Sequential everolimus for angiomyolipoma associated with tuberous sclerosis complex: a prospective cohort study

Abstract Background To evaluate the efficacy, safety and health economics of sequential everolimus in treating angiomyolipoma (AML) associated with tuberous sclerosis complex (TSC). Methods In this prospective cohort study, patients met the inclusion criteria received standard or sequential treatment according to their willingness. All patients received an initial dose of everolimus (10 mg oral, once a day) for 3 months. The standard treatment group maintained 10 mg QD for 12 months, while the sequential treatment group reduced the dose to 5 mg QD from the 4th month. The efficacy, serum everolimus concentration and safety were evaluated at 1, 3, 6, 9 and 12 months after treatment. The primary efficacy endpoint was the proportion of patients with confirmed angiomyolipoma response of at least a 50% reduction in the total volume of target AML relative to baseline. Results Between June 1, 2016 and June 1, 2017, a total of 53 patients were included. Twenty-three patients received standard treatment, 30 patients received sequential treatment. At 1, 3, 6, 9 and 12 months after treatment, the proportion of patients whose total target tumor volume decreased by ≥ 50% from baseline was 39.1% versus 36.7%, 43.5% versus 56.7%, 47.8% versus 50%, 47.8% versus 60% and 47.8% versus 23.3% respectively (P > 0.05 for all). The overall response rate of skin lesions in the two groups was 40.4%, and the response rates of skin lesions at different times were similar for two groups (P > 0.05 for all). Major adverse effects (AEs) included mouth ulceration, hypertriglyceridemia, hypercholesterolemia, menstrual disorders. There was no significant difference between the two groups in the incidence of AEs at 3 months after treatment. The incidence of overall and grade 3/4 AEs at 12 months after treatment were significantly lower in the sequential treatment group. The average direct cost of the two groups in 12 months was $15,466 and $11,120, respectively. Conclusions Compared to standard treatment, sequential treatment was equally effective, with a lower incidence of adverse events and a lower direct cost, suggesting that it may be an alternative treatment for AML associated with TSC.

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Evolution of electroencephalogram in infants with tuberous sclerosis complex and neurodevelopmental outcome: a prospective cohort study

Developmental Medicine & Child Neurology ◽

10.1111/dmcn.15073 ◽

2021 ◽

Author(s):

Jessie De Ridder ◽

Katarzyna Kotulska ◽

Paolo Curatolo ◽

Anna C Jansen ◽

Eleonora Aronica ◽

...

Keyword(s):

Cohort Study ◽

Tuberous Sclerosis ◽

Prospective Cohort Study ◽

Tuberous Sclerosis Complex ◽

Prospective Cohort ◽

Neurodevelopmental Outcome

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Treatment with liraglutide, a glucagon-like peptide-1 analogue, improves effectively the skin lesions of psoriasis patients with type 2 diabetes: A prospective cohort study

Diabetes Research and Clinical Practice ◽

10.1016/j.diabres.2019.03.002 ◽

2019 ◽

Vol 150 ◽

pp. 167-173 ◽

Cited By ~ 6

Author(s):

Xiangjin Xu ◽

Lu Lin ◽

Pin Chen ◽

Yunjie Yu ◽

Shengping Chen ◽

...

Keyword(s):

Type 2 Diabetes ◽

Cohort Study ◽

Prospective Cohort Study ◽

Prospective Cohort ◽

Skin Lesions ◽

Glucagon Like Peptide ◽

Glucagon Like Peptide 1

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Sirolimus improves seizure control in pediatric patients with tuberous sclerosis: A prospective cohort study

Seizure ◽

10.1016/j.seizure.2020.03.018 ◽

2020 ◽

Vol 79 ◽

pp. 20-26 ◽

Cited By ~ 2

Author(s):

Wen He ◽

Jian Chen ◽

Yang-Yang Wang ◽

Meng-Na Zhang ◽

Qian-Lu ◽

...

Keyword(s):

Cohort Study ◽

Tuberous Sclerosis ◽

Prospective Cohort Study ◽

Prospective Cohort ◽

Pediatric Patients ◽

Seizure Control

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Erratum to “Sirolimus improves seizure control in pediatric patients with tuberous sclerosis: A prospective cohort study” [Seizure: Eur. J. Epilepsy, 79 (2020) 20–26]

Seizure ◽

10.1016/j.seizure.2020.08.026 ◽

2020 ◽

Vol 81 ◽

pp. 342

Author(s):

Wen He ◽

Jian Chen ◽

Yang-Yang Wang ◽

Meng-Na Zhang ◽

Qian Lu ◽

...

Keyword(s):

Cohort Study ◽

Tuberous Sclerosis ◽

Prospective Cohort Study ◽

Prospective Cohort ◽

Pediatric Patients ◽

Seizure Control

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Increasing exercise adherence among elderly patients with chronic disease in primary care: a prospective cohort study

BMC Geriatrics ◽

10.1186/s12877-021-02572-5 ◽

2021 ◽

Vol 21 (1) ◽

Author(s):

Seth VanDerVeer ◽

Ronald Markert ◽

Brant Bickford ◽

Joseph Yuhas ◽

Paul Pikman ◽

...

