在小儿HSCT接受者接受埃维纳库马布后,危及生命的高甘油血症的快速解决:一个病例报告
Alice Fachin1, Chiara De Carlo2, Alessandra Maestro3
1Department of Medicine, Surgery and Health Sciences, University of Trieste, 34127 Trieste, Italy.
Pharmaceuticals (Basel, Switzerland)
|August 26, 2023
概括
埃维纳库马布有效地治疗了一名年轻男孩的严重高甘油三血症,证明了其作为儿童患者在其他治疗方法失败时安全和耐受良好选择的潜力.
科学领域:
- 药理学 药理学是指药理学的学科.
- 内分泌学 在内分泌学.
- 血液学 血液学 血液学
背景情况:
- 埃维纳库马布已被批准用于12岁及以上患者的同卵性家族性高胆固醇血症 (HoFH).
- 其降低甘油三的效果正在研究更广泛的应用.
- 耐火性高甘油三血症可能危及生命,特别是在患有复杂病史的儿科患者中.
研究的目的:
- 报告欧洲第一个12岁以下儿科患者使用埃维纳库马布的病例.
- 评估埃维纳库马布在治疗对其他治疗不耐药的严重高甘油三血症的疗效和安全性.
- 探索ANGPTL3抑制剂在儿科高甘油三血症管理中的潜力.
主要方法:
- 一个10岁男孩的病例报告,在血造细胞干细胞移植后患有耐火性过高甘油三血症.
- 用胰岛素治疗失败,无法进行脂质疏散.
- 作为治疗干预措施的埃维纳库马布的使用.
主要成果:
- 在埃维纳库马布治疗后观察到的甘油三和胆固醇水平的急剧和快速降低.
- 埃维纳库马布在这个儿科患者中是有效的,安全的,并且耐受良好.
- 在标准疗法不足时,成功管理危及生命的高甘油三血症.
结论:
- 埃维纳库马布作为治疗12岁以下儿科患者严重高甘油三血症的治疗方法具有前景.
- 抑制ANGPTL3可能是一个可行的治疗策略,用于儿科高甘油三血症,当其他选择是禁忌或无效.
- 这一案例凸显了埃维纳库马布的潜在扩展,超出其目前FDA批准的适用范围.
相关概念视频
01:29Venous Thrombosis III: Interprofessional Care
10
Venous thrombosis requires effective prevention and treatment strategies to improve patient outcomes and reduce potential complications.Prevention StrategiesHealthcare providers must prioritize preventing venous thromboembolism (VTE) for all adult patients upon admission. Interventions depend on bleeding and thrombosis risk, medical history, current medications, diagnoses, planned procedures, and patient preferences. Patients on bed rest should change positions every two hours and, if not...
10
01:29Pulmonary Embolism II: Diagnostic Studies and Interprofessional Care
10
Diagnosing Pulmonary EmbolismDiagnosing pulmonary embolism (PE) involves clinical assessment and advanced imaging tests. The preferred diagnostic tool is the spiral (helical) CT scan or CT angiography (CTA), which uses intravenous contrast media to visualize the pulmonary vasculature and identify emboli.A ventilation-perfusion (V/Q) scan is an alternative for patients unable to receive contrast media. This scan includes both perfusion and ventilation scanning. Perfusion scanning involves...
10
01:23Treatment for Pulmonary Arterial Hypertension: Prostacyclin Receptor Agonists
215
Prostacyclin receptor agonists are a class of therapeutic agents integral to managing pulmonary arterial hypertension (PAH). These drugs operate by mimicking the action of prostaglandin I2, or PGI2, a naturally occurring compound in the body.
These agonists bind to the IPR receptor situated on the plasma membrane of the pulmonary artery smooth muscle cells. This binding triggers a cascade of reactions known as the GS-AC-cAMP-PKA pathway. This pathway results in the relaxation of smooth muscle...
These agonists bind to the IPR receptor situated on the plasma membrane of the pulmonary artery smooth muscle cells. This binding triggers a cascade of reactions known as the GS-AC-cAMP-PKA pathway. This pathway results in the relaxation of smooth muscle...
215
01:18Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists
201
Endothelins (ETs) are potent vasoactive peptides critical in the human body's various physiological and pathological processes. One of the most promising therapeutic strategies for treating pulmonary arterial hypertension (PAH) involves counteracting the effects of these endothelins using a class of drugs known as endothelin receptor antagonists.
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme...
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme...
201