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HR-NLB1: Busulphan/melphalan improved 3-year OS to 60% in pediatric neuroblastoma

Issue: July 25, 2011

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2011 ASCO Annual Meeting

CHICAGO — Children with hard-to-treat neuroblastoma assigned to myeloablative chemotherapy with busulphan and melphalan had better OS and event-free survival compared with patients assigned to a myeloablative regimen of carboplatin, etoposide and melphalan.

Combined with recent results showing that an anti-GD2 ch14.18 antibody-based immune therapy can increase event-free survival and OS by 20% in high-risk patients, Ruth Ladenstein, MD, MBA, associate professor of pediatrics at the University of Vienna and St. Anna Children’s Cancer Research Institute, said researchers may soon be able to boost survival rates past 50% by choosing the right high-dose myeloablative regimen.

“This is the first time in pediatrics that we can show, in a specific disease, the superiority and the impact of a myeloablative high-dose regimen, with the specificity of busulphan, to act on resting tumor cells,” she said.

There are approximately 650 neuroblastomas diagnosed annually in the United States, but it is the most common cancer in the first year of life and accounts for 15% of childhood cancer deaths. About 40% of patients are considered high-risk.

In HR-NLB1, 563 children from 20 countries with stage IV high-risk disease with distant metastases or local disease with MYCN oncogene amplification were randomly assigned to either busulphan-melphalan (n=281) or carboplatin, etoposide and melphalan (n=282). At three years, event-free survival was 49% in the busulphan-melphalan arm vs. 33% in the carboplatin, etoposide and melphalan arm. Three-year OS also favored busulphan-melphalan, 60% vs. 48%.

Ladenstein added that busulphan-melphalan was also associated with lower rates of relapse and progression, 47% vs. 60%.

The treatment-related death rate was 3% for busulphan-melphalan and 5% for carboplatin, etoposide and melphalan. – by Jason Harris

For more information:

• Ladenstein RL. #2. Presented at: 2011 ASCO Annual Meeting.

Disclosure: Dr. Ladenstein reported no relevant financial disclosures.

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