Pegunigalsidase alfa, a PEGylated α-galactosidase A enzyme replacement therapy (ERT) for Fabry disease, has a longer plasma half-life than other ERTs administered intravenously every 2 weeks (E2W). BRIGHT (NCT03180840) was a phase III, open-label study in adults with Fabry disease, previously treated with agalsidase alfa or beta E2W for ≥3 years, who switched to 2 mg/kg pegunigalsidase alfa every 4 weeks (E4W) for 52 weeks. Primary objective assessed safety, including number of treatment-emergent adverse events (TEAEs). Thirty patients were enrolled (24 males); 23 previously received agalsidase beta. Pegunigalsidase alfa plasma concentrations remained above the lower limit of quantification throughout the 4-week dosing interval. Thirty-three of 182 TEAEs (in 9 patients) were considered treatment-related; all were mild/moderate. No patients developed de novo anti-drug antibodies (ADAs). In the efficacy analysis (n = 29), median (inter-quartile range) eGFR change from baseline over 52 weeks was -1.9 (-5.9; 1.8) mL/min/1.73 m2 (n = 28; males [n = 22]: -2.4 [-5.2; 3.2]; females [n = 6]: -0.7 [-9.2; 2.0]). Overall, median eGFR slope was -1.9 (-8.3; 1.9) mL/min/1.73 m2/year (ADA-negative [n = 20]: -1.2 [-6.4; 2.6]; ADA-positive [n = 9]: -8.4 [-11.6; -1.0]). Lyso-Gb3 concentrations were low and stable in females, with a slight increase in males (9/24 ADA-positive). The BRIGHT study results suggest that 2 mg/kg pegunigalsidase alfa E4W is tolerated well in stable adult patients with Fabry disease. Due to the low number of patients in this study, more research is needed to demonstrate the effects of pegunigalsidase alfa given E4W. Further evidence, outside of this clinical trial, should be factored in for physicians to prolong the biweekly ERT intervals to E4W. TAKE-HOME MESSAGE: Treatment with 2 mg/kg pegunigalsidase alfa every 4 weeks could offer a new treatment option for patients with Fabry disease.

• Klíčová slova
• Fabry disease, eGFR, enzyme replacement therapy, lysosomal storage disorders, lyso‐Gb3, pegunigalsidase alfa,
• MeSH
• alfa-galaktosidasa * aplikace a dávkování   terapeutické užití   MeSH
• dospělí MeSH
• enzymová substituční terapie * metody   MeSH
• Fabryho nemoc * farmakoterapie   MeSH
• lidé středního věku MeSH
• lidé MeSH
• mladý dospělý MeSH
• polyethylenglykoly aplikace a dávkování   MeSH
• rekombinantní proteiny * aplikace a dávkování   terapeutické užití   MeSH
• rozvrh dávkování léků MeSH
• senioři MeSH
• sfingolipidy krev   MeSH
• trihexosylceramidy krev   MeSH
• výsledek terapie MeSH
• Check Tag
• dospělí MeSH
• lidé středního věku MeSH
• lidé MeSH
• mladý dospělý MeSH
• mužské pohlaví MeSH
• senioři MeSH
• ženské pohlaví MeSH
• Publikační typ
• časopisecké články MeSH
• klinické zkoušky, fáze III MeSH
• multicentrická studie MeSH
• práce podpořená grantem MeSH
• Názvy látek
• agalsidase alfa MeSH Prohlížeč
• agalsidase beta MeSH Prohlížeč
• alfa-galaktosidasa * MeSH
• globotriaosyl lysosphingolipid MeSH Prohlížeč
• polyethylenglykoly MeSH
• rekombinantní proteiny * MeSH
• sfingolipidy MeSH
• trihexosylceramidy MeSH

• Publikační typ
• dopisy MeSH
• komentáře MeSH

The rapid pace of name changes of medically important fungi is creating challenges for clinical laboratories and clinicians involved in patient care. We describe two sources of name change which have different drivers, at the species versus the genus level. Some suggestions are made here to reduce the number of name changes. We urge taxonomists to provide diagnostic markers of taxonomic novelties. Given the instability of phylogenetic trees due to variable taxon sampling, we advocate to maintain genera at the largest possible size. Reporting of identified species in complexes or series should where possible comprise both the name of the overarching species and that of the molecular sibling, often cryptic species. Because the use of different names for the same species will be unavoidable for many years to come, an open access online database of the names of all medically important fungi, with proper nomenclatural designation and synonymy, is essential. We further recommend that while taxonomic discovery continues, the adaptation of new name changes by clinical laboratories and clinicians be reviewed routinely by a standing committee for validation and stability over time, with reference to an open access database, wherein reasons for changes are listed in a transparent way.

• Klíčová slova
• fungi, nomenclature, taxonomy,
• MeSH
• databáze faktografické MeSH
• fylogeneze MeSH
• houby * genetika   MeSH
• lidé MeSH
• Check Tag
• lidé MeSH
• Publikační typ
• časopisecké články MeSH
• práce podpořená grantem MeSH

BACKGROUND: The IMACS Registry compiles and analyzes worldwide data from patients undergoing implantation of durable left ventricular assist devices. METHODS: Data encompassing 16,286 LVAD recipients from 4 collectives and 24 individual hospitals was collected and analyzed. In this 3rd annual report we compare and contrast outcomes, adverse events and risks factors between axial flow and centrifugal flow device recipients. RESULTS: Significant differences were found in the baseline characteristics of axial vs centrifugal flow LVAD recipients. Survival was similar between pump types. INTERMACS profile 1-3 constitute 85% of implants. A survival gap persists in destination therapy compared to bridge patients. RVAD need and delay impact survival dramatically. Centrifugal flow outperforms axial flow recipients in regards to GI bleeding and freedom from hemocompatibility related adverse events. No significant difference in the actuarial freedom from all strokes or either stroke subtype (hemorrhagic or ischemic) was seen among the two types of pumps. New end points to guide decision making are proposed. CONCLUSIONS: We demonstrate a transition from axial to centrifugal flow with four-year survival that approximates 60%. A high frequency of adverse events remains an impediment to the wider adoption of these technologies. In the future, composite study endpoints examining life quality and adverse events beyond survival may help in shared decision making prior to MCS implant, and may provide the requisite data to support extension of MCS therapy into the lesser ill heart failure population.

• Klíčová slova
• IMACS, International Society for Heart and Lung Transplantation, axial flow, centrifugal flow, continuous-flow left ventricular assist devices, mechanical circulatory support,
• MeSH
• asistovaná cirkulace MeSH
• dospělí MeSH
• lidé středního věku MeSH
• lidé MeSH
• mezinárodní spolupráce MeSH
• podpůrné srdeční systémy * MeSH
• protézy - design * MeSH
• registrace MeSH
• senioři MeSH
• společnosti lékařské MeSH
• srdeční selhání chirurgie   MeSH
• transplantace srdce a plic MeSH
• výroční zprávy jako téma MeSH
• Check Tag
• dospělí MeSH
• lidé středního věku MeSH
• lidé MeSH
• mužské pohlaví MeSH
• senioři MeSH
• ženské pohlaví MeSH
• Publikační typ
• časopisecké články MeSH
• srovnávací studie MeSH