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Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease.
Genet Med. 2009 Mar; 11(3):210-9.GM

Abstract

PURPOSE

A clinical trial was conducted to evaluate the safety and efficacy of alglucosidase alfa in infants and children with advanced Pompe disease.

METHODS

Open-label, multicenter study of IV alglucosidase alfa treatment in 21 infants 3-43 months old (median 13 months) with minimal acid alpha-glucosidase activity and abnormal left ventricular mass index by echocardiography. Patients received IV alglucosidase alfa every 2 weeks for up to 168 weeks (median 120 weeks). Survival results were compared with an untreated reference cohort.

RESULTS

At study end, 71% (15/21) of patients were alive and 44% (7/16) of invasive-ventilator free patients remained so. Compared with the untreated reference cohort, alglucosidase alfa reduced the risk of death by 79% (P < 0.001) and the risk of invasive ventilation by 58% (P = 0.02). Left ventricular mass index improved or remained normal in all patients evaluated beyond 12 weeks; 62% (13/21) achieved new motor milestones. Five patients were walking independently at the end of the study and 86% (18/21) gained functional independence skills. Overall, 52% (11/21) of patients experienced infusion-associated reactions; 95% (19/20) developed IgG antibodies to recombinant human lysosomal acid alpha-glucosidase; no patients withdrew from the study because of safety concerns.

CONCLUSIONS

In this population of infants with advanced disease, biweekly infusions with alglucosidase alfa prolonged survival and invasive ventilation-free survival. Treatment also improved indices of cardiomyopathy, motor skills, and functional independence.

Authors+Show Affiliations

Division of Pediatric Endocrinology, Diabetology and Metabolism, Hôpital Debrousse, University Lyon, Lyon, France.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Clinical Trial
Journal Article
Multicenter Study
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

19287243

Citation

Nicolino, Marc, et al. "Clinical Outcomes After Long-term Treatment With Alglucosidase Alfa in Infants and Children With Advanced Pompe Disease." Genetics in Medicine : Official Journal of the American College of Medical Genetics, vol. 11, no. 3, 2009, pp. 210-9.
Nicolino M, Byrne B, Wraith JE, et al. Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease. Genet Med. 2009;11(3):210-9.
Nicolino, M., Byrne, B., Wraith, J. E., Leslie, N., Mandel, H., Freyer, D. R., Arnold, G. L., Pivnick, E. K., Ottinger, C. J., Robinson, P. H., Loo, J. C., Smitka, M., Jardine, P., Tatò, L., Chabrol, B., McCandless, S., Kimura, S., Mehta, L., Bali, D., ... Kishnani, P. S. (2009). Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease. Genetics in Medicine : Official Journal of the American College of Medical Genetics, 11(3), 210-9. https://doi.org/10.1097/GIM.0b013e31819d0996
Nicolino M, et al. Clinical Outcomes After Long-term Treatment With Alglucosidase Alfa in Infants and Children With Advanced Pompe Disease. Genet Med. 2009;11(3):210-9. PubMed PMID: 19287243.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease. AU - Nicolino,Marc, AU - Byrne,Barry, AU - Wraith,J Edmund, AU - Leslie,Nancy, AU - Mandel,Hanna, AU - Freyer,David R, AU - Arnold,Georgianne L, AU - Pivnick,Eniko K, AU - Ottinger,C J, AU - Robinson,Peter H, AU - Loo,John-Charles A, AU - Smitka,Martin, AU - Jardine,Philip, AU - Tatò,Luciano, AU - Chabrol,Brigitte, AU - McCandless,Shawn, AU - Kimura,Shigemi, AU - Mehta,L, AU - Bali,Deeksha, AU - Skrinar,Alison, AU - Morgan,Claire, AU - Rangachari,Lakshmi, AU - Corzo,Deya, AU - Kishnani,Priya S, PY - 2009/3/17/entrez PY - 2009/3/17/pubmed PY - 2009/9/26/medline SP - 210 EP - 9 JF - Genetics in medicine : official journal of the American College of Medical Genetics JO - Genet. Med. VL - 11 IS - 3 N2 - PURPOSE: A clinical trial was conducted to evaluate the safety and efficacy of alglucosidase alfa in infants and children with advanced Pompe disease. METHODS: Open-label, multicenter study of IV alglucosidase alfa treatment in 21 infants 3-43 months old (median 13 months) with minimal acid alpha-glucosidase activity and abnormal left ventricular mass index by echocardiography. Patients received IV alglucosidase alfa every 2 weeks for up to 168 weeks (median 120 weeks). Survival results were compared with an untreated reference cohort. RESULTS: At study end, 71% (15/21) of patients were alive and 44% (7/16) of invasive-ventilator free patients remained so. Compared with the untreated reference cohort, alglucosidase alfa reduced the risk of death by 79% (P < 0.001) and the risk of invasive ventilation by 58% (P = 0.02). Left ventricular mass index improved or remained normal in all patients evaluated beyond 12 weeks; 62% (13/21) achieved new motor milestones. Five patients were walking independently at the end of the study and 86% (18/21) gained functional independence skills. Overall, 52% (11/21) of patients experienced infusion-associated reactions; 95% (19/20) developed IgG antibodies to recombinant human lysosomal acid alpha-glucosidase; no patients withdrew from the study because of safety concerns. CONCLUSIONS: In this population of infants with advanced disease, biweekly infusions with alglucosidase alfa prolonged survival and invasive ventilation-free survival. Treatment also improved indices of cardiomyopathy, motor skills, and functional independence. SN - 1530-0366 UR - https://www.unboundmedicine.com/medline/citation/19287243/Clinical_outcomes_after_long_term_treatment_with_alglucosidase_alfa_in_infants_and_children_with_advanced_Pompe_disease_ L2 - http://dx.doi.org/10.1097/GIM.0b013e31819d0996 DB - PRIME DP - Unbound Medicine ER -