Analysis of number needed to treat for droxidopa in patients with symptomatic neurogenic orthostatic hypotensionReport as inadecuate

Analysis of number needed to treat for droxidopa in patients with symptomatic neurogenic orthostatic hypotension - Download this document for free, or read online. Document in PDF available to download.

BMC Neurology

, 16:143

Movement disorders


BackgroundDroxidopa is an orally active prodrug that significantly improved dizziness-lightheadedness measured using the Orthostatic Hypotension Symptom Assessment OHSA Item 1 in patients with neurogenic orthostatic hypotension nOH caused by primary autonomic failure Parkinson disease, multiple system atrophy, and pure autonomic failure, dopamine β-hydroxylase deficiency, or nondiabetic autonomic neuropathy. The efficacy and safety of droxidopa were assessed by determining the number needed to treat NNT and the number needed to harm NNH.

MethodsData collected in randomized, placebo-controlled clinical studies in adults with a clinical diagnosis of symptomatic nOH were pooled for efficacy and safety analyses. NNT and NNH were calculated as reciprocals of the risk difference difference in event rates for droxidopa versus placebo.

ResultsThe NNT for droxidopa for improvement in OHSA Item 1 was <10. The NNH for adverse events AEs leading to discontinuation in the pooled studies was 81. The likelihood of being helped or harmed LHH calculated from pooled analysis of the NNT for ≥2 units of improvement in OHSA Item 1 score and the NNH for discontinuations due to AEs were 7.8, 8.8, 3.1, and 3.5 for weeks 1, 2, 4, and 8 after randomization, respectively.

ConclusionsDroxidopa is efficacious for treatment of nOH, with an NNT below 10 and an acceptable tolerability profile with NNH ranging from 23 to 302 in the pooled analysis of frequently occurring AEs. Based on the LHH for the pooled analysis at week 1, droxidopa is 7.8 times more likely than placebo to show a clinical benefit than result in discontinuation because of an AE.

Trial identifiers: NCT00782340, first received October 29, 2008; NCT00633880, first received March 5, 2008; and NCT01176240, first received July 30, 2010.

KeywordsNeurogenic orthostatic hypotension Droxidopa nOH treatment benefit Number needed to treat Number needed to harm Risk reduction AbbreviationsAEAdverse Event

DBHDDopamine β-hydroxylase Deficiency

DBPDiastolic Blood Pressure

FDAUS Food and Drug Administration

LHHLikelihood of Being Helped or Harmed

MSAMultiple System Atrophy

NDANNondiabetic Autonomic Neuropathy

NNHNumber Needed to Harm

NNTNumber Needed to Treat

nOHNeurogenic Orthostatic Hypotension

NSNot Significant

OHDASOrthostatic Hypotension Daily Activity Scale

OHQOrthostatic Hypotension Questionnaire

OHSAOrthostatic Hypotension Symptom Assessment

PDParkinson Disease

SBPSystolic Blood Pressure

TEAETreatment-emergent Adverse Event

TID3 Times Daily

Gerald J. Rowse and Pamela Vo were Lundbeck employees at the time of manuscript development.

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Author: Clément François - Gerald J. Rowse - L. Arthur Hewitt - Pamela Vo - Robert A. Hauser



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