Table 3.

Median changes in plasma biomarkers from baseline to 4 y

End point	Baseline median (min, max)	4-y median (min, max)	Change from baseline median (min, max)	Percent change from baseline median (min, max)
Normalized chitotriosidase activity, nmol/h/mL	605 (0, 10 761); n = 99	363 (4, 3916); n = 30	−310 (−3397, 859); n = 26	−63 (−98, 81); n = 26
Plasma GL-1, μg/mL	5.10 (2.2, 16.9); n = 155	2.00 (2.0, 5.3); n = 42	−3.35 (−8.4, 0.4); n = 42	−60.36 (−80.8, 8.2); n = 42
Plasma GM3, μg/mL	13.0 (7, 30); n = 136	6.0 (4, 15); n = 43	−7.0 (−23, 0); n = 29	−55.6 (−82, 0); n = 29
Plasma ceramide, mg/L	3.90 (2.2, 8.3); n = 154	3.80 (2.5, 6.4); n = 43	−0.60 (−2.4, 1.6); n = 43	−13.33 (−40.0, 66.7); n = 43
Plasma sphingomyelin, μg/mL	318.0 (200, 596); n = 154	434.0 (224, 645); n = 43	133.0 (0, 362); n = 43	41.8 (0, 127); n = 43
Plasma macrophage inflammatory protein 1β, pg/mL	51.65 (9.3, 433.8); n = 152	55.30 (15.6, 193.7); n = 38	−21.78 (−279.3, 114.4); n = 38	−31.45 (−83.3, 289.3); n = 38

Normalized chitotriosidase: values were doubled for patients who had heterozygous chitotriosidase genotypes for the common null (24 bp duplication) mutation. Patients who have a chitotriosidase genotyping category of “homozygous mutation” have no expected chitotriosidase activity; therefore, their values for below the level of quantification were set equal to missing for this analysis. Normal ranges: chitotriosidase 4-120 nmol/h/mL; plasma GL-1, ≤2.0-6.6 μg/mL; GM3, 5-21 μg/mL; ceramide, 1.8-6.5 μg/mL; sphingomyelin, <200-703 μg/mL; macrophage inflammatory protein 1β, 27.3-77.2 pg/mL.

Max, maximum; min, minimum.