En mann i 50-årene med høyt ferritinnivå og økende kognitiv svikt

Bjørk, Marte Helene; Gjerde, Ivar; Tzoulis, Charalampos; Ulvik, Rune J.; Bindoff, Laurence A.

doi:10.4045/tidsskr.14.1115

Cited by 4 publications

(1 citation statement)

References 17 publications

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“…Honnør til forfatterne som skrev og til redaktøren som så behovet for en ny sjanger! For det andre to kasuistikker i serien Noe å laere av (2,3). Kasuistikkene illustrerer at medisinen går fremover ikke bare gjennom Cochranebevegelsens arbeid, men også som en frukt av akkumulert klinisk erfaring, bedre basalmedisinsk innsikt og ny forståelse av sykdomsmekanismer.…”

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Re: Legehelter – og antihelter

Wyller¹

2015

Tidsskriftet

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Re: Legehelter – og antihelter

Wyller¹

2015

Tidsskriftet

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New view of aceruloplasminemia: Systematic review and meta-analysis tracking dots from onset to disease development and iron-related features

Ketata,

Ellouz

2023

Rare

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Effects of iron chelation therapy on the clinical course of aceruloplasminemia: an analysis of aggregated case reports

Vroegindeweij

Wilson

Langendonk

2020

Orphanet J Rare Dis

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Background: Aceruloplasminemia is a rare genetic iron overload disorder, characterized by progressive neurological manifestations. The effects of iron chelation on neurological outcomes have only been described in case studies, and are inconsistent. Aggregated case reports were analyzed to help delineate the disease-modifying potential of treatment. Methods: Data on clinical manifestations, treatment and neurological outcomes of treatment were collected from three neurologically symptomatic Dutch patients, who received deferiprone with phlebotomy as a new therapeutic approach, and combined with other published cases. Neurological outcomes of treatment were compared between patients starting treatment when neurologically symptomatic and patients without neurological manifestations. Results: Therapeutic approaches for aceruloplasminemia have been described in 48 patients worldwide, including our three patients. Initiation of treatment in a presymptomatic stage of the disease delayed the estimated onset of neurological manifestations by 10 years (median age 61 years, SE 5.0 vs. median age 51 years, SE 0.6, p = 0.001). Although in 11/20 neurologically symptomatic patients neurological manifestations remained stable or improved during treatment, these patients were treated significantly shorter than patients who deteriorated neurologically (median 6 months vs. median 43 months, p = 0.016). Combined iron chelation therapy with deferiprone and phlebotomy for up to 34 months could be safely performed in our patients without symptomatic anemia (2/3), but did not prevent further neurological deterioration. Conclusions: Early initiation of iron chelation therapy seems to postpone the onset of neurological manifestations in aceruloplasminemia. Publication bias and significant differences in duration of treatment should be considered when interpreting reported treatment outcomes in neurologically symptomatic patients. Based on theoretical grounds and the observed long-term safety and tolerability in our study, we recommend iron chelation therapy with deferiprone in combination with phlebotomy for aceruloplasminemia patients without symptomatic anemia.

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En mann i 50-årene med høyt ferritinnivå og økende kognitiv svikt

Cited by 4 publications

References 17 publications

Re: Legehelter – og antihelter

Re: Legehelter – og antihelter

New view of aceruloplasminemia: Systematic review and meta-analysis tracking dots from onset to disease development and iron-related features

Effects of iron chelation therapy on the clinical course of aceruloplasminemia: an analysis of aggregated case reports

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