Age at onset of first signs or symptoms predicts age at loss of ambulation in Duchenne and Becker Muscular Dystrophy: Data from the MD STAR net

Emma Ciafaloni, Anil Kumar, Ke Liu, Shree Pandya, Christina Westfield, Deborah J. Fox, Kristin M Caspers Conway, Christopher M Cunniff, Katherine Mathews, Nancy West, Paul A. Romitti, Michael P. McDermott

Research output: Contribution to journalArticle

3 Citations (Scopus)

Abstract

PURPOSE: We investigated the prognostic utility of onset age at first signs and symptoms (SS) to predict onset age at loss of ambulation (LOA) for childhood-onset Duchenne and Becker Muscular Dystrophies (DBMD). METHODS: Our cohort comprised male cases with DBMD ascertained by the population-based Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet). Adjusted hazard ratios (HRs) and 95% confidence intervals (CIs) were estimated using Cox proportional hazards models for associations between onset ages of first SS and LOA. Covariates controlled for were corticosteroid use, family history of DBMD, birth year, race/ethnicity, and MD STARnet site. Onset age at first SS was considered as a continuous and as a categorical variable. RESULTS: A one-year increase in onset age at first SS was significantly associated with a 10% reduction in annual risk of LOA (HR = 0.90, CI = 0.870.94). Treating onset age at first SS as a categorical variable yielded a similar association (≥ 5 years: referent; ≥ 3 to <5 years: HR = 1.36, CI = 1.021.81; 18 months to <3 years: HR = 1.72, CI = 1.312.26; <18 months: HR = 1.52, CI = 1.142.02). CONCLUSIONS: Earlier onset age at first SS is associated with earlier onset age at LOA and may have clinical utility in differentiating childhood-onset Duchenne and Becker muscular dystrophies.

Original languageEnglish (US)
Pages (from-to)5-11
Number of pages7
JournalJournal of Pediatric Rehabilitation Medicine
Volume9
Issue number1
DOIs
StatePublished - Feb 27 2016
Externally publishedYes

Fingerprint

Duchenne Muscular Dystrophy
Age of Onset
Signs and Symptoms
Walking
Confidence Intervals
Muscular Dystrophies
Proportional Hazards Models
Research
Adrenal Cortex Hormones
Parturition

Keywords

  • ambulation
  • Becker Muscular Dystrophy
  • Duchenne Muscular Dystrophy

ASJC Scopus subject areas

  • Rehabilitation
  • Physical Therapy, Sports Therapy and Rehabilitation
  • Pediatrics, Perinatology, and Child Health

Cite this

Age at onset of first signs or symptoms predicts age at loss of ambulation in Duchenne and Becker Muscular Dystrophy : Data from the MD STAR net. / Ciafaloni, Emma; Kumar, Anil; Liu, Ke; Pandya, Shree; Westfield, Christina; Fox, Deborah J.; Conway, Kristin M Caspers; Cunniff, Christopher M; Mathews, Katherine; West, Nancy; Romitti, Paul A.; McDermott, Michael P.

In: Journal of Pediatric Rehabilitation Medicine, Vol. 9, No. 1, 27.02.2016, p. 5-11.

Research output: Contribution to journalArticle

Ciafaloni, E, Kumar, A, Liu, K, Pandya, S, Westfield, C, Fox, DJ, Conway, KMC, Cunniff, CM, Mathews, K, West, N, Romitti, PA & McDermott, MP 2016, 'Age at onset of first signs or symptoms predicts age at loss of ambulation in Duchenne and Becker Muscular Dystrophy: Data from the MD STAR net', Journal of Pediatric Rehabilitation Medicine, vol. 9, no. 1, pp. 5-11. https://doi.org/10.3233/PRM-160361
Ciafaloni, Emma ; Kumar, Anil ; Liu, Ke ; Pandya, Shree ; Westfield, Christina ; Fox, Deborah J. ; Conway, Kristin M Caspers ; Cunniff, Christopher M ; Mathews, Katherine ; West, Nancy ; Romitti, Paul A. ; McDermott, Michael P. / Age at onset of first signs or symptoms predicts age at loss of ambulation in Duchenne and Becker Muscular Dystrophy : Data from the MD STAR net. In: Journal of Pediatric Rehabilitation Medicine. 2016 ; Vol. 9, No. 1. pp. 5-11.
@article{0a0512eecb244df287b0c3a7b916fe0b,
title = "Age at onset of first signs or symptoms predicts age at loss of ambulation in Duchenne and Becker Muscular Dystrophy: Data from the MD STAR net",
abstract = "PURPOSE: We investigated the prognostic utility of onset age at first signs and symptoms (SS) to predict onset age at loss of ambulation (LOA) for childhood-onset Duchenne and Becker Muscular Dystrophies (DBMD). METHODS: Our cohort comprised male cases with DBMD ascertained by the population-based Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet). Adjusted hazard ratios (HRs) and 95{\%} confidence intervals (CIs) were estimated using Cox proportional hazards models for associations between onset ages of first SS and LOA. Covariates controlled for were corticosteroid use, family history of DBMD, birth year, race/ethnicity, and MD STARnet site. Onset age at first SS was considered as a continuous and as a categorical variable. RESULTS: A one-year increase in onset age at first SS was significantly associated with a 10{\%} reduction in annual risk of LOA (HR = 0.90, CI = 0.870.94). Treating onset age at first SS as a categorical variable yielded a similar association (≥ 5 years: referent; ≥ 3 to <5 years: HR = 1.36, CI = 1.021.81; 18 months to <3 years: HR = 1.72, CI = 1.312.26; <18 months: HR = 1.52, CI = 1.142.02). CONCLUSIONS: Earlier onset age at first SS is associated with earlier onset age at LOA and may have clinical utility in differentiating childhood-onset Duchenne and Becker muscular dystrophies.",
keywords = "ambulation, Becker Muscular Dystrophy, Duchenne Muscular Dystrophy",
author = "Emma Ciafaloni and Anil Kumar and Ke Liu and Shree Pandya and Christina Westfield and Fox, {Deborah J.} and Conway, {Kristin M Caspers} and Cunniff, {Christopher M} and Katherine Mathews and Nancy West and Romitti, {Paul A.} and McDermott, {Michael P.}",
year = "2016",
month = "2",
day = "27",
doi = "10.3233/PRM-160361",
language = "English (US)",
volume = "9",
pages = "5--11",
journal = "Journal of Pediatric Rehabilitation Medicine",
issn = "1874-5393",
publisher = "IOS Press",
number = "1",

