囊性纤维化的生长障碍和治疗。
Growth failure and treatment in cystic fibrosis.
机构信息
Departments of Internal Medicine and Pediatrics, Division of Endocrinology, Virginia Commonwealth University, Richmond, VA, USA.
Department of Internal Medicine, University of Kansas Medical Center, Kansas City, KS, USA.
出版信息
J Cyst Fibros. 2019 Oct;18 Suppl 2(Suppl 2):S82-S87. doi: 10.1016/j.jcf.2019.08.010.
Abstract
Poor growth has long been a characteristic feature of cystic fibrosis (CF) and is significantly linked to lung function and overall health status. Improvements in pulmonary and nutrition care for patients with cystic fibrosis (CF) have resulted in better growth outcomes; however, height gains have not paralleled the improvements in weight in children with CF, and patients with more severe CF mutations remain significantly more affected. Many factors affect the growth hormone-IGF-1 axis and the growth plate of the long bones, including the chronic inflammatory state associated with CF. There are also increasing data on the direct effects of CFTR on bone and implications for CFTR modulators in attaining optimal growth. Treatments aimed at improving growth in CF are also reviewed here.
摘要
长期以来，生长不良一直是囊性纤维化（CF）的特征之一，与肺功能和整体健康状况密切相关。对囊性纤维化（CF）患者的肺部和营养护理的改善导致了更好的生长结果；然而，身高的增长并没有与 CF 儿童的体重增长相匹配，并且具有更严重 CF 突变的患者仍然受到显著影响。许多因素会影响生长激素-IGF-1 轴和长骨的生长板，包括与 CF 相关的慢性炎症状态。也有越来越多的数据表明 CFTR 对骨骼的直接影响，以及 CFTR 调节剂在实现最佳生长方面的影响。本文还回顾了旨在改善 CF 生长的治疗方法。
相似文献
1
Growth failure and treatment in cystic fibrosis.
J Cyst Fibros. 2019 Oct;18 Suppl 2(Suppl 2):S82-S87. doi: 10.1016/j.jcf.2019.08.010.
2
The relationship between insulin secretion, the insulin-like growth factor axis and growth in children with cystic fibrosis.
Clin Endocrinol (Oxf). 2002 Mar;56(3):383-9. doi: 10.1046/j.1365-2265.2002.01484.x.
3
Somatic growth in cystic fibrosis.
Curr Opin Endocrinol Diabetes Obes. 2020 Feb;27(1):38-46. doi: 10.1097/MED.0000000000000522.
4
Growth failure in children with cystic fibrosis.
J Pediatr Endocrinol Metab. 2012;25(5-6):393-405. doi: 10.1515/jpem-2012-0012.
5
Recombinant growth hormone therapy for cystic fibrosis in children and young adults.
Cochrane Database Syst Rev. 2018 Dec 17;12(12):CD008901. doi: 10.1002/14651858.CD008901.pub4.
6
[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.
7
Cystic-fibrosis related-diabetes (CFRD) is preceded by and associated with growth failure and deteriorating lung function.
J Pediatr Endocrinol Metab. 2017 Aug 28;30(8):815-821. doi: 10.1515/jpem-2017-0005.
8
Most Short Children with Cystic Fibrosis Do Not Catch Up by Adulthood.
Nutrients. 2021 Dec 10;13(12):4414. doi: 10.3390/nu13124414.
9
Growth hormone treatment in children with cystic fibrosis.
Minerva Pediatr. 2002 Oct;54(5):365-71.
10
Growth status in children with cystic fibrosis based on the National Cystic Fibrosis Patient Registry data: evaluation of various criteria used to identify malnutrition.
J Pediatr. 1998 Mar;132(3 Pt 1):478-85. doi: 10.1016/s0022-3476(98)70024-1.
引用本文的文献
1
Cystic fibrosis management in pediatric population-from clinical features to personalized therapy.
Front Pediatr. 2024 May 10;12:1393193. doi: 10.3389/fped.2024.1393193. eCollection 2024.
2
Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2-5 Years with Cystic Fibrosis and at Least One Allele.
Am J Respir Crit Care Med. 2023 Jul 1;208(1):59-67. doi: 10.1164/rccm.202301-0084OC.
3
Serum levels of hormones regulating appetite in patients with cystic fibrosis - a single-center, cross-sectional study.
