Efficacy of vinblastine in central nervous system Langerhans cell histiocytosis: a nationwide retrospective study.

Abstract

BACKGROUND:

Vinblastine (VBL) is the standard treatment for systemic Langerhans cell histiocytosis (LCH), but little is known about its efficacy in central nervous system (CNS) mass lesions.

METHODS:

A retrospective chart review was conducted. Twenty patients from the French LCH Study Group register met the inclusion criteria. In brief, they had CNS mass lesions, had been treated with VBL, and were evaluable for radiologic response.

RESULTS:

The median age at diagnosis of LCH was 11.5 years (range: 1-50). Intravenous VBL 6 mg/m2 was given in a 6-week induction treatment, followed by a maintenance treatment. The median total duration was 12 months (range: 3-30). Eleven patients received steroids concomitantly. Fifteen patients achieved an objective response; five had a complete response (CR: 25%), ten had a partial response (PR: 50%), four had stable disease (SD: 20%) and one patient progressed (PD: 5%). Of interest, four out of the six patients who received VBL without concomitant steroids achieved an objective response. With a median follow-up of 6.8 years, the 5-year event-free and overall survival was 61% and 84%, respectively. VBL was well-tolerated and there were no patient withdrawals due to adverse events.

CONCLUSION:

VBL, with or without steroids, could potentially be a useful therapeutic option in LCH with CNS mass lesions, especially for those with inoperable lesions or multiple lesions. Prospective clinical trials are warranted for the evaluation of VBL in this indication.

• Authors: Sophie Ng Wing Tin, Nadine Martin-Duverneuil, Ahmed Idbaih, Catherine Garel, Maria Ribeiro, Judith Landman Parker, Anne-Sophie Defachelles, Anne Lambilliotte, Mohamed Barkaoui, Martine Munzer, Martine Gardembas, Jean Sibilia, Patrick Lutz, Renato Fior, Michel Polak, Alain Robert, Olivier Aumaitre, Dominique Plantaz, Corinne Armari-Alla, Thierry Genereau, Perrine Marec Berard, Ghislain Nokam Talom, Jean-Loup Pennaforte, Hubert Ducou Le Pointe, Marie-Anne Barthez, Gérard Couillault, Julien Haroche, Karima Mokhtari, Jean Donadieu, Khê Hoang-Xuan, French LCH study group
• Journal: Orphanet journal of rare diseases (Orphanet J Rare Dis) Vol. 6 Pg. 83 (Dec 12 2011) ISSN: 1750-1172 [Electronic] England
• PMID: 22151964 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)

Chemical References

• Antineoplastic Agents, Phytogenic
• Steroids
• Vinblastine

Topics

• Adolescent
• Adult
• Antineoplastic Agents, Phytogenic (therapeutic use)
• Central Nervous System (drug effects, pathology)
• Central Nervous System Diseases (drug therapy, mortality)
• Child
• Child, Preschool
• Female
• Histiocytosis, Langerhans-Cell (drug therapy, mortality)
• Humans
• Infant
• Male
• Middle Aged
• Retrospective Studies
• Steroids (therapeutic use)
• Treatment Outcome
• Vinblastine (therapeutic use)
• Young Adult