Ritivoiu Mirela-Elena, Drăgoi Cristina Manuela, Matei Dumitru, Stan Iustina Violeta, Nicolae Alina Crenguţa, Craiu Mihai, Dumitrescu Ion-Bogdan, Ciolpan Alina Angelica
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.
Pharmaceutics. 2023 Jan 3;15(1):162. doi: 10.3390/pharmaceutics15010162.
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.
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。