Experts have identified CLN2-specific disease management strategies that can greatly impact patient outcomes
CLN2 disease management support starts with a multidisciplinary care team
Multiple specialties can work together to provide optimal outcomes for children with CLN2 disease. This approach to optimize care is essential for patients and families.1
Dr Angela Schulz, MD, PhD Children’s Hospital NCL Specialty Clinic International Center of Lysosomal Storage Disorders (ICLD) University Medical Center Hamburg-Eppendorf Hamburg, Germany
Key aspects of disease management are covered in this video, including the multidisciplinary team approach, medication strategies, and the importance of physical therapy.
CLN2-specific care aims to maintain function for as long as possible and address quality of life
The goals of CLN2 disease care should evolve as the disease progresses
Experts have noted that reassessing the goals of care and management is important throughout the course of the disease.1
Early in the disease: Goals strive to maintain function for as long as possible
Later in the disease: Goals evolve toward maintenance of quality of life and pain control as functions are lost
Understanding the needs of families across the spectrum of care can make an important difference in the approach to CLN2-specific management strategies.1
Though these are CLN2 disease-specific management strategies, some may apply to the management of other NCLs.
CLN2 disease–specific care can positively impact the quality of life of the child and the family.
This concept of care can be defined by the broader idea of supporting the body, spirit, and mind of both the child and the family to optimize quality of life.1
Experts recommend discussions be initiated early, prior to physical and emotional exhaustion, with ongoing dialogue establishing trust with the palliative care team and ensuring parents’ goals are met
Home nursing can assist with everyday care to manage pain and prevent complications
Various types of hospice care can help provide respite for families, not just at end of life
Family and individual therapy ranges from marriage and family to grief counseling, to help guide decision-making and plan for transitions as the disease progresses
Reference:1. Williams RE, Adams HR, Blohm M, et al. Expert opinion on the management of CLN2 disease. Poster session presented at: The 12th Annual WORLD Symposium; February – March 2016; San Diego, CA.