在日本的儿童BRAF V600E突变脑瘤中使用未经批准和非标签药物的访问计划
Maya Suzuki1,2, Yuhki Koga1,3, Terumi Kawasaki2
1Department of Pediatrics. Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan.
Pediatric blood & cancer
|January 6, 2025
概括
访问计划为患有BRAF V600突变质瘤的儿科患者提供了研究中的达布拉费尼布加上特拉美替尼布. 这种方法促进了对严重的儿科脑瘤未经批准的药物的早期获取.
科学领域:
- 儿科瘤学 儿科瘤学
- 神经瘤学神经瘤学
- 临床药理学 临床药理学
背景情况:
- 研究性药物获取计划对于儿科癌症至关重要.
- 在治疗严重的儿科疾病时,非标签药物的使用是常见的.
- 日本患者提出的医疗保健服务促进了获得未经批准的治疗方法的机会.
研究的目的:
- 为了评估dabrafenib加上trametinib在患有BRAF V600突变质瘤的儿科患者的疗效和安全性.
- 评估日本患者提出的医疗保健服务在提供未经批准的药物早期获取方面的实用性.
主要方法:
- 一项临床研究 (jRCTs071210071) 在日本患者提出的医疗保健服务中进行.
- 四名患有BRAF V600突变质瘤的儿科患者 (2 种低级,2 种高级) 接受了达布拉费尼布加上特拉美丁尼布.
- 在获得监管部门批准之前,收集了关于疗效和安全性的数据.
主要成果:
- 该研究成功为儿科患者提供未经批准和非标签的dabrafenib加上trametinib.
- 纳入了患有低级和高级质瘤的患者.
- 招募患者的中位年龄为10.5岁.
结论:
- 患者提出的医疗保健服务可以有效地为儿童质瘤提供试验药物.
- 对儿科患者来说,需要优化计划时间表和数据收集.
- 国际医疗保健系统应该考虑调整此类计划,以实现公平的药物获取.
更多相关视频
相关概念视频
02:56Treatment Resistent Cancers
Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...
01:02Prescription, Nonprescription and Orphan Drugs
Prescription drugs require a prescription from a medical practitioner and can only be obtained from a pharmacy. They have many applications, including treating pain, anxiety, and hypertension.
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
01:26FDA Approved Drugs: Changes to Approved Drugs
Post-approval, manufacturers may modify an approved new or generic drug product. Such modifications can encompass alterations in the Active Pharmaceutical Ingredient (API), manufacturing process, formulation, batch size, manufacturing site, and container closure system (FDA Guidance for Industry, April 2004). Often, a drug product may undergo multiple changes.These modifications require careful evaluation to determine their potential impact on the drug product's identity, strength, quality,...
01:23Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption
Understanding the physiological differences in the pediatric population is crucial for effective pharmacotherapy. Neonates, infants, and children exhibit significant variations in gastric pH, gastric emptying time, intestinal transit time, and biliary function. These variations profoundly affect oral drug absorption, necessitating a nuanced approach to pediatric dosing.Neonates present with a unique physiological profile, having a gastric pH greater than 4 and faster and more irregular gastric...
01:17Pharmacokinetics in Pediatric Patients: Drug Distribution
Drug distribution in the pediatric population exhibits unique challenges and considerations due to the physiological differences between children, particularly neonates and infants, and adults. A crucial aspect of pediatric pharmacology is understanding how these differences impact the pharmacokinetics of various drugs, necessitating age-specific dosing strategies to ensure efficacy and safety.Neonates and infants have a higher total body water content, ~75%–90% of their body weight, compared...
01:24Pharmacokinetics in Pediatric Patients: Drug Metabolism
In pediatric care, understanding the nuances of hepatic drug metabolism is crucial, as it significantly differs from that of adults. This divergence is primarily due to the developmental stage of drug-metabolizing enzymes, which affects how medications are processed in the body. In neonates, for instance, the activity of Phase I enzymes—critical for the initial breakdown of drugs—is markedly reduced, functioning at just 20–40% of the levels seen in adults. This reduction poses a challenge in...