Perioperative management of children with glycogen storage disease type II—Pompe disease

Summary Background Pompe disease is a rare metabolic disorder caused by a deficiency of the lysosomal enzyme acid α‐glucosidase. Glycogen accumulation damages skeletal, cardiac, and smooth muscles, causing a progressive and debilitating muscle weakness and cardiomyopathy. As life expectancy has much...

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Published in:	Pediatric anesthesia Vol. 28; no. 5; pp. 428 - 435
Main Authors:	Bosman, Linelot, Hoeks, Sanne E., González Candel, Antonia, Hout, Hannerieke J. M., Ploeg, Ans T., Staals, Lonneke M.
Format:	Journal Article
Language:	English
Published:	France Wiley Subscription Services, Inc 01-05-2018
Subjects:	adverse events Anesthesia, General - adverse effects Anesthesia, General - methods Child Child, Preschool Cohort Studies congenital anomalies and syndromes Enzyme Replacement Therapy Enzymes Female Glycogen Storage Disease Type II - physiopathology Glycogen Storage Disease Type II - therapy Humans Infant Male muscle disorders Perioperative Care - methods Postoperative Care - methods Postoperative Complications - etiology Retrospective Studies Surgical Procedures, Operative - adverse effects Surgical Procedures, Operative - methods muscle disorders congenital anomalies and syndromes adverse events infant child
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Summary:	Summary Background Pompe disease is a rare metabolic disorder caused by a deficiency of the lysosomal enzyme acid α‐glucosidase. Glycogen accumulation damages skeletal, cardiac, and smooth muscles, causing a progressive and debilitating muscle weakness and cardiomyopathy. As life expectancy has much improved since the introduction of enzyme replacement therapy an increasing number of patients are referred for surgical procedures. Due to the potential cardiopulmonary complications, these patients form a high‐risk group for the anesthesiologist. Aims In this study, we investigated the incidence of perioperative complications in children with Pompe disease treated in our hospital since the introduction of enzyme replacement therapy. Methods Anesthetic and perioperative data of children with Pompe disease treated between 1999 and 2015 in the Erasmus MC—Sophia Children's Hospital, University Medical Centre, Rotterdam, The Netherlands, were collected, retrospectively. Results Of the 65 children with Pompe disease, 34 patients underwent in total 77, mostly low‐risk, surgical procedures. Twenty‐one children had the classic infantile form and 13 had a nonclassic presentation of Pompe disease. In 13 (16.8%) procedures, 1 or more perioperative complications occurred. Perioperative desaturation was the main complication (12.9%), followed by arrhythmia (3.8%) and heart failure requiring diuretic treatment (2.6%). One child died 2 days postoperatively, but this was considered unrelated to the procedure. Conclusion Despite the potentially high anesthetic risk for children with Pompe disease under enzyme replacement therapy, the incidence of perioperative complications in our study was relatively low. Our data suggest that with proper precautionary measures and a critical choice of timing of the operation, general anesthesia in children with Pompe disease could be relatively safe nowadays.
Bibliography:	Funding information The project was done with financial support of the Sectie Kinderanesthesiologie (Dutch Society for Pediatric Anesthesiology) of the Dutch Society for Anesthesiology (grant number 2014). ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23
ISSN:	1155-5645 1460-9592
DOI:	10.1111/pan.13361