LCH-S-2005

LCH-S-2005 (2005-2010)

This open-label, prospective, non-randomized, phase 2 trial, was based on the promising results of a previous pilot trial of the French LCH Study Group. The prescribed treatment consisted of at least two 5-day courses of cytarabine (Ara-C) 1g/m²/day plus cladribine (2-CdA) 9mg/m²/day, followed by maintenance therapy. The primary end-point of this salvage trial was the response rate after two Ara-C/2-CdA courses.⁸ Twenty-seven patients with MS-LCH, risk organ involvement and resistant to at least 1 course of steroids and vinblastine, were enrolled on this trial. The overall response rate after two courses was 92% (7% non-active disease and 85% active disease, better). The study achieved an excellent 5-year survival of 85%, but the toxicity of the regimen was very high (at least 2/4 deaths were due to treatment toxicity) and required highest level of supportive therapy.¹

^REFERENCES:

1. Donadieu J, Bernard F, van Noesel M, Barkaoui M, Bardet O, Mura R, et al. Cladribine and cytarabine in refractory multisystem Langerhans cell histiocytosis: results of an international phase 2 study. Blood, 2015, 126(12):1415-23.