Tagraxofusp as first-line treatment for blastic plasmacytoid dendritic cell neoplasm

Acedo, Rocío Díaz; Muñoz, María Ángeles Domínguez; Laguna, Clara Navajas; Morales‐Camacho, Rosario M.; Pilo, Isabel Simón; Ruiz-Mateos, Virgilio Pablo Calama; Ramírez, Manuel Yébenes; Medina, María Vahí Sánchez de; Criado, Silvia Artacho; Rodríguez-Pérez, Aitana; Couto, María Carmen

doi:10.1080/10428194.2022.2042685

Cited by 7 publications

(4 citation statements)

References 11 publications

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“…It has also been approved as first‐line therapy for adult patients with BPDCN in the European Union as of January 2021. Although tagraxofusp serves as an efficacious therapy in reducing burden of disease, it does involve a serious side effect profile, most encountered in cycle 1 of therapy, including hepatotoxicity (88%), thrombocytopenia (49%), and capillary leak syndrome (19%), which must be noted by all team members on the health care team administering this novel agent and should always be administered in an inpatient setting for the first cycle 22–25 . Given its demonstrated efficacy in achieving clinical response, CD123‐targeted based therapy will continue to be an integral backbone of treatment in both treatment‐naïve and refractory BPDCN.…”

Section: Introductionmentioning

confidence: 99%

Blastic plasmacytoid dendritic cell neoplasm (BPDCN): A promising future in the era of targeted therapeutics

Adimora

Wilson

Pemmaraju

2022

Cancer

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Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy arising from precursor dendritic cells. BPDCN cells characteristically express several markers on their cell surfaces including CD123, CD4, and CD56. Because of its rarity and challenging clinical presentation, there was no standard of care in managing BPDCN for decades and its prognosis overall was poor. However, as understanding of this rare neoplasm has increased, so have treatment options. The conventional cytotoxic chemotherapy regimens once used in the treatment of BPDCN were modest in their impact on disease relapse until paired with hematopoietic stem cell transplant. Although recent data suggest that there still remains a role for chemotherapeutic agents, targeted modalities have expanded the overall BPDCN treatment landscape. The CD123-targeted agent, tagraxofusp, was the first Food and Drug Administration-approved monotherapy in the treatment of BPDCN. Since its inception, several CD123-targeted and other cell-surface agents have been investigated, with many agents still in the preclinical stages. Although relapsed/refractory disease and central nervous system disease both remain formidable areas of research, there are several promising therapeutic approaches that could have a significant impact on the trajectory of treatment. This review will provide detailed insight on the novel drugs currently in use and those being explored in the management of BPDCN.

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Section: Introductionmentioning

confidence: 99%

Blastic plasmacytoid dendritic cell neoplasm (BPDCN): A promising future in the era of targeted therapeutics

Adimora

Wilson

Pemmaraju

2022

Cancer

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“…We identified 13 case reports of tagraxofusp first-line treatment for BPDCN outside clinicals trials in the literature, with patients at a median age of 63 years (range 12-81), ten (76.9%) male and three female, who received a median of three cycles tagraxofusp (Table 3) (36)(37)(38)(39)(40)(41)(42)(43)(44)(45)(46)(47). Of these, eight patients achieved a CR (61.5%), two a PR (15.4%) and two had stable disease, with a median response duration of 9 months for CR and 4 months for CR and PR combined.…”

Section: Discussionmentioning

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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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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“…While Tagraxofusp has proven efficacious in reducing disease burden, it does come with a notable side effect profile, most frequently encountered in the first cycle of therapy. Notable side effects include hepatotoxicity (88%), thrombocytopenia (49%), and capillary leak syndrome (CLS) (19%) [48,50]. Despite facing numerous obstacles, the effectiveness of CD123-targeted therapy in eliciting a clinical response establishes its role as a crucial element in the management of BPDCN for patients who are undergoing initial treatment as well as those with refractory disease.…”

Section: Anti-cd123 Tagraxofuspmentioning

confidence: 99%

Breakthrough in Blastic Plasmacytoid Dendritic Cell Neoplasm Cancer Therapy Owing to Precision Targeting of CD123

Zanotta,

Galati,

De Filippi

et al. 2024

IJMS

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Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive hematologic cancer originating from the malignant transformation of plasmacytoid dendritic cell precursors. This malignancy progresses rapidly, with frequent relapses and a poor overall survival rate, underscoring the urgent need for effective treatments. However, diagnosing and treating BPDCN have historically been challenging due to its rarity and the lack of standardized approaches. The recognition of BPDCN as a distinct disease entity is recent, and standardized treatment protocols are yet to be established. Traditionally, conventional chemotherapy and stem cell transplantation have been the primary methods for treating BPDCN patients. Advances in immunophenotyping and molecular profiling have identified potential therapeutic targets, leading to a shift toward CD123-targeted immunotherapies in both clinical and research settings. Ongoing developments with SL-401, IMGN632, CD123 chimeric antigen receptor (CAR) T-cells, and bispecific antibodies (BsAb) show promising advancements. However, the therapeutic effectiveness of CD123-targeting treatments needs improvement through innovative approaches and combinations of treatments with other anti-leukemic drugs. The exploration of combinations such as CD123-targeted immunotherapies with azacitidine and venetoclax is suggested to enhance antineoplastic responses and improve survival rates in BPDCN patients. In conclusion, this multifaceted approach offers hope for more effective and tailored therapeutic interventions against this challenging hematologic malignancy.

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Tagraxofusp as first-line treatment for blastic plasmacytoid dendritic cell neoplasm

Cited by 7 publications

References 11 publications

Blastic plasmacytoid dendritic cell neoplasm (BPDCN): A promising future in the era of targeted therapeutics

Blastic plasmacytoid dendritic cell neoplasm (BPDCN): A promising future in the era of targeted therapeutics

Real-world evidence on tagraxofusp for blastic plasmacytoid dendritic cell neoplasm – collected cases from a single center and case reports

Breakthrough in Blastic Plasmacytoid Dendritic Cell Neoplasm Cancer Therapy Owing to Precision Targeting of CD123

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