Notice: Users may be experiencing issues with displaying some pages on stanfordhealthcare.org. We are working closely with our technical teams to resolve the issue as quickly as possible. Thank you for your patience.
 

See our updated masking policy »

Stanford Health Care

Phase II evaluation of IPI-926, an oral Hedgehog inhibitor, in patients with myelofibrosis LEUKEMIA & LYMPHOMA Sasaki, K., Gotlib, J. R., Mesa, R. A., Newberry, K. J., Ravandi, F., Cortes, J. E., Kelly, P., Kutok, J. L., Kantarjian, H. M., Verstovsek, S. 2015; 56 (7): 2092-2097

Abstract

The clinical safety and efficacy of IPI-926 was evaluated in 14 patients with myelofibrosis in a phase II study. Patients received 160 mg IPI-926 orally in continuous 28-day cycles. The median treatment duration was 5.1 months, and all patients had discontinued treatment by 7.5 months. Nine patients discontinued due to lack of response as determined by the treating physician, two after developing acute leukemia and one due to disease progression/loss of response. Twelve patients had slight reductions in spleen size (less than 50% from baseline), but symptoms did not improve consistently. One patient achieved transfusion independence lasting 5 months. Reductions in GLI1 mRNA and protein levels, JAK2V617F allele burden, degree of fibrosis or cytokine levels were observed in some patients, but were not significant when evaluated for the cohort. Low-grade gastrointestinal/liver abnormalities were the most common toxicities. The results did not support continued evaluation of IPI-926 as a monotherapy in myelofibrosis.

View details for DOI 10.3109/10428194.2014.984703

View details for Web of Science ID 000359888700028

View details for PubMedID 25641433