Multiple Genomic Alterations, Including a Novel AFF4::IRF1 Fusion Gene, in a Treatment-Refractory Blastic Plasmacytoid Dendritic-Cell Neoplasm: A Case Report and Literature Review

Sahin, Yavuz; Wang, Y. Lynn; Pei, Jianming; Mansoor, Nashwa; Styler, Michael; Testa, Joseph R.; Nejati, Reza

doi:10.3390/ijms25010305

Cited by 2 publications

(2 citation statements)

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“…Tagraxofusp was discontinued due to AEs in two cases (15.4%), with ≥ grade 3 CLS in five cases (38.5%) and other ≥ grade 3 AEs in four cases (30.8%). Interestingly, similar to one of our patients, one of the reported cases achieved a CR lasting 4 months after only three doses of tagraxofusp ( 45 ). However, reported cases were younger and had less comorbidities than our cohort.…”

Section: Discussionsupporting

confidence: 87%

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: Discussionsupporting

confidence: 87%

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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“…Innovation can be disruptive and wellstrategized but may fail in the absence of key stakeholders. For example, an innovative gene therapy might complete successful clinical trials but without collaboration with patient associations, the therapy will neither receive regulatory approval nor reach the targeted patient population 76 .…”

Section: Collaboration and Opennessmentioning

confidence: 99%

A Framework for Innovation in Obesity

Levine

2024

MRAJ

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The intersection of innovation and obesity presents a significant opportunity for businesses to address a pressing public health challenge while capitalizing on a growing market demand. This abstract explores the potential for businesses, both existing and new, to innovate within sectors such as food, healthcare, technology, and education to promote healthier lifestyles and combat obesity. With the obesity market estimated at $80 billion annually, entrepreneurs, healthcare providers, and technology developers have ample room for growth and innovation. Existing businesses can reformulate products, offer new services, and advocate for supportive policies, while new ventures can enter the market with innovative solutions targeting obesity-related services and products. Moreover, the DACMAR (Disrupt, Adopt, Collaborate, Manage, Adapt, and Resource) framework provides a structured approach for innovators to navigate the complexities of obesity management. Through the DACMAR framework, stakeholders can drive meaningful progress and innovation in addressing obesity. This framework emphasizes the importance of challenging the status quo, fostering partnerships, effective resource management, and dynamic adaptation to changing circumstances. Similarly, by applying the DACMAR framework's principles, investors can systematically rank different innovations in the obesity domain, ensuring market-readiness, identify go-to-market gaps and align investments with investors’ strategic objectives. This enables objective de-risking to improve investment returns. Furthermore, effective leadership is crucial in driving innovation and change, particularly in complex issues like obesity. Various leadership styles, including transformational, innovative, collaborative, servant, adaptive, and strategic, play essential roles in shaping the direction and impact of obesity prevention and treatment efforts. By leveraging diverse leadership approaches, stakeholders can work together to develop innovative solutions that improve outcomes for individuals and communities affected by obesity. Keywords: obesity, innovation, leadership, business

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Multiple Genomic Alterations, Including a Novel AFF4::IRF1 Fusion Gene, in a Treatment-Refractory Blastic Plasmacytoid Dendritic-Cell Neoplasm: A Case Report and Literature Review

Cited by 2 publications

References 49 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

A Framework for Innovation in Obesity

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