Background:
Delandistrogene moxeparvovec-rokl is an adeno-associated virus (AAV) based gene therapy designed to deliver a functional micro-dystrophin transgene to patients with Duchenne Muscular Dystrophy (DMD). Given the complexity of gene therapy and the potential for immune-mediated adverse events, a standardized pre- and post-infusion monitoring protocol was developed. This protocol was developed based on prescribing information resulting from gene replacement therapy clinical trials, ACTION DMD harmonization protocols, and expert consensus which improve patient safety and optimize clinical outcomes. After development, this protocol and follow-up was uniquely managed by a physician assistant and nursing team, with oversight and frequent discussions with neuromuscular physicians.
Objectives:
To describe a comprehensive clinical monitoring protocol developed by Children’s Health neuromuscular team to assess safety, manage adverse events, and guide post-treatment care in patients receiving delandistrogene gene therapy. This protocol is executed primarily by an experienced Neuromuscular Physician Assistant and Gene Therapy Nurse Navigator, with a focus on protocol adherence, safety, patient education/communication, and timely clinical interventions.
Results:
Based on our model, the team was able to detect and successfully manage acute liver injury and other adverse systemic reactions in several patients. The collaborative nature of the team dynamic allowed for minimal care gaps and improved family adherence during a difficult and often emotional time. This collaborative environment ensured safe treatment by relying heavily on consistent, proactive monitoring and communication.
Conclusions:
PAs and RNs can be central to the safe delivery of Delandistrogene gene therapy in pediatric DMD patients. A clearly defined, team-driven monitoring protocol enables early identification of adverse events, consistent patient engagement, and successful post-gene therapy outcomes with appropriate oversight from neuromuscular physicians. This model demonstrates how the unique skills of an interdisciplinary team can be utilized to optimize gene therapy treatment in patients with DMD.