Longer Disease Duration Research Articles

Health state utility values in patients with Ankylosing Spondylitis: a systematic review and meta-analysis.

Ankylosing spondylitis (AS) is a chronic condition that requires lifelong treatment and results in a serious disease burden. Health state utility values (HSUVs) are a valuable tool for quantifying this burden and conducting cost-utility analysis. We conducted a systematic review and meta-analysis to obtain estimates of HSUVs in patients with AS, explored potential sources of heterogeneity, and compared pooled patient HSUVs with population norms. We searched PubMed, Embase, Web of science, Cochrane database and Scopus until July, 2023 to obtain eligible studies. The methodological quality of the included studies was assessed using the ROBINS-I checklist. Forty-two publications involving 11,354 participants were included in this systematic review. The most commonly used instrument is the EQ-5D (38 studies). The estimated HSUVs for patients with AS from all available studies was pooled as 0.62 (95% CI 0.59 to 0.65). The pooled mean utility estimates from the random effects meta-analysis for SF-6D, EQ-5D-3L, EQ-5D-5L, and HUI3 were 0.65 (95% CI 0.62,0.68), 0.63 (95% CI 0.59,0.66), 0.60 (95% CI 0.42,0.79), and 0.48 (95% CI 0.43,0.53), respectively. For the EQ-5D-3L we conducted stratified meta-analyses and meta-regression based on key subgroups. The pooled estimates of EQ-5D-3L were lower for patients published before 2010, with high disease activity, long duration of disease, and in developed countries. Pooled estimates of HSUVs for people with AS were substantially lower than population norms. These estimates provide robust evidence that can inform the economic evaluation of new therapies for individuals with AS.

Volumetric changes of the enteric nervous system under physiological and pathological conditions measured using x-ray phase-contrast tomography.

Full-thickness biopsies of the intestinal wall may be used to study and assess damage to the neurons of the enteric nervous system (ENS), that is, enteric neuropathy. The ENS is difficult to examine due to its localization deep in the intestinal wall and its organization with several connections in diverging directions. Histological sections used in clinical practice only visualize the sample in a two-dimensional way. X-ray phase-contrast micro-computed tomography (PC-μCT) has shown potential to assess the cross-sectional thickness and volume of the ENS in three dimensions (3D). The aim of this study was to explore the potential of PC-μCT to evaluate its use to determine the size of the ENS. Full-thickness biopsies of ileum obtained during surgery from five controls and six patients clinically diagnosed with enteric neuropathy and dysmotility were included. Punch biopsies of 1 mm in diameter and 1 cm in length, from an area containing myenteric plexus, were extracted from paraffin blocks, and scanned with synchrotron-based PC-μCT without any staining. The microscopic volumetric structure of the neural tissue (consisting of both ganglia and fascicles) could be determined in all samples. The ratio of neural tissue volume/total tissue volume was higher in controls than in patients with enteric neuropathy (P = 0.013). The patient with the longest disease duration had the lowest ratio. The assessment of neural tissue can be performed in an objective, standardized way, to ensure reproducibility and comparison under physiological and pathological conditions. Further evaluation is needed to examine the role of this method in the diagnosis of enteric neuropathy.

Sleep Quality is Poor in Rheumatoid Arthritis Patients and Correlates with Anxiety, Depression, and Poor Quality of Life.

