At the 15th
International Conference on Neuronal Ceroid Lipfuscinosis (Batten Disease) held in Boston, MA, best practice guidelines for managing neuronal ceroid lipofuscinosis type 2 (CLN2 disease) or Batten disease were presented.
Managing Batten Disease
Regarding how best to manage CLN2, an online survey was conducted using 24 disease experts (healthcare professionals and patient advocates) with a smaller group participating in a discussion of management practices.â€¨
The experts noted that the main goal of managing CLN2 is to maintain a good quality of life.
And to do that requires a multidisciplinary team approach that may include cardiologist, dentist, dietician, gastroenterologist, home nurse, neurodevelopmental therapist, neurologist, neurodisability expert, neuromuscular specialist, occupational therapist, ophthalmologist, orthopedists, palliative care team, pediatrician, physical therapist, pulmonologist, sleep therapist, social worker, and speech therapist.
This team will coordinate to manage the numerous aspects of the disease that require care, including seizure control, management of movement disorders, physical/occupational therapy, nutrition, eye care, pain management, sleep, social strategies, and palliative/end of life considerations. For example, the goal of antiepileptic medication is to achieve sufficient seizure control to support function balancing the side effects. Further, school and home environments should be adapted to accommodate physical and cognitive/behavioral impairments for ongoing benefit of maintaining social interactions. The panel also recommended that physical, occupational and speech therapies should be initiated early and assessed frequently, including use of adaptive devices to support function and independence.
Common medicines used by experts to manage CLN2 include:
The multidisciplinary team approach must also balance the medical needs of the child with other aspects of life as shown in the figure below.
Finally, the goals of care need to evolve as the disease progresses.
Williams RE, Adams HR, Blohm M, et al. Expert opinion on the management of CLN2 disease. Presented at the 15th
International Conference on Neuronal Ceroid Lipfuscinosis (Batten Disease); Boston, MA; October 5-8, 2016. Abstract P17.
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