Front Endocrinol (Lausanne). 2022 Oct 13;13:992667. doi: 10.3389/fendo.2022.992667. eCollection 2022.
4
New insight into the importance of formulation variables on parenteral growth hormone preparations: potential effect on the injection-site pain.
Front Endocrinol (Lausanne). 2022 Oct 3;13:963336. doi: 10.3389/fendo.2022.963336. eCollection 2022.
5
Appetite stimulants for people with cystic fibrosis.
Cochrane Database Syst Rev. 2022 Sep 23;9(9):CD008190. doi: 10.1002/14651858.CD008190.pub3.
6
Evaluation of Growth Hormone Deficiency in Children with Cystic Fibrosis.
Adv Biomed Res. 2022 Jul 29;11:62. doi: 10.4103/abr.abr_285_20. eCollection 2022.
7
The association between Sodium Urinary Discharge (FENa) and growth parameters in pediatrics with cystic fibrosis.
Am J Clin Exp Urol. 2022 Aug 15;10(4):246-251. eCollection 2022.
8
Case report of a Hispanic female with cystic fibrosis and short stature.
Respir Med Case Rep. 2022 Aug 12;39:101726. doi: 10.1016/j.rmcr.2022.101726. eCollection 2022.
9
Trends in Growth and Maturation in Children with Cystic Fibrosis Throughout Nine Decades.
Front Endocrinol (Lausanne). 2022 Jul 12;13:935354. doi: 10.3389/fendo.2022.935354. eCollection 2022.
10
A Phase 3, Open-Label Study of Lumacaftor/Ivacaftor in Children 1 to Less Than 2 Years of Age with Cystic Fibrosis Homozygous for .
Am J Respir Crit Care Med. 2022 Nov 15;206(10):1239-1247. doi: 10.1164/rccm.202204-0734OC.
本文引用的文献
1
Recombinant growth hormone therapy for cystic fibrosis in children and young adults.
Cochrane Database Syst Rev. 2018 Dec 17;12(12):CD008901. doi: 10.1002/14651858.CD008901.pub4.
2
Energy Balance and Mechanisms of Weight Gain with Ivacaftor Treatment of Cystic Fibrosis Gating Mutations.
J Pediatr. 2018 Oct;201:229-237.e4. doi: 10.1016/j.jpeds.2018.05.018. Epub 2018 Jul 18.
3
Reduced bone length, growth plate thickness, bone content, and IGF-I as a model for poor growth in the CFTR-deficient rat.
PLoS One. 2017 Nov 30;12(11):e0188497. doi: 10.1371/journal.pone.0188497. eCollection 2017.
4
The safety of lumacaftor and ivacaftor for the treatment of cystic fibrosis.
Expert Opin Drug Saf. 2017 Nov;16(11):1305-1311. doi: 10.1080/14740338.2017.1372419. Epub 2017 Sep 21.
5
Effects of Diagnosis by Newborn Screening for Cystic Fibrosis on Weight and Length in the First Year of Life.
JAMA Pediatr. 2017 Jun 1;171(6):546-554. doi: 10.1001/jamapediatrics.2017.0206.
6
Risk factors for mortality before age 18 years in cystic fibrosis.
Pediatr Pulmonol. 2017 Jul;52(7):909-915. doi: 10.1002/ppul.23715. Epub 2017 Apr 24.
7
Growth deficits in cystic fibrosis mice begin in utero prior to IGF-1 reduction.
PLoS One. 2017 Apr 6;12(4):e0175467. doi: 10.1371/journal.pone.0175467. eCollection 2017.
8
Cystic-fibrosis related-diabetes (CFRD) is preceded by and associated with growth failure and deteriorating lung function.
J Pediatr Endocrinol Metab. 2017 Aug 28;30(8):815-821. doi: 10.1515/jpem-2017-0005.
9
Growth in Prepubertal Children With Cystic Fibrosis Treated With Ivacaftor.
Pediatrics. 2017 Feb;139(2). doi: 10.1542/peds.2016-2522.
10
BMI fails to identify poor nutritional status in stunted children with CF.
J Cyst Fibros. 2017 Jan;16(1):158-160. doi: 10.1016/j.jcf.2016.11.005.
Zotero 插件