Blastic plasmocitoid dendritic cell neoplasm with leukemic spread: a GIMEMA survey

Valentini, Caterina Giovanna; Piciocchi, Alfonso; Facchetti, Fabio; Guolo, Fabio; Pulsoni, Alessandro; Vignetti, Marco; Pagano, Livio

doi:10.1182/bloodadvances.2021005802

Cited by 12 publications

(8 citation statements)

References 19 publications

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“…Flow cytometry can assist in diagnosing BPDCN in cases with involvement of the blood and/or BM, which is often detected during staging procedures. A recent survey showed that half of the Italian hematologists applied a panel including CD123, CD303, and TCL1 only in selected cases, after the exclusion of other forms of acute leukemia 11 . Regrettably, the limitations in the composition and usage of phenotypic panels lead to delay in diagnosis and subsequent treatment.…”

Section: Unmet Clinical Needsmentioning

confidence: 99%

Unmet Clinical Needs and Management Recommendations for Blastic Plasmacytoid Dendritic Cell Neoplasm: A Consensus-based Position Paper From an Ad Hoc International Expert Panel

et al. 2023

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Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a hematological malignancy characterized by recurrent skin nodules, an aggressive clinical course with rapid involvement of hematological organs, and a poor prognosis with overall survival. The rarity of the disease results in a few large-scale studies, a lack of controlled clinical trials for its management, and a lack of evidence-based guidelines. Here, we present a review of unmet clinical needs on the management of BPDCN by a panel of eleven experts involved in the research and clinical practice of BPDCN. Recommendations and proposals were achieved by multiple-step formalized procedures to reach a consensus after a comprehensive analysis of the scientific literature. The panel analyzed the critical issues of diagnostic pathway, prognostic stratification, therapy for young and fit patients and elderly and unfit patients, indication for allotransplant and for autotransplant, indication for central nervous system prophylaxis, and management of pediatric BPDCN patients. For each of these issues, consensus opinions were provided and, when appropriate, proposals for advancement in clinical practice were addressed. The hope is that this comprehensive overview will serve to improve the practice of BPDCN and inform the design and implementation of new studies in the field.

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Section: Unmet Clinical Needsmentioning

confidence: 99%

Unmet Clinical Needs and Management Recommendations for Blastic Plasmacytoid Dendritic Cell Neoplasm: A Consensus-based Position Paper From an Ad Hoc International Expert Panel

et al. 2023

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“…In another recently published Italian cohort of 68 patients, four patients presented with frontline CNS disease and two developed CNS recurrence (70% routine CSF exams at diagnosis). 7 These studies indicate the importance of CNS involvement in BPDCN, but also show, that routine CNS testing and prophylactic intrathecal treatment have not been implemented so far, despite clinical practice guidelines (NCCN 2022 8 ) and some data suggesting a benefit of acute lymphoblastic (ALL)-like treatment protocols including intrathecal treatment. 9,10 The anti-CD123 antibody-immunotoxin conjugate tagraxofusp-erzs (SL-401) received Food and Drug Administration and European Medicines Agency approval as upfront single agent after the results of a single arm phase I/II trial.…”

Section: Treatment Of Leptomeningeal Disease In Blastic Plasmacytoid ...mentioning

confidence: 99%

“…Our case adds to recently published data on CNS involvement in BPDCN. 7,8,12 Furthermore, it conveys three important messages: first, LP is an essential initial diagnostic procedure in BPDCN, also in the context of therapy with tagraxofusp-erzs. Due to their distinct morphology and aberrant immunophenotype BPDCN cells can be easily detected (Figure 1B and C).…”

Section: Treatment Of Leptomeningeal Disease In Blastic Plasmacytoid ...mentioning

confidence: 99%

Treatment of leptomeningeal disease in blastic plasmacytoid dendritic cell neoplasm following tagraxofusp-erzs induction

et al. 2022

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“…Most patients present with skin and bone marrow (BM) involvement; a small proportion present with the disease in other extramedullary sites, in particular, lymph nodes (LN) and the central nervous system (CNS) [8][9][10]. A recent United States study indicated that BPDCN may have a bimodal age distribution with the first peak seen in children and adolescents [1].…”

Section: Introductionmentioning

confidence: 99%

Efficacy and manageable safety of tagraxofusp in blastic plasmacytoid dendritic cell neoplasm: a case series of pediatric and adolescent/young adult patients

Pemmaraju,

Cuglievan,

Lasky

et al. 2024

eJHaem

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Blastic plasmacytoid dendritic cell neoplasm (BPDCN) predominantly occurs in adults ≥60 years old; 10–20% of cases are pediatric or adolescent/young adult (AYA) patients. Tagraxofusp (TAG, Elzonris®) is the only approved treatment for BPDCN; in the US it is approved for patients aged ≥2 years. Data on treating pediatric and AYA BPDCN patients are limited. We present a case series of pediatric and AYA patients with BPDCN treated with TAG. Eight patients (five newly diagnosed; three relapsed/refractory [R/R]), aged 2–21 years, received 12 mcg/kg TAG. Seven patients were female; most had skin (n = 6) and/or bone marrow (n = 5) involvement. No new safety signals were identified. Grade 3 adverse events were headache (n = 1) and transaminitis (n = 2). Three patients with newly diagnosed BPDCN achieved complete response, one achieved partial response, and one had stable disease (SD). One patient with R/R BPDCN achieved a minor response; one had SD. Seven patients (88%) were bridged to stem cell transplant: 80% of newly diagnosed patients and 100% of R/R patients. Five patients remained alive at last follow‐up. These cases highlight the efficacy and safety of TAG in pediatric and AYA patients for whom there is no other approved BPDCN therapy.

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Blastic plasmocitoid dendritic cell neoplasm with leukemic spread: a GIMEMA survey

Cited by 12 publications

References 19 publications

Unmet Clinical Needs and Management Recommendations for Blastic Plasmacytoid Dendritic Cell Neoplasm: A Consensus-based Position Paper From an Ad Hoc International Expert Panel

Unmet Clinical Needs and Management Recommendations for Blastic Plasmacytoid Dendritic Cell Neoplasm: A Consensus-based Position Paper From an Ad Hoc International Expert Panel

Treatment of leptomeningeal disease in blastic plasmacytoid dendritic cell neoplasm following tagraxofusp-erzs induction

Efficacy and manageable safety of tagraxofusp in blastic plasmacytoid dendritic cell neoplasm: a case series of pediatric and adolescent/young adult patients

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