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

Research output: Contribution to journalArticle

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 Two were withdrawn for treatment-related adverse effects.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

Fingerprint

Thalassemia
Iron
Chelation Therapy
Agranulocytosis
Iron Overload
Liver
Arthralgia
Ferritins
Neutropenia
Weights and Measures
Therapeutics

Keywords

  • deferasirox
  • deferiprone
  • iron overload
  • thalassemia

ASJC Scopus subject areas

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

Cite this

Hammond, John ; Thompson, Alexis A ; Fogel, Mark A. ; Hammond, Katherine ; Kokroko, Jolene ; Kwiatkowski, Janet L. / Combination Oral Chelation in Adult Patients with Transfusion-dependent Thalassemia and High Iron Burden. In: Journal of pediatric hematology/oncology. 2019 ; Vol. 41, No. 1. pp. E47-E50.
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Combination Oral Chelation in Adult Patients with Transfusion-dependent Thalassemia and High Iron Burden. / Hammond, John; Thompson, Alexis A; Fogel, Mark A.; Hammond, Katherine; Kokroko, Jolene; Kwiatkowski, Janet L.

In: Journal of pediatric hematology/oncology, Vol. 41, No. 1, 01.01.2019, p. E47-E50.

Research output: Contribution to journalArticle

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