E

2020 ◽  
pp. 275-307
Author(s):  
Sean Ainsworth
Keyword(s):  
Adverse Effects ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Recombinant Human Erythropoietin ◽  
Eye Drops ◽  
Enzyme Replacement ◽  
Maternal Treatment ◽  
Human Erythropoietin

This chapter presents information on neonatal drugs that begin with E, including use, pharmacology, adverse effects, fetal and infant implications of maternal treatment, treatment, and supply of Enemas, laxatives, and suppositories, Enoxaparin, Enzyme replacement therapy, Epoetin (recombinant human erythropoietin = rEPO), Epoprostenol and other prostanoids (iloprost and teoprostinil), Erythromycin, Esomeprazole, Ethambutol, and Eye drops (and ointments)

scholarly journals Switch from enzyme replacement therapy to oral chaperone migalastat for treating fabry disease: real-life data

2020 ◽  
Vol 28 (12) ◽  
pp. 1662-1668 ◽  
Author(s):  
Eleonora Riccio ◽  
◽  
Mario Zanfardino ◽  
Lucia Ferreri ◽  
Ciro Santoro ◽  
...  
Keyword(s):  
Adverse Effects ◽  
Enzyme Replacement Therapy ◽  
Fabry Disease ◽  
Replacement Therapy ◽  
Treatment Options ◽  
Real Life ◽  
Left Ventricular ◽  
Pharmacological Chaperone ◽  
Enzyme Replacement ◽  
Neurologic Function

AbstractThe treatment options for Fabry disease (FD) are enzyme replacement therapy (ERT) with agalsidase alfa or beta, and the oral pharmacological chaperone migalastat. Since few data are available on the effects of switching from ERT to migalastat, we performed a single-center observational study on seven male Fabry patients (18–66 years) to assess the effects of the switch on renal, cardiac, and neurologic function, health status, pain, lyso-Gb3, α-Gal A activity and adverse effects. Data were retrospectively collected at time of diagnosis of FD (baseline, T0), and after 12 months of ERT (T1), and prospectively after 1 year of therapy with migalastat (T2). No patient died or reported renal, cardiac, or cerebrovascular events during the study period. The predefined measures for cardiac, renal and neurologic function, and FD-related symptoms and questionnaires were stable between baseline and the switch, and remained unchanged with migalastat. However, a significant improvement was observed in left ventricular mass index from baseline to T2 (p = 0.016), with a significative difference between the treatments (p = 0.028), and in median proteinuria from T2 vs T1 (p = 0.048). Moreover, scores of the BPI improved from baseline to T1, and remained stable with migalastat. Plasma lyso-Gb3 levels significantly decreased from baseline to T1 (P = 0.007) and T2 (P = 0.003), while did not significantly differ between the two treatments. α-Gal A activity increased from T0 to T2 (p < 0.0001). The frequency of adverse effects under migalastat and ERT was comparable (28% for both drugs). In conclusion, switching from ERT to migalastat is valid, safe and well tolerated.

Cardiac and motor improvement with enzyme replacement therapy in a patient with infantile-onset Pompe disease

2005 ◽  
Vol 36 (02) ◽  
Author(s):  
M Smitka ◽  
M von der Hagen ◽  
A Kaindl ◽  
C Gilitzer ◽  
J Dumontier ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Enzyme Replacement ◽  
Motor Improvement

RESPONSE TO ENZYME REPLACEMENT THERAPY IN PATIENTS WITH GAUCHER DISEASE TYPE I

2019 ◽  
Vol 22 (06) ◽  
pp. 103-117
Author(s):  
Mays Al-Tai ◽  
Deia Al-Asady ◽  
Rula Hamid
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Gaucher Disease ◽  
Disease Type ◽  
Type I ◽  
Enzyme Replacement

Correlation Between Recombinant Human Erythropoietin Dose and Inflammatory Status in Dialysed Patients

2017 ◽  
Vol 68 (2) ◽  
pp. 354-357 ◽  
Author(s):  
Andrei Niculae ◽  
Cristiana David ◽  
Razvan Florin Ion Dragomirescu ◽  
Ileana Peride ◽  
Flavia Liliana Turcu ◽  
...  
Keyword(s):  
Adverse Effects ◽  
Recombinant Human Erythropoietin ◽  
Best Practice ◽  
Daily Practice ◽  
Therapeutic Benefit ◽  
Chronic Dialysis ◽  
Dialysis Patients ◽  
Protein Levels ◽  
Human Erythropoietin ◽  
Inflammatory Status

