Female sex and overweight are associated with a lower quality of life in patients with myasthenia gravis: a single center cohort study.

BACKGROUND: Myasthenia gravis (MG) affects individuals as a chronic autoimmune disease for many years. Commonly, chronic diseases significantly reduce the patients' quality of life. Aiming to improve the future quality of life in MG, this study assessed the factors impacting quality of life. As gender-specific medicine is becoming increasingly important, this study also focused on understanding gender differences in the outcome of MG. METHODS: The study is a combined monocentric, retrospective and prospective database analysis of patient records based on 2,370 presentations of 165 patients with clinically, serologically and/or electrophysiologically confirmed MG over an observation period of up to 47 years. The data collection included the following parameters: antibody status, disease severity, age, medication use, gender, and disease duration. In addition, a prospective survey was conducted on the quality of life using the Myasthenia gravis-specific 15-item Quality of Life scale (MG-QoL15) and on the activities of daily living using the MG-specific Activities of Daily Living scale (MG-ADL). RESULTS: Of the 165 patients, 85 were male (51.5%) and 80 were female (48.5%). The remaining baseline characteristics (e.g. age and antibody status) were consistent with other myasthenia gravis cohorts. A high body mass index (BMI) (p = 0.005) and a high disease severity (p < 0.001) were significantly associated with lower disease-specific quality of life. Additionally, the quality of life in women with MG was significantly reduced compared to male patients (19.7 vs. 13.0 points in the MG-QoL15, p = 0.024). Gender differences were also observable in terms of the period between initial manifestation and initial diagnosis and women were significantly more impaired in their activities of daily living (MG-ADL) than men (4.8 vs. 3.0 points, p = 0.032). CONCLUSION: Women with MG had significantly poorer disease specific quality of life compared to men as well as patients with a higher BMI. In order to improve the quality of life, gender-specific medicine and further investigation regarding a modification of the quality of life by lowering the BMI are essential and necessary. TRIAL REGISTRATION: Study approval by the Ethics Committee of the University Medical Center Göttingen was granted (number 6/5/18).

Blinded sample size reestimation for negative binomial regression with baseline adjustment.

In randomized clinical trials, it is standard to include baseline variables in the primary analysis as covariates, as it is recommended by international guidelines. For the study design to be consistent with the analysis, these variables should also be taken into account when calculating the sample size to appropriately power the trial. Because assumptions made in the sample size calculation are always subject to some degree of uncertainty, a blinded sample size reestimation (BSSR) is recommended to adjust the sample size when necessary. In this article, we introduce a BSSR approach for count data outcomes with baseline covariates. Count outcomes are common in clinical trials and examples include the number of exacerbations in asthma and chronic obstructive pulmonary disease, relapses, and scan lesions in multiple sclerosis and seizures in epilepsy. The introduced methods are based on Wald and likelihood ratio test statistics. The approaches are illustrated by a clinical trial in epilepsy. The BSSR procedures proposed are compared in a Monte Carlo simulation study and shown to yield power values close to the target while not inflating the type I error rate.

Changes in the oral health-related quality of life in adult patients with intellectual disabilities after dental treatment under general anesthesia.

OBJECTIVES: This study aimed to assess self- and proxy-rated changes in the oral health-related quality of life (OHRQoL) in adult patients with intellectual disabilities after dental treatment in general anesthesia. METHODS: The OHRQoL in 52 adult patients with intellectual and developmental disabilities was assessed prior to dental treatment in general anesthesia (t0), 2 to 3 (t1) and 12 weeks (t2) postoperatively. Therefore, patients and primary cargivers were surveyed using either the 5-item short form of the Oral Health Impact Profile translated into easy language (OHIP-G5-easy, self-rating, n = 27) or the 14-item short form of the Oral Health Impact Profile (OHIP-G14, proxy-rating, n = 51), respectively. OHIP-G5-easy and OHIP-G14 summary scores were statistically analyzed with non-parametric mixed effects models and spearman correlation (p < 0.05). Additionally, the effect of demographic, anamnestic, oral health and treatment factors on QHRQoL or on changes of OHRQoL were analyzed using univariate and multivariate ordinal logistic regressions (p < 0.05). RESULTS: Self- (t0: 5.8 ± 4.6, t1: 4.3 ± 5.9, t2: 2.0 ± 3.9) and proxy-ratings (t0: 18.8 ± 11.6, t1: 10.1 ± 11.2, t2: 2.7 ± 5.3) revealed a significant improvement of OHRQoL after dental treatment in general anesthesia. Data showed a moderate correlation (p < 0.001, rSp = 0.43). OHRQoL at baseline was affected by the etiology of cognitive impairment and by the periodontal status (OHIP-G14). Improvement of OHRQoL was associated with the etiology of cognitive impairment (OHIP-G14) and the extent of dental treatment (OHIP-G5-easy). CONCLUSION: Self- and proxy-ratings revealed a significant improvement of OHRQoL in patients with intellectual disabilities after dental treatment in general anesthesia. CLINICAL RELEVANCE: Assessment of OHRQoL promotes patient-centred care of adults with intellectual disabilities.