}

TY - JOUR

T1 - Age at onset of first signs or symptoms predicts age at loss of ambulation in Duchenne and Becker Muscular Dystrophy

T2 - Data from the MD STAR net

AU - Ciafaloni, Emma

AU - Kumar, Anil

AU - Liu, Ke

AU - Pandya, Shree

AU - Westfield, Christina

AU - Fox, Deborah J.

AU - Conway, Kristin M Caspers

AU - Cunniff, Christopher M

AU - Mathews, Katherine

AU - West, Nancy

AU - Romitti, Paul A.

AU - McDermott, Michael P.

PY - 2016/2/27

Y1 - 2016/2/27

N2 - PURPOSE: We investigated the prognostic utility of onset age at first signs and symptoms (SS) to predict onset age at loss of ambulation (LOA) for childhood-onset Duchenne and Becker Muscular Dystrophies (DBMD). METHODS: Our cohort comprised male cases with DBMD ascertained by the population-based Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet). Adjusted hazard ratios (HRs) and 95% confidence intervals (CIs) were estimated using Cox proportional hazards models for associations between onset ages of first SS and LOA. Covariates controlled for were corticosteroid use, family history of DBMD, birth year, race/ethnicity, and MD STARnet site. Onset age at first SS was considered as a continuous and as a categorical variable. RESULTS: A one-year increase in onset age at first SS was significantly associated with a 10% reduction in annual risk of LOA (HR = 0.90, CI = 0.870.94). Treating onset age at first SS as a categorical variable yielded a similar association (≥ 5 years: referent; ≥ 3 to <5 years: HR = 1.36, CI = 1.021.81; 18 months to <3 years: HR = 1.72, CI = 1.312.26; <18 months: HR = 1.52, CI = 1.142.02). CONCLUSIONS: Earlier onset age at first SS is associated with earlier onset age at LOA and may have clinical utility in differentiating childhood-onset Duchenne and Becker muscular dystrophies.

AB - PURPOSE: We investigated the prognostic utility of onset age at first signs and symptoms (SS) to predict onset age at loss of ambulation (LOA) for childhood-onset Duchenne and Becker Muscular Dystrophies (DBMD). METHODS: Our cohort comprised male cases with DBMD ascertained by the population-based Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet). Adjusted hazard ratios (HRs) and 95% confidence intervals (CIs) were estimated using Cox proportional hazards models for associations between onset ages of first SS and LOA. Covariates controlled for were corticosteroid use, family history of DBMD, birth year, race/ethnicity, and MD STARnet site. Onset age at first SS was considered as a continuous and as a categorical variable. RESULTS: A one-year increase in onset age at first SS was significantly associated with a 10% reduction in annual risk of LOA (HR = 0.90, CI = 0.870.94). Treating onset age at first SS as a categorical variable yielded a similar association (≥ 5 years: referent; ≥ 3 to <5 years: HR = 1.36, CI = 1.021.81; 18 months to <3 years: HR = 1.72, CI = 1.312.26; <18 months: HR = 1.52, CI = 1.142.02). CONCLUSIONS: Earlier onset age at first SS is associated with earlier onset age at LOA and may have clinical utility in differentiating childhood-onset Duchenne and Becker muscular dystrophies.

KW - ambulation

KW - Becker Muscular Dystrophy

KW - Duchenne Muscular Dystrophy

UR - http://www.scopus.com/inward/record.url?scp=84959895423&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84959895423&partnerID=8YFLogxK

U2 - 10.3233/PRM-160361

DO - 10.3233/PRM-160361

M3 - Article

C2 - 26966795

AN - SCOPUS:84959895423

VL - 9

SP - 5

EP - 11

JO - Journal of Pediatric Rehabilitation Medicine

JF - Journal of Pediatric Rehabilitation Medicine

SN - 1874-5393

IS - 1

ER -