months 1.9±0.1; 12-months 1.9±0.1; P< 0.001). Patient age and duration of symptoms were not associated with outcome. 82.3% of patients reported improvement on TCQoL. There was not a significant difference in the proportion of patients reporting improvement on TCQoL by cyst size (small 90.9% vs. large 77.9%; P = 0.066). Our longitudinal series demonstrated patient-reported improvement following surgery for symptomatic sacral Tarlov cysts using a validated disease-specific health-related quality of life scale through 12 months following surgery. Patient age and preoperative duration of symptoms were not correlated with outcome.

Achievement of Minimal Symptom Expression and Effect on Disease-specific Measures in Acetylcholine Receptor Antibody-positive Participants with Generalized Myasthenia Gravis Treated with Efgartigimod in ADAPT/ADAPT+ (P10-11.009)

Achievement of Minimal Symptom Expression and Effect on Disease-specific Measures in Acetylcholine Receptor Antibody-positive Participants with Generalized Myasthenia Gravis Treated with Efgartigimod in ADAPT/ADAPT+ (P10-11.009)

Modelling Treatment Sequences in Immunology: Optimizing Patient Outcomes.

For some immune-mediated disorders, despite the range of therapies available there is limited evidence on which treatment sequences are best for patients and healthcare systems. We investigated how their selection can impact outcomes in an Italian setting. A 3-year state-transition treatment-sequencing model calculated potential effectiveness improvements and budget reallocation considerations associated with implementing optimal sequences in ankylosing spondylitis (AS), Crohn's disease (CD), non-radiographic axial spondyloarthritis (NR-AxSpA), plaque psoriasis (PsO), psoriatic arthritis (PsA), rheumatoid arthritis (RA), and ulcerative colitis (UC). Sequences included three biological or disease-modifying treatments, followed by best supportive care. Disease-specific response measures were selected on the basis of clinical relevance, data availability, and data quality. Efficacy was differentiated between biologic-naïve and experienced populations, where possible, using published network meta-analyses and real-world data. All possible treatment sequences, based on reimbursement as of December 2022 in Italy (analyses' base country), were simulated. Sequences with the best outcomes consistently employed the most efficacious therapies earlier in the treatment pathway. Improvements to prescribing practice are possible in all diseases; however, most notable was UC, where the per-patient 3-year average treatment failure was 37.3% higher than optimal. The results focused on the three most crowded and prevalent immunological sub-condition diseases in dermatology, rheumatology, and gastroenterology: PsO, RA, and UC, respectively. By prescribing from within the top 20% of the most efficacious sequences, the model found a 15.1% reduction in treatment failures, with a 1.59% increase in drug costs. Prescribing more efficacious treatments earlier provides a greater opportunity to improve patient outcomes and minimizes treatment failures.

Construct validity of EQ-5D-5L among patients with inflammatory bowel disease—a study based on real-world data from the Swedish Inflammatory Bowel Disease Registry

ObjectivesThe Swedish Inflammatory Bowel Disease Registry (SWIBREG) includes approximately 84% of all patients with inflammatory bowel disease (IBD) treated with immunomodulators, biologics or surgery in Sweden. Data on health-related quality of life (HRQoL) have been collected using EQ-5D-5L in the registry since 2012. Nevertheless, there are few studies assessing the validity of EQ-5D-5L in this patient population. Thus, the aim of this study was to assess the construct validity of EQ-5D-5L amongst patients with IBD (ulcerative colitis and Crohn’s disease).MethodsIndividual-level data on EQ-5D-5L and other disease-specific measures were extracted from SWIBREG. Known-groups validity was assessed by analysing whether the EQ-5D-5L captured expected differences between patient groups with different activity levels of the disease. Convergent validity was assessed by analysing whether the reported problems in the dimensions of EQ-5D-5L, EQ VAS, and the EQ-5D-5L index value correlated, as hypothesized, with the four dimensions in the Short Health Scale, a symptom index question, and the Physician Global Assessment (PGA) score.ResultsIn total, 9769 patients with IBD were included in the study. Patients with active IBD reported more health problems in the EQ-5D-5L descriptive system than patients being in remission. The effect sizes for the differences in reported problems between patients with active and inactive disease were at least small (≥0.1) or medium (≥0.3) in all dimensions except self-care. Differences in the mean EQ-5D-5L index and EQ-VAS score between patients with active and inactive disease were statistically significant (p < 0.001) and larger than pre-defined cut-offs for minimally important differences (>0.08 for the index and >11.0 for EQ-VAS). The analysis of convergent validity showed that EQ-5D-5L results correlated as expected with the disease-specific measures in 16 of the 21 analyses. In total, 22 (79%) of the 28 hypotheses were supported.ConclusionThe findings support the construct validity of EQ-5D-5L amongst patients with IBD and contribute to the scarce literature on the validity of the five-level version of EQ-5D in this patient population. These findings have important implications for the choice of HRQoL measure in routine health care registries like SWIBREG as well as for future clinical or health economic studies considering using EQ-5D-5L as a measure of HRQoL.

