Venetoclax in relapsed/refractory blastic plasmacytoid dendritic cell neoplasm with central nervous system involvement: a case report and review of the literature

Abstract

Background We describe a patient with blastic plasmacytoid dendritic cell neoplasm with central nervous system involvement and the outcome of venetoclax use in this setting. Case presentation A 54-year-old Caucasian male was referred to the Haematology Unit with an enlarged inguinal lymph node which was diagnostic of a blastic plasmacytoid dendritic cell neoplasm. The staging revealed disseminated disease (skin, visceral, lymph nodes, and bone marrow). He received chemotherapy with an acute myeloid leukaemia-like regime. Afterwards, he underwent allogeneic haematopoietic stem cell transplantation, though it was not successful, showing a relapse 14 months later with hepatic and central nervous system dissemination. Intrathecal chemotherapy was administered, and venetoclax (anti-bcl2 agent) was started in an off-label indication based on most recent literature. The disease halted its course for 3 months. In the end, the patient’s disease progressed and so he succumbed due to infectious complications. Conclusions Venetoclax monotherapy seems not enough to control the disease progression under CNS involvement and other treatments should be investigated.

Cytomorphological and immunophenotypic characteristics of blastic plasmacytoid dendritic cell neoplasm involving central nervous system: a case report and review of literature

“…The malignant cells in our case had blast‐like morphology with scant amounts of agranular cytoplasm. Like previous studies 5‐7,12 where the presence of intracytoplasmic vacuoles in at least few cells has been considered as a distinctive cytomorphological characteristic of BPDCN, we found this feature in our case (Table 2). Akin to previous reports, 5‐7,12 we also found prominent nucleoli in a majority of cells in our case.…”

“…Like previous studies 5‐7,12 where the presence of intracytoplasmic vacuoles in at least few cells has been considered as a distinctive cytomorphological characteristic of BPDCN, we found this feature in our case (Table 2). Akin to previous reports, 5‐7,12 we also found prominent nucleoli in a majority of cells in our case. In all reported studies, only a few immunophenotypic markers have been used for assessment of the CSF sample.…”

Blastic plasmacytoid dendritic cell neoplasm (BPDCN): A promising future in the era of targeted therapeutics

“…The majority of patients followed in this case series had significant comorbidities, which could suggest that venetoclax and azacitidine may serve as a reasonable alternative treatment option for patients with an inability to tolerate CD123-based and cytotoxic chemotherapy regimens. 42 In addition, Phase 1 clinical trials are currently under way to determine the maximum tolerated dose, time to response, and duration of remission in patients with relapsed or refractory BPDCN treated with a combination of tagraxofusp, azacitidine, and venetoclax (NCT03113643). It is also the subject of study of a Phase 2 clinical trial in combination with decitabine to evaluate overall treatment response (NCT03404193).…”

“…Furthermore, 1 case series following 10 patients treated with relapsed/refractory BPDCN with a regimen of venetoclax and azacitidine showed that although the combination did have short‐lived effects of clinical remission, it could serve as a promising bridge to allogeneic stem cell transplant. The majority of patients followed in this case series had significant comorbidities, which could suggest that venetoclax and azacitidine may serve as a reasonable alternative treatment option for patients with an inability to tolerate CD123‐based and cytotoxic chemotherapy regimens 42 . In addition, Phase 1 clinical trials are currently under way to determine the maximum tolerated dose, time to response, and duration of remission in patients with relapsed or refractory BPDCN treated with a combination of tagraxofusp, azacitidine, and venetoclax (NCT03113643).…”

Primary Blastic Plasmacytoid Dendritic Cell Neoplasm: a US Population-Based Study

“…However, univariate, and multivariate cox analysis showed that those therapies (including chemotherapy and radiotherapy) had no impact on the survival outcomes of primary BPDCN at all. Although there were numerous introductions of novel drugs such as Tagraxofusp and Venetoclax for the treatment of BPDCN, which have shown promising results 17,18 . In the current study, based on population analysis of primary BPDCN patients, we found that patients diagnosed between 2015 and 2019 had no significant different OS and DSS than those diagnosed between 2000 and 2004, reflecting the development status of the novel therapies.…”