Retinoic acid therapy in "degenerative-like" neuro-Langerhans cell histiocytosis:: A prospective pilot study

被引：43

作者：

Idbaih, A

Donadieu, J

Barthez, MA

Geissmann, F

Bertrand, Y

Hermine, O

Brugières, L

Genereau, T

Thomas, C

Hoang-Xuan, K

机构：

[1] Grp Hosp Pitie Salpetriere, Federat Neurol Mazarim, F-75634 Paris, France

[2] Hop Trousseau, F-75571 Paris, France

[3] CHU Tours, Serv Neurol Pediat, Tours, France

[4] CHU Necker, Lab Anatomopathol, Paris, France

[5] Hop Debrousse, Serv Hematol Infantile, Lyon, France

[6] CHU Necker, Serv Hematol, Paris, France

[7] Inst Gustave Roussy, Serv Pediat, Villejuif, France

[8] CHU St Antoine, Serv Med Interne, Paris, France

[9] CHU Nantes, Serv Hematol Oncol Pediat, Nantes, France

来源：

PEDIATRIC BLOOD & CANCER | 2004年 / 43卷 / 01期

关键词：

central nervous system; cerebellar ataxia; Langerhans' cell histiocytosis; retinoic acid; treatment;

D O I：

10.1002/pbc.20040

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

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. (C) 2004 Wiley-Liss, Inc.

引用

页码：55 / 58

页数：4

共 12 条

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