重型β地中海贫血铁过载患者胰腺磁共振T2*检测及影响因素分析

目的探讨重型β地中海贫血(β-TM)患者胰腺铁过载状况及其与临床各监测指标之间的关系。方法 51例β-TM患者,进行胰腺MRI T2*检测。按是否有铁过载分为两组,组间比较性别、年龄、空腹血糖、血红蛋白(Hb);与血清铁蛋白(SF)、心脏T2*、肝脏T2*及输血时间进行相关性分析;比较铁螯合剂去铁胺联合去铁酮及单用地拉罗司(DFO+DFP组与DFX组)对胰腺T2*和心脏T2*的影响。结果 51例患者中有42例胰腺铁过载,占83%。胰腺铁过载与胰腺无铁过载组间比较,性别、年龄、空腹血糖、Hb差异无显著性(P>0.05);肝脏T2*、SF、输血时间与胰腺T2*之间呈低度相关(r值分别为-0.36、0.35及0.48);心脏T2*与胰腺T2*之间呈中度相关(r=0.50);心脏铁过载患者中91%有胰腺铁过载,89%胰腺无铁过载患者中心脏也无铁过载发生;DFX治疗组胰腺T2*显著高于DFO+DFP组(U=-2.23,P=0.03),但心脏T2*两组间差异无显著性(U=-0.67,P=0.51)。结论胰腺铁过载发生普遍;年龄、空腹血糖、是否足量输血对胰腺铁过载影响不明显;SF、肝脏T2*不能准确反映胰腺铁过载的情况,输血时间对胰腺铁过载影响不明显;胰腺与心脏有相同的铁动力学,可以通过胰腺铁过载来预测心脏铁过载;DFX可有效降低胰腺铁过载,但对心脏铁过载疗效有待进一步观察。     

不同铁螯合剂治疗重型β地中海贫血铁过载的临床研究

目的 比较去铁胺(DFO)、去铁酮(DFP)、地拉罗司(DFX)三种不同铁螯合剂治疗重型β地中海贫血(β-TM)铁过载患者5年的临床疗效.方法 随机选择规律输血的β3-TM铁过载患者31人,按DFO、DFP和DFX分为三个治疗组,分别对血清铁蛋白(SF)、心脏MRIT2*、肝脏MRIT2*值变化进行分析.结果 服药5年后,三组SF均明显降低、心脏MRIT2*、肝脏MRIT2*明显升高(P＜0.05);组间比较,三组SF水平5年连续监测差异无显著性(P＞0.05);心脏MRIT2*组间比较差异无显著性(P＞0.05),但与基线相比,第2年DFP组心脏MRIT2*值上升幅度明显高于DFX组、DFO组(P＜0.05),后3年差异无显著性;5年连续监测肝脏MRIT2* DFO组高于DFP组、DFX组(P＜0.05),与基线相比,DFO组第2年肝脏MRIT2*值上升幅度明显高于DFP组、DFX组(P＜0.05),后3年差异无显著性.结论 三种铁螯合剂均能有效降低重型β-TM患者SF浓度、去除心脏和肝脏铁沉积;短期内DFP降低心脏铁负荷优于DFO及DFX,DFO降低肝脏铁负荷优于DFP及DFX.     

