Efficacy and manageable safety of tagraxofusp in blastic plasmacytoid dendritic cell neoplasm: a case series of pediatric and adolescent/young adult patients
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...
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| Format: | Article |
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Wiley
2024-02-01
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| Series: | eJHaem |
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| Online Access: | https://doi.org/10.1002/jha2.856 |
| _version_ | 1827342583663165440 |
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| author | 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 |
| author_facet | 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 |
| author_sort | Naveen Pemmaraju |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-07T22:09:39Z |
| format | Article |
| id | doaj.art-f0c19f1aedd446e39c2ceaabeb4efd0f |
| institution | Directory Open Access Journal |
| issn | 2688-6146 |
| language | English |
| last_indexed | 2024-03-07T22:09:39Z |
| publishDate | 2024-02-01 |
| publisher | Wiley |
| record_format | Article |
| series | eJHaem |
| spelling | doaj.art-f0c19f1aedd446e39c2ceaabeb4efd0f2024-02-23T16:50:43ZengWileyeJHaem2688-61462024-02-0151616910.1002/jha2.856Efficacy and manageable safety of tagraxofusp in blastic plasmacytoid dendritic cell neoplasm: a case series of pediatric and adolescent/young adult patientsNaveen Pemmaraju0Branko Cuglievan1Joseph Lasky2Albert Kheradpour3Nobuko Hijiya4Anthony S. Stein5Soheil Meshinchi6Craig A. Mullen7Emanuele Angelucci8Luciana Vinti9Tariq I. Mughal10Anna B. Pawlowska11Department of Leukemia The University of Texas MD Anderson Cancer Center HoustonTexas USADepartment of Leukemia The University of Texas MD Anderson Cancer Center HoustonTexas USACure 4 The Kids Foundation Las VegasNevada USADepartment of Pediatric Hematology and Oncology Loma Linda University Children's Hospital Loma LindaCalifornia USADivision of Pediatric Oncology, Hematology, and Stem Cell Transplantation Columbia University Irving Medical Center New YorkNew York USADepartment of Hematology and Hematopoietic Cell Transplantation City of Hope National Medical Center DuarteCalifornia USADepartment of Pediatrics University of Washington School of Medicine SeattleWashington USADivision of Pediatric Hematology/OncologyDepartment of Pediatrics Golisano Children's HospitalUniversity of Rochester Rochester New York USAHematology and Cellular Therapy Unit, IRCCS Ospedale Policlinico San Martino GenovaItalyDepartment of Paediatric Haematology/Oncology, Cell and Gene Therapy Bambino Gesù Children's Hospital, IRCCS RomeItalyDivision of Hematology‐Oncology Tufts University Medical School BostonMassachusetts USADepartment of Pediatrics City of Hope National Medical Center DuarteCalifornia USAAbstract 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.https://doi.org/10.1002/jha2.856AYA BPDCNCD123pediatric BPDCNplasmacytoid dendritic cellstagraxofusp |
| spellingShingle | 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 Efficacy and manageable safety of tagraxofusp in blastic plasmacytoid dendritic cell neoplasm: a case series of pediatric and adolescent/young adult patients eJHaem AYA BPDCN CD123 pediatric BPDCN plasmacytoid dendritic cells tagraxofusp |
| title | Efficacy and manageable safety of tagraxofusp in blastic plasmacytoid dendritic cell neoplasm: a case series of pediatric and adolescent/young adult patients |
| title_full | Efficacy and manageable safety of tagraxofusp in blastic plasmacytoid dendritic cell neoplasm: a case series of pediatric and adolescent/young adult patients |
| title_fullStr | Efficacy and manageable safety of tagraxofusp in blastic plasmacytoid dendritic cell neoplasm: a case series of pediatric and adolescent/young adult patients |
| title_full_unstemmed | Efficacy and manageable safety of tagraxofusp in blastic plasmacytoid dendritic cell neoplasm: a case series of pediatric and adolescent/young adult patients |
| title_short | Efficacy and manageable safety of tagraxofusp in blastic plasmacytoid dendritic cell neoplasm: a case series of pediatric and adolescent/young adult patients |
| title_sort | efficacy and manageable safety of tagraxofusp in blastic plasmacytoid dendritic cell neoplasm a case series of pediatric and adolescent young adult patients |
| topic | AYA BPDCN CD123 pediatric BPDCN plasmacytoid dendritic cells tagraxofusp |
| url | https://doi.org/10.1002/jha2.856 |
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