Clinical observation on human alpha glucosidase in treatment of five patients with glycogen storage disease Ⅱ

Objective: To evaluate the effect of enzyme replacement therapy (ERT) on glycogen storage disease typeⅡ(GSDⅡ). Method: The clinical data of three juvenile onset and two infant onset GSDⅡpatients were collected from First Affiliated Hospital of Sun Yat-sen University in October 2015 to July 2016.Pati...

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Veröffentlicht in:Zhonghua er ke za zhi = Chinese journal of pediatrics. - 1960. - 54(2016), 11 vom: 02. Nov., Seite 829-833
1. Verfasser: Xu, L L (VerfasserIn)
Weitere Verfasser: Zhang, L D, Liang, Y J, Tang, W, Huang, X Q, Pei, Y X, Cheng, Y C, Huang, H M, Zhang, C
Format: Online-Aufsatz
Sprache:Chinese
Veröffentlicht: 2016
Zugriff auf das übergeordnete Werk:Zhonghua er ke za zhi = Chinese journal of pediatrics
Schlagworte:Case Reports Journal Article alpha-Glucosidases EC 3.2.1.20
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520 |a Objective: To evaluate the effect of enzyme replacement therapy (ERT) on glycogen storage disease typeⅡ(GSDⅡ). Method: The clinical data of three juvenile onset and two infant onset GSDⅡpatients were collected from First Affiliated Hospital of Sun Yat-sen University in October 2015 to July 2016.Patient 1 was female, the age of onset was 15 months. Patient 2 was male, the age of onset was 20 months. Patient 3 was female, the sister of patient 2, the age of onset was 47 months. Patient 4 was male, the age of onset was 5 months. Patient 5 was male, the age of onset was 1 month.The age at the start of ERT of the 5 patients was 32, 31, 56, 34, and 3 months respectively and the duration of ERT was 19, 9, 4, 5, 5 doses respectively.ERT was administered at 20 mg/kg every 2 weeks.Dexamethasone was regularly given before each infusion. Result: ERT was well tolerated, only one time, Patient 1 developed tachycardia and hypertension without using dexamethasone.Patient 2 underwent successfully ventilator weaning.Patient 1 underwent a tracheotomy, also needed mechanical ventilation treatment.Patient 4 noninvasive ventilation was tried. Conclusion: Recombinant human alpha-glucosidase treatment was effective and well tolerated in patients with GSDⅡ 
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700 1 |a Liang, Y J  |e verfasserin  |4 aut 
700 1 |a Tang, W  |e verfasserin  |4 aut 
700 1 |a Huang, X Q  |e verfasserin  |4 aut 
700 1 |a Pei, Y X  |e verfasserin  |4 aut 
700 1 |a Cheng, Y C  |e verfasserin  |4 aut 
700 1 |a Huang, H M  |e verfasserin  |4 aut 
700 1 |a Zhang, C  |e verfasserin  |4 aut 
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