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Vitamin A Analogues


In studies, a vitamin A analogue, retinoic acid, has activity against minimal residual neuroblastoma (disease that can not be identified by routine methods).

May work even in tumors that are resistant to chemotherapy and/or radiation.

Retinoic acid works by:

  • Decreasing proliferation
  • Decreasing expression of N-MYC
  • Causing morphologic differentiation (from immature Neuroblastoma to the more mature form)

Toxicities include:

  • Cheilitis
  • Dry skin
  • Hypertriglyceridemia
  • Hypercalcemia

In a randomized trial (CCG-3891), patients with high risk neuroblastoma were randomly assigned to receive a 6 month course of isotretinoin versus observation alone (after chemotherapy and autologous bone marrow transplant). The 3 year event free survival, for the patients who received isotretinoin was about 50% vs 35% for those who did not. The difference was not statistically significant, but there was a strong trend towards improved survival with isotretinoin.

Retinoic Acid has now been incorporated into the standard treatment for high risk neuroblastoma.








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