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地拉罗司治疗输血依赖型骨髓增生异常综合征患者的心脏效应:TELESTO 研究。

Cardiac effects of deferasirox in transfusion-dependent patients with myelodysplastic syndromes: TELESTO study.

机构信息

Cardiovascular Disease Unit, IRCCS Ospedale Policlinico San Martino, Genova, Italy.

School of Medicine, Ruijin Hospital, Shanghai Jiao Tong University, Shanghai, China.

出版信息

Br J Haematol. 2024 May;204(5):2049-2056. doi: 10.1111/bjh.19316. Epub 2024 Feb 11.

DOI:10.1111/bjh.19316
PMID:38343073
Abstract

Iron overload from repeated transfusions has a negative impact on cardiac function, and iron chelation therapy may help prevent cardiac dysfunction in transfusion-dependent patients with myelodysplastic syndromes (MDS). TELESTO (NCT00940602) was a prospective, placebo-controlled, randomised study to evaluate the iron chelator deferasirox in patients with low- or intermediate-1-risk MDS and iron overload. Echocardiographic parameters were collected at screening and during treatment. Patients receiving deferasirox experienced a significant decrease in the composite risk of hospitalisation for congestive heart failure (CHF) or worsening of cardiac function (HR = 0.23; 95% CI: 0.05, 0.99; nominal p = 0.0322) versus placebo. No significant differences between the arms were found in left ventricular ejection fraction, ventricular diameter and mass or pulmonary artery pressure. The absolute number of events was low, but the enrolled patients were younger than average for patients with MDS, with no serious cardiac comorbidities and a modest cardiovascular risk profile. These results support the effectiveness of deferasirox in preventing cardiac damage caused by iron overload in this patient population. Identification of patients developing CHF is challenging due to the lack of distinctive echocardiographic features. The treatment of iron overload may be important to prevent cardiac dysfunction in these patients, even those with moderate CHF risk.

摘要

反复输血导致的铁过载对心脏功能有负面影响,铁螯合疗法可能有助于预防骨髓增生异常综合征(MDS)依赖输血患者的心脏功能障碍。TELESTO(NCT00940602)是一项前瞻性、安慰剂对照、随机研究,旨在评估铁螯合剂地拉罗司在低危或中危-1 风险 MDS 伴铁过载患者中的疗效。在筛选和治疗期间收集了超声心动图参数。接受地拉罗司治疗的患者因充血性心力衰竭(CHF)或心脏功能恶化而住院的复合风险显著降低(HR=0.23;95%CI:0.05,0.99;名义 p=0.0322),与安慰剂相比。左心室射血分数、心室直径和质量或肺动脉压在两组之间无显著差异。事件的绝对数量较低,但纳入的患者比 MDS 患者的平均年龄小,没有严重的心脏合并症和中等心血管风险特征。这些结果支持地拉罗司在预防该患者人群中铁过载引起的心脏损伤方面的有效性。由于缺乏独特的超声心动图特征,识别发生 CHF 的患者具有挑战性。即使是中危 CHF 风险的患者,治疗铁过载对于预防心脏功能障碍也可能很重要。

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