地中海贫血患者肝脏T2*、血清铁蛋白预测心肌铁过载价值研究

目的定量评估地中海贫血(thalassemia,TM)患者心肌和肝脏铁沉积,探讨肝脏T2*、血清铁蛋白(serum ferritin,SF)预测心铁过载价值。材料与方法应用3.0 T磁共振梯度多回波序列扫描基因确诊并满足要求的地中海贫血患者113例,测量其心肌和肝脏T2*值,所有受试者扫描前后7 d、间隔1 w实验室两次检测血清铁蛋白。对心肌T2*、肝脏T2*、SF分度诊断并应用秩相关分析3者间的关系,采用受试者工作特征曲线(receiver operating characteristic curve,ROC曲线),评价肝脏T2*0.70 ms、SF2500μg/L时预测心铁过载及SF300μg/L预测肝铁过载诊断效能及最佳阈值。结果 113例TM患者的铁沉积分度诊断如下:心肌正常94例,轻度6例,中度10例,重度3例;肝脏正常13例,轻度31例,中度29例,重度25例,极重度15例;血清铁蛋白正常1例,轻度48例,中度17例,重度35例,极重度12例。秩相关分析显示心肌T2*-肝脏T2*(r_s=0.267,P=0.004)、心肌T2*-SF(r_s=-0.63,P=0.000)、肝脏T2*-SF(r_s=-0.641,P=0.000)三者间轻中度相关但无规律性。以心肌T2*10 ms诊断心铁沉积异常,肝脏T2*0.70 ms、SF2500μg/L预测心铁过载准确性分别为0.788和0.833,敏感度分别为80.0%、73.3%,特异性分别为70.4%、63.3%;以肝脏T2*3.57 ms诊断肝铁过载,SF300μg/L预测肝铁过载ROC曲线下面积为0.719,敏感度为94.0%,特异性为15.4%。结论在一定范围内,MRI T2*扫描可直接定量体内器官铁沉积,心铁过载、肝铁沉积、SF三者间轻中度相关但无规律性;以肝脏T2*、血清铁蛋白预测心肌铁异常价值较低,而血清铁蛋白预测肝铁过载尚不可靠。     

地拉罗司与去铁胺治疗重型β-地中海贫血铁过载患儿的对比研究

目的探讨地拉罗司与去铁胺治疗重型β-地中海贫血铁过载患儿的临床疗效。方法收集重型β-地中海贫血铁过载患儿47例,按照不同治疗方案分为两组:观察组26例给予地拉罗司治疗,对照组21例给予去铁胺治疗。治疗12个月后比较两组患儿的治疗效果、血清铁蛋白(SF)、不良反应以及心脏、肝脏磁共振成像(MRI)T2~*检查结果。结果治疗12个月后,两组总有效率比较差异无统计学意义(P0.05);观察组总控制率明显高于对照组,差异有统计学意义(P0.05);观察组心脏、肝脏T2~*值明显高于对照组,差异有统计学意义(P0.05);观察组在治疗12个月后SF水平明显低于对照组,差异有统计学意义(P0.05);观察组不良反应发生率明显低于对照组,差异有统计学意义(P0.05)。结论地拉罗司治疗重型β-地中海贫血铁过载的近期效果明显优于去铁胺,明显降低了SF水平,尤其对肝脏去铁效果更为明显,且减少了不良反应的发生,值得临床重视。     

