Deflazacort
Brand names: Calcort, Emflaza (US)
Deflazacort is an oral glucocorticoid corticosteroid used for its anti-inflammatory and immunosuppressant effects, including in Duchenne muscular dystrophy.
Adult dose
Paediatric dose
Dose adjustments
UK SPC §4.2, verbatim: 'In renally impaired patients, no special precautions other than those usually adopted in patients receiving glucocorticoid therapy are necessary.'
UK SPC §4.2, verbatim: 'In patients with hepatic impairment, blood levels of deflazacort may be increased. Therefore, the dose of deflazacort should be carefully monitored and adjusted to the minimum effective dose.'
Dose independently re-sourced from UK Summary of Product Characteristics (SPC) via the eMC; US FDA prescribing information (openFDA / DailyMed) — cross-check; US labelling may differ from UK — clinician-reviewed. Always confirm against the product SmPC and your local formulary before prescribing.
⚠️ UNIT: mg/kg/DAY — a total daily dose given once daily, not a per-dose figure. VERBATIM §4.2 paediatric population, in full: 'There has been limited exposure of children to deflazacort in clinical trials. In children, the indications for glucocorticoids are the same as for adults, but it is important that the lowest effective dosage is used. Alternate day administration may be appropriate (see section 4.4). Doses of deflazacort usually lie in the range 0.25 – 1.5 mg/kg/day. The following ranges provide general guidance: Juvenile chronic arthritis: The usual maintenance dose is between 0.25 – 1.0 mg/kg/day. Nephrotic syndrome: Initial dose of usually 1.5 mg/kg/day followed by down titration according to clinical need. Bronchial asthma: On the basis of the potency ratio, the initial dose should be between 0.25 – 1.0 mg/kg deflazacort on alternate days.' || WHY dosePerKg IS 0.25: the SPC gives a RANGE (0.25 – 1.5 mg/kg/day) and no single figure, so the calculator is seeded with the label's own lower bound and the label's own upper bound is carried in maxDose. Nothing has been averaged, scaled or derived. Choose within 0.25–1.5 mg/kg/day by indication using the label's own bands above — juvenile chronic arthritis 0.25–1.0 mg/kg/day; nephrotic syndrome usually 1.5 mg/kg/day initially, then down-titrated; bronchial asthma 0.25–1.0 mg/kg on ALTERNATE DAYS (note the alternate-day schedule for asthma, which the calculator's daily figure does not express). || concentration is NULL because the UK source product is a 6 mg TABLET, not a liquid — this bundle contains no UK suspension strength, so no volume can be calculated. || THE 0.9 mg/kg/day FIGURE IS US LABELLING FOR ONE INDICATION ONLY and is NOT the UK general paediatric dose: the openFDA provider's US label for 'Deflazacort Oral Suspension' states 'The recommended oral dosage of deflazacort oral suspension is approximately 0.9 mg/kg/day once daily', licensed for Duchenne muscular dystrophy, in patients 2 years of age and older, at a suspension strength of 22.75 mg/mL, with a US instruction to give one third of the dose with moderate or strong CYP3A4 inhibitors and to avoid moderate or strong CYP3A4 inducers. It is recorded here for reference and clearly attributed; it must not be applied to asthma, croup, nephrotic syndrome, juvenile chronic arthritis or any other indication, and it is not the figure the calculator uses. || SAFETY: the SPC itself warns 'There has been limited exposure of children to deflazacort in clinical trials.' Verify any paediatric dose against a children's formulary and current prescribing references before prescribing.
Contraindications
- UK SPC §4.3: 'Hypersensitivity to the active substance, deflazacort or any of the excipients listed in section 6.1.'
- UK SPC §4.3: 'Systemic infection unless specific anti-infective therapy is employed.'
- UK SPC §4.3: 'Patients receiving live virus immunisation (see section 4.4 and 4.5).'
- Not a labelled contraindication but a §4.4 warning that behaves like one in practice: 'Chickenpox is of particular concern since this normally minor illness may be fatal in immunosuppressed patients. Patients (or parents of children) without a definite history of chicken pox should be advised to avoid close personal contact with chickenpox or herpes zoster and, if exposed, they should seek urgent medical attention.'
Side effects
- §4.8 Endocrine — Uncommon: suppression of the hypothalamic-pituitary-adrenal axis, amenorrhoea, Cushingoid facies. Not known: growth suppression in infancy, childhood and adolescence; steroid withdrawal syndrome.
- §4.8 Metabolism and nutrition — Common: weight gain. Uncommon: impaired carbohydrate tolerance with increased requirement for anti-diabetic therapy; sodium and water retention with hypertension; potassium loss and hypokalaemic alkalosis when co-administered with beta-2-agonist and xanthines. Not known: negative protein and calcium balance, increased appetite; tumour lysis syndrome reported with Calcort in patients with haematological malignancies.
