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Effects of exercise training during infusion on late-onset Pompe disease patients receiving enzyme replacement therapy.
Mol Genet Metab 2012; 107(4):669-73MG

Abstract

Pompe disease is an autosomal recessive disorder caused by the deficiency of acid α-glucosidase resulting in lysosomal accumulation of glycogen and abnormal autophagic function. The late-onset form of the disease is characterized by progressive skeletal and respiratory muscle dysfunction. Enzyme replacement therapy (ERT, Genzyme Corporation, Cambridge, MA, USA) was recently introduced and resulted in significant prolongation of the life expectancy of the patients with the infantile form while the results were less significant for the late-onset form. It has been postulated that the weak influence of ERT in late-onset patients might be due to a non-effective delivery of the recombinant enzyme to the skeletal muscles perhaps due to the relatively low blood flow to the resting skeletal muscles during infusion. Exercise training acutely increases the blood flow to the exercising muscles. Thus, it was hypothesized that exercise training during enzyme infusion might increase the effectiveness of the ERT therapy. Five late-onset Pompe disease patients receiving ERT and following regular exercise training for approximately 10 months, followed a 6-month period of exercise training during infusion of the recombinant enzyme. Before and after this period, body composition, isometric strength and 6 minute walking distance were determined. Analysis of the results revealed that none of these parameters changed significantly after the 6-month intervention period (e.g. 6 minute walking distance before: 532±31 m, vs. after: 527±29 m, P=0.246). These results suggest that exercise training during infusion may not add significant functional changes in late-onset Pompe patients receiving ERT and undergoing regular exercise training.

Authors+Show Affiliations

1st Department of Neurology, School of Medicine, University of Athens, Eginition Hospital, Greece. gterzis@phed.uoa.grNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article

Language

eng

PubMed ID

23146291

Citation

Terzis, Gerasimos, et al. "Effects of Exercise Training During Infusion On Late-onset Pompe Disease Patients Receiving Enzyme Replacement Therapy." Molecular Genetics and Metabolism, vol. 107, no. 4, 2012, pp. 669-73.
Terzis G, Krase A, Papadimas G, et al. Effects of exercise training during infusion on late-onset Pompe disease patients receiving enzyme replacement therapy. Mol Genet Metab. 2012;107(4):669-73.
Terzis, G., Krase, A., Papadimas, G., Papadopoulos, C., Kavouras, S. A., & Manta, P. (2012). Effects of exercise training during infusion on late-onset Pompe disease patients receiving enzyme replacement therapy. Molecular Genetics and Metabolism, 107(4), pp. 669-73. doi:10.1016/j.ymgme.2012.10.020.
Terzis G, et al. Effects of Exercise Training During Infusion On Late-onset Pompe Disease Patients Receiving Enzyme Replacement Therapy. Mol Genet Metab. 2012;107(4):669-73. PubMed PMID: 23146291.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Effects of exercise training during infusion on late-onset Pompe disease patients receiving enzyme replacement therapy. AU - Terzis,Gerasimos, AU - Krase,Argyro, AU - Papadimas,Giorgos, AU - Papadopoulos,Constantinos, AU - Kavouras,Stavros A, AU - Manta,Panagiota, Y1 - 2012/10/24/ PY - 2012/09/02/received PY - 2012/10/21/revised PY - 2012/10/21/accepted PY - 2012/11/14/entrez PY - 2012/11/14/pubmed PY - 2013/4/26/medline SP - 669 EP - 73 JF - Molecular genetics and metabolism JO - Mol. Genet. Metab. VL - 107 IS - 4 N2 - Pompe disease is an autosomal recessive disorder caused by the deficiency of acid α-glucosidase resulting in lysosomal accumulation of glycogen and abnormal autophagic function. The late-onset form of the disease is characterized by progressive skeletal and respiratory muscle dysfunction. Enzyme replacement therapy (ERT, Genzyme Corporation, Cambridge, MA, USA) was recently introduced and resulted in significant prolongation of the life expectancy of the patients with the infantile form while the results were less significant for the late-onset form. It has been postulated that the weak influence of ERT in late-onset patients might be due to a non-effective delivery of the recombinant enzyme to the skeletal muscles perhaps due to the relatively low blood flow to the resting skeletal muscles during infusion. Exercise training acutely increases the blood flow to the exercising muscles. Thus, it was hypothesized that exercise training during enzyme infusion might increase the effectiveness of the ERT therapy. Five late-onset Pompe disease patients receiving ERT and following regular exercise training for approximately 10 months, followed a 6-month period of exercise training during infusion of the recombinant enzyme. Before and after this period, body composition, isometric strength and 6 minute walking distance were determined. Analysis of the results revealed that none of these parameters changed significantly after the 6-month intervention period (e.g. 6 minute walking distance before: 532±31 m, vs. after: 527±29 m, P=0.246). These results suggest that exercise training during infusion may not add significant functional changes in late-onset Pompe patients receiving ERT and undergoing regular exercise training. SN - 1096-7206 UR - https://www.unboundmedicine.com/medline/citation/23146291/Effects_of_exercise_training_during_infusion_on_late_onset_Pompe_disease_patients_receiving_enzyme_replacement_therapy_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S1096-7192(12)00407-6 DB - PRIME DP - Unbound Medicine ER -