An individually, modified approach to desensitize infants and young children with Pompe disease, and significant reactions to alglucosidase alfa infusions

Molecular Genetics and Metabolism

Volumn 104, Issue 1-2, 2011, Pages 118-122

  El Gharbawy, Areeg H ^a   Mackey, Joanne ^a   DeArmey, Stephanie ^a   Westby, Greg ^a   Grinnell, Sherry G ^b   Malovrh, Peggy ^b   Conway, Robert ^c   Kishnani, Priya S ^a  

^a DUKE UNIVERSITY   (United States)

^b Michigan State University and Sparrow Health System   (United States)

^c MICHIGAN STATE UNIVERSITY   (United States)

Author keywords

Alglucosidase alfa; Desensitization; Hypersensitivity reactions; Infantile Pompe disease

Indexed keywords

ADRENALIN; DEXAMETHASONE; DIPHENHYDRAMINE; IMMUNOGLOBULIN E; IMMUNOGLOBULIN G; PARACETAMOL; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; TRYPTASE;

ACUTE RESPIRATORY FAILURE; AGITATION; ANTIBODY TITER; ANXIETY DISORDER; ARTICLE; ASSISTED VENTILATION; BRADYCARDIA; CASE REPORT; CHILD; DESENSITIZATION; DRUG DOSE INCREASE; DRUG DOSE REDUCTION; DRUG HYPERSENSITIVITY; DRUG INFUSION; DRUG TOLERANCE; DRUG WITHDRAWAL; ENZYME BLOOD LEVEL; ENZYME REPLACEMENT; FLUSHING; GENE MUTATION; GLYCOGEN STORAGE DISEASE TYPE 2; HEART LEFT VENTRICLE HYPERTROPHY; HOMOZYGOSITY; HUMAN; HYPOTENSION; HYPOXIA; IRRITABILITY; LETHARGY; LOW DRUG DOSE; MALE; MOTOR DYSFUNCTION; MUSCLE BIOPSY; MUSCLE WEAKNESS; PREMEDICATION; PRESCHOOL CHILD; PRIORITY JOURNAL; RASH; RECOMMENDED DRUG DOSE; RESPIRATORY DISTRESS; SECRETION; SIDE EFFECT; SKIN FIBROBLAST; TACHYCARDIA; TRACHEAL SECRETION; TRACHEOSTOMY; TREATMENT WITHDRAWAL; URTICARIA; WESTERN BLOTTING; WHEEZING;

ALPHA-GLUCOSIDASES; CHILD; DESENSITIZATION, IMMUNOLOGIC; ENZYME REPLACEMENT THERAPY; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; IMMUNOGLOBULIN E; INDIVIDUALIZED MEDICINE; INFANT; INFUSIONS, INTRAVENOUS; MALE; RECURRENCE;

EID: 80052523018     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2011.07.004     Document Type: Article

References (17)

