Blastic plasmacytoid dendritic cell neoplasms: results of an international survey on 398 adult patients

Abstract

The purpose of this study is to describe the clinical and prognostic features and to evaluate the outcome of different therapeutic approaches among patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) who have been diagnosed and treated in different institutions. A total of 398 patients from 75 centers were included in the study. Treatment consisted of non-Hodgkin lymphoma (NHL)–like regimens in 129 (32.8%) patients and acute leukemia (AL)–like regimens in 113 (23.5%) patients. In 61 (15.5%) and 16 (4.1%) patients, chemotherapy was followed by allogeneic and autologous hematopoietic stem cell transplantation (HSCT), respectively. Twenty-seven (6.9%) patients received radiotherapy, 6 (1.5%) received new agents, and 62 (15.7%) received palliative care. After a median follow-up of 12 months, median overall survival (OS) was 18 months. Patients who received NHL/AL-like regimens, followed by allogeneic HSCT, had the best outcome; median OS was not reached. OS was 65 months for patients who underwent autologous HSCT; 18 months and 14 months, respectively, for those treated with AL-like and NHL-like regimens without consolidation; and 4 months for those receiving palliative care (P < .001). In BPDCN, chemotherapy with lymphoma- or AL-like regimens, followed by transplantation, represents the therapeutic strategy associated with the best outcome. Consolidation with allogeneic HSCT, when feasible, appears superior to autologous HSCT.

Targeting CD123 in hematologic malignancies: identifying suitable patients for targeted therapy

“…Progress in this field is impacted by the use of accurate methods for detecting CD123 expression in candidate malignancies. Unlike in BPDCN, where CD123 is overexpressed uniformly in malignant pDCs [64,86], its expression in other malignancies is limited to certain cell types and sometimes the microenvironment, pre-inflammatory, and inflammatory contexts [124]. Advances in detection techniques, such as FC, CyTOF, and CODEX V R , might overcome these limitations, thus allowing the individualization of therapy.…”

“…Currently, tagraxofusp is the only FDA-approved CD123-targeted therapy; it is approved for the treatment of newly diagnosed and relapsed/refractory (R/R) BPDCN in adult and pediatric patients [19]. MB-102 T cells are genetically modified to express a costimulatory CD123-specific chimeric antigen receptor (CAR) and a truncated epidermal growth factor receptor (CD123CAR-CD28-CD3f-EGFRt) that were granted Orphan Drug Designation in 2018 for the treatment of BPDCN and in 2019 for AML [86]. Table 2 depicts some of the anti-CD123 therapies that are being tested for the treatment of CD123þ hematologic malignancies.…”

A case report of blastic plasmacytoid dendritic cell neoplasm in a hispanic child

“…Other dendritic cell markers not tested in this case are CD68, CD123 and BDCA-2/CD303 [14] . This patient also showed positivity for terminal deoxynucleotidyl transferase (TdT), which is associated to a better prognosis [8] .…”

“…Current literature, based on case reports in children supports the use of acute leukemia treatment regimens as the most beneficial option [7] . While, recently, a larger cohort in adults has reported the best outcomes for AL-like regimen followed by allogenic hematopoietic stem cell transplantation [8] . Although there is no standardized treatment for BPDCN, one study reported that adults treated with AML-like, ALL-like and high-dose methotrexate with asparaginase (Aspa-MTX) chemotherapies showed increased survival and remission compared to other treatments such as CHOP-like (classical regimen used in the treatment of non-Hodgkin lymphomas and combining cyclophosphamide, doxorubicin, vincristine, and prednisone) and not otherwise specified (NOS) regimens (all other drugs alone or in combination) [9] .…”

Blastic Plasmacytoid Dendritic Cell Neoplasm: Progress in Cell Origin, Molecular Biology, Diagnostic Criteria and Therapeutic Approaches

“…Children also were less likely than adult patients (27% vs. 57%, P<0.01) to relapse after complete remission. We also listed the response rates and survival outcomes with different regimens of the main BPDCN patients reported in literature [8,73,75,86,[91][92][93][94][95] (table 2).…”