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IGF-1 in autosomal dominant cerebellar ataxia - open-label trial

BACKGROUND: The objective of this clinical open-label trial was to test the safety, tolerability and efficacy of IGF-1 therapy for autosomal dominant cerebellar ataxia (ADCA) patients. RESULTS: A total of 19 molecularly confirmed patients with SCA3, 1 patient with SCA6 and 6 patients with SCA7 compl...

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Autores principales: Sanz-Gallego, Irene, Rodriguez-de-Rivera, Francisco J, Pulido, Irene, Torres-Aleman, Ignacio, Arpa, Javier
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4552149/
https://www.ncbi.nlm.nih.gov/pubmed/26331037
http://dx.doi.org/10.1186/s40673-014-0013-8
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author Sanz-Gallego, Irene
Rodriguez-de-Rivera, Francisco J
Pulido, Irene
Torres-Aleman, Ignacio
Arpa, Javier
author_facet Sanz-Gallego, Irene
Rodriguez-de-Rivera, Francisco J
Pulido, Irene
Torres-Aleman, Ignacio
Arpa, Javier
author_sort Sanz-Gallego, Irene
collection PubMed
description BACKGROUND: The objective of this clinical open-label trial was to test the safety, tolerability and efficacy of IGF-1 therapy for autosomal dominant cerebellar ataxia (ADCA) patients. RESULTS: A total of 19 molecularly confirmed patients with SCA3, 1 patient with SCA6 and 6 patients with SCA7 completed our study. They were 8 females and 18 males, 28 to 74 years of age (average ± SD: 49.3 ± 14.1). Patients were treated with IGF-1 therapy with a dosage of 50 μg/kg twice a day for 12 months. The efficacy of this therapy was assessed by change from baseline on the scale for the assessment and rating of ataxia (SARA). Ten patients, consecutively selected, continued their assigned dosages in a second year open-label extension trial. A statistically significant improvement in SARA scores was observed for patients with SCA3, patients with SCA7 and all patients grouped together after the first year of IGF-1 therapy, while a stabilization of the disease was confirmed during the second year (extension study). The single patient with SCA6 showed 3 improvement points in SARA score after 3 four-month periods of IGF-1 therapy when compared with baseline measurements. Our data indicate that IGF-1 is safe and well tolerated in general. CONCLUSIONS: Our data, in comparison with results from previous cohorts, indicate a trend for IGF-1 treatment to stabilize the disease progression for patients with SCA, indicating that IGF-1 therapy is able to decrease the progressivity of ADCA.
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spelling pubmed-45521492015-09-01 IGF-1 in autosomal dominant cerebellar ataxia - open-label trial Sanz-Gallego, Irene Rodriguez-de-Rivera, Francisco J Pulido, Irene Torres-Aleman, Ignacio Arpa, Javier Cerebellum Ataxias Research BACKGROUND: The objective of this clinical open-label trial was to test the safety, tolerability and efficacy of IGF-1 therapy for autosomal dominant cerebellar ataxia (ADCA) patients. RESULTS: A total of 19 molecularly confirmed patients with SCA3, 1 patient with SCA6 and 6 patients with SCA7 completed our study. They were 8 females and 18 males, 28 to 74 years of age (average ± SD: 49.3 ± 14.1). Patients were treated with IGF-1 therapy with a dosage of 50 μg/kg twice a day for 12 months. The efficacy of this therapy was assessed by change from baseline on the scale for the assessment and rating of ataxia (SARA). Ten patients, consecutively selected, continued their assigned dosages in a second year open-label extension trial. A statistically significant improvement in SARA scores was observed for patients with SCA3, patients with SCA7 and all patients grouped together after the first year of IGF-1 therapy, while a stabilization of the disease was confirmed during the second year (extension study). The single patient with SCA6 showed 3 improvement points in SARA score after 3 four-month periods of IGF-1 therapy when compared with baseline measurements. Our data indicate that IGF-1 is safe and well tolerated in general. CONCLUSIONS: Our data, in comparison with results from previous cohorts, indicate a trend for IGF-1 treatment to stabilize the disease progression for patients with SCA, indicating that IGF-1 therapy is able to decrease the progressivity of ADCA. BioMed Central 2014-10-02 /pmc/articles/PMC4552149/ /pubmed/26331037 http://dx.doi.org/10.1186/s40673-014-0013-8 Text en © Sanz-Gallego et al.; licensee BioMed Central Ltd. 2014 This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly credited. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
spellingShingle Research
Sanz-Gallego, Irene
Rodriguez-de-Rivera, Francisco J
Pulido, Irene
Torres-Aleman, Ignacio
Arpa, Javier
IGF-1 in autosomal dominant cerebellar ataxia - open-label trial
title IGF-1 in autosomal dominant cerebellar ataxia - open-label trial
title_full IGF-1 in autosomal dominant cerebellar ataxia - open-label trial
title_fullStr IGF-1 in autosomal dominant cerebellar ataxia - open-label trial
title_full_unstemmed IGF-1 in autosomal dominant cerebellar ataxia - open-label trial
title_short IGF-1 in autosomal dominant cerebellar ataxia - open-label trial
title_sort igf-1 in autosomal dominant cerebellar ataxia - open-label trial
topic Research
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4552149/
https://www.ncbi.nlm.nih.gov/pubmed/26331037
http://dx.doi.org/10.1186/s40673-014-0013-8
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