Low-dose therapy trumps high-dose therapy again in the treatment of Gaucher disease.

The paper by De Fost et al. in the current issue of Blood contains important data with regard to dose-sensitivity in the treatment of Gaucher disease, but the title is misleading. It implies that high-dose enzyme replacement therapy may be superior to low-dose enzyme replacement therapy. However, it is apparent that “superiority” in response is limited to two surrogate markers, serum chitotriosidase and marrow fat content as measured by MRI. Importantly, the improvement in hematologic findings, organomegaly, and frequency of skeletal crises, is equivalent in low-dose and high dose therapy. Contrary to what De Fost et al. imply, neither serum chitotriosidase nor marrow fat are clearly related to disease severity, and we aver that the response of surrogates is of little importance selecting a program of treatment for a patient. Given the very high cost of enzyme replacement therapy, the study by De Fost et al. is another compelling reason for physicians not to use high-dose therapy in the treatment of Gaucher disease. For personal use only. on September 14, 2017. by guest www.bloodjournal.org From