A Controlled Trial of Inhaled Bronchodilators in Familial Dysautonomia

Bat el Bar-Aluma*, Ori Efrati, Horacio Kaufmann, Jose Alberto Palma, Lucy Norcliffe-Kaufmann

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

5 Scopus citations

Abstract

Background: Chronic lung disease is a leading cause of premature death in patients with familial dysautonomia (FD). A significant number of patients have obstructive airway disease, yet it is not known whether this is pharmacologically reversible. Methods: We conducted a double-blind, placebo-controlled, randomized clinical trial comparing the beta 2 agonist albuterol with the muscarinic blocker ipratropium bromide in patients homozygous for the IKBKAP founder mutation. Albuterol, ipratropium bromide, and placebo were administered on 3 separate days via nebulizer in the seated position. Airway responsiveness was evaluated using spirometry and impulse oscillometry 30 min post dose. Cardiovascular effects were evaluated by continuous monitoring of blood pressure, RR intervals, cardiac output, and systemic vascular resistance. Results: A total of 14 patients completed the trial. Neither active agent had significant detrimental effects on heart rate or rhythm or blood pressure. Albuterol and ipratropium were similar in their bronchodilator effectiveness causing significant improvement in forced expiratory volume in 1-s (FEV1, p = 0.002 and p = 0.030). Impulse oscillometry measures were consistent with a reduction in total airway resistance post nebulization (resistance at 5 Hz p < 0.006). Conclusion: Airway obstruction is pharmacologically reversible in a number of patients with FD. In the short term, both albuterol and ipratropium were well tolerated and not associated with major cardiovascular adverse events.

Original languageEnglish
Pages (from-to)93-101
Number of pages9
JournalLung
Volume196
Issue number1
DOIs
StatePublished - 1 Feb 2018

Funding

FundersFunder number
Daphna Vilozni
National Institutes of Health
National Institute of Neurological Disorders and StrokeU54NS065736
Dysautonomia Foundation
Food and Drug Administration

    Keywords

    • Autonomic
    • Bronchodilators
    • Familial dysautonomia/HSAN III

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