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Table 2 Pre-specified surrogate outcomes reflecting Gaucher disease activity

From: Plasma chitotriosidase activity versus CCL18 level for assessing type I Gaucher disease severity: protocol for a systematic review with meta-analysis of individual participant data

Outcomes Definitiona
Primary outcome The primary outcome is a composite of one or more individual components among the following:
- Liver volume >1.25 MN
- Spleen volume >5 MN
- Hemoglobin concentration <11.0 g/dL (<10.0 g/dL for patients 6 to 59 months of age)
- Platelet count <100 × 109/mL
Secondary outcomes 1. Composite of one or more individual components among the following:
- Liver volume >2.5 MN
- Spleen volume >15 MN
- Hemoglobin concentration <8.0 g/dL (<7.0 g/dL for patients 6 to 59 months of age)
- Platelet count <50 × 109/mL
2. Symptomatic bone manifestations with imaging confirmation including the following:
- Osteonecrosis
- Fracture
  1. Abbreviation: MN multiple of normal
  2. aOrgan volumes will be expressed as multiples of normal (MN) adjusted for body weight. The normal spleen volume will be computed as 2 mL/kg multiplied by weight in kilograms in patient who did not undergo any splenectomy procedure. The normal liver volume will be computed as 25 mL/kg multiplied by weight in kilograms (see the “Methods” section)