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

doi:10.1097/MPH.0000000000001269

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

Pediatrics

Research output: Contribution to journal › Article › peer-review

8 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 Two were withdrawn for treatment-related adverse effects.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 language	English (US)
Pages (from-to)	E47-E50
Journal	Journal of pediatric hematology/oncology
Volume	41
Issue number	1
DOIs	https://doi.org/10.1097/MPH.0000000000001269
State	Published - Jan 1 2019

Keywords

deferasirox
deferiprone
iron overload
thalassemia

ASJC Scopus subject areas

Pediatrics, Perinatology, and Child Health
Hematology
Oncology

Access to Document

10.1097/MPH.0000000000001269

Cite this

@article{047e5d8dd23347c883c051186c124fb0,

title = "Combination Oral Chelation in Adult Patients with Transfusion-dependent Thalassemia and High Iron Burden",

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 Two were withdrawn for treatment-related adverse effects.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.",

keywords = "deferasirox, deferiprone, iron overload, thalassemia",

author = "John Hammond and Thompson, {Alexis A.} and Fogel, {Mark A.} and Katherine Hammond and Jolene Kokroko and Kwiatkowski, {Janet L.}",

note = "Funding Information: Supported by a grant from the Cooley{\textquoteright}s Anemia Foundation. Funding Information: J.L.K. has consulted for Agios and Ionis Pharmaceuticals and is an investigator in studies sponsored by Apopharma, Novartis Phar-maceuticals, 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. Publisher Copyright: {\textcopyright} 2018 Wolters Kluwer Health, Inc. All rights reserved.",

year = "2019",

month = jan,

day = "1",

doi = "10.1097/MPH.0000000000001269",

language = "English (US)",

volume = "41",

pages = "E47--E50",

journal = "Journal of pediatric hematology/oncology",

issn = "1077-4114",

publisher = "Lippincott Williams and Wilkins",

number = "1",

}

TY - JOUR

T1 - Combination Oral Chelation in Adult Patients with Transfusion-dependent Thalassemia and High Iron Burden

AU - Hammond, John

AU - Thompson, Alexis A.

AU - Fogel, Mark A.

AU - Hammond, Katherine

AU - Kokroko, Jolene

AU - Kwiatkowski, Janet L.

N1 - Funding Information: Supported by a grant from the Cooley’s Anemia Foundation. Funding Information: J.L.K. has consulted for Agios and Ionis Pharmaceuticals and is an investigator in studies sponsored by Apopharma, Novartis Phar-maceuticals, 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. Publisher Copyright: © 2018 Wolters Kluwer Health, Inc. All rights reserved.

PY - 2019/1/1

Y1 - 2019/1/1

N2 - 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.

AB - 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.

KW - deferasirox

KW - deferiprone

KW - iron overload

KW - thalassemia

UR - http://www.scopus.com/inward/record.url?scp=85052814277&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85052814277&partnerID=8YFLogxK

U2 - 10.1097/MPH.0000000000001269

DO - 10.1097/MPH.0000000000001269

M3 - Article

C2 - 30080754

AN - SCOPUS:85052814277

SN - 1077-4114

VL - 41

SP - E47-E50

JO - Journal of pediatric hematology/oncology

JF - Journal of pediatric hematology/oncology

IS - 1

ER -

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

Abstract

Keywords

ASJC Scopus subject areas

Access to Document

Other files and links

Fingerprint

Cite this