Patients with Glycogen Storage Disease type II (GSDII), an inheritable metabolic myopathy also known as Pompe disease, are considered to be at risk for severe COVID-19 due to a reduced respiratory ...function and a tendency to be overweight. However, so far little is known about the course of SARS-CoV-2 infection and side effects of COVID-19 vaccinations in patients with GSDII.
169 Dutch Pompe patients are followed at the Erasmus MC Rotterdam. During the COVID-19 pandemic patients were requested to directly inform their physicians about SARS-CoV-2 infection. Infected patients were interviewed regularly by telephone until their symptoms subsided. Furthermore, all patients eligible for vaccination on 16-7-2021 (≥ 17 years, n = 122) were asked to complete a questionnaire.
To date, fifteen patients (8.9% of our cohort) reported a SARS-CoV-2 infection (classic infantile Pompe disease n = 5, late onset n = 10). No patients were admitted to hospital or needed intensivation of ventilatory support. All patients made a recovery within 19 days. 41.8% of patients filled in our questionnaire regarding vaccination, of whom 98% were vaccinated. Besides one case of perimyocarditis, only mild side effects were reported.
Overall, patients with Pompe disease showed mild symptoms from infection with SARS-CoV-2. All patients made a full recovery. Side effects after vaccination were mostly mild.
Cerebral arterial air embolism (CAE) is a serious and potentially dangerous condition that can interrupt the blood supply to the brain and cause stroke. One of the promising gas mixtures for ...emergency treatment of air embolism is an oxygen-helium mixture.
We modeled CAE in awake rats by injecting air into the common carotid artery. Immediately after CAE, animals were either untreated or underwent hyperbaria, oxygen inhalation, heated air inhalation, or helium-oxygen mixture inhalation. Body temperature, locomotor activity, respiratory and cardiovascular parameters were monitored in the animals before CAE modeling, and 3 and 24 h after CAE modeling.
After 3 hours of CAE modeling in awake rats, depression of the nervous, cardiovascular and respiratory systems, as well as decreased body temperature were observed. 24 h after CAE modeling multifocal cerebral ischemia was observed. Normobaric helium-oxygen mixture inhalation, on par with hyperbaric treatment, restored body temperature, locomotor activity, respiratory volume, respiratory rate, and blood pressure 3 hours after CAE, and prevented the formation of ischemic brain damage lesions 24 h after CAE.
Thus, inhalation of a heated oxygen-helium gas mixture (O2 30% and He 70%) immediately after CAE improves the physiological condition of the animals and prevents the foci of ischemic brain damage formation.
In 2011 a 12 weeks personalized exercise training program in 23 mildly affected adult late onset Pompe patients (age 19.6-70.5 years) improved endurance, muscle strength and function. Data on ...long-term effects of this program or of other physical activity in Pompe disease are absent. This retrospective cohort study aimed to explore effects of long-term healthy physical activity according to the WHO norm and the former exercise training program on the disease course. A total of 29 adult late onset Pompe patients were included: 19 former exercise training program participants and 10 comparable control patients. Patients, who based on interviews, met the 2010 WHO healthy physical activity norm (active, n = 16) performed better on endurance (maximal cardiopulmonary exercise test), muscle strength and function compared to patients not meeting this norm (inactive, n = 13) (p < 0.05). Majority of the outcomes, including endurance and manually tested muscle strength, tended to be higher in the active patients of the 2011 training cohort who continued the program compared to active control patients (p > 0.05). In Pompe disease long-term healthy physical activity according to the 2010 WHO norm leads to physical benefits and a personalized exercise training program may have additional favorable effects and both should be recommended as standard of care.
Terpenoids from the Plant Ferula ferganensis Babekov, A. U.; Yuldasheva, N. M.; Ismailova, A. M. ...
Chemistry of natural compounds,
09/2022, Letnik:
58, Številka:
5
Journal Article
Male Wistar rats aged 10 months were assigned to groups according to the initial level of systolic BP: hypertensive (systolic BP >115 mm Hg) and normotensive (systolic BP <115 mm Hg). The animals ...were injected intraperitoneally with 100 μg/kg taxifolin daily for 7 days. Systolic BP and HR were measured on the next day after single taxifolin administration and on the next day after 7-day injection course. In the group of hypertensive animals, systolic BP markedly decreased on the next day after the first injection; this decrease became even more pronounced (to the level of normotensive animals) at the end of the taxifolin course. In the group of normotensive animals, systolic BP remained unchanged. Hence, we demonstrate the possibility of course administration of taxifolin for BP normalization in hypertensive patients.
