Long-Term Outcomes of a Cultured Autologous Dermo-Epidermal Skin Substitute in Children: 5-Year Results of a Phase I Clinical Trial.

Abstract

Limited donor sites and poor long-term outcomes with standard treatment for large skin defects remain a huge problem. An autologous, bilayered, laboratory-grown skin substitute (denovoSkin) was developed to overcome this problem and has shown to be safe in 10 pediatric patients in a Phase I clinical trial after transplantation. The goal of this article was to report on 48-month long-term results. The pediatric participants of the phase I clinical trial were followed up at yearly visits up to 5 years after transplantation. Safety parameters, including the occurrence of adverse events, possible deviations of vital signs, and changes in concomitant therapy as well as additional parameters regarding skin stability, scar quality, and tumor formation, were assessed. Furthermore, scar maturation was photographically documented. Of the 10 patients treated with denovoSkin in this phase I clinical trial, 7 completed the 5-year follow-up period. Skin substitutes continued to be deemed safe, remained stable, and practically unchanged, with no sign of fragility and no tumor formation at clinical examination. Scar quality, captured using the Patient and Observer Scar Assessment Scale, was evaluated as close to normal skin. Transplantation of this laboratory-grown skin substitute in children is to date considered safe and shows encouraging functional and aesthetical long-term results close to normal skin. These results are promising and highlight the potential of a life-saving therapy for large skin defects. A multicentre, prospective, randomized, phase II clinical trial is currently ongoing to further evaluate the safety and efficacy of this novel skin substitute. Clinicaltrials.gov identifier NCT02145130.