Individualized prediction of changes in 6-minute walk distance for patients with Duchenne muscular dystrophy

PLoS ONE

Volumn 11, Issue 10, 2016, Pages

  Goemans, Nathalie ^a   Vanden Hauwe, Marleen ^a   Signorovitch, James ^b,c   Swallow, Elyse ^b   Song, Jinlin ^b   Ward, Susan J ^d   Gregg, Keith ^e   Champion, Giles ^e   Binks, Michael ^f   Luo, Xiaohui ^g   Ong, Tuyen ^g   Spiegel, Robert ^g   Kaye, Ed ^h   Guey, Lin ⁱ   Josiah, Serene ⁱ   Siffel, Csaba ⁱ   Charnas, Lawrence ^j  

^a UNIVERSITY HOSPITALS LEUVEN   (Belgium)

^b ANALYSIS GROUP   (United States)

^c Trajectory Analysis Project (TAP) Collaboration   (United States)

^d cTAP   (United States)

^e BIOMARIN PHARMACEUTICAL INC   (United States)

^f PFIZER INC   (United States)

^g PTC THERAPEUTICS   (United States)

^h AVI BIOPHARMA INC   (United States)

ⁱ SHIRE   (United States)

^j Parent Project Muscular Dystrophy   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

DEFLAZACORT; PREDNISONE; STEROID;

ADOLESCENT; AGE; ARTICLE; BODY HEIGHT; BODY WEIGHT; CHILD; CONTROLLED STUDY; DRUG SUBSTITUTION; DRUG USE; DRUG WITHDRAWAL; DUCHENNE MUSCULAR DYSTROPHY; FEMALE; FOLLOW UP; HUMAN; MAJOR CLINICAL STUDY; MALE; MOBILIZATION; MULTIVARIATE ANALYSIS; PERSONALIZED MEDICINE; PHYSICAL ACTIVITY; PREDICTION; PREDICTIVE VALUE; PRESCHOOL CHILD; PROGNOSIS; REGRESSION ANALYSIS; SCHOOL CHILD; SIX MINUTE WALK TEST; STATISTICAL MODEL; SUPINE POSITION; TREATMENT DURATION; VALIDATION PROCESS; EXERCISE TEST; GENOTYPE; MUSCULAR DYSTROPHY, DUCHENNE; PATHOPHYSIOLOGY; THEORETICAL MODEL;

ADOLESCENT; AGE FACTORS; CHILD; CHILD, PRESCHOOL; EXERCISE TEST; FOLLOW-UP STUDIES; GENOTYPE; HUMANS; MALE; MODELS, THEORETICAL; MUSCULAR DYSTROPHY, DUCHENNE; PROGNOSIS; STEROIDS;

EID: 84991327511     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0164684     Document Type: Article

References (40)

