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4234 Clinical Features and Treatment in Patients Diagnosed with Blastic Plasmacytoid Dendritic Cell Neoplasm: Interim Analysis from the Pethema Epidemiologic Registry (EPI-BLAS study)

Program: Oral and Poster Abstracts
Session: 613. Acute Myeloid Leukemias: Clinical and Epidemiological: Poster III
Hematology Disease Topics & Pathways:
Acute Myeloid Malignancies, AML, Diseases, Myeloid Malignancies
Monday, December 11, 2023, 6:00 PM-8:00 PM

Irene Navarro Vicente1,2*, Pilar Lloret Madrid1,2*, Antonio Solana-Altabella, PharmD2,3*, Pilar Martinez Sanchez, M.D.4*, Maria Angeles Foncillas5*, Carlos Cervero, MD6*, Victor Noriega7*, Maria Paz Garrastazul Sánchez8*, Jose Maria Alonso Alonso9*, Beatriz De Rueda Ciller10*, Pilar Herrera Puente11*, Susana Vives12*, Josefina Serrano, MD13*, Lourdes Hermosín, PhD14*, Fernando Jesús Ramos-Ortega, MD15*, Teresa Bernal16*, Jesús Lorenzo Algarra17*, Mercedes Colorado18*, Raimundo García-Boyero19*, Juan Miguel Bergua Burgues20*, Lisette Costilla-Barriga21*, Tamara Castaño, MD22*, Jorge Labrador23*, Armando Mena Duran24*, Maria Dolores Madrigal Toscano25*, Maria Del Mar Hermosilla-Fernandez26*, Carmen Couto27*, Esther Perez Santaolla, MD28*, Jose Mario Mariz, MD29, Sandra Casal Marini30*, Cristina Gil, MD31*, David Martinez-Cuadron, PhD1,2* and Pau Montesinos, PhD, MD1*

1Hematology Department, Hospital Universitari i Politècnic La Fe, Valencia, Spain
2Hematology Department, Instituto de Investigación Sanitaria La Fe (IISLAFE), Valencia, Spain
3Pharmacy Department, Hospital Universitari i Politècnic La Fe, Valencia, Spain
4Hematology Department, Hospital Universitario 12 de Octubre, Madrid, Spain
5Hospital Universitario Infanta Leonor, Madrid, ESP
6Hospital Virgen de la Luz, Cuenca, Spain
7Hospital Clínico de A Coruña, A Coruña, Spain
8Hospital Universitario Puerta del Mar, Cádiz, Spain
9Hospital Rio Carrión, Palencia, Palencia, ESP
10Hospital Miguel Servet, Zaragoza, Spain
11Hospital Universitario Ramon y Cajal, Madrid, ESP
12Hospital Germans Trias i Pujol, Badalona, ESP
13Hospital Universitario Reina Sofía, IMIBIC, Cordoba, ESP
14Department of Hematology, Hospital Jerez de la Frontera, Jerez de la Frontera, Cádiz, Spain
15Hospital Universitario de León, León, Spain
16Hospital Universitario Central de Asturias, Instituto Universitario (IUOPA), Instituto de Investigación del Principado de Asturias (ISPA), Oviedo, Spain
17Hospital General de Albacete, Albacete, ESP
18Hospital Universitario Marqués De Valdecilla, Santander, Spain
19Hospital General De CastellóN, CastellóN, ESP
20Hospital San Pedro de Alcántara, Caceres, Spain
21Hospital San Jorge, Zaragoza, ESP
22Hospital Fundación Jiménez Díaz, Madrid, Spain
23Hospital Universitario de Burgos, Universidad Isabel I, Burgos, ESP
24Hospital General de Valencia, Valencia, ESP
25Hospital Universitario Virgen Macarena, Sevilla, ESP
26Hospital San Pedro, Logroño, ESP
27Hospital Virgen de Valme, Sevilla, ESP
28Hospital Donostia, Donostia, Spain
29Instituto Português Oncologia do Porto Francisco Gentil, Oporto, Portugal
30Centro Hospitalar e Universitário de Coimbra, Coimbra, Portugal
31Hospital General Universitario de Alicante, Alicante, ESP


BPDCN (Blastic Plasmacytoid Dendritic Cell Neoplasm) is a rare and aggressive hematological neoplasm, and due to its rarity there are scarce registry studies analyzing the disease in real-world. The proposed study seeks to fill this knowledge gap and gain insights into various aspects of the disease, including demographics, presentation features, biological data, treatment patterns, and individual patient outcomes. Ultimately, the study aims to shed light on how to best manage BPDCN and identify patients who could benefit from curative treatments, including front-line agents followed by allogeneic stem cell transplant (allo-HSCT) and novel therapies, like IL3R-diphteric toxin compound (SL-140; Tagraxofusp) which showed remarkable activity in front-line therapy.


