Retinoic acid therapy in "degenerative-like" neuro-langerhans cell histiocytosis: a prospective pilot study.

Abstract:

BACKGROUND:Degenerative-like neuro-Langerhans cell histiocytosis (DN-LCH) is a rare complication of LCH marked by progressive cerebellar ataxia. No treatment has so far been shown to slow this progression. PROCEDURE:All-trans retinoic acid (ATRA) was administered orally at a dose of 45 mg/m(2) daily for 6 weeks and then 2 weeks every month for 1 year. The endpoints were clinical status at 1 year (assessed with rating scales for ataxia and disability), adverse effects, and changes in neurological abnormalities on MRI. RESULTS:Ten patients were studied. The treatment was well tolerated. All the patients were clinically stable at the end of the study. No MRI changes were noted. CONCLUSIONS:DN-LCH appeared to be stable during ATRA therapy, but further studies are required to appreciate the possible long-term benefits of ATRA.

journal_name

Pediatr Blood Cancer

journal_title

Pediatric blood & cancer

authors

Idbaih A,Donadieu J,Barthez MA,Geissmann F,Bertrand Y,Hermine O,Brugières L,Genereau T,Thomas C,Hoang-Xuan K

doi

10.1002/pbc.20040

keywords:

subject

Has Abstract

pub_date

2004-07-01 00:00:00

pages

55-8

issue

1

eissn

1545-5009

issn

1545-5017

journal_volume

43

pub_type

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