Patient-reported impact of myasthenia gravis in the real world: findings from a digital observational survey-based study (MyRealWorld MG).

OBJECTIVES: This study aims to explore the impact of myasthenia gravis (MG) - in terms of treatments, side effects, comorbidities, psychological health and work or study- in the real world from a patient perspective. DESIGN AND PARTICIPANTS: This is a prospective, observational, digital, longitudinal study. Adults diagnosed with MG residing in the USA, Japan, Germany, the UK, Italy, Spain or Canada were eligible to participate in the study. There were no other exclusion criteria. Participants used a bespoke smartphone application to confirm eligibility, provide consent and enter data about their MG into a profile, a tracker to record MG-related events and a series of patient-reported outcome instruments. 1693 participants completed at least 1 survey and were included in this analysis. RESULTS: Results are presented as a percentage of respondents to each survey question. The study population was largely female (69% of 1586 respondents), with an average age of 49.9 years (SD 14.8). In the previous 12 months, 83.7% of 1412 respondents confirmed that they had received one or more routine treatments for MG, and 67.1% of 255 respondents confirmed that they had experienced a side effect in the previous month. Commonly experienced comorbidities reported by 966 respondents were thyroid problems, hypertension and anxiety, experienced by 37.5%, 31.4% and 28.0% of respondents, respectively.According to 889 respondents to the Hospital Anxiety and Depression Scale survey, 52.7% and 43.2% had a score indicative of at least mild anxiety and mild depression, respectively. Of 257 respondents, 33.0% reported experiencing a work or study impact in the past month. CONCLUSIONS: This analysis of baseline characteristics of the MyRealWorld MG study population indicates that, despite current treatments, patients experience notable burden. Further scheduled analyses will develop a longitudinal picture of MG burden. TRIAL REGISTRATION NUMBER: NCT04176211.

Patient-reported burden of myasthenia gravis: baseline results of the international prospective, observational, longitudinal real-world digital study MyRealWorld-MG.

OBJECTIVES: Myasthenia gravis (MG) is a rare, chronic, autoimmune neuromuscular disease which can affect functional and mental aspects of health and health-related quality of life (HRQoL). This study aims to obtain detailed knowledge of the impact of MG on HRQoL in a broad population from the perspective of the patient. DESIGN: Prospective, observational, digital, longitudinal real-world study. SETTING: Adult patients with MG from seven countries (USA, Japan, Germany, UK, Italy, Spain and Canada) downloaded a mobile application onto their phones and entered data about themselves and their MG. OUTCOME MEASURES: Data was collected using the following general and disease-specific patient-reported outcome measurements: EuroQol 5 Domains Health-Related Quality of Life Questionnaire (EQ-5D-5L), Myasthenia Gravis Activities of Daily Living (MG-ADL), Myasthenia Gravis Quality of Life 15-item revised scale (MG-QoL-15r), Hospital Anxiety and Depression Scale (HADS) and Health Utilities Index III (HUI3). Patients were categorised by their self-assessed Myasthenia Gravis Foundation of America (MGFA) class (I-V). RESULTS: Baseline results of 841 participants (mean age 47 years, 70% women) are reported . The distribution across the MGFA classes was: 13.9%, 31.0%, 38.1%, 15.5% and 1.6% for classes I-V. The MGFA class was a strong predictor of all aspects of HRQoL, measured with disease-specific and with generic instruments. The domains in which patients with MG most frequently mentioned problems were usual activities, anxiety and depression, tiredness, breathing and vision. The mean total MG-ADL Score was positively associated with increasing MGFA classes: 2.7, 4.4, 6.3 and 8.4 for MGFA classes I-IV. Mean baseline EQ-5D-5L utility was also associated with MGFA classes and was 0.817, 0.766, 0.648 and 0.530 for MGFA class I-IV. CONCLUSIONS: MG has a large impact on key aspects of health and HRQoL. The impact of this disease increases substantially with increasing disease severity.

Comparing the treatment patterns of patients with schizophrenia treated with olanzapine and quetiapine in the Pennsylvania Medicaid population.

OBJECTIVE: Compare treatment patterns for patients with schizophrenia treated with olanzapine versus quetiapine in the Pennsylvania Medicaid population. METHODS: Patients (18-64 years) with a diagnosis of schizophrenia (ICD-9-CM: 295.xx) and treated with olanzapine or quetiapine were identified from the Pennsylvania Medicaid claims database (1999-2003). Patients were continuously enrolled in the 12-month pre- and 12-month post-initiation periods. To control for selection bias, propensity score method with optimal matching algorithm was used to match patients from the two treatment groups. The key study outcomes including rates of augmentation, polypharmacy, discontinuation, and switching were analyzed using Kaplan-Meier survival analysis. Medication possession ratio and use of concurrent psychotropic drugs were also compared between the two groups. RESULTS: A total of 2321 quetiapine and 6929 olanzapine patients were identified. In all, 2321 pairs of patients were matched between the two groups and they had similar baseline characteristics. Over the 12-month study period, olanzapine patients had a better medication adherence (0.47 vs. 0.43; p < 0.0001), and were less likely to use other psychotropic medications concomitantly (all p < 0.05). Olanzapine patients had a significantly lower risk of augmentation and polypharmacy with other antipsychotics. The 6-month augmentation rates with antipsychotics were 12.9% and 16.7% for olanzapine and quetiapine, respectively (p < 0.05); the polypharmacy rates with any antipsychotics were 12.5% and 18.6% for olanzapine and quetiapine, respectively (p < 0.001). No significant differences were observed for discontinuation and switching between the two treatment groups. Sensitivity analysis with a 60-day minimum monotherapy requirement showed similar results. LIMITATIONS: This study's limitations include the analysis of a single Medicaid state, which may limit the generalizability to the entire Medicaid population with schizophrenia or to all patients with schizophrenia. CONCLUSION: This large Medicaid claims database analysis showed that olanzapine patients were significantly more compliant to treatment and less likely to augment or have polypharmacy with antipsychotics during the course of treatment compared to quetiapine patients.