deflazacort
Sources réglementaires consultées
Indications approuvées
- Maladies rhumatismales, auto-immunes, dermatologiques, allergiques, respiratoires, hématologiques et autres nécessitant un glucocorticoïde systémique selon le RCP.
Contre-indications
Absolues
- Pour une utilisation dépassant la substitution ou l’urgence brève : ulcère peptique, tuberculose active, herpès oculaire, varicelle, mycose systémique et période pré- et post-vaccinale.
- Hypersensibilité au déflazacort ou aux excipients.
Mises en garde cliniques
- Mise en garde majeure · Utiliser la dose minimale efficace et diminuer progressivement après un traitement prolongé ; un arrêt brutal peut provoquer une insuffisance surrénalienne. — CIMA/AEMPS, ficha técnica 73873
- Mise en garde majeure · Il peut accroître la sensibilité aux infections, réactiver des infections latentes et en masquer les signes. Éviter les vaccins vivants aux doses immunosuppressives. — CIMA/AEMPS, ficha técnica 73873
- Mise en garde majeure · Surveiller la glycémie, la pression artérielle, les électrolytes, la santé osseuse, les symptômes psychiatriques ainsi que les complications oculaires et gastro-intestinales pendant le traitement systémique. — CIMA/AEMPS, ficha técnica 73873
Interactions médicamenteuses
- ModéréeInhibiteurs ou inducteurs du CYP3A4
Mécanisme: Ils peuvent augmenter ou diminuer l’exposition systémique au glucocorticoïde.
Recommandation: Surveiller l’efficacité et la toxicité et n’ajuster que sous supervision clinique.
CIMA/AEMPS, ficha técnica 73873
- SévèreVaccins vivants
Mécanisme: L’immunosuppression peut favoriser une infection disséminée et réduire la réponse vaccinale.
Recommandation: Éviter les vaccins vivants pendant un traitement immunosuppresseur ; planifier la vaccination avec l’équipe soignante.
CIMA/AEMPS, ficha técnica 73873
- ModéréeAntidiabétiques, diurétiques et AINS
Mécanisme: Les glucocorticoïdes peuvent compromettre le contrôle glycémique, aggraver la perte de potassium et augmenter le risque gastro-intestinal.
Recommandation: Surveiller la glycémie et les électrolytes et envisager une gastroprotection ou des alternatives selon le risque.
CIMA/AEMPS, ficha técnica 73873
Effets indésirables
Rares mais graves
Insuffisance surrénalienne, infection opportuniste, hémorragie gastro-intestinale, psychose et ostéonécrose
Grossesse et allaitement
Pendant la grossesse, n’utiliser que si le bénéfice dépasse le risque fœtal potentiel. Il passe dans le lait et peut affecter la croissance et la production stéroïdienne du nourrisson ; individualiser l’allaitement.
Bibliographie récente (PubMed)
Corticosteroids improve strength and function in boys with Duchenne muscular dystrophy. However, there is uncertainty regarding the optimum regimen and dosage. To compare efficacy and adverse effects of the 3 most frequently prescribed corticosteroid regimens in boys with Duchenne muscular dystrophy. 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). 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). 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. 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 rise/s] for daily prednisone vs intermittent prednisone [P = .003]; 0.0
The objective of this study was to describe predictors of loss of ambulation in Duchenne muscular dystrophy (DMD). This systematic review and meta-analysis included searches of MEDLINE ALL, Embase, and the Cochrane Database of Systematic Reviews from January 1, 2000, to December 31, 2022, for predictors of loss of ambulation in DMD. Search terms included "Duchenne muscular dystrophy" as a Medical Subject Heading or free text term, in combination with variations of the term "predictor". Risk of bias was assessed using the Newcastle-Ottawa Scale. We performed meta-analysis pooling of hazard ratios of the effects of glucocorticoids (vs. no glucocorticoid therapy) by fitting a common-effect inverse-variance model. The bibliographic searches resulted in the inclusion of 45 studies of children and adults with DMD from 17 countries across Europe, Asia, and North America. Glucocorticoid therapy was associated with delayed loss of ambulation (overall meta-analysis HR deflazacort/prednisone/prednisolone: 0.44 [95% CI: 0.40-0.48]) (n = 25 studies). Earlier onset of first signs or symptoms, earlier loss of developmental milestones, lower baseline 6MWT (i.e.,<350 vs. ≥350 metres and <330 