Treatment of neurodegenerative CNS disease in Langerhans cell histiocytosis with a combination of intravenous immunoglobulin and chemotherapy

Abstract

The IVIG-containing treatment may be promising for ND-CNS-LCH; however, its effectiveness remains to be further tested in more patients as well as in a randomized trial.

Management of Langerhans Cell Histiocytosis (LCH)-Induced Central Diabetes Insipidus and Its Associated Endocrinological/Neurological Sequelae

“…Established ND disease is very difficult to treat. Although several reports have suggested that all-trans retinoic acid or a combination of vincristine/AraC and intravenous high dose gamma-globulin (IVIG) may have some efficacy (35-37), it remains unclear whether any specific type of initial systemic chemotherapy for multifocal LCH patients can limit the later occurrence of ND disease (38). There is an urgent need for research that can identify an innovative therapy for such cases.…”

Neurodegenerative central nervous system disease as late sequelae of Langerhans cell histiocytosis. Report from the Japan LCH Study Group

“…18 The usefulness of brain MRI, particularly T2W1 and FLAIR imaging, to detect the strong signals at the cerebellar dentate nuclei, as well as severe cerebellar atrophy, has been well established in the diagnosis of ND-CNS-LCH disease. [7][8][9][10][11][12] In this series, only 5 patients had a chance to be diagnosed early from characteristic brain MRI findings prior to the development of neurological symptoms (Group 1). Since the remaining 6 patients in Group 2 were not examined by MRI in their follow-up until when neurological symptoms were noted, any comparison between these two groups is problematic.…”

“…With this aim, we previously reported therapeutic results with a combination therapy of IVIG and chemotherapy in pediatric ND-CNS-LCH patients, 12 since LCH CNS lesions share some common features with the pathogenesis of multiple sclerosis, and the efficacy of IVIG therapy has been reported in patients with multiple sclerosis. 19,20 Extended observation observed a trend for the delayed progression of the disease in the IVIG treated patients as seen from the change in the EDSS scores.…”

“…[7][8][9][10][11] The diagnosis of ND-CNS-LCH disease is made by characteristic brain MRI findings and/or symptoms of cerebral or cerebellar neurologic dysfunction. [7][8][9][10][11][12] The long-term outcome of ND-CNS-LCH disease is bleak. However, longitudinal follow-up studies from the initial LCH episodes in such cases have rarely been reported.…”

Endocrine manifestations of Langerhans cell histiocytosis diagnosed in adults

“…Appropriate therapy is selected based on the extent and site of disease involvement [5], among several options, such as conservative therapy (follow-up, topical steroids, hormone-replacement or local excision), radiation therapy, chemotherapy, anti-CD1a monoclonal antibodies and/or organ or stem cell transplantation [4,7,22,23].…”