Advertisement

Long-term outcomes of steroid therapy for Duchenne muscular dystrophy in Japan

      Abstract

      Introduction

      Corticosteroids are effective for improving motor function in patients with Duchenne muscular dystrophy (DMD), but there is no consensus on a regimen that balances efficacy and side effects.

      Methods

      Data from three groups of DMD patients were retrospectively analyzed: those treated with 0.75 mg/kg/day prednisolone every day (daily group, n = 51); those treated with 1 mg/kg/day prednisolone on alternate days (intermittent group, n = 36), and those not treated with steroids (nontreatment group, n = 42).

      Results

      Although the age of ambulation loss did not differ significantly among the groups, the hazard ratios for ambulation loss relative to the nontreatment group were 0.24 (95% confidence interval [CI]: 0.11–0.54) in the daily group and 0.34 (95% CI: 0.19–0.62) in the intermittent group. The percentage of predicted forced vital capacity increased until 9.6 years of age (to 94.1%) in the daily group, until 8.8 years of age (to 96.9%) in the intermittent group, and until 7.2 years of age (to 87.6%) in the nontreatment group. Weight gain was the most frequently observed side effect in the treated groups. Height was significantly lower in the daily than in the nontreatment group. Other side effects were observed, but no patient discontinued therapy. There were no marked differences in benefits and side effects between the two treated groups.

      Discussion

      This is the first assessment of long-term outcomes of different steroid therapy regimens in Japanese DMD patients. Benefits and side effects, except height, did not differ significantly between steroid regimens.

      Keywords

      To read this article in full you will need to make a payment

      Purchase one-time access:

      Academic & Personal: 24 hour online accessCorporate R&D Professionals: 24 hour online access
      One-time access price info
      • For academic or personal research use, select 'Academic and Personal'
      • For corporate R&D use, select 'Corporate R&D Professionals'

      Subscribe:

      Subscribe to Brain and Development
      Already a print subscriber? Claim online access
      Already an online subscriber? Sign in
      Institutional Access: Sign in to ScienceDirect

