title: Blastic plasmacytoid dendritic cell neoplasm frequently shows occult central nervous system involvement at diagnosis and benefits from intrathecal therapy.
abstract: Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare aggressive myeloid neoplasm which shows a high rate of central nervous system (CNS) recurrence and overall survival (OS) of <1 year. Despite this, screening for CNS involvement is not routinely performed at diagnosis and intrathecal (IT) prophylaxis is not regularly administered in BPDCN. Here, we prospectively evaluated 13 consecutive BPDCN patients for the presence of CNS involvement by flow cytometry. Despite none of the patients presented with neurological symptoms, occult CNS involvement was detected in 6/10 cases evaluated at diagnosis and 3/3 studied at relapse/progression. BPDCN patients evaluated at diagnosis received IT treatment -either CNS prophylaxis (n = 4) or active therapy (n = 6)- and all but one remain alive (median follow-up of 20 months). In contrast, all three patients assessed at relapse/progression died. The potential benefit of IT treatment administered early at diagnosis on OS and CNS recurrence-free survival of BPDCN was further confirmed in a retrospective cohort of another 23 BPDCN patients. Our results show that BPDCN patients studied at diagnosis frequently display occult CNS involvement; moreover, they also indicate that treatment of occult CNS disease might lead to a dramatically improved outcome of BPDCN.
authors: Cancer Research Centre (IBMCC, USAL-CSIC), Institute for Biomedical Research of Salamanca (IBSAL) and Department of Medicine and Cytometry Service (NUCLEUS Research Support Platform), University of Salamanca (USAL), Salamanca, Spain.
authors: Lourdes
authors: L
authors: Martín-Martín
authors: Cancer Research Centre (IBMCC, USAL-CSIC), Institute for Biomedical Research of Salamanca (IBSAL) and Department of Medicine and Cytometry Service (NUCLEUS Research Support Platform), University of Salamanca (USAL), Salamanca, Spain.
authors: Julia
authors: J
authors: Almeida
authors: Hematology Department, Institut Catalá d'Oncologia, Hospital Duran i Reynals, University of Barcelona, Institut d'Investigació Biomèdica de Bellvitge, Barcelona, Spain.
authors: Helena
authors: H
authors: Pomares
authors: Hematology Department, Institut Catalá d'Oncologia, Hospital Duran i Reynals, University of Barcelona, Institut d'Investigació Biomèdica de Bellvitge, Barcelona, Spain.
authors: Eva
authors: E
authors: González-Barca
authors: Hematology Department, University Hospital of Fuenlabrada, Madrid, Spain.
authors: Pilar
authors: P
authors: Bravo
authors: Hematology Department, University Hospital Joan XXIII, Tarragona, Spain.
authors: Teresa
authors: T
authors: Giménez
authors: Hematology Department, Infanta Leonor Hospital, Madrid, Spain.
authors: Cecilia
authors: C
authors: Heras
authors: Hematology Department, Hospital of Segovia, Segovia, Spain.
authors: José-Antonio
authors: JA
authors: Queizán
authors: Hematology Department, University Hospital Morales Meseguer, Murcia, Spain.
authors: Elena
authors: E
authors: Pérez-Ceballos
authors: Hematology Department, Hospital of León, León, Spain.
authors: Violeta
authors: V
authors: Martínez
authors: Hematology Department, University Hospital of Santiago, Santiago de Compostela, Spain.
authors: Natalia
authors: N
authors: Alonso
authors: Hematology Department, University Hospital Miguel Servet, Zaragoza, Spain.
authors: Carlota
authors: C
authors: Calvo
authors: Hematology Department, General Yagüe Hospital, Burgos, Spain.
authors: Rodolfo
authors: R
authors: Álvarez
authors: Hematology Department and IBSAL, University Hospital of Salamanca, Salamanca, Spain.
authors: María Dolores
authors: MD
authors: Caballero
authors: Cancer Research Centre (IBMCC, USAL-CSIC), Institute for Biomedical Research of Salamanca (IBSAL) and Department of Medicine and Cytometry Service (NUCLEUS Research Support Platform), University of Salamanca (USAL), Salamanca, Spain.
authors: Alberto
authors: A
authors: Orfao
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002490:Central Nervous System / Q000473:pathology*; D016543:Central Nervous System Neoplasms / Q000556:secondary* / Q000628:therapy*; D002648:Child; D003713:Dendritic Cells / Q000473:pathology*; D005260:Female; D019337:Hematologic Neoplasms / Q000401:mortality / Q000473:pathology* / Q000628:therapy*; D006801:Humans; D008297:Male; D008875:Middle Aged; D009364:Neoplasm Recurrence, Local; D011446:Prospective Studies; D012189:Retrospective Studies; D016896:Treatment Outcome; D055815:Young Adult
keywords: ALL therapy; blastic plasmacytoid dendritic cell neoplasm; central nervous system; flow cytometry; intrathecal prophylaxis