沙利度胺对β地贫患者生长发育和脏器参数的影响

Effects of thalidomide on height growth and organ parameters in patients withβ-thalassemia

目的:探究沙利度胺对β地中海贫血患者身高增长和脏器参数的影响。方法:52例β地中海贫血患者予以沙利度胺治疗,同时进行为期2年的治疗随访,观察其治疗前后身高增长情况、第二性征发育、女性月经情况及肝脾超声和心脏超声、心脏MRI T2*和肝脏MRI T2*。结果:52例患者中完成2年观察期的有48例,其中输血依赖型(TDT)34例,非输血依赖型(NTDT)14例。48例完成2年观察期的患者中,以其中28例<18岁的患者为观察对象,在身高方面,结果显示其在观察期内身高增长正常,与同龄人相当(P>0.05)。治疗前后第二性征发育迟缓及女性月经状态比较差异均无统计学意义(P>0.05)。与治疗前相比,患者治疗后肝脏、脾脏大小均未见明显改变(P>0.05);患者治疗后左心射血分数(LVEF)和三尖瓣反流速度(TRV)均未见明显改变(P>0.05);患者治疗后心脏T2*值和肝脏T2*值均增大(P<0.05)。结论:β地中海贫血患者采用沙利度胺治疗后,身高增长均正常,沙利度胺不影响第二性征发育,对女性月经可能无影响,肝脾肿大、LVEF和三尖瓣反流无明显改变,但心脏、肝脏铁沉积有所改善。

Objective:To explore the effects of thalidomide on height growth and organ parameters in patients withβ-thalassemia.Methods:52 patients withβ-thalassemia were selected,all of whom were treated with thalidomide and followed up for 2 years to observe their height growth,secondary sexual delay and female menstrual status,liver and spleen ultrasound,cardiac ultrasound,cardiac MRI T2*,and liver MRI T2*before and after treatment.Results:Among 52 patients,48 completed a 2-year observation period,including 34 cases of transfusion dependent(TDT)and 14 cases of non transfusion dependent(NTDT).Among the 48 patients who completed a 2-year observation period,in terms of height,28 patients under the age of 18 were observed,and the results showed that their height growth during the observation period was normal,comparable to their peers(P>0.05).There was no significant difference in secondary sexual delay and female menstrual status before and after treatment(P>0.05).Compared with before treatment,there was no significant change in the size of the patient's liver and spleen after treatment(P>0.05).Compared with before treatment,there were no significant changes in left ventricular ejection fraction(LVEF)and tricuspid regurgitation velocity(TRV)in patients after treatment(P>0.05).Compared with before treatment,the T2*values of the heart and liver in patients increased after treatment(P<0.05).Conclusion:After treatment with thalidomide,patients withβ-thalassemia showed normal height growth,and thalidomide did not affect the development of secondary sexual characteristics.It may not have an impact on female menstruation,and there is no significant change in hepatosplenomegaly,LVEF,and tricuspid regurgitation,but iron deposition in the heart and liver improved.