      References

        • Hutter O.F.
        The membrane hypothesis of Duchenne muscular dystrophy: quest for functional evidence.
        J Inherit Metab Dis. 1992; 15: 565-577
        • McDonald C.M.
        • Abresch R.T.
        • Carter G.T.
        • Fowler Jr., W.M.
        • Johnson E.R.
        • Kilmer D.D.
        • et al.
        Profiles of neuromuscular diseases. Duchenne muscular dystrophy.
        Am J Phys Med Rehabil. 1995; 74: S70-S92
        • Drachman D.B.
        • Toyka K.V.
        • Myer E.
        Prednisone in Duchenne muscular dystrophy.
        Lancet. 1974; 2: 1409-1412
        • Manzur A.Y.
        • Kuntzer T.
        • Pike M.
        • Swan A.
        Glucocorticoid corticosteroids for Duchenne muscular dystrophy.
        Cochrane Database Syst Rev. 2008; 1CD003725
        • Rahman M.M.
        • Hannan M.A.
        • Mondol B.A.
        • Bhoumick N.B.
        • Haque A.
        Prednisolone in Duchenne muscular dystrophy.
        Bangladesh Med Res Counc Bull. 2001; 27: 38-42
        • Mendell J.R.
        • Moxley R.T.
        • Griggs R.C.
        • Brooke M.H.
        • Fenichel G.M.
        • Miller J.P.
        • et al.
        Randomized, double-blind six-month trial of prednisone in Duchenne’s muscular dystrophy.
        N Engl J Med. 1989; 320: 1592-1597
        • Fenichel G.M.
        • Mendell J.R.
        • Moxley 3rd, R.T.
        • Griggs R.C.
        • Brooke M.H.
        • Miller J.P.
        • et al.
        A comparison of daily and alternate-day prednisone therapy in the treatment of Duchenne muscular dystrophy.
        Arch Neurol. 1991; 48: 575-579
        • Pradhan S.
        • Ghosh D.
        • Srivastava N.K.
        • Kumar A.
        • Mittal B.
        • Pandey C.M.
        • et al.
        Prednisolone in Duchenne muscular dystrophy with imminent loss of ambulation.
        J Neurol. 2006; 253: 1309-1316
        • Connolly A.M.
        • Schierbecker J.
        • Renna R.
        • Florence J.
        High dose weekly oral prednisone improves strength in boys with Duchenne muscular dystrophy.
        Neuromuscul Disord. 2002; 12: 917-925
        • Escolar D.M.
        • Hache L.P.
        • Clemens P.R.
        • Cnaan A.
        • McDonald C.M.
        • Viswanathan V.
        • et al.
        Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy.
        Neurology. 2011; 77: 444-452
        • Biggar W.D.
        • Harris V.A.
        • Eliasoph L.
        • Alman B.
        Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade.
        Neuromuscul Disord. 2006; 16: 249-255
        • King W.M.
        • Ruttencutter R.
        • Nagaraja H.N.
        • Matkovic V.
        • Landoll J.
        • Hoyle C.
        • et al.
        Orthopedic outcomes of long-term daily corticosteroid treatment in Duchenne muscular dystrophy.
        Neurology. 2007; 68: 1607-1613
        • Ricotti V.
        • Ridout D.A.
        • Scott E.
        • Quinlivan R.
        • Robb S.A.
        • Manzur A.Y.
        • et al.
        Long-term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy.
        J Neurol Neurosurg Psychiatry. 2013; 84: 698-705
        • Moxley 3rd, R.T.
        • Ashwal S.
        • Pandya S.
        • Connolly A.
        • Florence J.
        • Mathews K.
        • et al.
        Practice parameter: corticosteroid treatment of Duchenne dystrophy: report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society.
        Neurology. 2005; 11: 13-20
        • Gloss D.
        • Moxley 3rd, R.T.
        • Ashwal S.
        • Oskoui M.
        Practice guideline update summary: corticosteroid treatment of Duchenne muscular dystrophy: report of the Guideline Development Subcommittee of the American Academy of Neurology.
        Neurology. 2016; 86: 465-472
        • Nishima S.
        Flow-volume curve in healthy and asthmatic children in the non-attack stage (in Japanese).
        Rinsho to Kenkyu. 1977; 54: 525-530
        • Miller M.R.
        • Hankinson J.
        • Brusasco V.
        • Burgos F.
        • Casaburi R.
        • Coates A.
        • et al.
        Standardisation of spirometry.
        Eur Respir J. 2005; 26: 319-338
        • Cnaan A.
        • Laird N.M.
        • Slasor P.
        Using the general linear mixed model to analyse unbalanced repeated measures and longitudinal data.
        Stat Med. 1997; 16: 2349-2380
        • Griggs R.C.
        • Herr B.E.
        • Reha A.
        • Elfring G.
        • Atkinson L.
        • Cwik V.
        • et al.
        Corticosteroids in Duchenne muscular dystrophy: major variations in practice.
        Muscle Nerve. 2013; 48: 27-31
        • Hoffman E.P.
        • Reeves E.
        • Damsker J.
        • Nagaraju K.
        • McCall J.M.
        • Connor E.M.
        • et al.
        Novel approaches to corticosteroid treatment in Duchenne muscular dystrophy.
        Phys Med Rehabil Clin N Am. 2012; 23: 821-828
        • Moxley 3rd, R.T.
        • Pandya S.
        • Ciafaloni E.
        • Fox D.J.
        • Campbell K.
        Change in natural history of Duchenne muscular dystrophy with long-term corticosteroid treatment: implications for management.
        J Child Neurol. 2010; 25: 1116-1129
        • Henricson E.K.
        • Abresch R.T.
        • Cnaan A.
        • Hu F.
        • Duong T.
        • Arrieta A.
        • et al.
        The cooperative international neuromuscular research group Duchenne natural history study: glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures.
        Muscle Nerve. 2013; 48: 55-67
        • Mayer O.H.
        • Finkel R.S.
        • Rummey C.
        • Benton M.J.
        • Glanzman A.M.
        • Flickinger J.
        • et al.
        Characterization of pulmonary function in Duchenne Muscular Dystrophy.
        Pediatr Pulmonol. 2015; 50: 487-494
        • Nève V.
        • Cuisset J.M.
        • Edmé J.L.
        • Carpentier A.
        • Howsam M.
        • Leclerc O.
        • et al.
        Sniff nasal inspiratory pressure in the longitudinal assessment of young Duchenne muscular dystrophy children.
        Eur Respir J. 2013; 42: 671-680
        • Bonifati M.D.
        • Ruzza G.
        • Bonometto P.
        • Berardinelli A.
        • Gorni K.
        • Orcesi S.
        • et al.
        A multicenter, double-blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy.
        Muscle Nerve. 2000; 23: 1344-1347
        • Backman E.
        • Henriksson K.G.
        Low-dose prednisolone treatment in Duchenne and Becker muscular dystrophy.
        Neuromuscul Disord. 1995; 5: 233-241
      1. Practical guideline for duchenne muscular dystrophy (DMD). Nankodo Co., Ltd, Tokyo2014 ([in Japanese])
        • Nakamura H.
        • Kimura E.
        • Mori-Yoshimura M.
        • Komaki H.
        • Matsuda Y.
        • Goto K.
        • et al.
        Characteristics of Japanese Duchenne and Becker muscular dystrophy patients in a novel Japanese national registry of muscular dystrophy (Remedy).
        Orphanet J Rare Dis. 2013; 8: 60
        • Takeuchi F.
        • Yonemoto N.
        • Nakamura H.
        • Shimizu R.
        • Komaki H.
        • Mori-Yoshimura M.
        • et al.
        Prednisolone improves walking in Japanese Duchenne muscular dystrophy patients.
        J Neurol. 2013; 260: 3023-3029