Enzyme Replacement Therapy Decreases Left Ventricular Mass Index in Patients with Hunter Syndrome?

Valle, Daniel Almeida do; Cirino, Raphael Henrique Déa; Santos, Mara Lúcia Schmitz Ferreira; Pellissari, Eliana Costa; Scola, Rosana Hermínia

doi:10.1007/s00246-019-02267-0

Cited by 3 publications

(2 citation statements)

References 22 publications

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“…Patients with HF have reduced CO, inadequate perfusion of the renal arteries, reduced glomerular filtration, and increased venous pressure [10]. Patients will have symptoms of water and sodium retention with elevated urea nitrogen and blood creatinine levels, which severely impair their liver and kidney functions [11]. The sympathetic nervous system and inflammatory responses affect the cardiac and renal functions.…”

Section: Discussionmentioning

confidence: 99%

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Prognostic risk factors in patients with refractory heart failure treated with continuous veno‐venous hemofiltration

Mao

Zhong

2022

Ther Apher Dial

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Introduction Investigation of the prognostic factors in patients with refractory heart failure (HF) undergoing continuous veno‐venous hemofiltration (CVVH). Methods Clinical data of 146 patients with refractory HF between May 2018 and December 2020 were retrospectively analyzed and divided into survival and death groups according to the prognosis. Vital signs, inflammatory markers, and renal function parameters were compared before and after treatment. Results Central venous pressure levels were lower, whereas serum levels of brain natriuretic peptide, oxygen saturation, and cardiac output were higher after treatment (p < 0.05). Heart rate, systolic and diastolic blood pressures, serum levels of C‐reactive protein, interleukin‐6, tumor necrosis factor‐α, interleukin‐8, blood urea nitrogen, creatinine, and 24‐h urinary protein were lower after treatment (p < 0.05). Conclusion CVVH improved renal function and regulated blood pressure and vital signs in patients with refractory HF. Age, APACHE II score, disease duration, and hypotension were risk factors affecting the prognosis.

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Section: Discussionmentioning

confidence: 99%

“…Patients will have symptoms of water and sodium retention with elevated urea nitrogen and blood creatinine levels, which severely impair their liver and kidney functions [11]. The sympathetic nervous system and inflammatory responses affect the cardiac and renal functions.…”

Section: Discussionmentioning

confidence: 99%

Prognostic risk factors in patients with refractory heart failure treated with continuous veno‐venous hemofiltration

Mao

Zhong

2022

Ther Apher Dial

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Metabolic Cardiomyopathies and Cardiac Defects in Inherited Disorders of Carbohydrate Metabolism: A Systematic Review

Conte,

Sam,

Lefeber

et al. 2023

IJMS

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Heart failure (HF) is a progressive chronic disease that remains a primary cause of death worldwide, affecting over 64 million patients. HF can be caused by cardiomyopathies and congenital cardiac defects with monogenic etiology. The number of genes and monogenic disorders linked to development of cardiac defects is constantly growing and includes inherited metabolic disorders (IMDs). Several IMDs affecting various metabolic pathways have been reported presenting cardiomyopathies and cardiac defects. Considering the pivotal role of sugar metabolism in cardiac tissue, including energy production, nucleic acid synthesis and glycosylation, it is not surprising that an increasing number of IMDs linked to carbohydrate metabolism are described with cardiac manifestations. In this systematic review, we offer a comprehensive overview of IMDs linked to carbohydrate metabolism presenting that present with cardiomyopathies, arrhythmogenic disorders and/or structural cardiac defects. We identified 58 IMDs presenting with cardiac complications: 3 defects of sugar/sugar-linked transporters (GLUT3, GLUT10, THTR1); 2 disorders of the pentose phosphate pathway (G6PDH, TALDO); 9 diseases of glycogen metabolism (GAA, GBE1, GDE, GYG1, GYS1, LAMP2, RBCK1, PRKAG2, G6PT1); 29 congenital disorders of glycosylation (ALG3, ALG6, ALG9, ALG12, ATP6V1A, ATP6V1E1, B3GALTL, B3GAT3, COG1, COG7, DOLK, DPM3, FKRP, FKTN, GMPPB, MPDU1, NPL, PGM1, PIGA, PIGL, PIGN, PIGO, PIGT, PIGV, PMM2, POMT1, POMT2, SRD5A3, XYLT2); 15 carbohydrate-linked lysosomal storage diseases (CTSA, GBA1, GLA, GLB1, HEXB, IDUA, IDS, SGSH, NAGLU, HGSNAT, GNS, GALNS, ARSB, GUSB, ARSK). With this systematic review we aim to raise awareness about the cardiac presentations in carbohydrate-linked IMDs and draw attention to carbohydrate-linked pathogenic mechanisms that may underlie cardiac complications.

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Intravenous Idursulfase for the Treatment of Mucopolysaccharidosis Type II: A Systematic Literature Review

Al-Hertani,

Pathak,

Evuarherhe

et al. 2024

IJMS

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Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is a rare, X-linked disorder caused by deficient activity of the enzyme iduronate-2-sulfatase. Signs and symptoms typically emerge at 1.5–4 years of age and may include cognitive impairment, depending on whether patients have the neuronopathic or non-neuronopathic form of the disease. Treatment is available in the form of enzyme replacement therapy (ERT) with recombinant iduronate-2-sulfatase (idursulfase). A systematic literature review was conducted to assess the evidence regarding efficacy, effectiveness, and safety of ERT with intravenous idursulfase for MPS II. Electronic databases were searched in January 2023, and 33 eligible articles were found. These were analyzed to evaluate the effects of intravenous idursulfase and the overall benefits and disadvantages in patient subgroups. Studies showed that intravenous idursulfase treatment resulted in improved short- and long-term clinical and patient-centered outcomes, accompanied by a favorable safety profile. Patients with non-neuronopathic MPS II had more pronounced improvements in clinical outcomes than those with neuronopathic MPS II. In addition, the review identified that improvements in clinical outcomes are particularly apparent if intravenous idursulfase is started early in life, strengthening previous recommendations for early ERT initiation to maximally benefit patients. This review provides a comprehensive summary of our current knowledge on the efficacy of ERT in different populations of patients with MPS II and will help to inform the overall management of the disease in an evolving treatment landscape.

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Enzyme Replacement Therapy Decreases Left Ventricular Mass Index in Patients with Hunter Syndrome?

Cited by 3 publications

References 22 publications

Prognostic risk factors in patients with refractory heart failure treated with continuous veno‐venous hemofiltration

Prognostic risk factors in patients with refractory heart failure treated with continuous veno‐venous hemofiltration

Metabolic Cardiomyopathies and Cardiac Defects in Inherited Disorders of Carbohydrate Metabolism: A Systematic Review

Intravenous Idursulfase for the Treatment of Mucopolysaccharidosis Type II: A Systematic Literature Review

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