目的:评估获得性血友病(AHA)腹膜后和下肢肌肉骨骼出血的危险因素,并对其残疾和对生存的影响进行客观评估。
方法:我们纳入了2017年11月至2023年5月的49例AHA患者。研究了任何腹膜后或/和下肢出血表现的发生。在临床随访中,我们寻找压迫性股神经病变和股四头肌肌萎缩。所有AHA患者在最后一次出血事件后一年进行下肢功能评分(LEFS)。
结果:我们的AHA队列中有61.2%的患者出现任何腹膜后和/或下肢肌肉骨骼表现。这些患者的EACH2/ISTH标准大出血百分比更高(90%vs.57%,p=.01),输血需求(86%vs.57%的患者,p=.03),和止血旁路产品(90%与63%,p=.02)。高血压(HR2.6,95%CI1.1-5.9,p=0.02),存在自身免疫性疾病(HR13,95%CI1.7-99,p=0.01),和抑制剂水平>20BU(HR2.695%CI1.0-6.8,p=.04)显着预测腹膜后/下肢临床表现。最常见的后遗症是四肢萎缩(30.6%)和股神经麻痹(20.4%)。50岁以下的四核萎缩和LEFS评分与死亡率增加相关(分别为HR3,95%CI1.1-8.6和HR12,95%CI3.3-45)。
结论:AHA合并腹膜后/下肢出血受累的严重程度更高,显示高致残率和最差的生存结局。我们的AHA患者股四头肌萎缩和LEFS量表评分低于50个预测死亡率。
OBJECTIVE: To assess risk factors of
retroperitoneal and lower extremity musculoskeletal bleed in acquired haemophilia (AHA) and perform an objective assessment of disability and influence on survival.
METHODS: We included 49 patients with AHA from November 2017 to May 2023. The occurrence of any
retroperitoneal or/and lower extremities bleeding manifestation was investigated. On clinical follow-up, we search for compressive femoral neuropathy and quadriceps amyotrophy. The lower extremity functional scale (LEFS) was carried out one year after the last bleeding event in all AHA patients.
RESULTS: A 61.2% of patients in our AHA cohort presented with any
retroperitoneal and/or lower extremities musculoskeletal manifestation. Those patients had higher percentage of major bleeding EACH2/ISTH criteria (90% vs. 57%, p = .01), needs of blood transfusions (86% vs. 57% of patients, p = .03), and haemostatic by-pass products (90% vs. 63%, p = .02). Hypertension (HR 2.6, 95% CI 1.1-5.9, p = .02), presence of autoimmune disease (HR 13, 95% CI 1.7-99, p = .01), and inhibitor level > 20 BU (HR 2.6 95% CI 1.0-6.8, p = .04) significantly predicted
retroperitoneal/lower extremities clinical manifestations. Most frequent sequelae were quad atrophy (30.6%) and femoral nerve palsy (20.4%). Quad atrophy and LEFS scores under 50 were associated with increased mortality (HR 3, 95% CI 1.1-8.6 and HR 12, 95% CI 3.3-45, respectively).
CONCLUSIONS: AHA with
retroperitoneal/lower extremities bleeding involvement is of greater severity and shows high disability and worst survival outcomes. Quadriceps atrophy and LEFS scale scoring under 50 predicted mortality in our AHA patients.