Sleep quality is poor in most rheumatoid arthritis (RA) patients. We planned this study to see the association of sleep quality with anxiety, depression, and quality of life (QOL) in Indian RA patients. One hundred twelve RA patients and 93 controls were included in this cross-sectional study. Sleep quality was assessed by Pittsburgh Sleep Quality Index (PSQI). Quality of life was assessed using the World Health Organisation Quality of Life (WHOQOL-BREF). Anxiety and depression were assessed using the hospital anxiety and depression scale (HADS). The disease activity was measured by Disease Activity Score for 28 joints with 3 variables. Functional disability was measured by using the Indian version of the Health Assessment Questionnaire (HAQ). Poor sleep quality was seen in 103(92%) of the patients and 26(28%) controls (p<0.0001). RA patients had significantly higher PSQI scores as compared to the control group (10.8±3.7 vs.3.9±1.2, P<0.0001). Poor sleepers were more likely to be females (90% vs.0%, p = 0.02), and had longer disease duration (5.2±4.8 years vs. 2.8±2 years, p=0.01). Poor sleepers had more pain (VAS 42.2±23.6 mm vs.16.2±11.6 mm, p<0.001) and poor functional status (HAQ 1.2±0.5 vs. 0.6±0.4, p<0.01). There was a significant effect of the physical domain of WHOQOL-BREF, anxiety, and age on poor sleep quality. Poor sleep quality is seen in a majority of RA patients and correlates with anxiety, depression, and poor QOL. RA patients need to be routinely assessed for sleep quality and factors affecting poor sleep quality need to be addressed.

Exploring the experience of multiple sclerosis patients in Turkey: Insights from a national survey

Objective: To evaluate sociodemographic profile, clinical characteristics, disability and treatment status of multiple sclerosis (MS) patients in Turkey with respect to patient perspectives and expectations. Methods: A total of 2,176 MS patients participated in this cross-sectional questionnaire survey including items on sociodemographic, disease and treatment characteristics, daily life and perspectives and expectations. Results: Mean (SD) patient age was 36.4(9.4) years and 76.3% of patients were females. The numbness/weakness in the extremities (57.3%) was the most common presenting symptom. Overall, 56.8% reported treatment switch (due to attacks in 47.3%), while 22.2% reported physical disability and 39.7% reported work-related problems. Males had higher rate of MS-related physical disability (33.0% vs. 19.0%, p&lt;0.001) than females. Use of an assistive device was a more common in patients with longer disease duration (≥15 years; 39.0%) and in those under IV treatment (64.0%). Nearly half of patients reported significant concerns related to uncertainty of the future and impaired quality of life as well as lack of hope for future improvement. The majority of patients reported that they would prefer less frequent SC injection dosing and 43.3% reported preference for monthly high- efficacy SC injection. Conclusion: This nationwide questionnaire-based study in Turkish MS patients revealed the altered disability status with respect to sociodemographic profile, and altered treatment expectations specific to the route of administration, in addition to significant concerns regarding the uncertainty of the future, impaired quality of life and lack of hope for future improvement in nearly half of patients.

Psoriatic nail involvement in Malaysia: A 14-year registry review (2007-2020)

Nail psoriasis affects 20% to 30% of psoriasis patients and is an early predictor of psoriatic arthritis (PsA). We evaluated the prevalence, clinical characteristics, and impact on quality of life of patients with nail psoriasis. We conducted a multicenter retrospective cohort study of patients registered with The Malaysian Psoriasis Registry from January 1, 2007 to December 31, 2020. Of the 24,147 patients, 13,081 (54.2%) had nail psoriasis. Patients with nail psoriasis had later onset of psoriasis (34.0 ± 16.6 years vs 32.9 ± 17.6 years, P < .001) and longer disease duration (11.4 ± 10.5 years vs 8.5 ± 9.4 years, P < .01), with a man-to-woman ratio of 1.2:1. They were more likely to have a family history of psoriasis, cardiometabolic diseases, smoking history, higher body mass index, severe disease, PsA, face and scalp involvement, and higher mean Dermatology Life Quality Index scores (9.36 ± 6.84 vs 8.87 ± 6.60). Systemic treatment and biologics were more commonly prescribed in this cohort (25.0% vs 13.2%, P < .001). Overall, 54.2% of the Malaysian Psoriasis Registry patients had nail involvement. Nail psoriasis was associated with longer duration of psoriasis, older age of onset, male sex, and a family history of psoriasis. It proved to be an important predictor for PsA, severe psoriasis, face and scalp involvement, increased cardiometabolic risk, and a greater impairment of quality of life.

