■结缔组织病(CTD)是肺动脉高压(PAH)的第二常见原因。目前,关于CTD-PAH的临床数据很少.我们的研究旨在评估Macitentan治疗CTD-PAH的疗效和安全性。
■在这项回顾性研究中,纳入2020年4月至2021年11月在苏州大学附属第一医院诊断为CTD-PAH的患者。在患者中,9人改用Macitentan单一疗法,而23人接受初始联合疗法。平均随访时间为24周。六分钟步行距离(6MWD),世界卫生组织功能类(WHO-FC),血清N末端脑钠肽前体(NT-proBNP),评估用药前后的超声心动图参数。记录并比较不良反应。
■治疗24周后,6MWD,NT-proBNP,通过超声估计的收缩期肺动脉压(sPAP),马刺坦单药治疗组三尖瓣反流压力梯度(TRPG)和三尖瓣环平面收缩期偏移(TAPSE)差异有统计学意义(Z=-2.67,Z=-2.67,t=6.20,t=5.60,t=-3.04,P<0.05)。右心室内径(RVD)差异无统计学意义,右心房直径(RAD),升主动脉根部内径(AAO)和左心室舒张末期内径(LVEDd)(P>0.05)。服药24周后,WHO-FCIII/IV级症状的患者人数分别从6降至3,1降至0(P<0.05),WHO-FCI/II级症状患者的症状分别从0增加到2,2增加到4(P<0.05)。治疗24周后,6MWD,NT-proBNP,LVEDD,马西坦联合西地那非治疗组sPAP和TRPG比较,差异有统计学意义(Z=-4.11,Z=-3.74,Z=-3.83,t=6.88,t=6.54,P<0.001)。RVD的显著差异,RAD,发现TAPSE(t=3.46,t=3.69,t=-3.12,P<0.05)。AAO组间差异无统计学意义(P>0.05)。出现WHO-FCIII/IV级症状的患者人数分别从16例下降到8例、5例下降到0例(P<0.05),WHO-FCI/II级症状患者的症状分别从0增加到1,2增加到14(P<0.001)。6MWD治疗前后差异无统计学意义,NT-proBNP,RVD,RAD,AAO,LVEDD,sPAP,两组间TRPG和TAPSE比较(P>0.05)。丙氨酸转氨酶(ALT)差异无统计学意义,天冬氨酸转氨酶(AST),血清肌酐(Scr)和血红蛋白(Hb)在0~24周之间(P>0.05)。
■CTD-PAH患者的运动耐量和心功能在马西坦治疗后明显改善,这是很好的耐受性。因此,Macitentan可能是一种有效、安全的CTD-PAH靶向药物。
UNASSIGNED: Connective tissue disease (CTD) is the second most common cause of the pulmonary arterial hypertension (PAH). Currently, clinical data concerning CTD-PAH is scarce. Our study aimed to assess the efficacy and safety of macitentan in the treatment of CTD-PAH.
UNASSIGNED: In this retrospective study, patients diagnosed with CTD-PAH at The First Affiliated Hospital of Soochow University from April 2020 to November 2021 were included. Of the patients, 9 were switched to macitentan monotherapy whereas 23 received initial combination therapy. The mean follow-up time was 24 weeks. Six-minute walking distance (6MWD), World Health Organization functional class (WHO-FC), serum N-terminal pro-brain natriuretic peptide (NT-proBNP), and echocardiography parameters before and after medication were assessed. Adverse reactions were also recorded and compared.
UNASSIGNED: After 24 weeks of treatment, 6MWD, NT-proBNP, systolic pulmonary artery pressure (sPAP) estimated by ultrasound, tricuspid regurgitation pressure gradient (TRPG) and tricuspid annular plane systolic excursion (TAPSE) in the macitentan monotherapy group revealed significant differences (Z=-2.67, Z=-2.67, t=6.20, t=5.60, t=-3.04, P<0.05). There were no statistically significant differences in right ventricular diameter (RVD), right atrial diameter (RAD), ascending aortic root inner diameter (AAO) and left ventricular end-diastolic diameter (LVEDd) (P>0.05). After 24 weeks of medication, the number of patients with WHO-FC grade III/IV symptoms decreased from 6 to 3, 1 to 0 respectively (P<0.05), and that of patients with WHO-FC grade I/II symptoms increased from 0 to 2, 2 to 4 respectively(P<0.05). After 24 weeks of treatment, 6MWD, NT-proBNP, LVEDd, sPAP and TRPG in the macitentan combined with sildenafil treatment group revealed statistically significant differences (Z=-4.11, Z=-3.74, Z=-3.83, t=6.88, t=6.54, P<0.001). Significant differences in RVD, RAD, and TAPSE were found (t=3.46, t=3.69, t=-3.12, P<0.05). There were no statistically significant variances in AAO between the groups (P>0.05). The number of patients with WHO-FC grade III/IV symptoms decreased from 16 to 8, 5 to 0 respectively (P<0.05), and that of patients with WHO-FC grade I/II symptoms increased from 0 to 1, 2 to 14 respectively (P<0.001). There were no statistically significant differences before and after treatment in 6MWD, NT-proBNP, RVD, RAD, AAO, LVEDd, sPAP, TRPG and TAPSE between the two groups (P>0.05). There were no statistically significant differences in alanine aminotransferase (ALT), aspartate aminotransferase (AST), serum creatinine (Scr) and hemoglobin (Hb) between 0 and 24 weeks (P>0.05).
UNASSIGNED: Exercise tolerance and cardiac function in patients with CTD-PAH were significantly improved after treatment with macitentan, which was well tolerated. Therefore, macitentan may be an effective and safe targeted drug for CTD-PAH.