Prevalence of duchenne and becker muscular dystrophies in the United States

Paul A. Romitti, Yong Zhu, Soman Puzhankara, Katherine A. James, Sarah K. Nabukera, Gideon K D Zamba, Emma Ciafaloni, Christopher M Cunniff, Charlotte M. Druschel, Katherine D. Mathews, Dennis J. Matthews, F. John Meaney, Jennifer G. Andrews, Kristin M. Caspers Conway, Deborah J. Fox, Natalie Street, Melissa M. Adams, Julie Bolen

Research output: Contribution to journalArticle

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Abstract

OBJECTIVE: To estimate prevalence of childhood-onset Duchenne and Becker muscular dystrophies (DBMD) in 6 sites in the United States by race/ethnicity and phenotype (Duchenne muscular dystrophy [DMD] or Becker muscular dystrophy [BMD]). METHODS: In 2002, the Centers for Disease Control and Prevention established the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) to conduct longitudinal, population-based surveillance and research of DBMD in the United States. Six sites conducted active, multiple-source case finding and record abstraction to identify MD STARnet cases born January 1982 to December 2011. We used cross-sectional analyses to estimate prevalence of DBMD per 10 000 boys, ages 5 to 9 years, for 4 quinquennia (1991-1995, 1996-2000, 2001-2005, and 2006-2010) and prevalence per 10 000 male individuals, ages 5 to 24 years, in 2010. Prevalence was also estimated by race/ethnicity and phenotype. RESULTS: Overall, 649 cases resided in an MD STARnet site during ≥1 quinquennia. Prevalence estimates per 10 000 boys, ages 5 to 9 years, were 1.93, 2.05, 2.04, and 1.51, respectively, for 1991-1995, 1996-2000, 2001-2005, and 2006-2010. Prevalence tended to be higher for Hispanic individuals than non-Hispanic white or black individuals, and higher for DMD than BMD. In 2010, prevalence of DBMD was 1.38 per 10 000 male individuals, ages 5 to 24 years. CONCLUSIONS: We present population-based prevalence estimates for DBMD in 6 US sites. Prevalence differed by race/ethnicity, suggesting potential cultural and socioeconomic influences in the diagnosis of DBMD. Prevalence also was higher for DMD than BMD. Continued longitudinal surveillance will permit us to examine racial/ethnic and socioeconomic differences in treatment and outcomes for MD STARnet cases.

Original languageEnglish (US)
Pages (from-to)513-521
Number of pages9
JournalPediatrics
Volume135
Issue number3
DOIs
StatePublished - Mar 1 2015

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Duchenne Muscular Dystrophy
Muscular Dystrophies
Research
Population Surveillance
Phenotype
Centers for Disease Control and Prevention (U.S.)
Hispanic Americans
Catalytic Domain
Cross-Sectional Studies

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Medicine(all)

Cite this

Romitti, P. A., Zhu, Y., Puzhankara, S., James, K. A., Nabukera, S. K., Zamba, G. K. D., ... Bolen, J. (2015). Prevalence of duchenne and becker muscular dystrophies in the United States. Pediatrics, 135(3), 513-521. https://doi.org/10.1542/peds.2014-2044

Prevalence of duchenne and becker muscular dystrophies in the United States. / Romitti, Paul A.; Zhu, Yong; Puzhankara, Soman; James, Katherine A.; Nabukera, Sarah K.; Zamba, Gideon K D; Ciafaloni, Emma; Cunniff, Christopher M; Druschel, Charlotte M.; Mathews, Katherine D.; Matthews, Dennis J.; Meaney, F. John; Andrews, Jennifer G.; Caspers Conway, Kristin M.; Fox, Deborah J.; Street, Natalie; Adams, Melissa M.; Bolen, Julie.

In: Pediatrics, Vol. 135, No. 3, 01.03.2015, p. 513-521.