Telaah Kualitas Hidup Pasien Penyakit Kronik di Kalimantan Barat

Background: The long duration of chronic disease affecting the patient’s quality of life. Every disease including chronic disease has a different duration and prognosis. The instrument that being used in measuring the quality of life can be very important factor. Quality of life assessment has a benefit by evaluate the treatment of patients with chronic disease, moreover the good quality of life will also increase the patient's life expectancy. The purpose of this article are to describe and identify instruments that being used to measure quality of life patients with chronic disease in West Kalimantan.Methods: This study conduct to review the original research articles with the keywords "quality of life", "chronic disease", and "West Kalimantan". Twenty three research articles were obtained in Google Scholar and met the inclusion criteria. The generic instruments such as WHOQOL-BREF and EQ5D are the most widely used to measure the patient’s quality of life.Result: The distribution of chronic diseases that have been assessed were hypertension, chronic renal failure, diabetes mellitus, COPD, pulmonary tuberculosis, asthma, myocardial infarction, stroke, CHD, osteoarthritis, HIV / AIDS, and psoriasis. The disease with the most impact on quality of life is hypertension (6 of 23 articles). There were 53.07% patients has a good quality of life; 15.97% patients has a moderate quality of life; and 30.96% patients has a bad quality of life.Conclusion : Most of the hypertensive patients has a good quality of life compared to other disease groups. Health interventions were needed to improve the quality of life for patients with chronic diseases.

Cognitive function in Parkinson's disease: associations with perivascular space in basal ganglia.

Cognitive impairment is one of the most common symptoms of Parkinson's disease (PD), and may be detectable through changes in neural features visualized by magnetic resonance imaging (MRI). Mild cognitive impairment is a transitional state between normal aging and dementia, and early recognition of Parkinson's disease with mild cognitive impairment (PD-MCI) can help improve the quality of life and treatment for patients. This study investigated the association of enlarged perivascular space (EPVS) and white matter hyperintensity (WMH) with PD-MCI. This study aimed to evaluate whether EPVS and WMH can be used as potential MRI markers for PD-MCI. This retrospective study involved 200 patients with PD who underwent cranial MRI in our hospital from April 2021 to April 2022. Patients were divided into those with no cognitive impairment (PD-NCI) or mild cognitive impairment. Uni- and multivariate logistic regression analyzed associations of EPVS, WMH, and clinicodemographic characteristics with cognitive decline. Univariate regression identified severe EPVS in basal ganglia, severe WMH, older age, late-onset, male sex, low educational level, longer duration of disease, low triglycerides, low uric acid, and low scores on the Mini-mental State Exam as risk factors for PD-MCI. After adjusting for clinicodemographic risk factors in multivariate regression, low education level and EPVS in basal ganglia remained risk factors for cognitive impairment. Severe EPVS in basal ganglia and poor education, but not WMH, are independent risk factors of PD-MCI. Our findings suggest that non-invasive detection of EPVS in basal ganglia by MRI may be a valuable early indicator of cognitive decline in PD patients.

Effects of disease-modifying therapies on remyelination in multiple sclerosis; evaluation via visual evoked potential test

BackgroundAssessment of the visual pathway, which is frequently affected by MS, provides the opportunity to measure the remyelination of acute and chronic MS lesions in vivo and non-invasively. VEP can be used in this context. Amplitude is a parameter of axonal loss, whereas latency is an in vivo biomarker of myelin repair. This study aimed to evaluate DMT's neuroprotective and pro-remyelinating potential by evaluating VEP latency and amplitude in MS patients. Materials and methodsA total of 74 patients with relapsing MS who had no evidence of optic neuritis were included in the study. Patient data were retrospectively analyzed and recorded. In the VEP test, latency above 118 ms and amplitude below 5.0 μV were considered abnormal. Classified according to DMTs (injectables, teriflunomide, dimethyl fumarate, fingolimod, cladribine, and alemtuzumab). Visual evoked potential tests, clinical features, and cerebrospinal fluid examinations were evaluated by three independent neurologists (one of whom was also a clinical neurophysiologist). ResultsThe mean age at diagnosis was 29.2 ± 9.01, and the mean age at first VEP was 34.97 ± 10.64. In women, latency was lower, and amplitude was higher. The mean differences in latency and amplitude were, respectively, latency prolonged by 0.7 ms on the right and 0.5 ms on the left, and amplitude increased by 0.6 μV on the right and 0.37 μV on the left. However, these changes were not statistically significant. Latency worsening was more prominent in those with longer disease duration (p = 0.011). Those with amplitude or latency worsening had higher EDSS (p = 0.016 and 0.013, respectively). DMTs did not affect these changes. ConclusionProlonged latency is associated with a long disease duration. Deterioration in both amplitude and latency is evident in high EDSS. These results may be an indirect consequence of axonal degeneration dominating remyelination. DMTs do not ameliorate impaired remyelination and neurodegeneration but seem to be sufficient for short-term maintenance of the current state.