不同铁鳌合剂治疗重型β地中海贫血铁过载的临床研究

目的比较去铁胺(DFO)、去铁酮(DFP)、地拉罗司(DFX)三种不同铁螯合剂治疗重型β地中海贫血(β-TM)铁过载患者5年的临床疗效。方法随机选择规律输血的β-TM铁过载患者31人,按DFO、DFP和DFX分为三个治疗组,分别对血清铁蛋白(SF)、心脏MRIT2*、肝脏MRIT2*值变化进行分析。结果服药5年后,三组SF均明显降低、心脏MRIT2*、肝脏MRIT2*明显升高(P0.05);组间比较,三组SF水平5年连续监测差异无显著性(P0.05);心脏MRIT2*组间比较差异无显著性(P0.05),但与基线相比,第2年DFP组心脏MRIT2*值上升幅度明显高于DFX组、DFO组(P0.05),后3年差异无显著性;5年连续监测肝脏MRIT2*DFO组高于DFP组、DFX组(P0.05),与基线相比,DFO组第2年肝脏MRIT2*值上升幅度明显高于DFP组、DFX组(P0.05),后3年差异无显著性。结论三种铁螯合剂均能有效降低重型β-TM患者SF浓度、去除心脏和肝脏铁沉积;短期内DFP降低心脏铁负荷优于DFO及DFX,DFO降低肝脏铁负荷优于DFP及DFX。     

重型β地中海贫血铁过载患儿心脏与肝脏磁共振T2*检测及其相关因素分析

目的探讨重型β地中海贫血(β-TM)患儿心脏、肝脏铁过载状况,以及与临床监测指标之间的关系。方法 2010年6月,根据自愿的原则,从规律输血治疗的80例≥7岁的β-TM患儿中,选择51例,进行心脏磁共振T2*(心脏T2*)及肝脏磁共振T2*(肝脏T2*)检测。检测结果与年龄、血清铁蛋白(SF)、心脏左室射血分数(LVEF)、输血年限、去铁年限、输血前血红蛋白(Hb)进行比较。结果 51例患儿中11例(21.6%)发生心肌铁过载,其中轻度3例,中度3例,重度5例;肝脏铁过载43例(84.3%),其中轻度14例,中度17例,重度12例。心脏T2*与SF、LVEF、肝脏T2*之间无相关性,SF与肝脏T2*呈正相关(r=0.558,P<0.01)。心肌铁过载患儿输血年限大于心肌铁正常患儿(P<0.05),而肝脏铁过载发生率差异无统计学意义(P>0.05)。11例心肌铁过载患儿中,2例LVEF降低。结论β-TM患儿SF值可反映机体肝脏铁过载的情况,但不能预测心肌铁过载;随着输血年限的增加,心肌铁过载风险亦加大;心肌铁过载与肝脏铁过载之间存在着不一致性,肝脏铁过载不能成为提示心肌铁过载的依据。心肌铁过载患儿LVEF可以在...     

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目的探讨重型β珠蛋白生成障碍性贫血(beta-thalassaemia,简称β-TM)患儿长期输血、去铁治疗与铁过载的关系。方法深圳市第二人民医院2001年成立"地贫之友"与"地贫服务队",对β-TM患儿进行规范性的长期输血和去铁治疗。每3个月监测血清铁蛋白浓度(SF)、肝肾功能、心肌酶谱、心功能、心脏和肝脾B超、血糖和尿糖。2001年2月至2010年6月对其中51例患儿进行核磁共振检测心脏T2*、左心室射血分数(LVEF)、肝脏T2*、胰腺T2*和垂体T2*。根据治疗方法分为足疗程去铁胺+去铁酮(DFO+DFP)联合去铁治疗组(足疗程联合组)10例、不足疗程DFO+DFP联合去铁治疗组(不足疗程联合组)31例、单用足疗程地拉罗司(DFX)去铁治疗组(单用DFX组)10例。根据SF质量浓度分为SF≤2000μg/L组(A组)12例、SF~3000μg/L组(B组)17例、SF>3000μg/L组(C组)22例。结果各组LVEF、心脏T2*、垂体T2*值差异无统计学意义(P>0.05);足疗程联合组肝脏T2*高于不足疗程联合组(P<0.05),单用DFX组肝脏T2*、胰腺T2*、垂体T2*均高于足疗程联合组和不足疗程联合组(P<0.05)。足疗程联合组SF低于不足疗程联合组,单用DFX组SF低于足疗程联合组和不足疗程联合组,差异均具有统计学意义(P<0.05)。C组肝脏T2*和胰腺T2*明显低于A组和B组,差异具有统计学意义(P<0.05)。心肌铁过载11例(21.6%),肝脏铁过载43例(84.3%)。SF与心脏T2*无相关性(r=0.254,P>0.05),与肝脏T2*呈中度负相关(r=0.558,P<0.01)。结论足疗程DFO+DFP联合去铁治疗和单用足疗程DFX去铁治疗均能有效降低血清铁蛋白浓度,动员肝脏组织铁,效果优于不足疗程DFO+DFP联合去铁治疗。不同去铁方式均能减轻心脏铁过载。     

