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Weekend vs Daily Prednisone in DMD

Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy

Year of Publication: 2011

Authors: Escolar DM, Hache LP, Clemens PR, ..., Connolly AM

Journal: Neurology

Citation: Escolar DM et al. Neurology. 2011 Aug 2;77(5):444-52. DOI: 10.1212/WNL.0b013e318227b164. PMID: 21753160. PMCID: PMC3146308

Link: https://pubmed.ncbi.nlm.nih.gov/21753160/

Clinical Question

Is weekend high-dose prednisone equivalent to daily prednisone in preserving muscle strength in DMD, and does it offer a better side-effect profile?

Bottom Line

Weekend prednisone (5 mg/kg Saturday + Sunday) was equivalent to daily prednisone (0.75 mg/kg/day) in preserving QMT arm and leg strength over 12 months. Weekend dosing preserved growth (6.6 vs 4.1 cm height gain, p=0.002) and bone density (lumbar Z-score +0.26 vs -0.30, p=0.001) significantly better than daily dosing. Class I evidence for weekend prednisone as a safer alternative.

        Major Points
        Equivalence met for QMT arm score (p=0.009 lower bound, p<0.0001 upper bound) and QMT leg score
QMT elbow flexors and all timed function tests (10m walk, 4-stair climb, supine-to-stand) also equivalent
Weekend dosing significantly preserved height: 6.6 cm vs 4.1 cm growth (p=0.002)
Weekend dosing preserved bone density: lumbar spine Z-score +0.26 vs -0.30 (p=0.001)
BMI at 12 months did not differ between groups
Class I evidence for weekend prednisone as safe and effective alternative to daily dosing

      

Design

Study Type: Multicenter, international, prospective, 12-month, randomized, double-blind equivalence study

Randomization: 1

Blinding: Double-blind (daily group received placebo on weekends; weekend group received placebo on weekdays)

Enrollment Period: November 2003 to November 2007

Follow-up Duration: 12 months

Centers: 12

Countries: USA, Canada, Australia, Israel, India

Sample Size: 64

Analysis: Equivalence analysis with pre-specified margins; 77 screened, 64 randomized (32 per group)

Inclusion Criteria

Ambulant boys with DMD
Age 4 to 10 years
Steroid-naive
Clinical muscle weakness
Reproducible QMT biceps within 15%

Exclusion Criteria

Female carriers
Carnitine, creatine, or other supplements within 3 months
Cardiomyopathy
Positive PPD (tuberculosis screening)
No prior chickenpox and no varicella immunization

Arms

Field	Weekend prednisone	Control
Intervention	Prednisone 5 mg/kg on Saturday + 5 mg/kg on Sunday (10 mg/kg total per week), placebo Monday-Friday	Prednisone 0.75 mg/kg/day every day, placebo on weekends
Duration	12 months	12 months

Outcomes

Outcome	Type	Control	Intervention	P-value
QMT arm score and QMT leg score at 12 months (equivalence design)	Primary	Daily: reference standard	Weekend: equivalent	Equivalence established
QMT elbow flexors	Secondary	Daily	Equivalent	Equivalence met
Timed function tests (10m walk, 4-stair climb, supine-to-stand)	Secondary	Daily	All equivalent	All equivalence met
MVV and MIP (respiratory)	Secondary	Daily	Equivalent	Equivalence met
FVC% and FEV1% (respiratory)	Secondary	Daily	NOT equivalent	Equivalence NOT met
MMT, grip strength, knee extension	Secondary	Daily	NOT equivalent	Equivalence NOT met
BMI at 12 months	Adverse	Daily	Weekend -- no difference	NS
Height change at 12 months	Adverse	Daily: 4.1 cm	Weekend: 6.6 cm	p=0.002 (weekend preserved growth)
Lumbar spine Z-score change	Adverse	Daily: -0.30	Weekend: +0.26	p=0.001 (weekend preserved bone density)
Behavior (CBCL)	Adverse	Daily	No difference	NS
Dose reductions needed	Adverse	Daily: 3 patients	Weekend: 2 patients
SAEs	Adverse	Daily: 6 (mostly disease progression)	Weekend: 6 (mostly disease progression)

