Suppr超能文献

个性化医疗时代囊性纤维化患儿外分泌性胰腺功能不全的当前及未来治疗方法

Current and Future Therapeutic Approaches of Exocrine Pancreatic Insufficiency in Children with Cystic Fibrosis in the Era of Personalized Medicine.

作者信息

Ritivoiu Mirela-Elena, Drăgoi Cristina Manuela, Matei Dumitru, Stan Iustina Violeta, Nicolae Alina Crenguţa, Craiu Mihai, Dumitrescu Ion-Bogdan, Ciolpan Alina Angelica

机构信息

Faculty of Medicine, "Carol Davila" University of Medicine and Pharmacy, 050474 Bucharest, Romania.

Alessandrescu-Rusescu National Institute for Mother and Child Health, 020395 Bucharest, Romania.

出版信息

Pharmaceutics. 2023 Jan 3;15(1):162. doi: 10.3390/pharmaceutics15010162.

Abstract

This review presents current updates of pancreatic enzyme replacement therapy in children with cystic fibrosis based on literature published in the last decade and some special considerations regarding pancreatic enzyme replacement therapy in the era of new therapies, such as cystic fibrosis transmembrane conductance regulator modulator therapies. Few articles evaluate the efficacy of pancreatic enzyme replacement therapy in the pediatric population, and most studies also included children and adults with cystic fibrosis. Approximately 85% of cystic fibrosis patients have exocrine pancreatic insufficiency and need pancreatic enzyme replacement therapy. Fecal elastase is the most commonly used diagnostic test for exocrine pancreatic insufficiency, although this value can fluctuate over time. While it is used as a diagnostic test, it cannot be used for monitoring the effectiveness of pancreatic enzyme replacement therapy and for adjusting doses. Pancreatic enzyme replacement therapy, the actual treatment for exocrine pancreatic insufficiency, is essential in children with cystic fibrosis to prevent malabsorption and malnutrition and needs to be urgently initiated. This therapy presents many considerations for physicians, patients, and their families, including types and timing of administration, dose monitoring, and therapy failures. Based on clinical trials, pancreatic enzyme replacement therapy is considered effective and well-tolerated in children with cystic fibrosis. An important key point in cystic fibrosis treatment is the recent hypothesis that cystic fibrosis transmembrane conductance regulator modulators could improve pancreatic function, further studies being essential. Pancreatic enzyme replacement therapy is addressed a complication of the disease (exocrine pancreatic insufficiency), while modulators target the defective cystic fibrosis transmembrane conductance regulator protein. Exocrine pancreatic insufficiency in cystic fibrosis remains an active area of research in this era of cystic fibrosis transmembrane conductance regulator modulator therapies. This new therapy could represent an example of personalized medicine in cystic fibrosis patients, with each class of modulators being addressed to patients with specific genetic mutations.

摘要

本综述基于过去十年发表的文献,介绍了囊性纤维化患儿胰酶替代疗法的最新进展,以及在新疗法时代(如囊性纤维化跨膜传导调节因子调节剂疗法)关于胰酶替代疗法的一些特殊考虑。很少有文章评估胰酶替代疗法在儿科人群中的疗效,大多数研究还纳入了患有囊性纤维化的儿童和成人。大约85%的囊性纤维化患者存在外分泌性胰腺功能不全,需要胰酶替代疗法。粪便弹性蛋白酶是外分泌性胰腺功能不全最常用的诊断测试,尽管该值可能随时间波动。虽然它被用作诊断测试,但不能用于监测胰酶替代疗法的有效性和调整剂量。胰酶替代疗法是外分泌性胰腺功能不全的实际治疗方法,对囊性纤维化患儿预防吸收不良和营养不良至关重要,需要紧急启动。这种疗法给医生、患者及其家属带来了许多需要考虑的问题,包括给药类型和时间、剂量监测以及治疗失败等。基于临床试验,胰酶替代疗法在囊性纤维化患儿中被认为是有效且耐受性良好的。囊性纤维化治疗中的一个重要关键点是最近的假说,即囊性纤维化跨膜传导调节因子调节剂可能改善胰腺功能,进一步的研究至关重要。胰酶替代疗法针对的是该疾病的一种并发症(外分泌性胰腺功能不全),而调节剂针对的是有缺陷的囊性纤维化跨膜传导调节因子蛋白。在囊性纤维化跨膜传导调节因子调节剂疗法的这个时代,囊性纤维化中的外分泌性胰腺功能不全仍然是一个活跃的研究领域。这种新疗法可能代表了囊性纤维化患者个性化医疗的一个例子,每一类调节剂都针对具有特定基因突变的患者。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4b8f/9862205/7de206f90d78/pharmaceutics-15-00162-g001.jpg