Research output: Contribution to journalArticle

Romitti, PA, Zhu, Y, Puzhankara, S, James, KA, Nabukera, SK, Zamba, GKD, Ciafaloni, E, Cunniff, CM, Druschel, CM, Mathews, KD, Matthews, DJ, Meaney, FJ, Andrews, JG, Caspers Conway, KM, Fox, DJ, Street, N, Adams, MM & Bolen, J 2015, 'Prevalence of duchenne and becker muscular dystrophies in the United States', Pediatrics, vol. 135, no. 3, pp. 513-521. https://doi.org/10.1542/peds.2014-2044
Romitti PA, Zhu Y, Puzhankara S, James KA, Nabukera SK, Zamba GKD et al. Prevalence of duchenne and becker muscular dystrophies in the United States. Pediatrics. 2015 Mar 1;135(3):513-521. https://doi.org/10.1542/peds.2014-2044
Romitti, Paul A. ; Zhu, Yong ; Puzhankara, Soman ; James, Katherine A. ; Nabukera, Sarah K. ; Zamba, Gideon K D ; Ciafaloni, Emma ; Cunniff, Christopher M ; Druschel, Charlotte M. ; Mathews, Katherine D. ; Matthews, Dennis J. ; Meaney, F. John ; Andrews, Jennifer G. ; Caspers Conway, Kristin M. ; Fox, Deborah J. ; Street, Natalie ; Adams, Melissa M. ; Bolen, Julie. / Prevalence of duchenne and becker muscular dystrophies in the United States. In: Pediatrics. 2015 ; Vol. 135, No. 3. pp. 513-521.
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abstract = "OBJECTIVE: To estimate prevalence of childhood-onset Duchenne and Becker muscular dystrophies (DBMD) in 6 sites in the United States by race/ethnicity and phenotype (Duchenne muscular dystrophy [DMD] or Becker muscular dystrophy [BMD]). METHODS: In 2002, the Centers for Disease Control and Prevention established the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) to conduct longitudinal, population-based surveillance and research of DBMD in the United States. Six sites conducted active, multiple-source case finding and record abstraction to identify MD STARnet cases born January 1982 to December 2011. We used cross-sectional analyses to estimate prevalence of DBMD per 10 000 boys, ages 5 to 9 years, for 4 quinquennia (1991-1995, 1996-2000, 2001-2005, and 2006-2010) and prevalence per 10 000 male individuals, ages 5 to 24 years, in 2010. Prevalence was also estimated by race/ethnicity and phenotype. RESULTS: Overall, 649 cases resided in an MD STARnet site during ≥1 quinquennia. Prevalence estimates per 10 000 boys, ages 5 to 9 years, were 1.93, 2.05, 2.04, and 1.51, respectively, for 1991-1995, 1996-2000, 2001-2005, and 2006-2010. Prevalence tended to be higher for Hispanic individuals than non-Hispanic white or black individuals, and higher for DMD than BMD. In 2010, prevalence of DBMD was 1.38 per 10 000 male individuals, ages 5 to 24 years. CONCLUSIONS: We present population-based prevalence estimates for DBMD in 6 US sites. Prevalence differed by race/ethnicity, suggesting potential cultural and socioeconomic influences in the diagnosis of DBMD. Prevalence also was higher for DMD than BMD. Continued longitudinal surveillance will permit us to examine racial/ethnic and socioeconomic differences in treatment and outcomes for MD STARnet cases.",
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T1 - Prevalence of duchenne and becker muscular dystrophies in the United States

AU - Romitti, Paul A.

AU - Zhu, Yong

AU - Puzhankara, Soman

AU - James, Katherine A.

AU - Nabukera, Sarah K.

AU - Zamba, Gideon K D

AU - Ciafaloni, Emma

AU - Cunniff, Christopher M

AU - Druschel, Charlotte M.

AU - Mathews, Katherine D.

AU - Matthews, Dennis J.

