Departamento de Farmacología, Farmacia y Tecnología Farmacéutica, I+D Farma (GI-1645), Facultad de Farmacia, Materials Institute (iMATUS) and Health Research Institute of Santiago de Compostela (IDIS), Universidade de Santiago de Compostela, 15782 Santiago de Compostela, Spain.
Servicio de Neonatología, Unidad de Diagnóstico y Tratamiento de Enfermedades Metabólicas Congénitas, Health Research Institute of Santiago de Compostela (IDIS), Hospital Clínico Universitario de Santiago de Compostela, Universidad de Santiago de Compostela, RICORS, CIBERER, MetabERN, 15706 Santiago de Compostela, Spain.
Nutrients. 2023 Dec 25;16(1):61. doi: 10.3390/nu16010061.
The incidence of Inborn Error of Intermediary Metabolism (IEiM) diseases may be low, yet collectively, they impact approximately 6-10% of the global population, primarily affecting children. Precise treatment doses and strict adherence to prescribed diet and pharmacological treatment regimens are imperative to avert metabolic disturbances in patients. However, the existing dietary and pharmacological products suffer from poor palatability, posing challenges to patient adherence. Furthermore, frequent dose adjustments contingent on age and drug blood levels further complicate treatment. Semi-solid extrusion (SSE) 3D printing technology is currently under assessment as a pioneering method for crafting customized chewable dosage forms, surmounting the primary limitations prevalent in present therapies. This method offers a spectrum of advantages, including the flexibility to tailor patient-specific doses, excipients, and organoleptic properties. These elements are pivotal in ensuring the treatment's efficacy, safety, and adherence. This comprehensive review presents the current landscape of available dietary products, diagnostic methods, therapeutic monitoring, and the latest advancements in SSE technology. It highlights the rationale underpinning their adoption while addressing regulatory aspects imperative for their seamless integration into clinical practice.
先天性代谢缺陷（IEiM）疾病的发病率可能较低，但总体而言，它们影响了全球约 6-10%的人口，主要影响儿童。为了避免患者发生代谢紊乱，精确的治疗剂量和严格遵守规定的饮食和药物治疗方案至关重要。然而，现有的饮食和药物产品存在适口性差的问题，这给患者的依从性带来了挑战。此外，根据年龄和药物血药浓度频繁调整剂量也使治疗更加复杂。半固态挤出（SSE）3D 打印技术目前正在被评估为一种开创性的方法，用于制作定制的可咀嚼剂型，克服了现有疗法中普遍存在的主要限制。该方法具有一系列优势，包括能够为患者定制剂量、赋形剂和感官特性的灵活性。这些因素对于确保治疗的疗效、安全性和依从性至关重要。本综述全面介绍了现有的饮食产品、诊断方法、治疗监测以及 SSE 技术的最新进展。它强调了采用这些技术的基本原理，同时解决了将其无缝整合到临床实践中所必需的监管方面的问题。
