Preliminary Results from an Observational Multicenter Study of Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm Treated with Tagraxofusp in the European Expanded Access Program

Deconinck, Éric; Anant, Madhu; Manteigas, David; Paley, Carole; Riggi, Marcello; Herling, Marco; Angelucci, Emanuele

doi:10.1182/blood-2022-159942

Cited by 4 publications

(5 citation statements)

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“…At the same time, he has tolerated tagraxofusp well and thus has benefited much from the availability of tagraxofusp. Apart from the above-mentioned clinical trials, real-world outcome data from tagraxofusp-treated BPDCN patients are limited to case reports and conference abstracts reporting early results ( 35 ).…”

Section: Discussionmentioning

confidence: 99%

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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: 99%

“…Long-term results of an expanded cohort of the STML-401-0114 trial recently indicated an overall response rate of 75%, a median CR duration of 24.9 months, and a median OS of 15.8 months after four treatment cycles, with overall good safety profile ( 35 ).…”

Section: Introductionmentioning

confidence: 99%

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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“…Of note, initial "realworld" clinical practice data from a European expanded access program were recently presented confirming a 67% CR rate (10 of 15) in previously untreated patients, with a similar safety profile and no deaths related to CLS. 35 The STML-401-0114 data resulted in US Food and Drug Administration (FDA) approval for tagraxofusp in 2018, for patients ≥2 years with newly diagnosed or R/R BPDCN.…”

Section: Tagraxofusp As a Single Agent (Stml-401-0114)mentioning

confidence: 99%

“…Of note, initial “real-world” clinical practice data from a European expanded access program were recently presented confirming a 67% CR rate (10 out of 15) in previously untreated patients, with a similar safety profile and no deaths related to CLS. 35 …”

Section: Tagraxofusp As a Single Agentmentioning

confidence: 99%

Tagraxofusp for blastic plasmacytoid dendritic cell neoplasm

Luskin

Lane

2023

haematol

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Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy that presents with characteristic dark purple skin papules, plaques, and tumors, but may also involve the bone marrow, blood, lymph nodes, and central nervous system. The disease, which commonly affects older men but can also present in children, is associated with a distinct immunophenotype including universal expression of CD123, the alpha chain of the interleukin 3 receptor. Recently, tagraxofusp, a CD123-targeting drug consisting of the ligand for CD123, interleukin 3, conjugated to a truncated diphtheria toxin payload was approved for treatment of BPDCN. This was the first agent specifically approved for BPDCN and the first CD123 targeted agent in oncology. Here, we review the development of tagraxofusp and the key preclinical insights and clinical data that led to approval. Tagraxofusp treatment is associated with a unique toxicity, capillary leak syndrome (CLS), which can be severe but is manageable with proper patient selection and monitoring, early recognition, and directed intervention. We outline our approach to the use of tagraxofusp and discuss open questions in treatment of BPDCN. Overall, tagraxofusp represents a unique targeted therapy and a step forward in meeting an unmet need for patients with this rare disease.

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“… 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). Nonetheless, the drug is still under evaluation by the regulatory agencies of most European countries, whereas it has been available only in Germany since January 2023 and secondly in Italy, where the Italian Medicines Agency-AIFA has approved the reimbursement of tagraxofusp in March 2023.…”

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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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Preliminary Results from an Observational Multicenter Study of Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm Treated with Tagraxofusp in the European Expanded Access Program

Cited by 4 publications

References 0 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

Tagraxofusp for blastic plasmacytoid dendritic cell neoplasm

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

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