- MeSH
- Cardiology * organization & administration MeSH
- Patient Care Planning MeSH
- Publication type
- Newspaper Article MeSH
- Interview MeSH
Kardiomyopatie představují širokou skupinu onemocnění, která vyžadují specifické diagnostické a léčebné postupy. Na rozdíl od častých onemocnění zahrnuje tato skupina některé vzácnější jednotky, jejichž diagnóza vyžaduje vysoký stupeň expertizy a dostupnost moderních zobrazovacích a laboratorních diagnostických metod. V současné době se mimoto začíná objevovat specifická léčba pro některá tato onemocnění (obstrukční hypertrofická kardiomyopatie, srdeční amyloidóza, Fabryho a Pompeho choroba a další). Tato léčba spadá většinou do kategorie vysoce nákladných léčiv, podléhá řadě indikačních omezení a vyžaduje nadstandardní a převážně vysoce intenzivní monitoraci nemocných. V součtu tak diagnostika a léčba kardiomyopatií vyžaduje centralizaci v expertních centrech s dostatečnou materiální i personální výbavou a jednání s plátci péče tak, aby tato centra měla přístup k vybraným typům léčby pro nemocné, o něž se stará. Tento dokument sestavený Pracovní skupinou pro choroby myokardu a perikardu České kardiologické společnosti představuje konsenzus odborníků specifikující , jak by měla být specializovaná pracoviště pro kardiomyopatie v současné době vybavena. Přepokládá se, že tato centra budou formálně ustavena a budou moci poskytovat celé spektrum péče včetně inovativních a regulovaných léčiv.
Cardiomyopathies encompass a diverse group of diseases requiring specialized diagnostic and therapeutic approaches. Unlike more common conditions, some rarer forms demand advanced expertise and access to modern imaging and diagnostic tools. Recently, targeted treatments for diseases like obstructive hypertrophic cardiomyopathy, cardiac amyloidosis, Fabry, and Pompe disease have emerged. These therapies are often high-cost, subject to strict indications, and necessitate intensive patient monitoring. Given the complexity, diagnosis and treatment should be centralized in expert centers with adequate resources and coor- dination with healthcare payers to ensure an access to these treatments. This document, prepared by the Working Group on Myocardial and Pericardial Diseases of the Czech Society of Cardiology, outlines expert consensus on the necessary infrastructure for specialized cardiomyopathy units, which are expected to offer comprehensive care, including innovative and regulated therapies.
- MeSH
- Cardiac Imaging Techniques classification MeSH
- Cardiology organization & administration MeSH
- Cardiomyopathies * diagnostic imaging drug therapy therapy MeSH
- Humans MeSH
- Multimodal Imaging methods MeSH
- Heart Diseases diagnostic imaging drug therapy therapy MeSH
- Check Tag
- Humans MeSH
- Publication type
- Practice Guideline MeSH
- MeSH
- Echocardiography * methods MeSH
- Cardiovascular Diseases diagnostic imaging MeSH
- Humans MeSH
- Check Tag
- Humans MeSH
- Publication type
- Review MeSH
- MeSH
- Cardiology * history organization & administration instrumentation trends MeSH
- Cardiovascular Diseases prevention & control MeSH
- Humans MeSH
- Personal Narratives as Topic MeSH
- Primary Prevention methods MeSH
- Education, Professional trends MeSH
- Patient Education as Topic MeSH
- Check Tag
- Humans MeSH
- Publication type
- Newspaper Article MeSH
- Interview MeSH
- MeSH
- Myocardial Infarction * prevention & control MeSH
- Secondary Prevention * methods MeSH
- Publication type
- Newspaper Article MeSH
- Interview MeSH
BACKGROUND: Pegunigalsidase alfa is a novel, PEGylated α-galactosidase-A enzyme-replacement therapy approved in the EU and US to treat patients with Fabry disease (FD). OBJECTIVE/METHODS: BRIDGE is a phase 3 open-label, switch-over study designed to assess safety and efficacy of 12 months of pegunigalsidase alfa (1 mg/kg every 2 weeks) treatment in adults with FD who had been previously treated with agalsidase alfa (0.2 mg/kg every 2 weeks) for ≥ 2 years. RESULTS: Twenty-seven patients were screened; 22 met eligibility criteria; and 20 (13 men, 7 women) completed the study. Pegunigalsidase alfa was well-tolerated, with 97% of treatment-emergent adverse events (TEAEs) being of mild or moderate severity. The incidence of treatment-related TEAEs was low, with 2 (9%) discontinuations due to TEAEs. Five patients (23%) reported infusion-related reactions. Overall mean (SD; n = 22) baseline estimated glomerular filtration rate (eGFR) was 82.5 (23.4) mL/min/1.73 m2 and plasma lyso-Gb3 level was 38.3 (41.2) nmol/L (men: 49.7 [45.8] nmol/L; women: 13.8 [6.1] nmol/L). Before switching to pegunigalsidase alfa, mean (standard error [SE]) annualized eGFR slope was - 5.90 (1.34) mL/min/1.73 m2/year; 12 months post-switch, the mean eGFR slope was - 1.19 (1.77) mL/min/1.73 m2/year; and mean plasma lyso-Gb3 reduced by 31%. Seven (35%) out of 20 patients were positive for pegunigalsidase alfa antidrug antibodies (ADAs) at ≥ 1 study timepoint, two of whom had pre-existing ADAs at baseline. Mean (SE) changes in eGFR slope for ADA-positive and ADA-negative patients were + 5.47 (3.03) and + 4.29 (3.15) mL/min/1.73 m2/year, respectively, suggesting no negative impact of anti-pegunigalsidase alfa ADAs on eGFR slope. CONCLUSION: Pegunigalsidase alfa may offer a safe and effective treatment option for patients with FD, including those previously treated with agalsidase alfa. TRN: NCT03018730. Date of registration: January 2017.
- MeSH
- alpha-Galactosidase therapeutic use MeSH
- Adult MeSH
- Enzyme Replacement Therapy methods MeSH
- Fabry Disease * drug therapy MeSH
- Isoenzymes adverse effects MeSH
- Humans MeSH
- Antibodies therapeutic use MeSH
- Recombinant Proteins therapeutic use MeSH
- Treatment Outcome MeSH
- Check Tag
- Adult MeSH
- Humans MeSH
- Male MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
