Abstract
BackgroundAdult Langerhans cell histiocytosis (LCH) is an orphan disease. Chemotherapy is usually reserved to patients presenting with single system multifocal (SS-m) or multisystem (MS) disease but due to the lack of randomized studies no standard first line therapy has been defined yet. Pediatric regimens based on the vinblastine/prednisone backbone are not well tolerated in adults and probably less effective. We previously demonstrated high efficacy of the dose dense polichemotherapy regimen MACOP-B in 7 adult patients with SS-m or MS-LCH, in terms of high response rate and durable responses. Here we report an update of these data with the purpose of evaluating the long term efficacy of MACOP-B in adult LCH.MethodsClinical data of all adult LCH patients (n = 17) diagnosed and treated at our Institution during the past 20-year period were retrospectively reviewed.ResultsA total of 11 patients (6 with SS-m and 5 with MS-LCH) were treated with MACOP-B from 1995 to 2014. The overall response rate was confirmed to be 100 %, with a complete response of 73 % and a partial response rate of 27 %. Overall progression free survival was 64 %, and disease free survival after achievement of initial CR was 87 %. Overall survival rate was 82 % after 6.7 years of median follow-up.ConclusionsThese data confirm high activity of MACOP-B in adult LCH, indicating that a substantial fraction of patients achieve long lasting responses and can be cured with this therapeutic approach.
Highlights
Adult Langerhans cell histiocytosis (LCH) is an orphan disease
We previously described our experience with the dose dense short course polichemotherapy regimen MACOP-B (Methotrexate, Doxorubicin, Cyclophosphamide, Vincristine, Bleomycin, Prednisone) in 7 adult patients affected by system multifocal (SS-m) or MS LCH [9], demonstrating high activity in terms of overall response rate (ORR) and long term disease control, despite the lack of maintenance therapy and an overall treatment duration of only 3 months
One patient with pulmonary LCH who relapsed after prior vinblastine/prednisone was monitored with a wait and see approach after smoking cessation
Summary
Adult Langerhans cell histiocytosis (LCH) is an orphan disease. Chemotherapy is usually reserved to patients presenting with single system multifocal (SS-m) or multisystem (MS) disease but due to the lack of randomized studies no standard first line therapy has been defined yet. Derenzini et al BMC Cancer (2015) 15:879 been made in the treatment of pediatric LCH, this therapeutic strategy is unlikely to be successful in adults, as the only prospective randomized trial evaluating the efficacy of vinblastine/prednisone regimen in adults (LCHA1 trial) was prematurely closed for unacceptable toxicities (vinblastine related neurotoxicity and detrimental effects of prolonged steroid therapy) [3]. Available data do not support the use of pediatric regimens in adult LCH, indicating that the concept of mild induction followed by maintenance therapy probably cannot be translated to adults. Alternative approaches tested in adult LCH include nucleoside analogs such as cytarabine and cladribine (2-CDA) [6, 8] but no consensus on the best frontline treatment strategy has been reached yet
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