- §4.8 Infections — Uncommon: increased susceptibility and severity of infections with suppression of clinical symptoms and signs; opportunistic infections; recurrence of dormant tuberculosis. Not known: candidiasis.
- §4.8 Musculoskeletal — Uncommon: osteoporosis, vertebral and long bone fractures. Rare: muscle wasting. Not known: avascular osteonecrosis; tendonitis and tendon rupture when co-administered with quinolones; myopathy (acute myopathy may be precipitated by non-depolarising muscle relaxants); negative nitrogen balance.
- §4.8 Cardiac — Not known: heart failure; hypertrophic cardiomyopathy in preterm infants.
- §4.8 Nervous system — Uncommon: headache, vertigo. Not known: restlessness; increased intra-cranial pressure with papilloedema in children (pseudotumour cerebri), usually after treatment withdrawal; aggravation of epilepsy.
- §4.8 Psychiatric — Uncommon: depressed and labile mood, behavioural disturbances. Not known: irritable, euphoric, suicidal thoughts, psychotic reactions (including mania, delusions, hallucinations, aggravation of schizophrenia), anxiety, sleep disturbances, cognitive dysfunction including confusion and amnesia. The label adds: 'Reactions are common and may occur in both adults and children. In adults, the frequency of severe reactions has been estimated to be 5 – 6%. Psychological effects have been reported on withdrawal of corticosteroids; the frequency is unknown.'
- §4.8 Eye — Not known: vision blurred, increased intra-ocular pressure, glaucoma, papilloedema, posterior subcapsular cataracts. ⚠️ The §4.8 eye row is where the source-fetch limit truncates this section, so the adverse-effect list above is NOT complete.
- §4.8 Reproductive — Not known: menstrual irregularity.
Monitoring
- §4.4: 'Frequent patient review is required to appropriately titrate the dose against disease activity.' Give the lowest possible dose for the minimum period, as a single morning dose or single morning dose on alternate days where possible.
- Adrenal suppression, §4.4: 'Glucocorticoid induced suppression of hypothalamic-pituitary-adrenal function is dependent on dose and duration of treatment. … Adrenal cortical atrophy develops during prolonged therapy and may persist for years after stopping treatment. During prolonged therapy, any intercurrent illness, trauma or surgical procedure will require a temporary increase in dosage.'
- Steroid treatment card, §4.4: 'Patients should carry "Steroid treatment" cards which give clear guidance on the precautions to be taken to minimise risk and which provide details of prescriber, drug, dosage and the duration of treatment.'
- Watch for steroid withdrawal syndrome on stopping, §4.4: 'typical symptoms include fever, anorexia, nausea, lethargy, malaise, myalgia, arthralgias, rhinitis, conjunctivitis, desquamation of the skin and painful itchy skin nodules, weakness, hypotension and weight loss. This may occur in patients even without evidence of adrenal insufficiency.'
- Infection surveillance, §4.4: 'Suppression of the inflammatory response and immune function increases the susceptibility to infections and their severity. The clinical presentation may often be atypical and serious infections such as septicaemia and tuberculosis may be masked and may reach an advanced stage before being recognised.'
- Chickenpox / herpes zoster exposure in a non-immune patient — §4.4 directs urgent medical attention and passive immunisation with varicella zoster immunoglobulin for exposed non-immune patients. ⚠️ §4.4 is truncated at the source-fetch limit part-way through this sentence, so the full VZIG instruction was not retrieved.
- Growth in children — §4.8 lists growth suppression in infancy, childhood and adolescence as a labelled effect; monitor growth on prolonged treatment.
Clinical monograph
How it works
It binds glucocorticoid receptors to modulate gene expression, suppressing inflammation and immune responses.
Prescribing in practice
- Prolonged use causes adrenal suppression, so it must not be stopped abruptly and the dose should be tapered gradually; patients require sick-day guidance.
- Long-term corticosteroid use carries risks including growth suppression in children, osteoporosis, weight gain, hyperglycaemia and increased infection susceptibility.
- Live vaccines should generally be avoided during immunosuppressive doses, and exposure to chickenpox or measles in non-immune patients needs prompt assessment.
Monitoring
Monitor growth in children, weight, blood pressure, blood glucose and bone health during long-term treatment.
Counselling the patient
- Never stop this medicine suddenly; carry a steroid alert card and follow sick-day advice if unwell.
- Avoid contact with chickenpox or measles if you have not had them, and report any exposure.
- Report increased thirst, frequent infections or mood changes to your team.
Evidence & guidelines
Corticosteroids such as deflazacort are recommended to preserve muscle function in Duchenne muscular dystrophy.
Reference: SmPC Calcort; DMD Care Considerations Lancet Neurol 2018; FDA Approval Emflaza (deflazacort) for DMD 2017; Drug verified in RxNorm (NLM); confirm dosing against the manufacturer SPC (eMC). Verify against your local formulary and current prescribing references before prescribing. The structured dose values shown have been reviewed by a clinician. Monograph status: clinician-reviewed (2026-07-04).
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