Unmet Clinical Needs and Management Recommendations for Blastic Plasmacytoid Dendritic Cell Neoplasm: A Consensus-based Position Paper From an Ad Hoc International Expert Panel
Livio Pagano,
Pier Luigi Zinzani,
Stefano Pileri,
Pietro Quaglino,
Branko Cuglievan,
Emilio Berti,
Naveen Pemmaraju,
Francesco Onida,
Rein Willemze,
Alberto Orfao,
Giovanni Barosi
Affiliations
Livio Pagano
1 Hematology Unit, Fondazione Policlinico Universitario Agostino Gemelli—IRCCS, Rome, Italy
Pier Luigi Zinzani
3 IRCCS Azienda Ospedaliero-Universitaria di Bologna, Istituto di Ematologia “Seràgnoli”, Bologna, Italy
Stefano Pileri
5 IEO—European Institute of Oncology IRCCS (Milan) and Bologna University School of Medicine, Italy
Pietro Quaglino
6 Dermatology Clinic, Department of Medical Sciences, Città Della Salute e Della Scienza of Turin, University of Turin, Italy
Branko Cuglievan
7 Division of Pediatrics and Patient Care, The University of Texas MD Anderson Cancer Center, Houston, TX, USA
Emilio Berti
8 Dermatology Unit, La Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy
Naveen Pemmaraju
10 Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX, USA
Francesco Onida
11 Università degli Studi di Milano, Italy
Rein Willemze
13 Department of Dermatology, Leiden University Medical Center, Leiden, The Netherlands
Alberto Orfao
14 Cancer Research Center (IBMCC, USAL/CSIC), Department of Medicine, Universidad de Salamanca, Biomedical Research Institute of Salamanca and Spanish Network on Mastocytosis (REMA), Salamanca, Spain
Giovanni Barosi
16 Center for the Study of Myelofibrosis, IRCCS Policlinico S. Matteo Foundation, Pavia, Italy
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a hematological malignancy characterized by recurrent skin nodules, an aggressive clinical course with rapid involvement of hematological organs, and a poor prognosis with overall survival. The rarity of the disease results in a few large-scale studies, a lack of controlled clinical trials for its management, and a lack of evidence-based guidelines. Here, we present a review of unmet clinical needs on the management of BPDCN by a panel of eleven experts involved in the research and clinical practice of BPDCN. Recommendations and proposals were achieved by multiple-step formalized procedures to reach a consensus after a comprehensive analysis of the scientific literature. The panel analyzed the critical issues of diagnostic pathway, prognostic stratification, therapy for young and fit patients and elderly and unfit patients, indication for allotransplant and for autotransplant, indication for central nervous system prophylaxis, and management of pediatric BPDCN patients. For each of these issues, consensus opinions were provided and, when appropriate, proposals for advancement in clinical practice were addressed. The hope is that this comprehensive overview will serve to improve the practice of BPDCN and inform the design and implementation of new studies in the field.
