Objectives: Myasthenia gravis (MG) may be refractory to traditional therapies. Quality of life (QOL) and disease burden in patients with refractory and nonrefractory MG were compared using Myasthenia Gravis Foundation of America MG Patient Registry data. Methods: Adults aged 18 years or older with MG diagnosed ≥2 years before enrollment were included. Participants with refractory MG had received ≥2 previous and 1 current MG treatment and had MG Activities of Daily Living Scale total score ≥6 at enrollment; other participants had nonrefractory MG. MG QOL 15-item scale (MG-QOL15) scores were compared. Results: In total, 56 participants with refractory and 717 participants with nonrefractory MG enrolled. Participants with refractory MG had significantly higher mean (SD) MG-QOL15 total scores [31.4 (11.1) vs. 20.8 (15.0), P < 0.0001] and were more likely to have had exacerbations, emergency department visits, and recent hospitalizations. Conclusions: Participants with refractory MG experience worse QOL and greater clinical burden than those with nonrefractory disease.

The objective of this study was to assess the incidence and clinical presentation of acute rheumatic fever in children and young adults in the north west of Western Australia--a region known as the Kimberley. In this five year retrospective study, residents aged less than 30 years, suspected of having had acute rheumatic fever from 1988 to 1992, were identified and their hospital and clinic records were reviewed. There were 96 cases of acute rheumatic fever that met the revised Jones criteria during the five year period. There were 59 initial attacks and 37 recurrences that occurred in 81 people, of whom 80 were of Aboriginal descent. The overall incidence of acute rheumatic fever in the Kimberley region for those aged under 30 years is 136 cases per 100,000 per year and in the Aboriginal population is 241 per 100,000 per year. The group most at risk are Aboriginal school children aged 5-14 years who accounted for 50 cases and have an annual incidence of 375 cases per 100,000 per year. The clinical presenting features were carditis in 50 per cent, polyarthritis in 59 per cent, chorea in 22 per cent, and subcutaneous nodules in 1 per cent. The hospitalization rate was 80 per cent for new cases and 73 per cent for recurrences with average stays of 14.6 and 1.5 days respectively. Rheumatic heart disease (RMD) developed in 31 of the cases; two required surgery and there was one death attributable to RHD in this group. Rheumatic fever continues to be a significant health problem in the Aboriginal population in this region and the recurrence rate, despite secondary prophylaxis, is also unacceptably high.

Objective: In the absence of head-to-head comparisons, the objective of this study was to conduct a network meta-analysis (NMA) to indirectly compare the relative efficacy and safety of rimegepant, ubrogepant, and lasmiditan for the acute treatment of migraine. Methods: A systematic literature review was conducted to identify randomized controlled trials (RCTs) of rimegepant, ubrogepant, and lasmiditan in adults with acute migraine. Outcomes included sustained pain freedom and -relief 2-48 hours post-dose, and adverse events. No RCTs were identified that directly compared these interventions. Therefore, a fixed-effects Bayesian NMA was conducted by identifying a connected (via comparison to placebo) network of RCTs. Results: Five RCTs were identified as follows: rimegepant study 303 (n = 1,466), ubrogepant ACHIEVE I and II (n = 1,672 and n = 1,686, respectively), and lasmiditan SAMURAI and SPARTAN (n = 2,231 and n = 3,005, respectively). Efficacy outcomes (pain freedom and relief at 2, 24, 48 hours) tended to be highest for lasmiditan 200 mg and rimegepant followed lower doses of lasmiditan and all doses of ubrogepant. However, lasmiditan 200 mg was also associated with higher rates of adverse events, particularly somnolence and dizziness. Conclusions: Lasmiditan, rimegepant, and ubrogepant all performed significantly better than placebo with respect to pain freedom and pain relief. Efficacy results were similar for rimegepant and lasmiditan with rimegepant having higher rates of pain freedom and relief than lower doses of lasmiditan, while somnolence and dizziness outcomes were lower for rimegepant than higher doses of lasmiditan.