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

被引：9

作者：

Hammond, John ^{[1
]}

Thompson, Alexis A. ^{[4
,5
]}

Fogel, Mark A. ^{[2
]}

Hammond, Katherine ^{[4
]}

Kokroko, Jolene ^{[1
]}

Kwiatkowski, Janet L. ^{[1
,3
]}

机构：

[1] Childrens Hosp Philadelphia, Div Hematol, 3401 Civ Ctr Blvd, Philadelphia, PA 19104 USA

[2] Childrens Hosp Philadelphia, Div Cardiol, 34th St & Civic Ctr Blvd, Philadelphia, PA 19104 USA

[3] Univ Penn, Dept Pediat, Perelman Sch Med, Philadelphia, PA 19104 USA

[4] Ann & Robert H Lurie Childrens Hosp Chicago, Div Hematol Oncol, Chicago, IL 60611 USA

[5] Northwestern Univ, Dept Pediat, Feinberg Sch Med, Chicago, IL 60611 USA

来源：

JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY | 2019年 / 41卷 / 01期

关键词：

thalassemia; iron overload; deferasirox; deferiprone; MYOCARDIAL IRON; DEFERIPRONE THERAPY; DEFERASIROX; SAFETY; DEFEROXAMINE; TRIAL; RISK;

D O I：

10.1097/MPH.0000000000001269

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

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 Two were withdrawn for treatment-related adverse effects.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.

引用

页码：E47 / E50

页数：4

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