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Biochemical Changes in Blood of Patients with Duchenne Muscular Dystrophy Treated with Granulocyte-Colony Stimulating Factor.
Sienkiewicz, Dorota; Kulak, Wojciech; Paszko-Patej, Grazyna; Okurowska-Zawada, Bozena; Sienkiewicz, Jerzy; Kulak, Piotr.
Afiliación
  • Sienkiewicz D; Department of Pediatric Rehabilitation, Medical University of Bialystok, Bialystok, Poland.
  • Kulak W; Department of Pediatric Rehabilitation, Medical University of Bialystok, Bialystok, Poland.
  • Paszko-Patej G; Department of Pediatric Rehabilitation, Medical University of Bialystok, Bialystok, Poland.
  • Okurowska-Zawada B; Department of Pediatric Rehabilitation, Medical University of Bialystok, Bialystok, Poland.
  • Sienkiewicz J; Bialystok Technical University, Bialystok, Poland.
  • Kulak P; Department of Pediatric Radiology, Medical University of Bialystok, Bialystok, Poland.
Biomed Res Int ; 2019: 4789101, 2019.
Article en En | MEDLINE | ID: mdl-31001554
INTRODUCTION: In addition to the "gold standard" of therapy-steroids and gene therapy-there are experimental trials using granulocyte-colony stimulating factor (G-CSF) for patients with Duchenne muscular dystrophy (DMD). The aim of this study was to present the biochemical changes in blood after repeating cycles of granulocyte-colony stimulating factor G-CSF therapy in children with DMD. MATERIALS AND METHODS: Nineteen patients, aged 5 to 15 years, with diagnosed DMD confirmed by genetic tests, participated; nine were in wheelchairs, and ten were mobile and independent. Patients had a clinical assessment and laboratory tests to evaluate hematological parameters and biochemistry. G-CSF (5µg/kg/day) was given subcutaneously for five days during five nonconsecutive months over the course of a year. RESULTS: We found a significant elevation of white blood cells, and the level of leucocytes returned to norm after each cycle. No signs of any inflammatory process were found by monitoring C-reactive protein. We did not detect significant changes in red blood cells, hemoglobin, and platelet levels or coagulation parameters. We found a significant elevation of uric acid, with normalization after finishing each treatment cycle. A significant decrease of the mean value activity of aspartate transaminase (AST) and alanine transaminase (ALT) of the G-CSF treatment was noted. After each five days of therapy, the level of cholesterol was significantly lowered. Also, glucose concentration significantly decreased after the fourth cycle. CONCLUSIONS: G-SCF decreased the aminotransferases activity, cholesterol level, and glucose level in patients with DMD, which may be important for patients with DMD and metabolic syndrome.
Asunto(s)

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Factor Estimulante de Colonias de Granulocitos / Distrofia Muscular de Duchenne Tipo de estudio: Observational_studies Límite: Adolescent / Child / Child, preschool / Female / Humans / Male Idioma: En Revista: Biomed Res Int Año: 2019 Tipo del documento: Article País de afiliación: Polonia Pais de publicación: Estados Unidos

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Factor Estimulante de Colonias de Granulocitos / Distrofia Muscular de Duchenne Tipo de estudio: Observational_studies Límite: Adolescent / Child / Child, preschool / Female / Humans / Male Idioma: En Revista: Biomed Res Int Año: 2019 Tipo del documento: Article País de afiliación: Polonia Pais de publicación: Estados Unidos