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 Two were withdrawn for treatment-related adverse effects.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.
*Division of Hematology
§Division of Cardiology, Children’s Hospital of Philadelphia
∥Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA
†Division of Hematology/Oncology, Ann & Robert H. Lurie Children’s Hospital of Chicago
‡Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL
Supported by a grant from the Cooley’s Anemia Foundation.
J.L.K. has consulted for Agios and Ionis Pharmaceuticals and is an investigator in studies sponsored by Apopharma, Novartis Pharmaceuticals, and bluebird bio. A.A.T. has consulted for Novartis, Celgene, and bluebird bio and receives research funding from Amgen, Baxalta, bluebird bio, Celgene, Eli Lilly, Mast, and Novartis. The remaining authors declare no conflict of interest.
Reprints: Janet L. Kwiatkowski, MD, MSCE, Children’s Hospital of Philadelphia, Division of Hematology, 3401 Civic Center Boulevard, Philadelphia, PA 19104 (e-mail: firstname.lastname@example.org).
Received August 14, 2017
Accepted June 14, 2018