Clinical and experimental rheumatology最新文献

Interstitial lung disease (ILD) affects a significant proportion of adults and children with idiopathic inflammatory myopathies (IIM-ILD and JIIM-ILD). Despite its major impact on mortality and therapeutic decision‑making, robust studies and clinical trials to inform evidence‑based practice are strikingly scarce. Heterogeneous clinical manifestations, variable testing practices, and lack of widely accepted nomenclature and standardised endpoint definitions magnify the challenges to design clinical trials for this novel disease subtype.Thus, the Myositis Clinical Trials Consortium (MCTC) developed the IIMILD working group (WG) to bridge the gaps and address the challenges unique to these patients. The IIM-ILD WG will accomplish these objectives by leveraging MCTC's global network of over 960 members, including physicians from multiple specialties, researchers, industry collaborators, and patient-support organisations. Collectively, the paper emphasises the need for structured phenotyping, unified terminology, and validated outcome measures as indispensable prerequisites for designing rigorous, multicentre trials in patients with IIM-ILD and JIIM-ILD. By leveraging the MCTC platform, the IIM-ILD Working Group will accelerate therapeutic development and ultimately improve outcomes for adults and children afflicted with IIM-associated ILD.

Objectives: Idiopathic inflammatory myopathies are systemic autoimmune diseases that can be associated with debilitating muscle weakness and significant limitations in daily activities. Physical activity monitors (PAM) are recognised measures of the frequency and intensity of physical activities. The aim of this study is to investigate the psychometric properties and performance of Fitbit® as a wearable activity tracker in IIM.

Methodds: Myositis Patient Centred Tele-Research (MyPACER) is a multi-centre observational prospective study conducted over 6 months recruiting patients with dermatomyositis (DM), necrotising myopathy (NM) and polymyositis (PM). The study had two cohorts, a Tele-Research Cohort (TRC, remote enrolment) and a Centre-Based Cohort (CBC, in person enrolment). Functional and patient-reported outcome measures were completed monthly. Participants were asked to use their wrist-worn Fitbit® for 7 consecutive days per month. Average daily steps per minute and average peak 1-minute cadence were evaluated as PAM measures.

Results: A total of 120 IIM patients (mean age 55.5±13.43; 75% females; 81% White) were enrolled; 82 in the TRC group and 38 in the CBC group. There were 51% DM, 39% PM, and 9% NM. The TRC and CBC cohorts were similar in demographics and disease subtypes. Gender, race/ethnicity, or disease subtypes were not associated with PAM measures. Compliance with Fitbit® wear protocol was very high, with similar results for remote or local recruitment. Average steps/min and average peak 1-minute cadence showed strong test-retest reliability [r=0.89 (p<0.0001 and r=0.86 (p= 0.0001)]. A longitudinal significant positive correlation was found between physical activity metrics and patient-reported and functional measures.

Conclusions: In a large IIM cohort, Fitbit® PAM variables demonstrate favourable compliance and psychometric properties with strong test-retest reliability and validity. PAMs can complement current measurements to remotely track patient performance, quality of life, and disease activity.

Objectives: The objective of this study is to assess the differences in environmental exposures of juvenile dermatomyositis (JDM) patients in Brazil versus the United States (U.S.).

Methods: JDM patients from 4 centres [3 U.S. (n=66), 1 Brazil (n=36)] were enrolled. Exposures during pregnancy were assessed by questionnaire, including occupational exposures, sources of inhalable pollution near the mother's home and work, and exposure to tobacco/alcohol.

