在爱尔兰儿科队列中使用托法西替尼的结果
Niamh Ryan1, Sarah Cooper1,2, Anna Dominik1,2
1From the National Centre for Pediatric Gastroenterology, Children's Health Ireland, Dublin, Ireland.
JPGN reports
|August 21, 2023
概括
托法西替尼在儿科性结肠炎中有效诱导缓解,特别是在与生物药物相结合时. 对托法西提尼布治疗的早期反应是关键的,因为在16周内未能达到缓解预测了切除术.
科学领域:
- 胃肠病学 胃肠病学
- 儿科炎症性肠病 儿科炎症性肠病
- 药理学 药理学是指药理学的学科.
背景情况:
- 儿童性结肠炎 (UC) 往往比成人疾病更严重.
- 托法西提尼布是一种Janus激酶抑制剂,已被批准用于UC治疗,但儿科数据仍然有限.
- 需要对托法西提尼布在儿科UC使用的真实数据,特别是在组合疗法中.
研究的目的:
- 评估托法西提尼布在全国儿科UC患者队列中的实际有效性和结果.
- 评估托法西提尼布作为单一疗法的疗效与儿科UC的组合疗法.
- 在接受托法西提尼布的儿科UC患者中确定治疗成功或失败的预测因素.
主要方法:
- 从2019年到2022年接受托法西提尼布治疗的15名儿科UC患者的回顾性分析.
- 收集的数据包括基线特征,儿科性结肠炎活动指数 (PUCAI) 评分,以及之前/同时接受的治疗.
- 主要结局是8周临床缓解;二次结局评估到最大后续.
主要成果:
- 在15名患者中,有8名患者在8周内实现了临床缓解;在5名因弗利克西马布双疗中的4名患者有反应.
- 平均PUCAI得分从治疗前的48.7显著下降到第8周的16.7 (P=0.0004).
- 四名患者需要进行大肠切除术,所有患者在第16周未能达到缓解;一个患上带状带,另一个患上带状带.
结论:
- 托法西提尼布,特别是与生物药物如因弗利克西马布结合使用,在耐药儿科UC中显示出有效性.
- 在第16周早期实现临床缓解是持续反应的强有力的预测指标.
- 在第16周未能实现缓解与更高的胆管切除的可能性有关.
相关概念视频
Drugs for Treatment of Crohn's Disease in IBD Using Immunomodulatory Agents
195
Crohn's disease is an inflammatory bowel disorder marked by chronic inflammation of the GI tract. Various treatment strategies for Crohn's disease are employed, such as immunomodulatory agents, glucocorticoids, and biologics or anti-TNF therapy. Azathioprine (Imuran), a commonly used immunomodulatory drug for Crohn's disease, is converted in the body to mercaptopurine, which inhibits purine biosynthesis and cell proliferation. Both are utilized in severe cases of Inflammatory Bowel...
195
Drugs for Treatment of Crohn's Disease in IBD Using Biologic Agents: Anti-TNF
173
Tumor Necrosis Factor (TNF), a proinflammatory cytokine, contributes significantly to the inflammation seen in Crohn's disease. It exists as soluble TNF and membrane-bound TNF, with actions mediated through TNF receptors (TNFR). TNFR activation leads to the release of proinflammatory cytokines, T-cell activation, collagen production, and leukocyte migration, all contributing to inflammation in Crohn's disease. Anti-TNF monoclonal antibodies, namely infliximab (Remicade), adalimumab...
173
Drugs for Treatment of Crohn's Disease in IBD Using Glucocorticoids
150
Glucocorticoids, a class of anti-inflammatory drugs, are pivotal in treating moderate to severe Crohn's disease by inducing remission. They exhibit their anti-inflammatory action by inhibiting the production of inflammatory cytokines such as tumor necrosis factor (TNF)-α, interleukin (IL)-1, and chemokines like IL-8. In addition, they reduce the expression of inflammatory cell adhesion molecules and inhibit gene transcription of nitric oxide synthase, phospholipase A2, cyclooxygenase-2...
150
Drugs for Treatment of Ulcerative Colitis in IBD
173
Ulcerative colitis is a chronic inflammatory condition primarily affecting the colon and rectum. The primary drugs used in the treatment of ulcerative colitis are aminosalicylates. They exhibit anti-inflammatory and immunosuppressive properties. They modulate inflammatory mediators and inhibit the activity of nuclear factor κB (NF-κB). Aminosalicylates also reduce inflammation by inhibiting prostaglandin and leukotriene production and decreasing neutrophil chemotaxis and superoxide...
173
Cystic Fibrosis: Management
194
Cystic fibrosis (CF) is an autosomal recessive disorder that predominantly affects individuals of Northern European descent, occurring at a rate of 1 in 3500. It is caused by a genetic mutation in a gene on chromosome 7, most commonly the ΔF508 mutation, that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This results in thicker mucus secretions and obstruction pathologies in multiple organs, including the lungs and sinuses.
Sinus disease and chronic...
Sinus disease and chronic...
194
Clinical Trials: Overview
3.0K
Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
3.0K
![Dynamic Imaging of Chimeric Antigen Receptor T Cells with [18F]Tetrafluoroborate Positron Emission Tomography/Computed Tomography](/_next/image?url=https%3A%2F%2Fcloudfront.jove.com%2FCDNSource%2Fteasers%2F62334.jpg&w=3840&q=50)

