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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.
When might retinoid therapy be used?
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, nosebleeds, muscle and joint pains, and changes in the nails are also possible.
Bloodwork may be done before and while taking the drug to watch for possible effects of the drug on the liver or cholesterol levels.
Retinoids can also make the skin more sensitive to the sun, so doctors typically advise that prolonged sun exposure be limited.
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- References
Developed by the American Cancer Society medical and editorial content team with medical review and contribution by the American Society of Clinical Oncology (ASCO).
Dome JS, Rodriguez-Galindo C, Spunt SL, Santana VM. Chapter 92: Pediatric solid tumors. In: Neiderhuber JE, Armitage JO, Doroshow JH, Kastan MB, Tepper JE, eds. Abeloff’s Clinical Oncology. 6th ed. Philadelphia, PA. Elsevier; 2020.
Matthay KK, Villablanca JG, Seeger RC, et al. Treatment of high-risk neuroblastoma with intensive chemotherapy, radiotherapy, autologous bone marrow transplantation, and 13-cis-retinoic acid. New England Journal of Medicine. 1999;341(16):1165-1173.
National Cancer Institute. Neuroblastoma Treatment (PDQ). 2024. Accessed at https://www.cancer.gov/types/neuroblastoma/hp/neuroblastoma-treatment-pdq on March 24, 2025.
Park JR, Hogarty MD, Bagatell R, et al. Chapter 23: Neuroblastoma. In: Blaney SM, Adamson PC, Helman LJ, eds. Pizzo and Poplack’s Principles and Practice of Pediatric Oncology. 8th ed. Philadelphia Pa: Lippincott Williams & Wilkins; 2021.
Pinto NR, Applebaum MA, Volchenboum SL, et al. Advances in risk classification and treatment strategies for neuroblastoma. J Clin Oncol. 2015: 30;3008-3017.
Shohet JM, Nuchtern JG, Foster JH. Treatment and prognosis of neuroblastoma. UpToDate. 2025. Accessed at https://www.uptodate.com/contents/treatment-and-prognosis-of-neuroblastoma on March 24, 2025.
Last Revised: June 26, 2025
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