Deferasirox improves hematologic and hepatic function with effective reduction of serum ferritin and liver iron concentration in transfusional iron overload patients with myelodysplastic syndrome or aplastic anemia

Cheong, June Won; Kim, Hyeoung Joon; Lee, Kyoo Hyung; Yoon, Sung Soo; Lee, Jae Hoon; Park, Hee Sook; Kim, Ho Young; Shim, Hyeok; Seong, Chu-Myong; Kim, Chul Soo; Chung, Joo-Seop; Hyun, Myung Soo; Jo, Deog Yeon; Jung, Chul Won; Sohn, Sang Kyun; Yoon, Hwi Joong; Kim, Byung Soo; Joo, Young-Don; Park, Chi Young; Min, Yoo Hong

doi:10.1111/trf.12507

Cited by 45 publications

(34 citation statements)

References 34 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…The efficacy and safety of once-daily deferasirox treatment in reducing or maintaining body iron has been demonstrated in several studies involving large numbers of patients with a variety of transfusion-dependent anemias [13][14][15][16][17][18][19] including subpopulations of patients with rare anemias, such as MDS and AA [13,15,[20][21][22][23][24][25][26][27] . However, studies evaluating oral iron chelation therapy among Japanese patients, in particular, are lacking.…”

mentioning

confidence: 99%

Deferasirox Decreases Liver Iron Concentration in Iron-Overloaded Patients with Myelodysplastic Syndromes, Aplastic Anemia and Other Rare Anemias

et al. 2015

View full text Add to dashboard Cite

Iron overload in transfusion-dependent patients with rare anemias can be managed with chelation therapy. This study evaluated deferasirox efficacy and safety in patients with myelodysplastic syndromes (MDS), aplastic anemia (AA) or other rare anemias. A 1-year, open-label, multicenter, single-arm, phase II trial was performed with deferasirox (10-40 mg/kg/day, based on transfusion frequency and therapeutic goals), including an optional 1-year extension. The primary end point was a change in liver iron concentration (LIC) after 1 year. Secondary end points included changes in efficacy and safety parameters (including ophthalmologic assessments) overall as well as in a Japanese subpopulation. Overall, 102 patients (42 with MDS, 29 with AA and 31 with other rare anemias) were enrolled; 57 continued into the extension. Mean absolute change in LIC was -10.9 mg Fe/g dry weight (d.w.) after 1 year (baseline: 24.5 mg Fe/g d.w.) and -13.5 mg Fe/g d.w. after 2 years. The most common drug-related adverse event was increased serum creatinine (23.5%), predominantly in MDS patients. Four patients had suspected drug-related ophthalmologic abnormalities. Outcomes in Japanese patients were generally consistent with the overall population. Results confirm deferasirox efficacy in patients with rare anemias, including a Japanese subpopulation. The safety profile was consistent with previous studies and ophthalmologic parameters generally agreed with baseline values (EUDRACT 2006-003337-32).

show abstract

mentioning

confidence: 99%

Deferasirox Decreases Liver Iron Concentration in Iron-Overloaded Patients with Myelodysplastic Syndromes, Aplastic Anemia and Other Rare Anemias

et al. 2015

View full text Add to dashboard Cite

show abstract

“…Measurement of serum ferritin levels is convenient, inexpensive and also widely used as a marker of iron overload in transfusional iron overload settings. A significant positive correlation between serum ferritin and LIC has been established in small observational studies across a variety of patients with various underlying diseases, including patients with the main NTDT subtypes (Pakbaz et al, 2007;Taher et al, 2008;Lal et al, 2011;Cheong et al, 2014;Eghbali et al, 2014). However, it was also noted that the ratio of serum ferritin to LIC was lower than that observed in regularly transfused patients (Origa et al, 2007;Pakbaz et al, 2007;Taher et al, 2008), suggesting that further investigation of this relationship is needed before serum ferritin can be widely used in clinical practice to monitor and treat iron overload in NTDT patients.…”

