Category: Statistics

Clin Exp Rheumatol. 2026 Jan 29. doi: 10.55563/clinexprheumatol/0yu9gm. Online ahead of print.

ABSTRACT

OBJECTIVES: To evaluate the clinical response of Janus kinase inhibitors (JAKi) across subtypes of idiopathic inflammatory myopathies (IIM) in a cohort of patients with refractory disease.

METHODS: We conducted a retrospective analysis of all adult IIM patients treated with JAKi at our centre. Treatment response was assessed based on changes in muscle strength, serum biomarkers of muscle damage and inflammation, pulmonary function, radiological evolution of interstitial lung disease (ILD) and corticosteroid dosage.

RESULTS: Ten IIM patients who were previously or currently receiving JAKi therapy were identified. Six patients were female and the mean age was 52.7 years (standard deviation [SD] 13.91). Anti-synthetase syndrome was the most common subtype (n=5, 50%). At baseline, median manual muscle test 8 score was 136 (interquartile range [IQR] 25.5) and, after therapy, was 147 (IQR 8), representing a statistically significant increase (p<0.05). Corticosteroid dose reduction was also statistically significant (p<0.05), with the median daily dose decreasing from 10mg to 2.5mg. Additionally, four patients were able to discontinue corticosteroid therapy. In six patients with ILD, diffusion capacity for carbon monoxide improved significantly (p<0.01), from 68.33% (IQR 19.31) to 93% (IQR 7.27). No significant changes were observed in serum inflammatory markers, creatine kinase, forced expiratory volume first second or forced vital capacity.

CONCLUSIONS: JAKi therapy appears to be clinically effective, well tolerated, and safe in patients with refractory IIM, with a particular benefit in ILD. The steroid sparing effect was also a major outcome. Future prospective and controlled studies are warranted to confirm these preliminary results and better define the therapeutic potential of JAKi in IIM.

PMID:41678164 | DOI:10.55563/clinexprheumatol/0yu9gm

Eur Geriatr Med. 2026 Feb 12. doi: 10.1007/s41999-025-01363-0. Online ahead of print.

ABSTRACT

PURPOSE: To deliver an effective training intervention to staff in temporary elder care institutions in municipalities.

SETTINGS: A geriatric department at a university hospital in collaboration with 6 surrounding municipalities.

INTERVENTION: The training intervention was planned using self-determination theory as framework. Important elements were relation building, giving nursing staff a choice concerning themes of teaching sessions (supports autonomy) and selection of cases, supporting competence development through case-based discussions. Study design and statistical approach: before and after the intervention, an electronic questionnaire was mailed to staff at care institutions in the municipalities. The questionnaire contained questions drawn from the Basic Psychological Need Satisfaction and Frustration Scale (BPNSFS). Differences concerning competences between before and after the intervention were assessed using Wilcoxon signed-rank test.

KEY FINDINGS: 55 persons answered the BPNSFS before and after the intervention. For all items in BPNSFS, the results after compared to before improved. However, the improvement was significant only for the sum of all items with a mean difference of 0.13, p = 0.05, and for one single item (I feel capable at what I do at work), with a mean difference of 0.15, p = 0.02.

CONCLUSIONS: We have shown that self-determination theory has the potential to provide a theoretical framework for planning training interventions among staff in temporary elder care institutions. It is possible to measure the effect using an instrument developed within the framework of self-determination theory (BPNSFS).

PMID:41678141 | DOI:10.1007/s41999-025-01363-0

Adv Ther. 2026 Feb 12. doi: 10.1007/s12325-026-03504-8. Online ahead of print.

ABSTRACT

INTRODUCTION: Delamanid (Deltyba®), a medicinal product with an orphan designation for the treatment of tuberculosis, received a conditional marketing authorisation in the European Union (EU) based on phase 2 data, while phase 3 trial was ongoing. The list of adverse drug reactions (ADRs) in the original Summary of Product Characteristics (SmPC) contained all adverse events (AEs) considered related by the investigator that were reported in at least one of the 321 patients receiving delamanid. The safety profile observed after completion of the phase 3 clinical trial, post-marketing studies and spontaneous reports from post-marketing appeared different from what the initial SmPC was indicating. A comprehensive analysis was undertaken aiming to provide evidence for identification of a well-substantiated and clinically useful delamanid safety profile.

METHODS: In support of the process of ADR identification, a statistical methodology of data from controlled clinical trials was introduced based on the estimation of risk difference and risk ratio for identification of a potential risk from delamanid in high and low incidence situations. Final medical assessment was supported by this statistical analysis of data from controlled clinical trials, any signal from all other Otsuka-sponsored clinical trials, along with data from post-marketing solicited and unsolicited sources.

RESULTS: ADRs were either retained, added to or removed from the ADR list with the same or modified frequency. Thus, the total number of ADRs listed in the original SmPC was significantly reduced.