Criticisms

Small sample size (64 patients) limits power for equivalence testing
12-month follow-up may miss long-term differences in efficacy or safety
Some secondary measures (MMT, grip, knee, FVC, FEV1) did NOT meet equivalence -- suggesting weekend may not be fully equivalent on all measures
Femoral bone density not assessed (only lumbar spine)
Only compared two dosing regimens; did not test intermittent daily or alternate-day dosing

Funding

Muscular Dystrophy Association (MDA), General Clinical Research Center (GCRC), NIH (K23 RR16281-01)

Based on: Weekend vs Daily Prednisone in DMD (Neurology, 2011)

Authors: Escolar DM, Hache LP, Clemens PR, ..., Connolly AM

Citation: Escolar DM et al. Neurology. 2011 Aug 2;77(5):444-52. DOI: 10.1212/WNL.0b013e318227b164. PMID: 21753160. PMCID: PMC3146308

Content summarized and formatted by NeuroTrials.ai.

Weekend vs Daily Prednisone in DMD

(2011)

Objective

To determine whether weekend prednisone dosing is equivalent to daily dosing in preserving muscle strength in DMD

Study Summary

• Weekend prednisone (10 mg/kg/week) was equivalent to daily (0.75 mg/kg/day) for QMT arm and leg scores at 12 months
• Side-effect profiles did not differ between regimens

Intervention

Prednisone 10 mg/kg/week (weekend) vs prednisone 0.75 mg/kg/day (daily)

Inclusion Criteria

Boys aged 4-10 years with Duchenne muscular dystrophy

Study Design

Arms: Weekend prednisone 10 mg/kg/week, Daily prednisone 0.75 mg/kg/day

Patients per Arm: 32 per arm (64 total)

Outcome

• Equivalence met for QMT arm and leg scores between weekend and daily dosing
• QMT elbow flexors also showed equivalence
• Class I evidence that weekend prednisone is as safe and effective as daily prednisone over 12 months

Bottom Line

Major Points

Equivalence met for QMT arm score (p=0.009 lower bound, p<0.0001 upper bound) and QMT leg score
QMT elbow flexors and all timed function tests (10m walk, 4-stair climb, supine-to-stand) also equivalent
Weekend dosing significantly preserved height: 6.6 cm vs 4.1 cm growth (p=0.002)
Weekend dosing preserved bone density: lumbar spine Z-score +0.26 vs -0.30 (p=0.001)
BMI at 12 months did not differ between groups
Class I evidence for weekend prednisone as safe and effective alternative to daily dosing

Study Design

Study Type: Multicenter, international, prospective, 12-month, randomized, double-blind equivalence study
Randomization: Yes
Blinding: Double-blind (daily group received placebo on weekends; weekend group received placebo on weekdays)
Sample Size: 64
Follow-up: 12 months
Centers: 12
Countries: USA, Canada, Australia, Israel, India

Primary Outcome

Definition: QMT arm score and QMT leg score at 12 months (equivalence design)

Control	Intervention	HR/OR	P-value
Daily: reference standard	Weekend: equivalent	- (Arm score: equivalence met (p=0.009 lower, p<0.0001 upper); Leg score: equivalence met)	Equivalence established

Limitations & Criticisms

Small sample size (64 patients) limits power for equivalence testing
12-month follow-up may miss long-term differences in efficacy or safety
Some secondary measures (MMT, grip, knee, FVC, FEV1) did NOT meet equivalence -- suggesting weekend may not be fully equivalent on all measures
Femoral bone density not assessed (only lumbar spine)
Only compared two dosing regimens; did not test intermittent daily or alternate-day dosing

Citation

Escolar DM et al. Neurology. 2011 Aug 2;77(5):444-52. DOI: 10.1212/WNL.0b013e318227b164. PMID: 21753160. PMCID: PMC3146308

View Original Publication →

Weekend vs Daily Prednisone in DMD

Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy

Clinical Question

Bottom Line

Major Points

Design

Inclusion Criteria

Exclusion Criteria

Arms

Outcomes

Criticisms

Funding

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