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Monographs of Topical Drugs that Have Caused Contact Allergy/Allergic Contact Dermatitis
Published in Anton C. de Groot, Monographs in Contact Allergy, 2021
A 63-year-old woman developed an acute itchy, maculopapular eruption of the trunk, arms and legs after taking deflazacort as an adjuvant for chemotherapy. She had suffered from eczematous dermatitis in the past after using various topical corticosteroids for lichen planus, including desoximetasone. Patch tests were positive to deflazacort 1% alc., desoximetasone 1% alc. and 8 other corticosteroids (9). This also was a case of systemic contact dermatitis.
Expression of Cell Adhesion Molecules in Allergic Disorders of the Eye
Published in Bruce S. Bochner, Adhesion Molecules in Allergic Disease, 2020
Giorgio Walter Canonica, Antonio Scordamaglia, Francesca Paolieri, Nicolò Fiorino, Giovanni Passalacqua, Giorgio Ciprandi
Deflazacort, a new systemic corticosteroid, reduces ICAM-1 in the early as well in the late phase of the allergic response induced by allergen-specific challenge (20). Further studies on topical antihistamines (such as azelastine and levocabastine) have demonstrated their antiallergic activity (Table 4).
D
Published in Caroline Ashley, Aileen Dunleavy, John Cunningham, The Renal Drug Handbook, 2018
Caroline Ashley, Aileen Dunleavy, John Cunningham
Deflazacort is immediately converted by plasma esterases to the pharmacologically active metabolite (D 21-OH). It is 40% protein-bound and has no affinity for corticosteroid-binding-globulin (transcortin). Elimination takes place primarily through the kidneys; 70% of the administered dose is excreted in the urine. The remaining 30% is eliminated in the faeces. Metabolism of D 21-OH is extensive; only 18% of urinary excretion represents D 21-OH. The metabolite of D 21-OH, deflazacort 6-beta-OH, represents one third of the urinary elimination.
Perspectives on the advances in the pharmacotherapeutic management of Duchenne muscular dystrophy
Published in Expert Opinion on Pharmacotherapy, 2022
Kelsie D. Kracht, Nicole L. Eichorn, Daniel J. Berlau
Although corticosteroids are critical to controlling inflammation in DMD patients, precise dosing of these medications has been difficult. Because patients with DMD need these medications for lifelong treatment, they are at high risk for side effects from chronic steroid use. Lower doses of these medications can reduce these chronic effects, but at the cost of reduced anti-inflammatory effect. This seems to be the case for prednisone and to some extent deflazacort, however there is some hope that newer synthetic steroid medications, such as vamorolone, might have decreased adverse outcomes. Nevertheless, patients, physicians, and caregivers need to work together to create a therapeutic plan that provides therapeutic benefit, which minimizes adverse effects. Imperative to the discussion is the cost of deflazacort. Deflazacort was developed as a safer alternative to prednisone but has limited use due to cost. In 2019, the Institute for Clinical and Economic Review (ICER) reported the annual treatment cost of prednisone (0.75 mg/kg/day) to be $550 and $81,400 for deflazacort (0.9 mg/kg/day) for a 40 kg patient [103]. This disparity makes it difficult to provide the best medication for all patients when cost is a factor.
Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies
Published in Expert Opinion on Investigational Drugs, 2021
Corticosteroids, which comprise a class of steroid hormones produced in the adrenal cortex, lower inflammation, offer effective therapy for many patients with DMD [21]. These drugs bind to the glucocorticoid receptor, which regulates many pathways involved in immune response and metabolism [22]. For example, inflammatory NF-κB signaling is inhibited by corticosteroids [23,24]. Although the mechanism of action is not fully understood, research points to the effectiveness of corticosteroids [25] in delaying loss of motor function, supporting their prescription to many DMD patients. According to one study in DMD patients, corticosteroids inhibit the loss of strength [26]. However, until recently, clinical trials have only extensively examined deflazacort (brand name Emflaza®) and prednisone. Deflazacort was approved in 2017 by the FDA [27]. Although prednisone is frequently prescribed for DMD, it has not been approved specifically for DMD [28]. In other words, prednisone has been prescribed off-label, which refers to a medication used differently from its FDA approved usage as a pharmacotherapeutic standard of care, for the treatment of DMD. A meta-analysis indicated that, compared to prednisone-treated patients, deflazacort-treated patients experienced lower declines based on 6-minute walk distance and the North Star Ambulatory Assessment (NSAA) [29]. Though widely prescribed, corticosteroids are controversial because of their common adverse effects on body mass, sleep quality, and bone integrity in DMD patients [30,31] Patients cite ‘side effects’ and ‘not enough benefit’ as their primary reasons to discontinue corticosteroids [32].
The potential and benefits of repurposing existing drugs to treat rare muscular dystrophies
Published in Expert Opinion on Orphan Drugs, 2018
Hesham M. Ismail, Olivier M. Dorchies, Leonardo Scapozza
They act to delay the loss of independent ambulation and the onset of cardiac and respiratory failure. Their mechanism of action is still debated and was attributed to a potent anti-inflammatory effect, anabolic effect, promotion of muscle-specific gene expression, correction of Ca2+ dysregulation, and activation of the calcineurin pathway among other mechanisms still being investigated [47–50]. However, corticosteroid treatment is accompanied by a variety of undesirable effects, including hyperactivity, weight gain, bone fragility, hypertension, and susceptibility to diabetes mellitus and infections. According to some authors, another corticoid, deflazacort, provides an alternative to the more commonly used prednisolone with less severe undesirable effects [51]. However, this is highly disputed and is reflected in a recent study that shows lack of standardized treatment protocols in clinics specialized in DMD treatment [52]. In fact, optimized treatment protocols can make both agents attain a comparable efficacy with minimal undesired effects, which highlights the need for standardized treatment protocols [53]. Another factor that can play a role in variation of patient-to-patient response is the genetic polymorphism in the glucocorticoid receptor. Since last year, deflazacort is sold in the United States under the trade name Emflaza for DMD patients aged 5 and above. A novel approach to improve the therapeutic profile of glucocorticoids is the use of glucocorticoid receptor modulators. In fact, the undesirable effects of standard glucocorticoid receptor agonists result from formation of the receptor homodimer that transcribes the relevant genes via the glucocorticoid response elements. A series of reports provided evidence that vamorolone (VBP15), a ‘dissociative’ steroidal compound, is capable of transrepressing these genes without transactivation [54]. The authors of this study showed in vitro NF-κB inhibitory activity in mdx myoblasts and myotubes, and demonstrated improvements in the disease phenotype of mdx mice. Vamorolone is currently undergoing Phase IIa clinical trials (NCT02760264).