Combination Oral Chelation in Adult Patients with Transfusion-dependent Thalassemia and High Iron Burden

John Hammond, Alexis A. Thompson, Mark A. Fogel, Katherine Hammond, Jolene Kokroko, Janet L. Kwiatkowski*

*Corresponding author for this work

Research output: Contribution to journalArticle

1 Scopus citations

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 Two were withdrawn for treatment-related adverse effects.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.

Original languageEnglish (US)
Pages (from-to)E47-E50
JournalJournal of pediatric hematology/oncology
Volume41
Issue number1
DOIs
StatePublished - Jan 1 2019

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Keywords

  • deferasirox
  • deferiprone
  • iron overload
  • thalassemia

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

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