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Systemic Diseases and the Skin
Published in Ayşe Serap Karadağ, Lawrence Charles Parish, Jordan V. Wang, Roxburgh's Common Skin Diseases, 2022
Jana Kazandjieva, Razvigor Darlenski, Nikolai Tsankov
Management: Phlebotomy is usually performed once or twice weekly to obtain a ferritin level of less than 50 mcg/L. Although chelation is not the most effective treatment method, deferoxamine, deferiprone, and deferasirox are used for some patients.
Overview of Companion Diagnostics (CDx) for Precision Medicine
Published in Il-Jin Kim, Companion Diagnostics (CDx) in Precision Medicine, 2019
Only one imaging assay (Ferriscan) has been approved for screening liver iron concentration for selecting non-transfusion-dependent thalassemia patients for deferasirox treatment (Table 1.1).5 This is the CDx assay approved for a patient test for a disease other than cancer.
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Published in Caroline Ashley, Aileen Dunleavy, John Cunningham, The Renal Drug Handbook, 2018
Caroline Ashley, Aileen Dunleavy, John Cunningham
During clinical trials, increases in serum creatinine of >33% on ≥2 consecutive occasions (sometimes above the upper limit of the normal range) occurred in about 36% of patients. These were dose-dependent. Cases of acute renal failure have been reported following post-marketing use of deferasirox.
A Case of Toxic Epidermal Necrolysis Caused by Deferasirox
Published in Hemoglobin, 2019
Zulaiha Muda, Sin Toun Loh, Begum Sabeera, Ida Shahnaz Othman, Hishamshah Ibrahim
Toxic epidermal necrolysis (TEN) is a rare but serious dermatological emergency characterized by diffuse exfoliation of skin and mucous membranes. It can be complicated with sepsis, renal impairment, and respiratory failure. The average reported mortality rate of TEN is 25.0–35.0%. Drugs are assumed or identified as the main cause of TEN in most cases. Deferasirox (DFX) is widely used as an effective chelation agent for iron overload in thalassemia. To the best of our knowledge, there has not been any reported case of TEN caused by DFX. We report a 14-year-old boy who has transfusion-dependent thalassemia (TDT), treated with DFX FCT (film-coated tablet), who developed TEN within 2 weeks of initiation of the drug. He presented with fever, vomiting, and diarrhea. Four days later, he developed an oral ulcer and pruritic skin lesions that progressed into blister and bullae formation involving 30.0–40.0% of his bodysurface area (BSA). Deferasirox was stopped. His clinical course was complicated with bilateral pseudomembranous conjunctivitis, renal impairment, pneumonitis, and staphylococcus coagulase negative sepsis. His highest SCORe of TEN (SCORTEN) scale was 3. He was nursed in the pediatric intensive care unit, requiring intravenous immunoglobulin, prednisolone, and antibiotics. Successful management TEN is presented and discussed.
Long-term safety and efficacy of deferasirox in patients with myelodysplastic syndrome, aplastic anemia and other rare anemia in Taiwan
Published in Hematology, 2019
Bor-Sheng Ko, Ming-Chih Chang, Tzeon-Jye Chiou, Te-Kau Chang, Yeu-Chin Chen, Sheng-Fung Lin, Cheng-Shyong Chang, Yin-Che Lu, Su-Peng Yeh, Tsai-Yun Chen, Wei-Shou Hwang
During the 3-year observational period, reported AEs were mostly mild (58.0%) or moderate (27.2%) in severity, and only two grade three skin rash events in two patients with MDS resulted in temporary drug discontinuation. Resuming deferasirox with a lower dose and subsequent dose increments led to disease improvement and therefore permanent drug discontinuation was avoided in these patients. The incidence of skin rash with deferasirox among patients with MDS was higher in our study (23.7%) compared with the EPIC study (6.7%), which enrolled mainly Caucasian patients, while the incidence was lower for abdominal pain (5.3% vs. 7.6%) and diarrhea (13.2% vs. 32.6%). Similarly, the higher incidence of skin rash with deferasirox was also observed in a previous study in Taiwanese patients with β-thalassemia [19]. Different AE profile of deferasirox between Taiwanese and Caucasian patients was therefore speculated. Overall death percentage was 29.1%, which seemed higher compared to earlier reports, however, smaller size of the patient population, patients with more comorbidities and longer observation period might have affected this.
Diagnosis, management and response criteria of iron overload in myelodysplastic syndromes (MDS): updated recommendations of the Austrian MDS platform
Published in Expert Review of Hematology, 2018
Peter Valent, Reinhard Stauder, Igor Theurl, Klaus Geissler, Thamer Sliwa, Wolfgang R. Sperr, Peter Bettelheim, Heinz Sill, Michael Pfeilstöcker
With regard to dosage, we recommend to start with a relatively low dose of the drug (360–720 mg/day) and to adjust the dose from there to the clinical response that can be measured by determining serum ferritin levels in the follow-up. In this regard, it is worth noting that the new formulation of the drug (film coated tablets) differs from the older formulation (tablets to dissolve) regarding the available free substance, and that therefore the recommended standard dose also changed (from higher to lower doses). Frequent side effects of deferasirox are gastrointestinal cramping and diarrhea as well as impairment of the kidney function. All other side effects are rare and usually, no cytopenia occurs. In the case of intolerance or insufficient chelation under deferasirox, one of the two alternative chelators, desferoxamine or deferiprone, can be applied. However, these drugs may also produce side effects. For example, deferiprone has been described to cause neutropenia (up to 10% of treated MDS patients), gastrointestinal symptoms, arthropathy, and transient changes in liver enzymes. The major disadvantage of desferoxamine is that it needs to be applied though continuous intravenous or subcutaneous injection which may cause local skin reactions or even infections.