RESUMO
BACKGROUND: Juvenile myasthenia gravis (JMG) is a rare autoimmune disease that causes fatigable muscle weakness in children aged <18 years. There is currently no curative treatment or internationally accepted standard of care for JMG. The objective is to investigate relationships between clinical presentation, antibody status, severity of disease onset, electrodiagnostic evaluation, and response to therapy in JMG. METHODS: This study was a retrospective chart review. Congenital myasthenic syndromes were excluded. Data on demographics, treatments, and outcomes were collected. Disease severity was evaluated using Myasthenia Gravis Foundation of America (MGFA) clinical classifications. RESULTS: We identified 84 patients with JMG at Children's Medical Center Dallas between January 2014 and February 2022. It was found that 52% of patients presented with ocular JMG (median onset age 4.5 years) and 48% with generalized JMG (median onset age 11.5 years); 81% tested positive for acetylcholine receptor antibodies. Patients were 17% non-Hispanic white, 29% Hispanic, 39% black, and 12% Asian. There was a significant difference in average MGFA scores between ethnicities (P = 0.047) and age groups (P = 0.004), with postpubertal patients having higher average MGFA scores than prepubertal patients. Seventy-one percent of patients who underwent thymectomy experienced a decrease in MGFA scores postprocedure. CONCLUSIONS: Our study showed that there were significant differences in disease severity between ethnicities and age groups and that most patients who underwent thymectomy showed clinical improvement. These outcomes highlight the need for additional therapies in the treatment of JMG and the importance of extending clinical trials to the pediatric population.
Assuntos
Miastenia Gravis , Humanos , Miastenia Gravis/terapia , Miastenia Gravis/diagnóstico , Miastenia Gravis/fisiopatologia , Criança , Masculino , Feminino , Estudos Retrospectivos , Texas , Adolescente , Pré-Escolar , Resultado do Tratamento , Timectomia , Índice de Gravidade de Doença , Idade de InícioRESUMO
BACKGROUND: Increased incidence and life expectancy have resulted in a growing population of patients with metastatic breast cancer, and these patients experience high rates of morbidity and premature mortality. Increased physical activity (PA) is consistently associated with improved health and disease outcomes among early-stage survivors. However, there is a paucity of research on PA in patients with metastatic breast cancer, and existing PA interventions have exhibited low feasibility because of their focus on intense PA and/or requirement of on-site visits. Mobile health (mHealth)-based PA interventions may be particularly useful for patients with metastatic breast cancer because they allow for remote monitoring, which facilitates individual tailoring of PA recommendations to patients' abilities and may minimize participant burden. However, no studies have examined mHealth PA interventions in patients with metastatic breast cancer. OBJECTIVE: We aim to address these critical research gaps by testing a highly tailored technology-based intervention to promote PA of any intensity (ie, light, moderate, or vigorous) by increasing daily steps in patients with metastatic breast cancer. The primary aim of this study is to test the feasibility and acceptability of the Fit2ThriveMB intervention. We will also examine outcome patterns suggesting the efficacy of Fit2ThriveMB on symptom burden, quality of life, and functional performance. METHODS: The Fit2ThriveMB trial is a two-arm pilot randomized controlled trial that will compare the effects of a smartphone-delivered, home-based PA intervention and an attention-control education condition on PA and quality of life in low-active female patients with metastatic breast cancer. A subsample (n=25) will also complete functional performance measures. This innovative trial will recruit 50 participants who will be randomized into the study's intervention or control arm. The intervention will last 12 weeks. The Fit2ThriveMB intervention consists of a Fitbit, coaching calls, and the Fit2ThriveMB smartphone app that provides self-monitoring, a tailored goal-setting tool, real-time tailored feedback, app notifications, and a group message board. Assessments will occur at baseline and post intervention. RESULTS: The Fit2ThriveMB study is ongoing. Data collection ended in February 2021. CONCLUSIONS: Data from this study will provide the preliminary effect sizes needed to assemble an intervention that is to be evaluated in a fully powered trial. In addition, these data will provide essential evidence to support the feasibility and acceptability of using a technology-based PA promotion intervention, a scalable strategy that could be easily integrated into care, among patients with metastatic breast cancer. TRIAL REGISTRATION: ClinicalTrials.gov NCT04129346; https://clinicaltrials.gov/ct2/show/NCT04129346. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): DERR1-10.2196/24254.
RESUMO
BACKGROUND: Increased activity is beneficial during chemotherapy, but treatment-related symptoms may be a barrier. This study examines the relationship between daily fluctuations in symptoms and activity during chemotherapy. METHODS: Women undergoing chemotherapy for breast cancer [n = 67; M age = 48.6 (SD = 10.3)] wore an accelerometer 24 hours/day and received four text prompts/day to rate symptoms for 10 consecutive days at the beginning, middle, and end of chemotherapy. Mixed-effects models were used to examine the between and within-person relationships between symptom ratings on a given day and moderate to vigorous physical activity (MVPA) and light physical activity (LPA) on that day and the following day controlling for relevant covariates and using the Bonferroni correction for multiple comparisons. RESULTS: For MVPA and LPA, within-person associations were statistically significant for same day affect, fatigue, pain, walking, activities of daily living (ADL) physical function, and cognitive function. Previous day anxiety was associated with next day LPA. Every one point worse symptom rating than an individual's overall average was associated with: (i) between 1.49 (pain) and 4.94 (fatigue) minutes less MVPA and between 4.48 (pain) and 24.72 (ADL physical function) minutes less LPA that day, and (ii) 11.28 minutes less LPA the next day. No between-person effects were significant for MVPA or LPA. CONCLUSIONS: Daily within-person variations in symptoms were associated with MVPA and LPA during chemotherapy for breast cancer. IMPACT: Future work should explore relationships between symptoms and activity further and identify whether tailoring to symptoms enhances efficacy of physical activity promotion interventions during chemotherapy.