Here are the latest figures from Dr Sholler's DFMO trial presented in an abstract for SIOP 2016.
There are a handful more patients included since the last abstract published earlier this year for ANR 2016, which was previously copy+pasted in this thread.
MAINTENANCE DFMO INCREASES SURVIVAL IN HIGH RISK NEUROBLASTOMA
G. Saulnier-Sholler1,2, W. Ferguson3, G. Bergendahl1, K. Neville4, D. Eslin5, W. Roberts6, R. Wada7, J. Oesterheld8, D. Mitchell1, J. Foley1, V. Brown9, N. Parikh10, F. Eshun11, P. Zage12, J. Rawwas13, S. Sencer13, D. Pankiewicz1, M. Quinn1, M. Rich1, J. Kraveka14
High Risk Neuroblastoma (HRNB) remains a challenge, accounting for 15% of all paediatric cancer deaths. While most attain remission, the natural history of HRNB is well documented with approximately half of patients relapsing within 5 years after completion of immunotherapy. This study evaluated the effectiveness of the ODC inhibitor difluoromethylornithine (DFMO) as a maintenance therapy to prevent relapse in HRNB patients after the completion of standard therapy.
This study was a single agent, multicenter study, enrolling from June 2012 to February 2016. Patients received 27 four-week cycles of oral DFMO at a dose of 750 ± 250 mg/m2 twice daily. Event free survival (EFS) and overall survival (OS) were determined on an intention-to-treat basis.
A total of 101 patients received DFMO, 98 were eligible for the intention to treat (ITT) analysis. For all ITT patients, EFS was 89% (± 4%) and OS 98% (±2%) at 2 years. For the subgroup of patients (n=79) who had been previously enrolled on ANBL0032 prior to starting this study, the 2 year EFS was 92% (± 4%) and OS 98% (±2%). DFMO was well tolerated, with grade 2-3 transaminitis being the most common toxicity reported in <10 % of patients.
DFMO at 750 ± 250 mg /m2 BID following completion of standard therapy for HRNB is safe and associated with improved EFS and OS in children with HRNB. DFMO is now currently undergoing further evaluation in a prospective confirmatory study for additional safety and pharmacokinetic analysis.
Original link can be found here (on page 351): http://www.siop2016.kenes.com/landing/Documents/PBC_Abstracts.pdf