Gene therapy and hospital pharmacy: the experience of the Hospital Pharmacy unit of L. Vanvitelli university hospital

Background: Since hospital use of gene therapy requires close cooperation between doctors and pharmacists to ensure correct drug handling and administration, it is essential to define standard operating procedures (SOPs) for each phase of the process to ensure therapeutic success in compliance with the safety of patient and healthcare professionals. Among the available gene therapy medicines, voretigene neparvovec is used to treat hereditary dystrophy of the retina caused by biallelic mutations in the RPE65 gene. The aim of this article is to describe the experience of the Hospital Pharmacy unit of L. Vanvitelli university hospital in the management of treatment with voretigene neparvovec in the first two pediatric patients treated in Italy. Methods: SOPs were developed for all phases of gene therapy management process (procurement, receipt, storage, handling, transport of the finished product and waste disposal). Particular attention was paid to the training of personnel involved, to ensure maximum compliance with the procedures at all stages of the process. Results: SOPs were developed according to the European Association of Hospital Pharmacist (EAHP) policies and in full agreement with the operating procedures established by the manufacturer, ensuring correct storage and handling of the drug, as well as safe administration to patients. Conclusions: This example confirms the importance of close collaboration in a multidisciplinary team between hospital pharmacists, ophthalmologists experienced in the treatment of patients with hereditary retinal dystrophies and surgeons experienced in subretinal surgery to ensure the correct management of gene therapy with voretigene neparvovec, through compliance with shared operating procedures.

Autotranslocation of the pigment epithelium-choroid complex in treatment of age-related macular degeneration scarring stage

The gold standard of the neovascular age-related macular degeneration treatment is the intravitreal administration of angiogenesis inhibitors. In subretinal macular fibrosis, antiangiogenic therapy is not effective. In such cases, subretinal surgery is used, in particular, autotranslocation of pigment epithelium-choroid complex. This paper presents a case of successful use of this method in a 77 y.o. female patient with subretinal fibrosis in the macular area as an outcome of neovascular age-related macular degeneration. An original method of translocation of pedicled pigment epithelium-choroid complex from the paramacular area to the macula was used. In 24 months, the visual acuity increased from 0.01 to 0.07; the central fixation was restored; the absolute positive central scotoma disappeared. During all the post-operative follow-up period, the full-rate pigment epithelium-choroid perfusion in the choroid of the translocated flap, the loss of choroidal neovascularization activity signs and of indications for intravitreal administration of angiogenesis inhibitors were proved.