Hemojuvelin hemochromatosis receiving iron chelation therapy with deferasirox: improvement of liver disease activity, cardiac and hematological function

Maeda, Takashi; Nakamaki, Tsuyoshi; Saito, Bungo; Nakashima, Hidetoshi; Ariizumi, Hirotsugu; Yanagisawa, Kouji; Hattori, Ai; Tatsumi, Yasuaki; Hayashi, Hisao; Suzuki, Kenshi; Tomoyasu, Shigeru

doi:10.1111/j.1600-0609.2011.01693.x

Cited by 16 publications

(10 citation statements)

References 10 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The lack of serious side effects in the patients studied indicates its safety, and the salutary reduction in all parameters of iron overload (SF, TS, and LIC) to normal or near normal values confirms its efficacy. These findings are similar to previous anecdotal reports and a phase 1/2 dose‐escalation trial of deferasirox for the treatment of iron overload in patients with hereditary hemochromatosis . In the study by Phatak et al .…”

Section: Discussionsupporting

confidence: 91%

“…described hepatotoxicity induced by deferasirox in a patient with hemochromatosis and recommended not using it in a disease that is already known to cause liver damage. Our study and another study, however, showed improved liver function after treatment with deferasirox . Moreover, although SCD and thalassemia are also known to cause liver damage, the use of deferasirox in these diseases was safe .…”

Section: Discussioncontrasting

confidence: 47%

See 1 more Smart Citation

Deferasirox in patients with iron overload secondary to hereditary hemochromatosis: results of a 1‐yr Phase 2 study

Cançado

Melo

Bastos

et al. 2015

European J of Haematology

View full text Add to dashboard Cite

This open-label, prospective, phase 2 study evaluated the safety and efficacy of deferasirox (10 ± 5 mg/kg/d) in patients with hereditary hemochromatosis (HH) and iron overload refractory to or intolerant of phlebotomy. Ten patients were enrolled and all completed the 12-month treatment period. There were significant decreases from baseline to end of study (i.e., 12 months) in median serum ferritin (P < 0.001), mean transferrin saturation (P < 0.05), median liver iron concentration (P < 0.001), and mean alanine aminotransferase (P < 0.05). The median time to achieve serum ferritin reduction ≥50% compared to baseline was 7.53 months. The most common adverse events were mild, transient diarrhea (n = 5) and nausea (n = 2). No patient experienced an increase in serum creatinine that exceeded the upper limit of normal. These data confirm that deferasirox was well tolerated and effective in reducing iron burden in patients with hereditary hemochromatosis and could be a safe alternative to phlebotomy in selected patients.

show abstract

Section: Discussionsupporting

confidence: 91%

Section: Discussioncontrasting

confidence: 47%

Deferasirox in patients with iron overload secondary to hereditary hemochromatosis: results of a 1‐yr Phase 2 study

Cançado

Melo

Bastos

et al. 2015

European J of Haematology

View full text Add to dashboard Cite

show abstract

“…Our study confirms that a small number of Japanese people are affected by iron overload disorders caused by a variety of genotypes (CP, HAMP, HJV, TFR2, and SLC40A1) and that the hepcidin system plays different roles in the conditions caused by each genotype. Regarding the clinical features and genetic backgrounds of these patients, several cases of each condition have already been reported in detail 7,8,19–24 . However, this study is the first to systematically assess the clinicopathological features of Japanese patients with defined genetic backgrounds.…”

Section: Discussionmentioning

confidence: 99%

Clinicopathological study of Japanese patients with genetic iron overload syndromes

Hattori

Miyajima

Tomosugi

et al. 2012

Pathology International

Self Cite

View full text Add to dashboard Cite

In addition to hemochromatosis, aceruloplasminemia and ferroportin disease may be complicated by iron-induced multiple organ damage. Therefore, clinicopathological features should be evaluated in a wider range of genetic iron disorders. This study included 16 Japanese patients with genetic iron overload syndromes. The responsible genes were CP in four, HAMP in one, HJV in three, TFR2 in five, and SLC40A1 in three patients. No phenotype dissociation was observed in patients with the CP, TFR2, or HAMP genotypes. Two of the three patients with the HJV genotype displayed classic hemochromatosis instead of the juvenile type. Patients with the SLC40A1 genotype were affected by mild iron overload (ferroportin A) or severe iron overload (ferroportin B). Transferrin saturation was unusually low in aceruloplasminemia patients. All patients, except those with ferroportin disease, displayed low serum hepcidin-25 levels. Liver pathology showed phenotype-specific changes; isolated parenchymal iron loading in aceruloplasminemia, periportal fibrosis associated with heavy iron overload in both parenchymal and Kupffer cells of ferroportin B, and parenchyma-dominant iron-loading cirrhosis in hemochromatosis. In contrast, diabetes occurred in all phenotypes of aceruloplasminemia, hemochromatosis, and ferroportin disease B. In conclusion, clinicopathological features were partially characterized in Japanese patients with genetic iron overload syndromes.

show abstract

“…In transfusional iron overload, deferasirox has been extensively used at higher dosages (usually 20 to 40 mg/kg/day) according to the amount of transfusional iron loading even in children [7]. At the best of our knowledge there are only two reports on the use of deferasirox in the treatment of iron overload in JH in humans [8,9] and none in children. In a murine model of JH characterized by massive iron overload, deferasirox effectively reduced liver and heart iron at a dosage of 30 -100 mg/kg/day [10].…”

Section: Discussionmentioning

confidence: 99%

Efficacy of deferasirox for the treatment of iron overload in a child affected by Juvenile Hemochromatosis

Masera¹,

Cattoni²,

Decimi³

et al. 2013

CRCM

View full text Add to dashboard Cite

We report the case of a 7 years old girl with Juvenile Hemochromatosis, due to homozygous mutation of HJV, which had increased serum iron indices and liver iron overload in the absence of any clinical sign of disease. Oral iron chelation with low dose deferasirox showed good efficacy and no side effects. The oral iron chelator deferasirox could be a valid option for removing excess iron in early Juvenile Hemochromatosis.

show abstract

Hemojuvelin hemochromatosis receiving iron chelation therapy with deferasirox: improvement of liver disease activity, cardiac and hematological function

Cited by 16 publications

References 10 publications

Deferasirox in patients with iron overload secondary to hereditary hemochromatosis: results of a 1‐yr Phase 2 study

Deferasirox in patients with iron overload secondary to hereditary hemochromatosis: results of a 1‐yr Phase 2 study

Clinicopathological study of Japanese patients with genetic iron overload syndromes

Efficacy of deferasirox for the treatment of iron overload in a child affected by Juvenile Hemochromatosis

Contact Info

Product

Resources

About