Declarations

Funding The preparation of this review was not supported by any external funding.

Authorship and conflict of interest A. Markham is a salaried employee of Adis International Ltd/Springer Nature and declare no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.

Ethics approval, Consent to participate, Consent for publication, Availability of data and material, Code availability Not applicable

Additional information about this Adis Drug Review can be found here

Abstract

The CD123-directed cytotoxin tagraxofusp (ELZONRIS^®) is the first treatment approved in the EU for first-line treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults. BPDCN is a rare disease that affects mainly older patients and is usually treated with acute leukaemia-type multi-drug chemotherapy regimens followed, if possible, by stem cell transplantation (SCT). In the largest prospective trial conducted in BPDCN to date, treatment with tagraxofusp led to high rates of early and durable response in patients with previously untreated BPDCN, and enabled bridging to SCT in half of those who responded. The tolerability profile of tagraxofusp is well characterised, with most adverse events occurring during the first cycle of treatment, and many older patients who did not undergo transplantation were able to tolerate long term treatment, leading to prolonged responses. Tagraxofusp thus represents an important advance in the treatment of BPDCN, particularly for older patients who are unable to tolerate the multidrug chemotherapy regimens that until now have been the mainstay of treatment.

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