AU - Meaney, F. John

AU - Andrews, Jennifer G.

AU - Caspers Conway, Kristin M.

AU - Fox, Deborah J.

AU - Street, Natalie

AU - Adams, Melissa M.

AU - Bolen, Julie

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N2 - OBJECTIVE: To estimate prevalence of childhood-onset Duchenne and Becker muscular dystrophies (DBMD) in 6 sites in the United States by race/ethnicity and phenotype (Duchenne muscular dystrophy [DMD] or Becker muscular dystrophy [BMD]). METHODS: In 2002, the Centers for Disease Control and Prevention established the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) to conduct longitudinal, population-based surveillance and research of DBMD in the United States. Six sites conducted active, multiple-source case finding and record abstraction to identify MD STARnet cases born January 1982 to December 2011. We used cross-sectional analyses to estimate prevalence of DBMD per 10 000 boys, ages 5 to 9 years, for 4 quinquennia (1991-1995, 1996-2000, 2001-2005, and 2006-2010) and prevalence per 10 000 male individuals, ages 5 to 24 years, in 2010. Prevalence was also estimated by race/ethnicity and phenotype. RESULTS: Overall, 649 cases resided in an MD STARnet site during ≥1 quinquennia. Prevalence estimates per 10 000 boys, ages 5 to 9 years, were 1.93, 2.05, 2.04, and 1.51, respectively, for 1991-1995, 1996-2000, 2001-2005, and 2006-2010. Prevalence tended to be higher for Hispanic individuals than non-Hispanic white or black individuals, and higher for DMD than BMD. In 2010, prevalence of DBMD was 1.38 per 10 000 male individuals, ages 5 to 24 years. CONCLUSIONS: We present population-based prevalence estimates for DBMD in 6 US sites. Prevalence differed by race/ethnicity, suggesting potential cultural and socioeconomic influences in the diagnosis of DBMD. Prevalence also was higher for DMD than BMD. Continued longitudinal surveillance will permit us to examine racial/ethnic and socioeconomic differences in treatment and outcomes for MD STARnet cases.

AB - OBJECTIVE: To estimate prevalence of childhood-onset Duchenne and Becker muscular dystrophies (DBMD) in 6 sites in the United States by race/ethnicity and phenotype (Duchenne muscular dystrophy [DMD] or Becker muscular dystrophy [BMD]). METHODS: In 2002, the Centers for Disease Control and Prevention established the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) to conduct longitudinal, population-based surveillance and research of DBMD in the United States. Six sites conducted active, multiple-source case finding and record abstraction to identify MD STARnet cases born January 1982 to December 2011. We used cross-sectional analyses to estimate prevalence of DBMD per 10 000 boys, ages 5 to 9 years, for 4 quinquennia (1991-1995, 1996-2000, 2001-2005, and 2006-2010) and prevalence per 10 000 male individuals, ages 5 to 24 years, in 2010. Prevalence was also estimated by race/ethnicity and phenotype. RESULTS: Overall, 649 cases resided in an MD STARnet site during ≥1 quinquennia. Prevalence estimates per 10 000 boys, ages 5 to 9 years, were 1.93, 2.05, 2.04, and 1.51, respectively, for 1991-1995, 1996-2000, 2001-2005, and 2006-2010. Prevalence tended to be higher for Hispanic individuals than non-Hispanic white or black individuals, and higher for DMD than BMD. In 2010, prevalence of DBMD was 1.38 per 10 000 male individuals, ages 5 to 24 years. CONCLUSIONS: We present population-based prevalence estimates for DBMD in 6 US sites. Prevalence differed by race/ethnicity, suggesting potential cultural and socioeconomic influences in the diagnosis of DBMD. Prevalence also was higher for DMD than BMD. Continued longitudinal surveillance will permit us to examine racial/ethnic and socioeconomic differences in treatment and outcomes for MD STARnet cases.

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