Generalizability and treatment with sodium-glucose co-trasporter-2 inhibitors (SGLT2i) among patients with type 2 diabetes: an assessment using an Italian primary care database.

This study aimed to assess the proportions of type 2 diabetes (T2D) subjects meeting cardiovascular outcome trials (CVOTs) criteria for sodium-glucose cotransporter-2 inhibitors (SGLT-2i) and estimate SGLT2i utilization, along with associated demographic and clinical characteristics, in a primary care setting. T2D patients in Italy were selected between January 1, 2021, and December 31, 2022, from The Health Improvement Network (THIN®) database. Representativeness was determined by dividing patients meeting key inclusion criteria for four CVOTs (CANVAS, DECLARE-TIMI 58, EMPA-REG OUTCOME, VERTIS-CV) to the total T2D population. Demographic and clinical characteristics of eligible T2D subjects and SGLT2i users were compared, and logistic regression models assessed the likelihood of receiving SGLT2i. Out of 17,102 T2D patients, 8,828 met eligibility criteria for at least one CVOT. DECLARE-TIMI 58 exhibited the highest representativeness (51.1%), compared to CANVAS (21.1%), EMPA-REG OUTCOME (5.5%), and VERTIS-CV (4.9%) trials. Eligible CVOTs patients were older (74.6 vs. 68.3 years), with a longer disease duration (10.2 vs. 9.7 years), and higher established cardiovascular disease (CVD) prevalence (36.0 vs. 27.3%) compared to SGLT2i users. Less than 10% of eligible T2D patients received SGLT2i. Males (OR: 1.43; 95%CI: 1.24-1.66) were more likely to be prescribed SGLT2i than other antidiabetic drugs, while the elderly (80 + vs. 40-64 years, OR: 0.17; 95% CI: 0.14-0.22) were less likely. Eligible T2D patients with CVD reported an increased likelihood of receiving SGLT2is compared to other antidiabetics. This study highlights significant variability in the proportion of T2D subjects meeting SGLT2i CVOT inclusion criteria, with DECLARE-TIMI-58 being the most represented. Low SGLT2i prescription rates in the Italian primary care setting, along with substantial demographic and clinical differences between SGLT-2i users and T2D eligible patients, emphasize the need for targeted interventions to optimize the use of these medications in primary care settings.

Electrolytes as predictors of fibro fatigue scores in Long-COVID patients.

The complex effects of Long-COVID, a syndrome marked by enduring symptoms after COVID-19 infection, with an emphasis on patients' differing degrees of fibro fatigue (FF). Electrolyte disturbances may affect the severity of FF and may be used as a predictive tool for severe FF in Long-COVID patients. The aim is to use the electrolyte levels for prediction of the Long-COVID patients with high FF levels. The electrolyte levels, calcium, and magnesium, as well as albumin and C-reactive protein levels were measured in 120 Long-COVID patients and 60 controls. FF scale was used for scoring the fatigue severity in all subjects. Patients were divided into high-FF (FF score>25) and moderate-FF group (FF score<25). FF is the major effector on the serum biomarker levels. High-FF group had older people, longer disease durations, lower SpO2, higher CRP, and higher peak temperatures than the control group. High-FF group has a significant decrease in serum total and ionized calcium compared with the controls and low-FF group. After controlling the cofounders, the major factor controlling the levels of the measured biomarkers is the FF value (Partial η2 = 0.468). The ROC-AUC analysis showed that the peak body temperature, Low-SpO2, high-CRP, and low electrolytes can predict the high-FF in a patient with Long-COVID with a moderate sensitivity and specificities (61.6-70%). Long-COVID patients have an elevation in FF score. The decline in electrolytes can predict the severity of FF with moderate sensitivities and specificities.

