MPN-467 Findings From an Observational, Multicenter, Retrospective Analysis of Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm Treated With Tagraxofusp in the European Expanded Access Program

Herling, Marco; Deconinck, Éric; Anant, Madhu; Manteigas, David; Riggi, Marcello; Mughal, Tariq I.; Angelucci, Emanuele

doi:10.1016/s2152-2650(22)01461-6

Cited by 3 publications

(5 citation statements)

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“…Likewise, no environmental, inherited, or acquired genetic factors have been found to increase BPDCN risk. Retrospective review of European pediatric and adult patients with BPDCN on targeted therapy further confirms these demographic distributions [ 18 ].…”

Section: Epidemiologymentioning

confidence: 86%

Blastic plasmacytoid dendritic cell neoplasm: a comprehensive review in pediatrics, adolescents, and young adults (AYA) and an update of novel therapies

Cuglievan

Connors

et al. 2023

Leukemia

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Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy that can involve the bone marrow, peripheral blood, skin, lymph nodes, and the central nervous system. Though more common in older adults, BPDCN has been reported across all age groups, including infants and children. The incidence of pediatric BPDCN is extremely low and little is known about the disease. Pediatric BPDCN is believed to be clinically less aggressive but often with more dissemination at presentation than adult cases. Unlike adults who almost always proceed to a hematopoietic stem cell transplantation in first complete remission if transplant-eligible, the majority of children can be cured with a high-risk acute lymphoblastic leukemia-like regimen. Hematopoietic stem cell transplantation is recommended for children with high-risk disease, the definition of which continues to evolve, or those in relapse and refractory settings where outcomes continue to be dismal. Novel agents used in other hematologic malignancies and CD123 targeted agents, including chimeric antigen receptor T-cells and monoclonal/bispecific antibodies, are being brought into research and practice. Our goal is to provide a comprehensive review of presentation, diagnosis, and treatment by review of pediatric cases reported for the last 20 years, and a review of novel targeted therapies and therapies under investigation for adult and pediatric patients.

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Section: Epidemiologymentioning

confidence: 86%

Blastic plasmacytoid dendritic cell neoplasm: a comprehensive review in pediatrics, adolescents, and young adults (AYA) and an update of novel therapies

Cuglievan

Connors

et al. 2023

Leukemia

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“…G-banding staining was performed, and the karyotype was reported following the International System for Human Cytogenetic Nomenclature 2020 recommendations. The karyotype was complex (47, XY, −3, −5, of (7) (q21), 12, +13, of (20) (q12), + 3mar [3]/46, XY [5]). Next-generation sequencing (NGS) studies were performed using SOPHiA DDM™ Myeloid Solution (MYS, Sophia Genetics, Switzerland).…”

Section: Clinical Casementioning

confidence: 99%

“…Around 10-20% of patients with BPDCN have a previous diagnosis of acute myeloid leukaemia (AML), chronic myeloid leukaemia, chronic myelomonocytic leukaemia (CMML), or myelodysplastic syndromes [3][4][5][6][7]. The immunophenotypic profile is characterized by positivity for the CD56 and/or CD4 and CD123 markers and one other plasmacytoid dendritic cell (pDC) marker (TCF4, TCL1, CD303, or CD304) or the expression of any three pDC markers with negative results for other specific T-cell, B-cell, and myeloid cell markers [8].…”

Section: Introductionmentioning

confidence: 99%

Spontaneous Remission of Blastic Plasmacytoid Dendritic Cell Neoplasm: A Case Report

