Clinical and therapeutic aspects of childhood narcolepsy-cataplexy : a retrospective study of 51 children
STUDY OBJECTIVE: to report on symptoms and therapies used in childhood narcolepsy-cataplexy.
DESIGN, PATIENTS, AND SETTING: retrospective series of 51 children who completed the Stanford Sleep Inventory. HLA-DQB1*0602 typing (all tested, and 100% positive), polysomnography or Multiple Sleep Latency Test (76%), and cerebrospinal fluid hypocretin-1 measurements (26%, all with low levels) were also conducted. Prospective data on medication response was collected in 78% using a specially designed questionnaire.
MEASUREMENTS AND RESULTS: patients were separated into children with onset of narcolepsy prior to (53%), around (29%), and after (18%) puberty. None of the children had secondary narcolepsy. Clinical features were similar across puberty groups, except for sleep paralysis, which increased in frequency with age. Common features included excessive weight gain (84% ≥ 4 kg within 6 months of onset of narcolepsy) and earlier puberty (when compared with family members), notably in subjects who gained the most weight. Streptococcus-positive throat infections were reported in 20% of cases within 6 months of onset of narcolepsy. Polysomnographic features were similar across groups, but 3 prepubertal children did not meet Multiple Sleep Latency Test diagnostic criteria. Regarding treatment, the most used and continued medications were modafinil (84% continued), sodium oxybate (79%), and venlafaxine (68%). Drugs such as methylphenidate, tricyclic antidepressants, or selective serotonin reuptake inhibitors were often tried but rarely continued. Modafinil was reported to be effective for treating sleepiness, venlafaxine for cataplexy, and sodium oxybate for all symptoms, across all puberty groups. At the conclusion of the study, half of children with prepubertal onset of narcolepsy were treated "off label" with sodium oxybate alone or with the addition of one other compound. In older children, however, most patients needed more than 2 drugs.
CONCLUSION: this study reports on the clinical features of childhood narcolepsy and documents the safe use of treatments commonly used in adults in young children.
| Medienart: |
Artikel |
|---|
| Erscheinungsjahr: |
2010 |
|---|---|
| Erschienen: |
2010 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:33 |
|---|---|
| Enthalten in: |
Sleep - 33(2010), 11 vom: 01. Nov., Seite 1457-64 |
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
Aran, Adi [VerfasserIn] |
|---|
| Anmerkungen: |
Date Completed 10.01.2011 Date Revised 30.03.2022 published: Print Citation Status MEDLINE |
|---|
| Förderinstitution / Projekttitel: |
|
|---|
| PPN (Katalog-ID): |
NLM203842723 |
|---|
| LEADER | 01000naa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM203842723 | ||
| 003 | DE-627 | ||
| 005 | 20231223230908.0 | ||
| 007 | tu | ||
| 008 | 231223s2010 xx ||||| 00| ||eng c | ||
| 028 | 5 | 2 | |a pubmed24n0679.xml |
| 035 | |a (DE-627)NLM203842723 | ||
| 035 | |a (NLM)21102987 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a Aran, Adi |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Clinical and therapeutic aspects of childhood narcolepsy-cataplexy |b a retrospective study of 51 children |
| 264 | 1 | |c 2010 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ohne Hilfsmittel zu benutzen |b n |2 rdamedia | ||
| 338 | |a Band |b nc |2 rdacarrier | ||
| 500 | |a Date Completed 10.01.2011 | ||
| 500 | |a Date Revised 30.03.2022 | ||
| 500 | |a published: Print | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a STUDY OBJECTIVE: to report on symptoms and therapies used in childhood narcolepsy-cataplexy | ||
