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Long-Term Benefits of Tagraxofusp for Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm

Clinical trials frequently include multiple end points that mature at different times. The initial report, typically based on the primary end point, may be published when key planned co-primary or secondary analyses are not yet available. Clinical Trial Updates provide an opportunity to disseminate...

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Autores principales: Pemmaraju, Naveen, Sweet, Kendra L., Stein, Anthony S., Wang, Eunice S., Rizzieri, David A., Vasu, Sumithira, Rosenblat, Todd L., Brooks, Christopher L., Habboubi, Nassir, Mughal, Tariq I., Kantarjian, Hagop, Konopleva, Marina, Lane, Andrew A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Wolters Kluwer Health 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9462530/
https://www.ncbi.nlm.nih.gov/pubmed/35820082
http://dx.doi.org/10.1200/JCO.22.00034
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author Pemmaraju, Naveen
Sweet, Kendra L.
Stein, Anthony S.
Wang, Eunice S.
Rizzieri, David A.
Vasu, Sumithira
Rosenblat, Todd L.
Brooks, Christopher L.
Habboubi, Nassir
Mughal, Tariq I.
Kantarjian, Hagop
Konopleva, Marina
Lane, Andrew A.
author_facet Pemmaraju, Naveen
Sweet, Kendra L.
Stein, Anthony S.
Wang, Eunice S.
Rizzieri, David A.
Vasu, Sumithira
Rosenblat, Todd L.
Brooks, Christopher L.
Habboubi, Nassir
Mughal, Tariq I.
Kantarjian, Hagop
Konopleva, Marina
Lane, Andrew A.
author_sort Pemmaraju, Naveen
collection PubMed
description Clinical trials frequently include multiple end points that mature at different times. The initial report, typically based on the primary end point, may be published when key planned co-primary or secondary analyses are not yet available. Clinical Trial Updates provide an opportunity to disseminate additional results from studies, published in JCO or elsewhere, for which the primary end point has already been reported. Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive myeloid malignancy. We report long-term results, including data from the continued access phase, of the largest prospective BPDCN trial evaluating the CD123-targeted therapy tagraxofusp (TAG) in adults with treatment-naive and relapsed/refractory BPDCN. The primary outcome was complete response (CR) + clinical CR (CRc: CR with residual skin abnormality not indicative of active disease). Eighty-four (65 treatment-naive and 19 relapsed/refractory) of 89 patients received TAG 12 μg/kg once daily; the median follow-up was 34.0 months. For treatment-naive patients, the overall response rate was 75%; 57% achieved CR + CRc. The median time to remission was 39 (range, 14-131) days, and the median CR + CRc duration was 24.9 (95% CI, 3.8 to not reached) months. Nineteen patients (51%) with CR + CRc were bridged to stem-cell transplant, with a median CR + CRc duration of 22.2 (range, 1.5-57.4) months. Most common adverse events were increased alanine (64%) or aspartate (60%) aminotransferase and hypoalbuminemia (51%); most occurred in cycle 1 and were transient. Capillary leak syndrome occurred in 21% of patients (grade ≥ 3: 7%). In first-line patients with BPDCN, TAG monotherapy resulted in high and durable responses, allowing many to bridge to stem-cell transplant. TAG was generally well-tolerated with a predictable and manageable safety profile.
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spelling pubmed-94625302022-09-12 Long-Term Benefits of Tagraxofusp for Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm Pemmaraju, Naveen Sweet, Kendra L. Stein, Anthony S. Wang, Eunice S. Rizzieri, David A. Vasu, Sumithira Rosenblat, Todd L. Brooks, Christopher L. Habboubi, Nassir Mughal, Tariq I. Kantarjian, Hagop Konopleva, Marina Lane, Andrew A. J Clin Oncol CLINICAL TRIAL UPDATES Clinical trials frequently include multiple end points that mature at different times. The initial report, typically based on the primary end point, may be published when key planned co-primary or secondary analyses are not yet available. Clinical Trial Updates provide an opportunity to disseminate additional results from studies, published in JCO or elsewhere, for which the primary end point has already been reported. Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive myeloid malignancy. We report long-term results, including data from the continued access phase, of the largest prospective BPDCN trial evaluating the CD123-targeted therapy tagraxofusp (TAG) in adults with treatment-naive and relapsed/refractory BPDCN. The primary outcome was complete response (CR) + clinical CR (CRc: CR with residual skin abnormality not indicative of active disease). Eighty-four (65 treatment-naive and 19 relapsed/refractory) of 89 patients received TAG 12 μg/kg once daily; the median follow-up was 34.0 months. For treatment-naive patients, the overall response rate was 75%; 57% achieved CR + CRc. The median time to remission was 39 (range, 14-131) days, and the median CR + CRc duration was 24.9 (95% CI, 3.8 to not reached) months. Nineteen patients (51%) with CR + CRc were bridged to stem-cell transplant, with a median CR + CRc duration of 22.2 (range, 1.5-57.4) months. Most common adverse events were increased alanine (64%) or aspartate (60%) aminotransferase and hypoalbuminemia (51%); most occurred in cycle 1 and were transient. Capillary leak syndrome occurred in 21% of patients (grade ≥ 3: 7%). In first-line patients with BPDCN, TAG monotherapy resulted in high and durable responses, allowing many to bridge to stem-cell transplant. TAG was generally well-tolerated with a predictable and manageable safety profile. Wolters Kluwer Health 2022-09-10 2022-07-12 /pmc/articles/PMC9462530/ /pubmed/35820082 http://dx.doi.org/10.1200/JCO.22.00034 Text en © 2022 by American Society of Clinical Oncology https://creativecommons.org/licenses/by-nc-nd/4.0/Creative Commons Attribution Non-Commercial No Derivatives 4.0 License: https://creativecommons.org/licenses/by-nc-nd/4.0/
spellingShingle CLINICAL TRIAL UPDATES
Pemmaraju, Naveen
Sweet, Kendra L.
Stein, Anthony S.
Wang, Eunice S.
Rizzieri, David A.
Vasu, Sumithira
Rosenblat, Todd L.
Brooks, Christopher L.
Habboubi, Nassir
Mughal, Tariq I.
Kantarjian, Hagop
Konopleva, Marina
Lane, Andrew A.
Long-Term Benefits of Tagraxofusp for Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm
title Long-Term Benefits of Tagraxofusp for Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm
title_full Long-Term Benefits of Tagraxofusp for Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm
title_fullStr Long-Term Benefits of Tagraxofusp for Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm
title_full_unstemmed Long-Term Benefits of Tagraxofusp for Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm
title_short Long-Term Benefits of Tagraxofusp for Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm
title_sort long-term benefits of tagraxofusp for patients with blastic plasmacytoid dendritic cell neoplasm
topic CLINICAL TRIAL UPDATES
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9462530/
https://www.ncbi.nlm.nih.gov/pubmed/35820082
http://dx.doi.org/10.1200/JCO.22.00034
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