Retinoid Therapy for Neuroblastoma

Retinoids are chemicals that are related to vitamin A. They are known as differentiating agents because they are thought to help some cancer cells mature (differentiate) into normal cells.

In children with high-risk neuroblastoma, treatment with a retinoid called 13-cis-retinoic acid (isotretinoin) reduces the risk of the cancer coming back after high-dose chemotherapy and stem cell transplant. Most doctors now recommend 6 months of 13-cis-retinoic acid after the transplant. This drug is taken as a capsule, twice a day for 2 weeks, followed by 2 weeks off.

Researchers are now trying to develop more effective retinoids and to define the exact role of this approach in treating neuroblastoma.

Possible side effects

The most common side effect of 13-cis-retinoic acid is dry and cracked lips. Dry skin or eyes are also possible, as are nosebleeds, muscle and joint pains, and changes in the nails.

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Brodeur GM, Hogarty MD, Bagatell R, Mosse YP, Maris JM. Neuroblastoma. In: Pizzo PA, Poplack DG, eds. Principles and Practice of Pediatric Oncology. 7th ed. Philadelphia Pa: Lippincott Williams & Wilkins; 2016:772–792.

Pinto NR, Applebaum MA, Volchenboum SL, et al. Advances in risk classification and treatment strategies for neuroblastoma. J Clin Oncol. 2015: 30;3008-3017.

Last Medical Review: March 19, 2018 Last Revised: March 19, 2018

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