Castaño-Bonilla,

Mata,

Láinez-González

et al. 2024

Medicina

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Spontaneous remissions (SRs) in blastic plasmacytoid dendritic cell neoplasms (BPDCNs) are infrequent, poorly documented, and transient. We report a 40-year-old man presenting with bycitopenia and soft tissue infection. The bone marrow exhibited 3% abnormal cells. Immunophenotyping of these cells revealed the antigens CD45+ (dim), CD34+, CD117+, CD123+ (bright), HLA-DR+ (bimodal), CD56+ (bright), CD33+, CD13+, CD2+, and CD22+ (dim) and the partial expression of the CD10+, CD36+, and CD7+ antigens. All other myeloid, monocytic, and lymphoid antigens were negative. Genetic studies showed a complex karyotype and mutations in the TP53R337C and KRASG12D genes. On hospital admission, the patient showed a subcutaneous nodule on the right hand and left lower limb. Flow cytometry multiparameter (FCM) analysis showed the presence of 29% abnormal cells with the previously described immunophenotype. The patient was diagnosed with BPDCN. The patient was treated with broad-spectrum antibiotics for soft tissue infection, which delayed therapy for BPDCN. No steroids or chemotherapeutic or hypomethylating agents were administered. His blood cell counts improved and skin lesions disappeared, until the patient relapsed five months after achieving spontaneous remission. About 60% of abnormal cells were identified. No changes in immunophenotype or the results of genetic studies were observed. The patient underwent a HyperCVAD chemotherapy regimen for six cycles. Consolidation therapy was performed via allogeneic bone marrow transplantation with an HLA-unrelated donor. One year after the bone marrow transplant, the patient died due to the progression of his underlying disease, coinciding with a respiratory infection caused by SARS-CoV-2. In the available literature, SRs are often linked to infections or other stimulators of the immune system, suggesting that powerful immune activation could play a role in controlling the leukemic clone. Nevertheless, the underlying mechanism of this phenomenon is not clearly understood. We hypothesize that the immune system would force the leukemic stem cell (LSC) to undergo a state of quiescence. This loss of replication causes the LSC progeny to die off, resulting in the SR of BPDCN.

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“…In August 2019, the manufacturer launched an expanded access program (EAP) in Europe in order to guarantee patients’ access to tagraxofusp before its approval by the European Medicines Agency (EMA) and, at the same time, collect data on its efficacy and safety in everyday clinical practice. 8 The European EAP was a single-arm, retrospective, multicenter observational study involving both patients with treatment-naive BPDCN and other-relapsed/refractory BPDCN treated with tagraxofusp in real life in 6 European countries (France, Germany, Italy, Switzerland, Austria, and Spain). 8 The preliminary analysis of the EAP revealed extremely satisfactory data and a favorable risk-benefit profile of tagraxofusp, with an efficacy that appeared even higher than that reported in the registration trial.…”

mentioning

confidence: 99%

“… 8 The European EAP was a single-arm, retrospective, multicenter observational study involving both patients with treatment-naive BPDCN and other-relapsed/refractory BPDCN treated with tagraxofusp in real life in 6 European countries (France, Germany, Italy, Switzerland, Austria, and Spain). 8 The preliminary analysis of the EAP revealed extremely satisfactory data and a favorable risk-benefit profile of tagraxofusp, with an efficacy that appeared even higher than that reported in the registration trial. 9 Despite the excellent results, it was several years after FDA approval, on 7 January 2021, that the EMA granted marketing authorization in the European Union (EU) for tagraxofusp as frontline therapy in BPDCN, limiting its use as a monotherapy treatment to adult patients (aged ≥18 years).…”

mentioning

confidence: 99%

Tagraxofusp for blastic plasmacytoid dendritic cell neoplasm: a 2-speed cure in the United States and European Union

Valentini,

Pagano

2023

Blood Advances

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MPN-467 Findings From an Observational, Multicenter, Retrospective Analysis of Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm Treated With Tagraxofusp in the European Expanded Access Program

Cited by 3 publications

References 0 publications

Blastic plasmacytoid dendritic cell neoplasm: a comprehensive review in pediatrics, adolescents, and young adults (AYA) and an update of novel therapies

Blastic plasmacytoid dendritic cell neoplasm: a comprehensive review in pediatrics, adolescents, and young adults (AYA) and an update of novel therapies

Spontaneous Remission of Blastic Plasmacytoid Dendritic Cell Neoplasm: A Case Report

Tagraxofusp for blastic plasmacytoid dendritic cell neoplasm: a 2-speed cure in the United States and European Union

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