埃弗罗利斯对儿科结核硬化综合症患者的囊性病减轻负担:一个病例系列
Sumona Banerjee1, Louis Richard Feldenberg2
1Saint Louis University School of Medicine, 1402 S Grand Blvd, St. Louis, MO, 63104, USA. sumona.banerjee@health.slu.edu.
BMC nephrology
|October 25, 2024
概括
作为mTORC1抑制剂的Everolimus通过减少SEGA和发作,有效治疗结核性硬化综合体 (TSC). 这项研究强调了其在减轻儿科TSC患者囊性病负担方面的好处.
科学领域:
- 儿科脏病学 儿科脏病学
- 在瘤学瘤学.
- 遗传学 遗传学 是一个
背景情况:
- 结核性硬化综合体 (TSC) 是一种影响多个系统的遗传性疾病.
- TSC可以导致严重的并发症,如SEGA,脏血管脂瘤和囊性病.
- 埃弗罗利斯 (mTORC1抑制剂) 已被批准用于TSC,主要用于SEGA和,在成年人中已知有性益处.
研究的目的:
- 报告有关Everolimus对儿科TSC患者囊性病的影响的偶然发现.
- 评估Everolimus对脏囊负担的影响,并与SEGA和管理相结合.
主要方法:
- 三名患有TSC的儿科患者用everolimus治疗的病例报告.
- 在Everolimus治疗期间监测SEGA体积,发作频率和囊性病负担.
- 在一个患者停止使用everolimus后观察囊负担的变化.
主要成果:
- 在观察到的儿科患者中,Everolimus治疗导致囊性病的消失或减轻其负担.
- 在一个案例中,在停止服用everolimus后,囊负担保持稳定,而SEGA则恢复.
- 埃弗罗利斯表现出超越SEGA和发作的疗效,影响囊性脏并发症.
结论:
- 埃弗罗利斯在治疗儿科TSC患者的囊性病方面具有实用性,补充了其在治疗血管脂瘤方面的作用.
- 早期使用Everolimus可以保持功能,并预防囊造成的血和出血等并发症.
- 这表明everolimus在TSC的治疗作用更广泛,特别是在儿童的脏表现方面.
相关概念视频
Drug Elimination by Renal Route: Tubular Secretion
2.2K
Once the process of glomerular filtration is completed, blood carrying unfiltered drug molecules traverses through efferent arterioles and makes its way into the peritubular capillaries in the proximal tubule. A variety of carriers play a pivotal role in actively secreting drugs from these peritubular capillaries into the tubular fluid. The organic anion transporter transfers acidic drugs, against an electrochemical gradient, from the peritubular capillaries into the renal tubule cells and...
2.2K
Renal Failure: Dose Adjustments
67
In patients with renal impairment, drugs undergo significant changes in their pharmacokinetics, which require dosage adjustments to ensure safe and effective therapy.
Reduced renal clearance and elimination rate are common outcomes of renal impairment. These alterations lead to a prolonged elimination half-life and an altered apparent volume of distribution for drugs. As a result, dosage adjustments are typically necessary to maintain optimal drug levels in the body.
However, dosage adjustments...
Reduced renal clearance and elimination rate are common outcomes of renal impairment. These alterations lead to a prolonged elimination half-life and an altered apparent volume of distribution for drugs. As a result, dosage adjustments are typically necessary to maintain optimal drug levels in the body.
However, dosage adjustments...
67
Renal Drug Excretion: Tubular Secretion
144
Active tubular secretion is a robust, energy-demanding process that utilizes carrier systems to transport drugs into renal tubules. The active renal secretion systems include the organic anion transporter (OAT) for weak acids and the organic cation transporter (OCT) for weak bases. Structurally similar drugs can compete for the same transporter, potentially leading to drug accumulation and toxicity. However, this principle can be exploited therapeutically. One example is probenecid (Probalan),...
144
Nephrotic Syndrome III : Nursing Management
1
Nursing management for nephrotic syndrome adapts as the disease progresses, with strategies evolving to address advancing symptoms and complications.Early-Stage Management In the early stages, nursing interventions for nephrotic syndrome resemble those used in managing acute glomerulonephritis, focusing on symptom monitoring, fluid balance, and managing mild to moderate edema.Vital Signs: Regularly monitor blood pressure, pulse, respiratory rate, and temperature to promptly identify...
1
Drug Elimination by Renal Route: Tubular Reabsorption
3.2K
During the process of renal excretion, as the glomerular filtrate progresses to the distal convoluted tubule (DCT), drugs that are highly permeable, lipophilic, and nonionized undergo passive reabsorption from the tubular fluid into the surrounding peritubular capillaries. This reabsorption process restricts their elimination through the kidneys. However, the majority of drugs are either weak acids or weak bases, and their ionization level is dependent on pH. By altering the pH of urine, the...
3.2K
Antiepileptic Drugs: Potassium Channel Activators
147
Ezocgabine or retigabine, an antiepileptic drug of remarkable efficacy, has revolutionized the management of seizures. It is a potassium channel activator, explicitly targeting the family of Q subtype potassium channels. It enhances the transmembrane potassium currents, regulating neuronal excitability. This action stabilizes the resting membrane potential, a pivotal factor in mitigating the hyperexcitability that characterizes epilepsy.
Ezogabine has gained approval as an adjunctive treatment...
Ezogabine has gained approval as an adjunctive treatment...
147


