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. 2022 Jun;32(6):468-476.
doi: 10.1016/j.nmd.2022.04.008. Epub 2022 Apr 30.

Selected clinical and demographic factors and all-cause mortality among individuals with Duchenne muscular dystrophy in the Muscular Dystrophy Surveillance, Tracking, and Research Network

Pangaja Paramsothy  1 , Yinding Wang  2 , Bo Cai  3 , Kristin M Conway  4 , Nicholas E Johnson  5 , Shree Pandya  6 , Emma Ciafaloni  6 , Katherine D Mathews  7 , Paul A Romitti  4 , James F Howard Jr  8 , Catharine Riley  9
Affiliations

Selected clinical and demographic factors and all-cause mortality among individuals with Duchenne muscular dystrophy in the Muscular Dystrophy Surveillance, Tracking, and Research Network

Pangaja Paramsothy et al. Neuromuscul Disord. 2022 Jun.

Abstract

Population-based estimates of survival among individuals with Duchenne muscular dystrophy (DMD) living in the United States are lacking. It is also unclear whether the association between glucocorticoid use and all-cause mortality persists in the context of other common treatments (cardiac medication, cough-assist, bilevel positive airway pressure, and scoliosis surgery) observed to delay mortality. Among 526 individuals identified by the Muscular Dystrophy Surveillance, Tracking, and Research Network, the estimated median survival time from birth was 23.7 years. Current glucocorticoid users had a lower hazard of mortality than non-users. Individuals who ever had scoliosis surgery had a lower hazard of mortality than individuals who did not have scoliosis surgery. Individuals who ever used cough assist had a lower hazard of mortality than individuals who never used cough assist. Non-Hispanic Black individuals had a higher hazard of mortality than non-Hispanic White individuals. No differences in hazards of mortality were observed between ever versus never use of cardiac medication and ever versus never use of bilevel positive airway pressure. The glucocorticoid observation is consistent with the 2018 Care Considerations statement that glucocorticoid use continues in the non-ambulatory phase. Our observations may inform the clinical care of individuals living with DMD.

Keywords: Duchenne Muscular Dystrophy; Mortality; Survival.

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Conflict of interest statement

Declaration of Competing Interest Pangaja Paramsothy, Yinding Wang, Bo Cai, Kristin Conway, Shree Pandya, and Catharine Riley report none. Nicholas Johnson has received grant funding from NINDS (R01NS104010), NCATS (R21TR003184), CDC (U01DD001242) and the FDA (7R01FD006071-02). He receives royalties from the CCMDHI and the CMTHI. He receives research funds from Dyne, AveXis, CSL Behring, Vertex Pharmaceuticals, Fulcrum Therapeutics, ML Bio, Sarepta, and Acceleron Pharma. He has provided consultation for AveXis, AMO Pharma, Strongbridge BioPharma, Acceleron Pharma, Fulcrum Therapeutics, Dyne, Avidity, Arthex, and Vertex Pharmaceuticals. He receives licensing fees from the University of Rochester for the CCMDHI and CMTHI. He has received stock options from ML Bio. Emma Ciafaloni has received personal compensation for serving on advisory boards and/or as a consultant for Viela Bio, Avexis, Inc, Biogen, Medscape, Pfizer, PTC Therapeutics, Sarepta Therapeutics, Ra pharma, Wave, and Strongbridge Biopharma plc. She has received personal compensation for serving on a speaker's bureau for Biogen. She has received research and/or grant support from the Centers for Disease Control and Prevention, CureSMA, Muscular Dystrophy Association, National Institutes of Health, Orphazyme, the Patient-Centered Outcomes Research Institute, Parent Project Muscular Dystrophy, PTC Therapeutics, Santhera, Sarepta Therapeutics, Orphazyme, and the US Food and Drug Administration. She has received royalties from Oxford University Press and compensation from Medlink for editorial duties. Katherine Mathews receives research funding from NIH U54 NS053672 and U24 NS-107181, the Friedreich's Ataxia Research Alliance, the Centers for Disease Control and Prevention (U01 DD001248) and serves as a site PI for clinical research sponsored by PTC Therapeutics Inc, Sarepta Therapeutics Inc, Pfizer Inc, FibroGen Inc, AMO, BMS, Reata, Retrotope, and Italfarmaco. She is an advisory board member for Sarepta Therapeutics Inc and Dyne Therapeutics. Paul Romitti has received support from PTC Therapeutics Inc. for work unrelated to the current study James Howard Jr. has received research support from Alexion Pharmaceuticals, argenx BVBA, the Myasthenia Gravis Foundation of America, the Muscular Dystrophy Association, the National Institutes of Health (including the National Institute of Neurological Disorders and Stroke and the National Institute of Arthritis and Musculoskeletal and Skin Diseases), PCORI, Ra Pharmaceuticals (now UCB) and Takeda Pharmaceuticals; Honoraria from Alexion Pharmaceuticals, argenx BVBA, Immunovant Inc., Ra Pharmaceuticals (now UCB), Regeneron Pharmaceuticals, Sanofi US and Viela Bio Inc. and non-financial support from Alexion Pharmaceuticals, argenx BVBA, Ra Pharmaceuticals (now UCB) and Toleranzia AB. He receives research support from Research Triangle Institute (the Centers for Disease Control and Prevention (Atlanta, GA, USA)) in support of this study

Figures

Figure 1.
Figure 1.
Inclusion and Exclusion Criteria, Muscular Dystrophy Surveillance, Tracking, and Research Network,1982-2011
Figure 2.
Figure 2.
Kaplan- Meier Survival Curve from Birth, Muscular Dystrophy Surveillance, Tracking, and Research Network, 1982-2011. Red line is the overall estimated survival probability from birth with red plus signs representing censored individuals and blue shade region the 95% confidence interval.
Figure 3.
Figure 3.
Kaplan-Meier Survival Curve from Loss of Ambulation, Muscular Dystrophy Surveillance, Tracking, and Research Network, 1982-2011. Red line is the overall estimated survival probability from loss of ambulation with red plus signs representing censored individuals and blue shade region the 95% confidence interval.
See this image and copyright information in PMC

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