Section: Discussionmentioning

confidence: 95%

Defining serum ferritin thresholds to predict clinically relevant liver iron concentrations for guiding deferasirox therapy when MRI is unavailable in patients with non‐transfusion‐dependent thalassaemia

et al. 2014

View full text Add to dashboard Cite

SummaryLiver iron concentration (LIC) assessment by magnetic resonance imaging (MRI) remains the gold standard to diagnose iron overload and guide iron chelation therapy in patients with non‐transfusion‐dependent thalassaemia (NTDT). However, limited access to MRI technology and expertise worldwide makes it practical to also use serum ferritin assessments. The THALASSA (assessment of Exjade® in non‐transfusion‐dependent THALASSemiA patients) study assessed the efficacy and safety of deferasirox in iron‐overloaded NTDT patients and provided a large data set to allow exploration of the relationship between LIC and serum ferritin. Using data from screened patients and those treated with deferasirox for up to 2 years, we identified clinically relevant serum ferritin thresholds (for when MRI is unavailable) for the initiation of chelation therapy (>800 μg/l), as well as thresholds to guide chelator dose interruption (<300 μg/l) and dose escalation (>2000 μg/l). (clinicaltrials.gov identifier: NCT00873041).

show abstract

“…Several case descriptions 10,11 and published cohorts [12][13][14][15][16][17][18] have reported improved erythropoiesis and a decreased transfusion requirement in mds patients treated with iron chelation.…”

Section: Discussionmentioning

confidence: 99%

“…A recent study reported improved erythropoiesis in a cohort of mds patients (n = 43) and aplastic anemia patients (n = 53) treated with deferasirox over a 1-year period 15 . The mean hemoglobin level for the group had increased at the end of the study compared with baseline (7.62 ± 2.65 g/dL vs. 6.26 ± 0.94 g/dL, p = 0.002).…”

Section: Discussionmentioning

confidence: 99%

A Case of Transfusion Independence in a Patient with Myelodysplastic Syndrome Using Deferasirox, Sustained for Two Years after Stopping Therapy

Sanford

Hsia

2015

Current Oncology

View full text Add to dashboard Cite

Patients with myelodysplastic syndrome (mds) experience clinical complications related to progressive marrow failure and have an increased risk of developing acute myeloid leukemia. Frequent red blood cell transfusion can lead to clinical iron overload and is associated with decreased survival in mds patients. Iron chelation therapy reduces markers of iron overload and prevents end-organ damage.Here, we present the case of a patient with lowrisk mds with transfusional iron overload. He was treated for 2 years with an oral iron chelator, deferasirox, and after 12 months of treatment, he experienced a hemoglobin increase of more than 50 g/L, becoming transfusion-independent. He has remained transfusion-independent, with a normal hemoglobin level, for more than 2 years since stopping chelation therapy. Hematologic and erythroid responses have previously been reported in mds patients treated with iron chelation. The durability of our patient’sresponse suggests that iron chelation might alter the natural history of mds in some patients.

show abstract

Deferasirox improves hematologic and hepatic function with effective reduction of serum ferritin and liver iron concentration in transfusional iron overload patients with myelodysplastic syndrome or aplastic anemia

Cited by 45 publications

References 34 publications

Deferasirox Decreases Liver Iron Concentration in Iron-Overloaded Patients with Myelodysplastic Syndromes, Aplastic Anemia and Other Rare Anemias

Deferasirox Decreases Liver Iron Concentration in Iron-Overloaded Patients with Myelodysplastic Syndromes, Aplastic Anemia and Other Rare Anemias

Defining serum ferritin thresholds to predict clinically relevant liver iron concentrations for guiding deferasirox therapy when MRI is unavailable in patients with non‐transfusion‐dependent thalassaemia

A Case of Transfusion Independence in a Patient with Myelodysplastic Syndrome Using Deferasirox, Sustained for Two Years after Stopping Therapy

Contact Info

Product

Resources

About