Early Deferasirox Treatment in a Patient with Myelodysplastic Syndrome Results in a Long-Term Reduction in Transfusion Requirements

Capalbo, Silvana; Spinosa, Giuseppina; Franzese, Maria Grazia; Palumbo, Gaetano

doi:10.1159/000209206

Cited by 24 publications

(18 citation statements)

References 10 publications

(3 reference statements)

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“…[18][19][20][21][22]25 Interestingly, in one case, improvements in transfusional requirements and hemoglobin levels observed after 3 months of deferasirox treatment were reversed following deferasirox interruption, but regained when deferasirox was resumed. 25 The IWG 2000 criteria, which classify hematologic responses as major or minor depending on the extent of the improvement, 2 were used in a recent retrospective analysis of eight transfused patients (seven patients with MDS, one patient with myelofibrosis) treated with deferasirox (seven patients) and deferoxamine (one patient).…”

Section: Discussionmentioning

confidence: 99%

“…[6][7][8][9][10][11][12][13][14][15] In addition to reports of reduction in iron burden, 16,17 a number of recently published case reports and studies have reported improvements in hematologic parameters and transfusion requirements during iron chelation therapy with deferasirox. [17][18][19][20][21][22][23][24][25][26] There is also limited evidence of hematologic improvement in patients with MDS treated with deferoxamine, 27,28 although the exact mechanism of the hematologic response to iron chelators is unknown.…”

Section: Introductionmentioning

confidence: 99%

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Hematologic responses to deferasirox therapy in transfusion-dependent patients with myelodysplastic syndromes

et al. 2012

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BackgroundReductions in transfusion requirements/improvements in hematologic parameters have been associated with iron chelation therapy in transfusion-dependent patients, including those with myelodysplastic syndromes; data on these reductions/improvements have been limited to case reports and small studies. Design and MethodsTo explore this observation in a large population of patients, we report a post-hoc analysis evaluating hematologic response to deferasirox in a cohort of iron-overloaded patients with myelodysplastic syndromes enrolled in the Evaluation of Patients' Iron Chelation with Exjade ® (EPIC) study using International Working Group 2006 criteria. ResultsTwo-hundred and forty-seven, 100 and 50 patients without concomitant medication for myelodysplastic syndromes were eligible for analysis of erythroid, platelet and neutrophil responses, respectively. Erythroid, platelet and neutrophil responses were observed in 21.5% (53/247), 13.0% (13/100) and 22.0% (11/50) of the patients after a median of 109, 169 and 226 days, respectively. Median serum ferritin reductions were greater in hematologic responders compared with non-responders at end of study, although these differences were not statistically significant. A reduction in labile plasma iron to less than 0.4 μmol/L was observed from week 12 onwards; this change did not differ between hematologic responders and non-responders. ConclusionsThis analysis suggests that deferasirox treatment for up to 1 year could lead to improvement in hematologic parameters in some patients with myelodysplastic syndromes.Key words: myelodysplastic syndromes, deferasirox, iron overload, iron chelation therapy, hematologic response. Haematologica 2012;97(9):1364-1371. doi:10.3324/haematol.2011 This is an open-access paper. Citation: Gattermann N, Finelli C, Della Porta M, Fenaux P, Stadler M, Guerci-Bresler A, Schmid M, Taylor K, Vassilieff D, Habr D, Marcellari A, Roubert B, and Rose C. Hematologic responses to deferasirox therapy in transfusion-dependent patients with myelodysplastic syndromes. Hematologic responses to deferasirox therapy in transfusion-dependent patients with myelodysplastic syndromes

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Hematologic responses to deferasirox therapy in transfusion-dependent patients with myelodysplastic syndromes

et al. 2012

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“…Although reports are emerging on hematologic improvements associated with iron chelation therapy in patients with myelodysplastic syndromes, 8,[15][16][17][18][19][20][21] there are only a few small studies reporting such improvements in patients with AA. [7][8][9][10] This post hoc analysis of data from the EPIC trial presented here indicates that in addition to reducing iron burden, treatment with deferasirox for 1 year in patients with AA is associated with improvements in hematologic parameters, whether administered concomitantly with immunosuppressive therapy or not.…”

Section: Discussionmentioning

confidence: 99%

“…[8][9][10] These findings add to the evidence in patients with AA and other bone marrow failure conditions, such as myelodysplastic syndromes. [15][16][17][18][19][20][21] There are some limitations to these post hoc analyses. Since patients had been diagnosed with AA for a considerable time prior to the study (as demonstrated by a mean transfusion duration of approximately 5 years), details of the methodology used for diagnosis is not discussed in this paper.…”

Section: Discussionmentioning

confidence: 99%

Hematologic responses in patients with aplastic anemia treated with deferasirox: a post hoc analysis from the EPIC study

et al. 2013

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“…99 Other studies in deferasiroxchelated MDS patients also observe reduced RBC transfusion requirements. [100][101][102] In one study, 16% of patients achieved transfusion independence by 12 months with a median hemoglobin of 8 g. In this study, all patients reduced RBC transfusion frequency by 67% after 12 months of treatment. 85 Most recently, chelation with deferasirox demonstrated a 1.5-to 1.8-g/dL increase in hemoglobin despite a decrease in RBC transfusion requirements in patients with low-risk MDS.…”

Section: Iron Chelation and Improved Erythropoiesis In Mdsmentioning

confidence: 56%

Impact of iron overload and potential benefit from iron chelation in low-risk myelodysplastic syndrome

Shenoy

Vallumsetla

Rachmilewitz

et al. 2014

Blood

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Myelodysplastic syndromes (MDSs) are a group of heterogeneous clonal bone marrow disorders characterized by ineffective hematopoiesis, peripheral blood cytopenias, and potential for malignant transformation. Lower/intermediate-risk MDSs are associated with longer survival and high red blood cell (RBC) transfusion requirements resulting in secondary iron overload. Recent data suggest that markers of iron overload portend a relatively poor prognosis, and retrospective analysis demonstrates that iron chelation therapy is associated with prolonged survival in transfusion-dependent MDS patients. New data provide concrete evidence of iron’s adverse effects on erythroid precursors in vitro and in vivo. Renewed interest in the iron field was heralded by the discovery of hepcidin, the main serum peptide hormone negative regulator of body iron. Evidence from β-thalassemia suggests that regulation of hepcidin by erythropoiesis dominates regulation by iron. Because iron overload develops in some MDS patients who do not require RBC transfusions, the suppressive effect of ineffective erythropoiesis on hepcidin may also play a role in iron overload. We anticipate that additional novel tools for measuring iron overload and a molecular-mechanism–driven description of MDS subtypes will provide a deeper understanding of how iron metabolism and erythropoiesis intersect in MDSs and improve clinical management of this patient population.

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Early Deferasirox Treatment in a Patient with Myelodysplastic Syndrome Results in a Long-Term Reduction in Transfusion Requirements

Cited by 24 publications

References 10 publications

Hematologic responses to deferasirox therapy in transfusion-dependent patients with myelodysplastic syndromes

Hematologic responses to deferasirox therapy in transfusion-dependent patients with myelodysplastic syndromes

Hematologic responses in patients with aplastic anemia treated with deferasirox: a post hoc analysis from the EPIC study

Impact of iron overload and potential benefit from iron chelation in low-risk myelodysplastic syndrome

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