磁共振成像(T2*)检测重型β-地中海贫血患儿心脏、肝脏铁负荷及左心室射血分数的研究

目的观察我院重型β-地中海贫血(β-TM)患儿血清铁蛋白(SF)和体内铁沉积状况及对心脏功能的影响。方法检测我院28例重型β-TM患儿SF水平,并由我院放射科运用磁共振成像T2*(MRI T2*)技术检测心脏及肝脏铁沉积状况及左室射血分数(LVEF)。结果本组28例患儿中男16例,女12例,中位年龄11.2岁(7岁1个月~16岁11个月)。28例患儿中SF轻度升高者4例(14.3%),中度升高者8例(28.6%),高度升高者16例(57.1%)。肝脏MRI T2*检测结果 :肝铁轻度沉积2例(7.1%),中度沉积8例(28.6%),重度沉积18例(54.8%);心脏MRI T2*检测结果 :心铁正常16例(57.1%),轻度铁沉积4例(14.3%),中度铁沉积4例(14.3%),重度铁沉积4例(14.3%);LVEF降低者19例(67.9%),4例心脏重度铁沉积患儿中有3例(75%)LVEF明显降低(≤55%)。本组患儿SF水平与肝脏T2*值(r=-0.467,P=0.036)、心脏T2*值(r=-0.486,P=0.01)呈明显负相关;SF与输血量呈正相关(r=0.634,P=0.043;r=0.067,P=0.124)而与去铁治疗呈负相关(r=-0.526,P=0.043),SF和心脏T2*值与LVEF均无相关性(r=-0.154,P=0.306;r=0.067,P=0.124)。结论重型β-TM患儿SF增高及肝脏重度铁沉积发生率高,虽然心脏重度铁沉积患儿发生率不高,但心脏重度铁沉积患儿多有LVEF明显降低。     

重型β地中海贫血患者肝脏胰腺和心脏铁负荷磁共振成像T2*的多中心研究

目的 探讨使用磁共振成像T2*（MRI T2*）方法检测重型β地中海贫血（重型β地贫）患者肝脏、胰腺和心脏铁过载状况,并评估其有效性。方法 总结2010年5月至2010年12月中国广东及广西省共7所医院的153例重型β地贫患者的临床资料,对其肝脏、胰腺、心脏MRI T2*与年龄、血清铁蛋白作相关性分析,并按肝脏不同铁沉积等级比较患者心脏和（或）胰腺铁沉积的情况。结果 心脏铁沉积重度37例, 轻度21例, 正常95例。肝脏、胰腺和心脏MRI T2*值与血清铁蛋白水平有相关性,但与年龄无相关性,血清铁蛋白水平也与年龄无关。肝脏与胰腺T2*值（r = 0.529, P = 0.000）、肝脏与心脏的T2*值（r = 0.369,P = 0.000）以及胰腺与心脏T2*值（r = 0.715,P = 0.000）有相关性。随着肝脏铁沉积等级的增加,患者同时合并心和胰腺的比例也随之增大（χ2=20.78,P = 0.000）。结论 肝脏、胰腺、心脏三器官间的MRI T2*值有相关性。随着肝脏铁沉积等级增加,患者合并心脏、胰腺铁沉积的比例逐渐增高。     

