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01 April 2011

Two-year follow-up of Sanfilippo Disease patients treated with a genistein-rich isoflavone extract: Assessment of effects on cognitive functions and general status of patients

Ewa PiotrowskaABCD, Joanna Jakobkiewicz-BaneckaACDE, Agnieszka MaryniakBCD, Anna Tylki-SzymanskaACDEF, Ewa PukA, Anna LiberekCF, Alicja WegrzynDEF, Barbara CzartoryskaCD, Monika Slominska-WojewodzkaEF, Grzegorz WegrzynADEFG

DOI: 10.12659/MSM.881715

Med Sci Monit 2011; 17(4): CR196-202


Background: Mucopolysaccharidoses (MPS) are inherited metabolic disorders caused by deficiencies in enzymes involved in degradation of glycosaminoglycans. MPS type III (Sanfilippo disease) is clinically characterized mainly by progressive and severe behavioral disturbances and cognitive dysfunction. Recent 1-year experimental treatment of 10 patients with a genistein (4’, 5, 7-trihydroxyisoflavone)-rich extract resulted in improvement of tested parameters, including cognitive and behavioral functions.
Material/Methods: Eight pediatric patients with Sanfilippo disease were enrolled into the study. The modified version of the Brief Assessment Examination was used to assess cognitive functions. Moreover, 18 different parameters concerning changes in conditions of patients were assessed by their parents.
Results: During the first year of the treatment, an improvement of cognitive functions in 7 patients and stabilization in 1 patient were assessed, while after the third year (2-year follow-up) further improvement was observed in 2 patients, stabilization in 3 patients and some deterioration in 3 patients. Monitoring of general and behavioral symptoms revealed improvement in all patients after the first year of the treatment, further improvement in 5 patients, and deterioration in 3 patients during the next 2 years.
Conclusions: We conclude that the treatment of Sanfilippo patients with a genistein-rich soy isoflavone extract (called gene expression-targeted isoflavone therapy [GET IT]) may be effective in either inhibition (in some patients) or slowing down (in other patients) of behavioral and cognitive problems over a longer period. An increased dose of genistein may improve the efficacy of the treatment.

Keywords: Mucopolysaccharidosis III - physiopathology, Genistein - therapeutic use, Follow-Up Studies, Cognition - physiology, Child, Preschool, Child, Adolescent, Parents, Phytotherapy, Plant Extracts - therapeutic use

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Medical Science Monitor eISSN: 1643-3750
Medical Science Monitor eISSN: 1643-3750