Efficacy and Safety of Low-Dose Cytarabine Combined With Thalidomide in Adult Patients With Untreated Langerhans Cell Histiocytosis: A Prospective, Multicenter, Single-Arm Study

What this Trial Is About

This research aims to evaluate the effectiveness and safety of a treatment combining low-dose cytarabine with thalidomide for adults newly diagnosed with Langerhans Cell Histiocytosis (LCH), a rare disease affecting multiple or single systems with multiple lesions. The study focuses on patients who have not received prior systemic treatment and seeks to understand how this treatment impacts event-free survival over a three-year period. Participants will receive cytarabine at 100 mg/m² daily from days 1 to 5 combined with thalidomide 100 mg daily from days 1 to 35, repeated every 35 days for 12 cycles. After this initial phase, participants will continue with thalidomide alone at 100 mg daily from days 1 to 28 every 28 days for an additional 12 cycles, totaling 24 treatment cycles. The study is a multicenter, single-arm trial assessing this combined and maintenance therapy in adults. Throughout the study, participants will be monitored for event-free survival as the primary outcome, along with overall survival, overall response rate, and adverse events up to three years from enrollment. Safety and treatment effects will be carefully tracked, and patient assessments will include clinical evaluations and laboratory tests during treatment and follow-up. The total duration of participation covers the treatment cycles and extends to three years for outcome measurement.

Efficacy and Safety of Low-Dose Cytarabine Combined With Thalidomide in Adult Patients With Untreated LCH