Intravenous Immunoglobulin Therapy in Pediatric Narcolepsy: A Nonrandomized, Open-Label, Controlled, Longitudinal Observational Study.

Abstract

STUDY OBJECTIVES:

Previous case reports of intravenous immunoglobulins (IVIg) in pediatric narcolepsy have shown contradictory results.

METHODS:

This was a nonrandomized, open-label, controlled, longitudinal observational study of IVIg use in pediatric narcolepsy with retrospective data collection from medical files obtained from a single pediatric national reference center for the treatment of narcolepsy in France. Of 56 consecutively referred patients with narcolepsy, 24 received IVIg (3 infusions administered at 1-mo intervals) in addition to standard care (psychostimulants and/or anticataplectic agents), and 32 continued on standard care alone (controls).

RESULTS:

For two patients in each group, medical files were unavailable. Of the 22 IVIg patients, all had cerebrospinal fluid (CSF) hypocretin ≤ 110 pg/mL and were HLA-DQB1*06:02 positive. Of the 30 control patients, 29 were HLA-DQB1*06:02 positive and of those with available CSF measurements, all 12 had hypocretin ≤ 110 pg/mL. Compared with control patients, IVIg patients had shorter disease duration, shorter latency to sleep onset, and more had received H1N1 vaccination. Mean (standard deviation) follow-up length was 2.4 (1.1) y in the IVIg group and 3.9 (1.7) y in controls. In multivariate-adjusted linear mixed-effects analyses of change from baseline in Ullanlinna Narcolepsy Scale (UNS) scores, high baseline UNS, but not IVIg treatment, was associated with a reduction in narcolepsy symptoms. On time-to-event analysis, among patients with high baseline UNS scores, control patients achieved a UNS score < 14 (indicating remission) less rapidly than IVIg patients (adjusted hazard ratio 0.18; 95% confidence interval: 95% confidence interval: 0.03, 0.95; p = 0.043). Shorter or longer disease duration did not influence treatment response in any analysis.

CONCLUSIONS:

Overall, narcolepsy symptoms were not significantly reduced by IVIg. However, in patients with high baseline symptoms, a subset of IVIg-treated patients achieved remission more rapidly than control patients.

COMMENTARY:

A commentary on this article appears in this issue on page 363.

• Authors: Michel Lecendreux, Johanna Berthier, Jennifer Corny, Olivier Bourdon, Claire Dossier, Christophe Delclaux
• Journal: Journal of clinical sleep medicine : JCSM : official publication of the American Academy of Sleep Medicine (J Clin Sleep Med) Vol. 13 Issue 3 Pg. 441-453 (Mar 15 2017) ISSN: 1550-9397 [Electronic] United States
• PMID: 28095967 (Publication Type: Journal Article, Observational Study)
• Copyright: © 2017 American Academy of Sleep Medicine

Chemical References

• Immunoglobulins, Intravenous

Topics

• Child
• Female
• France
• Humans
• Immunoglobulins, Intravenous (therapeutic use)
• Longitudinal Studies
• Male
• Narcolepsy (drug therapy)
• Retrospective Studies
• Treatment Outcome