前瞻性3.0TMRI梯度多回波序列参数优化可行性研究

目的探讨缩短首回波(Minimum TE,TEmin)及扫描时间(Scan Time,ST)的3.0 T梯度多回波序列参数优化可行性。材料与方法在3.0 T扫描仪上,优化梯度多回波序列参数作为研究,与常规序列对照,分别扫描MR专用水模(含0~3.2 m M氯化锰盐酸混合液小瓶15只)、45例志愿者肝脏(包括重度铁沉积10例和非重度铁沉积35例),记录两序列TEmin和ST并由接受过良好培训的影像医师利用专业软件测算水模、肝T2*或R2*值。应用配对样本t检验或两相关样本非参数检验分析组间T2*值、TEmin和ST差异性,P0.05为差异有统计学意义;采用两变量相关分析与曲线拟合探讨水模R2*与其浓度相关性。结果 (1)水模、志愿者肝扫描两序列TEmin和ST组间差异有统计学意义(P=0.000),研究序列TEmin和ST分别为(0.62±0.02)ms、(9.3±0.8)s,与对照序列相比分别减少约53.0%、45.0%;(2)水模或志愿者肝扫描两序列间T2*差异在非重度铁沉积组无统计学意义(P水模/肝=0.814/0.201),而在重度铁沉积组有统计学差异(P=0.001),研究序列对重度铁沉积肝T2*检测可信度较高;(3)研究序列、重复及次月扫描水模各小瓶T2*值三组间差异无统计学意义(P=0.584),水模R2*与浓度(Ph C)高度线性相关(r=0.986,P=0.000),通过直线回归分析,得到Ph C与R2*拟合直线斜率和截距分别为7.008,0.036。结论在一定铁沉积浓度范围内,3.0 T MRI梯度多回波优化序列对活体肝扫描具有可行性,并提高重度铁过载检测可信度。     

输血相关铁过载评估及联合疗法与单一疗法比较研究

目的探讨去铁酮(DFP)、去铁胺(DFO)联合方案对比单一疗法中DFP与DFO的疗效。方法收集2011年1月～2016年12月收治地中海贫血患者164例,评估接受联合方案(DFO+DFP,n=51)和DFP (n=39)或DFO (n=74)病患的疗效。心肌、肝铁含量的评估采用MRI T2*时间测量。结果三种方案铁摄取的差异无统计学意义(F=0.207,P=0.819),铁排泄的差异有统计学意义(F=8.196,P=0.019)。三种方案随访中铁蛋白水平与治疗前比较,均有明显下降。联合方案在治疗后1个月、2个月、3个月的血清铁蛋白的平均值下降最明显,差异有统计学意义(F=5.5 27,P=0.044),而在后期三组间下降的差异无统计学意义(F=1.492,P=0.298)。三种方案对心脏功能比较并无明显差异。另外,三组间在心肌T2*(P=0.012)及肝T2*(P=0.019)方面的差异有统计学意义。结论地中海贫血患者中,联合方案在清除铁超载方面优于DFO或DFP单一疗法,联合治疗并没有显示对心脏功能的额外影响。     

重型β地中海贫血肝脏铁沉积的MRI与病理学相关分析

目的 通过与组织病理学对照,探讨MRI评价重型β地中海贫血肝脏铁沉积的价值.方法 对35例重型β地中海贫血患者行肝脏、脾脏MR检查并进行肝脏穿刺活检,在TSE T2WI上测量肝脏/肌肉、脾脏/肌肉信号强度比,与血清铁蛋白、肝穿刺活检中的肝含铁血黄素细胞百分比之间进行相关性分析.结果 35例地中海贫血的肝/肌信号强度为(0.53±0.24),脾/肌信号强度比为(2.47±1.71),血清铁蛋白为(2912.77±1646.01)μg/L,肝脏含铁血黄素细胞百分比为(61.57%±18.26%,中位数60.00%).肝/肌信号强度比与血清铁蛋白、肝含铁血黄素细胞百分比均呈负相关(r＝-0.38,-0.44,P均<0.05),脾/肌信号强度比与血清铁蛋白负相关(r＝-0.41,P=0.03),但脾/肌信号强度比、血清铁蛋白与含铁血黄素细胞百分比均无相关性.结论 MRI测量肝脏信号强度比可作为无创伤性动态评估肝脏铁负荷的一个补充方法.     

