Treatment of children with refractory/relapse high risk Langerhans cell histiocytosis with the combination of cytarabine, vindesine and prednisone

Abstract

Abstract Background: The patients with multisystem and risk organ involvement Langerhans cell histiocytosis (MS-RO+ LCH) have poor prognosis. The patients with MS-RO+ LCH who failed front-line therapy have a high mortality rate and the standard salvage treatment has not been established. The combination of cytarabine (Ara-c), vincristine (VCR) and prednisone might be effective in refractory/relapse MS-RO+ LCH, with low toxicity. Methods: We retrospectively analyzed pediatric refractory/relapse MS-RO+ LCH patients treated with the low-dose Ara-c (100mg/m2/d×5days) or high-dose Ara-c (500mg/m2/d×5days) combined with vindesine (VDS) and prednisone in a single center. The efficacy, long term outcomes and adverse events were analyzed. Results: From January 2013 to December 2016, 13 patients receiving the low-dose Ara-c chemotherapy (LAC) and 7 patients receiving the high-dose Ara-c chemotherapy (HAC) were enrolled in the study. 11 (84.64%) of the 13 patients treated with the LAC regimen and 6 (85.71%) of the 7 patients treated with the HAC regimen had response after four courses of the therapy. All patients in the study were alive during follow-up, and the 5-year event-free survival rate (EFS) was 42.98% and 85.71% in the LAC and HAC groups. The most frequent adverse event was Grade 1/2 myelosuppression, which was observed in 38.46% (5/13) and 42.86% (3/7) of patients who received the LAC and HAC regimen. Conclusions: A combination of Ara-c, VDS and prednisone might be effective as a salvage treatment in some patients with refractory/relapse MS-RO+ LCH , with low toxicity and 5-year overall survival rate of 100%. The high-dose Ara-c regimen was associated with numerically higher EFS rate.