Pivekimab Sunirine in Blastic Plasmacytoid Dendritic Cell Neoplasm

A Andrew A. Lane (Dana-Farber Cancer Institute, Harvard Medical School, Boston, Massachusetts, United States) L Luca Mazzarella (2Early Drug Development for Innovative Therapies Division, IRCCS IEO - Istituto Europeo di Oncologia, Milan, Italy) D David A. Sallman (6Department of Malignant Hematology, Moffitt Cancer Center, Tampa, FL) M Matthew L. Ulrickson (3Section of Hematology, Banner MD Anderson Cancer Center, Gilbert, AZ) G Gary J. Schiller (David Geffen School of Medicine at UCLA, Los Angeles, California, United States) H Harry P. Erba (Duke University, Durham, North Carolina, United States) E Eunice S. Wang (30Roswell Park Cancer Institute, Buffalo, NY) R Roland B. Walter D Daniel J. DeAngelo (1Dana-Farber Cancer Institute, Boston, MA) Y Yining Du (21AbbVie Inc., North Chicago, United States) S Sribalaji Lakshmikanthan (AbbVie, Inc, North Chicago, IL) J Jalaja Potluri (AbbVie Inc., North Chicago, IL, United States) H Hagop M. Kantarjian (Department of Leukemia The University of Texas MD Anderson Cancer Center Houston Texas USA) N Naval G. Daver (Department of Leukemia The University of Texas MD Anderson Cancer Center Houston Texas USA) N Navel Daver N Naveen Pemmaraju (The University of Texas MD Anderson Cancer Center, Houston, Texas, United States) E Eunice Wang (13Roswell Park Comprehensive Cancer Center, Buffalo, United States) D Daniel Deangelo (2Dana Farber Cancer Institute, Boston, United States) A Andrew Lane (2Broad Institute of MIT and Harvard, Cambridge, United States) H Harry Erba (28Duke University Medical Center, Durham, United States) D David Sallman (Moffitt Cancer Cancer and Research Institute, Tampa, Florida, United States) R Roland Walter (1University of Washington, Medicine (Hematology/Oncology), Seattle, United States) A Ahmed Aribi (1City of Hope, Duarte, United States) M Matthew Ulrickson (28Banner MD Anderson Cancer Center, Gilbert, AZ) G Gary Schiller (7David Geffen School of Medicine at UCLA, Los Angeles, United States) P Pau Montesinos (Hospital Universitari i Politecnic La Fe, Valencia, Spain) E Enrico Derenzini (17Oncohematology Division, IEO European Institute of Oncology IRCCS, Department of Health Sciences, University of Milan, Milan, Italy) L Luca Mazzella M Maria Benedetta Giannini (9IRCCS Istituto Romagnolo per lo Studio dei Tumori (IRST) Dino Amadori, Meldola, Italy) G Giovanni Martinelli G Giovanni Marconi (31Hematology Unit, Ospedale S. Maria delle Croci, University of Bologna, Ravenna, Italy) E Eric Deconinck (14Hopital Jean Minjoz, Besancon, France) O Ollivier Legrand (14Hôpital Saint-Antoine, Service d'hématologie clinique et de thérapie cellulaire, Paris, France) D Delphine Lebon (10CHU Amiens Picardie, Unité d'Hématologie clinique et Thérapie Cellulaire, Amiens, France) V Valerio Maisano (7Department of Hematology, Institut Paoli-Calmettes, INSERM UMR 1068, CNRS UMR725, CNRS, Aix-Marseille University, Marseille, France)

Abstract

PURPOSE Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a unique myeloid malignancy with CD123 interleukin-3 receptor-α overexpression and poor prognosis. METHODS This phase I/II, open-label, multicenter study evaluated pivekimab sunirine (PVEK), a novel CD123 antibody-drug conjugate, 0.045 mg/kg once every 3 weeks, in adults with frontline (no previous systemic therapy and de novo BPDCN or coexisting hematologic malignancy) or relapsed/refractory BPDCN (ClinicalTrials.gov identifier: NCT03386513 ) The primary end point in the primary analysis population (PAP; frontline de novo) was composite complete response (CCR; CR+ clinical CR) rate. RESULTS Of 84 patients, 33 had frontline BPDCN (22 de novo [20 in PAP]; 11 with previous or concomitant malignancy) and 51 had relapsed/refractory disease. The median (range) age was 72 (63-76) years. In the PAP (n = 20), the CCR rate was 75% (95% CI, 51 to 91; n = 15; median duration: 10.6 [95% CI, 3.8 to not reached] months) and the median overall survival (OS) was 16.6 (95% CI, 7.2 to not reached) months. Eight of these 15 (53%) patients proceeded to stem-cell transplant (SCT). The corresponding rate for relapsed/refractory disease was 14% (95% CI, 6 to 26; n = 7; median duration: 9.2 [95% CI, 2.4 to not reached] months), and the median OS was 5.8 (95% CI, 3.9 to 8.4) months. Adverse events (AEs) included peripheral edema (54%), fatigue (26%), and infusion-related reactions (26%). Grade ≥3 events included neutropenia (16%), thrombocytopenia (14%), and peripheral edema (12%). Serious AEs included pneumonia (6%) and febrile neutropenia (5%). Two on-treatment cases of reversible veno-occlusive disease (VOD) occurred. Of the total 19 patients who proceeded to SCT, VOD was reported in five patients (four with relapsed/refractory BPDCN). CONCLUSION PVEK, with convenient dosing, led to high, durable responses, especially in frontline BPDCN, and a manageable safety profile.

