Maintenance therapy with difluoromethylornithine safe, effective in high-risk neuroblastoma
Difluoromethylornithine (DFMO) maintenance medication in high-risk neuroblastoma (HRNB) patients is associated with better survival outcomes, a recent study has shown.
The study included HRNB patients with histologically confirmed disease who took maintenance DFMO to prevent relapse following completion of standard therapy (stratum 1; n=101; mean age 3.5 years; 56 percent male) or after salvage therapy for relapsed disease (stratum 2; n=39; mean age 3.2 years; 72 percent male).
Over a median follow-up period of 3.5 years, participants in stratum 1 demonstrated a mean 2-year event-free survival rate of 84.4±4 percent. Overall survival was likewise high at 97±2 percent. Survival rates remained high even in patients with high-risk features such as status of the MYCN gene.
In the subset of stratum 1 participants who were given DFMO after the completion of the Children’s Oncology Group antibody clinical trial, event-free and overall survival rates were also high (86±4 percent and 97±2 percent).
In stratum 2, the 2-year event-free and overall survival rates were low at 51±8 percent and 84±6 percent, respectively. These values were lower in patients with relapsed disease (35±11 percent and 80±9 percent, respectively) and higher in those with primary refractory disease (68±11 percent and 89±7 percent, respectively).
In terms of safety, no serious adverse events were reported. Majority (67 percent) of the patients reported no treatment-related adverse events. Grade 2–3 transaminitis was the most common adverse event, and none of the cases required suspension of the DFMO treatment.