重型β地中海贫血患儿生长发育异常及其与铁超负荷的关系

目的：探讨重型β-地中海贫血患儿生长发育状态及其与铁超负荷的关系。方法：于2007年7~8月对50例长期在该院儿科定期输血的重型β 地中海贫血患儿进行身高、体重和性发育评价，并与1995年中国0~18岁儿童体重、身高百分位数参考值比较，同时检测输血前血常规、肝功能和血清铁蛋白水平。结果：24例（48%）重型β-地中海贫血患儿表现身材矮小，其中15例同时伴体重低下。≥10岁者21例，仅7例出现自发性青春期发育，Tanner II~III期；≥14岁者8例，其中4例尚无性征发育。身高低于第10百分位者（n=31）与身高高于第10百分位者(n=19)分组比较，前者血清铁蛋白水平显著增高（8 239.2±5 865.5 vs 5 028.1±3 885.7 mg/L, P<0.05），输血前Hb水平显著降低(68.2±12.3 vs 79.7±14.5 g/L, P<0.05) ，肝脏显著增大(P<0.05)。而体重低于第10百分位者（n=20）与体重高于第10百分位者（n=30）分组比较，前者仅血清铁蛋白水平差异具有显著意义（9 165.5±6 042.5 vs 5 567.3±4 447.3 mg/L, P<0.05）。结论：接受中等量输血和不正规除铁治疗的重型β-地中海贫血患儿常伴有身材矮小、体重低下和性发育迟缓，其生长发育异常与体内铁严重超负荷有关。     

地中海贫血铁代谢特点及器官铁沉积的检测

输血依赖型地中海贫血(重型地贫,TDT)和非输血依赖型地贫(NTDT)患者由于长期反复输血和肠道铁吸收增加导致铁过载,过多的铁沉积于心、肝、胰腺及垂体等组织器官,引起组织细胞损伤和器官功能衰竭,临床表现为心功能衰竭、肝硬化、糖尿病和生长发育障碍等,最终引起死亡。心脏核磁共振(MRI)T2~*和肝脏MRI R2值可以很好地反映心脏和肝脏的铁负荷。在NTDT患者血清铁蛋白(SF)常会低估体内铁过载的程度。检测脏器铁沉积情况及选择合适的螯合剂祛铁治疗是减低地贫患者铁过载并发症的必要手段。     

珠海地区重型β-地中海贫血患儿的生存状况

目的　从行政区域范围了解珠海地区重型β-地中海贫血（β-TM）患儿生存状况。方法　分析2014年1月至2015年12月广东珠海≤14岁β-TM患儿的治疗依从性、生长发育和器官功能状况以及β-TM年出生人数。结果　研究期间珠海共62例，57例（91.9%）依从调查和随访。57例并发症发生率：脾大77.2%，肝大61.4%，生长迟缓59.6%，ALT升高22.8%，亚临床甲状腺功能减退14.0%，左心射血分数降低7.0%，胰岛素抵抗5.3%。不规范输血主要影响脾大和心功能，而不规范祛铁则影响血清铁蛋白、生长发育、ALT、内分泌系统和心功能；血清铁蛋白多数未得到良好控制。22.8%和36.2%患儿不规范输血和祛铁治疗，医保覆盖和知识宣教是首位和次位影响因素。新出生β-TM 逐年减少。结论　珠海的调查结果在一定程度上反映我国中上发达地区β-TM患儿的生存状况；医疗保障、宣教及规范治疗仍是今后工作的重点。     

Effect of Deferasirox Chelation on Liver Iron and Total Body Iron Concentration

Objective

To determine efficacy of Deferasirox (DFX) on total body iron and liver iron concentration (LIC) as estimated by serum ferritin (SF) and liver MRI T2*.

Methods

Thirty patients had baseline MRI T2* of the liver performed to determine LIC before starting DFX therapy and classified as normal >6.3 milliseconds (ms), mild 6.3?2.7 ms, moderate 2.7?1.4 ms and severe iron overload <1.4 ms. DFX was given 25–35 mg/kg/d. The serum ferritin (SF) level was estimated every 3 monthly. Liver iron is expressed as liver R2*?=?1000/T2*. The primary end point of the study was to determine change in SF and liver MRI R2* values after 18 mo of therapy.