The EPI-BLAS is a retrospective, multicenter, non-interventional chart review study of patients diagnosed with BPDCN. Approximately 150 patients treated at PETHEMA participating sites in Spain will be included in final analyses. The review will focus on medical records of patients diagnosed with BPDCN, and the data will be entered into an electronic case report form (eCRF) for all patients who meet the inclusion and exclusion criteria. For this analysis, we included a first cohort of 68 adult patients (≥18 years old) who were diagnosed BPDCN between January 2010 and January 2022.


The median age at diagnosis was 66 years (range 18-87 years). The majority of the patients (74%) was de novo, while 26% of them had a prior history of neoplastic disease [43% of them had prior solid neoplasm (thyroid cancer, prostate adenocarcinoma, melanoma and breast ductal carcinoma) and 57% suffered from hematological neoplasms as large granular lymphocytic leukemia, myelodysplastic syndromes, non-Hodgkin T-cell lymphoma and chronic myelomonocytic leukemia]. Previous anti-cancer therapy was received by 36 patients. Table 1 shows main baseline characteristics of BPDCN patients.

Regarding first-line treatment, 34 patients (50%) received AML-like regimens [19 underwent anthracycline and cytarabine (3+7 regimen), 4 FLAGIDA, 4 received FLUGA (fludarabine plus low-dose cytarabine), 1 cytarabine alone, and 1 azacitidine alone]; 9 (13%) acute lymphoblastic leukemia-like regimens; 8 patients (12%) were treated with lymphoma–like regimens; 6 (9%) received medication within clinical trial; 2 (3%) compassionate use of Tagraxofusp, and 9 (13%) supportive care only.

Among 59 treated patients, front-line treatment resulted in 27 (47%) complete remission, 1 (2%) morphologic leukemia-free state (MLFS), 6 (10%) partial remission, 14 (24%) showed resistance, 7 (12%) died during induction (causes of death were cerebral bleeding [3] and multiorgan failure [4]), and 4 (7%) had not available response. Of among 42 treated patients with available post-remission treatment, 9 (21%) received an allogeneic hematopoietic stem cell transplant in first CR. With a median follow-up of 9.6 months, the median overall survival of the cohort was 8.58 months. Median OS was 6 months in patients not transplanted and not reached among those transplanted in first CR (p=0.000695) (Figure 1).


Our preliminary analysis confirms the dismal prognosis of this rare entity (median OS of 8.6 months). We found large heterogeneity of therapeutic approaches, low complete remission rates and scarce rates of allogeneic transplant in the front-line. Improvement of outcomes through well-defined treatment protocols and innovative agents are needed for this high unmet need disease.

Disclosures: Martinez Sanchez: Pfizer: Honoraria, Other: Support for attending meetings; Jazzpharma: Honoraria, Other: Support for attending meetings; Incyte: Honoraria; Abbvie: Other: Support for attending meetings; BMS: Other: support for attending meetings. Bergua Burgues: Hospital San Pedro de Alcántara. Servicio de Hematologia. Cáceres. SPAIN: Current Employment; Daychii: Consultancy; Fundesalud. Grants of Europena funds.Daychii: Research Funding. Martinez-Cuadron: Otsuka: Consultancy, Other: Travel, Accommodations; Astellas: Consultancy, Speakers Bureau; Pfizer: Other: Travel, Accommodations. Montesinos: Janssen: Speakers Bureau; Celgene: Consultancy; Daiichi Sankyo: Consultancy, Research Funding; BMS: Consultancy, Other, Research Funding; Pfizer: Consultancy, Research Funding, Speakers Bureau; Abbvie: Consultancy, Research Funding, Speakers Bureau; Jazz pharma: Consultancy, Research Funding, Speakers Bureau; Menarini-Stemline: Consultancy, Research Funding; Kura oncology: Consultancy; Ryvu: Consultancy; Astellas: Consultancy, Speakers Bureau; Novartis: Consultancy, Research Funding; Takeda: Consultancy, Research Funding; GILEAD: Consultancy; OTSUKA: Consultancy; BEIGENE: Consultancy; INCYTE: Consultancy; NERVIANO: Consultancy.

*signifies non-member of ASH