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NEUROLOGY 2005;65:275-279
© 2005 American Academy of Neurology

A clinical rating scale for Batten disease

Reliable and relevant for clinical trials

F. J. Marshall, MD, E. A. de Blieck, MPA, J. W. Mink, MD, PhD, FAAN, L. Dure, MD, H. Adams, PhD, S. Messing, MA, MS, P. G. Rothberg, PhD, E. Levy, BA, T. McDonough, BA, J. DeYoung, MD, M. Wang, BA, D. Ramirez-Montealegre, MD, J. M. Kwon, MD and D. A. Pearce, PhD

From the University of Rochester School of Medicine and Dentistry (Drs. Marshall, Mink, Adams, Rothberg, Ramirez-Montealegre, Kwon, and Pearce, E.A. de Blieck, S. Messing, E. Levy, and T. McDonough), NY; Department of Pediatrics (Dr. Dure), University of Alabama–Birmingham; Dartmouth College (Dr. DeYoung), Hanover, NH; and University of Pennsylvania (M. Wang), Philadelphia.

Address correspondence and reprint requests to Dr Marshall, Clinical Trials Coordination Center, Department of Neurology, University of Rochester School of Medicine and Dentistry, 1351 Mt. Hope Ave., Suite 223, Rochester, NY 14620; e-mail: fred.marshall{at}ctcc.rochester.edu

Background: Batten disease (juvenile neuronal ceroid lipofuscinosis [JNCL]) is an autosomal recessive neurodegenerative disorder characterized by blindness, seizures, and relentless decline in cognitive, motor, and behavioral function. Onset is in the early school years, with progression to death typically by late adolescence. Development of a clinical instrument to quantify severity of illness is a prerequisite to eventual assessment of experimental therapeutic interventions

Objective: To develop a clinical rating instrument to assess motor, behavioral, and functional capability in JNCL.

Methods: A clinical rating instrument, the Unified Batten Disease Rating Scale (UBDRS), was developed by the authors to assess motor, behavioral, and functional capability in JNCL. Children with verified JNCL were evaluated independently by three neurologists. Intraclass correlation coefficients (ICCs) were used to estimate the interrater reliability for total scores in each domain. Interrater reliability for scale items was assessed with weighted {kappa} statistics

Results: Thirty-one children with confirmed JNCL (10 boys, 21 girls) were evaluated. The mean age at symptom onset was 6.1 ± 1.6 years, and the mean duration of illness was 9.0 ± 4.4 years. The ICCs for the domains were as follows: motor = 0.83, behavioral = 0.68, and functional capability = 0.85.

Conclusions: The Unified Batten Disease Rating Scale (UBDRS) is a reliable instrument that effectively tests for neurologic function in blind and demented patients. In its current form, the UBDRS is useful for monitoring the diverse clinical findings seen in Batten disease.

Additional material related to this article can be found on the Neurology Web site. Go to www.neurology.org and scroll down the Table of Contents for the July 26 issue to find the title link for this article.

Funded by a grant from the Batten's Disease Support and Research Association.

Received December 30, 2004. Accepted in final form April 11, 2005.




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