肝硬化患者肝脏R2值与血清铁蛋白含量的相关性研究

目的：应用磁共振多回波T2*扫描定量计算肝硬化患者肝脏R2*值,并与血清铁蛋白含量进行相关性分析。材料和方法：对32例健康人和42例临床诊断为乙型肝炎后肝硬化和酒精性肝硬化患者行磁共振多回波T2*序列扫描,并采集静脉血样本测量血清铁蛋白浓度。应用SPIN软件分别测量健康人和肝硬化患者肝脏右叶前段和右叶后段R2*值,取其平均值与血清铁蛋白浓度进行Pearson相关性分析。结果：32例健康人和42例肝硬化患者肝脏R2*值分别为（67.02±12.32） Hz和（85.30±28.48） Hz,经两独立样本t检验,显示二者有显著差异,t=3.335,P=0.001。42例肝硬化患者血清铁蛋白浓度为（165.5±104.4） ng/mL（均值±标准差）,R2*值与血清铁蛋白浓度的相关性分别为r=0.710,P=0.000。结论：肝硬化患者肝脏R2*值明显高于健康人,证明肝硬化患者肝脏铁浓度明显高于健康人。肝硬化患者肝脏R2*值与血清铁蛋白浓度有很好的相关性,证明R2*可以用于无创活体定量测量乙型肝炎后肝硬化和酒精性肝硬化患者肝脏内铁浓度。     

轻型β地中海贫血人群HbA2水平与缺铁水平的相关性分析

目的探讨轻型β地中海贫血人群的血红蛋白A2(HbA2)水平与缺铁水平的相关性。方法将研究对象分为健康人群组(n=504)、缺铁性贫血组(n=126)、轻型β地中海贫血组(n=107)和轻型β地中海贫血合并缺铁性贫血组(n=84),分析各组的HbA2水平和缺铁水平,并使用Pearson相关性分析探讨两者的相关性。结果非地中海贫血组中的缺铁人群与不缺铁人群的HbA2水平差异无统计学意义(P=0.525);但血清铁水平差异有统计学意义(P0.001)。地中海贫血组中缺铁人群与不缺铁人群的HbA2水平差异也无统计学意义(P=0.764),但血清铁水平差异显著(P0.001)。各研究组男女之间HbA2水平差异不显著;轻型β地中海贫血合并缺铁性贫血组患者HbA2水平和机体的血清铁水平之间并无显著相关性(r=0.053,P0.05)。结论缺铁的严重程度可以影响HbA2水平,轻型β地中海贫血合并缺铁性贫血患者的HbA2水平和血清铁之间并无显著相关性。     

骨髓增生异常综合征患者MRI检查心脏T2~*值与心脏铁过载关系的初步探讨

目的:检测骨髓增生异常综合征(MDS)患者MRI检查中心脏T2*值和心脏铁过载的相关指标——左心室射血分数(LVEF),并探讨心脏T2*值与心脏功能的关系。方法:20例MDS患者行MRI检查,得到心脏、肝脏T2*数值,并换算成心铁、肝铁浓度(LIC),同时进行心脏超声检测,得到LVEF。结果 :MDS患者的心脏T2*值与LVEF间存在相关性(r=0.565,P=0.009),而心脏T2*值与国际预后积分系统(IPSS)评分、患者病程时间、LIC、SF间则均无相关性(P>0.05)。不同WHO分型、IPSS分层、性别、是否输血、近一年输血量患者间的心脏T2*值无统计学差异。结论:心脏T2*值对MDS患者心脏功能有一定预测性,比同期的LIC及血清铁蛋白可能更有价值。     

