Author Interviews, Cancer Research, Leukemia / 21.12.2015 Interview with: Renaud Capdeville, MD Global Program Head Oncology Global Development Novartis Medical Research: What is the background for this study? What are the main findings? Dr. Capdeville: RATIFY (Randomized AML Trial in FLT3 in patients <60 Years Old; CALGB 10603) was a Phase III, international, randomized, placebo-controlled, double-blind group trial of newly-diagnosed (Acute myeloid leukemia, acute myelogenous leukemia) AML patients aged 18 to less than 60 who have a FLT3 mutation. In the study, patients who received the investigational compound PKC412 (midostaurin) plus standard induction and consolidation chemotherapy experienced a 23% improvement in overall survival (OS) (hazard ratio [HR] = 0.77, P =0.0074) compared with those treated with standard induction and consolidation chemotherapy alone. The median OS for patients in the PKC412 (midostaurin) treatment group was 74.7 months (95% confidence interval [CI]: 31.7, not attained), versus 25.6 months (95% CI: 18.6, 42.9) for patients in the placebo group. The data, collected and analyzed in partnership with the Alliance for Clinical Trials in Oncology, are from the largest clinical trial in FLT3-mutated AML to date, with 3,279 patients screened and 717 study participants from around the world. (more…)
Author Interviews, Hematology / 11.12.2015 Interview with: Dr. Elena Santagostino, MD PhD Angelo Bianchi Bonomi Hemophilia and Thrombosis Center Ca' Granda Foundation Maggiore Hospital Policlinico, Milan, Italy Medical Research: What is the background for this study? What are the main findings? Dr. Santagostino: Two of our abstracts presented at the 57th ASH Annual Meeting are part of the PROLONG-9FP clinical program evaluating the efficacy and safety of CSL Behring’s investigational long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP).  The first is an oral presentation on results from two Phase III studies and an ongoing extension study of rIX-FP for routine prophylaxis in previously-treated people with hemophilia B. The two Phase III studies included adolescents and adults (ages 12 to 61) treated with dosing intervals of up to 14 days for 12 to 18 months, and children (ages 1 to 11) who received seven-day prophylaxis treatment for approximately 12 months. Overall, the median annualized spontaneous bleeding rate (AsBR) was 0.00 for all treatment intervals. The extension study is examining longer treatment intervals, including 10- and 14-day intervals in patients younger than 12 and more prolonged treatment intervals in patients older than 18, and so far has reported favorable long-term tolerability with no serious adverse reactions such as the development of inhibitors to factor IX or antibodies to rIX-FP. A second abstract reported on a surgical sub-study in these trials found that a single pre-operative dose of rIX-FP maintained hemostasis during surgery with responses rated by investigators as “excellent” or “good.” Oven a 14-day perioperative period, patients needed six or seven infusions, and none developed inhibitors to factor IX or antibodies to rIX-FP. (more…)
Author Interviews, Cancer Research, Chemotherapy, Hematology, Immunotherapy / 09.12.2015 Interview with: Dr. Ajai Chari MD Associate Professor Medicine, Hematology and Medical Oncology Tisch Cancer Institute Mount Sinai School of Medicine New York, NY Medical Research: What is the background for this study? Dr. Chari: This is a heavily pretreated population where the median progression free survival (PFS) of the pomalidomide dexamethasone is only 4 months and ORR is only 31%. While the anti CD38 monoclonal antibody daratumumab has single agent has activity in this setting, patients with rapidly progressive disease need combination therapy to achieve rapid and deep responses. Pomalidomide also upregulates CD38 on MM cells and like daratumumab, increases the effector cell activity against myeloma. Thus, there is a strong preclinical and clinical rationale for combining daratumumab with pomalidomide and dexamethasone. (more…)