Development and validation of the HDSIM™ assessment system: a measure of hemorrhoid disease symptom impact.

Hemorrhoid disease (HD) affects 10 million people in the US at any given time, and 50% of the US population will develop symptomatic hemorrhoids during their lifetime. Approximately 60% of people with hemorrhoids experience symptoms. Despite its prevalence and impact on quality of life, the existing patient-reported outcome measures of HD have not been validated using standard psychometric methods. The present study thus aimed to develop the Hemorrhoid Disease Symptom Impact Measure™ (HDSIM™) assessment system, a patient-reported measure of HD symptoms and impact for use in HD clinical research. On the basis of results from qualitative cognitive interviews, we generated the conceptual model and item pool. A cross-sectional web-based survey (n = 1066) was done, including a randomly selected retest subsample (n = 100) 1-2weeks later. The survey sample was selected to be evenly distributed across mild, moderate, and severe levels of disease and to be nationally representative of the general United States population in terms of race, gender, and region. Existing disease-specific measures of symptoms and generic measures of quality of life and well-being were compared to the new tool for construct validation. The HDSIM system includes 38 items representing six conceptual-model-driven subscales, aligning with the conceptual model: Symptoms at Worst, Symptoms at Best, Bowel Health Impact, Life Impact, Mental Health Impact, and Manageability. Psychometric analyses documented that the subscales had excellent internal consistency reliability, cross-sectional construct validity (i.e., convergent and divergent validity, known groups validity), test-retest stability, and longitudinal construct validity (i.e., responsiveness). The HDSIM system is fit for purpose in hemorrhoid disease clinical trials research. Since measures are validated in an iterative manner over many studies and over time, the present study results should be considered preliminary.

The PlexiQoL, a patient-reported outcome measure on quality of life in neurofibromatosis type 1-associated plexiform neurofibroma: translation, cultural adaptation and validation into the Dutch language for the Netherlands.

Half of the patients with Neurofibromatosis type 1 (NF1) develop one or more tumours called plexiform neurofibromas, which can have a significant impact on Quality of Life (QoL). The PlexiQoL questionnaire is a disease-specific QoL measure for adults with NF1-associated plexiform neurofibromas. The aim of this study was to adapt and validate a Dutch version of the PlexiQoL for the Netherlands. The PlexiQoL was translated using the dual-panel methodology, followed by cognitive debriefing interviews to assess face and content validity. The psychometric properties were evaluated by administering the questionnaire on two separate occasions to a sample of adults with NF1 and plexiform neurofibromas. Feasibility was evaluated by the presence of floor/ceiling effects. Reliability was assessed by evaluating Cronbach's alpha coefficient and test-retest reliability, using Spearman's rank correlation coefficients. Mann-Whitney U tests were used to check for known group validity. The Nottingham Health Profile (NHP) questionnaire was used as comparator questionnaire to evaluate convergent validity. The translation and cognitive debriefing interviews resulted in a Dutch version of the PlexiQoL that reflected the original concept and underlying semantic meanings of the UK English version. Forty participants completed the validation survey. The Dutch PlexiQoL demonstrated excellent internal consistency (Cronbach's α 0.825) and test-retest reliability (Spearman correlation coefficient 0.928). The questionnaire detected differences in PlexiQoL scores between participants based on self-reported general health and disease severity. Convergent validity was confirmed for relevant NHP subsections. The Dutch PlexiQoL demonstrated excellent psychometric properties and can be reliably used to measure plexiform neurofibroma-related QoL in adults with NF1 in the Netherlands.

Emotional atypical arousal ratings for unpleasant stimuli in patients with Parkinson's disease.