A severe asthma phenotype of excessive airway Haemophilus influenzae relative abundance associated with sputum neutrophilia.

Severe asthma (SA) encompasses several clinical phenotypes with a heterogeneous airway microbiome. We determined the phenotypes associated with a low α-diversity microbiome. Metagenomic sequencing was performed on sputum samples from SA participants. A threshold of 2 standard deviations below the mean of α-diversity of mild-moderate asthma and healthy control subjects was used to define those with an abnormal abundance threshold as relative dominant species (RDS). Fifty-one out of 97 SA samples were classified as RDSs with Haemophilus influenzae RDS being most common (n=16), followed by Actinobacillus unclassified (n=10), Veillonella unclassified (n=9), Haemophilus aegyptius (n=9), Streptococcus pseudopneumoniae (n=7), Propionibacterium acnes (n=5), Moraxella catarrhalis (n=5) and Tropheryma whipplei (n=5). Haemophilus influenzae RDS had the highest duration of disease, more exacerbations in previous year and greatest number on daily oral corticosteroids. Hierarchical clustering of RDSs revealed a C2 cluster (n=9) of highest relative abundance of exclusively Haemophilus influenzae RDSs with longer duration of disease and higher sputum neutrophil counts associated with enrichment pathways of MAPK, NF-κB, TNF, mTOR and necroptosis, compared to the only other cluster, C1, which consisted of 7 Haemophilus influenzae RDSs out of 42. Sputum transcriptomics of C2 cluster compared to C1 RDSs revealed higher expression of neutrophil extracellular trap pathway (NETosis), IL6-transignalling signature and neutrophil activation. We describe a Haemophilus influenzae cluster of the highest relative abundance associated with neutrophilic inflammation and NETosis indicating a host response to the bacteria. This phenotype of severe asthma may respond to specific antibiotics.

Increased periventricular thalamic damage gradient in multiple sclerosis detected by quantitative gradient echo MRI

ObjectiveThalamic tissue damage in multiple sclerosis (MS) follows a ‘surface-in’ gradient from the ventricular surface. The clinical consequences of this gradient are not completely understood. Using quantitative gradient-recalled echo (qGRE) MRI, we evaluated a periventricular thalamic gradient of tissue integrity in MS and its relationship with clinical variables. MethodsStructural and qGRE MRI scans were acquired for a cohort of MS patients and healthy controls (HC). qGRE-derived R2t* values were used as a measure of tissue integrity. Thalamic segmentations were divided into 1-mm concentric bands radiating from the ventricular surface, excluding the CSF-adjacent band. Median R2t* values within these bands were used to calculate the periventricular thalamic gradient. ResultsWe included 44 MS patients and 17 HC. R2t* increased slightly with distance from the ventricular surface in HC. MS patients had a steeper periventricular thalamic gradient compared to HC (mean slope 0.55 vs. 0.36; p < 0.001), which correlated with longer disease duration (β = 0.001 /year; p = 0.027) and higher Expanded Disability Status Scale (EDSS) score (β = 0.07 /EDSS point; p = 0.019). Left and right thalamus were symmetrically affected. ConclusionsWe detected an increased thalamic gradient in MS in vivo using qGRE MRI, which correlated with disease duration and greater clinical disability. These findings further support the ‘surface-in’ pathology hypothesis in MS and suggest a CSF-mediated process given symmetric bi-thalamic involvement.

Exploring Analysis Approaches for Using the Dopamine Transporter Striatal Binding Ratio in Early- to Mid-Stage Parkinson's Disease Modification Trials.

