Heparan sulphate inhibits CXCL12-mediated hematopoietic cell migration and engraftment in mucopolysaccharidosis type I mice

Abstract

reduced 50% of their normal therapeutic dosage of imiglucerase. After 6–12 month, 9 of them switched to velaglucerase alfa treatment. They have been followed for more than 12 months since drug switching. Clinic, biochemical and imaging parameters have been evaluated every 3–6 months. Velaglucerase alfa was administered at the dosage preceding the shortage period (range 60 to 94 U/kg/month). Results: Imiglucerase reduction was generally well tolerated in the majority of the patients, while only few showed GD symptoms recurrence (mainly asthenia and bone pain). One year after enzyme switch, asthenia improved in all patients and bone pain in two of them. At biochemical level, hemoglobin increased in 3 patients and platelet in 1 patient. Imaging analysis of organ volumes and femurs storage did not show significant modifications. No side effects were recorded. Conclusions: Velaglucerase alfa demonstrated to be an effective therapeutic alternative to imiglucerase in treating Gaucher disease.