Deferasirox Might Be Effective for Microcytic Anemia and Neurological Symptoms Associated with Aceruloplasminemia: A Case Report and Review of the Literature

Miyake, Zenshi; Nakamagoe, Kiyotaka; Yoshida, Katsumi; Kondo, Tadashi; Tamaoka, Akira

doi:10.2169/internalmedicine.4178-19

Cited by 9 publications

(2 citation statements)

References 21 publications

(25 reference statements)

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“…Based on a literature review, it was suggested that chelation treatment in neurologically symptomatic patients rarely leads to improvement while a long-term treatment in neurologically asymptomatic patients may prevent the occurrence of neuropsychiatric symptoms [151]. Others have documented improvement of neurological symptoms in 50% of patients with neurological symptoms [152]. An aggregated case study analyzed chelation treatment outcomes in 24 symptomatic and 24 asymptomatic patients and concluded that clinical improvement was described in almost half of the symptomatic patients while chelation postponed symptom onset by approximately 10 years in asymptomatic patients [153].…”

Section: Neurodegenerations With Brain Iron Accumulation (Nbia) Groupmentioning

confidence: 99%

Cerebral Iron Deposition in Neurodegeneration

et al. 2022

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Disruption of cerebral iron regulation appears to have a role in aging and in the pathogenesis of various neurodegenerative disorders. Possible unfavorable impacts of iron accumulation include reactive oxygen species generation, induction of ferroptosis, and acceleration of inflammatory changes. Whole-brain iron-sensitive magnetic resonance imaging (MRI) techniques allow the examination of macroscopic patterns of brain iron deposits in vivo, while modern analytical methods ex vivo enable the determination of metal-specific content inside individual cell-types, sometimes also within specific cellular compartments. The present review summarizes the whole brain, cellular, and subcellular patterns of iron accumulation in neurodegenerative diseases of genetic and sporadic origin. We also provide an update on mechanisms, biomarkers, and effects of brain iron accumulation in these disorders, focusing on recent publications. In Parkinson’s disease, Friedreich’s disease, and several disorders within the neurodegeneration with brain iron accumulation group, there is a focal siderosis, typically in regions with the most pronounced neuropathological changes. The second group of disorders including multiple sclerosis, Alzheimer’s disease, and amyotrophic lateral sclerosis shows iron accumulation in the globus pallidus, caudate, and putamen, and in specific cortical regions. Yet, other disorders such as aceruloplasminemia, neuroferritinopathy, or Wilson disease manifest with diffuse iron accumulation in the deep gray matter in a pattern comparable to or even more extensive than that observed during normal aging. On the microscopic level, brain iron deposits are present mostly in dystrophic microglia variably accompanied by iron-laden macrophages and in astrocytes, implicating a role of inflammatory changes and blood–brain barrier disturbance in iron accumulation. Options and potential benefits of iron reducing strategies in neurodegeneration are discussed. Future research investigating whether genetic predispositions play a role in brain Fe accumulation is necessary. If confirmed, the prevention of further brain Fe uptake in individuals at risk may be key for preventing neurodegenerative disorders.

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Section: Neurodegenerations With Brain Iron Accumulation (Nbia) Groupmentioning

confidence: 99%

Cerebral Iron Deposition in Neurodegeneration

et al. 2022

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“…The iron chelator deferasirox (DFX) can improve anemia, as observed in patients with transfusional iron overload, such as patients with myelodysplastic syndrome (MDS), 1-4 myeloproliferative neoplasms, 5 aplastic anemia, 2,6 pure red cell aplasia, 7 and the iron metabolism disorder aceruloplasminemia. 8 How DFX improves anemia is unclear. It may increase erythropoietin production, improve iron availability for hematopoietic tissue, 9 and modify the bone marrow (BM) microenvironment by reducing oxidative stress 10,11 and inflammation.…”

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confidence: 99%