骨髓MRI在重型β-地中海贫血造血干细胞移植中的价值

目的 探讨骨髓MRI在重型β地中海贫血造血干细胞移植(HSCT)中的价值。方法 对27例拟行HSCT重型地中海贫血患儿的股骨骨髓进行MRI检查。进行FSE的T1WI及T2WI、STIR、同相位及反相位快速场回波序列(FFE)股骨近段斜冠状位成像。在T1WI、T2WI及STIR图像上判断红黄骨髓的分布,并测量股骨近段红骨髓总面积及其百分比。在同相位、反相位FFE图像上序列判断骨髓是否有铁沉积。比较不同病情及不同移植结局患者的红骨髓面积及其百分比及骨髓内铁沉积,并与临床实验室指标进行相关性分析。运用Logistic回归分析红骨髓面积及其百分比、骨髓铁沉积与HSCT结局之间的关系。结果 27例重型地中海贫血中,10例出现骨髓铁沉积,骨髓铁沉积与临床指标无相关性(P>0.05),对HSCT结局无影响(P＝0.775)。红骨髓面积及其百分比与病程及血清铁蛋白正相关(r=0.727、0.428,P<0.05;r=0.487、0.511,P＝0.01、0.006)。27例中2例死于预处理,余25例进行了HSCT。进行HSCT的25例中,移植成功及移植失败病例中红骨髓面积及其百分比分别为(7.94±2.71) mm²(90.25%±4.14%),(10.54±3.31) mm²(94.54%±2.93%),两组之间均有统计学差异(P＝0.04、0.01)。回归分析显示红骨髓面积百分比与HSCT结局密切相关(OR=1.383,P＝0.005)。结论 MRI上股骨近段红骨髓面积百分比可作为重型地中海贫血HSCT病情分类的一个独立危险因素。     

初诊2型糖尿病患者肝脏脂肪沉积的~1H MRS量化及其与血清甘油三酯和胰岛素抵抗的关系研究

目的分析初诊2型糖尿病(T2DM)患者肝脏脂肪含量与血清甘油三酯(TG)、身高体重指数(BMI)及胰岛素抵抗的关系。方法在3.0T磁共振系统应用单体素点分辨波谱序列(PRESS)对32例初诊T2DM患者和32例无糖尿病志愿者的肝脏进行1H MRS扫描,应用SAGE软件测量其脂肪和水的峰下面积,计算肝脏脂肪分数。分析肝脏脂肪含量与BMI、TG以及胰岛素抵抗指数(HOMA-IR)的相关性。结果 32例初诊T2DM患者和32例无糖尿病志愿者的平均肝脏脂肪分数分别为24.84(8.79~33.21)%和6.50(3.31~16.85)%,差异有统计学意义(P=0.006)。相关性分析表明,初诊T2DM患者的肝脏脂肪含量与TG(r=0.615,P=0.000)、BMI(r=0.557,P=0.001)中度相关,与HOMA-IR低度相关(r=0.355,P=0.046)。无糖尿病志愿者的肝脏脂肪含量与TG高度相关(r=0.740,P=0.000),与BMI(r=0.666,P=0.000)中度相关。结论初诊T2DM患者的肝脏脂肪含量高于无糖尿病人群,且与TG、BMI及HOMA-IR有相关性。     

铁超负荷兔模型3.0TMRI定量肝铁沉积可行性研究

目的探讨3.0T MRI定量肝脏铁沉积的可行性。材料与方法 44只雄性新西兰家兔,随机分为实验组30只,对照组2只,验证组12只。实验组及验证组每周一肌注右旋糖酐铁15mg/kg;实验组于1～15周周日随机选取2只行肝脏3.0T MRI检查,测量T2值（R2=1/T1）,肝脏与肌肉信号强度比值（SIR）。2只对照组检查时间点同实验组。验证组分别在1～5、8、9及11～15周周日随机选取1只检查。实验组、验证组检查结束即处死,取出肝脏,用原子吸收分光光度计测量肝铁浓度（LIC）并行病理检查。对照组于15周处死,处置如实验组。结果随着注射铁剂增多,病理显示肝脏铁沉积加重。实验组肝脏T2值、SIR及LIC的范围分别为0.3～1.5ms、10.2～48.3、1.3～9.1mg/g干重,中位数分别为1.0ms、18.9、4.6mg/g干重。LIC与R2值、SIR均呈线性相关（R2：r=0.948,P=0.000;SIR：r=-0.845,P=0.000）。通过直线回归分析,分别得到R2、SIR与LIC拟合直线的斜率为96.426、-5.924,截距为-0.920、10.581。验证组实际LIC与利用R2、SIR通过预测公式得出的LIC两者组内相关系数（ICC）分别为0.953、0.914。结论一定LIC范围内使用3.0T MRI定量肝铁沉积具有可行性。     

Myocardial iron loading in patients with thalassemia major on deferoxamine chelation.