vs. ≥330 metres), and lower baseline NSAA were associated with earlier loss of ambulation (n = 5 studies). Deletion of exons 3-7, proximal mutations (upstream intron 44), single exon 45 deletions, and mutations amenable of skipping exon 8, exon 44, and exon 53, were associated with prolonged ambulation; distal mutations (intron 44 and downstream), deletion of exons 49-50, and mutations amenable of skipping exon 45, and exon 51 were associated with earlier loss of ambulation (n = 13 studies). Specific single-nucleotide polymorphisms in CD40 gene rs1883832, LTBP4 gene rs10880, SPP1 gene rs2835709 and rs11730582, and TCTEX1D1 gene rs1060575 (n = 7 studies), as well as race/ethnicity and level of family/patient deprivation (n = 3 studies), were associated with loss of ambulation. Treatment with atal
Use of glucocorticoid in various diseases including rheumatology and respiratory diseases is on the rise because of its prompt beneficial effects. This culminates in osteoporosis and fragility fractures. Judicious use of glucocorticoid hence calls for attention with regard to the dose schedule, route of administration and accompanying enhancing factors. Institution of proper therapeutic management as per WHO risk stratification with anabolic and/or resorptive drugs like bisphosphonates, teriparatide or denosumab is necessary to prevent the eventuality of fragility fractures. Even otherwise, knowledge of glucocorticoid, its metabolism, various dose schedules, adverse effects are areas worth discussing.
Duchenne muscular dystrophy (DMD) is a severe degenerative disease that remains incurable. Its prognosis has been transformed by multidisciplinary care, which has significantly improved the life expectancy of patients. Corticosteroids have been used for over 20 years and have modified the care of young boys with DMD: They delay the loss of ambulation, support the gradual introduction of technical mobility aids, and reduce the frequency of scoliosis surgery. Corticoids are started and maintained at the theoretical dose of 0.75 mg/kg/day of prednisone/prednisolone or 0.9 mg/kg/day of deflazacort, adjusting the dose to weight if the benefit-risk ratio favors treatment. If introduced early enough, when muscle degeneration is not too advanced, they prolong the ability to climb stairs, rise from the floor, walk, and bring the hands to the mouth. They have been suggested to contribute to delaying the onset of respiratory failure and cardiomyopathy. The well-known side effects of corticosteroids require specific management (to prevent obesity and osteoporosis) and/or adjustment of their dosage. The only reasons to discontinue treatment when the patient and family are adhering to it are the uncontrolled side effects. Thus, in many countries including France, as of late 2025, the only routinely prescribed symptomatic treatment for DMD remains conventional corticosteroids, which will be discussed in this article, followed by a description of vamorolone, a dissociative steroidal compound, and givinostat, a histone deacetylase inhibitor.
Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder characterized by progressive muscle degeneration and weakness, caused by mutations in the dystrophin gene. DMD has effects in early age with significantly shortened lifespan and deteriorated quality of life in the second decade, creating an urgent need to develop better therapeutic options. Corticosteroid medication therapy is an integral tool for the management of DMD and several therapeutic options have been recently approved for use. A comprehensive literature search was completed to examine efficacy and safety profiles of the three corticosteroid medications available for use in DMD patients. The review presents information about the three agents through clinical trials, significant preclinical trials, and comparative studies. Managing DMD takes a multidisciplinary approach, although long-term corticosteroid therapy remains a significant therapeutic tool. Based on the available published studies, unequivocal comparison between the benefits of the three medications cannot yet be made. When selecting a medication for a patient, the decision-making process will most likely rely on the minor differences in the adverse effect profiles. Whichever medication is utilized will surely be a part of a larger regimen that includes other novel therapeutic agents.