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Paediatric clinical study of 3D printed personalised medicines for rare metabolic disorders
Lucía Rodríguez-Pombo1, María José de Castro-López2, Paula Sánchez-Pintos2
1Departamento 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.
Insights
Three-dimensional printing (3DP) offers personalized medicine for children with rare diseases. This novel technology creates chewable medications with precise doses, improving adherence and quality of life.
Area of Science:
- Pharmaceutical Technology
- Rare Diseases
- Pediatric Medicine
Background:
- Rare diseases affect millions globally, predominantly children, necessitating precise medication.
- Conventional pharmaceutical compounding struggles to meet the specific dosage and adherence needs for rare disease patients.
- Three-dimensional printing (3DP) presents a novel solution for personalized medicine, enabling tailored dosage forms.
Purpose of the Study:
- To evaluate the efficacy and acceptability of 3D printed chewable medicines for children with rare metabolic disorders.
- To compare 3D printed medicines with conventionally compounded treatments.
- To explore patient preferences and real-time feedback using flavors and a mobile app.
Main Methods:
- Preparation of 3D printed chewable amino acid medicines (citrulline, isoleucine, valine) in a hospital setting.
- Clinical evaluation in six children, comparing 3D printed formulations to conventional medicines.
- Incorporation of varied flavors and a mobile app for real-time patient feedback.
Main Results:
- 3D printed medicines effectively controlled amino acid levels, comparable to conventional methods.
- Chewable formulations demonstrated narrower and more consistent citrulline level deviations within target ranges.
- Patients reported good acceptance of chewable formulations, suggesting improved adherence and quality of life.
- 3DP successfully combined two active ingredients in a single formulation for the first time.
Conclusions:
- 3D printing is a viable technology for creating personalized, effective, and well-accepted medications for pediatric rare metabolic disorders.
- This study highlights the potential of 3DP to enhance patient adherence and quality of life in managing rare diseases.
- The successful co-formulation of multiple actives using 3DP offers a promising approach to simplify treatment regimens.
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.
