Phase II study of doxercalciferol for the treatment of myelodysplastic syndrome

Abstract

We conducted a phase II trial of doxercalciferol, a vitamin D₂ analogue, in 15 patients with MDS. Each received doxercalciferol 12.5 µg orally daily for 12 weeks. Nine of 15 patients completed the prescribed course and of these, six had stable disease. No patient had a response (IWG criteria) and overall eight patients experienced progressive disease while on therapy. Two patients with chronic myelomonocytic leukemia (CMML) had a marked rise in monocytes on study. Overall the treatment was well tolerated. One patient was removed from study due to hypercalcemia. We conclude that short-term treatment with doxercalciferol has limited activity in patients with MDS.

Keywords:

• Doxercalciferol
• myelodysplastic syndrome
• phase II study