DOSING PATTERNS ASSOCIATED WITH DEFLAZACORT USE IN DUCHENNE MUSCULAR DYSTROPHY (DMD)- OBSERVATIONS FROM DEFLAZACORT EARLY ACCESS PROGRAM (EAP) IN THE U.S
Author(s)
Narayanan S, Souza M
PTC Therapeutics, Inc., South Plainfield, NJ, USA
Presentation Documents
OBJECTIVES: Deflazacort (EMFLAZA™) is the first Food and Drug Administration (FDA)-approved corticosteroid treatment for DMD in patients >=5yrs of age. Deflazacort is available both as tablet (6/18/30/36mg) and oral suspension. Objective of this analysis is to evaluate real-world dosing patterns associated with deflazacort in EAP in the U.S. METHODS: Deflazacort EAP was an open label study implemented between Sept 2015 and May 2017, per following inclusion criteria: patients >=5yrs of age with DMD who were ineligible/unable/otherwise unwilling to enroll in a clinical study while marketing application (for deflazacort) was under preparation/review; child or adolescent patients (<18yrs) weighed >=13kg, with body mass index (BMI) <40kg/m2, and were up-to date on childhood vaccinations; adult patients (>18yrs) had 18.5kg/m2<BMI<40 kg/m2. Patients with history of hypersensitivity or allergic reaction to steroids or their formulations and those who, in physician judgment, had a history/current medical condition that could pose safety/risk to patient were excluded. Protocol-recommended dose of deflazacort was 0.9 mg/kg/day and physicians adjusted dose per clinical judgment. RESULTS: In total, 860 eligible DMD patient records were included in the analysis. Patient characteristics included - mean age:12.8yrs, % prescribed tablet/oral suspension:86.1%/13.9%, % prescribed once-daily dosing:89.9%. Weight varied from 14.8-136kg. Mean daily protocol-recommended dose vs. actual-prescribed dose was 40.4mg (STD:18.8mg) vs. 27.0mg (STD:10.9mg); the corresponding mean difference in daily protocol-recommended vs. actual-prescribed dosing was 13.3mg (STD:16.8mg) across the cohort; this mean daily dose difference increased as the patient weight increased. Corresponding mean % variance of actual-prescribed dose from protocol-recommended dose was: 10-19kg:+0.5%, 20-29kg: -5.2%, 30-39kg: -17.7%, 40-49kg: -27.6%, 50-59kg: -37.5%, 60-69kg: -43.0%, 70-79kg: -54.0%, 80-89kg: - 51.9%, 90-99kg: -57.1%, 100-109kg: -57.7%, 110-119kg: -66.4%, >=120kg: -63.4%. CONCLUSIONS: In this cohort of EAP patients on deflazacort, the actual prescribed dose was lower than the protocol-recommended dose and the variance increased with patient weight.
Conference/Value in Health Info
2017-11, ISPOR Europe 2017, Glasgow, Scotland
Value in Health, Vol. 20, No. 9 (October 2017)
Code
PSY156
Topic
Health Service Delivery & Process of Care
Topic Subcategory
Prescribing Behavior, Treatment Patterns and Guidelines
Disease
Neurological Disorders