BACKGROUND: Heart failure secondary to myocardial iron loading remains the leading cause of death in thalassemia major (TM). We used cardiovascular magnetic resonance (CMR) to assess the prevalence of myocardial iron overload and ventricular dysfunction in a large cohort of TM patients maintained on conventional chelation treatment with deferoxamine. METHODS: A mobile CMR scanner was transported from London, UK, to Sardinia, Italy where 167 TM patients were assessed for myocardial iron loading, B-natriuretic peptide (BNP), and ferritin. In patients with myocardial iron loading CMR assessments of ventricular function were also made. RESULTS: Myocardial iron loading (T2* < 20 ms) was present in 108 (65%) patients, which was severe (T2* < 8 ms) in 22 (13%). Impaired (< 56%) left ventricular (LV) ejection fraction (EF) was present in 5%, 20% and 62% of patients with mild, moderate or severe iron loading. Increasing myocardial iron was related to impaired LVEF (Rs = 0.57, p < 0.001), weakly related to serum ferritin (Rs = -0.34, p < 0.001), and not related to liver iron (Rs = 0.11, p = 0.26). BNP was weakly related to myocardial iron (Rs = -0.35, p < 0.001) and was abnormal in only 5 patients. CONCLUSIONS: Myocardial siderosis was found in two-thirds of thalassemia major patients on maintenance deferoxamine treatment. This was combined with a high prevalence of impaired LV function, the severity of which tracked the severity of iron deposition. BNP was not useful to assess myocardial siderosis.     

Biopsy-based calibration of T2* magnetic resonance for estimation of liver iron concentration and comparison with R2 Ferriscan

Background

There is a need to standardise non-invasive measurements of liver iron concentrations (LIC) so clear inferences can be drawn about body iron levels that are associated with hepatic and extra-hepatic complications of iron overload. Since the first demonstration of an inverse relationship between biopsy LIC and liver magnetic resonance (MR) using a proof-of-concept T2* sequence, MR technology has advanced dramatically with a shorter minimum echo-time, closer inter-echo spacing and constant repetition time. These important advances allow more accurate calculation of liver T2* especially in patients with high LIC.

Methods

Here, we used an optimised liver T2* sequence calibrated against 50 liver biopsy samples on 25 patients with transfusional haemosiderosis using ordinary least squares linear regression, and assessed the method reproducibility in 96 scans over an LIC range up to 42 mg/g dry weight (dw) using Bland-Altman plots. Using mixed model linear regression we compared the new T2*-LIC with R2-LIC (Ferriscan) on 92 scans in 54 patients with transfusional haemosiderosis and examined method agreement using Bland-Altman approach.

Results

Strong linear correlation between ln(T2*) and ln(LIC) led to the calibration equation LIC = 31.94(T2*)^-1.014. This yielded LIC values approximately 2.2 times higher than the proof-of-concept T2* method. Comparing this new T2*-LIC with the R2-LIC (Ferriscan) technique in 92 scans, we observed a close relationship between the two methods for values up to 10 mg/g dw, however the method agreement was poor.

Conclusions

New calibration of T2* against liver biopsy estimates LIC in a reproducible way, correcting the proof-of-concept calibration by 2.2 times. Due to poor agreement, both methods should be used separately to diagnose or rule out liver iron overload in patients with increased ferritin.     

The reproducibility of cardiac and liver T2* measurement in thalassemia major using two different software packages

