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Biomarkers in Gaucher disease
platelet count <100x109/L, spleen volume >5 MN, and liver vol- ume >1.25 MN. The secondary outcomes included symptomatic bone events, a composite of hemoglobin concentration <8 g/dL (<7 g/dL for patients 12-59 months of age), platelet count <50x109/L, spleen volume >15 MN, and liver volume >2.5 MN, and individual components of the primary and secondary com- posite outcomes. Bone events included skeletal fracture,
osteonecrosis, or avascular necrosis that occurred within the pre- vious 12 months of biomarker analysis.21 All outcomes and cut-off values for continuous parameters were set according to published guidelines or previous studies,22,23 and were pre-specified.21
Statistical analysis
We performed logarithm transformation for chitotriosidase
Figure 1. Identification and selection of studies for the meta-analysis of individual participant data. *Seventy three additional participants (222 observations) had splenectomy at base- line. IDP: individual participant data.
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