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
  • [31] A Multi-Centre Prospective Study of the Efficacy and Safety of Alglucosidase Alfa in Chinese Patients With Infantile-Onset Pompe Disease
    Zhu, Diqi
    Zhu, Jiacong
    Qiu, Wenjuan
    Wang, Benzhen
    Liu, Lin
    Yu, Xiaodan
    Ou, Zhenheng
    Shan, Guangsong
    Wang, Jian
    Li, Bin
    Chen, Xiaokang
    Liu, Cong
    Li, Zipu
    Fu, Lijun
    FRONTIERS IN PHARMACOLOGY, 2022, 13
  • [32] Unwarranted, long term, alglucosidase alfa enzyme replacement therapy in two non-Pompe disease patients
    Karam, Chafic
    Ragole, Thomas
    Moshe-Lilie, Orly
    Chahin, Nizar
    CLINICAL NEUROLOGY AND NEUROSURGERY, 2020, 196
  • [33] Long-term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: Prospective analysis from the French Pompe Registry
    Semplicini, Claudio
    De Antonio, Marie
    Taouagh, Nadjib
    Bhin, Anthony
    Bouhour, FranOoise
    Echaniz-Laguna, Andoni
    Magot, Armelle
    Nadaj-Pakleza, Aleksandra
    Orlikowski, David
    Sacconi, Sabrina
    Salort-Campana, Emmanuelle
    Sole, Guilhem
    Tard, Cline
    Zagnoli, Fabien
    Hogrel, Jean-Yves
    Hamroun, Dalil
    Lafort, Pascal
    JOURNAL OF INHERITED METABOLIC DISEASE, 2020, 43 (06) : 1219 - 1231
  • [34] Pregnancy during enzyme replacement therapy with alglucosidase alfa over a 14-year period in late-onset Pompe disease
    Yanagihara, Chie
    Hayasaka, Yuki
    Kageyama, Yasufumi
    NEUROLOGY AND CLINICAL NEUROSCIENCE, 2023, 11 (02): : 97 - 99
  • [35] Efficacy and safety of enzyme replacement therapy with alglucosidase alfa for the treatment of patients with infantile-onset Pompe disease: a systematic review and metanalysis
    Dornelles, A. D.
    Junges, A. P. P.
    Krug, B.
    Goncalves, C.
    de Oliveira Junior, H. A.
    Schwartz, I. V. D.
    FRONTIERS IN PEDIATRICS, 2024, 12
  • [36] An individually, modified approach to desensitize infants and young children with Pompe disease, and significant reactions to alglucosidase alfa infusions
    El-Gharbawy, Areeg H.
    Mackey, Joanne
    DeArmey, Stephanie
    Westby, Greg
    Grinnell, Sherry G.
    Malovrh, Peggy
    Conway, Robert
    Kishnani, Priya S.
    MOLECULAR GENETICS AND METABOLISM, 2011, 104 (1-2) : 118 - 122
  • [37] Real-world outcomes from a series of patients with late onset Pompe disease who switched from alglucosidase alfa to avalglucosidase alfa
    Carter, Chris
    Boggs, Tracy
    Case, Laura E.
    Kishnani, Priya
    FRONTIERS IN GENETICS, 2024, 15
  • [38] Pharmacokinetics of Alglucosidase Alfa Manufactured at the 4000-L Scale in Participants with Pompe Disease: A Phase 3/4 Open-Label Study
    Nicolas, Xavier
    Hurbin, Fabrice
    Periquet, Magali
    Richards, Susan
    Sensinger, Charlotte
    Welch, Karen
    An Haack, Kristina
    CLINICAL PHARMACOLOGY IN DRUG DEVELOPMENT, 2023, 12 (12): : 1185 - 1193
  • [39] Desensitization of olipudase alfa-induced anaphylaxis in a child with chronic neurovisceral acid sphingomyelinase deficiency
    Fiori, Laura
    Tagi, Veronica Maria
    Montanari, Chiara
    Gambino, Mirko
    Carlevatti, Veronica
    Zizzo, Carmela
    D'Auria, Enza
    Dilillo, Dario
    Verduci, Elvira
    Zuccotti, Gianvincenzo
    MOLECULAR GENETICS AND METABOLISM REPORTS, 2024, 40
  • [40] Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study
    van der Ploeg, Ans
    Carlier, Pierre G.
    Carlier, Robert-Yves
    Kissel, John T.
    Schoser, Benedikt
    Wenninger, Stephan
    Pestronk, Alan
    Barohn, Richard J.
    Dimachkie, Mazen M.
    Goker-Alpan, Ozlem
    Mozaffar, Tahseen
    Pena, Loren D. M.
    Simmons, Zachary
    Straub, Volker
    Guglieri, Michela
    Young, Peter
    Boentert, Matthias
    Baudin, Pierre-Yves
    Wens, Stephan
    Shafi, Raheel
    Bjartmar, Carl
    Thurberg, Beth L.
    MOLECULAR GENETICS AND METABOLISM, 2016, 119 (1-2) : 115 - 123
12345