CONCLUSION: A combination of statistical parameters and medical judgement should be considered for the selection of undesirable effects for the product label and for the safety risk classification.

PMID:41678139 | DOI:10.1007/s12325-026-03504-8

Eur J Clin Microbiol Infect Dis. 2026 Feb 12. doi: 10.1007/s10096-026-05404-9. Online ahead of print.

ABSTRACT

PURPOSE : Chlamydia psittaci pneumonia (CPP) remains underdiagnosed due to nonspecific clinical manifestations. This study assessed the clinical utility of targeted next-generation sequencing (tNGS) in optimizing CPP diagnosis and antimicrobial stewardship, with a focus on empirical quinolone efficacy.

METHODS: We conducted a retrospective cohort study of 35 CPP patients (November 2022-October 2023) diagnosed by tNGS of respiratory specimens (8 sputum/27 bronchoalveolar lavage fluid [BALF]). Data included epidemiological history, laboratory findings, imaging features, therapeutic interventions, and clinical outcomes. Statistical comparisons between severe and non-severe CPP were performed using Student’s t-test and Mann-Whitney U tests.

RESULTS: Median diagnostic delay post-admission was 4 days (IQR:3-5). Fever predominated as initial presentation (97.1%), with 45.7% reporting avian contact. Leukocyte counts were normal/mildly elevated,, yet neutrophil ratio (83.86 ± 6.17%) and D-dimer (1.31 ± 0.86 mg/L) were notably increased. All patients showed elevated CRP (175.52 ± 87.62 mg/L) and ESR (70.00 ± 22.62 mm/h). Severe CPP cases (n = 8) exhibited higher CRP (p = 0.041) and procalcitonin (p = 0.013) than non-severe cases. Common comorbidities included hepatic dysfunction (68.6%) and pleural effusion (34.3%). Polymicrobial co-infections occurred more frequently in severe CPP cases than in non-severe cases (OR = 21.07, 95% CI:1.11-402.30). tNGS-guided diagnosis prompted antibiotic adjustment in 60.0% of patients (21/35) to targeted quinolone, tetracycline, or combination therapy. Clinical recovery was achieved in 97.1%, with 2.9% mortality.

CONCLUSIONS: tNGS enhances early CPP diagnosis and targeted antimicrobial adjustment. Quinolones demonstrate high efficacy as empirical treatment. The strong association between severe CPP and polymicrobial co-infections necessitates comprehensive pathogen screening. Study limitations include a single-center design and a small sample size, warranting validation through prospective multicenter studies.

PMID:41678126 | DOI:10.1007/s10096-026-05404-9

J Clin Sleep Med. 2026 Jan 5;22(1):10. doi: 10.1007/s44470-025-00014-2.

ABSTRACT

STUDY OBJECTIVES: Evidence of dairy’s association with sleep is limited. Therefore, we evaluated the association between dairy product consumption and self-reported and objective sleep outcomes in U.S. adults.

METHODS: Participants included 23,480 men and women (mean age 46.7 ± 17.3 years) from the 2005-2020 National Health and Nutrition Examination Survey. Dairy intake (total and sub-types) was estimated from two 24-h dietary recalls. We categorized average daily dairy intake as low, moderate, and high based on sex-specific tertiles. Sleep duration (< 7, 7-9 (ref.), ≥ 9 h/day) and trouble sleeping (yes/no) were obtained from questionnaires. Objective sleep measures were derived from accelerometer data in a subsample (n = 4,972). Survey-weighted multivariate logistic and linear regression were used to evaluate dairy intake in relation to self-reported and objective sleep outcomes, respectively.

RESULTS: Mean (SD) total dairy intake was 1.76 (1.34) cup-eq/day. High total dairy intake was associated with 16% and 23% lower odds of short and long sleep duration, respectively (all P-trend ≤ 0.01), and lower odds of trouble sleeping (OR_{high vs. low}=0.86; 95% CI 0.75-1.00; P-trend < 0.05). Similar associations between milk and sleep duration, and high-fat dairy and trouble sleeping were observed (all P-trend < 0.03). Moreover, high vs. low total dairy intake was associated with a higher sleep regularity index, and fermented dairy intake was associated with less variable sleep midpoint, and lower odds of poor sleep (all P-trend < 0.03).

CONCLUSIONS: Dairy consumption is associated with improved odds of adequate sleep, no trouble sleeping, and less variable sleep in U.S. adults. However, additional studies are warranted to assess causality. Current Knowledge/Study Rationale: Emerging research suggests that diet influences sleep health; however, evidence on the specific role of dairy products remains limited. We examined the association between dairy product consumption (total and subtypes) and both self-reported and objective sleep metrics in a nationally representative sample of U.S. adults. Study Impact: Dairy intake, especially of fermented dairy products, is associated with better sleep patterns, via improved odds of having adequate sleep, no trouble sleeping, and less sleep variability. Longitudinal studies and clinical interventions are needed to investigate causal associations and to clarify underlying mechanisms.

