Cargando…
An overview of the Cure SMA membership database: Highlights of key demographic and clinical characteristics of SMA members
BACKGROUND: The Cure SMA database is one of the largest patient reported databases for people affected with SMA. OBJECTIVE: The purpose of this study was to examine a subset of affected SMA persons with types I, II, and III from a patient reported database. METHODS: Individuals with SMA were selecte...
Autores principales: | , , , , , , , |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
IOS Press
2018
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6004903/ https://www.ncbi.nlm.nih.gov/pubmed/29614694 http://dx.doi.org/10.3233/JND-170292 |
_version_ | 1783332605344612352 |
---|---|
author | Belter, Lisa Cook, Suzanne F. Crawford, Thomas O. Jarecki, Jill Jones, Cynthia C. Kissel, John T. Schroth, Mary Hobby, Kenneth |
author_facet | Belter, Lisa Cook, Suzanne F. Crawford, Thomas O. Jarecki, Jill Jones, Cynthia C. Kissel, John T. Schroth, Mary Hobby, Kenneth |
author_sort | Belter, Lisa |
collection | PubMed |
description | BACKGROUND: The Cure SMA database is one of the largest patient reported databases for people affected with SMA. OBJECTIVE: The purpose of this study was to examine a subset of affected SMA persons with types I, II, and III from a patient reported database. METHODS: Individuals with SMA were selected from the database using a date of first contact to Cure SMA between 2010 and 2016. Data analyzed included age at diagnosis, number of weeks from SMA diagnosis to contacting Cure SMA, and geographic distribution of individuals. RESULTS: A total of 1,966 individuals with SMA were included in the analysis. Of these individuals, 51.9% had type I, 32.3% had type II, and 15.8% had type III. The average age of diagnosis for type I patients was 5.2 months, 22.1 months for type II, and 97.8 months for type III. From published incidence, about 59.0% of affected individuals in the US are registered in the Cure SMA database. CONCLUSIONS: The Cure SMA database is a unique and robust source of patient reported data that informs on the burden of illness and supports the development of new therapeutic modalities. |
format | Online Article Text |
id | pubmed-6004903 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2018 |
publisher | IOS Press |
record_format | MEDLINE/PubMed |
spelling | pubmed-60049032018-06-25 An overview of the Cure SMA membership database: Highlights of key demographic and clinical characteristics of SMA members Belter, Lisa Cook, Suzanne F. Crawford, Thomas O. Jarecki, Jill Jones, Cynthia C. Kissel, John T. Schroth, Mary Hobby, Kenneth J Neuromuscul Dis Research Report BACKGROUND: The Cure SMA database is one of the largest patient reported databases for people affected with SMA. OBJECTIVE: The purpose of this study was to examine a subset of affected SMA persons with types I, II, and III from a patient reported database. METHODS: Individuals with SMA were selected from the database using a date of first contact to Cure SMA between 2010 and 2016. Data analyzed included age at diagnosis, number of weeks from SMA diagnosis to contacting Cure SMA, and geographic distribution of individuals. RESULTS: A total of 1,966 individuals with SMA were included in the analysis. Of these individuals, 51.9% had type I, 32.3% had type II, and 15.8% had type III. The average age of diagnosis for type I patients was 5.2 months, 22.1 months for type II, and 97.8 months for type III. From published incidence, about 59.0% of affected individuals in the US are registered in the Cure SMA database. CONCLUSIONS: The Cure SMA database is a unique and robust source of patient reported data that informs on the burden of illness and supports the development of new therapeutic modalities. IOS Press 2018-05-29 /pmc/articles/PMC6004903/ /pubmed/29614694 http://dx.doi.org/10.3233/JND-170292 Text en © 2018 – IOS Press and the authors. All rights reserved https://creativecommons.org/licenses/by-nc/4.0/ This is an open access article distributed under the terms of the Creative Commons Attribution Non-Commercial (CC BY-NC 4.0) License (https://creativecommons.org/licenses/by-nc/4.0/) , which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited. |
spellingShingle | Research Report Belter, Lisa Cook, Suzanne F. Crawford, Thomas O. Jarecki, Jill Jones, Cynthia C. Kissel, John T. Schroth, Mary Hobby, Kenneth An overview of the Cure SMA membership database: Highlights of key demographic and clinical characteristics of SMA members |
title | An overview of the Cure SMA membership database: Highlights of key demographic and clinical characteristics of SMA members |
title_full | An overview of the Cure SMA membership database: Highlights of key demographic and clinical characteristics of SMA members |
title_fullStr | An overview of the Cure SMA membership database: Highlights of key demographic and clinical characteristics of SMA members |
title_full_unstemmed | An overview of the Cure SMA membership database: Highlights of key demographic and clinical characteristics of SMA members |
title_short | An overview of the Cure SMA membership database: Highlights of key demographic and clinical characteristics of SMA members |
title_sort | overview of the cure sma membership database: highlights of key demographic and clinical characteristics of sma members |
topic | Research Report |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6004903/ https://www.ncbi.nlm.nih.gov/pubmed/29614694 http://dx.doi.org/10.3233/JND-170292 |
work_keys_str_mv | AT belterlisa anoverviewofthecuresmamembershipdatabasehighlightsofkeydemographicandclinicalcharacteristicsofsmamembers AT cooksuzannef anoverviewofthecuresmamembershipdatabasehighlightsofkeydemographicandclinicalcharacteristicsofsmamembers AT crawfordthomaso anoverviewofthecuresmamembershipdatabasehighlightsofkeydemographicandclinicalcharacteristicsofsmamembers AT jareckijill anoverviewofthecuresmamembershipdatabasehighlightsofkeydemographicandclinicalcharacteristicsofsmamembers AT jonescynthiac anoverviewofthecuresmamembershipdatabasehighlightsofkeydemographicandclinicalcharacteristicsofsmamembers AT kisseljohnt anoverviewofthecuresmamembershipdatabasehighlightsofkeydemographicandclinicalcharacteristicsofsmamembers AT schrothmary anoverviewofthecuresmamembershipdatabasehighlightsofkeydemographicandclinicalcharacteristicsofsmamembers AT hobbykenneth anoverviewofthecuresmamembershipdatabasehighlightsofkeydemographicandclinicalcharacteristicsofsmamembers AT belterlisa overviewofthecuresmamembershipdatabasehighlightsofkeydemographicandclinicalcharacteristicsofsmamembers AT cooksuzannef overviewofthecuresmamembershipdatabasehighlightsofkeydemographicandclinicalcharacteristicsofsmamembers AT crawfordthomaso overviewofthecuresmamembershipdatabasehighlightsofkeydemographicandclinicalcharacteristicsofsmamembers AT jareckijill overviewofthecuresmamembershipdatabasehighlightsofkeydemographicandclinicalcharacteristicsofsmamembers AT jonescynthiac overviewofthecuresmamembershipdatabasehighlightsofkeydemographicandclinicalcharacteristicsofsmamembers AT kisseljohnt overviewofthecuresmamembershipdatabasehighlightsofkeydemographicandclinicalcharacteristicsofsmamembers AT schrothmary overviewofthecuresmamembershipdatabasehighlightsofkeydemographicandclinicalcharacteristicsofsmamembers AT hobbykenneth overviewofthecuresmamembershipdatabasehighlightsofkeydemographicandclinicalcharacteristicsofsmamembers |