Combination Oral Chelation in Adult Patients With Transfusion-dependent Thalassemia and High Iron Burden. Issue 1 (January 2019)

• Record Type: Journal Article
• Authors: Hammond, John; Thompson, Alexis A.; Fogel, Mark A.; Hammond, Katherine; Kokroko, Jolene; Kwiatkowski, Janet L.
• Is Part Of: Journal of pediatric hematology/oncology. Volume 41:Issue 1(2019)
• Publication Date: 2019-01
• Subjects: thalassemia -- iron overload -- deferasirox -- deferiprone; Pediatric hematology -- Periodicals; Tumors in children -- Periodicals; 618.9215
• DOI: 10.1097/MPH.0000000000001269
• Languages: English
• ISSNs: 1077-4114

Abstract

Abstract : An open-label, pilot study was conducted to evaluate deferasirox/deferiprone combination chelation therapy in adult patients with transfusion-dependent thalassemia and severe iron overload. Enrollment proved difficult. Nine patients (median age, 27.4 y; ferritin, 4965 ng/mL; liver iron concentration, 28.5 mg/g dry weight; cardiac T2*, 13.3 ms) received treatment. Two were withdrawn for treatment-related adverse effects. Arthralgia (4 patients) and gastrointestinal symptoms (5 patients) were common; no episodes of neutropenia/agranulocytosis occurred. Adherence difficulties were common. Of 6 patients with 12 to 18 months follow-up, 3 showed improvement in cardiac T2* and 2 in liver iron. Combination oral chelation may be effective but adverse effects and adherence challenges may limit efficacy.