In b-thalassemia major (TM) multiple blood transfusions are needed for survival. As a consequence these patients present iron overload in different organs, including heart and liver. Magnetic resonance imaging using a bright blood gradient echo sequence has been successfully used for the quantification of tissue iron. The aim is to evaluate of the accuracy and precision in the evaluation of liver and myocardial T2* values in TM using two different analytical software solutions. Thirty TM patients aged 20–56 years (mean age 37, 11M/19F) were scanned in a GE 1.5 T CVI system. Each scan included the measurement of heart and liver T2* and the left ventricular ejection fraction using standard techniques. The analysis of T2* of heart and liver was done using the two different analytical software solutions: the “Functool” protocol by GE and the T2* module of QMassMR v7.4 by Medis medical imaging systems bv, Leiden, The Netherlands. The cardiac and liver T2* measurements showed that both software solutions allow reproducible measurements with low intra-observer variations (accuracy < 0.3 ms, precision < 2 ms). There is a small but significant difference between the two solutions of 2.4 ms in cardiac and of 1.5 ms in liver measurements. However, from the clinical point of view these differences (<2 ms) are small with negligible impact on the patient’s treatment management. The comparison of the T2* measurements using the two analytical software solutions proved that both techniques enable reproducible measurements for the evaluation of iron overload in heart and liver.     

Detection of myocardial iron overload by two-dimensional speckle tracking in patients with beta-thalassaemia major: a combined echocardiographic and T2* segmental CMR study

We aimed to evaluate the role of two-dimensional speckle tracking imaging (2DSTI) in detecting early changes of myocardial deformation in patients affected by thalassemia major (TM) and its relation with myocardial iron overload (MIO) detected by T2* cardiovascular magnetic resonance (CMR). We studied 28 TM patients (15 males, 37.4?±?10 years). All patients underwent CMR and echocardiography in the same day. Segmental and global T2* values were measured. Values of global longitudinal strain (GLS) were derived from the three apical views, while radial and circumferential strain were obtained as average strain from the short axis views at basal, mid and apical level. Six patients (21.4%) showed significant MIO (global heart T2*?<?20 ms). GLS showed a significant correlation with T2* values (R?=??0.49; P?=?0.001) and it was significantly lower in patients with a significant MIO than in those with no significant MIO (?18.3?±?2 vs. ?21.3?±?2.7, P?=?0.02). No significant difference was found for radial and circumferential strain in relation to the severity of MIO. Patients with impaired GLS (<?19.5%) had a significant higher risk of showing significant MIO (Odds-ratio-OR?=?17; 95%). GLS is related with global T2* in TM patients. Moreover, GLS can identify TM patients with severe MIO detected by CMR.     

The efficacy of tissue Doppler imaging in predicting myocardial iron load in patients with beta-thalassemia major: correlation with T2* cardiovascular magnetic resonance

Tissue Doppler imaging (TDI) can detect myocardial dysfunction related to iron load in patients with beta-thalassemia major (TM). We aimed to assess the efficacy of pulsed-wave TDI (PW-TDI) in predicting myocardial iron load in patients with TM using T2* magnetic resonance (MR) as the gold-standard non-invasive diagnostic test. 33 asymptomatic TM patients, mean aged 18 ± 6 years (6–31) with normal left ventricular (LV) global systolic function were evaluated by conventional echocardiography and PW-TDI. Results were compared with 20 age and sex-matched controls. TDI measures included myocardial systolic (Sm), early (Em) and late (Am) diastolic velocities at basal and middle segments of septal and lateral LV wall. Myocardial iron deposition were measured in 29/33 patients by T2* MR. TM patients were also subgrouped according to those with iron load (T2* ≤ 20 ms) and those without (T2* > 20 ms). Mean T2* was 12.3 ± 7.8 ms (4–31.3). Abnormal myocardial iron load (T2* < 20 ms) was found in 25/29 (86%) patients. The following TDI measures were lower in patients than in controls: basal septal Em (P < 0.001) and Am (P < 0.05), mid-septal Am (P < 0.05), mid-lateral LV wall Sm (P < 0.05) and Am (P < 0.05). Regional myocardial dysfunction were more prominent in patients with T2* ≤ 20 ms. Mid-septal Sm and Em significantly correlated with mid-septal T2*(r = 0.44, P = 0.023 and r = 0.54, P = 0.004, respectively). The PW-TDI parameters and the cut-off values for predicting presence of myocardial iron load were determined. PW-TDI technique was found both sensitive and specific in predicting presence of myocardial iron load in TM patients with normal LV global systolic function. Therefore, it can be used for screening of TM patients.     

