Evidence map›Paper›PMID 23553478›Full record

Trial reportNeurology2013

Hyperdopaminergic crises in familial dysautonomia: a randomized trial of carbidopa.

Lucy Norcliffe-Kaufmann, Jose Martinez, Felicia Axelrod, Horacio Kaufmann

Registry-linked trialOpen access · bronzeAbstract readRandomized Controlled Trial
In one paragraph

Trial report in Neurology, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02553265 (Carbidopa in Familial Dysautonomia), which is not on this map. Cited by 22 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
22citing papers in PubMed, 1 pooled it
1.1field-weighted citation impact, top 25% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT02553265 phase2completednot on this mapstarted 2015, after this paper: background citation

Carbidopa in Familial Dysautonomia: Phase-II Study, Investigational New Drug (IND) 117435, Date: 01/07/13

TypeinterventionalSponsorNYU Langone HealthRan2015 to 2019Enrolled22ConditionsDysautonomia, Familial, Baroreflex Failure SyndromeArmsCarbidopa Low-Dose, Placebo, Carbidopa High-Dose
3 · Its place in the literature

Who cites it

22 citing papers in PubMed, 1 synthesis or guideline pooled it, 40 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Trial
  4. Review
  5. Review
  6. Article
  7. Carbidopa: beyond Parkinson's disease.Clinical autonomic research : official journal of the Clinical Autonomic Research Society · 2025
    Review
  8. Article
  9. Familial dysautonomia.Clinical autonomic research : official journal of the Clinical Autonomic Research Society · 2023
    Review
  10. Article
  11. Article
  12. Genetic pain loss disorders.Nature reviews. Disease primers · 2022
    Review
  13. Article
  14. Gastrointestinal motility disorders in neurologic disease.The Journal of clinical investigation · 2021
    Review
  15. Review
  16. Ambulatory blood pressure profiles in familial dysautonomia.Clinical autonomic research : official journal of the Clinical Autonomic Research Society · 2018
    Article
  17. Cyclic vomiting syndrome: the nervous system has the guts.Clinical autonomic research : official journal of the Clinical Autonomic Research Society · 2018
    Article
  18. Intranasal dexmedetomidine for adrenergic crisis in familial dysautonomia.Clinical autonomic research : official journal of the Clinical Autonomic Research Society · 2017
    Article
  19. Dexmedetomidine for refractory adrenergic crisis in familial dysautonomia.Clinical autonomic research : official journal of the Clinical Autonomic Research Society · 2017
    Article
  20. Observational
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors at 1 institution in 1 country.

Lucy Norcliffe-KaufmannDysautonomia Center, New York University School of Medicine, New York, NY, USA.
Jose Martinez
Felicia Axelrod
Horacio Kaufmann
Dysautonomia Foundation · US

Funding

Carbidopa for the treatment of nausea and vomiting in familial dysautonomiaIND #R01FD003731 · FDA · NEW YORK UNIVERSITY SCHOOL OF MEDICINE · PI KAUFMANN, HORACIO · 2009 to 2010
$366k
FDA HHS R01- FD003731-02
6 · The paper itself

Abstract

objectiveThe purpose of this study was to determine whether carbidopa (Lodosyn), an inhibitor of dopa-decarboxylase that blocks the synthesis of dopamine outside the brain, is an effective antiemetic in patients with familial dysautonomia (FD) and hyperdopaminergic nausea/retching/vomiting attacks.

methodsWe enrolled 12 patients with FD in an open-label titration and treatment study to assess the safety of carbidopa. We then conducted a randomized, double-blind, placebo-controlled, crossover study to evaluate its antiemetic efficacy.

resultsPrevious fundoplication surgery in each patient studied prevented vomiting, but all of the subjects experienced severe cyclical nausea and uncontrollable retching that was refractory to standard treatments. Carbidopa at an average daily dose of 480 mg (range 325-600 mg/day) was well tolerated. In the double-blind phase, patients experienced significantly less nausea and retching while on carbidopa than on placebo (p < 0.03 and p < 0.02, respectively). Twenty-four-hour urinary dopamine excretion was significantly lower while on carbidopa (147 ± 32 µg/gCr) than while on placebo (222 ± 41µg/gCr, p < 0.05).

conclusionsCarbidopa is a safe and effective antiemetic in patients with FD, likely by reducing the formation of dopamine outside the brain. CLASSIFICATION OF EVIDENCE: This study provides Class II evidence that carbidopa is effective in reducing nausea/retching/vomiting in patients with FD.

Indexed as

AdolescentAdultAntiemeticsCarbidopaChildCross-Over StudiesDopamine AgentsDouble-Blind MethodDysautonomia, FamilialFemaleHumansMaleNauseaVomitingYoung AdultAntiemeticsCarbidopaDopamine Agents

Identifiers

PMID23553478
PMCPMC3662326
OpenAlexW2166682821

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.