Abstract:Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an exceedingly rare disorder categorized under acute myeloid leukemia by the World Health Organization. Phenotypically, malignant cells coexpress CD4(+) and CD56(+) without coexpressing common lymphoid or myeloid lineage markers. BPDCN frequently expresses CD123, TCL1, BDCA-2, and CD2AP. Restriction of CD2AP expression to plasmacytoid dendritic cells makes it a useful tool to help confirm diagnosis. Clonal complex chromosome aberrations are described in t… Show more
“…Chemotherapy with AML-type, acute lymphoblastic leukemia (ALL)-type or non-Hodgkin lymphoma-like regimens can yield complete remission (CR) rates of 40-90% [7,8]. Treatment with ALL-type regimens is associated with higher CR rates and/or overall survival (OS) [7,9,10].…”
Section: Introductionmentioning
confidence: 99%
“…Treatment with ALL-type regimens is associated with higher CR rates and/or overall survival (OS) [7,9,10]. However, remissions are usually short term with a median OS of 12-18 months [8]. Recent data suggest that the median OS of patients who received an allogeneic-Hematopoeitic Cell Transplant (allo-HCT) were superior to those who received chemotherapy alone (22.7 months vs. 7.1 months; P 5 0.03) [7].…”
“…Chemotherapy with AML-type, acute lymphoblastic leukemia (ALL)-type or non-Hodgkin lymphoma-like regimens can yield complete remission (CR) rates of 40-90% [7,8]. Treatment with ALL-type regimens is associated with higher CR rates and/or overall survival (OS) [7,9,10].…”
Section: Introductionmentioning
confidence: 99%
“…Treatment with ALL-type regimens is associated with higher CR rates and/or overall survival (OS) [7,9,10]. However, remissions are usually short term with a median OS of 12-18 months [8]. Recent data suggest that the median OS of patients who received an allogeneic-Hematopoeitic Cell Transplant (allo-HCT) were superior to those who received chemotherapy alone (22.7 months vs. 7.1 months; P 5 0.03) [7].…”
“…Unfortunately, prospective series of chemotherapy in BPDCN are lacking. We direct the reader to a series of comprehensive retrospective chemotherapy reviews (more than 4 patients per series) in Pagano et al, 31 Kharfan-Dabaja et al, 37 and most recently Laribi et al 36 We have not re-created the information contained in these articles, but we have proon thr basis of comorbidity and transplant eligibility, as shown in Figure 2. When deciding which therapy is most appropriate, it is important to remember that, although those with early-stage and isolated cutaneous lesions often have very good performance status, the majority of patients have advanced disease (stage III to IV) as defined by Ann Arbor staging and a median age older than 60 years at diagnosis.…”
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare myeloid malignancy with no defined standard of care. BPDCN presents most commonly with skin lesions with or without extramedullary organ involvement before leukemic dissemination. As a result of its clinical ambiguity, differentiating BPDCN from benign skin lesions or those of acute myeloid leukemia with leukemia cutis is challenging. BPDCN is most easily defined by the phenotype CD4 1 CD56
1
CD1231 lineage -MPO -, although many patients will present with variable expression of CD4, CD56, or alternate plasmacytoid markers, which compounds the difficulty in differentiating BPDCN from other myeloid or lymphoid malignancies. Chromosomal aberrations are frequent, and the mutational landscape of BPDCN is being rapidly characterized although no obvious molecular target for chemoimmunotherapy has been identified. Chemotherapy regimens developed for acute myeloid leukemia, acute lymphoid leukemia, and myelodysplastic syndrome have all been used to treat BPDCN. Relapse is frequent, and overall survival is quite poor. Allogeneic transplantation offers a chance at prolonged remission and possible cure for those who are eligible; unfortunately, relapse remains high ranging from 30% to 40%. Novel therapies such as SL-401, a diphtheria toxin conjugated to interleukin-3 (IL-3) is commonly overexpressed in BPDCN and other aggressive myeloid malignancies and has shown considerable promise in ongoing clinical trials. Future work with SL-401 will define its place in treating relapsed or refractory disease as well as its role as a first-line therapy or bridge to transplantation.
Learning Objectives• To understand the clinical and laboratory approach for differentiating BPDCN from acute myeloid leukemia with cutaneous manifestations • To consider appropriate first-line and salvage treatments for BPDCN, including the utility of hematopoietic allogeneic stem cell transplantation and novel agents in various patient populations
“…Allogeneic hematopoietic stem cell transplantation (allo-HSCT) with myeloablative conditioning (MAC) during remission has shown encouraging results for patients with BPDCN. 3,[12][13][14][15][16][17] Meanwhile, only a few small case series were described that showed the benefit of high-dose chemotherapy followed by autologous hematopoietic stem cell transplantation (auto-HSCT) or reduced-intensity conditioning (RIC) allo-HSCT. 12,14,15,18,19 Because BPDCN is a disease of advanced age, clarifying the role of the RIC regimen or auto-HSCT in BPDCN is crucial.…”
Key Points• Auto-HSCT in CR1 provides long-term remission in BPDCN patients.• RIC allo-HSCT and MAC allo-HSCT results are comparable.We sought to clarify the role of high-dose chemotherapy followed by autologous hematopoietic stem cell transplantation (auto-HSCT) and allogeneic hematopoietic stem cell transplantation (allo-HSCT) to treat blastic plasmacytoid dendritic cell neoplasm (BPDCN). We retrospectively identified 25 BPDCN patients (allo-HSCT, n 5 14; auto-HSCT, n 5 11) from registry data of the Japan Society for Hematopoietic Cell Transplantation and analyzed clinicopathologic data and clinical outcomes after transplantation. The median age at HSCT was 58 years (range, 17-67 years). All 11 patients who underwent auto-HSCT were in the first complete remission (CR1). With a median follow-up of 53.5 months, the overall survival rates at 4 years for patients who underwent auto-HSCT and allo-HSCT were 82% and 53% (P 5 .11), respectively, and progression-free survival rates were 73% and 48% (P 5 .14), respectively. Auto-HSCT for BPDCN in CR1 appears to provide promising results and deserves further evaluation in the setting of prospective trials. (Blood. 2015;125(23):3559-3562)
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