Rodríguez-Pombo, Lucía;
De Castro-López, María José;
Sánchez-Pintos, Paula;
Giraldez-Montero, Jose Maria;
Januskaite, Patricija;
Duran-Piñeiro, Goretti;
Dolores Bóveda, M Dolores;
... Couce, Maria L; + view all
(2024)
Paediatric clinical study of 3D printed personalised medicines for rare metabolic disorders.
International Journal of Pharmaceutics
, 657
, Article 124140. 10.1016/j.ijpharm.2024.124140.
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Abstract
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.
| Type: | Article |
|---|---|
| Title: | Paediatric clinical study of 3D printed personalised medicines for rare metabolic disorders |
| Location: | Netherlands |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1016/j.ijpharm.2024.124140 |
| Publisher version: | http://dx.doi.org/10.1016/j.ijpharm.2024.124140 |
| Language: | English |
| Additional information: | Copyright © 2024 The Author(s). Published by Elsevier B.V. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/). |
| Keywords: | Semi-solid extrusion 3D printing; Pediatrics; Direct ink writing; Polypills; Patient acceptability of formulations; Additive manufacturing of drug products; Precision pharmaceuticals and drug delivery systems |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > UCL School of Pharmacy |
| URI: | https://discovery-pp.ucl.ac.uk/id/eprint/10191925 |
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