Deutsche medizinische Wochenschrift
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Givosiran is a small synthetic double-stranded siRNA (small interfering RNA) conjugated with N-acetyl-galactosamine (GalNAc) for specific hepatocyte targeting via the asialoglycoprotein receptor. A prospective randomized multicenter study (Envision) demonstrated the clinical efficacy of monthly subcutaneous injection of Givosiran for the prevention of attacks of acute hepatic porphyria (AHP). This leads to highly selective transcriptional inhibition of the key hepatic enzyme, aminolaevulinate synthase 1, that is overexpressed in AHP. ⋯ This innovative guided siRNA therapy has opened up the possibility to selectively inhibit the expression of any hepatocyte gene whose overexpression that causes pathology, which can be considered a milestone development in hepatology. However, currently this treatment with givosiran is very costly. Moreover, since some patients experience worsening of kidney function and elevated aminotransferases, monthly monitoring of these parameters is necessary in the first half year of treatment.
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Dtsch. Med. Wochenschr. · Aug 2021
[Update on primary spontaneous pneumothorax - conservative or primarily surgical therapy?]
Spontaneous pneumothorax is a potentially life-threatening situation. Therefore, it is mandatory to treat it safely. The incidence is approximately 10 out of 100 000 residents per year. ⋯ After diagnosis pneumothorax is traditionally treated by an insertion of a thoracic tube. Recently, thoracic ultrasound gained influence in diagnosis of pneumothorax and primarily conservative treatment strategies have been shown to be safe and equally effective in particular groups of patients. This article aims to present and discuss these upcoming strategies.
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Dtsch. Med. Wochenschr. · Aug 2021
[Organ donation: A treatment option in end-of life intensive care?]
A newly issued policy statement of the German Medical Association considers organ donation as an integral part of end-of-life intensive care in patients with devastating brain afflictions. Hence, patients' wishes towards organ donation and medical suitability should be evaluated when prognosis is considered futile and goals of treatment need to be reconsidered. ⋯ Thus, a potential recovery of transplantable organs is supported by appropriate intensive care treatment. Decisions to employ extended intensive care options (like extracorporeal circulatory support or cardiopulmonary resuscitation) in potential organ donors should be carefully outbalanced with patients' wishes, organ donation being considered an achievable goal and even potential frictions in medical teams.
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Interventional radiology plays a crucial role in oncology. The most common interventional treatments are transarterial embolisation as well as percutaneous thermal ablations. Transarterial embolisation, such as transarterial chemoembolization (TACE) or selective internal radiation therapy (SIRT) are well established, usually palliatively intended treatment options for primary and secondary hepatic malignancies. ⋯ New interventional treatments are high intensity focused ultrasound (HIFU) and irreversible electroporation (IRE). HIFU allows a non-invasive, imaging-guided thermoablation that is currently certified for uterine myoma, prostate cancer and bone tumors. IRE is a minimal invasive non-thermal ablation that is especially established for locally advanced tumors that show a close relationship to large vessels, for example pancreatic cancer.