PMID:41678097 | DOI:10.1007/s44470-025-00014-2

J Clin Sleep Med. 2026 Feb 6;22(1):28. doi: 10.1007/s44470-025-00028-w.

NO ABSTRACT

PMID:41678095 | DOI:10.1007/s44470-025-00028-w

J Clin Sleep Med. 2025 Dec 22;22(1):7. doi: 10.1007/s44470-025-00019-x.

ABSTRACT

BACKGROUND: Adherence to positive airway pressure (PAP) therapy in obstructive sleep apnoea (OSA) remains suboptimal. Previous cognitive behavioural therapy (CBT) has relied on passive education approaches and has yielded mixed results.

OBJECTIVE: To evaluate whether personalised video footage showing one’s own sleep-disordered breathing events (Self-Viewing of Sleep Apnoea Videos, SVSV) improves PAP adherence.

METHODS: In this randomised controlled trial, 228 treatment-naïve patients with moderate to severe OSA were assigned to either the SVSV or standard CBT-only (control) group. All participants received standard CBT before PAP initiation. The SVSV group additionally viewed video footage of apnoeas from their own diagnostic polysomnography. The primary outcome was good adherence, defined as ≥ 4 h of sleep per night on ≥ 70% of nights for 90 days. Secondary outcomes included the number of days with adequate use, mean daily usage time, and apnoea-hypopnoea index (AHI) during PAP.

RESULTS: Good adherence was achieved in 89.5% of the SVSV and 78.1% of the control group (absolute difference, 11.4%; 95% confidence interval (CI), 1.94% to 20.86%). Compared with the control group, the SVSV group had more days with adequate usage (mean difference of 9.05 days; CI, 2.29 to 15.81) and longer daily usage (mean difference of 0.55 h; CI, 0.13 to 0.98). AHI during PAP therapy was similar between the two groups.

CONCLUSIONS: This study supports the clinical utility of patient-specific visual feedback to improve PAP adherence. SVSV-a simple, low-cost, and scalable strategy-resulted in a higher proportion of patients achieving good adherence and greater usage. 1. Current Knowledge/Study Rationale: Adherence to positive airway pressure therapy in obstructive sleep apnoea is often inadequate, limiting its therapeutic benefit. Previous educational approaches, including video-based cognitive behavioural therapy, have yielded inconsistent effects on adherence. 2. Study Impact: This prospective, randomised controlled trial shows that allowing patients to view video segments of their own sleep-disordered breathing events, extracted from diagnostic polysomnography, significantly improves positive airway pressure adherence. This personalised self-viewing intervention is simple, cost-effective, and easily integrated into existing clinical workflows, with particular value for underserved populations and individuals with limited health literacy.

PMID:41678091 | DOI:10.1007/s44470-025-00019-x

Invest New Drugs. 2026 Feb 12. doi: 10.1007/s10637-026-01602-6. Online ahead of print.

ABSTRACT

Cholangiocarcinoma (CCA) is a rare and aggressive biliary tract cancer with a poor prognosis. Despite recent advancements in molecularly targeted therapies for patients with IDH1 mutations and FGFR2 fusions, the efficacy of integrating these agents with first-line chemotherapy has not been established. This multicenter, Phase Ib dose de-escalation study, conducted prior to the establishment of chemoimmunotherapy as the standard first-line therapy, evaluated the safety and preliminary efficacy of combining ivosidenib or pemigatinib with gemcitabine and cisplatin in patients with advanced CCA. Eligible patients with no disease progression after three cycles of standard chemotherapy were assigned to receive either ivosidenib or pemigatinib based on their mutation status and chemotherapy. The primary endpoints were to evaluate safety, tolerability, the maximum tolerated dose, and the recommended Phase II dose. Secondary endpoints included progression-free survival (PFS), overall survival (OS), and best response profile. The study opened in January 2020 and closed early in November 2022, enrolling eight patients – seven in the ivosidenib arm (Arm A) and one in the pemigatinib arm (Arm B). Enrollment in Arm B was constrained due to challenges in identifying FGFR2 fusion/rearrangement-positive patients in the first-line setting. Treatment-related toxicities of grade 3 or higher occurred in four patients (66.7%) in Arm A. Regarding efficacy, one patient (16.7%) in Arm A achieved a partial response, while the remaining five had stable disease, with a median progression-free survival (PFS) of 15.4 months in Arm A and a median overall survival (OS) of 22.9 months. Given the selection bias and small sample size, these efficacy analyses must be interpreted with caution. Adding ivosidenib to gemcitabine and cisplatin in this study demonstrated a challenging safety profile in advanced CCA. Further research and dose optimization are warranted to confirm these findings and optimize the integration of targeted therapies into first-line regimens.

