Paediatric clinical study of 3D printed personalised medicines for rare metabolic disorders.

Autor: Rodríguez-Pombo L; 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., de Castro-López MJ; 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, IDIS, RICORS, CIBERER, MetabERN, Spain., Sánchez-Pintos P; 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, IDIS, RICORS, CIBERER, MetabERN, Spain., Giraldez-Montero JM; Pharmacy Department, Xerencia de Xestión Integrada de Santiago de Compostela, SERGAS, Travesía Choupana s/n, Santiago de Compostela 15706, Spain., Januskaite P; Department of Pharmaceutics, UCL School of Pharmacy, University College London, 29-39 Brunswick Square, London WC1N 1AX, UK., Duran-Piñeiro G; Pharmacy Department, Xerencia de Xestión Integrada de Santiago de Compostela, SERGAS, Travesía Choupana s/n, Santiago de Compostela 15706, Spain., Dolores Bóveda M; 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, IDIS, RICORS, CIBERER, MetabERN, Spain., Alvarez-Lorenzo C; 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., Basit AW; Department of Pharmaceutics, UCL School of Pharmacy, University College London, 29-39 Brunswick Square, London WC1N 1AX, UK; FABRX Ltd., Henwood House, Henwood, Ashford, Kent TN24 8DH, UK; FABRX Artificial Intelligence, Carretera de Escairón, 14, Currelos (O Saviñao), CP 27543, Spain., Goyanes A; 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; Department of Pharmaceutics, UCL School of Pharmacy, University College London, 29-39 Brunswick Square, London WC1N 1AX, UK; FABRX Ltd., Henwood House, Henwood, Ashford, Kent TN24 8DH, UK; FABRX Artificial Intelligence, Carretera de Escairón, 14, Currelos (O Saviñao), CP 27543, Spain. Electronic address: a.goyanes@fabrx.co.uk., Couce ML; 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, IDIS, RICORS, CIBERER, MetabERN, Spain. Electronic address: maria.luz.couce.pico@sergas.es.
Jazyk: angličtina
Zdroj: International journal of pharmaceutics [Int J Pharm] 2024 May 25; Vol. 657, pp. 124140. Date of Electronic Publication: 2024 Apr 19.
DOI: 10.1016/j.ijpharm.2024.124140
Abstrakt: Rare diseases are infrequent, but together they affect up to 6-10 % of the world's population, mainly children. Patients require precise doses and strict adherence to avoid metabolic or cardiac failure in some cases, which cannot be addressed in a reliable way using pharmaceutical compounding. 3D printing (3DP) is a disruptive technology that allows the real-time personalization of the dose and the modulation of the dosage form to adapt the medicine to the therapeutic needs of each patient. 3D printed chewable medicines containing amino acids (citrulline, isoleucine, valine, and isoleucine and valine combinations) were prepared in a hospital setting, and the efficacy and acceptability were evaluated in comparison to conventional compounded medicines in six children. The inclusion of new flavours (lemon, vanilla and peach) to obtain more information on patient preferences and the implementation of a mobile app to obtain patient feedback in real-time was also used. The 3D printed medicines controlled amino acid levels within target levels as well as the conventional medicines. The deviation of citrulline levels was narrower and closer within the target concentration with the chewable formulations. According to participants' responses, the chewable formulations were well accepted and can improve adherence and quality of life. For the first time, 3DP enabled two actives to be combined in the same formulation, reducing the number of administrations. This study demonstrated the benefits of preparing 3D printed personalized treatments for children diagnosed with rare metabolic disorders using a novel technology in real clinical practice.
Competing Interests: Declaration of competing interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: Abdul W. Basit reports a relationship with FABRX Ltd. that includes: employment and equity or stocks. Alvaro Goyanes reports a relationship with FABRX Ltd. that includes: employment and equity or stocks. If there are other authors, they declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.
(Copyright © 2024 The Author(s). Published by Elsevier B.V. All rights reserved.)
Databáze: MEDLINE
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