Efficacy and manageable safety of tagraxofusp in blastic plasmacytoid dendritic cell neoplasm: a case series of pediatric and adolescent/young adult patients
Naveen Pemmaraju,
Branko Cuglievan,
Joseph Lasky,
Albert Kheradpour,
Nobuko Hijiya,
Anthony S. Stein,
Soheil Meshinchi,
Craig A. Mullen,
Emanuele Angelucci,
Luciana Vinti,
Tariq I. Mughal,
Anna B. Pawlowska
Affiliations
Naveen Pemmaraju
Department of Leukemia The University of Texas MD Anderson Cancer Center HoustonTexas USA
Branko Cuglievan
Department of Leukemia The University of Texas MD Anderson Cancer Center HoustonTexas USA
Joseph Lasky
Cure 4 The Kids Foundation Las VegasNevada USA
Albert Kheradpour
Department of Pediatric Hematology and Oncology Loma Linda University Children's Hospital Loma LindaCalifornia USA
Nobuko Hijiya
Division of Pediatric Oncology, Hematology, and Stem Cell Transplantation Columbia University Irving Medical Center New YorkNew York USA
Anthony S. Stein
Department of Hematology and Hematopoietic Cell Transplantation City of Hope National Medical Center DuarteCalifornia USA
Soheil Meshinchi
Department of Pediatrics University of Washington School of Medicine SeattleWashington USA
Craig A. Mullen
Division of Pediatric Hematology/OncologyDepartment of Pediatrics Golisano Children's HospitalUniversity of Rochester Rochester New York USA
Emanuele Angelucci
Hematology and Cellular Therapy Unit, IRCCS Ospedale Policlinico San Martino GenovaItaly
Luciana Vinti
Department of Paediatric Haematology/Oncology, Cell and Gene Therapy Bambino Gesù Children's Hospital, IRCCS RomeItaly
Tariq I. Mughal
Division of Hematology‐Oncology Tufts University Medical School BostonMassachusetts USA
Anna B. Pawlowska
Department of Pediatrics City of Hope National Medical Center DuarteCalifornia USA
Abstract Blastic plasmacytoid dendritic cell neoplasm (BPDCN) predominantly occurs in adults ≥60 years old; 10–20% of cases are pediatric or adolescent/young adult (AYA) patients. Tagraxofusp (TAG, Elzonris®) is the only approved treatment for BPDCN; in the United States it is approved for patients aged ≥2 years. Data on treating pediatric and AYA BPDCN patients are limited. We present a case series of pediatric and AYA patients with BPDCN treated with TAG. Eight patients (five newly diagnosed; three relapsed/refractory [R/R]), aged 2–21 years, received 12 mcg/kg TAG. Seven patients were female; most had skin (n = 6) and/or bone marrow (n = 4) involvement. No new safety signals were identified. Grade 3 adverse events were headache (n = 1) and transaminitis (n = 2). Three patients with newly diagnosed BPDCN achieved complete response, one achieved partial response, and one had stable disease (SD). One patient with R/R BPDCN achieved a minor response; one had SD. Seven patients (88%) were bridged to stem cell transplant: 80% of newly diagnosed patients and 100% of R/R patients. Five patients remained alive at last follow‐up. These cases highlight the efficacy and safety of TAG in pediatric and AYA patients for whom there is no other approved BPDCN therapy.