| 520 | |a DESIGN, PATIENTS, AND SETTING: retrospective series of 51 children who completed the Stanford Sleep Inventory. HLA-DQB1*0602 typing (all tested, and 100% positive), polysomnography or Multiple Sleep Latency Test (76%), and cerebrospinal fluid hypocretin-1 measurements (26%, all with low levels) were also conducted. Prospective data on medication response was collected in 78% using a specially designed questionnaire | ||
| 520 | |a MEASUREMENTS AND RESULTS: patients were separated into children with onset of narcolepsy prior to (53%), around (29%), and after (18%) puberty. None of the children had secondary narcolepsy. Clinical features were similar across puberty groups, except for sleep paralysis, which increased in frequency with age. Common features included excessive weight gain (84% ≥ 4 kg within 6 months of onset of narcolepsy) and earlier puberty (when compared with family members), notably in subjects who gained the most weight. Streptococcus-positive throat infections were reported in 20% of cases within 6 months of onset of narcolepsy. Polysomnographic features were similar across groups, but 3 prepubertal children did not meet Multiple Sleep Latency Test diagnostic criteria. Regarding treatment, the most used and continued medications were modafinil (84% continued), sodium oxybate (79%), and venlafaxine (68%). Drugs such as methylphenidate, tricyclic antidepressants, or selective serotonin reuptake inhibitors were often tried but rarely continued. Modafinil was reported to be effective for treating sleepiness, venlafaxine for cataplexy, and sodium oxybate for all symptoms, across all puberty groups. At the conclusion of the study, half of children with prepubertal onset of narcolepsy were treated "off label" with sodium oxybate alone or with the addition of one other compound. In older children, however, most patients needed more than 2 drugs | ||
| 520 | |a CONCLUSION: this study reports on the clinical features of childhood narcolepsy and documents the safe use of treatments commonly used in adults in young children | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Research Support, Non-U.S. Gov't | |
| 650 | 4 | |a DQB1*0602 | |
| 650 | 4 | |a HLA | |
| 650 | 4 | |a MSLT | |
| 650 | 4 | |a Narcolepsy | |
| 650 | 4 | |a cataplexy | |
| 650 | 4 | |a childhood | |
| 650 | 4 | |a hypocretin | |
| 650 | 4 | |a modafinil | |
| 650 | 4 | |a orexin | |
| 650 | 4 | |a sodium oxybate | |
| 650 | 4 | |a venlafaxine | |
| 650 | 7 | |a Adjuvants, Anesthesia |2 NLM | |
| 650 | 7 | |a Antidepressive Agents, Second-Generation |2 NLM | |
| 650 | 7 | |a Benzhydryl Compounds |2 NLM | |
| 650 | 7 | |a Central Nervous System Stimulants |2 NLM | |
| 650 | 7 | |a Cyclohexanols |2 NLM | |
| 650 | 7 | |a Drosophila Proteins |2 NLM | |
| 650 | 7 | |a Nerve Tissue Proteins |2 NLM | |
| 650 | 7 | |a Nuclear Proteins |2 NLM | |
| 650 | 7 | |a Transcription Factors |2 NLM | |
| 650 | 7 | |a pros protein, Drosophila |2 NLM | |
| 650 | 7 | |a Venlafaxine Hydrochloride |2 NLM | |
| 650 | 7 | |a 7D7RX5A8MO |2 NLM | |
| 650 | 7 | |a Sodium Oxybate |2 NLM | |
| 650 | 7 | |a 7G33012534 |2 NLM | |
| 650 | 7 | |a Modafinil |2 NLM | |
| 650 | 7 | |a R3UK8X3U3D |2 NLM | |
| 700 | 1 | |a Einen, Mali |e verfasserin |4 aut | |
| 700 | 1 | |a Lin, Ling |e verfasserin |4 aut | |
| 700 | 1 | |a Plazzi, Guiseppe |e verfasserin |4 aut | |
| 700 | 1 | |a Nishino, Seiji |e verfasserin |4 aut | |
| 700 | 1 | |a Mignot, Emmanuel |e verfasserin |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t Sleep |d 1986 |g 33(2010), 11 vom: 01. Nov., Seite 1457-64 |w (DE-627)NLM00225574X |x 1550-9109 |7 nnns |
| 773 | 1 | 8 | |g volume:33 |g year:2010 |g number:11 |g day:01 |g month:11 |g pages:1457-64 |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |d 33 |j 2010 |e 11 |b 01 |c 11 |h 1457-64 | ||