Elzonris (tagraxofusp-erzs)

In this trial, tagraxofusp monotherapy in MF was well tolerated, without cumulative myelotoxicity, and with symptom score improvements, warranting further investigation in combination therapy. This trial was registered at www.clincaltrials.gov as #NCT02268253.

During tagraxofusp treatment, strict monitoring for early recognition of CLS symptoms and directed intervention is essential for managing CLS; with this approach CLS is typically mild and occurs mostly in cycle 1, without recurrence. We discuss patient selection and optimization, monitoring, and early intervention for successful identification and optimized management of CLS in real-world practice.

Given the poor prognosis associated with this subtype, CD123-targeted therapy, such as tagraxofusp-erzs, alone or in combination with agents like azacitidine and venetoclax, may represent a rational therapeutic approach. To our knowledge, this represents the third case report of RUNX1 rearrangement in pDC-AML and may provide valuable insights for future research.

Tagraxofusp, a first-in-class CD123-directed antibody conjugate comprising human interleukin-3 and truncated diphtheria toxin, represents a welcome addition to the treatment armamentarium for BPDCN...Several questions remain unanswered-namely, the potential benefit of post-transplant consolidation or maintenance strategies to mitigate the risk of relapse or progression, and the role of next-generation sequencing as a prognostic tool and for better defining depth of remission and measurable residual disease, among others. We believe that further improvement in the prognosis of BPDCN will require large collaborative efforts, considering the rarity of this disease.

In this case series, we describe our institutional experience treating BPDCN both pre- and post-tagraxofusp approval. We summarize six cases, the majority of which occurred in elderly patients, and highlight unique challenges of treating BPDCN at a center that serves a large rural/frontier area with variable access to specialty care.

Tagraxofusp demonstrated a manageable safety profile with limited clinical efficacy in CMML. This trial was registered at www.ClinicalTrials.gov as #NCT02268253.

A core specialized interdisciplinary team coupled with AP-led management optimizes tagraxofusp treatment. This paper reviews best practices for the clinical management of patients with BPDCN receiving tagraxofusp in the context of the US package insert and APs' real-world management approaches.

Treatment-related non-hematologic grade 3/4 adverse events (AEs) or serious AEs were reported in 12 patients (46%); 9 patients (35%) had treatment-related grade 3/4 hematologic AEs. These real-world findings showed no new safety signals and confirmed that tagraxofusp is the first-line treatment of choice for most patients with BPDCN.

P1, N=72, Recruiting, Dana-Farber Cancer Institute | Trial completion date: May 2026 --> May 2027 | Trial primary completion date: May 2025 --> May 2026

The median overall survival is 18-24 months. Potential risk factors for shortened survival include older age, overt bone marrow involvement, and genetic abnormalities.

P2, N=33, Not yet recruiting, French Innovative Leukemia Organisation

P1, N=36, Active, not recruiting, Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | Recruiting --> Active, not recruiting