التفاصيل البيبلوغرافية
| العنوان: |
Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial |
| المؤلفون: |
Guglieri, Michela, Bushby, Kate, McDermott, Michael P., Hart, Kimberly A., Tawil, Rabi, Martens, William B., Herr, Barbara E., McColl, Elaine, Speed, Chris, Wilkinson, Jennifer, Kirschner, Janbernd, King, Wendy M., Eagle, Michelle, Brown, Mary W., Willis, Tracey, Griggs, Robert C., Straub, Volker, van Ruiten, Henriette, Childs, Anne-Marie, Ciafaloni, Emma, Shieh, Perry B., Spinty, Stefan, Maggi, Lorenzo, Baranello, Giovanni, Butterfield, Russell J., Horrocks, I. A., Roper, Helen, Alhaswani, Zoya, Flanigan, Kevin M., Kuntz, Nancy L., Manzur, Adnan, Darras, Basil T., Kang, Peter B., Morrison, Leslie, Krzesniak-Swinarska, Monika, Mah, Jean K., Mongini, Tiziana E., Ricci, Federica, von der Hagen, Maja, Finkel, Richard S., O’Reardon, Kathleen, Wicklund, Matthew, Kumar, Ashutosh, McDonald, Craig M., Han, Jay J., Joyce, Nanette, Henricson, Erik K., Schara-Schmidt, Ulrike, Gangfuss, Andrea, Wilichowski, Ekkehard, Barohn, Richard J., Statland, Jeffrey M., Campbell, Craig, Vita, Giuseppe, Vita, Gian Luca, Howard, James F., Hughes, Imelda, McMillan, Hugh J., Pegoraro, Elena, Bello, Luca, Burnette, W. Bryan, Thangarajh, Mathula, Chang, Taeun |
| المصدر: |
JAMA |
| بيانات النشر: |
American Medical Association |
| سنة النشر: |
2022 |
| مصطلحات موضوعية: |
Original Investigation, psy, envir |
| الوصف: |
IMPORTANCE: Corticosteroids improve strength and function in boys with Duchenne muscular dystrophy. However, there is uncertainty regarding the optimum regimen and dosage. OBJECTIVE: To compare efficacy and adverse effects of the 3 most frequently prescribed corticosteroid regimens in boys with Duchenne muscular dystrophy. DESIGN, SETTING, AND PARTICIPANTS: Double-blind, parallel-group randomized clinical trial including 196 boys aged 4 to 7 years with Duchenne muscular dystrophy who had not previously been treated with corticosteroids; enrollment occurred between January 30, 2013, and September 17, 2016, at 32 clinic sites in 5 countries. The boys were assessed for 3 years (last participant visit on October 16, 2019). INTERVENTIONS: Participants were randomized to daily prednisone (0.75 mg/kg) (n = 65), daily deflazacort (0.90 mg/kg) (n = 65), or intermittent prednisone (0.75 mg/kg for 10 days on and then 10 days off) (n = 66). MAIN OUTCOMES AND MEASURES: The global primary outcome comprised 3 end points: rise from the floor velocity (in rise/seconds), forced vital capacity (in liters), and participant or parent global satisfaction with treatment measured by the Treatment Satisfaction Questionnaire for Medication (TSQM; score range, 0 to 100), each averaged across all study visits after baseline. Pairwise group comparisons used a Bonferroni-adjusted significance level of .017. RESULTS: Among the 196 boys randomized (mean age, 5.8 years [SD, 1.0 years]), 164 (84%) completed the trial. Both daily prednisone and daily deflazacort were more effective than intermittent prednisone for the primary outcome (P < .001 for daily prednisone vs intermittent prednisone using a global test; P = .017 for daily deflazacort vs intermittent prednisone using a global test) and the daily regimens did not differ significantly (P = .38 for daily prednisone vs daily deflazacort using a global test). The between-group differences were principally attributable to rise from the floor velocity (0.06 rise/s [98.3% CI, 0.03 to 0.08 . |
| نوع الوثيقة: |
text |
| اللغة: |
English |
| العلاقة: |
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8984930Test/ |
| الإتاحة: |
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8984930Test/ |
| حقوق: |
undefined |
| رقم الانضمام: |
edsbas.30436B33 |
| قاعدة البيانات: |
BASE |