Once recombinant human erythropoietin (r-HuEPO) was introduced in daily practice, huge steps were made in combating the adverse effects induced by anemia in chronic kidney disease population. Still, r-HuEPO resistance and the doses ensuring the maximum therapeutic benefit remain matters of debate. The aim of our study was to assess the correlation between the presence and the degree of inflammation and the r-HuEPO requirements in chronic dialysis patients. We conducted a 2 years prospective study on 146 patients undergoing chronic dialysis treated with r-HuEPO. Based on their average CRP (C-reactive protein) levels, obtained from repeated samplings at 3 months interval, 3 groups were formed; we noted in each group the average values of r-HuEPO prescribed to achieve the optimum hemoglobin levels according to the dialysis best practice guidelines and all the adverse effects of the therapy. A direct correlation was observed between CRP levels and r-HuEPO requirements in the first 2 groups of patients (CRP under 6 mg/L and CRP values 6-20 mg/L), with significant increase in r-HuEPO doses between groups (p [ 0.001); the third group, CRP values over 20 mg/dL, showed a minor, insignificant increase in average r-HuEPO doses compared to mild inflammation group (p = 0.199) and more adverse effects of the therapy (p [ 0.05). Inflammation is an important determinant of anemia in chronic dialysis patients and can induce an increase in the doses of r-HuEPO. However, prescribing excessive r-HuEPO doses is not the answer in severe inflammatory status, due to lack of response and possible adverse effects.

Two siblings with attenuated MPS II form: Long term enzyme replacement therapy

2021 ◽  
Vol 132 (2) ◽  
pp. S107-S108
Author(s):  
Nato Vashakmadze ◽  
Leyla Namazova-Baranova ◽  
Natalia Zhurkova ◽  
Olga Gordeeva ◽  
Nina Fedorova ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Enzyme Replacement ◽  
Mps Ii

scholarly journals Development of the “Hamburg Best Practice Guidelines for ICV−Enzyme Replacement therapy (ERT) in CLN2 Disease” Based on 6 Years Treatment Experience in 48 Patients

2021 ◽  
pp. 088307382198915
Author(s):  
Christoph Schwering ◽  
Gertrud Kammler ◽  
Eva Wibbeler ◽  
Martin Christner ◽  
Johannes K.-M. Knobloch ◽  
...  
Keyword(s):  
Patient Safety ◽  
Adverse Events ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Practice Guidelines ◽  
Best Practice ◽  
Vision Loss ◽  
Neuronal Ceroid Lipofuscinosis ◽  
Enzyme Replacement ◽  
Best Practice Guidelines

Intracerebroventricular enzyme replacement therapy (ICV-ERT) for CLN2 disease represents the first approved treatment for neuronal ceroid lipofuscinosis (NCL) diseases. It is the first treatment where a recombinant lysosomal enzyme, cerliponase alfa, is administered into the lateral cerebral ventricles to reach the central nervous system, the organ affected in CLN2 disease. If untreated, CLN2 children show first symptoms such as epilepsy and language developmental delay at 2-4 years followed by rapid loss of motor and language function, vision loss, and early death. Treatment with cerliponase alfa has shown to slow the rapid neurologic decline. However, the mode of administration by 4 hour-long intracerebroventricular infusions every 14 days represents a potentially greater risk of infection compared to intravenous enzyme replacement therapies. The Hamburg NCL Specialty Clinic was the first site worldwide to perform intracerebroventricular enzyme replacement therapy in children with CLN2 disease. In order to ensure maximum patient safety, we analysed data from our center from more than 3000 intracerebroventricular enzyme replacement therapies in 48 patients over 6 years with regard to the occurrence of device-related adverse events and device infections. Since starting intracerebroventricular enzyme replacement therapy, we have also developed and continuously improved the “Hamburg Best Practice Guidelines for ICV–Enzyme Replacement Therapy (ERT) in CLN2 Disease.” Results from this study showed low rates for device-related adverse events and infections with 0.27% and 0.33%, respectively. Therefore, following our internal procedural guidelines has shown to improve standardization and patient safety of intracerebroventricular enzyme replacement therapy for CLN2 disease.

Description of a patient with infantile onset Pompe disease after 45 months of enzyme replacement therapy

2018 ◽  
Vol 123 (2) ◽  
pp. S124-S125
Author(s):  
Roberto Sandobal Pacheco ◽  
Diana Espinosa Villanueva ◽  
Adriana Alcnatara Salinas ◽  
Jorge A. Romero Ramirez ◽  
Jose Antonio Vasquez Galeana ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Enzyme Replacement
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