Alpelisib: A Novel Agent for PIK3CA-Related Overgrowth Spectrum.

Q2 Medicine Journal of Pediatric Pharmacology and Therapeutics Pub Date : 2023-01-01 Epub Date: 2023-11-23 DOI:10.5863/1551-6776-28.7.590
Katarina Nadjkovic, Kevin Lonabaugh
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Abstract

The aim of this review is to present the information a clinician will need when considering alpelisib therapy for a patient diagnosed with PIK3CA-related overgrowth spectrum (PROS). PROS is a condition caused by a somatic recessive gain-of-function mutation in the gene encoding phosphatidylinositol-3-kinase (PI3K). PROS is rare, affecting approximately 14 births per 1 million. PROS affects many different tissues including skin, bone, vascular, adipose, and connective tissues, thus its presentations vary widely. The presentation of PROS is often described as mosaic, as the disease typically does not affect all cells in the body. For patients two years of age and older requiring systemic therapy, alpelisib is an option which was recently granted accelerated approval by the US Food and Drug Administration (FDA) on April 5, 2022. Alpelisib is an inhibitor of PI3K, slowing the progression of existing lesions and preventing new lesions in patients with PROS. Important drug interactions exist with both CYP3A4 inducers and CYP2C9 substrates. Additionally, providers of patients receiving alpelisib should be aware of potential side effects including hypersensitivity, severe cutaneous adverse reactions, hyperglycemia, pneumonitis, diarrhea, and embryo-fetal toxicity. Despite the potential for adverse events, alpelisib has provided clinical benefit to many patients with PROS as evidenced by the current literature. This review collects and summarizes the currently available evidence, including a recently published case series and multiple case reports. Alpelisib is a promising new option for patients with PROS.

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Alpelisib:一种与pik3ca相关的过度生长谱的新型药物。
本综述的目的是提供临床医生在考虑对诊断为pik3ca相关过度生长谱(PROS)的患者使用alpelisib治疗时需要的信息。PROS是一种由编码磷脂酰肌醇-3激酶(PI3K)基因的体细胞隐性功能获得突变引起的疾病。PROS很少见,大约每100万新生儿中有14例。PROS影响许多不同的组织,包括皮肤、骨骼、血管、脂肪和结缔组织,因此其表现差异很大。PROS的表现通常被描述为马赛克,因为这种疾病通常不会影响体内的所有细胞。对于两岁及以上需要全身治疗的患者,alpelisib是最近于2022年4月5日获得美国食品和药物管理局(FDA)加速批准的一种选择。Alpelisib是一种PI3K抑制剂,在PROS患者中减缓现有病变的进展并预防新病变。重要的药物相互作用存在于CYP3A4诱导剂和CYP2C9底物。此外,患者接受alpelisib的提供者应该意识到潜在的副作用,包括过敏、严重的皮肤不良反应、高血糖、肺炎、腹泻和胚胎-胎儿毒性。尽管存在潜在的不良事件,但alpelisib已经为许多PROS患者提供了临床益处,这是目前文献所证明的。本综述收集并总结了目前可获得的证据,包括最近发表的病例系列和多个病例报告。Alpelisib对PROS患者来说是一个很有希望的新选择。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Journal of Pediatric Pharmacology and Therapeutics
Journal of Pediatric Pharmacology and Therapeutics Medicine-Pediatrics, Perinatology and Child Health
CiteScore
2.40
自引率
0.00%
发文量
90
期刊介绍: The Journal of Pediatric Pharmacology and Therapeutics is the official journal of the Pediatric Pharmacy Advocacy Group. JPPT is a peer-reviewed multi disciplinary journal that is devoted to promoting the safe and effective use of medications in infants and children. To this end, the journal publishes practical information for all practitioners who provide care to pediatric patients. Each issue includes review articles, original clinical investigations, case reports, editorials, and other information relevant to pediatric medication therapy. The Journal focuses all work on issues related to the practice of pediatric pharmacology and therapeutics. The scope of content includes pharmacotherapy, extemporaneous compounding, dosing, methods of medication administration, medication error prevention, and legislative issues. The Journal will contain original research, review articles, short subjects, case reports, clinical investigations, editorials, and news from such organizations as the Pediatric Pharmacy Advocacy Group, the FDA, the American Academy of Pediatrics, the American Society of Health-System Pharmacists, and so on.
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