PMID- 27538531 OWN - NLM STAT- MEDLINE DCOM- 20170102 LR - 20191210 IS - 1471-2377 (Electronic) IS - 1471-2377 (Linking) VI - 16 IP - 1 DP - 2016 Aug 18 TI - Analysis of number needed to treat for droxidopa in patients with symptomatic neurogenic orthostatic hypotension. PG - 143 LID - 10.1186/s12883-016-0665-5 [doi] LID - 143 AB - BACKGROUND: Droxidopa 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 beta-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). METHODS: Data 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. RESULTS: The 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. CONCLUSIONS: Droxidopa 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 REGISTRATIONS: ClinicalTrials.gov identifiers: NCT00782340 , first received October 29, 2008; NCT00633880 , first received March 5, 2008; and NCT01176240 , first received July 30, 2010. FAU - Francois, Clement AU - Francois C AD - Lundbeck LLC, 6 Parkway North, Deerfield, IL, 60015, USA. CFR@Lundbeck.com. FAU - Rowse, Gerald J AU - Rowse GJ AD - Lundbeck LLC, 6 Parkway North, Deerfield, IL, 60015, USA. FAU - Hewitt, L Arthur AU - Hewitt LA AD - Lundbeck LLC, 6 Parkway North, Deerfield, IL, 60015, USA. FAU - Vo, Pamela AU - Vo P AD - Lundbeck LLC, 6 Parkway North, Deerfield, IL, 60015, USA. FAU - Hauser, Robert A AU - Hauser RA AD - Parkinson's Disease and Movement Disorders Center, University of South Florida, 4001 E Fletcher Ave, Tampa, FL, 33596, USA. LA - eng SI - ClinicalTrials.gov/NCT00782340 SI - ClinicalTrials.gov/NCT00633880 SI - ClinicalTrials.gov/NCT01176240 PT - Journal Article PT - Randomized Controlled Trial DEP - 20160818 PL - England TA - BMC Neurol JT - BMC neurology JID - 100968555 RN - 0 (Antiparkinson Agents) RN - J7A92W69L7 (Droxidopa) SB - IM MH - Aged MH - Aged, 80 and over MH - Antiparkinson Agents/adverse effects/*pharmacology MH - Droxidopa/adverse effects/*pharmacology MH - Female MH - Humans MH - Hypotension, Orthostatic/*drug therapy/etiology MH - Male MH - Middle Aged MH - Nervous System Diseases/*complications MH - Outcome Assessment, Health Care/methods/*statistics & numerical data MH - Randomized Controlled Trials as Topic/*statistics & numerical data PMC - PMC4990877 OTO - NOTNLM OT - Droxidopa OT - Neurogenic orthostatic hypotension OT - Number needed to harm OT - Number needed to treat OT - Risk reduction OT - nOH treatment benefit EDAT- 2016/08/20 06:00 MHDA- 2017/01/04 06:00 PMCR- 2016/08/18 CRDT- 2016/08/20 06:00 PHST- 2015/12/22 00:00 [received] PHST- 2016/08/05 00:00 [accepted] PHST- 2016/08/20 06:00 [entrez] PHST- 2016/08/20 06:00 [pubmed] PHST- 2017/01/04 06:00 [medline] PHST- 2016/08/18 00:00 [pmc-release] AID - 10.1186/s12883-016-0665-5 [pii] AID - 665 [pii] AID - 10.1186/s12883-016-0665-5 [doi] PST - epublish SO - BMC Neurol. 2016 Aug 18;16(1):143. doi: 10.1186/s12883-016-0665-5.