Blastic plasmacytoid dendritic cell neoplasm
Blastic plasmacytoid dendritic cell neoplasm | |
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CD4+ CD56+ lymphoma |
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy. It was initially regarded as a form of lymphocyte-derived cutaneous lymphoma and alternatively named CD4+CD56+ hematodermic tumor, blastic NK cell lymphoma,[1] and agranular CD4+ NK cell leukemia.[2] Later, however, the disease was determined to be a malignancy of plasmacytoid dendritic cells rather than lymphocytes and therefore termed blastic plasmacytoid dendritic cell neoplasm. In 2016, the World Health Organization designated BPDCN to be in its own separate category within the myeloid class of neoplasms.[3] It is estimated that BPDCN constitutes 0.44% of all hematological malignancies.[4]
Blastic plasmacytoid dendritic cell neoplasm is an aggressive malignancy with features of
Blastic plasmacytoid dendritic cell neoplasm typically responds to chemotherapy regimens used to treat hematological malignancies. All too often, however, the disease rapidly recurs and does so in a more drug-resistant form.[5] Furthermore, the disease may occur in association with the myelodysplastic syndrome or transform to acute myeloid leukemia.[4] Consequently, BPDCN has a very low 5 year survival rate.[5] Current translational research studies on treating BPDCN have therefore focused on non-chemotherapeutic regimens that target the molecular pathways which may promote the disease.[6]
Presentation
Blastic plasmacytoid dendritic cell neoplasm occurs in children,
Pathophysiology
There are three types of
Blastic plasmacytoid dendritic cell neoplasm typically arises after the serial acquisition of multiple genetic abnormalities in pDC or their precursor cells.
Diagnosis
BPDCN is suggested by a biopsy of skin lesions which reveals the infiltration by medium-sized blast (i.e. immature) cells into the
Treatment
There have been no controlled studies to define the optimal treatment for BPDCN.
While few studies have reported on the treatment of BPDCN that has recurred following initial therapy, donor lymphocyte infusions coupled with alternative chemotherapy treatments have induced second complete or partial remissions in a few patients.[4]
Tagraxofusp-erzs
Prognosis
Due to the high rates of recurrence following initial therapy and the short overall survival times of individuals with BPDCN, prognosis of the disease is poor. However, further study of treatment regimens that include intrathecal chemotherapy and hematological stem cell transplantation in initial treatment regimens (see previous section) and newer non-chemotherapeutic drug treatments (see next section) may improve this situation.[8]
Research
UCART123
Venetoclax
References
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- ^ "Combination Chemotherapy (Methotrexate, L-asparaginase, Idarubicin and Dexamethasone) in Patients with Newly Diagnosed Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)". 19 November 2019.
- ^ "FDA approves first treatment for rare blood disease" (Press release). Food and Drug Administration. December 21, 2018.
- ^ a b McKee, Selina (2017-09-05). "FDA holds trials of Cellectis' cell therapy after patient death". www.pharmatimes.com. Retrieved 2017-10-08.
- ^ Melão, Alice (9 November 2017). "FDA Lifts Clinical Hold on Cellectis' UCART123 Trials in AML, BPDCN".
- ^ "Phase 1, Open-label Dose-escalation and Dose-expansion Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of a Single Dose of UCART123 (Allogeneic Engineered T-cells Expressing Anti-CD123 Chimeric Antigen Receptor), Administered in Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)". 29 July 2019.
- ^ Lane, Andrew (6 May 2021). "Phase 1 Study of Venetoclax, a BCL2 Antagonist, for Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)".