Phase II evaluation of IPI-926, an oral Hedgehog inhibitor, in patients with myelofibrosis

References

• Mesa RA, Verstovsek S, Cervantes F, et al. Primary myelofibrosis (PMF), post polycythemia vera myelofibrosis (post-PV MF), post essential thrombocythemia myelofibrosis (post-ET MF), blast phase PMF (PMF-BP): consensus on terminology by the international working group for myelofibrosis research and treatment (IWG-MRT). Leuk Res 2007;31:737–740.
   PubMed Web of Science ®Google Scholar
• Mehta J, Wang H, Iqbal SU, et al. Epidemiology of myeloproliferative neoplasms in the United States. Leuk Lymphoma 2014;55:595–600.
   PubMed Web of Science ®Google Scholar
• Tefferi A. Myelofibrosis with myeloid metaplasia. N Engl J Med 2000;342:1255–1265.
   PubMed Web of Science ®Google Scholar
• Barbui T, Barosi G, Birgegard G, et al. Philadelphia-negative classical myeloproliferative neoplasms:critical concepts and management recommendations from European LeukemiaNet. J Clin Oncol 2011;29:761–770.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Nagorney DS, Schwager S, et al. Palliative goals, patient selection, and perioperative platelet management:outcomes and lessons from 3 decades of splenectomy for myelofibrosis with myeloid metaplasia at the Mayo Clinic. Cancer 2006;107:361–370.
   PubMed Web of Science ®Google Scholar
• Kiladjian JJ, Chomienne C, Fenaux P. Interferon-alpha therapy in bcr-abl-negative myeloproliferative neoplasms. Leukemia 2008;22:1990–1998.
   PubMed Web of Science ®Google Scholar
• Thomas DA, Giles FJ, Albitar M, et al. Thalidomide therapy for myelofibrosis with myeloid metaplasia. Cancer 2006;106:1974–1984.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Yao X, Cripe LD, et al. Lenalidomide and prednisone for myelofibrosis: Eastern Cooperative Oncology Group (ECOG) phase 2 trial E4903. Blood 2010;116:4436–4438.
   PubMed Web of Science ®Google Scholar
• Quintas-Cardama A, Kantarjian HM, Manshouri T, et al. Lenalidomide plus prednisone results in durable clinical, histopathologic, and molecular responses in patients with myelofibrosis. J Clin Oncol 2009;27:4760–4766.
   PubMed Web of Science ®Google Scholar
• Manoharan A. Management of myelofibrosis with intermittent hydroxyurea. Br J Haematol 1991;77:252–254.
   PubMed Web of Science ®Google Scholar
• Cervantes F, Dupriez B, Pereira A, et al. New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment. Blood 2009;113:2895–28901.
   PubMed Web of Science ®Google Scholar
• Harrison C, Kiladjian JJ, Al-Ali HK, et al. JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. N Engl J Med 2012;366:787–798.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Mesa RA, Gotlib J, et al. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med 2012;366:799–807.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Mesa RA, Gotlib J, et al. Efficacy, safety and survival with ruxolitinib in patients with myelofibrosis: results of a median 2-year follow-up of COMFORT-I. Haematologica 2013;98:1865–1871.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Kantarjian HM, Estrov Z, et al. Long-term outcomes of 107 patients with myelofibrosis receiving JAK1/JAK2 inhibitor ruxolitinib: survival advantage in comparison to matched historical controls. Blood 2012;120:1202–1209.
   PubMed Web of Science ®Google Scholar
• Cervantes F, Vannucchi AM, Kiladjian JJ, et al. Three-year efficacy, safety, and survival findings from COMFORT-II, a phase 3 study comparing ruxolitinib with best available therapy for myelofibrosis. Blood 2013;122:4047–4053.
   PubMed Web of Science ®Google Scholar
• McGowan SE, McCoy DM. Platelet-derived growth factor-A and sonic hedgehog signaling direct lung fibroblast precursors during alveolar septal formation. Am J Physiol Lung Cell Mol Physiol 2013;305:L229–L239.
   PubMed Web of Science ®Google Scholar
• Greenbaum LE. Hedgehog signaling in biliary fibrosis. J Clin Invest 2008;118:3263–3265.
   PubMed Web of Science ®Google Scholar
• Pasca di Magliano M, Hebrok M. Hedgehog signalling in cancer formation and maintenance. Nat Rev Cancer 2003;3:903–911.
   PubMed Web of Science ®Google Scholar
• Ruiz i Altaba A, Sanchez P, Dahmane N. Gli and hedgehog in cancer: tumours, embryos and stem cells. Nat Rev Cancer 2002;2:361–372.
   PubMed Web of Science ®Google Scholar
• Chen JK, Taipale J, Cooper MK, et al. Inhibition of Hedgehog signaling by direct binding of cyclopamine to Smoothened. Genes Dev 2002;16:2743–2748.
   PubMed Web of Science ®Google Scholar
• Taipale J, Chen JK, Cooper MK, et al. Effects of oncogenic mutations in Smoothened and Patched can be reversed by cyclopamine. Nature 2000;406:1005–1009.
   PubMed Web of Science ®Google Scholar
• Olive KP, Jacobetz MA, Davidson CJ, et al. Inhibition of Hedgehog signaling enhances delivery of chemotherapy in a mouse model of pancreatic cancer. Science 2009;324:1457–1461.
   PubMed Web of Science ®Google Scholar
• Jimeno A, Weiss GJ, Miller WH Jr, et al. Phase I study of the Hedgehog pathway inhibitor IPI-926 in adult patients with solid tumors. Clin Cancer Res 2013;19:2766–2774.
   PubMed Web of Science ®Google Scholar
• Jamieson C, Cortes JE, Oehler V, et al. Phase 1 dose-escalation study of PF-04449913, an oral hedgehog (Hh) inhibitor, in patients with select hematologic malignancies. Blood 2011;118(Suppl. 1): Abstract 424.
   Google Scholar
• Swerdlow SH, Campo E, Harris NL, et al. WHO classification of tumours of haematopoietic and lymphoid tissues. 4th ed. Lyon: IARC; 2008.
   Google Scholar
• Simon R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials 1989;10:1–10.
   PubMedGoogle Scholar
• Tefferi A, Barosi G, Mesa RA, et al. International Working Group (IWG) consensus criteria for treatment response in myelofibrosis with myeloid metaplasia, for the IWG for Myelofibrosis Research and Treatment (IWG-MRT). Blood 2006;108:1497–1503.
   PubMed Web of Science ®Google Scholar
• Scherber R, Dueck AC, Johansson P, et al. The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF): international prospective validation and reliability trial in 402 patients. Blood 2011;118:401–408.
   PubMed Web of Science ®Google Scholar
• Calhoun ME, Mouton PR. Length measurement: new developments in neurostereology and 3D imagery. J Chem Neuroanat 2000;20:61–69.
   PubMed Web of Science ®Google Scholar
• Thiele J, Kvasnicka HM, Facchetti F, et al. European consensus on grading bone marrow fibrosis and assessment of cellularity. Haematologica 2005;90:1128–1132.
   PubMed Web of Science ®Google Scholar
• Humke EW, Dorn KV, Milenkovic L, et al. The output of Hedgehog signaling is controlled by the dynamic association between Suppressor of Fused and the Gli proteins. Genes Dev 2010;24:670–682.
   PubMed Web of Science ®Google Scholar
• Bhagwat N, Keller MD, Rampal RK, et al. Improved efficacy of combination of JAK2 and hedgehog inhibitors in myelofibrosis. Blood 2013;122(Suppl. 1): Abstract 666.
   PubMed Web of Science ®Google Scholar