Results: JDM mean age onset was 7.12 (SD±4.02) years for U.S. patients and 5.30 (SD±2.52) for Brazilians (p=0.004). During pregnancy, American mothers more frequently worked outside home than Brazilians (65.2% vs. 41.2%; p=0.032). Americans more often worked in offices (51.2% vs. 14.3%; p=0.027) and Brazilians, as teachers (28.6% vs. 4.8%; p=0.029). Americans commuted to work more frequently by subway (68.3% vs. 7.1%; p≤0.01), Brazilians, by bus (64.3% vs. 14.6%; p=0.001). Brazilians were more frequently exposed to dust (42.9% vs. 9.5%; p=0.01) and tobacco (50% vs. 23.1%; p=0.01). Places where Brazilians worked (35.7% vs. 9.1%; p=0.03) and lived (50% vs. 10.6%; p≤0.01) during pregnancy were closer to factories and quarries, as well as where the child was born (32.3% vs. 8.6%; p=0.007). After birth, Brazilian patients were more frequently exposed to tobacco, both through their fathers' smoking (26.5% vs. 6.2%; p=0.009) and other household residents (36.4% vs. 9.2%; p=0.002).

Conclusions: Earlier onset of symptoms, possibly related to early life environmental exposures, was observed in Brazilian patients. Their mothers lived and worked closer to factories and quarries, commuted to work by bus and were more exposed to dust and tobacco.

Objectives: This study aimed to identify risk and prognostic factors of Pneumocystis jirovecii pneumonia (PJP) in patients with anti-melanoma differentiation-associated gene 5 antibody-positive dermatomyositis (anti-MDA5+DM).

Methods: We conducted a retrospective cohort study of anti-MDA5+DM patients who underwent metagenomic next-generation sequencing analysis of bronchoalveolar lavage fluid or lung tissue at our center between January 2019 and February 2023. Eligible patients were stratified into PJP+ and PJP- groups based on PJP status. Potential risk factors and prognostic indicators for PJP were analysed using univariate and multivariate logistic regression analysis.

Results: A total of 107 anti-MDA5+DM patients were enrolled, of whom 47 were assigned to the PJP+ group. Multivariate logistic regression analysis revealed older age and high cumulative dosage of glucocorticoids within 3 months preceding PJP diagnosis were independent risk factors for PJP development. Conversely, prophylactic-dose trimethoprim-sulfamethoxazole (TMP/SMZ) was associated with a significantly reduced risk of PJP (all p<0.05). The 30-day mortality rate in the PJP+ group was 55.3%. Short disease duration and immunosuppressive therapy exposure, severe hypoxia, extensive radiological interstitial lung disease, moderate to severe acute respiratory distress syndrome, mechanical ventilation were associated with unfavourable prognosis (all p<0.05). Glucocorticoids therapy was more frenquently administered in survivors (p<0.05).

Conclusions: PJP significantly increases early mortality of anti-MDA5+DM patients. Clinicians should identify high-risk patients early and administer prophylactic-dose TMP/SMZ for PJP prophylaxis.

Objectives: To identify predictors of thrombotic events in patients with idiopathic inflammatory myopathies (IIM).

Methods: We conducted a retrospective study of a large, longitudinal IIM cohort followed at a single academic centre. We used Poisson regression models to estimate incidence-rate ratios (IRR) of prospective arterial and venous thrombotic events (ATE and VTE respectively).

Results: Thrombotic events occurred in 37 out of 312 patients (12%) over a median [interquartile range (IQR)] follow up time of 6[2-11] years after IIM diagnosis. Among patients with thrombotic events, 65% had VTE, which predominantly occurred within the first 3 years of IIM diagnosis, while 41% had ATE, which predominantly occurred after 10 years from IIM diagnosis. In predictive models, disease duration less than 1 year (IRR 6.49, 95%CI 1.89-22.35) was the strongest risk factor for VTE. Prior ATE was the strongest risk factor for prospective ATE (IRR 18.78, 95%CI 10.98-32.12). Traditional cardiovascular (CV) risk factors and higher levels of myositis activity and damage were other predictors of prospective ATE. The lactonase activity of PON1 was independent of the PON1 Q192R polymorphism and enhanced prediction of ATE in patients with high CV risk. IVIG was not associated with increased thrombotic risk in a high-risk population.

Conclusions: We report the incidence and risk factors for thrombotic events in a single-centre longitudinal IIM cohort, showing early occurrence of VTE and late onset of ATE. PON1 activity was predictive of ATE in a high-risk subgroup of IIM patients.