Parkinson's disease (PD) is a neurodegenerative disorder characterized by motor and non-motor symptoms, including alteration in emotional processing and recognition of emotions. We explored the effects of PD on the emotional behavioral ratings using a battery of affective visual stimuli selected from the International Affective Picture System (IAPS). Twenty-two patients diagnosed with idiopathic PD and 22 healthy controls (HC), matched by age, gender, and education, were enrolled in the study. Following a clinical assessment, each participant was asked to evaluate the arousal and valence of affective visual stimuli, and response time was recorded. Disease-specific measures including the MDS Unified Parkinson's Disease Rating Scale (MDS UPDRS) and the Non-Motor Symptom Scale (NMSS) were also collected. PD patients exhibited higher arousal responses compared to HC for negative/unpleasant pictures (scoring 7.32 ± 0.88 vs 5.43 ± 2.06, p < 0.001). The arousal response to negative/unpleasant pictures was correlated with measures of non-motor burden in PD (MDS UPDRS I and NMSS, rho = 0.480 and p = 0.023, rho = 0.533 and p = 0.010, respectively). Impaired emotional processing characterizes PD patients with mild disease and is related to the non-motor symptom burden. Given the importance of emotional processing for the development and maintenance of close interpersonal relationship and for coping with specific medical situations, it is crucial to direct PD patients towards therapeutic interventions focused on the recognition and processing of emotions.

Validation of the Comprehensive ICF Core Set for Multiple Sclerosis: A Turkish clinical perspective.

The aim of this study was to evaluate the results of the construct validity of the Comprehensive International Classification of Functioning, Disability and Health (ICF) Core Set for Multiple Sclerosis (MS) and to identify the impact of MS on specific health domains according to the ICF categories in Turkish individuals. Between December 2011 and December 2012, a total of 150 individuals with MS (69 males, 81 females; mean age: 39.8±8.0 years; range, 19 to 52 years) who were diagnosed by a neurologist at least six months previously were included. The Comprehensive ICF Core Set for MS was used to assess functioning, disability, and environmental factors in all participants. The Spearman correlation coefficient was used to determine construct validity of the ICF Core Set between the ICF components and disease-specific and general measurements. The most frequently coded impairments in the body functions component were b280 sensation of pain, b134 sleep functions, b1301 motivations, and b740 muscle endurance functions. The most frequently coded impairments in the body structures component were s110 structure of brain, s120 spinal cord and related structures and s750 structure of lower extremity. A significant problem was documented in 47 of the 53 categories of the activities and participation. Of the 38 categories in the environmental factors, 23 were identified as a facilitator, while 12 categories were identified as a barrier. All body function, structure, activities and participation and environmental factors categories showed a significant correlation with disease-specific and generic measures. Based on these results, the ICF Core Set may help to determine major facilitators or barriers to functioning and disability and a targeted rehabilitation approach in patients with MS.

Advancing Rheumatology Care Through Machine Learning.

Rheumatologic diseases are marked by their complexity, involving immune-, metabolic- and mechanically mediated processes which can affect different organ systems. Despite a growing arsenal of targeted medications, many rheumatology patients fail to achieve full remission. Assessing disease activity remains challenging, as patients prioritize different symptoms and disease phenotypes vary. This is also reflected in clinical trials where the efficacy of drugs is not necessarily measured in an optimal way with the traditional outcome assessment. The recent COVID-19 pandemic has catalyzed a digital transformation in healthcare, embracing telemonitoring and patient-reported data via apps and wearables. As a further driver of digital medicine, electronic medical record (EMR) providers are actively engaged in developing algorithms for clinical decision support, heralding a shift towards patient-centered, decentralized care. Machine learning algorithms have emerged as valuable tools for handling the increasing volume of patient data, promising to enhance treatment quality and patient well-being. Convolutional neural networks (CNN) are particularly promising for radiological image analysis, aiding in the detection of specific lesions such as erosions, sacroiliitis, or osteoarthritis, with several FDA-approved applications. Clinical predictions, including numerical disease activity forecasts and medication choices, offer the potential to optimize treatment strategies. Numeric predictions can be integrated into clinical workflows, allowing for shared decision making with patients. Clustering patients based on disease characteristics provides a personalized care approach. Digital biomarkers, such as patient-reported outcomes and wearables data, offer insights into disease progression and therapy response more flexibly and outside patient consultations. In association with patient-reported outcomes, disease-specific digital biomarkers via image recognition or single-camera motion capture enables more efficient remote patient monitoring.Digital biomarkers may also play a major role in clinical trials in the future as continuous, disease-specific outcome measurement facilitating decentralized studies. Prediction models can help with patient selection in clinical trials, such as by predicting high disease activity.Efforts are underway to integrate these advancements into clinical workflows using digital pathways and remote patient monitoring platforms.In summary, machine learning, digital biomarkers, and advanced imaging technologies hold immense promise for enhancing clinical decision support and clinical trials in rheumatology. Effective integration will require a multidisciplinary approach and continued validation through prospective studies.

