Deferasirox Might Be Effective for Microcytic Anemia and Neurological Symptoms Associated with Aceruloplasminemia: A Case Report and Review of the Literature.
Study Goal
The researchers aimed to evaluate the effects of iron chelation therapy (deferasirox) on anemia and cerebellar symptoms in a patient with aceruloplasminemia, a condition linked to low copper levels.
Results Summary
The study found that deferasirox improved the patient's anemia and cerebellar symptoms, including dysarthria and limb ataxia, suggesting potential benefits for similar cases within a year of treatment.
Population
A 64-year-old man with aceruloplasminemia, refractory microcytic anemia, diabetes mellitus, and low serum copper and ceruloplasmin levels.
Effective Dosage
Not specified
Duration
Less than one year
Interactions
None mentioned
| Intervention | Direction | Endpoint | Population | Dosage | Impact | Claim # |
|---|---|---|---|---|---|---|
Iron chelation therapy with deferasirox | decrease | anemia | 64-year-old man with aceruloplasminemia | - | improved | #1 |
Iron chelation therapy with deferasirox | decrease | cerebellar symptoms | 64-year-old man with aceruloplasminemia | - | improved | #2 |
Iron chelation therapy with deferasirox | decrease | dysarthria | 64-year-old man with aceruloplasminemia | - | improved | #3 |
Iron chelation therapy with deferasirox | decrease | limb ataxia | 64-year-old man with aceruloplasminemia | - | improved | #4 |
deferasirox | decrease | cerebellar symptoms with aceruloplasminemia | patients with aceruloplasminemia | in less than one year | might respond to | #5 |
The patient was a 64-year-old man presented with difficulty in walking, articulation, and swallowing, as well as cognitive impairment. He had refractory microcytic anemia and diabetes mellitus. His serum levels of iron, copper, and ceruloplasmin were low. Magnetic resonance imaging suggested iron deposition in the basal ganglia, thalami, cerebellar dentate nuclei, and cerebral and cerebellar cortices. He was diagnosed with aceruloplasminemia after a ceruloplasmin gene analysis. Iron chelation therapy with deferasirox improved his anemia and cerebellar symptoms, which included dysarthria and limb ataxia. The present study and previous reports indicate that cerebellar symptoms with aceruloplasminemia might respond to deferasirox in less than one year.