The dopamine transporter striatal binding ratio (DAT SBR) has been used as an outcome measure in Parkinson's disease (PD) trials of potential disease-modifying therapies; however, both patient characteristics and analysis approach potentially complicate its interpretation. The aim was to explore how well DAT SBR reflects PD motor severity across different striatal subregions and the relationship to disease duration, and side of onset. DAT SBR for the anterior and posterior putamen and caudate in both hemispheres was obtained using validated automated quantitative software on baseline scans of 132 patients recruited for the Exenatide PD2 and PD3 trials. Associations between mean and lateralized SBR subregions (posterior and anterior putamen and caudate) and summed and lateralized motor characteristics were explored using regression analysis. Analyses were repeated considering disease duration and limiting analysis to the less-affected hemisphere. Lateralized bradykinesia was most consistently associated with the loss of DAT uptake in the contralateral anterior putamen. There was much higher variance in the posterior putamen, and in all regions in those with longer duration disease, although bradykinesia remained robustly associated with anterior putaminal DAT uptake even in longer-duration patients. Restricting analyses to the less-affected side did not usefully reduce the variance compared to the overall cohort. These data suggest that DAT SBR could be a useful biomarker in disease-modifying trials, but a focus on anterior striatal subregions and incorporating disease duration into analyses may improve its utility.

Predictors of Diabetic Retinopathy in Type 2 Diabetes: A Cross-Sectional Study.

Type 2 diabetes mellitus (T2DM) represents one of the most impacting health issues of the modern era, as it is associated with an extensive range of comorbidities. Diabetic retinopathy (DR) is one the utmost severe diabetes complications as it is one of the major causes of vision loss among these patients. Our present research aims to evaluate the most frequent risk factors related to the occurrence of DR in T2DM patients. This study consisted of a research group of 302 participants, priorly diagnosed with T2DM, that were evaluated for the most important risk factors related to the occurrence of DR. Patients had a median age of 64 years, 48% of them being women, with a 12-year median duration of DM and presenting a deficient glycaemic control echoed by a median HbA1C value of 7.5%. From the total number of participants, the total prevalence of DR in different stages of severity was 34.8% with a 95% CI. Statistically significant values were found regarding DM duration (p = 0.007), HbA1c > 7.2% (p = 0.001) and patients aged over 67 years (p = 0.0035), all these parameters being directly linked to DR. Older patients with T2DM that have a longer disease duration and simultaneous comorbidities present a higher risk of DR development, consequently a stringent management of these pathologies is needed.

Differential Analysis of Venous Sinus Diameters: Unveiling Vascular Alterations in Patients with Multiple Sclerosis.

Alterations in the cerebral venous system have been increasingly recognized as a significant component of the pathophysiology of multiple sclerosis (MS). This study aimed to explore the relationship between venous sinus diameter and MS to understand potential vascular alterations in MS patients compared with controls. We sought to determine whether these alterations were correlated with disease characteristics such as duration, lesion type, and disability score. This study included 79 MS patients diagnosed according to the 2017 McDonald criteria and 67 healthy individuals. Magnetic resonance imaging (MRI) scans via a 1.5 Tesla system provided measurements of the superior sagittal sinus, right and left transverse sinus, sinus rectus, and venous structures. Statistical analysis was conducted via SPSS, employing independent sample t tests, ANOVA, chi-square tests, and Pearson correlation analysis, with the significance level set at p < 0.05. This study revealed significant differences in venous sinus diameter between MS patients and controls, with MS patients exhibiting larger diameters. Specifically, patients with brainstem and spinal lesions had larger diameters in certain sinus regions. No significant correlations were found between venous sinus diameter and demographic factors, expanded disability status scale scores, or lesion counts. However, a significant increase in perivenular lesions was noted in patients with longer disease durations. The findings indicate notable vascular alterations in MS patients, particularly in venous sinus diameters, suggesting a potential vascular component in MS pathology. The lack of correlation with conventional clinical and MRI metrics highlights the complexity of MS pathology. These insights underscore the need for further research, particularly longitudinal studies, to elucidate the role of venous changes in MS progression and their potential as therapeutic targets.

