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Attempted enzyme replacement using human amnion membane implantations in mucopolysaccharidoses

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Journal of Inherited Metabolic Disease

Summary

Amnion membrane implantation has been proposed as an approach to enzyme replacement in mucopolysaccharidoses. Human amnion membranes have been subcutaneously implanted in the abdominal wall in 19 patients with mucopolysaccharidoses (MPS I, II and III). A protocol was developed for the objective evaluation of experimental treatments of these patients. Systematic evaluation of the clinical status before and 6 months after amnion membrane implantation reveals no change in function except improvement in joint mobility. The sum of all joint movements showed improvement from baseline values to 6 months after implantation by ANOVA followed bypost-hoc analysis (p<0.056). The only specific joint movements to significantly improve after 6 months were shoulder extension (p<0.01) and hip internal rotation (p<0.05). Serial measurements of the deficient lysosomal enzyme activity in serum and white blood cells did not increase in any patient after amnion membrane implantation. Urinary glycosaminoglycan excretion decreased transiently in 2 of 10 patients after implantation, but a second amnion membrane implantation did not result in any change. Biopsy of the implantation site in 10 patients 6 months after amnion membrane implantation revealed a foreign-body reaction with giant cell formation and fibrosis and no recognizable amnion membrane tissue. We conclude that human amnion membrane implantation is not an effective therapy in mucopolysaccharidoses.

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Authors and Affiliations

  1. Human Genetics Branch, National Institute of Child Health and Human Development, USA

    J. Muenzer & M. A. Zasloff

  2. Genetics and Biochemistry Branch, National Institute of Diabetes, Digestive and Kidney Diseases, USA

    E. F. Neufeld

  3. Developmental and Metabolic Neurology Branch, National Institute of Neurological and Communicative Disorders and Stroke, USA

    G. Constantopoulos

  4. Ophthalmic Genetics and Clinical Services Branch, National Eye Institute, USA

    R. C. Caruso & M. I. Kaiser-Kupfer

  5. Audiology, USA

    A. Pikus

  6. Rehabilitation Medicine Departments, Warren Grant Magnuson Clinical Center, National Institutes of Health, USA

    J. Danoff & R. R. Berry

  7. Surgery Branch, Division of Cancer Treatment, National Cancer Institute, National Institutes of Health, 20892, Bethesda, MD, USA

    H. D. McDonald

  8. Laboratory of Medical Genetics, University of Alabama, 35294, Birmingham, AL, USA

    J. N. Thompson & L. Rodén

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  1. J. Muenzer
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  2. E. F. Neufeld
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  3. G. Constantopoulos
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  4. R. C. Caruso
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  12. M. A. Zasloff
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Muenzer, J., Neufeld, E.F., Constantopoulos, G. et al. Attempted enzyme replacement using human amnion membane implantations in mucopolysaccharidoses. J Inherit Metab Dis 15, 25–37 (1992). https://doi.org/10.1007/BF01800340

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