Cardiac structure and function during ageing in energetically compromised Guanidinoacetate N-methyltransferase (GAMT)-knockout mice – a one year longitudinal MRI study

Background

In thalassemia major (TM), severe cardiac siderosis can be treated by continuous parenteral deferoxamine, but poor compliance, complications and deaths occur. Combined chelation therapy with deferiprone and deferoxamine is effective for moderate myocardial siderosis, but has not been prospectively examined in severe myocardial siderosis.

Methods

T2* cardiovascular magnetic resonance (CMR) was performed in 167 TM patients receiving standard subcutaneous deferoxamine monotherapy, and 22 had severe myocardial siderosis (T2* < 8 ms) with impaired left ventricular (LV) function. Fifteen of these patients received combination therapy with subcutaneous deferoxamine and oral deferiprone with CMR follow-up.

Results

At baseline, deferoxamine was prescribed at 38 ± 10.2 mg/kg for 5.3 days/week, and deferiprone at 73.9 ± 4.0 mg/kg/day. All patients continued both deferiprone and deferoxamine for 12 months. There were no deaths or new cardiovascular complications. The myocardial T2* improved (5.7 ± 0.98 ms to 7.9 ± 2.47 ms; p = 0.010), with concomitant improvement in LV ejection fraction (51.2 ± 10.9% to 65.6 ± 6.7%; p < 0.001). Serum ferritin improved from 2057 (CV 7.6%) to 666 (CV 13.2%) μg/L (p < 0.001), and liver iron improved (liver T2*: 3.7 ± 2.9 ms to 10.8 ± 7.3 ms; p = 0.006).

Conclusion

In patients with severe myocardial siderosis and impaired LV function, combined chelation therapy with subcutaneous deferoxamine and oral deferiprone reduces myocardial iron and improves cardiac function. This treatment is considerably less onerous for the patient than conventional high dose continuous subcutaneous or intravenous deferoxamine monotherapy, and may be considered as an alternative. Very prolonged tailored treatment with iron chelation is necessary to clear myocardial iron, and alterations in chelation must be guided by repeated myocardial T2* scans.

Trial registration

This trial is registered as NCT00103753     

Magnetic resonance evaluation of liver and myocardial iron deposition in thalassemia intermedia and b-thalassemia major

Introduction b-Thalassemia major (TM) and thalassemia intermedia (TI) are forms of inherited hemoglobinopathies. Our aim was to evaluate a population of asymptomatic TM and TI patients using cardiovascular magnetic resonance (CMR). We hypothesized that the TI group could be differentiated from the TM group based on T2*. We also hypothesized that the TI group would demonstrate significantly higher cardiac output compared to the TM group. Patients and methods Twenty-one consecutive TI patients aged 23(19–25) years, 21 TM patients and 21 age and sex matched controls were studied. Evaluation of heart, liver T2* relaxation time and right and left ventricular parameters was performed using a 1.5 T system. Results Myocardial and liver T2* values were significantly higher in TI patients compared to TM (34.35 ± 2.36 vs 15.77 ± 3.53 m, P < 0.001 and 5.12 ± 6.52 vs 1.36 ± 0.53 ms, P < 0.001, respectively). Controls had myocardial T2* 35.07 ± 4.52 ms (similar to TI patients, but significantly increased compared to TM patients, P < 0.001) and liver T2* 26.28 ± 2.37 ms (significantly increased compared to both TI and TM patients, P < 0.001). Left ventricular end-diastolic (LVEDV), end-systolic (LVESV) volumes and left ventricular ejection fraction (LVEF) were higher in TI patients compared to TM (P < 0.001). Stroke volume (LVSV), cardiac output (LVCO) and cardiac index (LVCI) were similarly increased in TI patients compared to TM (P < 0.001). Right ventricular end-diastolic volume (RVEDV), right ventricular end-systolic volume (RVESV) and right ventricular ejection fraction (RVEF) were higher in TI patients compared to TM (P < 0.001). Conclusions Although in TM iron plays a crucial role in the evolution of the disease, in TI the high output cardiac state seems to be the most prominent finding.