Real-world analysis of brain atrophy in multiple sclerosis patients with an artificial intelligence based software tool

BackgroundAtrophy of white and grey matter volumes occurs early in the brains of people with multiple sclerosis (pwMS) and has great clinical relevance. In clinical trials, brain atrophy can be quantified by magnetic resonance imaging (MRI) with automated software tools.MethodsIn this study, we analyze volumes of various brain regions with the software “md brain” based on routine MRI scans of 53 pwMS in a real-world setting. We compare brain volumes of pwMS with an EDSS ≥ 3.5 and a disease duration ≥ 10 years to the brain volumes of pwMS with an EDSS < 3.5 and a disease duration < 10 years as well as with or without immunotherapy.ResultspwMS with an EDSS ≥ 3.5 and a disease duration ≥ 10 years had significantly lower volumes of the total brain, the grey matter and of the frontal, temporal, parietal and occipital lobe regions as compared to pwMS with an EDSS < 3.5 and a disease duration < 10 years. Regional brain volumes were significantly lower in pwMS without immunotherapy.ConclusionsThe study showed that higher EDSS, longer disease duration and absence of immunotherapy was associated with lower volumes in a number of brain regions. Further real-world studies may include larger patient cohorts in longitudinal analyses.

Distribution of Serum Testosterone Concentrations in IBD Males and Associations With Inflammatory Bowel Disease Activity.

An inverse relationship exists between inflammation and testosterone concentrations in non-inflammatory bowel disease (IBD) immune conditions but has not been objectively explored in the IBD male population. We aimed to characterize the distribution of testosterone concentrations in a cohort of males with IBD and identify any relationship between testosterone levels and disease activity. We conducted a prospective cross-sectional study of male IBD patients. Demographics, disease characteristics, sex-hormone concentration, gonadotropins, C-reactive protein, fecal calprotectin, and patient-reported outcomes on quality of life and erectile function were collected. Relationships between disease activity, biomarkers, patient-reported outcome scores, and testosterone levels were analyzed using univariate and multivariate linear regression analyses. A total of 85 male IBD patients were included with a mean age 44±14.1 years, of which 49.4% had Crohn's disease. Mean testosterone concentration was 15.4±5.2 nmol/L and 17.6% had a serum testosterone <10.4 nmol/L. Active disease was associated with lower testosterone concentrations in univariate analysis (β ± SE = -0.25 ± -1.99, P = .02) but not in multivariate analysis (β -0.18±1.75, P = .06). Testosterone concentrations were independently associated with sex hormone-binding globulin levels (β ± SE = 0.45±0.04, P < .0001) and a younger age (β ± SE = -0.32±0.04, P <.0001). Erectile function scores (5-item International Index of Erectile Function) were lower in IBD patients with a longer duration of disease (β ± SE = -0.24±0.006, P = .04). Lower testosterone concentrations in men with IBD may reflect confounding from other factors and are not independently associated with disease activity. Greater awareness and screening for sexual dysfunction should occur in males with IBD, particularly in those with a longer disease duration.

Exploring the educational needs of patients with cutaneous lymphoma using an educational needs assessment tool.

Cutaneous T-cell lymphomas (CTCL) are a group of rare non-Hodgkin lymphomas characterized by initial localization of malignant T-lymphocytes in the skin. Support and information from nurses and patient support groups have proven useful for patients with CTCL, but little is known about the educational needs of these patients. To investigate the self-reported educational needs among CTCL patients using an educational needs assessment tool and to explore differences related to sex, age, disease duration, clinical stage, and education. This observational single center study analyzed 70 patients with CTCL in routine dermatological outpatient care. The patients were asked to complete a questionnaire to capture their educational needs in regard to CTCL. The questionnaire was inspired by the educational needs assessment tool, designed and validated for patients with rheumatoid disease. The questionnaire included a general question, "In general, how much information do you want to receive about your lymphoma disease?", and five domains covering information relating to disease process (6 items), treatment (4 items), feelings (2 items), self-management of itch, sleep, and rest (2 items), and support systems (3 items). The domain scores ranged from 0 to 18 and the total score from 0 to 51, with a higher score indicating a greater need for education. When asked "In general, how much information do you need?", females wanted to know more compared with males (2.6 vs. 2.1, p=0.006), and patients with higher education wanted to know more than patients with lower education (2.5 vs. 2.0, p=0.025). The domains concerning treatment (80%) and disease process (75%) revealed the greatest needs for education. Patients with a disease duration <2 years reported a greater educational need for the domain support system, compared with patients with longer disease duration. Patients with lower education reported a greater educational need about feelings compared with patients with higher education. We found that 65% of the CTCL patients in the cohort, particularly females, expressed a need for education, especially regarding disease process and treatment. A deeper understanding of the educational needs would enable healthcare providers to give personalized information.

