The cooperative international neuromuscular research group Duchenne natural history study--a longitudinal investigation in the era of glucocorticoid therapy: design of protocol and the methods used
Craig M McDonald, Erik K Henricson, R Ted Abresch, Jay J Han, Diana M Escolar, Julaine M Florence, Tina Duong, Adrienne Arrieta, Paula R Clemens, Eric P Hoffman, Avital Cnaan, Cinrg Investigators, V Vishwanathan, S Chidambaranathan, W Douglas Biggar, Jean K Mah, Mar Tulinius, Robert Leshner, Andrew Kornberg, Monique Ryan, Yoram Nevo, Alberto Dubrovsky, Nancy Kuntz, Sherilyn Driscoll, Anne Connolly, Alan Pestronk, Jean Teasley, Tulio Bertorini, Kathryn North, Hanna Kolski, Jose Carlo, Ksenija Gorni, Timothy Lotze, John Day, Michelle Cregan, Erica Goude, Merete Glick, Linda Johnson, Nanette Joyce, Alina Nicorici, Andrew Skalsky, Amanda Witt, Bethany Lipa, Suresh Kumar, Laila Eliasoph, Elizabeth Hosaki, Angela Gonzales, Vivien Harris, Angela Chiu, Edit Goia, Jennifer Thannhauser, Lori Walker, Caitlin Wright, Mehrnaz Yousefi, Ann-Christine Alhander, Lisa Berglund, Ann-Berit Ekstrom, Anna-Karin Kroksmark, Ulrika Sterky, Marissa Birkmeier, Sarah Kaminski, Katie Parker, Kate Carroll, Katy DeValle, Rachel Kennedy, Dani Villano, Adina Bar Leve, Itai Shurr, Elana Wisband, Debbie Yaffe, Luz Andreone, Jose Corderi, Lilia Mesa, Lorena Levi, Krista Coleman-Wood, Ann Hoffman, Wendy Korn-Petersen, Duygu Selcen, Hoda Abdel-Hamid, Christopher Bise, Ann Craig, Lauren Hache, Sarah Hughes, Casey Nguyen, Jason Weimer, Paul Golumbak, Glenn Lopate, Justin Malane, Betsy Malkus, Kenkicki Nozaki, Renee Renna, Jeanine Schierbacker, Catherine Seiner, Charlie Wulf, Susan Blair, Barbara Grillo, Karen Jones, Eugenio Monasterio, Judy Clift, Cassandra Feliciano, Masanori Igarashi, Rachel Young, Kristy Rose, Richard Webster, Stephanie Wicks, Lucia Chen, Cameron Kennedy, Brenda Deliz, Sheila Espada, Pura Fuste, Carlos Luciano, Luca Capone, Maria Beneggi, Valentina Morettini, Anjali Gupta, Robert McNeil, Amy Erickson, Marcia Margolis, Cameron Naughton, Gareth Parry, David Walk, Naomi Bartley, Paola Canelos, Robert Casper, Lauren Hache, Corinne Ingram, Fengming Hu, Mohammad Ahmed, Angela Zimmerman, Craig M McDonald, Erik K Henricson, R Ted Abresch, Jay J Han, Diana M Escolar, Julaine M Florence, Tina Duong, Adrienne Arrieta, Paula R Clemens, Eric P Hoffman, Avital Cnaan, Cinrg Investigators, V Vishwanathan, S Chidambaranathan, W Douglas Biggar, Jean K Mah, Mar Tulinius, Robert Leshner, Andrew Kornberg, Monique Ryan, Yoram Nevo, Alberto Dubrovsky, Nancy Kuntz, Sherilyn Driscoll, Anne Connolly, Alan Pestronk, Jean Teasley, Tulio Bertorini, Kathryn North, Hanna Kolski, Jose Carlo, Ksenija Gorni, Timothy Lotze, John Day, Michelle Cregan, Erica Goude, Merete Glick, Linda Johnson, Nanette Joyce, Alina Nicorici, Andrew Skalsky, Amanda Witt, Bethany Lipa, Suresh Kumar, Laila Eliasoph, Elizabeth Hosaki, Angela Gonzales, Vivien Harris, Angela Chiu, Edit Goia, Jennifer Thannhauser, Lori Walker, Caitlin Wright, Mehrnaz Yousefi, Ann-Christine Alhander, Lisa Berglund, Ann-Berit Ekstrom, Anna-Karin Kroksmark, Ulrika Sterky, Marissa Birkmeier, Sarah Kaminski, Katie Parker, Kate Carroll, Katy DeValle, Rachel Kennedy, Dani Villano, Adina Bar Leve, Itai Shurr, Elana Wisband, Debbie Yaffe, Luz Andreone, Jose Corderi, Lilia Mesa, Lorena Levi, Krista Coleman-Wood, Ann Hoffman, Wendy Korn-Petersen, Duygu Selcen, Hoda Abdel-Hamid, Christopher Bise, Ann Craig, Lauren Hache, Sarah Hughes, Casey Nguyen, Jason Weimer, Paul Golumbak, Glenn Lopate, Justin Malane, Betsy Malkus, Kenkicki Nozaki, Renee Renna, Jeanine Schierbacker, Catherine Seiner, Charlie Wulf, Susan Blair, Barbara Grillo, Karen Jones, Eugenio Monasterio, Judy Clift, Cassandra Feliciano, Masanori Igarashi, Rachel Young, Kristy Rose, Richard Webster, Stephanie Wicks, Lucia Chen, Cameron Kennedy, Brenda Deliz, Sheila Espada, Pura Fuste, Carlos Luciano, Luca Capone, Maria Beneggi, Valentina Morettini, Anjali Gupta, Robert McNeil, Amy Erickson, Marcia Margolis, Cameron Naughton, Gareth Parry, David Walk, Naomi Bartley, Paola Canelos, Robert Casper, Lauren Hache, Corinne Ingram, Fengming Hu, Mohammad Ahmed, Angela Zimmerman
Abstract
Contemporary natural history data in Duchenne muscular dystrophy (DMD) is needed to assess care recommendations and aid in planning future trials.
Methods: The Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study (DMD-NHS) enrolled 340 individuals, aged 2-28 years, with DMD in a longitudinal, observational study at 20 centers. Assessments obtained every 3 months for 1 year, at 18 months, and annually thereafter included: clinical history; anthropometrics; goniometry; manual muscle testing; quantitative muscle strength; timed function tests; pulmonary function; and patient-reported outcomes/health-related quality-of-life instruments.
Results: Glucocorticoid (GC) use at baseline was 62% present, 14% past, and 24% GC-naive. In those ≥6 years of age, 16% lost ambulation over the first 12 months (mean age 10.8 years).
Conclusions: Detailed information on the study methodology of the CINRG DMD-NHS lays the groundwork for future analyses of prospective longitudinal natural history data. These data will assist investigators in designing clinical trials of novel therapeutics.
Copyright © 2013 Wiley Periodicals, Inc.
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Source: PubMed