Kim Il-Hwan, Moon Joon-Ho, Lim Sung-Nam, Sohn Sang-Kyun, Kim Hoon-Gu, Lee Gyeong-Won, Kim Yang-Soo, Lee Ho-Sup, Kwon Ki-Young, Kim Sung-Hyun, Park Kyung-Tae, Chung Joo-Seop, Lee Won-Sik, Lee Sang-Min, Hyun Myung-Soo, Kim Hawk, Ryoo Hun-Mo, Bae Sung-Hwa, Joo Young-Don
Department of Hemato-Oncology, Inje University Haeundae Paik Hospital, Busan.
Department of Hematology/Oncology, Kyungpook National University Hospital, Daegu.
Transfusion. 2015 Jul;55(7):1613-20. doi: 10.1111/trf.13036. Epub 2015 Mar 11.
Patients receiving red blood cell (RBC) transfusions are at risk of iron overload, which can cause significant organ damage and is an important cause of morbidity and mortality.
This study was an open-label, single-arm, prospective clinical study to evaluate the efficacy and safety of deferasirox (DFX) in patients with aplastic anemia (AA), myelodysplastic syndrome (MDS), or acute myeloid leukemia (AML). Patients with serum ferritin levels of at least 1000 ng/mL and ongoing transfusion requirements were enrolled. DFX was administered for up to 1 year. A total of 100 patients were enrolled.
Serum ferritin levels decreased significantly following treatment (from 2000 to 1650 ng/mL, p = 0.004). The median absolute reduction in serum ferritin levels was -65 ng/mL in AA (p = 0.037), -647 ng/mL in lower-risk MDS (MDS-LR; p = 0.007), and -552 ng/mL in higher-risk MDS (MDS-HR)/AML (p = 0.482). Mean labile plasma iron (LPI) levels decreased from 0.24 μmol/L at baseline to 0.03 μmol/L at 1 year in all patients (p = 0.036). The mean LPI reduction in each group was -0.17 μmol/L in AA, -0.21 μmol/L in MDS-LR, and -0.30 μmol/L in MDS-HR/AML. Gastrointestinal disorders were commonly observed among groups (16.0%). DFX was temporarily skipped for adverse events in seven patients (7.0%) and was permanently discontinued in 11 patients (11.0%).
DFX reduced serum ferritin and LPI levels in patients with transfusional iron overload. Despite the relatively high percentage of gastrointestinal side effects, DFX was tolerable in all subgroups.
接受红细胞（RBC）输血的患者存在铁过载风险，铁过载可导致严重器官损害，是发病和死亡的重要原因。
本研究为开放标签、单臂、前瞻性临床研究，旨在评估地拉罗司（DFX）对再生障碍性贫血（AA）、骨髓增生异常综合征（MDS）或急性髓系白血病（AML）患者的疗效和安全性。纳入血清铁蛋白水平至少为1000 ng/mL且有持续输血需求的患者。DFX给药长达1年。共纳入100例患者。
治疗后血清铁蛋白水平显著降低（从2000 ng/mL降至1650 ng/mL，p = 0.004）。AA患者血清铁蛋白水平的中位绝对降低值为-65 ng/mL（p = 0.037），低危MDS（MDS-LR）患者为-647 ng/mL（p = 0.007），高危MDS（MDS-HR）/AML患者为-552 ng/mL（p = 0.482）。所有患者的平均不稳定血浆铁（LPI）水平从基线时的0.24 μmol/L降至1年时的0.03 μmol/L（p = 0.036）。每组的平均LPI降低值在AA患者中为-0.17 μmol/L，MDS-LR患者中为-0.21 μmol/L，MDS-HR/AML患者中为-0.30 μmol/L。各组中常见胃肠道疾病（16.0%）。7例患者（7.0%）因不良事件暂时停用DFX，11例患者（11.0%）永久停药。
DFX降低了输血性铁过载患者的血清铁蛋白和LPI水平。尽管胃肠道副作用发生率相对较高，但DFX在所有亚组中均可耐受。
