Evaluation of the efficacy of ursodiol for prevention of hepatotoxicity in patients receiving gemtuzumab ozogamicin and inotuzumab ozogamicin.

Gemtuzumab ozogamicin (GO) and inotuzumab ozogamicin (InO) are indicated for newly diagnosed or relapsed/refractory CD33-positive acute myeloid leukemia and relapsed/refractory B-cell precursor acute lymphoblastic leukemia respectively. Patients undergoing therapy with these agents are at increased risk for hepatotoxicity. Forty-nine patients received either GO or InO with concomitant ursodiol (n=14) or no ursodiol (n=35) for hepatotoxicity prophylaxis. Hepatotoxicity occurred in 2 (14%) patients in the ursodiol group compared to 15 (43%) patients in the no ursodiol group (p=0.10). Median days (17 versus 11; p=0.66) and doses (4 versus 2; p=0.28) to development of hepatotoxicity were higher in the ursodiol versus no ursodiol group. After adjusting for concomitant hepatotoxic medications and prior chemotherapy, ursodiol did not significantly reduce the incidence of hepatotoxicity. Ursodiol prophylaxis was associated with a similar incidence of hepatotoxicity compared to no ursodiol, but may delay the time to occurrence.