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Dr Michael Lim, a senior consultant at the Paediatric Pulmonary and Sleep Division, National University Hospital, Singapore, speaks to Roshini Claire Anthony on the rare disease that is cystic fibrosis.

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Maintenance therapy with difluoromethylornithine safe, effective in high-risk neuroblastoma

09 Oct 2018

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.

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Most Read Articles
6 days ago

Dr Michael Lim, a senior consultant at the Paediatric Pulmonary and Sleep Division, National University Hospital, Singapore, speaks to Roshini Claire Anthony on the rare disease that is cystic fibrosis.

4 days ago
Susceptibility‐guided therapy is as effective as empiric modified bismuth quadruple therapy for the first-line treatment of Helicobacter pylori infection, with both yielding excellent eradication rates, as shown in a recent trial.
6 days ago
It appears that long-term consumption of fish and omega-3 fatty acid does not influence the risk of incident hypertension in middle-aged and older men, suggests a US study.
2 days ago
Cabozantinib is not better than mitoxantrone-prednisone at relieving pain in patients with metastatic castration-resistant prostate cancer (mCRPC) with symptomatic bone metastasis, a recent study suggests.