1. Chung J, Smith AL, Hughes SC, Niizawa G, Abdel-Hamid HZ, Naylor EW, et al. Twenty-year follow-up of newborn screening for patients with muscular dystrophy. Muscle Nerve. 2015.
2. Emery AEH, Muntoni F, Quinlivan R. Duchenne muscular dystrophy. 4th ed. Oxford: Oxford University Press; 2015.
3. Ishikawa Y, Miura T, Ishikawa Y, Aoyagi T, Ogata H, Hamada S, et al. Duchenne muscular dystrophy: survival by cardio-respiratory interventions. Neuromuscul Disord. 2011; 21(1):47-51. doi: 10.1016/j.nmd.2010.09.006 PMID: 21144751
4. LoMauro A, D'Angelo MG, Aliverti A. Assessment and management of respiratory function in patients with Duchenne muscular dystrophy: current and emerging options. Ther Clin Risk Manag. 2015; 11:1475-88. doi: 10.2147/TCRM.S55889 PMID: 26451113
5. Bushby K, Finkel R, Wong B, Barohn R, Campbell C, Comi GP, et al. Ataluren treatment of patients with nonsense mutation dystrophinopathy. Muscle Nerve. 2014; 50(4):477-87. doi: 10.1002/mus.24332 PMID: 25042182
6. Hoffman EP, Connor EM. Orphan drug development in muscular dystrophy: update on two large clinical trials of dystrophin rescue therapies. Discov Med. 2013; 16(89):233-9. PMID: 24229740
7. Voit T, Topaloglu H, Straub V, Muntoni F, Deconinck N, Campion G, et al. Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study. Lancet Neurol. 2014; 13(10):987-96. doi: 10.1016/S1474-4422(14)70195-4 PMID: 25209738
8. Mendell JR, Goemans N, Lowes LP, Alfano LN, Berry K, Shao J, et al. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy. Ann Neurol. 2016; 79(2):257-71. doi: 10.1002/ana.24555 PMID: 26573217
9. Pane M, Mazzone ES, Sivo S, Sormani MP, Messina S, A DA, et al. Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes. PLoS One. 2014; 9(10):e108205. doi: 10.1371/journal.pone.0108205 PMID: 25271887
10. McDonald CM, Henricson EK, Abresch RT, Florence J, Eagle M, Gappmaier E, et al. The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study. Muscle Nerve. 2013; 48(3):343-56. doi: 10.1002/mus.23902 PMID: 23681930
11. Merlini L, Gennari M, Malaspina E, Cecconi I, Armaroli A, Gnudi S, et al. Early corticosteroid treatment in 4 Duchenne muscular dystrophy patients: 14-year follow-up. Muscle Nerve. 2012; 45(6):796-802. doi: 10.1002/mus.23272 PMID: 22581531
12. Bushby K, Connor E. Clinical outcome measures for trials in Duchenne muscular dystrophy: report from International Working Group meetings. Clin Investig (Lond). 2011; 1(9):1217-35.
13. Ricotti V, Ridout DA, Pane M, Main M, Mayhew A, Mercuri E, et al. The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials. J Neurol Neurosurg Psychiatry. 2016; 87(2):149-55. doi: 10.1136/jnnp-2014-309405 PMID: 25733532
14. Bakker JP, De Groot IJ, Beelen A, Lankhorst GJ. Predictive factors of cessation of ambulation in patients with Duchenne muscular dystrophy. Am J Phys Med Rehabil. 2002; 81(12):906-12. doi: 10.1097/01.PHM.0000034954.48339.F5 PMID: 12447089
15. Shklyar I, Pasternak A, Kapur K, Darras BT, Rutkove SB. Composite biomarkers for assessing Duchenne muscular dystrophy: an initial assessment. Pediatr Neurol. 2015; 52(2):202-5. doi: 10.1016/j.pediatrneurol.2014.09.014 PMID: 25447928
16. Ohmann C. Prognostic scores and design of clinical studies. Infection. 1998; 26(5):342-4. PMID: 9795803
17. Venur VA, Ahluwalia MS. Prognostic scores for brain metastasis patients: use in clinical practice and trial design. Chin Clin Oncol. 2015; 4(2):18. doi: 10.3978/j.issn.2304-3865.2015.06.01 PMID: 26112804
18. Motzer RJ, Escudier B, Oudard S, Hutson TE, Porta C, Bracarda S, et al. Phase 3 trial of everolimus for metastatic renal cell carcinoma: final results and analysis of prognostic factors. Cancer. 2010; 116(18):4256-65. doi: 10.1002/cncr.25219 PMID: 20549832
19. Vickers AJ. How to randomize. J Soc Integr Oncol. 2006; 4(4):194-8. PMID: 17022927
20. Kernan WN, Viscoli CM, Makuch RW, Brass LM, Horwitz RI. Stratified randomization for clinical trials. J Clin Epidemiol. 1999; 52(1):19-26. PMID: 9973070
21. Simon R, Blumenthal GM, Rothenberg ML, Sommer J, Roberts SA, Armstrong DK, et al. The role of nonrandomized trials in the evaluation of oncology drugs. Clin Pharmacol Ther. 2015; 97(5):502-7. doi: 10.1002/cpt.86 PMID: 25676488
