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Development of a practical dietitian road map for the nutritional management of phenylketonuria (PKU) patients on pegvaliase
Molecular Genetics and Metabolism Reports ( IF 1.9 ) Pub Date : 2021-05-25 , DOI: 10.1016/j.ymgmr.2021.100771
Júlio César Rocha 1, 2, 3 , Heather Bausell 4 , Amaya Bélanger-Quintana 5 , Laurie Bernstein 6 , Hülya Gökmen-Özel 7 , Alexandra Jung 8 , Anita MacDonald 9 , Fran Rohr 10 , Esther van Dam 11 , Margret Heddrich-Ellerbrok 12
Affiliation  

Background

The metabolic dietitian/nutritionist (hereafter ‘dietitian’) plays an essential role in the nutritional management of patients with phenylketonuria (PKU), including those on pegvaliase. Currently, more educational support and clinical experience is needed to ensure that dietitians are prepared to provide optimal nutritional management and counselling of pegvaliase-treated patients.

Methods

Via a face-to-face data-review meeting, followed by a virtual consolidation meeting, a group of expert dietitians and one paediatrician discussed and developed a series of recommendations on the nutritional evaluation and management of patients receiving pegvaliase. The consensus group consisted of 10 PKU experts: six dietitians and one paediatrician from Europe and three dietitians from the US. One European and three US dietitians had experience with pegvaliase-treated patients.

Results

The consensus group recommended that a physician, dietitian and nurse are part of the pegvaliase treatment team. Additionally, a psychologist/counsellor should be included if available. Practical proposals for the nutritional evaluation of pegvaliase-treated patients at baseline, during the induction and titration phases and for long-term maintenance were developed. The consensus group suggested assessment of blood Phe at least monthly or every 2 weeks in the event of low blood Phe (i.e., blood Phe <30 μmol/L). It may be appropriate to increase blood Phe monitoring when adjusting protein intake and/or pegvaliase dose. It was recommended that natural protein intake is increased by 10–20 g increments if blood Phe concentrations decrease to <240 μmol/L in patients who are not meeting the dietary reference intake for natural protein of 0.8 g/kg. It was proposed that with pegvaliase treatment blood Phe levels could be maintained <240 μmol/L but more evidence on the safety of achieving physiological blood Phe levels is necessary before any recommendation on the lower blood Phe target can be given. Finally, both patients and dietitians should have access to educational resources to optimally support patients receiving pegvaliase.

Conclusion

This practical road map aims to provide initial recommendations for dietitians monitoring patients with PKU prescribed pegvaliase. Given that practical experience with pegvaliase is still limited, nutritional recommendations will require regular updating once more evidence is available and clinical experience evolves.



中文翻译:

苯丙酮尿症 (PKU) 患者使用聚乙二醇化酶进行营养管理的实用营养师路线图的开发

背景

代谢营养师/营养师(以下简称“营养师”)在苯丙酮尿症 (PKU) 患者的营养管理中发挥着重要作用,包括那些服用聚乙二醇化酶的患者。目前,需要更多的教育支持和临床经验,以确保营养师准备好为接受培格瓦利酶治疗的患者提供最佳的营养管理和咨询。

方法

通过面对面的数据审查会议和随后的虚拟整合会议,一组专家营养师和一名儿科医生讨论并制定了一系列关于接受培伐利亚酶治疗的患者的营养评估和管理的建议。共识小组由10名北大专家组成:来自欧洲的六名营养师和一名儿科医生以及来自美国的三名营养师。一名欧洲营养师和三名美国营养师对接受聚乙二醇化酶治疗的患者有过经验。

结果

共识小组建议医生、营养师和护士是培格瓦利亚酶治疗团队的一部分。此外,如果有的话,应包括心理学家/顾问。制定了在基线、诱导和滴定阶段以及长期维持期间对聚乙二醇化酶治疗患者进行营养评估的实用建议。共识小组建议在低血 Phe(即血 Phe <30 μmol/L)的情况下至少每月或每 2 周评估一次血 Phe。在调整蛋白质摄入量和/或聚乙二醇化酶剂量时,增加血液 Phe 监测可能是合适的。如果患者的血液 Phe 浓度降低至 <240 μmol/L,而未达到 0.8 g/kg 的天然蛋白质膳食参考摄入量,则建议将天然蛋白质摄入量增加 10-20 g。有人提议,通过聚乙二醇化酶治疗,血液 Phe 水平可以保持 <240 μmol/L,但在给出任何关于较低血液 Phe 目标的建议之前,需要更多关于达到生理血 Phe 水平的安全性的证据。最后,患者和营养师都应该获得教育资源,以最佳地支持接受聚乙二醇化酶治疗的患者。

结论

该实用路线图旨在为营养师监测 PKU 处方聚乙二醇化酶患者提供初步建议。鉴于聚乙二醇化酶的实际经验仍然有限,一旦有更多证据可用且临床经验不断发展,营养建议将需要定期更新。

更新日期:2021-05-25
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