Rationale: Patients with cystic fibrosis periodically experience pulmonary exacerbations. Previous studies have noted that some patients' lung function (FEV 1 ) does not improve with treatment. Objectives: To determine the proportion of patients treated for a pulmonary exacerbation that does not recover to spirometric baseline, and to identify factors associated with the failure to recover to spirometric baseline. Methods: Cohort study using the Cystic Fibrosis Foundation Patient Registry from [2003][2004][2005][2006]. We randomly selected one pulmonary exacerbation treated with intravenous antibiotics per patient and compared the best FEV 1 in the 3 months after treatment with the best FEV 1 in the 6 months before treatment. Recovery to baseline was defined as any FEV 1 in the 3 months after treatment that was greater than or equal to 90% of the baseline FEV 1 . Multivariable logistic regression was used to estimate associations with the failure to recover to baseline FEV 1 . Measurements and Main Results: Of 8,479 pulmonary exacerbations, 25% failed to recover to baseline FEV 1 . A higher risk of failing to recover to baseline was associated with female sex; pancreatic insufficiency; being undernourished; Medicaid insurance; persistent infection with Pseudomonas aeruginosa, Burkholderia cepacia complex, or methicillin-resistant Staphylococcus aureus; allergic bronchopulmonary aspergillosis; a longer time since baseline spirometric assessment; and a larger drop in FEV 1 from baseline to treatment initiation. Conclusions: For a randomly selected pulmonary exacerbation, 25% of patients' pulmonary function did not recover to baseline after treatment with intravenous antibiotics. We identified factors associated with the failure to recover to baseline, allowing clinicians to identify patients who may benefit from closer monitoring and more aggressive treatment.Keywords: cystic fibrosis; pulmonary exacerbation; pulmonary function tests; spirometry; antibiotics Lung disease is the major source of morbidity and mortality for patients with cystic fibrosis (CF) (1). One of the hallmarks of CF lung disease is the pulmonary exacerbation, characterized by an increase in pulmonary symptoms, decrease in pulmonary function, loss of energy, weight loss, and changes in physical findings (2). Pulmonary exacerbations have been linked to increased mortality (3, 4); higher health care costs (5); reduced quality of life (6); and a faster subsequent decline in pulmonary function (FEV 1 ) (7). In 2008, 38% of patients with CF in the Cystic Fibrosis Foundation Patient Registry (CFFPR) were treated with intravenous (IV) antibiotics for at least one pulmonary exacerbation (8).Previous studies have noted that some patients' FEV 1 values do not improve with IV antibiotic treatment (9-11). In a retrospective analysis of the placebo arm of an inhaled tobramycin trial, Smith and coworkers (9) showed that 23 out of 77 patients' FEV 1 did not improve after treatment with IV antibiotics to levels achieved at the clinic visit before the exacerbat...

We assessed the influence of race, sex, and puberty upon clinical features and outcome in 115 patients with autoimmune juvenile myasthenia gravis (JMG). These demographic variables influenced not only disease incidence but also disease severity, response to therapy, and outcome, despite comparable therapeutic strategies. Among white patients, those with prepubertal onset had low incidence and equal sex ratio; the incidence in females increased during and after puberty; males had lesser disease severity than females during and after puberty (p < 0.05); spontaneous remissions were most frequent (44%, p = 0.001) and persistence of active JMG for more than 10 years was least frequent (p = 0.05) in patients with prepubertal onset; remissions were more frequent after early than late thymectomy (p = 0.03); and final disease severity was less after early than late thymectomy. Black patients had similar incidence, disease severity, and sex ratio (F:M = 2:1) with pre-, peri-, or postpubertal disease onset; infrequent spontaneous or treatment-induced remissions; and the same final disease severity after early or late thymectomy. These observations imply that race and sex hormones modify the clinical features and outcome of JMG; spontaneous remissions are common in white patients with prepubertal disease onset; early thymectomy may be more beneficial than late thymectomy in white patients; and the role of thymectomy in the youngest patients is uncertain. We suggest that demographic factors should be considered when evaluating past and future therapeutic strategies for JMG.

Article abstract-In an open-label study, 12 patients with refractory MG or who were taking only corticosteroids and required additional immunosuppression received mycophenolate mofetil 1 g twice daily for 6 months. A reduction of three points in a quantified MG score and two points in a manual muscle test or a reduction of 50% in corticosteroid dose defined efficacy. Eight patients improved, beginning after 2 weeks to 2 months. No major side effects were observed. NEUROLOGY 2001;56:97-99 E. Ciafaloni, MD; J.M. Massey, MD; B. Tucker-Lipscomb, RN, BSN, APN; and D.B. Sanders, MD Mycophenolate mofetil (MM) (CellCept, Roche Laboratories, Nutley, NJ) is a novel and potent immunosuppressive agent. It blocks purine synthesis in activated T and B lymphocytes and selectively inhibits their proliferation while leaving other cell lines intact.1 It has been proven effective in preventing organ rejection in renal transplant patients when used with corticosteroids and cyclosporine.2,3 It has a strong safety profile and no major organ toxicity or mutagenic effect.2-5 Immunosuppressive therapy is effective in MG, and the use of corticosteroids, azathioprine, and cyclosporine have changed the prognosis of this autoimmune disease. Unfortunately, these agents all carry serious side effects, and some patients do not tolerate them or respond adequately to them. The possibility of a new effective and safe immunosuppressive agent to add to our MG treatment armamentarium is very attractive. The use of MM in MG has not been studied, but successful treatment of one patient with severe, refractory MG suggested its potential role in this disease. who had or had not undergone thymectomy and who had or did not have elevated acetylcholine receptor antibodies (AChR Ab) were eligible for the study. Two groups of patients were studied: 1) patients with refractory MG defined by a quantified MG (QMG) 8 score of at least 10 and a manual muscle test (MMT) score (figure) of at least 5, despite treatment with corticosteroids and azathioprine for at least 2 years or cyclosporine for at least 1 year; and 2) patients taking corticosteroids as sole therapy for at least 8 months and requiring additional immunosuppression to heighten the clinical improvement or to reduce the corticosteroid side effects. No plasmapheresis, IV immunoglobulin treatment, or change in medication dose was permitted for 3 months before enrollment. Anticholinesterase medications were continued, and examinations were performed 4 hours after the most recent dose. MM was administered in an oral dose of 1 g every 12 hours for 6 months, and clinic visits were performed every 2 months. The primary measure of efficacy was a reduction of at least 3 points in the QMG and at least 2 points in the MMT, or a reduction of at least 50% in corticosteroid dose for at least 3 months without worsening of the QMG and MMT scores. An activities of daily living (ADL) 9 questionnaire was also administered by phone every week for the first month and at clinic visits every 2 months thereafter, and was ...

The authors report a retrospective analysis of the use of mycophenolate mofetil (MyM) in 85 patients with autoimmune myasthenia gravis. The Myasthenia Gravis Foundation of America (MGFA) postintervention status (PIS) was used to characterize the treatment response in each patient. Sixty-two patients (73%) achieved a PIS status indicating improvement. Quantitative strength testing performed on the majority of patients before and after treatment also improved. Side effects to MyM were observed in 27% of patients but required discontinuation in only 6%.