Interim 1-Year Radiographic and Clinical Outcomes Following Anterior Cervical Discectomy and Fusion Using Hydroxyapatite-Infused Polyetheretherketone Interbody Cages.

This is a multicenter observational registry analysis of 1-year radiographic and clinical outcomes following anterior cervical discectomy and fusion (ACDF) using hydroxyapatite (HA)-infused polyetheretherketone (PEEK) intervertebral cages. Radiographic and clinical outcome data were collected preoperatively and at 6 weeks, 3 months, 6 months, and 12 months postoperatively. To assess fusion, dynamic flexion-extension radiographs were independently evaluated with a validated method. Clinical outcomes were assessed using the following disease-specific measures: Neck Disability Index (NDI) and visual analog scale (VAS) for neck, left arm, and right arm pain. Patient satisfaction was also evaluated. A total of 789 ACDF patients (men: 51.5%/women: 48.5%; mean body mass index: 29.9 kg/m2) were included at the time of analysis, and 1565 segments have been operated. Successful fusion was confirmed in 91.3% of all operated levels after 6 months and 92.2% after 12 months. Mean NDI scores improved significantly (P < 0.01) preoperatively (46.3, n = 771) to postoperatively (12 months: 25.2, n = 281). Consistently, mean VAS neck (preoperative: 64.2, n = 770; 12 months: 28.6, n = 278), VAS right arm (preoperative: 42.6, n = 766; 12 months: 20.4, n = 277), and VAS left arm (preoperative: 41.1, n = 768; 12 months: 20.8, n = 277) decreased significantly (P < 0.01). Patients reported high satisfaction rates after surgery with no significant changes in postoperative patient satisfaction between 6 weeks and 12 months (95.1%, n = 273). ACDF with HA-infused PEEK cages demonstrates promising radiographic and clinical outcomes, supporting the potential benefits of incorporating HA into PEEK cages to enhance fusion rates and improve patient outcomes. This study demonstrates a >90% fusion rate by level with reliable improvements in patient reported outcomes, along with a high rate of patient satisfaction, in a large patient cohort undergoing ACDF with HA-infused PEEK cages. 2 .

The development of a cryptoglandular anal fistula quality of life scale (AF-QoL).

Quality of life (QoL) is a crucial and core outcome in assessing the effectiveness of treatments for cryptoglandular anal fistula. Despite its extensive impact, there is a lack of patient-centred, disease-specific QoL measurement instruments of adequate quality. The aim of this study is to develop a disease-specific measurement instrument that can accurately measure QoL for patients with cryptoglandular anal fistula. Semi-structured qualitative patient interviews and a systematic review of current instruments were used to generate items for the draft instrument. This underwent successive rounds of cognitive interviews to refine its wording and structure. Individual item and overall scale content validity were determined by asking experts to rate the relevance of each item and those deemed irrelevant were removed. The final instrument then underwent psychometric testing and test-retest analysis to determine its sensitivity and stability. A total of 148 patients were involved in item generation, scale development and psychometric testing. A 22-item measurement instrument has been developed; it is scored on a scale of 0-100, where 0 indicates the worst QoL and 100 demonstrates perfect QoL. The scale demonstrates excellent internal consistency (Cronbach-α = 0.927), strong content and construct validity [correlation with Perianal Disease Activity Index = -0.713, Hospital Anxiety and Depression Anxiety (-0.659) and Depression (-0.673) subscales and Short Form-12 physical (0.609) and mental (0.589) component scales] and strong reliability and responsiveness. We have developed a cryptoglandular Anal Fistula Quality of Life scale (AF-QoL), a comprehensive, disease-specific patient reported outcome measure assessing QoL in patients with cryptoglandular anal fistula.

