Evidence supporting the use of: Vitamin A (mixed)
For the health condition: Leukemia

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Synopsis

Source of validity: Scientific
Rating (out of 5): 4

Vitamin A (and its derivatives, particularly all-trans retinoic acid, or ATRA) has a scientifically validated role in the treatment of a specific subtype of leukemia known as acute promyelocytic leukemia (APL), which is a form of acute myeloid leukemia (AML). This therapeutic application arose from research in the late 20th century demonstrating that pharmacological doses of ATRA could induce differentiation of the abnormal promyelocytes characteristic of APL, leading to remission in many patients. The mechanism involves binding to retinoic acid receptors and promoting the maturation of leukemic cells, thus counteracting the block in differentiation caused by the PML-RARα fusion protein in APL. ATRA (a form of vitamin A) is now standard therapy for APL, typically used in combination with arsenic trioxide or chemotherapy. However, this use is highly specific; vitamin A supplementation in the form of "mixed" carotenoids or retinyl esters is not standard for other types of leukemia, nor is it recommended as a general leukemia therapy outside of APL and the retinoid class. The evidence for ATRA in APL is robust, with numerous clinical trials and decades of clinical experience supporting its use, justifying a high evidence rating for this particular application.

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