PMID:41678089 | DOI:10.1007/s10637-026-01602-6

J Clin Sleep Med. 2026 Jan 29;22(1):22. doi: 10.1007/s44470-025-00009-z.

ABSTRACT

STUDY OBJECTIVES: For Veterans living with a serious mental illness (SMI), insomnia is prevalent and harmful. Cognitive Behavioral Therapy for Insomnia (CBT-I) is an effective treatment, but Veterans with SMI experience significant environmental, psychological, and systemic barriers to receiving and benefiting from it. There is limited clinical guidance on using CBT-I with Veterans with SMI. We developed provider guidelines and patient materials for conducting CBT-I when these barriers are present for people with SMI (CBT-I for SMI) and evaluated the acceptability and preliminary efficacy of CBT-I for SMI in an assessor-blind randomized controlled trial.

METHODS: Forty-seven Veterans with insomnia and SMI were randomized to either CBT-I for SMI (n = 26) or Health and Wellness (HW; n = 21), an active control condition. At baseline, post-treatment, and 3-month follow-up, participants completed: actigraphy and daily sleep diaries for two weeks and self-report measures of insomnia and functioning. At 3-month follow-up, participants completed satisfaction ratings of treatment.

RESULTS: CBT-I for SMI participants had high treatment satisfaction and attendance. At post, compared to HW, CBT-I caused statistically significantly greater reductions in diary-measured time-in-bed, increases in diary-measured sleep efficiency, reductions in actigraphy-measured time-in-bed and total sleep time, and improvements in self-reported insomnia severity and sleep-related functioning; relative to CBT-I, HW improved community participation.

CONCLUSIONS: CBT-I for SMI is acceptable to Veterans with SMI and improves sleep and functioning. Future research should examine how sleep mediates effective functional gains, identify how CBT-I could be integrated within recovery centers, and develop preventative interventions to curtail insomnia-associated functional decline. People with serious mental illnesses experience challenges to receiving and benefiting from Cognitive Behavioral Therapy for Insomnia. To support providers and patients, we developed guidelines and materials for navigating these challenges while conducting Cognitive Behavioral Therapy for Insomnia with people with serious mental illness. In the first study to compare Cognitive Behavioral Therapy for Insomnia conducted with our materials and guidelines to an active treatment control, we found that our guideline-led Cognitive Behavioral Therapy for Insomnia improved self-reported insomnia severity, sleep-related functioning, and actigraphy and diary-measured sleep of Veterans with serious mental illness and insomnia. Veterans with serious mental illness can participate in, derive satisfaction from, and benefit from Cognitive Behavioral Therapy for Insomnia when this treatment is appropriately tailored.

PMID:41678078 | DOI:10.1007/s44470-025-00009-z

J Clin Sleep Med. 2026 Feb 11;22(1):32. doi: 10.1007/s44470-025-00036-w.

ABSTRACT

STUDY OBJECTIVES: Obstructive sleep apnea (OSA) impairs sleep and respiration, and sub-optimal adherence to its gold-standard CPAP therapy compels development of alternative approaches. This study investigates the effects of steam-assisted respiratory muscle training (RMT) on polysomnographic (PSG) outcomes in patients with OSA.

METHODS: In a 12-week open-label prospective pilot study, 60 working participants with mild to moderate OSA underwent individualized inspiratory and expiratory resistance training with adjunctive steam inhalation. PSG was conducted pre- and post-intervention. Primary outcomes included changes in respiratory indices (AHI, ODI₃, CT₉₀) and sleep quality metrics (sleep efficiency, WASO). Statistical analyses included the Shapiro-Wilk normality test, Paired T, Welch, or Wilcoxon comparing visits, Wilson CIs reporting responders, Mann-Whitney and Fisher assessing associations, regression predicting change, with significance set at p < 0.05.

RESULTS: Of 60 participants, 33 completed the study. Primary outcomes-respiratory indices and sleep continuity metrics-remained unchanged (all p > 0.05). Secondary analyses showed reduced REM latency, increased REM duration, and fewer periodic limb movements and arousal-related events (all p < 0.05). Regression analysis indicated that greater height and BMI were associated with fewer PLM, whereas larger waist circumference predicted more PLM.

DISCUSSION: Steam-assisted RMT did not significantly alter respiratory or sleep continuity indices but was associated with modest changes in REM architecture and limb movements. These findings should be interpreted cautiously, as exploratory observations in a non-controlled pilot setting. Larger randomized, sham-controlled trials with objective adherence monitoring are warranted to confirm these preliminary results.

PMID:41678040 | DOI:10.1007/s44470-025-00036-w