The relationship between anti-idursulfase antibody status and safety and efficacy outcomes in attenuated mucopolysaccharidosis II patients aged 5years and older treated with intravenous idursulfase

Molecular Genetics and Metabolism

Volumn 110, Issue 3, 2013, Pages 303-310

  Barbier, A J ^a   Bielefeld, B ^a   Whiteman, D A H ^a   Natarajan, M ^a   Pano, A ^a   Amato, D A ^a  

^a SHIRE   (United States)

Author keywords

Antibodies; Idursulfase; Mucopolysaccharidosis II; Treatment outcome

Indexed keywords

CREATININE; GLYCOSAMINOGLYCAN; IDURONATE 2 SULFATASE; IMMUNOGLOBULIN G ANTIBODY; NEUTRALIZING ANTIBODY; PLACEBO;

ADOLESCENT; ADULT; ANTIBODY RESPONSE; ARTICLE; CHILD; CONTROLLED STUDY; CREATININE URINE LEVEL; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG SAFETY; FOLLOW UP; FORCED VITAL CAPACITY; FRAMESHIFT MUTATION; GENOTYPE; HUMAN; HUNTER SYNDROME; IMMUNOGENICITY; INFUSION RELATED REACTION; LIVER WEIGHT; MAJOR CLINICAL STUDY; MISSENSE MUTATION; MUCOPOLYSACCHARIDOSIS; MULTICENTER STUDY; PHASE 2 CLINICAL TRIAL; PHASE 3 CLINICAL TRIAL; PRESCHOOL CHILD; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; RECOMMENDED DRUG DOSE; RISK FACTOR; SCHOOL CHILD; SPLEEN WEIGHT; TREATMENT DURATION; TREATMENT OUTCOME;

%FVC; 6MWT; AB+; AB-; ADVERSE EVENTS; AES; ANTIBODIES; ANTIBODY NEGATIVE; ANTIBODY POSITIVE; CONFORMATION-SPECIFIC ANTIBODY ASSAY; CSA; ELISA; ENZYME-LINKED IMMUNOSORBENT ASSAY; FORCED VITAL CAPACITY; FVC; GAG; GLYCOSAMINOGLYCAN; IDURSULFASE; INFUSION-RELATED ADVERSE EVENTS; IRAE; MPS; MUCOPOLYSACCHARIDOSIS; MUCOPOLYSACCHARIDOSIS II; NAB+; NAB-; NEUTRALIZING ANTIBODY NEGATIVE; NEUTRALIZING ANTIBODY POSITIVE; PAB+; PERCENT PREDICTED FORCED VITAL CAPACITY; PERSISTENTLY ANTIBODY POSITIVE; PERSISTENTLY NEUTRALIZING ANTIBODY POSITIVE; PNAB+; RADIOIMMUNOPRECIPITATION; RIP; SAE; SERIOUS ADVERSE EVENT; SIX-MINUTE WALK TEST; TEAE; TREATMENT EMERGENT ADVERSE EVENT; TREATMENT OUTCOME; UGAG; URINARY GLYCOSAMINOGLYCAN;

ADMINISTRATION, INTRAVENOUS; ADOLESCENT; ADULT; ANTIBODIES; CHILD; CHILD, PRESCHOOL; ENZYME REPLACEMENT THERAPY; GENOTYPE; GLYCOPROTEINS; GLYCOSAMINOGLYCANS; HUMANS; IDURONATE SULFATASE; LIVER; MUCOPOLYSACCHARIDOSIS II; ORGAN SIZE; SPLEEN; TREATMENT OUTCOME; YOUNG ADULT;

EID: 84885426525     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2013.08.002     Document Type: Article

References (16)

