儿童骨和软组织肉瘤患者的多向激酶抑制剂疗法 - - 一个单一中心的经验中心
Anna Mohás1, Klára Horváth1, Zsuzsanna Jakab2
1Pediatric Centre, Tűzoltó Street Department, Semmelweis University, Budapest, Hungary.
Cancer medicine
|May 9, 2025
概括
多向激酶抑制剂 (mTKI) 在儿科耐火性肉瘤中表现有前途,改善了生存结果. 这些药物提供了息的选择,减缓了进展,并维持了有限治疗选择的患者的生活质量.
科学领域:
- 在瘤学瘤学.
- 儿科血液学/瘤学
- 药理学 药理学是指药理学的学科.
背景情况:
- 儿科患者复发或耐火软组织和骨肉瘤的预后不佳.
- 多向激酶抑制剂 (mTKI) 在各种恶性瘤中有效,既可作为初始疗法,也可作为挽救疗法.
研究的目的:
- 追溯评估多向激酶抑制剂 (mTKI) 治疗在多种小儿肉瘤患者群体中的临床结果.
- 评估mTKIs在患有复发性或耐火性肉瘤的儿科患者的疗效和安全性.
主要方法:
- 18名患有骨髓瘤,突肉瘤,软骨瘤或骨髓瘤的儿科患者接受了索拉费尼布,雷戈拉费尼布或帕佐帕尼布.
- 在一线和二线化疗后,治疗适用于进展,不可手术,复发或耐化疗的疾病.
- 对骨肉瘤患者的生存结果与回顾性,非随机对照组进行了比较.
主要成果:
- 患者在开始mTKI治疗时的中位数年龄为16.5岁,中位数进展时间为4个月.
- 总体响应率为16%.
- 在接受mTKIs和对照组的骨髓瘤患者之间观察到生存分布的显著差异 (p=0.004),表明使用mTKIs的生存率有所改善.
结论:
- 多向激酶抑制剂 (mTKIs) 在儿科肉瘤患者中耐受性良好.
- mTKIs代表了耐火性骨肉瘤的可行的治疗选择,作为缓解治疗来减缓疾病进展.
- 用mTKI治疗可以帮助儿童患有晚期肉瘤的患者保持良好的生活质量.
相关概念视频
02:57Targeted Cancer Therapies
7.4K
The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
There are several types of targeted therapies against...
7.4K
02:50Combination Therapies and Personalized Medicine
4.8K
Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
4.8K
02:34Inhibition of Cdk Activity
4.6K
The orderly progression of the cell cycle depends on the activation of Cdk protein by binding to its cyclin partner. However, the cell cycle must be restricted when undergoing abnormal changes. Most cancers correlate to the deregulated cell cycle, and since Cdks are a central component of the cell cycle, Cdk inhibitors are extensively studied to develop anticancer agents. For instance, cyclin D associates with several Cdks, such as Cdk 4/6, to form an active complex. The cyclin D-Cdk4/6 complex...
4.6K
01:27Transducer Mechanism: Enzyme-Linked Receptors
2.3K
Enzyme-linked receptors are cell-surface receptors acting as an enzyme or associating with an enzyme intracellularly. They make excellent drug targets. Drugs can bind to the extracellular ligand-binding domain or directly affect their enzymatic domain and alter their activity.
Major types that are helpful drug targets include:
Major types that are helpful drug targets include:
2.3K
02:49Cancer Therapies
7.4K
Cancer therapies are various modes of treatment, such as surgery, radiation therapy, and chemotherapy that are administered to cancer patients.
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...
7.4K