Subgroup stratification and outcome in recently diagnosed generalized myasthenia gravis.

OBJECTIVE: To describe disease outcomes of myasthenia gravis (MG) subgroups and which factors influence outcomes by reviewing individual patient records of a representative cohort. METHODS: We performed a retrospective analysis of 199 patients with generalized MG and disease onset after the year 2000 who were treated at 2 tertiary referral centers in Austria. We stratified patients as early- and late-onset acetylcholine receptor antibody-positive, muscle-specific tyrosine kinase (MuSK) antibody-positive, and seronegative patients and patients with thymoma regardless of antibody status. We evaluated patients' symptom severity and treatment regimens and the occurrence of life-threatening events at yearly time points for up to 10 years. RESULTS: Minimal manifestation status or better was eventually achieved and sustained for >1 year by 125 (63%) patients. Forty percent (66 of 165 patients) showed an early response to treatment, which predicted a benign disease course later on. In contrast, 19% of patients, who remained symptomatic for 2 years after disease onset despite immunosuppressive therapy, were more treatment resistant in the following years. The strongest predictor of outcome was the diagnostic subgroup. Patients with MuSK-MG had a much better outcome than previously reported. CONCLUSION: Our data give an update on the disease course of generalized MG in the new century. Diagnostic subgroups and response to treatment within the first 2 years help to predict the long term outcome.

Frequency and clinical features of treatment-refractory myasthenia gravis.

BACKGROUND: To investigate the frequency and characterize the clinical features of treatment-refractory myasthenia gravis in an Austrian cohort. METHODS: Patient charts of 126 patients with generalized myasthenia gravis and onset between 2000 and 2016 were analyzed retrospectively. Patients were classified as treatment-refractory according to strict, predefined criteria. These mandated patients being at least moderately symptomatic (i.e., MGFA class III) or needing either maintenance immunoglobulins or plasma exchange therapy for at least 1 year in spite of two adequately dosed immunosuppressive drugs. Clinical features and outcome at last follow-up were compared to treatment-responsive patients. RESULTS: 14 out of 126 patients (11.1%) met these criteria of treatment-refractory myasthenia gravis. Treatment-refractory patients had more frequent clinical exacerbations and more often received rescue treatments or a further escalation of immunosuppressive therapies. They also remained more severely affected at last follow-up. An early onset of myasthenia gravis was associated with a higher risk for a refractory course. CONCLUSION: A small subgroup of patients with generalized myasthenia gravis do not respond sufficiently to standard therapies. Refractory disease has considerable implications for both patients and health care providers and highlights an unmet need for new treatment options.

Iodinated contrast agents in patients with myasthenia gravis: a retrospective cohort study.

Currently, it has not been satisfactorily established, whether modern low-osmolality iodinated contrast agents (ICAs) used in computed tomography (CT) studies are a risk factor for exacerbation of myasthenic symptoms. The rate of acute adverse events as well as delayed clinical worsening up to 30 days were analyzed in 73 patients with confirmed myasthenia gravis (MG) who underwent contrast-enhanced CT studies and compared to 52 patients who underwent unenhanced CT studies. One acute adverse event was documented. 12.3% of MG patients experienced a delayed exacerbation of symptoms after ICA administration. The rate of delayed severe exacerbation was higher in the contrast-enhanced group. Alternative causes for the exacerbation of MG-related symptoms were more likely than ICA administration in all cases. ICA administration for CT studies in MG patients should not be withheld if indicated, but patients particularly those with concomitant acute diseases should be carefully monitored for exacerbation of symptoms.

Sexual and Psychosocial Functioning in Women with MRKHS after Neovaginoplasty According to Wharton-Sheares-George: A Case Control Study.

BACKGROUND: Mayer-Rokitansky-Küster-Hauser syndrome (MRKHS) has a major impact on a woman's psychological and sexual well-being. In most of the studies that addressed treatment techniques, postoperative sexual function was reported to be satisfactory; however, comparatively few studies have additionally provided an accurate analysis of psychosocial functions in MRKHS patients following different kinds of neovaginal treatment. This study is to evaluate sexual and psychosocial functioning after creation of a neovagina according to Wharton-Sheares-George in women with MRKHS. METHODS: We performed a case-control-study using multiple measures to assess sexual and psychosocial functioning. Ten MRKHS patients and 20 controls of a University hospital and tertiary center for pediatric and adolescent gynecology were assessed. The follow-up assessment comprised 6 standardized questionnaires (Female Sexuality Function Index, FSFI; Patient Health Questionnaire, PHQ; Brief Symptom Inventory, BSI; World Health Organization Quality of Life Assessment, WHOQoL-BREF; Parental Bonding Instrument, PBI; and a German questionnaire on body image). The main outcome measures were sexual function, psychological status, quality of life, body image, and parental bonding styles. FINDINGS: Sexual function, psychological status (including depressive and somatic symptoms), quality of life, and own-body experience were at least as good in operated MRKHS patients as in controls. In some measures (FSFI, PHQ-15, psychological domain of the WHOQoL-BREF, and BSI Positive Symptom Total), patients scored significantly better than controls. The results of the PBI indicated a close and sustainable mother-daughter-relationship in MRKHS patients. CONCLUSIONS: We found no evidence for an impairment of sexual or psychosocial functioning in patients after neovaginoplasty according to Wharton-Sheares-George. MRKHS may not necessarily compromise sexual and psychological well-being, provided that the syndrome is properly managed by a multidisciplinary team of health professionals.