Neurodegenerative central nervous system Langerhans cell histiocytosis and coincident hydrocephalus treated with vincristine/cytosine arabinoside.
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.


METHODS
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.
Allen
Carl E
CE
Texas Children's Cancer Center/Hematology Service, Houston, TX 77030, USA.
Flores
Ricardo
R
Rauch
Ronald
R
Dauser
Robert
R
Murray
Jeffrey C
JC
Puccetti
Diane
D
Hsu
David A
DA
Sondel
Paul
P
Hetherington
Maxine
M
Goldman
Stan
S
McClain
Kenneth L
KL
D002493:Central Nervous System Diseases / Q000150:complications / Q000188:drug therapy*; D002648:Child; D002675:Child, Preschool; D003561:Cytarabine / Q000627:therapeutic use*; D006646:Histiocytosis, Langerhans-Cell / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D006849:Hydrocephalus / Q000150:complications / Q000188:drug therapy*; D007223:Infant; D008279:Magnetic Resonance Imaging; D008297:Male; D019636:Neurodegenerative Diseases / Q000150:complications / Q000188:drug therapy*; D012189:Retrospective Studies; D014750:Vincristine / Q000627:therapeutic use*
D003561:Cytarabine; D014750:Vincristine