Article Details

Volume / Issue Vol. 44, Issue 10
Published April 01, 2026
Pages 861-873
ISSN 0732-183X
Publisher Lippincott Williams & Wilkins

Journal Info

Journal of Clinical Oncology

Lippincott Williams & Wilkins

ISSN: 0732-183X Health Sciences

Authors (35)

A

Andrew A. Lane

Dana-Farber Cancer Institute, Harvard Medical School, Boston, Massachusetts, United States

L

Luca Mazzarella

2Early Drug Development for Innovative Therapies Division, IRCCS IEO - Istituto Europeo di Oncologia, Milan, Italy

D

David A. Sallman

6Department of Malignant Hematology, Moffitt Cancer Center, Tampa, FL

M

Matthew L. Ulrickson

3Section of Hematology, Banner MD Anderson Cancer Center, Gilbert, AZ

G

Gary J. Schiller

David Geffen School of Medicine at UCLA, Los Angeles, California, United States

H

Harry P. Erba

Duke University, Durham, North Carolina, United States

E

Eunice S. Wang

30Roswell Park Cancer Institute, Buffalo, NY

R

Roland B. Walter

D

Daniel J. DeAngelo

1Dana-Farber Cancer Institute, Boston, MA

Y

Yining Du

21AbbVie Inc., North Chicago, United States

S

Sribalaji Lakshmikanthan

AbbVie, Inc, North Chicago, IL

J

Jalaja Potluri

AbbVie Inc., North Chicago, IL, United States

H

Hagop M. Kantarjian

Department of Leukemia The University of Texas MD Anderson Cancer Center Houston Texas USA

N

Naval G. Daver

Department of Leukemia The University of Texas MD Anderson Cancer Center Houston Texas USA

N

Navel Daver

N

Naveen Pemmaraju

The University of Texas MD Anderson Cancer Center, Houston, Texas, United States

E

Eunice Wang

13Roswell Park Comprehensive Cancer Center, Buffalo, United States

D

Daniel Deangelo

2Dana Farber Cancer Institute, Boston, United States

A

Andrew Lane

2Broad Institute of MIT and Harvard, Cambridge, United States

H

Harry Erba

28Duke University Medical Center, Durham, United States

D

David Sallman

Moffitt Cancer Cancer and Research Institute, Tampa, Florida, United States

R

Roland Walter

1University of Washington, Medicine (Hematology/Oncology), Seattle, United States

A

Ahmed Aribi

1City of Hope, Duarte, United States

M

Matthew Ulrickson

28Banner MD Anderson Cancer Center, Gilbert, AZ

G

Gary Schiller

7David Geffen School of Medicine at UCLA, Los Angeles, United States

P

Pau Montesinos

Hospital Universitari i Politecnic La Fe, Valencia, Spain

E

Enrico Derenzini

17Oncohematology Division, IEO European Institute of Oncology IRCCS, Department of Health Sciences, University of Milan, Milan, Italy

L

Luca Mazzella

M

Maria Benedetta Giannini

9IRCCS Istituto Romagnolo per lo Studio dei Tumori (IRST) Dino Amadori, Meldola, Italy

G

Giovanni Martinelli

G

Giovanni Marconi

31Hematology Unit, Ospedale S. Maria delle Croci, University of Bologna, Ravenna, Italy

E

Eric Deconinck

14Hopital Jean Minjoz, Besancon, France

O

Ollivier Legrand

14Hôpital Saint-Antoine, Service d'hématologie clinique et de thérapie cellulaire, Paris, France

D

Delphine Lebon

10CHU Amiens Picardie, Unité d'Hématologie clinique et Thérapie Cellulaire, Amiens, France

V

Valerio Maisano

7Department of Hematology, Institut Paoli-Calmettes, INSERM UMR 1068, CNRS UMR725, CNRS, Aix-Marseille University, Marseille, France