PMID- 27637292
OWN - NLM
STAT- MEDLINE
DCOM- 20180125
LR  - 20180322
IS  - 1873-2364 (Electronic)
IS  - 0960-8966 (Linking)
VI  - 26
IP  - 11
DP  - 2016 Nov
TI  - SWORD: A simplified desensitization protocol for enzyme replacement therapy in 
      adult Pompe disease.
PG  - 801-804
LID - S0960-8966(16)30047-5 [pii]
LID - 10.1016/j.nmd.2016.07.006 [doi]
AB  - Pompe disease is an inherited lysosomal disease in which there is a decrease or 
      absence of acid alpha-glucosidase activity. This enzyme defect induces glycogen 
      storage in different tissues, especially muscle and heart, resulting in muscle 
      weakness, respiratory failure and heart disease. Substitutive enzyme replacement 
      therapy (ERT) dispensed every two weeks is the only treatment that has shown 
      benefits. However, this treatment induces hypersensitivity for half of the 
      treated patients. Reactions range from mild to severe, sometimes requiring ERT 
      suspension and anti-anaphylaxis drug administration. Understandably, high amount 
      of acid alpha-glucosidase infusion seems to be identified by the immune system as 
      a danger associated molecular pattern, and induce an immune reaction, involving 
      sometimes, but not always, immunoglobulin E (IgE) production and activating mast 
      and basophil polynuclear cells. Considering the lack of therapeutic alternatives 
      and the proved benefit of ERT, desensitization finds its place here. We hereby 
      report the case of a patient for whom a simplified desensitization protocol 
      ("SWORD": Start With One Regular Drop) was successfully achieved, allowing ERT to 
      be pursued, resulting eventually in clinical improvement.
CI  - Copyright (c) 2016. Published by Elsevier B.V.
FAU - Gallay, Laure
AU  - Gallay L
AD  - Department of Clinical Immunology, Edouard Herriot University Hospital, 5 place 
      d'Arsonvaal, 69437 Lyon Cedex 03, France; INMG, CNRS UMR 5310-INSERM U1217, 
      University Lyon 1, France.
FAU - Petiot, Philippe
AU  - Petiot P
AD  - Department of Neurology, Croix-Rousse Hospital, Hospices Civils de Lyon, Claude 
      Bernard University Lyon 1, Grande rue de la croix rousse, 69004 Lyon, France.
FAU - Durieu, Isabelle
AU  - Durieu I
AD  - Department of Internal and Vascular Medicine, Lyon Sud University Hospital, 69495 
      Pierre Benite, France.
FAU - Streichenberger, Nathalie
AU  - Streichenberger N
AD  - INMG, CNRS UMR 5310-INSERM U1217, University Lyon 1, France; Department of 
      Neuropathology, Hospices Civils de Lyon, Neurology and Neurosurgery Pierre 
      Wertheimer University Hospital, Boulevard Pinel, 69500 Bron, France.
FAU - Berard, Frederic
AU  - Berard F
AD  - Department of Allergy and Clinical Immunology, Hospital Center Lyon Sud, 69495 
      Pierre Benite, France; University Lyon 1, France. Electronic address: 
      Frederic.berard@chu-lyon.fr.
LA  - eng
PT  - Case Reports
PT  - Journal Article
DEP - 20160719
PL  - England
TA  - Neuromuscul Disord
JT  - Neuromuscular disorders : NMD
JID - 9111470
RN  - EC 3.2.1.3 (Glucan 1,4-alpha-Glucosidase)
SB  - IM
MH  - Adult
MH  - Clinical Protocols
MH  - Drug Hypersensitivity/*prevention & control
MH  - Enzyme Replacement Therapy/*adverse effects/*methods
MH  - Female
MH  - Glucan 1,4-alpha-Glucosidase/*administration & dosage/*adverse effects
MH  - Glycogen Storage Disease Type II/*drug therapy
MH  - Humans
OTO - NOTNLM
OT  - Desensitization
OT  - Enzyme replacement therapy
OT  - Hypersensitivity
OT  - Pompe disease
OT  - Simplified protocol
EDAT- 2016/10/30 06:00
MHDA- 2018/01/26 06:00
CRDT- 2016/09/18 06:00
PHST- 2016/02/06 00:00 [received]
PHST- 2016/05/30 00:00 [revised]
PHST- 2016/07/17 00:00 [accepted]
PHST- 2016/10/30 06:00 [pubmed]
PHST- 2018/01/26 06:00 [medline]
PHST- 2016/09/18 06:00 [entrez]
AID - S0960-8966(16)30047-5 [pii]
AID - 10.1016/j.nmd.2016.07.006 [doi]
PST - ppublish
SO  - Neuromuscul Disord. 2016 Nov;26(11):801-804. doi: 10.1016/j.nmd.2016.07.006. Epub 
      2016 Jul 19.