Platelet response to romiplostim amongst patients with newly diagnosed, persistent, and chronic immune thrombocytopenia in routine clinical practice in Denmark, Sweden, and Norway.

Patient characteristics and platelet responses at romiplostim initiation according to the duration of immune thrombocytopenia (ITP) are poorly understood. Amongst romiplostim-exposed adults with ITP lasting ≥6 months during 2009-2018 in Denmark, Sweden, and Norway, we examined characteristics at romiplostim initiation, romiplostim dosage, and durable platelet response (≥75% of measurements ≥50 × 109/L at 14-24 weeks) for subcohorts with newly diagnosed (duration <3 months), persistent (3-12 months), or chronic (>12 months) ITP initiating romiplostim. The 285 romiplostim initiators comprised 81 (28%) with newly diagnosed, 47 (16%) with persistent, and 157 (55%) with chronic ITP. More patients with newly diagnosed ITP than longer ITP duration, had low comorbidity levels, two or more prior ITP therapies, and previous bleeding requiring hospitalisation. The median romiplostim doses were similar across subcohorts. During treatment, median platelet counts were similar across subcohorts (75-76 × 109/L), and the durable platelet response was 64.6%, 52.9%, and 52.7% for newly diagnosed, persistent, and chronic ITP, respectively. After treatment cessation, the median platelet count was 138 × 109/L, 68 × 109/L, and 71 × 109/L, respectively. In conclusion, newly diagnosed patients, compared with romiplostim initiators with longer disease duration, had more severe ITP, higher frequency of durable platelet response, and higher median platelet count after cessation.

Multimodal, Longitudinal Profiling of SCA1 Identifies Predictors of Disease Severity and Progression.

Spinocerebellar ataxia type 1 (SCA1) is a rare autosomal dominant neurodegenerative disease. Objective surrogate markers sensitive to detect changes in disease severity are needed to reduce sample sizes in interventional trials and identification of predictors of faster disease progression would facilitate patient selection, enrichment, or stratification in such trials. We performed a prospective 1-year longitudinal, multimodal study in 34 ataxic SCA1 individuals and 21 healthy controls. We collected clinical, patient-reported outcomes, biochemical and magnetic resonance (MR) biomarkers at baseline and after 1 year. We determined 1-year progression and evaluated the potential predictive value of several baseline markers on 1-year disease progression. At baseline, multiple structural and spectroscopic MR markers in pons and cerebellum differentiated SCA1 from healthy controls and correlated with disease severity. Plasma and cerebrospinal fluid (CSF) neurofilament light (NfL) chain and CSF glial fibrillary acidic protein (GFAP) were elevated in SCA1. In longitudinal analysis, total brainstem and pontine volume change, inventory of non-ataxia signs (INAS) count, and SCA functional index (SCAFI) showed larger responsiveness compared to the Scale for Assessment and Rating of Ataxia (SARA). Longer disease duration, longer non-expanded CAG repeat length, and higher disease burden were associated with faster SARA increase after 1-year in the SCA1 group. Similarly, lower baseline brainstem, pontine, and cerebellar volumes, as well as lower levels of N-acetylaspartate and glutamate in the cerebellar white matter, were also associated with faster SARA increase. Our results guide the selection of the most sensitive measures of disease progression in SCA1 and have identified features associated with accelerated progression that could inform the design of clinical trials. ANN NEUROL 2024;96:774-787.