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Clinical Trial
. 2004 Jul;43(1):55-8.
doi: 10.1002/pbc.20040.

Retinoic Acid Therapy in "Degenerative-Like" Neuro-Langerhans Cell Histiocytosis: A Prospective Pilot Study

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Clinical Trial

Retinoic Acid Therapy in "Degenerative-Like" Neuro-Langerhans Cell Histiocytosis: A Prospective Pilot Study

Ahmed Idbaih et al. Pediatr Blood Cancer. .

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.

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