Results

All 30 patients had some degree of liver iron overload; 11 (36.6 %) had severe, 15 (50 %) had moderate while 4 (13.3 %) had mild overload. The pre-DFX therapy median SF of all was 3604.5 ng/mL (IQR 2357.0–5056.0) and median liver R2* was 574.71 Hz (IQR 411.3–770.8). After 18 mo, SF dropped significantly to a median of 2036.5 ng/mL (IQR 1700.0–3162.0) (p?=?0.0011), while median liver R2* decreased from 574.71 to 568.18Hz (IQR 393.4–803.2) which was not significant (p?=?0.986).

Conclusions

DFX monotherapy at the doses used decreases total body iron, but does not significantly decrease liver iron. It is well tolerated by Indian thalassemia patients, with observed side effects including rash, diarrhea, and transient albuminuria. MRI T2* (and derived R2*) can serve as useful method in non invasive monitoring of LIC in thalassemia patient management.     

Chronically transfused pediatric sickle cell patients are protected from cardiac iron overload

Iron overload is a major toxicity of chronic transfusions. Myocardial iron overload is associated with cardiac dysfunction. Cardiac and liver magnetic resonance imaging (MRI) was performed on 14 chronically transfused sickle cell disease (SCD) and non-sickle cell disease (non-SCD) patients seen at Vanderbilt Children's Hospital from 1 January 2000 to 10 March 2010. Retrospective review was conducted to assess cardiac T2*, liver T2*, ventricular dimensions and function, echocardiogram, length of transfusion, hemoglobin, and ferritin measurements. Ten patients had SCD and 4 had non-SCD, including α-thalassemia, β-thalassemia, and Diamond-Blackfan anemia. Cardiac T2* was normal in all SCD patients (mean 39 ± 12 ms), but abnormal in 3 of 4 non-SCD patients (mean 11.8 ± 2.4 ms). Liver T2* was similar between SCD (mean 6.2 ± 1.6 ms) and non-SCD patients (mean 5.9 ± 1.9 ms), and did not correlate with serum ferritin. Comparing SCD and non-SCD patients with similar transfusion duration, SCD patients had normal cardiac T2* and non-SCD patients had abnormal cardiac T2*. No patients had cardiomyopathy, but ventricular dilatation was common among SCD patients. Chronically transfused pediatric SCD patients are relatively spared of myocardial iron overload, which is unlikely to be due to lower total body iron burden in SCD patients than non-SCD patients.     

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??Abstracts?? Objective To study the T2* magnetic resonance imaging for evaluation of iron deposition of liver?? pancreas and heart in β- thalassemia major ??β-TM?? patients and the relationship of the serum ferritin with iron overload. Methods Measurement of hepatic?? pancreatic and cardiac MRI T2* was performed in 153 patients with β-TM from May 1?? 2010 to December 31?? 2010??The demographics and serum ferritin ??SF?? were collected. The rate of the iron overload in liver?? pancreas and heart was analyzed. The rates of combined pancreatic and cardiac iron overload in different hepatic iron overload groups were compared. Results According to MRI T2*?? there was serious hepatic iron overload in 85 cases?? moderate in 39?? mild in 21?? no iron overload in 8. Similarly?? there was pancreatic iron overload in 134?? no iron overload in 19. In terms of cardiac iron overload?? there was serious cardiac iron overload in 37 cases?? mild in 21?? no iron overload in 95. No correlation was found between hepatic?? pancreatic and cardiac MRI T2* and age or SF?? but hepatic MRI T2* correlated with pancreatic and cardiac MRI T2*??P = 0.000?? r = 0.529?? r = 0.369???? and pancreatic MRI T2* correlated with cardiac MRI T2* well ??P = 0.000?? r = 0.715??. As grade of hepatic iron overload increases?? the patients demonstrated higher rate of combination of pancreatic and cardiac iron overload simultaneously ??χ2 = 20.78??P = 0.000??.     

