MDS/Secondary AML Associated with Type I Gaucher Disease

Conclusion]We deeply indicate that the combination of enzyme replacement therapy and azacitidine therapy is useful for cases of MDS/s-AML associated with Gaucher disease. There is a possibility that the reduction of the enzyme is involved not only in Gaucher disease but in the ontogeny of blood diseases such as MDS. The effects of enzyme replacement therapy of Gaucher's disease for MDS/s-AML should be discussed.DisclosuresNo relevant conflicts of interest to declare.

Source: Blood - November 21, 2018 Category: Hematology Authors: Okamoto, A., Yamamoto, K., Eguchi, G., Kanai, Y., Yamaguchi, T., Maeda, Y. Tags: 613. Acute Myeloid Leukemia: Clinical Studies Source Type: research