Mean percentage change in hematologic values, organ values, and biomarkers in phase 1/2 trial. A marked increase in hemoglobin concentration (A) and platelet count (B) is observed during weeks 25 and 37 along with a marked reduction in liver (C) and spleen volumes (D). Although biomarker sampling for chitotriosidase (E) and CCL18 (F) was incomplete, a general decrease in both biomarkers relative to baseline is observed per patient over time.