1. Neufeld E.F., Muenzer J. The mucopolysaccharidoses. The Metabolic and Molecular Bases of Inherited Disease 2001, vol. III:3421-3452. McGraw-Hill, New York. C.R. Scriver, A.L. Beaudet, W.S. Sly, D. Valle (Eds.).
2. Bach G., Eisenberg F., Cantz M., Neufeld E.F. The defect in the Hunter syndrome: deficiency of sulfoiduronate sulfatase. Proc. Natl. Acad. Sci. U. S. A. 1973, 70:2134-2138.
3. Wraith J.E., Beck M., Giugliani R., Clarke J., Martin R., Muenzer J. Initial report from the Hunter Outcome Survey. Genet. Med. 2008, 10:508-516.
4. Jones S.A., Almassy Z., Beck M., Burt K., Clarke J.T., Giugliani R., Hendriksz C., Kroepfl T., Lavery L., Lin S.P., Malm G., Ramaswami U., Tincheva R., Wraith J.E. Mortality and cause of death in mucopolysaccharidosis type II-a historical review based on data from the Hunter Outcome Survey (HOS). J. Inherit. Metab. Dis. 2009, 32:534-543.
5. Martin R., Beck M., Eng C., Giugliani R., Harmatz P., Munoz V., Muenzer J. Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome). Pediatrics 2008, 121:e377-e386.
6. Young I.D., Harper P.S. The natural history of the severe form of Hunter's syndrome: a study based on 52 cases. Dev. Med.Child Neurol. 1983, 25:481-489.
7. Muenzer J., Wraith J.E., Beck M., Giugliani R., Harmatz P., Eng C.M., Vellodi A., Martin R., Ramaswami U., Gucsavas-Calikoglu M., Vijayaraghavan S., Wendt S., Puga A.C., Ulbrich B., Shinawi M., Cleary M., Piper D., Conway A.M., Kimura A. A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet. Med. 2006, 8:465-473.
8. Muenzer J., Beck M., Eng C.M., Giugliani R., Harmatz P., Martin R., Ramaswami U., Vellodi A., Wraith J.E., Cleary M., Gucsavas-Calikoglu M., Puga A.C., Shinawi M., Ulbrich B., Vijayaraghavan S., Wendt S., Conway A.M., Rossi A., Whiteman D.A., Kimura A. Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome. Genet. Med. 2011, 13:95-101.
9. Hunt S.V. Perspectives from B cell immunology: fact and fancy. Int. J. Clin. Pharmacol. Ther. 2009, 47(Suppl. 1):S86-S99.
10. Sellos-Moura M., Barzegar S., Pan L., Shi P., Oommen S., Durant J., Ruiz J.A. Development of a panel of highly sensitive, equivalent assays for detection of antibody responses to velaglucerase alfa or imiglucerase enzyme replacement therapy in patients with Gaucher disease. J. Immunol. Methods 2011, 373:45-53.
11. Gupta S., Indelicato S.R., Jethwa V., Kawabata T., Kelley M., Mire-Sluis A.R., Richards S.M., Rup B., Shores E., Swanson S.J., Wakshull E. Recommendations for the design, optimization, and qualification of cell-based assays used for the detection of neutralizing antibody responses elicited to biological therapeutics. J. Immunol. Methods 2007, 321:1-18.
12. Froissart R., Moreira da Silva I., Guffon N., Bozon D., Maire I. Mucopolysaccharidosis type II-genotype/phenotype aspects. Acta Paediatr. Suppl. 2002, 91:82-87.
13. ATS statement: guidelines for the six-minute walk test. Am. J. Respir. Crit. Care Med. 2002, 166:111-117.
14. Standardization of Spirometry, 1994 Update. American Thoracic Society. Am. J. Respir. Crit. Care Med. 1994, 152(1995):1107-1136.
15. Anderson J.R., Cain K.C., Gelber R.D. Analysis of survival by tumor response. J. Clin. Oncol. 1983, 1:710-719.
16. Burton B.K., Whiteman D.A. Incidence and timing of infusion-related reactions in patients with mucopolysaccharidosis type II (Hunter syndrome) on idursulfase therapy in the real-world setting: a perspective from the Hunter Outcome Survey (HOS). Mol. Genet. Metab. 2011, 103:113-120.