Desensitization of an adult patient with Pompe disease and a history of anaphylaxis to alglucosidase alfa

被引：23
|
作者：
Lipinski, Shawn E. [1 ]
Lipinski, Michael J. [2 ]
Burnette, Autumn [3 ]
Platts-Mills, Thomas A. [3 ]
Wilson, William G. [1 ]
机构：
[1] Univ Virginia Hlth Syst, Dept Pediat, Div Med Genet, Charlottesville, VA 22908 USA
[2] Univ Virginia Hlth Syst, Dept Internal Med, Charlottesville, VA 22908 USA
[3] Univ Virginia Hlth Syst, Asthma & Allerg Dis Ctr, Charlottesville, VA 22908 USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2009年 / 98卷 / 03期
关键词：
Pompe disease; Enzyme replacement therapy; Lysosomal storage disease; Anaphylaxis; Allergy;
D O I：
10.1016/j.ymgme.2009.07.001
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
We report on the successful desensitization of an adult female with Pompe disease who had previously experienced anaphylaxis to intravenous alglucosidase alfa therapy. The starting alglucosidase alfa dose for desensitization was 10 mg/kg with gradual dose escalation and desensitization via serial dilution was completed over five infusions. This methodology serves as a means to desensitize patients with prior anaphylactic response to alglucosidase alfa so that enzyme replacement therapy can be utilized. (C) 2009 Elsevier Inc. All rights reserved.
引用
收藏
页码：319 / 321
页数：3
相关论文
共 50 条
  • [11] Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease
    Kanters, Tim A.
    van der Ploeg, Ans T.
    Kruijshaar, Michelle E.
    Rizopoulos, Dimitris
    Redekop, W. Ken
    Rutten-van Molken, Maureen P. M. H.
    Hakkaart-van Roijen, Leona
    ORPHANET JOURNAL OF RARE DISEASES, 2017, 12
  • [12] Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease
    Case, Laura E.
    Bjartmar, Carl
    Morgan, Claire
    Casey, Robin
    Charrow, Joel
    Clancy, John P.
    Dasouki, Majed
    DeArmey, Stephanie
    Nedd, Khan
    Nevins, Mary
    Peters, Heidi
    Phillips, Dawn
    Spigelman, Zachary
    Tifft, Cynthia
    Kishnani, Priya S.
    NEUROMUSCULAR DISORDERS, 2015, 25 (04) : 321 - 332
  • [13] Two successfully completed pregnancies in adult onset Pompe disease, under continued treatment with alglucosidase alfa
    Julie Van Houtte
    Jan L. De Bleecker
    Acta Neurologica Belgica, 2019, 119 : 147 - 149
  • [14] Two successfully completed pregnancies in adult onset Pompe disease, under continued treatment with alglucosidase alfa
    Van Houtte, Julie
    De Bleecker, Jan L.
    ACTA NEUROLOGICA BELGICA, 2019, 119 (01) : 147 - 149
  • [15] Enzyme replacement therapy with alglucosidase alfa in a late-onset Pompe disease patient during pregnancy
    Santos, Miguel Oliveira
    Evangelista, Teresinha
    Conceicao, Isabel
    NEUROMUSCULAR DISORDERS, 2018, 28 (11) : 965 - 968
  • [16] Alglucosidase alfa: 5 years of experience in late-onset Pompe disease
    Benedikt Schoser
    BMC Musculoskeletal Disorders, 14 (Suppl 2)
  • [17] Enzyme replacement therapy with alglucosidase alfa in Pompe disease: Clinical experience with rate escalation
    Desai, Ankit K.
    Walters, Crista K.
    Cope, Heidi L.
    Kazi, Zoheb B.
    DeArmey, Stephanie M.
    Kishnani, Priya S.
    MOLECULAR GENETICS AND METABOLISM, 2018, 123 (02) : 92 - 96
  • [18] SWORD: A simplified desensitization protocol for enzyme replacement therapy in adult Pompe disease
    Gallay, Laure
    Petiot, Philippe
    Durieu, Isabelle
    Streichenberger, Nathalie
    Berard, Frederic
    NEUROMUSCULAR DISORDERS, 2016, 26 (11) : 801 - 804
  • [19] Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature
    Khan, Aleena A.
    Case, Laura E.
    Herbert, Mrudu
    DeArmey, Stephanie
    Jones, Harrison
    Crisp, Kelly
    Zimmerman, Kanecia
    ElMallah, Mai K.
    Young, Sarah P.
    Kishnani, Priya S.
    GENETICS IN MEDICINE, 2020, 22 (05) : 898 - 907
  • [20] A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease.
    van der Ploeg, Ans T.
    Clemens, Paula R.
    Corzo, Deyanira
    Escolar, Diana M.
    Florence, Julaine
    Groeneveld, Geert Jan
    Herson, Serge
    Kishnani, Priya S.
    Laforet, Pascal
    Lake, Stephen L.
    Lange, Dale J.
    Leshner, Robert T.
    Mayhew, Jill E.
    Morgan, Claire
    Nozaki, Kenkichi
    Park, Dorothy J.
    Pestronk, Alan
    Rosenbloom, Barry
    Skrinar, Alison
    van Capelle, Carine I.
    van der Beek, Nadine A.
    Wasserstein, Melissa
    Zivkovic, Sasa A.
    NEW ENGLAND JOURNAL OF MEDICINE, 2010, 362 (15) : 1396 - 1406
12345