Skip to main page content
Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2010 Mar;54(3):416-23.
doi: 10.1002/pbc.22326.

Neurodegenerative Central Nervous System Langerhans Cell Histiocytosis and Coincident Hydrocephalus Treated With Vincristine/Cytosine Arabinoside

Affiliations
Free PMC article

Neurodegenerative Central Nervous System Langerhans Cell Histiocytosis and Coincident Hydrocephalus Treated With Vincristine/Cytosine Arabinoside

Carl E Allen et al. Pediatr Blood Cancer. .
Free PMC article

Abstract

Background: Central nervous system (CNS) complications of Langerhans cell histiocytosis (LCH) include mass lesions and a neurodegenerative (ND) syndrome with ataxia, dysarthria, dysmetria, learning and behavior difficulties and/or characteristic changes on brain MRIs. Hydrocephalus has rarely been reported in LCH. LCH lesions of the orbit, mastoid and temporal bones ("CNS-Risk" lesions) and diabetes insipidus predispose patients to ND-CNS-LCH. Treatment options have been limited and only a case series using trans-retinoic acid (ATRA) and intravenous immunoglobulin (IVIG) have been published.

Methods: We have used cytosine arabinoside (ARA-C) with or without vincristine to treat eight patients with ND-CNS LCH.

Patients: Seven male children and one young adult male with clinical and radiologic ND-CNS-LCH were treated with a regimen of vincristine 1.5 mg/m(2) on day 1 and ARA-C 100 mg/m(2) daily for 5 days or ARA-C alone monthly for 4-19 months. Seven patients were evaluated with an ataxia rating scale (ARS) and all with serial MRIs of the brain.

Results: Five of seven patients had decreases in their ARS scores and/or decreased T2 hyperintense lesions on MRI images. Grade 2 neutropenia was the most frequent adverse event. Vincristine-associated neuropathy occurred in two patients. Hydrocephalus caused symptoms and signs that confounded the diagnosis and management of ND-CNS-LCH in all four patients affected with both.

Conclusions: Subtle changes in neurologic function may be complicated by hydrocephalus. Vcr/ARA-C or ARA-C were an effective therapies for some ND-CNS LCH patients. A clinical trial using this and possibly other modalities such as IVIG or ATRA should be done.

Conflict of interest statement

Conflict of Interest Statement. Dr. McClain owns shares of Johnson and Johnson common stock. None of the other authors have any conflicts of interest.

Figures

Fig. 1
Fig. 1
Fig. 2
Fig. 2
Handwriting samples of Patient 1. A. Before treatment with vincristine/cytosine arabinoside. B: After 2 months of treatment.
Fig. 3
Fig. 3
Fig. 4
Fig. 4
Fig. 5
Fig. 5
Fig. 6
Fig. 6
Fig. 7
Fig. 7

Comment in

  • VCR/AraC chemotherapy and ND-CNS-LCH.
    Imashuku S, Shioda Y, Tsunematsu Y, Imamura T, Morimoto A; Japan LCH Study Group. Imashuku S, et al. Pediatr Blood Cancer. 2010 Jul 15;55(1):215-6. doi: 10.1002/pbc.22478. Pediatr Blood Cancer. 2010. PMID: 20310001 No abstract available.

Similar articles

See all similar articles

Cited by 15 articles

See all "Cited by" articles

MeSH terms

Feedback