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 Two were withdrawn for treatment-related adverse effects.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
收藏
页码：E47 / E50
页数：4
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