DOES SURGERY FOR IBD DELIVER ON ITS “PROMIS”ES? IMPACT OF SURGERY ON SHORT-TERM PATIENT REPORTED OUTCOMES FROM THE CROHN’S &amp; COLITIS FOUNDATION’S SURGICAL INNOVATIONS, RESEARCH, &amp; QUALITY COLLABORATIVE

Abstract BACKGROUND As part of the Crohn’s &amp; Colitis Foundation’s IBD Plexus, a novel prospective, longitudinal multicenter surgical inception cohort was implemented. In this first report from the first 100 patients enrolled, we aimed to assess patient reported outcomes before and after IBD surgery. We hypothesized surgery is associated with improved patient reported outcomes. METHODS Beginning in Aug. 2022 patients were prospectively enrolled into a multicenter IRB-approved observational longitudinal cohort at 6 centers with 17 enrolling surgeons. Inclusion criteria: adults undergoing major abdominopelvic surgery for a known diagnosis of IBD. Baseline demographics, intra-operative details, 30-day outcomes, and patient reported outcomes were recorded at baseline and 30-days postoperatively. Overall health status was assess using the PROMIS General Health measures, symptom specific measures: PROMIS Pain Interference, Fatigue, and Sleep scales, and disease-specific measures: the Crohn’s &amp; Ulcerative Colitis Questionnaire items. Figures represent frequency (proportion), median (range). P-values represent Wilcoxon rank-sum test or chi-squared test or Fisher’s exact test. RESULTS A total of 103 patients were enrolled to date; median age 43 (20 – 74), 49% female, diagnoses: Crohns (64%), ulcerative colitis (35%), and IBDu (1%). A total of 69 (67%) patients completed surveys, and 58 (56%) patients completed the post-operative survey. In terms of overall health status within 7 days, improved overall median pain scores (3 vs. 2, p=0.04), overall health (p=0.04), physical health (p=0.03), and less fatigue (p=0.05) were observed after surgery compared to baseline (Table 1), while overall quality of life, mental health, social measures, and emotional problems were similar. Patients reported more difficulties with activities of daily living after surgery (p=0.01). In terms of symptoms within 7 days, less fatigue (p&amp;lt;0.05), but no difference in pain interference or sleep disturbance was observed after surgery (p&amp;gt;0.05). In terms of disease-specific outcomes within 2 weeks, fewer days feeling unwell (5 vs. 3, p=0.02), bloated (5 vs 2, p=0.04), or needing to rush to the toilet (5 vs 1, p=0.007), and no difference in feeling tired, upset, abdominal pain, or nocturnal bowel movements was observed after surgery. CONCLUSIONS In a prospective longitudinal cohort, surgery was associated with improved pain and physical health scores, with fewer days feeling unwell, fatigued, bloating, and fecal urgency. On the other hand, mental, social, and emotional health did not change 30-days after surgery. Our data suggest patients are still recovering 30-days after surgery, and longer follow-up may reveal further improvements. Targeted psychosocial interventions and support at home early in the acute post-operative period may improve the quality of recovery after surgery for patients with IBD. Table 1 Table 2

Health-related quality of life with standard and curative therapies in thalassemia: A narrative literature review.

Health-related quality of life (HRQOL) is a patient-reported outcome that assesses the impact of a disease or illness on different domains of a patient's life. Different general and disease-specific measures can be used to evaluate HRQOL. This article aimed to summarize the evidence for HRQOL among patients with transfusion-dependent (TDT) and non-transfusion-dependent thalassemia (NTDT). We included HRQOL data related to standard therapy with blood transfusions, iron chelation, and/or luspatercept in TDT and NTDT, as well as curative therapies for TDT, including hematopoietic stem cell transplant (HSCT) and gene therapy. Patients with thalassemia had worse HRQOL scores compared to the general population, and chronic pain was seen to increase in frequency and severity over time with age. NTDT patients reported worse physical health and functioning, mental health, general health, and vitality than TDT patients. However, TDT patients reported worse pain, change in health, and social support than NTDT. Most therapies improved overall HRQOL among thalassemia patients. Deferasirox, an oral iron chelator, was associated with more HRQOL benefits compared to deferoxamine, an intravenous iron chelator. Luspatercept showed clinically meaningful improvement in physical functioning among TDT and NTDT. Furthermore, HSCT and gene therapy were associated with better physical, emotional, and mental domains scores.