MRI T2*技术对中、重型β地中海贫血患者心脏及肝脏铁沉积状态的评估

目的：了解中、重型β地中海贫血患者体内铁沉积状况。方法：对39例中、重型β地中海贫血患者的输血、排铁的情况进行统计,检测患者体内铁蛋白水平,并运用MRI T2*技术检测心脏及肝脏铁沉积状况。结果：患者血清铁蛋白水平最低为1500 ng/mL,最高达 11491 ng/mL。肝脏铁重度沉积者15例（38%）,中度沉积者15例（38%）,轻度沉积者7例（18%）,正常者 2例（5%）。 心脏铁重度沉积者7例（18%）,轻度沉积者5例（13%）,正常者27例（69%）。1例出现心律紊乱症状,4例年龄超过20岁者均呈现性腺功能发育不全。大多患者因家庭经济原因未能进行规律输血及排铁治疗,且开始排铁时间较晚。患者血清铁蛋白水平与开始排铁的时间、剂量密切相关。结论：未进行早期规律的输血和排铁治疗的地中海贫血患者,体内铁的沉积发生年龄早,易早期出现重要器官的功能损害而引发相关并发症,应引起临床医师和患者家属的高度重视并制定相应的诊疗措施提高患者的生活质量。     

Chronically Transfused Pediatric Sickle Cell Patients are Protected from Cardiac Iron Overload

Iron overload is a major toxicity of chronic transfusions. Myocardial iron overload is associated with cardiac dysfunction. Cardiac and liver magnetic resonance imaging (MRI) was performed on 14 chronically transfused sickle cell disease (SCD) and non-sickle cell disease (non-SCD) patients seen at Vanderbilt Children's Hospital from 1 January 2000 to 10 March 2010. Retrospective review was conducted to assess cardiac T2*, liver T2*, ventricular dimensions and function, echocardiogram, length of transfusion, hemoglobin, and ferritin measurements. Ten patients had SCD and 4 had non-SCD, including α-thalassemia, β-thalassemia, and Diamond-Blackfan anemia. Cardiac T2* was normal in all SCD patients (mean 39 ± 12 ms), but abnormal in 3 of 4 non-SCD patients (mean 11.8 ± 2.4 ms). Liver T2* was similar between SCD (mean 6.2 ± 1.6 ms) and non-SCD patients (mean 5.9 ± 1.9 ms), and did not correlate with serum ferritin. Comparing SCD and non-SCD patients with similar transfusion duration, SCD patients had normal cardiac T2* and non-SCD patients had abnormal cardiac T2*. No patients had cardiomyopathy, but ventricular dilatation was common among SCD patients. Chronically transfused pediatric SCD patients are relatively spared of myocardial iron overload, which is unlikely to be due to lower total body iron burden in SCD patients than non-SCD patients.     

T2* MRI in regularly transfused children with thalassemia intermedia: serum ferritin does not reflect liver iron stores

Nontransfused patients with thalassemia intermedia (TI) accumulate iron due to increased gastrointestinal absorption of iron. Recent studies using T2* MRI revealed that serum ferritin does not reflect the severity of iron overload in nontransfused TI patients. We evaluated the iron overload status in TI children on monthly transfusion. Based on serum ferritin levels, 11 such patients (mean age 13.18 ± 4.09?years), were classified into two groups, group 1 (six patients) and group 2 (five patients) with serum ferritin levels below and above 1000?ng/mL, respectively. T2* MRI assessments were done for evaluation of hepatic and cardiac iron status. Group 1 and group 2 had mean serum ferritin levels of 817.300 ± 244.690?ng/mL and 1983.80 ± 662.862?ng/mL, respectively (P = .003). T2* MRI showed comparable moderate to severe hepatic iron overload status in both. None of the patients had myocardial iron deposition. We conclude that serum ferritin does not reflect the hepatic iron overload status in our patients with TI on regular transfusion.