Calvo-Lerma Joaquim, Hulst Jessie M, Asseiceira Inês, Claes Ine, Garriga Maria, Colombo Carla, Fornés Victoria, Woodcock Sandra, Martins Tiago, Boon Mieke, Ruperto Mar, Walet Sylvia, Speziali Chiara, Witters Peter, Masip Etna, Barreto Celeste, de Boeck Kris, Ribes-Koninckx Carmen
Instituto de Investigación Sanitaria La Fe, Avenida Fernando Abril Martorell, 106, Torre A, planta 0, 46026 Valencia, Spain.
Erasmus Medical Center, Sophia Children's Hospital, Rotterdam, the Netherlands.
J Cyst Fibros. 2017 Jul;16(4):510-518. doi: 10.1016/j.jcf.2017.03.005. Epub 2017 Mar 17.
The New European guidelines have established the most updated recommendations on nutrition and pancreatic enzyme replacement therapy (PERT) in CF. In the context of MyCyFAPP project - a European study in children with CF aimed at developing specific tools for improvement of self-management - the objective of the current study was to assess nutritional status, daily energy and macronutrient intake, and PERT dosing with reference to these new guidelines.
Cross sectional study in paediatric patients with CF from 6 European centres. SD-scores for weight-for-age (WFA), height-for-age (HFA) and body mass index-for-age (BMI) were obtained. Through a specific 4-day food and enzyme-dose record, energy and macronutrients intake and PERT-use (LU/g lipids) were automatically calculated by the MyCyFAPP system. Comparisons were made using linear regression models.
The lowest quartiles for BMI and HFA were between 0 and -1SD in all the centres with no significant differences, and 33.5% of the patients had a SD-score <0 for all three parameters. The minimum energy intake recommendation was not reached by 40% of the children and mean nutrients intake values were 14%, 51% and 34% of the total energy for protein, carbohydrates and lipids respectively. When assessed per centre, reported PERT doses were in the recommended range in only 13.8% to 46.6% of the patients; from 5.6% up to 82.7% of children were above the recommended doses and 3.3% to 75% were below.
Among the 6 centres, a large variability and inconsistency with new guidelines on nutrition and PERT-use was found. Our findings document the lack of a general criterion to adjust PERT and suggest the potential benefit of educational and self-managerial tools to ensure adherence to therapies, both for clinical staff and families. They will be taken into account when developing these new tools during the next stages of MyCyFAPP Project.
欧洲新指南已制定了关于囊性纤维化（CF）患者营养及胰酶替代疗法（PERT）的最新建议。在MyCyFAPP项目（一项针对CF儿童的欧洲研究，旨在开发改善自我管理的特定工具）的背景下，本研究的目的是参照这些新指南评估营养状况、每日能量和宏量营养素摄入量以及PERT剂量。
对来自6个欧洲中心的CF儿科患者进行横断面研究。获取年龄别体重（WFA）、年龄别身高（HFA）和年龄别体重指数（BMI）的标准差评分。通过特定的4天食物和酶剂量记录，MyCyFAPP系统自动计算能量和宏量营养素摄入量以及PERT使用量（脂酶单位/克脂质）。使用线性回归模型进行比较。
所有中心BMI和HFA的最低四分位数在0至 -1标准差之间，无显著差异，33.5%的患者这三个参数的标准差评分均<0。40%的儿童未达到最低能量摄入推荐量，蛋白质、碳水化合物和脂质的平均营养素摄入量分别占总能量的14%、51%和34%。按中心评估时，仅13.8%至46.6%的患者报告的PERT剂量在推荐范围内；5.6%至82.7%的儿童高于推荐剂量，3.3%至75%低于推荐剂量。
在这6个中心中，发现营养和PERT使用方面与新指南存在很大差异和不一致。我们的研究结果表明缺乏调整PERT的通用标准，并提示教育和自我管理工具对临床工作人员和家庭确保坚持治疗可能具有益处。在MyCyFAPP项目的下一阶段开发这些新工具时将考虑这些结果。
