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 Volume 62, Number 7, April 13, 2004
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NEUROLOGY 2004;62:1066-1072
© 2004 American Academy of Neurology

Enzyme replacement therapy improves function of C-, A{delta}-, and Aß-nerve fibers in Fabry neuropathy

M. J. Hilz, MD PhD, M. Brys, MD, H. Marthol, MD, B. Stemper, MD and M. Dütsch, MD

From the Department of Neurology (Drs. Hilz, Brys, and Marthol), New York University, NY; and Department of Neurology (Drs. Stemper and Dütsch), University of Erlangen–Nuremberg, Erlangen, Germany.

Address correspondence and reprint requests to Dr. M.J. Hilz, Department of Neurology, New York University School of Medicine, 550 First Avenue, NB 7W11, New York, NY 10016; e-mail: max.hilz{at}neuro.med.uni-erlangen.de

Background: Peripheral neuropathy in Fabry disease predominantly involves small nerve fibers. Recently, enzyme replacement therapy (ERT) with recombinant human {alpha}-galactosidase A has become available.

Objective: To evaluate whether ERT improves Fabry neuropathy.

Methods: In 22 Fabry patients (age 27.9 ± 8.0 years) undergoing ERT with recombinant human {alpha}-galactosidase A (agalsidase beta) for 18 (n = 11) or 23 (n = 11) months and in 25 control subjects (age 29.0 ± 10.4 years), the authors performed quantitative sensory testing using the 4, 2, and 1 stepping algorithm (CASE IVTM). Detection thresholds of vibration (VDT) on the first toe were assessed; cold detection thresholds (CDT), heat-pain onset (HP 0.5), and intermediate heat-pain (HP 5.0) assessments were made on the dorsum of the feet. Patient values above mean + 2.5 SD of control values were considered abnormal.

Results: Before ERT, VDT, CDT, HP 0.5, and HP 5.0 were higher in patients than control subjects (p < 0.05). Following ERT, patients developed lower thresholds than prior to ERT for VDT (15.5 ± 3.5 vs 14.3 ± 4.1; p < 0.05), HP 0.5 (22.3 ± 6.7 vs 19.4 ± 1.3; p < 0.01), and HP 5.0 (27.3 ± 5.6 vs 22.5 ± 2.3; p < 0.01). Moreover, fewer patients had abnormal results of VDT (2 vs 4), CDT (7 vs 12), HP 0.5 (0 vs 9), and HP 5.0 (4 vs 20) after than before ERT.

Conclusions: ERT therapy with agalsidase beta significantly improves function of C-, A{delta}-, and Aß-nerve fibers and intradermal vibration receptors in Fabry neuropathy. Lack of recovery in some patients with abnormal cold or heat-pain perception suggests the need for early ERT, prior to irreversible nerve fiber loss.

Received August 22, 2003. Accepted in final form December 4, 2003.

Dr. M.J. Hilz has served as a consultant to and received honoraria from Genzyme Corp. (One Kendall Square, Cambridge, MA).




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