Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa

Ploeg, Ans T. van der; Barohn, Richard J.; Carlson, Lisa; Charrow, Joel; Clemens, Paula R.; Hopkin, Robert J.; Kishnani, Priya S.; Laforêt, Pascal; Morgan, Claire; Nations, Sharon P.; Pestronk, Alan; Plotkin, Horacio; Rosenbloom, Barry E.; Sims, Katherine B.; Tsao, Elisa

doi:10.1016/j.ymgme.2012.09.015

Cited by 96 publications

(101 citation statements)

References 11 publications

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“…The approval of recombinant human GAA (rhGAA) (Myozyme) for the treatment of Pompe disease resulted in a significant improvement of both life expectancy and quality of life of infantile PD patients7, although long-term follow up of children treated with enzyme replacement therapy (ERT) revealed occurrence of symptoms resulting from the incomplete correction of the enzyme deficiency in certain tissues8. Approval of ERT for LOPD patients followed that of pediatric subjects few years later, and long-term benefit of ERT in this population is still being assessed91011.…”

mentioning

confidence: 99%

Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients

Masat

Pascal

Antonio

et al. 2016

Sci Rep

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Immunogenicity of recombinant human acid-alpha glucosidase (rhGAA) in enzyme replacement therapy (ERT) is a safety and efficacy concern in the management of late-onset Pompe disease (LOPD). However, long-term effects of ERT on humoral and cellular responses to rhGAA are still poorly understood. To better understand the impact of immunogenicity of rhGAA on the efficacy of ERT, clinical data and blood samples from LOPD patients undergoing ERT for >4 years (n = 28) or untreated (n = 10) were collected and analyzed. In treated LOPD patients, anti-rhGAA antibodies peaked within the first 1000 days of ERT, while long-term exposure to rhGAA resulted in clearance of antibodies with residual production of non-neutralizing IgG. Analysis of T cell responses to rhGAA showed detectable T cell reactivity only after in vitro restimulation. Upregulation of several cytokines and chemokines was detectable in both treated and untreated LOPD subjects, while IL2 secretion was detectable only in subjects who received ERT. These results indicate that long-term ERT in LOPD patients results in a decrease in antibody titers and residual production of non-inhibitory IgGs. Immune responses to GAA following long-term ERT do not seem to affect efficacy of ERT and are consistent with an immunomodulatory effect possibly mediated by regulatory T cells.

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mentioning

confidence: 99%

Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients

Masat

Pascal

Antonio

et al. 2016

Sci Rep

Self Cite

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“…The Late-Onset Treatment Study and its extension trial concluded that the treatment of Pompe disease with ERT was associated with improved walking distance and stabilization of pulmonary function over an 18-month period when compared to baseline. 9 Thus, ERT may affect the natural history of this progressive and degenerative disease. Therefore it is essential that any patient with Pompe disease be recognized and diagnosed quickly.…”

Section: Sectionmentioning

confidence: 99%

Clinical Reasoning: A 38-year-old man with respiratory failure and progressive leg weakness

McIntosh

Karam²

2016

Neurology

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“…Разработка и включение с 2006 г. в стандарты лече-ния БП в США и странах Европы, а с 2014 г. в России ферментозаместительной терапии (ФЗТ) позволили снизить смертность пациентов с БП, уменьшить выра-женность клинических проявлений и улучшить каче-ство жизни больных с БП [8,9]. Возможность терапии с использованием ФЗТ привела к выявлению до 7 % случаев БППН при направленном диагностическом скрининге активности α-ГЗД по DBS суммарно у 500 больных из разных клинических групп -пациентов с бессимптомной и малосимптомной персистирующей гиперкреатинкиназемией, неклассифицируемыми ми-опатиями, гиперкреатинкиназемией ПКМД с неуста-новленным генетически дефектом [10][11][12].…”

Section: Introductionunclassified

Late-onset Pompe disease with phenotype of the limb-girdle muscular dystrophy

Kurbatov¹,

Никитин²,

Захарова³

2015

Neuromuscular Diseases

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Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa

Cited by 96 publications

References 11 publications

Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients

Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients

Clinical Reasoning: A 38-year-old man with respiratory failure and progressive leg weakness

Late-onset Pompe disease with phenotype of the limb-girdle muscular dystrophy

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