Venetoclax and Azacitidine in the Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm Refractory to Conventional Therapy

Azad, Farhan; Zhang, Jiahua; Miranda, Clive; Gravina, Matthew

doi:10.7759/cureus.33109

Cited by 4 publications

(6 citation statements)

References 18 publications

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“…In addition, tagraxofusp-resistant cells have been demonstrated to exhibit increased dependence on BCL2 ( 53 ). Although our findings did not show favorable responses to salvage treatment with Aza and/or Ven, these agents also hold promise for post-tagraxofusp relapse salvage treatment, as indicated by several case reports ( 36 , 40 , 41 ).…”

Section: Discussionmentioning

confidence: 44%

Real-world evidence on tagraxofusp for blastic plasmacytoid dendritic cell neoplasm – collected cases from a single center and case reports

Faustmann,

Schroeder,

Mix

et al. 2024

Front. Oncol.

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IntroductionBlastic plasmacytoid dendritic cell neoplasia (BPDCN) is a rare, aggressive hematologic malignancy. Until recently, the only curative treatment consisted of intensive chemotherapy, followed by hematopoietic cell transplantation (HCT) in eligible adult cases. Tagraxofusp, a CD123-targeted protein-drug conjugate and the first approved targeted treatment for BPDCN, might enhance outcomes especially in patients not eligible for intensive therapies.MethodsHere, we report real-world outcomes of five male patients with a median age of 79 years who received tagraxofusp as first-line treatment for BPDCN.ResultsTagraxofusp was found to be well-tolerated in this elderly cohort, with only one patient requiring discontinuation. Three patients responded to the treatment (two patients achieved a CR and one patient achieved a partial response), of which two subsequently underwent allogeneic (allo) HCT. One patient is alive and well after ≥ 4 years after alloHCT, and one patient shows sustained CR after now 13 cycles of tagraxofusp. The other three patients died of progressive disease 4-11 months after initiation of treatment.DiscussionIn line with results from 13 published cases outside clinical trials in the literature, sustained responses were associated with CR after tagraxofusp treatment and subsequent alloHCT. Our results provide real-world evidence for safety and efficacy of tagraxofusp as first-line treatment for BPDCN.

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Section: Discussionmentioning

confidence: 44%

Real-world evidence on tagraxofusp for blastic plasmacytoid dendritic cell neoplasm – collected cases from a single center and case reports

Faustmann,

Schroeder,

Mix

et al. 2024

Front. Oncol.

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“…In the present case, the patient presented with a TET2 mutation and was treated with the DNA methyltransferase inhibitor, azacytidine, in combination with venetoclax. Certain cases of BPDCN have been treated with azacitidine in combination with venetoclax and it was well tolerated (58,59). Moreover, azacitidine combined with tagraxofusp has been reported to be effective for BPDCN treatment, with azacitidine restoring sensitivity to tagraxofusp by restoring diphthamide biosynthesis protein 1 expression (60).…”

Section: Discussionmentioning

confidence: 99%

Simultaneous blastic plasmacytoid dendritic cell neoplasm and myelofibrosis: A case report

Luo,

Li,

et al. 2024

Oncol Lett

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Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an extremely rare and aggressive tumor with an unknown pathogenesis. Myelofibrosis (MF) is a type of myeloproliferative neoplasm. MF can be secondary to several hematological malignancies, including chronic myeloid leukemia, myelodysplastic syndrome and hairy cell leukemia. In the present report, a rare case of BPDCN secondary to MF is described. A 70-year-old male patient developed a large purplish-red rash with recurrent symptoms. BPDCN was confirmed by immunohistochemistry of a biopsy specimen and flow cytometry of bone marrow cells. Bone marrow histopathology revealed MF. Next-generation sequencing of peripheral blood revealed mutations in the Tet methylcytosine dioxygenase 2 and NRAS proto-oncogene GTPase genes. The patient underwent one cycle of chemoimmunotherapy, but the condition progressed, an infection developed and the patient eventually died. The present case suggests that BPDCN can occur in conjunction with MF and that the prognosis of such patients is poor. Pathological examination and genetic testing aided in the diagnosis and treatment. This case emphasizes the need to raise awareness of BPDCN among clinicians and to be alert to the potential for fatal infection in patients with BPDCN combined with MF following myelosuppression triggered during chemotherapy.

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“…Gangat et al reported on five patients with primary BPDCN and five patients with relapsed BPDCN who received VEN-combined HMA therapy, in which VEN-combined HMA therapy led to CR in patients with BPDCN [5]. In addition, a previous case report described a patient with BPDCN who received continuous VEN/AZA therapy and remained in CR [6]. However, because patients treated with VEN/AZA therapy often present with FN, several patients, especially older adults or those who are unfit, discontinue VEN/AZA therapy [11,12].…”

Section: Discussionmentioning

confidence: 99%

“…A recent study demonstrated that BPDCN cells target BCL2 proteins and are uniformly sensitive to VEN in vivo [4]. In addition, VEN-combined chemotherapy is reportedly effective in patients with primary and relapsed BPDCN [5,6].…”

Section: Introductionmentioning

confidence: 99%

Blastic Plasmacytoid Dendritic Cell Neoplasm in Long-Term Complete Remission After Venetoclax Monotherapy

Sasaki,

Murai,

Shiozawa

et al. 2024

Cureus

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Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive hematological malignancy associated with a poor prognosis and limited treatment options. Although allogeneic hematopoietic stem cell transplantation or intensive chemotherapy prolongs overall survival in patients with BPDCN, intensive chemotherapy is inappropriate for older or unfit patients. Venetoclax (VEN), an oral BCL2 inhibitor, is approved for use in patients with acute myeloid leukemia (AML). BPDCN cells require BCL2 protein and are uniformly sensitive to VEN in vivo. Moreover, patients with AML who have achieved complete remission after induction therapy are reportedly considered to receive VEN monotherapy as maintenance therapy, especially older patients. However, the efficacy of VEN monotherapy as a maintenance therapy for patients with BPDCN remains controversial. Recently, BPDCN has been classified into MYC+ and MYC-subtypes, which show clinical differences. Hence, BPDCN treatment strategies based on the MYC classification may be necessary. Here, we report a case of MYC-BPDCN in an older patient in long-term complete remission after VEN monotherapy following VEN and azacitidine induction chemotherapy.

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Venetoclax and Azacitidine in the Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm Refractory to Conventional Therapy

Cited by 4 publications

References 18 publications

Real-world evidence on tagraxofusp for blastic plasmacytoid dendritic cell neoplasm – collected cases from a single center and case reports

Real-world evidence on tagraxofusp for blastic plasmacytoid dendritic cell neoplasm – collected cases from a single center and case reports

Simultaneous blastic plasmacytoid dendritic cell neoplasm and myelofibrosis: A case report

Blastic Plasmacytoid Dendritic Cell Neoplasm in Long-Term Complete Remission After Venetoclax Monotherapy

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