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Enzyme replacement therapy for late‐onset Pompe disease

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Appendices

Appendix 1. Search methods ‐ electronic searches

Database/ Resource

Strategy

MEDLINE OvidSP (1946 – present)

#1 Glycogen Storage Disease Type II/

#2 (glycogen storage disease adj2 (type 2 or type II)).tw.

#3 pompe disease.tw.

#4 1 or 2 or 3

#5 Enzyme Replacement Therapy/

#6 (enzyme* adj2 replac*).tw.

#7 (alglucosidase or myozyme or lumizyme or genzyme).tw.

#8 5 or 6 or 7

#9 4 and 8

#10 ((late or adult) adj3 onset).tw.

#11 9 and 10

#12 randomized controlled trial.pt.

#13 controlled clinical trial.pt.

#14 randomized.ab.

#15 placebo.ab.

#16 drug therapy.fs.

#17 randomly.ab.

#18 trial.ab.

#19 groups.ab.

#20 12 or 13 or 14 or 15 or 16 or 17 or 18 or 19

#21 (animals not (humans and animals)).sh.

#22 20 not 21

#23 11 and 22

NOTE: Lines #12‐ #22 are the Cochrane Highly Sensitive Search Strategy for identifying randomized trials in MEDLINE: sensitivity‐maximizing version (2008 revision); Ovid format

Clinicaltrials.gov

[Advanced Search Form]

OTHER TERMS: alglucosidase OR myozyme OR lumizyme OR genzyme OR enzyme*

STUDY TYPE: Interventional Studies

CONDITION/ DISEASE: Pompe disease OR glycogen storage disease OR LOPD

WHO ICTRP

[Advanced Search]

CONDITION: Pompe disease OR glycogen storage disease OR LOPD

INTERVENTION: alglucosidase OR myozyme OR lumizyme OR genzyme OR enzyme*

RECRUITMENT STATUS: All

Appendix 2. Search methods ‐ handsearching

The following conference proceedings will be searched from 2000 onwards until one year prior to completion of the review.

1. SSIEM (Society for the Study of Inborn Errors of Metabolism) International Conference (2000 onwards).
2. Lysosomal Disease Network Annual WORLD Symposium (2000 onwards).