Automated therapy preparation of isoleucine formulations using 3D printing for the treatment of MSUD: first single-centre, prospective, crossover study in patients

Publication date: Available online 4 July 2019Source: International Journal of PharmaceuticsAuthor(s): Alvaro Goyanes, Christine M. Madla, Aysha Umerji, Goretti Duran Piñeiro, Jose Maria Giraldez Montero, María Jesús Lamas Diaz, Miguel Gonzalez Barcia, Farhan Taherali, Paula Sánchez-Pintos, Maria-Luz Couce, Simon Gaisford, Abdul W. BasitAbstractMaple syrup urine disease (MSUD) is a rare metabolic disorder with a worldwide prevalence of 1 in every 185,000 live births. However, certain populations display a significant overexpression of the disorder where incidence is reported to be 1 in every 52, 541 new-borns. The first-line therapy for MSUD involves a strict dietary leucine restriction and oral supplementation of isoleucine and valine. The dose administered to patients requires strict tailoring according to age, weight and blood levels. In current clinical practice, however, practitioners still have to prepare extemporaneous formulations due to the lack of suitable oral treatments for MSUD. Herein we evaluate for the first time the use of 3D printing in a hospital setting for the preparation of personalised therapies with the aim of improving safety and acceptability to isoleucine supplementation in paediatric patients suffering from MSUD. The study was a single-centre, prospective crossover experimental study. Four paediatric patients with MSUD (aged 3-16 years) were treated at the Clinic University Hospital in Santiago de Compostela, Spain which is a MSUD reference hos...
Source: International Journal of Pharmaceutics - Category: Drugs & Pharmacology Source Type: research