title: High efficacy of the MACOP-B regimen in the treatment of adult Langerhans cell histiocytosis, a 20 year experience.
abstract: BACKGROUND
  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. 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.
  
  
  METHODS
  Clinical data of all adult LCH patients (n = 17) diagnosed and treated at our Institution during the past 20-year period were retrospectively reviewed.
  
  
  RESULTS
  A 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.
  
  
  CONCLUSIONS
  These 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.
authors.affiliation: Department of Experimental, Diagnostic and Specialty Medicine- DIMES, Institute of Hematology and Medical Oncology L.A. Seragnoli, University of Bologna, Via Massarenti 9, 40138, Bologna, Italy. ederenzini@gmail.com.
authors.forename: Enrico
authors.initials: E
authors.lastname: Derenzini
authors.affiliation: Department of Experimental, Diagnostic and Specialty Medicine- DIMES, Institute of Hematology and Medical Oncology L.A. Seragnoli, University of Bologna, Via Massarenti 9, 40138, Bologna, Italy. vittorio.stefoni2@unibo.it.
authors.forename: Vittorio
authors.initials: V
authors.lastname: Stefoni
authors.affiliation: Department of Experimental, Diagnostic and Specialty Medicine- DIMES, Institute of Hematology and Medical Oncology L.A. Seragnoli, University of Bologna, Via Massarenti 9, 40138, Bologna, Italy. c.pellegrini@live.it.
authors.forename: Cinzia
authors.initials: C
authors.lastname: Pellegrini
authors.affiliation: Department of Experimental, Diagnostic and Specialty Medicine- DIMES, Institute of Hematology and Medical Oncology L.A. Seragnoli, University of Bologna, Via Massarenti 9, 40138, Bologna, Italy. letizia.gandolfi83@gmail.com.
authors.forename: Letizia
authors.initials: L
authors.lastname: Gandolfi
authors.affiliation: Department of Experimental, Diagnostic and Specialty Medicine- DIMES, Institute of Hematology and Medical Oncology L.A. Seragnoli, University of Bologna, Via Massarenti 9, 40138, Bologna, Italy. alessandro.broccoli@studio.unibo.it.
authors.forename: Alessandro
authors.initials: A
authors.lastname: Broccoli
authors.affiliation: Department of Experimental, Diagnostic and Specialty Medicine- DIMES, Institute of Hematology and Medical Oncology L.A. Seragnoli, University of Bologna, Via Massarenti 9, 40138, Bologna, Italy. bea.casadei@gmail.com.
authors.forename: Beatrice
authors.initials: B
authors.lastname: Casadei
authors.affiliation: Department of Experimental, Diagnostic and Specialty Medicine- DIMES, Institute of Hematology and Medical Oncology L.A. Seragnoli, University of Bologna, Via Massarenti 9, 40138, Bologna, Italy. federica.quirini@gmail.com.
authors.forename: Federica
authors.initials: F
authors.lastname: Quirini
authors.affiliation: Department of Experimental, Diagnostic and Specialty Medicine- DIMES, Institute of Hematology and Medical Oncology L.A. Seragnoli, University of Bologna, Via Massarenti 9, 40138, Bologna, Italy. lisa.argnani@unibo.it.
authors.forename: Lisa
authors.initials: L
authors.lastname: Argnani
authors.affiliation: Department of Experimental, Diagnostic and Specialty Medicine- DIMES, Institute of Hematology and Medical Oncology L.A. Seragnoli, University of Bologna, Via Massarenti 9, 40138, Bologna, Italy. lorenzo.tonialini@studio.unibo.it.
authors.forename: Lorenzo
authors.initials: L
authors.lastname: Tonialini
authors.affiliation: Department of Experimental, Diagnostic and Specialty Medicine- DIMES, Institute of Hematology and Medical Oncology L.A. Seragnoli, University of Bologna, Via Massarenti 9, 40138, Bologna, Italy. pierluigi.zinzani@unibo.it.
authors.forename: Pier Luigi
authors.initials: PL
authors.lastname: Zinzani
mesh_terms: D000293:Adolescent; D000328:Adult; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage*; D001761:Bleomycin / Q000008:administration & dosage; D003520:Cyclophosphamide / Q000008:administration & dosage; D018572:Disease-Free Survival; D004317:Doxorubicin / Q000008:administration & dosage; D005260:Female; D005500:Follow-Up Studies; D006646:Histiocytosis, Langerhans-Cell / Q000188:drug therapy* / Q000453:epidemiology* / Q000473:pathology; D006801:Humans; D008297:Male; D008727:Methotrexate / Q000008:administration & dosage; D008875:Middle Aged; D011239:Prednisolone / Q000008:administration & dosage; D012074:Remission Induction; D014750:Vincristine / Q000008:administration & dosage
chemical_list: D001761:Bleomycin; D014750:Vincristine; D004317:Doxorubicin; D003520:Cyclophosphamide; D011239:Prednisolone; D008727:Methotrexate