22. Kahan BC, Jairath V, Dore CJ, Morris TP. The risks and rewards of covariate adjustment in randomized trials: an assessment of 12 outcomes from 8 studies. Trials. 2014; 15:139. doi: 10.1186/1745-6215-15-139 PMID: 24755011
23. Czaplinski A, Haverkamp LJ, Yen AA, Simpson EP, Lai EC, Appel SH. The value of database controls in pilot or futility studies in ALS. Neurology. 2006; 67(10):1827-32. doi: 10.1212/01.wnl.0000244415.48221.81 PMID: 17130417
24. Goemans N, van den Hauwe M, Wilson R, van Impe A, Klingels K, Buyse G. Ambulatory capacity and disease progression as measured by the 6-minute-walk-distance in Duchenne muscular dystrophy subjects on daily corticosteroids. Neuromuscul Disord. 2013; 23(8):618-23. doi: 10.1016/j.nmd.2013.05.006 PMID: 23770101
25. McDonald CM, Henricson EK, Han JJ, Abresch RT, Nicorici A, Elfring GL, et al. The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy. Muscle Nerve. 2010; 41(4):500-10. doi: 10.1002/mus.21544 PMID: 19941337
26. McDonald CM, Henricson EK, Han JJ, Abresch RT, Nicorici A, Atkinson L, et al. The 6-minute walk test in Duchenne/Becker muscular dystrophy: longitudinal observations. Muscle Nerve. 2010; 42(6):966-74. doi: 10.1002/mus.21808 PMID: 21038378
27. Abramson NS, Kelsey SF, Safar P, Sutton-Tyrrell K. Simpson's paradox and clinical trials: what you find is not necessarily what you prove. Ann Emerg Med. 1992; 21(12):1480-2. PMID: 1443848
28. Moxley RT 3rd, Pandya S, Ciafaloni E, Fox DJ, Campbell K. Change in natural history of Duchenne muscular dystrophy with long-term corticosteroid treatment: implications for management. J Child Neurol. 2010; 25(9):1116-29. doi: 10.1177/0883073810371004 PMID: 20581335
29. Manzur AY, Kuntzer T, Pike M, Swan A. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev. 2008(1: ):CD003725. doi: 10.1002/14651858.CD003725.pub3 PMID: 18254031
30. Biggar WD, Harris VA, Eliasoph L, Alman B. Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade. Neuromuscul Disord. 2006; 16(4):249-55. doi: 10.1016/j.nmd.2006.01.010 PMID: 16545568
31. Dec GW. Steroid therapy effectively delays Duchenne's cardiomyopathy. J Am Coll Cardiol. 2013; 61(9):955-6. doi: 10.1016/j.jacc.2012.12.011 PMID: 23352780
32. Wilson PW, D'Agostino RB, Levy D, Belanger AM, Silbershatz H, Kannel WB. Prediction of coronary heart disease using risk factor categories. Circulation. 1998; 97(18):1837-47. PMID: 9603539
33. Wade DT. Measurement in neurological rehabilitation. Curr Opin Neurol Neurosurg. 1992; 5(5):682-6. PMID: 1392142
34. van den Bergen JC, Hiller M, Bohringer S, Vijfhuizen L, Ginjaar HB, Chaouch A, et al. Validation of genetic modifiers for Duchenne muscular dystrophy: a multicentre study assessing SPP1 and LTBP4 variants. J Neurol Neurosurg Psychiatry. 2015; 86(10):1060-5. doi: 10.1136/jnnp-2014-308409 PMID: 25476005
35. Bello L, Kesari A, Gordish-Dressman H, Cnaan A, Morgenroth LP, Punetha J, et al. Genetic modifiers of ambulation in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study. Ann Neurol. 2015; 77(4):684-96. doi: 10.1002/ana.24370 PMID: 25641372
36. Finanger EL, Russman B, Forbes SC, Rooney WD, Walter GA, Vandenborne K. Use of skeletal muscle MRI in diagnosis and monitoring disease progression in Duchenne muscular dystrophy. Phys Med Rehabil Clin N Am. 2012; 23(1):1-10, ix. doi: 10.1016/j.pmr.2011.11.004 PMID: 22239869
37. Mazzone E, Martinelli D, Berardinelli A, Messina S, D'Amico A, Vasco G, et al. North Star Ambulatory Assessment, 6-minute walk test and timed items in ambulant boys with Duchenne muscular dystrophy. Neuromuscul Disord. 2010; 20(11):712-6. doi: 10.1016/j.nmd.2010.06.014 PMID: 20634072
38. Humbertclaude V, Hamroun D, Bezzou K, Berard C, Boespflug-Tanguy O, Bommelaer C, et al. Motor and respiratory heterogeneity in Duchenne patients: implication for clinical trials. Eur J Paediatr Neurol. 2012; 16(2):149-60. doi: 10.1016/j.ejpn.2011.07.001 PMID: 21920787
39. Smith RA, Newcombe RG, Sibert JR, Harper PS. Assessment of locomotor function in young boys with Duchenne muscular dystrophy. Muscle Nerve. 1991; 14(5):462-9. doi: 10.1002/mus.880140513 PMID: 1870637
40. Henricson E, Abresch R, Han JJ, Nicorici A, Goude Keller E, Elfring G, et al. Percent-predicted 6-minute walk distance in duchenne muscular dystrophy to account for maturational influences. PLoS Curr. 2012; 4:RRN1297. doi: 10.1371/currents.RRN1297 PMID: 22306689