PDF(Pubmed)
Abstract：
UNASSIGNED: Wilson\'s disease (WD) is not an uncommon genetic disease in clinical practice. However, the current WD therapies have limitations. The effectiveness of stem cell therapy in treating WD has yet to be verified, although a few animal studies have shown that stem cell transplantation could partially correct the abnormal metabolic phenotype of WD. In this case report, we present the therapeutic effect of human amniotic fluid containing stem cells in one WD patient.
UNASSIGNED: A 22-year-old Chinese woman was diagnosed with WD 1 year ago in 2019. The available drugs were not effective in managing the progressive neuropsychiatric symptoms. We treated the patient with pre-cultured human amniotic fluid containing stem cells. Amniotic fluid was collected from pregnant women who underwent induced labor at a gestational age of 19-26 weeks, and then, the fluid was cultured for 2 h to allow stem cell expansion. Cultured amniotic fluid that contained amniotic fluid derived stem cells (AFSC) in the range of approximately 2.8-5.5 × 104/ml was administrated by IV infusion at a rate of 50-70 drops per minute after filtration with a 300-mu nylon mesh. Before the infusion of amniotic fluid, low-molecular-weight heparin and dexamethasone were successively administrated. The patient received a total of 12 applications of amniotic fluid from different pregnant women, and the treatment interval depended on the availability of amniotic fluid. The neuropsychiatric symptoms gradually improved after the stem cell treatment. Dystonia, which included tremor, chorea, dysphagia, dysarthria, and drooling, almost disappeared after 1.5 years of follow-up. The Unified Wilson\'s Disease Rating Scale score of the patient decreased from 72 to 10. Brain magnetic resonance imaging (MRI) showed a reduction in the lesion area and alleviation of damage in the central nervous system, along with a partial recovery of the lesion to the normal condition. The serum ceruloplasmin level was elevated from undetectable to 30.8 mg/L, and the 24-h urinary copper excretion decreased from 171 to 37 μg. In addition, amniotic fluid transplantation also alleviates hematopoietic disorders. There were no adverse reactions during or after amniotic fluid administration.
UNASSIGNED: Amniotic fluid administration, through which stem cells were infused, significantly improves the clinical outcomes in the WD patient, and the finding may provide a novel approach for managing WD effectively.
UNASSIGNED: A 22-year-old Chinese woman was diagnosed with WD 1 year ago in 2019. The available drugs were not effective in managing the progressive neuropsychiatric symptoms. We treated the patient with pre-cultured human amniotic fluid containing stem cells. Amniotic fluid was collected from pregnant women who underwent induced labor at a gestational age of 19-26 weeks, and then, the fluid was cultured for 2 h to allow stem cell expansion. Cultured amniotic fluid that contained amniotic fluid derived stem cells (AFSC) in the range of approximately 2.8-5.5 × 104/ml was administrated by IV infusion at a rate of 50-70 drops per minute after filtration with a 300-mu nylon mesh. Before the infusion of amniotic fluid, low-molecular-weight heparin and dexamethasone were successively administrated. The patient received a total of 12 applications of amniotic fluid from different pregnant women, and the treatment interval depended on the availability of amniotic fluid. The neuropsychiatric symptoms gradually improved after the stem cell treatment. Dystonia, which included tremor, chorea, dysphagia, dysarthria, and drooling, almost disappeared after 1.5 years of follow-up. The Unified Wilson\'s Disease Rating Scale score of the patient decreased from 72 to 10. Brain magnetic resonance imaging (MRI) showed a reduction in the lesion area and alleviation of damage in the central nervous system, along with a partial recovery of the lesion to the normal condition. The serum ceruloplasmin level was elevated from undetectable to 30.8 mg/L, and the 24-h urinary copper excretion decreased from 171 to 37 μg. In addition, amniotic fluid transplantation also alleviates hematopoietic disorders. There were no adverse reactions during or after amniotic fluid administration.
UNASSIGNED: Amniotic fluid administration, through which stem cells were infused, significantly improves the clinical outcomes in the WD patient, and the finding may provide a novel approach for managing WD effectively.
摘要：
■威尔逊病（WD）在临床实践中并不罕见。然而,目前的WD治疗有局限性。干细胞疗法治疗WD的有效性还有待验证,尽管一些动物研究表明干细胞移植可以部分纠正WD的异常代谢表型。在这个案例报告中,我们介绍了含干细胞的人羊水对一名WD患者的治疗效果。
■一名22岁的中国女性在2019年1年前被诊断出患有WD。可用的药物在控制进行性神经精神症状方面无效。我们用含有干细胞的预培养的人羊水治疗患者。羊水收集的孕妇在19-26周孕时进行引产，然后,将该液体培养2小时以允许干细胞扩增。含有羊水来源的干细胞(AFSC)的培养的羊水在约2.8-5.5×104/ml的范围内,在用300μm尼龙网过滤后,以每分钟50-70滴的速率通过IV输注施用。在输注羊水之前，依次给予低分子肝素和地塞米松.病人共接受了12次不同孕妇的羊水应用,治疗间隔取决于羊水的可用性。干细胞治疗后神经精神症状逐渐好转。肌张力障碍,其中包括震颤，舞蹈病,吞咽困难,构音障碍,流口水,1.5年随访后几乎消失.患者的统一威尔逊疾病评定量表评分从72降至10。脑磁共振成像（MRI）显示病变面积减少，中枢神经系统损伤减轻，随着病变部分恢复到正常状态。血清铜蓝蛋白水平从检测不到升高至30.8mg/L,24小时尿铜排泄量从171μg下降到37μg。此外,羊水移植还可以缓解造血障碍。在羊水给药期间或之后均未出现不良反应。
■羊水管理，通过它注入干细胞，显着改善WD患者的临床结局，这一发现可以提供一种有效管理WD的新方法。
■一名22岁的中国女性在2019年1年前被诊断出患有WD。可用的药物在控制进行性神经精神症状方面无效。我们用含有干细胞的预培养的人羊水治疗患者。羊水收集的孕妇在19-26周孕时进行引产，然后,将该液体培养2小时以允许干细胞扩增。含有羊水来源的干细胞(AFSC)的培养的羊水在约2.8-5.5×104/ml的范围内,在用300μm尼龙网过滤后,以每分钟50-70滴的速率通过IV输注施用。在输注羊水之前，依次给予低分子肝素和地塞米松.病人共接受了12次不同孕妇的羊水应用,治疗间隔取决于羊水的可用性。干细胞治疗后神经精神症状逐渐好转。肌张力障碍,其中包括震颤，舞蹈病,吞咽困难,构音障碍,流口水,1.5年随访后几乎消失.患者的统一威尔逊疾病评定量表评分从72降至10。脑磁共振成像（MRI）显示病变面积减少，中枢神经系统损伤减轻，随着病变部分恢复到正常状态。血清铜蓝蛋白水平从检测不到升高至30.8mg/L,24小时尿铜排泄量从171μg下降到37μg。此外,羊水移植还可以缓解造血障碍。在羊水给药期间或之后均未出现不良反应。
■羊水管理，通过它注入干细胞，显着改善WD患者的临床结局，这一发现可以提供一种有效管理WD的新方法。
