Abstract：
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.
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.
摘要：
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。
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。
