| Objective To observe and evaluate the efficiency and side effects of low-dose glucocorticoids therapy in children with Duchenne muscular dystrophy and to accumulate experience in the treatment and management.Methods 1.We collected clinical data of male children who were diagnosed with Duchenne muscular dystrophy by genetic testing.2.All patients were treated with glucocorticoid.The specific dose of prednisone was as follows: 0.75 mg/kg daily in ambulatory patients and 0.3-0.6 mg/kg daily in stretcher patients.All patients took vitamin D,calcium citrate and 10% potassium chloride solution at the same time.We observed these patients in 4 observational points for 6 months.4 observational points set at the beginning of treatment and the 1st,3rd and 6th month during this treatment.3.The main content of observation were muscle strength scores,the time of running 10 meters,the time of climbing4 stairs,the time of rising from supine to a standing position(Gowers’ time),the change of creatine kinase(CK)and the situation of growing development.4.SPSS20.0 software was applied for data analysis.Counting data was used for cases and percentages.Measurement data was used for average± standard.T test was used for the comparison between means.The difference was statistically significant with P value<0.05.Results 1.50 cases were collected in our study.4 cases were treated for 6 months,but they did not follow up as required.Another 24 cases were treated for less than 6 months,which was mainly related to worry about side effects and economic factors.Finally,22 cases of which finished 6 months of treatment.The age of onset was 4-12 years old.The average age of onset was 7.52+2.52 years old.72.7% of them were younger within 10 years old.22 cases were divided into three groups: the first group was 4-6 years old(9 cases),the second group was 7-9 years old(7 cases)and the third group was 10-12 years old(6 cases).2.We record these muscle strength,10-meter running time,four-stair climbing time and Gowers’ timeat4 observation points of each group.2.1 The average muscle strength score of the first group were 25,25,25 and 26.The second group were 23,23,24 and 23.The third group were 24,24,25 and 24.There was no significant difference in the change of muscle strength score at the three observational points during the treatment compared with the score at the at the beginning of treatment in three groups(P>0.05).After six months’ treatment,muscle strength of three groups maintained at the beginning level.2.2 The mean time of 10-meter running in the first group were 7.6,7.1,6.2,and 6.3 second.The second group was 8.6,7.3,7.4,and 6.9.The third group were 8.3,7.0,6.6,and 7.3.There was no significant difference in the change of 10-meter running time at the three observational points after treatment compared with the time at the beginning of treatment in three groups(P >0.05).After six months’ treatment,the 10-meter running time of three groups maintained at the beginning level.2.3 The mean time of climbing 4 stairs in the first group were 5.0,3.7,3.2,and 2.6 seconds.The second group were 4.0,4.6,3.9,and 4.4,The third group were 4.2,4.5,3.8,and 4.3.There was significant difference in the change of climbing 4 stairs time at the 3rd and 6th month after treatment after treatment compared with the time at the beginning of treatment in the first group(P<0.05),while those of the second group and the third group were no significant difference(P>0.05).After six months’ treatment,the four-stair climbing time of the first group significantly decreased,while the second and the third still maintained at the beginning level.2.4 The mean time of Gower in the first group were 6.7,5.2,4.0,and 4.4 second.The second group were 11.0,6.4,6.5,and 9.2.The third group 8.8,8.8,7.0,and 7.5.There was no significant difference in the change of Gowers’ time at the 1st month after treatment compared with the time at the beginning of treatment in the first group(P>0.05),while there was significant difference in the change of Gowers’ time at the 3rd and 6th month after treatment compared with the time at the beginning of treatment in the first group(P<0.05).Those of the second group and the third group had no significant difference(P>0.05).After six months’ treatment,the Gowers’ time of the first group significantly decreased,while the second and the third still maintained at the beginning level.2.5 The mean CK values of 22 patients at 4 observation points were(13612±9680),(9600±5692),(9077±5692),and(6848±2695)U/L,respectively.It decreased significantly before and after treatment(P< 0.05).The first group significantly decreased 3rd month after treatment(P < 0.05),while the second and the third groups had no significant difference before and after treatment(P >0.05).3.There was significant difference in the change of weight and height at the sixth month after treatment compared with the beginning of treatment.(P<0.05).Compared with the normal weight gain of children in half a year,the abnormal weight gain of three groups was 44%,57% and 67% respectively.Six cases obtain pelvic osteoporosis(27.2%).The adverse reactions were reduction or maintenance when the dosage of prednisone was adjusted.In three group,there were 5 cases of mild facial fullness(22.7%),14 cases of leukocytosis(63.6%),5 cases of excessive hair(22.7%),1 case of hypertension(4.5%),and 1 case of abnormal mental behavior(4.5%).There were no serious symptoms which had an affect on daily life and didn’t need treatment.Conclusions 1.Children with Duchenne muscular dystrophy treated with oral prednisone,CK value of which was significantly lower than that before treatment.It maybe related to the effect of Glucocorticoid that stabilized the myocyte membrane,reduced the release of CK into the blood and reduced the serum CK.2.Muscle strength and motor function of children with Duchenne muscular dystrophy could be improved or kept by taken prednisone(0.75mg/kg/d).Younger patients had obvious curative effect and fewer side effects compared with than older patients.DMD patients should be treated with-as early as possibie.3.Oral low dose prednisone had significant effect on the growth of weigth but had no effect on the growth of heigth in the short term.The effect of long-term treatment need further study.4.The dosage should be adjusted appropriately when patients with oral prednisone appeared significant weight gain and osteoporosis.The related side effects could be observed if it had no effect on daily life.5.Before treatment,doctors had better communicate with patients about the goal of treatment,monitoring and handling of side effects,approximate cost and so on,which were helpful to improve patient compliance. |
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