Keyword(s):

Blood Pressure ◽

Primary Care ◽

Heart Rate ◽

Cohort Study ◽

Chronic Disease ◽

Health Status ◽

Treatment Group ◽

Prospective Cohort Study ◽

Prospective Cohort ◽

Exercise Adherence

Abstract Background Currently, the evidence for exercise in maintaining health, well-being, and physical functioning is overwhelming. Despite these benefits, more than 50% of the population fails to meet the recommended exercise requirements for age and health status. In our study, we sought to provide a method to increase exercise adherence that was both effective and time-efficient for physicians and their patients. Methods The primary objective of this research study was to evaluate the effectiveness of a graded exercise protocol and biweekly monitoring on increasing the duration of aerobic exercise to 150 min per week in a population of elderly individuals with chronic disease. Secondarily, we evaluated for improvement in resting heart rate, blood pressure, body mass index (BMI), and cardiorespiratory fitness. The overall study design was a randomized, prospective cohort study with assessor blinding. Forty-five patients aged ≥60 years with multiple comorbidities were recruited from the Internal Medicine Clinic at Wright-Patterson AFB. Participants were randomized into a treatment or control arm and observed over a period of 34 weeks. Those in the treatment arm were given a graded walking protocol and received biweekly phone calls to evaluate compliance. Those in the control arm did not receive an intervention or biweekly monitoring. Measurements of heart rate, blood pressure, and BMI were taken quarterly in both groups. At the beginning and conclusion of the study, each participant completed a modified Balke treadmill test and Physical Activity Scale for the Elderly (PASE). Continuous variables were evaluated with the independent samples t-test, whereas categorical variables were evaluated with the chi-squared test. Results A greater percentage of the treatment group achieved the primary outcome (41.6% vs. 0%; p = 0.003). Those in the treatment group also had favorable improvements in heart rate response (− 2.4 beats/min vs. + 5.3 beats/min; p = 0.038) and PASE (+ 66 vs.-20; p < 0.001). No significant differences were observed between groups for mean change in heart rate, blood pressure, or BMI. Conclusion Guided, independent exercise and surveillance can be an effective tool in primary care practice to help patients reach the recommended levels of exercise for both age and health status.

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Butylphthalide Combined With Conventional Treatment Attenuates MMP-9 Levels and Increases VEGF Levels in Patients With Stroke: A Prospective Cohort Study

Frontiers in Neurology ◽

10.3389/fneur.2021.686199 ◽

2021 ◽

Vol 12 ◽

Author(s):

Yingqiong Xiong ◽

Juanjuan Liu ◽

Yang Xu ◽

Shu Xie ◽

Xinhua Zhou ◽

...

Keyword(s):

Cohort Study ◽

Treatment Group ◽

Prospective Cohort Study ◽

Prospective Cohort ◽

Conventional Treatment ◽

National Institutes Of Health ◽

Enzyme Linked Immunosorbent Assay ◽

Vascular Endothelial ◽

Conventional Treatment Group ◽

Endothelial Growth Factor

Background and Purpose: Butylphtalide increases the vascular endothelial growth factor (VEGF) and decreases matrix metalloproteinase (MMP)-9 in animal models of stroke and might be of use in the management of stroke. To explore whether butylphthalide combined with conventional treatment can change the levels of MMP-9 and VEGF and the National Institutes of Health Stroke Scale (NIHSS) scores of patients with stroke.Methods: This was a prospective cohort study involving inpatients admitted to the Jiangxi Provincial People's Hospital (January–June 2019) due to acute cerebral infarction. The patients received conventional treatments with or without butylphthalide. The changes in the NIHSS scores were compared between groups. Plasma MMP-9 and VEGF were measured by enzyme-linked immunosorbent assay.Results: A total of 24 patients were included in the conventional treatment group and 46 in the butylphthalide group. The butylphthalide group showed lower MMP-9 (130 ± 59 vs. 188 ± 65, p = 0.001) and higher VEGF (441 ± 121 vs. 378 ± 70, p = 0.034) levels on day 6 compared with the conventional treatment group. The changes in MMP-9 and VEGF were significant, starting on day 3 in the butylphthalide group but on day 6 in the conventional treatment group. There were no differences between the two groups in the NIHSS scores at admission and at discharge (p > 0.05). The overall response rate was higher in the butylphthalide group compared with the conventional treatment group (63.0 vs. 37.5%, p = 0.042).Conclusion: Butylphthalide combined with conventional treatment can decrease MMP-9 levels and increase VEGF levels. The patients showed the reduced NIHSS scores, possibly suggesting some improvement in prognosis after stroke. Still, the conclusions need to be confirmed in a larger sample and in different etiological subtypes of stroke.

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