Novel treatment of blastic plasmacytoid dendritic cell neoplasm

Abstract

Rationale:Blastic plasmacytoid dendritic cell neoplasm (BPDCN), derived from precursors of plasmacytoid dendritic cells, is a rare and aggressive malignancy with frequent cutaneous involvement. Although cutaneous lesions are often chemosensitive, BPDCN portends a poor prognosis as most patients relapse after developing drug resistance.Patient concerns:We report a case of a 65-year-old man who presented with a rapidly enlarging hyperpigmented plaque on his shoulder with subsequent similarly appearing macules and plaques on his chest, back, and neck.Diagnosis:Skin biopsy revealed a dense adnexocentric dermal infiltrate of immature blastoid cells without epidermal involvement. The infiltrate was immunoreactive for CD4, CD56, CD123, and Bcl-2, but negative for CD3, CD8, CD30, MPO, EBER, and ISH. The patient was diagnosed with BPDCN based on these cell markers.Intervention:Bone marrow biopsy and radiologic work-up showed no evidence of extracutaneous involvement. The patient attained partial remission after undergoing 2 rounds of cyclophosphamide, doxorubicin, vincristine, and prednisone (CHOP regimen) before autologous stem cell transplantation, however, he quickly relapsed and developed new cutaneous lesions.Outcomes:The patient was treated with venetoclax, a Bcl-2 inhibitor, and exhibits complete resolution of prior skin findings and continues to remain free of new cutaneous lesions 10 months posttreatment initiation with venetoclax.Lessons:Herein, we present a case that supports the use of venetoclax, a Bcl-2 inhibitor, in the off-label treatment of BPDCN with Bcl-2 overexpression. Only 1 prior case has reported the off-label use of venetoclax for the treatment of BPDCN. This case highlights a novel therapeutic option for BPDCN patients unresponsive to traditional treatment.

Blastic Plasmacytoid Dendritic Cell Neoplasm: A Case Report and Clinicopathological Review

“…Patients with BPDCN are usually treated with regimens used for other hematopoietic malignancies such as non-Hodgkin lymphoma (CHOP) and acute myeloid leukemia and lymphoma (cyclophosphamide/vincristine/doxorubicin/ dexamethasone alternating with methotrexate and cytarabine (hyper-CVAD)). Initially, patients commonly respond well to chemotherapy with complete resolution of skin lesions, but frequently relapse as chemotherapeutic drug resistance develops [16,17].…”

“…However, advanced age and comorbidities in many patients with BP-DCN limit the use of allogeneic BMT. Studies demonstrate that the best long-term prognosis is in younger patients who undergo allogeneic BMT after chemotherapy induced remission [15][16][17].…”

Blastic Plasmacytoid Dendritic Cell Neoplasm: State of the Art and Prospects

“…Several recently published reports have described the activity of venetoclax in the setting of BPDCN [89,110,111,112]. Venetoclax given as single agent or in combination with hypomethylating agents was able to induce meaningful clinical responses in relapsed/refractory patients.…”

The beneficial role of allogenic hematopoietic cell transplantation in blastic plasmacytoid dendritic cell neoplasm

“…Strong expression of Bcl-2 in BPDCN compared to pDC cells encouraged the use of venetoclax and the expression of CD38 suggests a potential to use daratumumab. Grushchak et al [56] described a patient with CR and survival of at least 10 months after 5-month venetoclax therapy, while Iversen et al [39] showed remissions after using daratumumab in monotherapy. Another protocol that has recently proved effective in individual cases consisting of bortezomib, lenalidomide, and dexamethasone.…”