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Prednisolone in Duchenne muscular dystrophy with imminent loss of ambulation

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Abstract

An open controlled trial of 0.75 mg/Kg/day prednisolone was conducted at a stage when the patients had started falling several times in a day and stopped on their attaining a chair bound stage, thus minimising the total period of steroid therapy. Out of the 67 DMD patients enrolled in this study, 44 were put on prednisolone therapy and 23 served as controls. All patients were followed-up at two-monthly intervals for two years and thereafter they continued to take their respective medications till their chair-bound stage; then the drug was gradually withdrawn. In the treatment group 24 patients could not continue the trial because of adverse effects – 14 due to excessive obesity, 3 due to measles, 4 due to pulmonary tuberculosis, 2 due to recurrent throat and chest infection and 1 due to an unexplained high leukocyte count. Of the remaining 20 patients in the treatment group, steroid therapy was stopped in 5 patients as there was no improvement in power in six months. Fifteen patients in the treatment group and 19 patients in the control group could be followed regularly for 2 years and then up to chair-bound stage. Outcome parameters included fall frequency, peak expiratory flow rate, limb muscle power, ability to lift weights, time taken in getting up from squatting position, walking 9 metres and climbing 13 stairs. Maximum improvement was noted between 2 and 4 months while mild improvement in some parameters continued up to six months. All parameters remained stabilised for 1 year or so, after which there was slight deterioration. Deterioration at 2 years was, however, less than the natural course of events noted in control patients. Prednisolone treated patients and controls became chair bound at the mean age of 169 ± 9 and 132 ± 8 months respectively. Till the ideal stage of the disease and the type or dosage of starting steroid therapy is defined by specially designed studies, 0.75 mg/Kg/day prednisolone therapy may be started in DMD patients at the stage of frequent falls ( > 10 / day) on walking or increased get-up time ( > 10 s) as observed while testing Gowers’ sign; this improves muscle power and timing of motor performance within 2–4 months of onset of therapy in about 75% of those who tolerate this therapy, with a possible gain of approximately 3 years in terms of independent walking.

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Acknowledgements

This work was supported by the intramural research grant of Sanjay Gandhi Postgraduate Institute of Medical Sciences, Lucknow, India. The help of Drs. Sneh Shashank and Rameshwar Prasad Gupta in data collection is gratefully acknowledged.

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Authors and Affiliations

  1. Department of Neurology, Sanjay Gandhi Postgraduate Institute of Medical Sciences, Raebareli Road, Lucknow, 226014, India

    Sunil Pradhan, Debabrata Ghosh, Niraj Kumar Srivastava & Ashok Kumar

  2. Dept. of Genetics, Sanjay Gandhi Postgraduate Institute of Medical Sciences, Raebareli Road, Lucknow , 226014, India

    Balraj Mittal

  3. Dept. of Bio-statistics, Sanjay Gandhi Postgraduate Institute of Medical Sciences, Raebareli Road, Lucknow, 226014, India

    Chandra Mani Pandey & Uttam Singh

Authors
  1. Sunil Pradhan
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  2. Debabrata Ghosh
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  3. Niraj Kumar Srivastava
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  4. Ashok Kumar
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  5. Balraj Mittal
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  6. Chandra Mani Pandey
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  7. Uttam Singh
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Correspondence to Sunil Pradhan.

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Received in revised form: 23 February 2006

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Pradhan, S., Ghosh, D., Srivastava, N.K. et al. Prednisolone in Duchenne muscular dystrophy with imminent loss of ambulation. J Neurol 253, 1309–1316 (2006). https://doi.org/10.1007/s00415-006-0212-1

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  • DOI: https://doi.org/10.1007/s00415-006-0212-1

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