α7-Type nicotinic acetylcholine receptor (α7-nAChR) promotes the growth and metastasis of solid tumors. Secreted Ly6/uPAR-Related Protein 1 (SLURP-1) is a specific negative modulator of α7-nAChR ...produced by epithelial cells. Here, we investigated mechanisms of antiproliferative activity of recombinant SLURP-1 in epidermoid carcinoma A431 cells and activity of SLURP-1 and synthetic 21 a.a. peptide mimicking its loop I (Oncotag) in a xenograft mice model of epidermoid carcinoma. SLURP-1 inhibited the mitogenic pathways and transcription factors in A431 cells, and its antiproliferative activity depended on α7-nAChR. Intravenous treatment of mice with SLURP-1 or Oncotag for 10 days suppressed the tumor growth and metastasis and induced sustained changes in gene and microRNA expression in the tumors. Both SLURP-1 and Oncotag demonstrated no acute toxicity. Surprisingly, Oncotag led to a longer suppression of pro-oncogenic signaling and downregulated expression of pro-oncogenic miR-221 and upregulated expression of KLF4 protein responsible for control of cell differentiation. Affinity purification revealed SLURP-1 interactions with both α7-nAChR and EGFR and selective Oncotag interaction with α7-nAChR. Thus, the selective inhibition of α7-nAChRs by drugs based on Oncotag may be a promising strategy for cancer therapy.
The fingerprint regions of infrared and Raman spectra of aqueous solutions of acetic acid at concentrations of 0.3 and 0.8 mole fractions and pure solutions were compared with the calculated data for ...terahertz (THz) spectroscopy. A molecular-dynamics simulation of THz spectroscopy of aqueous solutions of acetic acid was presented. THz spectroscopy was shown to be informative for determining the structural reorganization of water molecules in aqueous solutions of acetic acid. Special attention was paid to the collective dynamics of water molecules depending on the concentration of acetic acid.
Previously, it was shown that the non-conventional toxin WTX from the venom of the cobra
Naja kaouthia
, when administered intravenously, caused a decrease in blood pressure (BP) and an increase in ...heart rate (HR) in rats 13. To identify the site of the toxin molecule responsible for these effects, we studied the influence of synthetic peptide fragments of the WTX on BP and HR in normotensive male Sprague–Dawley rats under general anesthesia induced by Telazol and Xylazine. It was found that peptides corresponding to the WTX central polypeptide loop, stabilized by a disulfide bond, at intravenous injection at concentrations from 0.1 to 1.0 mg/mL caused a dose-dependent decrease in BP, with the HR increasing only in the first 5–10 min after administration. Thus, WTX fragments corresponding to the central polypeptide loop reproduce the decrease in blood pressure caused by the toxin.
Background
Enzyme replacement therapy (ERT) with alglucosidase alfa is the treatment for patients with Pompe disease, a hereditary metabolic myopathy. Home-based ERT is unavailable in many countries ...because of the boxed warning alglucosidase alfa received due to the risk of infusion-associated reactions (IARs). Since 2008, home infusions have been provided in The Netherlands.
Objectives
This study aimed to provide an overview of our experience with home-based infusions with alglucosidase alfa in adult Pompe patients, focusing on safety, including management of IARs.
Method
We analysed infusion data and IARs from adult patients starting ERT between 1999 and 2018. ERT was initially given in the hospital during the first year. Patients were eligible for home treatment if they were without IARs for multiple consecutive infusions and if a trained home nurse, with on-call back-up by a doctor, was available. The healthcare providers graded IARs.
Results
We analysed data on 18,380 infusions with alglucosidase alfa in 121 adult patients; 4961 infusions (27.0%) were given in hospital and 13,419 (73.0%) were given at home. IARs occurred in 144 (2.9%) hospital infusions and 113 (0.8%) home infusions; 115 (79.9% of 144) IARs in hospital and 104 (92.0% of 113) IARs at home were mild, 25 IARs (17.4%) in hospital and 8 IARs (7.1%) at home were moderate, and very few severe IARs occurred (4 IARs in hospital 2.8% and 1 IAR at home 0.9%). Only one IAR in the home situation required immediate clinical evaluation in the hospital.
Conclusion
Given the small numbers of IARs that occurred with the home infusions, of which only one was severe, we conclude that alglucosidase alfa can be administered safely in the home situation, provided the appropriate infrastructure is present.
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