Long-term enzyme replacement therapy for pompe disease with recombinant human alpha-glucosidase derived from Chinese hamster ovary cells

Journal of Child Neurology

Volumn 22, Issue 5, 2007, Pages 565-573

  Rossi, Massimiliano ^a   Parenti, Giancarlo ^a   Della Casa, Roberto ^a   Romano, Alfonso ^a   Mansi, Giuseppina ^a   Agovino, Teresa ^a   Rosapepe, Felice ^a   Vosa, Carlo ^a   Del Giudice, Ennio ^a   Andria, Generoso ^a  

^a UNIVERSITY OF NAPLES FEDERICO II   (Italy)

Author keywords

Enzyme replacement therapy; Glycogenosis type II; Pompe disease

Indexed keywords

OXYGEN; PARACETAMOL; PROMETHAZINE; RECOMBINANT ALPHA GLUCOSIDASE; RECOMBINANT ENZYME; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; UNCLASSIFIED DRUG;

ABDOMINAL PAIN; ADULT; ARTICLE; BLOOD PRESSURE; BRONCHUS SECRETION; CARDIOMYOPATHY; CASE REPORT; CHILD; CHO CELL; CLINICAL FEATURE; CONTROLLED STUDY; DRUG DOSE INCREASE; DRUG FEVER; DRUG RESPONSE; DRUG SAFETY; ENZYME REPLACEMENT; FATIGUE; FEMALE; GLYCOGEN STORAGE DISEASE TYPE 2; HEART RATE; HUMAN; HYPOXIA; IMMUNOGLOBULIN BLOOD LEVEL; IRRITABILITY; LONG TERM CARE; MALE; MOTOR PERFORMANCE; OTITIS; PRIORITY JOURNAL; PROGNOSIS; PROTEINURIA; RESPIRATORY FUNCTION; SIDE EFFECT; UPPER RESPIRATORY TRACT INFECTION;

ALPHA-GLUCOSIDASES; ANIMALS; ANIMALS, GENETICALLY MODIFIED; CHILD, PRESCHOOL; CHO CELLS; CRICETINAE; CRICETULUS; DISABILITY EVALUATION; DRUG ADMINISTRATION SCHEDULE; DRUG EVALUATION; FEMALE; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; INFANT; LONGITUDINAL STUDIES; MALE; MOTOR ACTIVITY; RECOMBINANT PROTEINS; TREATMENT OUTCOME;

EID: 34250744225     PISSN: 08830738     EISSN: None     Source Type: Journal    
DOI: 10.1177/0883073807302598     Document Type: Article

References (20)

1. Hirschhorn R., Reuser Ajj. Glycogen storage disease type II: Acid a-glucosidase (acid maltase) deficiency. In: Scriver CR, Sly WS, Childs, B, et al, eds. The Metabolic and Molecular Bases of Inherited Disease. New York, NY: McGraw-Hill; 2001: 3389-3420.
2. Kishnani PS, Howell RR Pompe disease in infants and children. J Pediatr. 2004; 144 (5 suppl): S35-S43.
3. Van den Hout HM, Hop W., van Diggelen OP, et al. The natural course of infantile Pompe's disease: 20 original cases compared with 133 cases from the literature. Pediatrics. 2003; 112: 332-340.
4. Hagemans ML, Winkel LP, Van Doorn PA, et al. Clinical manifestation and natural course of late-onset Pompe's disease in 54 Dutch patients. Brain. 2005; 128: 671-677.
5. Winkel LP, Hagemans ML, van Doorn PA, et al. The natural course of non-classic Pompe's disease; a review of 225 published cases. J Neurol. 2005; 252: 875-884.
6. Slonim AE, Bulone L., Ritz S., Goldberg T., Chen A., Martiniuk F. Identification of two subtypes of infantile acid maltase deficiency. J Pediatr. 2000; 137: 283-285.
7. Van den Hout H., Reuser AJ, Vulto AG, Loonen MC, Cromme-Dijkhuis A., Van der Ploeg AT Recombinant human alpha-glucosidase from rabbit milk in Pompe patients. Lancet. 2000; 56: 397-398.
8. Van den Hout JM, Reuser AJ, de Klerk JB, Arts WF, Smeitink JA, Van der Ploeg AT Enzyme therapy for Pompe disease with recombinant human alpha-glucosidase from rabbit milk. J Inherit Metab Dis. 2001; 24: 266-274.
9. Amalfitano A., Bengur AR, Morse RP, et al. Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: Results of a phase I/II clinical trial. Genet Med. 2001; 3: 132-138.
10. Van den Hout JM, Kamphoven JH, Winkel LP, et al. Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk. Pediatrics. 2004; 113: E448-e457.
11. Winkel LP, Van den Hout JM, Kamphoven JH, et al. Enzyme replacement therapy in late onset Pompe's disease: A 3-year follow-up. Ann Neur. 2004; 55: 495-502.
12. Klinge L., Straub V., Neudorf U., Voit T. Enzyme replacement therapy in classical infantile Pompe disease: Results of a 10-month follow-up study. Neuropediatrics. 2005; 36: 6-11.
13. Klinge L., Straub V., Neudorf U., et al. Safety and efficacy of recombinant acid alpha-glucosidase (rhGAA) in patients with classical infantile Pompe disease: Results of a phase II clinical trial. Neuromuscul Disord. 2005; 15: 24-31.
14. Brooke MH, Griggs RC, Mendell JR, Fenichel GM, Shumate JB, Pellegrino RJ et al. Clinical trial in Duchenne dystrophy. I. The design of the protocol. Muscle Nerve 1981; 4: 186-197.
15. Haley SM, Fragala MA, Aseltine R., Ni P., Skrinar AM Development of a disease-specific disability instrument for Pompe disease. Pediatr Rehabil. 2003; 6: 77-84.
16. Hagemans ML, Winkel LP, Hop WC, Reuser AJ, Van Doorn PA, Van der Ploeg AT Disease severity in children and adults with Pompe disease related to age and disease duration. Neurology. 2005; 64: 2139-2141.
17. Raben N., Danon M., Gilbert AL, et al. Enzyme replacement therapy in the mouse model of Pompe disease. Mol Genet Metab. 2003; 80: 159-169.
18. Reuser AJ, Van Den Hout H., Bijvoet AG, et al. Enzyme therapy for Pompe disease: From science to industrial enterprise. Eur J Pediatr. 2002; 161: S106-S111.
19. Sun B., Zhang H., Franco LM, et al. Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter. Mol Ther. 2005; 11: 889 898.
20. Parenti G., Zuppaldi A., Tuzzi MR, et al. Alpha-glucosidase enhancement in fibroblasts from patients with Pompe disease. J Inherit Metab Dis. 2005; 28 (suppl 1 383-P): 193.