Liver and Learn: Single Center Experience of Acute Liver Injury Post Treatment with Delandistrogene Moxeparvovec and Management Strategies

Topic:

Clinical Management

Poster Number: 45 S

Author(s):

Jennifer Momot, BSN, RN, CPN, Children's Hospital Colorado, Tiffany Lopez, Children's Hospital Colorado, Taylor Schwab, BSN, RN, CPN, AMB-BC, Children's Hospital Colorado, Kelli Johnson, CPNP, Children's Hospital Colorado, Hattie Foster, RN, Children's Hospital Colorado, Melissa Gibbons, MS, Children's Hospital Colorado, Meghan Moore Burk, PhD, PT, DPT, Children's Hospital Colorado, Carolyn Kelley, PT, DPT, Children's Hospital Colorado, Melissa Wright, MD, PhD, University of Colorado School of Medicine, Susan Apkon, Children’s Hospital Colorado, Michele Yang, MD, Children's Hospital Colorado

Objectives: Identify differences in laboratory values, treatments, and number of visits in patients with and without complications following gene transfer therapy in boys with Duchenne Muscular Dystrophy (DMD).

Methods: A retrospective chart review was performed to collect data on laboratory values, treatment strategies, patient outcomes, number of laboratory studies/clinic visits, and specialists consulted for each patient. Data was analyzed to identify differences between patients with and without post-treatment complications.

Results: Data was collected from five boys who were treated at Children’s Hospital Colorado with delandistrogene moxeparvovec. Three patients were on standard of care treatment with steroids prior to starting high dose prednisone/prednisolone regiment. Of these three, two patients were admitted to the hospital for acute liver injury between 5-7 weeks post treatment and treated with high dose IV methylprednisolone. Of the two patients requiring admission, one started treatment with sirolimus. The two steroid-naive boys had no post-treatment complications. On average, patients required 45 visits if post-treatment complications occurred, compared with 26 visits without complications.

Discussion: The treatment of post-gene therapy complications is still evolving with no standardized protocols for management of acute liver injury. Multiple subspecialists may be involved in the care of acute liver injury. Optimizing management to minimize morbidity and mortality is needed. Frequent appointments and laboratory testing additionally can become burdensome for both the families and the neuromuscular team and add additional costs to the treatment.

Conclusion: Close collaboration with subspecialty teams and between centers is needed to ensure recognition of complications that can occur following gene therapy. Optimizing immunosuppressant treatments and anticipating risks to minimize post-gene therapy complications is needed as centers develop more experience with management. The impact of the frequent appointments, consultations, and laboratory studies on families needs to be considered prior to initiation of any treatments.