In case anyone hasn't seen it, this is the abstract of Dr Sholler's presentation on her DFMO trial for ANR2016 taking place in Cairns next month. But it is only a summary and certainly there is information on the slide in the twitter picture that isn't in this summary e.g. median follow-up time.
DFMO maintains remission and increases overall survival in high risk neuroblastoma: results of a phase II prevention trial (#88)
Giselle Sholler 1 , William Ferguson 2 , Genevieve Bergandahl 1 , Jeffrey Bond 3 , Kathleen Neville 4 , Don Eslin 5 , William Roberts 6 , Randal Wada 7 , Javier Oesterheld 8 , Deanna Mitchell 1 , Jessica Foley 1 , Valerie Brown 9 , Nehal Parikh 10 , Francis Eshun 11 , Peter Zage 12 , Jawhar Rawwas 13 , Susan Sencer 13 , Debra Pankiewicz 14 , Monique Quinn 14 , Maria Rich 1 , Joseph Junewick 15 , Max Wicha 16 , Jacqueline Kraveka 17
Background: High Risk Neuroblastoma (HRNB) remains a challenge in pediatric oncology, accounting for 15% of all pediatric cancer deaths. While most patients are able to 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), which targets cancer stem cell pathways in HRNB, as a maintenance therapy to prevent relapse in HRNB patients who were in complete remission at the completion of standard therapy.
Methods: This study was an open label, single agent, multicenter study. Enrollment began in June 2012 and ended in February 2016. Subjects received 27 4-week cycles of oral DFMO at a dose of 500-1000 mg/m2 twice daily. Event free survival (EFS) and overall survival (OS) were determined on an intention-to-treat (ITT) basis.
Results: A total of 94 subjects received DFMO, 91 were eligible for the intention to treat (ITT) population. For all ITT subjects, EFS was 91% (± 4%) and OS 98% (+/-2%) at 2 years. For the subgroup of subjects (n=74) who were previously enrolled on the ANBL0032 study, the 2 year EFS was 95% (± 3%) and OS 98% (+/-2%). This is a significant improvement in comparison to ANBL0032 study which showed a conservative EFS of 76% 2 years post antibody therapy (p <0.01) and OS of 89% (p
Conclusions: Administration of DFMO at 500-1000mg/m2 BID is an effective and safe dose. Following the completion of standard therapy for high risk neuroblastoma DFMO treatment was associated with improved EFS and OS decreasing the high rate of relapse in children with HRNB. An additional prospective trial is ongoing to confirm these results.