Influence of Fresh Plasma Infusions on Patients with Genetic Mucopolysaccharidosis

Orii, Tadao; Yamaguchi, Mamoru; Minami, Ryoji; Ito, Hiroyoshi; Nakao, Tooru

doi:10.1620/tjem.114.385

Cited by 3 publications

(1 citation statement)

References 10 publications

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“…However, most subsequent studies (Dekaban et al, 1972;Erickson et al, 1972;Bartsocas et al, 1973;Booth and Nadler, 1973;Danes et aI., 1973;Orii et al, 1974) failed to demonstrate improvement except for two that reported moderate clinical benefit (Hussels et al, 1974;Yatziv et al, 1975). Brown et al (1982) attempted enzyme replacement by plasma exchange in Hunter syndrome.…”

Section: Ms Receivedmentioning

confidence: 99%

Attempted enzyme replacement using human amnion membane implantations in mucopolysaccharidoses

Muenzer

Neufeld

Constantopoulos

et al. 1991

J of Inher Metab Disea

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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 by post-hoc analysis (p less than 0.056). The only specific joint movements to significantly improve after 6 months were shoulder extension (p less than 0.01) and hip internal rotation (p less than 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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Section: Ms Receivedmentioning

confidence: 99%

Attempted enzyme replacement using human amnion membane implantations in mucopolysaccharidoses

Muenzer

Neufeld

Constantopoulos

et al. 1991

J of Inher Metab Disea

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Administration of iduronate sulfatase by plasma exchange to patients with the hunter syndrome: A clinical study

et al. 1982

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The Hunter syndrome (MPS II) is the only mucopolysaccharidosis in which there is appreciable activity of the deficient enzyme in normal plasma. We performed enzyme-replacement treatment by plasma exchange in five Hunter syndrome children. Carefully monitoring the cardiovascular status, we administered monthly single plasma volume exchanges for a 3 to 8 mo period. The results indicate a substantial gain of enzyme activity, persisting with a t50% = 19 +/- 5 hr. The maximal level and persistence of increased enzyme activity did not change after repeated exchanges, suggesting that immune responses were not elicited. Despite this, no demonstrable clinical benefit was apparent when the study group was compared with an age-matched control group of Hunter syndrome patients.

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Mucopolysaccharidoses

Muenzer

1986

Advances in Pediatrics

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Influence of Fresh Plasma Infusions on Patients with Genetic Mucopolysaccharidosis

Cited by 3 publications

References 10 publications

Attempted enzyme replacement using human amnion membane implantations in mucopolysaccharidoses

Attempted enzyme replacement using human amnion membane implantations in mucopolysaccharidoses

Administration of iduronate sulfatase by plasma exchange to patients with the hunter syndrome: A clinical study

Mucopolysaccharidoses

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