Synopsis

Source of validity: Scientific
Rating (out of 5): 3

Vitamin A and its derivatives, particularly all-trans retinoic acid (ATRA), have a well-established role in the treatment of a specific type of leukemia known as acute promyelocytic leukemia (APL), a subtype of acute myeloid leukemia (AML). The scientific basis for this use stems from the discovery that APL is characterized by a chromosomal translocation resulting in a fusion protein (PML-RARα) that disrupts normal retinoic acid signaling and blocks the differentiation of promyelocytes. Pharmacological doses of ATRA overcome this block, promoting the maturation of leukemic cells into non-proliferative granulocytes, leading to remission in many patients. This differentiation therapy is considered a paradigm shift in leukemia treatment and is supported by robust clinical evidence, making ATRA (a vitamin A derivative) a standard of care for APL.

However, the use of unspecified vitamin A (as opposed to pharmaceutical-grade ATRA or other retinoids) for other forms of leukemia lacks strong evidence and is not a standard or recommended therapy. The efficacy is specific to the mechanism present in APL, and generic vitamin A supplementation does not have the same effect or safety profile. Therefore, while there is scientific validation for vitamin A derivatives in a specific leukemia context (APL), this does not extend broadly to all leukemias or justify use of over-the-counter vitamin A supplements for leukemia treatment.

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