1. Hirschhorn R., Reuser A.J.J. Glycogen storage disease type II: Acid alpha-glucosidase (acid maltase) deficiency. The Metabolic & Molecular Bases of Inherited Disease 2001, 3389-3420. McGraw-Hill, New York. (8th edition). D. Valle, A.L. Beaudet, B. Vogelstein, K.W. Kinzler, S.E. Antonarakis, A. Ballabio (Eds.).
2. Myozyme Prescribing Information. Genzyme Corporation Available at:. http://www.Myozyme.com.
3. Kishnani P.S., Corzo D., Nicolino M., Byrne B., Mandel H., Hwu W.L., Leslie N., Levine J., Spencer C., McDonald M., Li J., Dumontier J., Halberthal M., Chien Y.H., Hopkin R., Vijayaraghavan S., Gruskin D., Bartholomew D., van der Ploeg A., Clancy J.P., Parini R., Morin G., Beck M., De la Gastine G.S., Jokic M., Thurberg B., Richards S., Bali D., Davison M., Worden M.A., Chen Y.T., Wraith J.E. Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease. Neurology 2007, 68:99-109.
4. Kishnani P.S., Hwu W.L., Mandel H., Nicolino M., Yong F., Corzo D. A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease. J. Pediatr. 2006, 148:671-676.
5. Kishnani P.S., Corzo D., Leslie N.D., Gruskin D., van der Ploeg A., Clancy J.P., Parini R., Morin G., Beck M., Bauer M.S., Jokic M., Tsai C.E., Tsai B.W., Morgan C., O'Meara T., Richards S., Tsao E.C., Mandel H. Early treatment with alglucosidase alfa prolongs long term survival of infants with Pompe disease. Pediatr. Res. 2009, 66:329-355.
6. Starzyk K., Richards S., Yee J., Smith S.E., Kingma W. The long-term international safety experience of imiglucerase therapy for Gaucher disease. Mol. Genet. Metab. 2007, 90:157-163.
7. Wilcox W.R., Banikazemi M., Guffon N., Waldek S., Lee P., Linthorst G.E., Desnick R.J., Germain D.P. Long-term safety and efficacy of enzyme replacement therapy for Fabry disease. Am. J. Hum. Genet. 2004, 75:65-74.
8. Wraith J.E., Clarke L.A., Beck M., Kolodny E.H., Pastores G.M., Muenzer J., Rapoport D.M., Berger K.I., Swiedler S.J., Kakkis E.D., Braakman T., Chadbourne E., Walton-Bowen K., Cox G.F. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-l-iduronidase (laronidase). J. Pediatr. 2004, 144:581-588.
9. Lenz H.J. Management and preparedness for infusion and hypersensitivity reactions. Oncologist 2007, 12:601-609.
10. Wilcock L.K., Francis J.N., Durham S.R. IgE-facilitated antigen presentation: role in allergy and the influence of allergen immunotherapy. Immunol. Allergy Clin. North Am. 2006, 26:333-347. viii-ix.
11. Burton B.K., Whiteman D.A.H. Incidence and timing of infusion-related reactions in patients with mucopolysaccharidosis type II (Hunter syndrome) on idursulfase. Mol. Genet. Metab. 2011, 103(2):113-120.
12. Bodensteiner D., Scott C.R., Sims K.B., Shepherd G.M., Cintron R.D., Germain D.P. Successful reinstitution of agalsidase beta therapy in Fabry disease patients with previous IgE-antibody or skin-test reactivity to the recombinant enzyme. Genet. Med. 2008, 10:353-358.
13. Kim K.H., Decker C., Burton B.K. Successful management of difficult infusion-associated reactions in a young patient with mucopolysaccharidosis type VI receiving recombinant human arylsulfatase B. Pediatrics 2008, 121:e714-e717.
14. Peroni D.G., Pescollderungg L., Piacentini G.L., Cassar W., Boner A.L. Effective desensitization to imiglucerase in a patient with type I Gaucher disease. J. Pediatr. 2009, 155:940-941.
15. Lipinski S.E., Lipinski M.J., Burnette A., Platts-Mills T.A., Wilson W.G. Desensitization of an adult patient with Pompe disease and a history of anaphylaxis to Alglucosidase alpha. Mol. Genet. Metab. 2009, 98:319-321.
16. Cernadas J.R., Brockow K., Romano A., Aberer W., Torres M.J., Bircher A., Campi P., Sanz M.L., Castells M., Demoly P., Pichler W.J. General considerations on rapid desensitization for drug hypersensitivity-a consensus statement. Allergy 2010, 65:1357-1366. For the European Network of Drug Allergy and the EAACI interest group on drug hypersensitivity.
17. Castells M., Tennant NM N.M., Sloane D.E., Hsu F.I., Barrett N.A., Hong D.I., et al. Hypersensitivity reactions to chemotherapy: outcomes and safety of rapid desensitization in 413 cases. J. Allergy Clin. Immunol. 2008, 122:574-580.