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Volume 13 No. 03
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Scientific Investigations

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

http://dx.doi.org/10.5664/jcsm.6500

Michel Lecendreux, MD1,2; Johanna Berthier, Medical Student3; Jennifer Corny, PharmD4; Olivier Bourdon, MD, PhD4,5; Claire Dossier, MD6; Christophe Delclaux, MD, PhD1,7
1AP-HP, Pediatric Sleep Center, Hospital Robert-Debré, Paris, France; 2National Reference Centre for Orphan Diseases, Narcolepsy, Idiopathic Hypersomnia and Kleine-Levin Syndrome (CNR Narcolepsie-Hypersomnie), Paris, France; 3Carol Davila University of Medicine and Pharmacy, Bucharest, Romania; 4Pharmacy Department, Hospital Robert-Debré, Paris, France; 5Pharmacy Faculty, Paris Descartes, Paris, France; 6Pediatric Nephrology Department, Hospital Robert-Debré, Paris, France; 7Paris Diderot University, Sorbonne Paris Cité, Paris, France

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.

Citation:

Lecendreux M, Berthier J, Corny J, Bourdon O, Dossier C, Delclaux C. Intravenous immunoglobulin therapy in pediatric narcolepsy: a nonrandomized, open-label, controlled, longitudinal observational study. J Clin Sleep Med. 2017;13(3):441–453.




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