Autologous Bone Marrow Transplantation - Blog Science Connections

ENSG 1—Randomized Study of High-Dose
Melphalan in Neuroblastoma
R. Pinkerton, J. Pritchard, J. de Kraker, D. Jones, S. Germond,
and S. Loue
The outcome for children with advanced neuroblastoma has improved with
the introduction of intensive chemotherapy, but, despite high initial remission
rates, prolonged survival remains limited to around 10% of patients with stage
4 disease who are older than 1 year at diagnosis. After almost any of the
current regimens, about 70% of children achieve partial or complete
responses following chemotherapy and surgery. In 1983 the European
Neuroblastoma Study Group (ENSG) set out to answer the question of
whether intensive consolidation chemotherapy would produce in such
patients better results than no further treatment, in terms of either diseasefree
progression or survival. Phase 2 studies with high-dose melphalan
followed by autologous bone marrow transplantation (AF3MT) had shown
activity in patients with relapsed or chemoresistant disease; therefore this was
a logical agent to evaluate. Because of the morbidity, prolonged hospitalization,
and emotional trauma of isolation nursing inevitable with high-dose
chemotherapy and ABMT procedures, we believed it essential to evaluate this
regimen with a prospective, randomized study rather than just a historical
comparison. The latter would have been particularly difficult due to the
recent introduction of more intensive conventional dose-induction regimens.
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