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.
Highlights
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare disease -i.e. < 1% acute myeloblastic leukemia cases (AML)- with a historically dismal prognosis
Despite most BPDCN patients treated with different intensive chemotherapy regimens (e.g. CHOP, AML-type and acute lymphoblastic leukemia (ALL)-type regimens) achieve complete remission (CR), the great majority relapse early after CR, with further resistance to therapy and a fatal outcome
Cerebrospinal fluid (CSF) samples positive for tumor plasmacytoid dendritic cells were found in 6/10 (60%) cases studied at diagnosis –median of 11 tumor cells/μl, representing 82% of the total cerebrospinal fluid (CSF) cellularity- in association with ≥20% bone marrow (BM) infiltration by tumor cells (6/6 vs. 1/4 cases, respectively; p=.03)
Summary
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare disease -i.e. < 1% acute myeloblastic leukemia cases (AML)- with a historically dismal prognosis. BPDCN prognosis is extremely poor, patients treated with ALL-type regimens that include CNS prophylaxis [1,2,3] and/or allogeneic hematopoietic stem cell transplantation (AHSCT) [7, 8] appear to show a better outcome. These findings suggest that occult CNS involvement might be already present at diagnosis in a significant fraction of BPDCN patients, and could contribute to explain the relatively high number of CNS recurrences [9]. The impact of CNS involvement and CNS-directed therapy in patient outcome was further validated in a retrospective series of 23 additional BPDCN patients
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