相似文献

2
[Chinese experts consensus statement: diagnosis and treatment of cystic fibrosis (2023)].
Zhonghua Jie He He Hu Xi Za Zhi. 2023 Apr 12;46(4):352-372. doi: 10.3760/cma.j.cn112147-20221214-00971.
3
Changes in fecal elastase-1 following initiation of CFTR modulator therapy in pediatric patients with cystic fibrosis.
J Cyst Fibros. 2023 Nov;22(6):996-1001. doi: 10.1016/j.jcf.2023.09.005. Epub 2023 Sep 26.
4
Pancreatic complications in children with cystic fibrosis.
Curr Opin Pediatr. 2020 Oct;32(5):661-667. doi: 10.1097/MOP.0000000000000934.
5
Cystic fibrosis transmembrane conductance regulator modulators and the exocrine pancreas: A scoping review.
J Cyst Fibros. 2023 Mar;22(2):193-200. doi: 10.1016/j.jcf.2022.08.008. Epub 2022 Aug 23.
6
Persistent recovery of pancreatic function in patients with cystic fibrosis after ivacaftor.
Pediatr Pulmonol. 2020 Dec;55(12):3381-3383. doi: 10.1002/ppul.25065. Epub 2020 Oct 22.
9
AGA Clinical Practice Update on the Epidemiology, Evaluation, and Management of Exocrine Pancreatic Insufficiency: Expert Review.
Gastroenterology. 2023 Nov;165(5):1292-1301. doi: 10.1053/j.gastro.2023.07.007. Epub 2023 Sep 20.
10
Time for a gut check: Pancreatic sufficiency resulting from CFTR modulator use.
Pediatr Pulmonol. 2019 Aug;54(8):E16-E18. doi: 10.1002/ppul.24353. Epub 2019 May 7.

引用本文的文献

1
Fecal calprotectin as a marker of intestinal inflammation in pediatric cystic fibrosis patients.
Eur J Pediatr. 2025 Sep 8;184(10):605. doi: 10.1007/s00431-025-06442-9.
4
Current and future therapeutic approaches of CFTR and airway dysbiosis in an era of personalized medicine.
J Family Med Prim Care. 2024 Jun;13(6):2200-2208. doi: 10.4103/jfmpc.jfmpc_1085_23. Epub 2024 Jun 14.
5
A review: Pancreatic enzymes in the treatment of chronic pancreatic insufficiency in companion animals.
J Vet Intern Med. 2024 Jul-Aug;38(4):2026-2033. doi: 10.1111/jvim.17096. Epub 2024 May 9.
6
Functional Consequences of CFTR Interactions in Cystic Fibrosis.
Int J Mol Sci. 2024 Mar 16;25(6):3384. doi: 10.3390/ijms25063384.
7
Exocrine Pancreatic Insufficiency in Children - Challenges in Management.
Pediatric Health Med Ther. 2023 Oct 26;14:361-378. doi: 10.2147/PHMT.S402589. eCollection 2023.

本文引用的文献

1
Molecular analysis of gene mutations among Iraqi cystic fibrosis patients.
Egypt J Med Hum Genet. 2021;22(1):45. doi: 10.1186/s43042-021-00164-x. Epub 2021 May 11.
2
Nutritional and metabolic management for cystic fibrosis in a post-cystic fibrosis transmembrane conductance modulator era.
Curr Opin Pulm Med. 2022 Nov 1;28(6):577-583. doi: 10.1097/MCP.0000000000000917. Epub 2022 Sep 14.
3
Personalized medicine with drugs targeting the underlying protein defect in cystic fibrosis: is monitoring of treatment response necessary?
Ther Adv Chronic Dis. 2022 Aug 5;13:20406223221108627. doi: 10.1177/20406223221108627. eCollection 2022.
4
The Exocrine Pancreas in Cystic Fibrosis in the Era of CFTR Modulation: A Mini Review.
Front Pediatr. 2022 Jun 27;10:914790. doi: 10.3389/fped.2022.914790. eCollection 2022.
6
Pancreatic Enzyme Replacement Therapy in Cystic Fibrosis.
Nutrients. 2022 Mar 23;14(7):1341. doi: 10.3390/nu14071341.
9
Understanding Cystic Fibrosis Comorbidities and Their Impact on Nutritional Management.
Nutrients. 2022 Feb 28;14(5):1028. doi: 10.3390/nu14051028.

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验