An Italian case series' description of thiamine responsive megaloblastic anemia syndrome: importance of early diagnosis and treatment.
Francesca Di CandiaValentina Di IorioNadia TintoRiccardo BonfantiClaudio IovinoFrancesco Maria RosanioLudovica FediFernanda IafuscoFrancesca ArrigoniRita MalesciFrancesca SimonelliAndrea RigamontiAdriana FranzeseEnza MozzilloPublished in: Italian journal of pediatrics (2023)
Early institution of high-dose thiamine supplementation seems to prevent the development of retinal changes and optic atrophy in TRMA patients. The spectrum of clinical manifestations is broad, and it is important to describe known Cases to gain a better understanding of this rare disease.