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320 Treatment Approaches for Blastic Plasmacytoid Dendritic Cell Neoplasm: Data from the Pethema and PALG Epidemiologic Registry

Program: Oral and Poster Abstracts
Type: Oral
Session: 615. Acute Myeloid Leukemias: Clinical and Epidemiological: Treatments and Outcomes in AML in Specific Age Groups, and in Blastic Plasmacytoid Dendritic Cell Neoplasms
Hematology Disease Topics & Pathways:
Lymphoid Leukemias, ALL, Acute Myeloid Malignancies, AML, Adult, Elderly, Chemotherapy, Supportive Care, Diseases, Treatment Considerations, Lymphoid Malignancies, Non-Biological therapies, Young adult , Myeloid Malignancies, Pharmacology, Study Population, Human
Saturday, December 7, 2024: 4:15 PM

Antonio Solana-Altabella, PharmD1,2*, Irene Navarro Vicente1,3*, Marta Sobas, MD4*, Josefina Serrano, MD5*, Magdalena Karasek, MD6*, Manuel Perez Encinas, MD7*, Jorge Labrador, MD8*, Cristina de la Fuente, MD9*, Olga Salamero, MD10*, Rafael Colmenares11*, Tamara Castaño, MD12*, Lorenzo Algarra, MD13*, María García-Fortes, MD14*, Maria Dunia De Miguel, MD15*, Fernanda Trigo, MD16*, Delfim Duarte, MD, PhD17, Virginia Pradillo Fernandez, MD18*, Maria Angeles Foncillas, MD19*, Victor Noriega Concepción, MD20*, Pedro Paúl Vidaller, MD21*, Teresa Bernal Del Castillo, MD, PhD22*, Juan Miguel Miguel Bergua Burgues23*, Gabriela Rodriguez Macias, MD24, Eduardo Rodriguez Arboli, MD MPhil25*, Isabel Simon, MD26*, Nerea Caminos, MD27*, Ada Esteban-Figuerola, MD28*, Joana Brioso Infante, MD, MSc29*, Monica Alejandra Romero, MD30*, Rolando Omar Vallansot, MD31*, Lukasz Targonski, MD32*, Emilia Ulenberg, MD33*, Krzysztof Lewandowski, MD34*, Anna Armatys, MD35*, Renata Guzicka-Kazmierczak, MD, PhD36*, Małgorzata Sobczyk-Kruszelnicka, MD37*, Andrzej Szczepaniak, MD38*, Beatriz De Rueda Ciller, MD39*, Esperanza Lavilla-Rubira, MD40*, Maria Paz Garrastazul Sánchez, MD41*, Jackeline Solano Tovar, MD42*, Cristina Insua Vilas, MD43*, Maria Dolores Madrigal, MD44*, Laura Lamarca Eraso, MD45*, Mar Tormo, MD46, Carmen Botella, MD, PhD47*, Mercedes Colorado, MD48*, Manuel Barrios García, MD, PhD49*, Rebeca Rodriguez-Veiga, MD, PhD3,50*, Pilar Lloret Madrid, MD3*, David Martinez Cuadron, MD, PhD1,3* and Pau Montesinos, PhD, MD3*

1Department of Hematology, Instituto de Investigación Sanitaria La Fe (IISLAFE), Valencia, Spain
2Department of Pharmacy, Hospital Universitari i Politècnic La Fe, Valencia, Spain
3Department of Hematology, Hospital Universitari i Politècnic La Fe, Valencia, Spain
4Department of Hematology, Blood Neoplasms and Bone Marrow Transplantation, Medical University of Wroclaw, Wroclaw, Poland
5Department of Hematology, Hospital Universitario Reina Sofía, IMIBIC.UCO, Cordoba, ESP
6Department of Hematology, Blood Neoplasms and Bone Marrow Transplantation, Wroclaw Medical University, Wroclaw, Poland
7Department of Hematology, Hospital Unviersitario de Santiago de Compostela, IDIS, Santiago de Compostela, ESP
8Department of Hematology, Hospital Universitario de Burgos, Universidad Isabel I, Burgos, Spain
9Department of Hematology, Institut Català d’Oncologia - Hospital Universitari Germans Trias i Pujol, Institut de Recerca Contra la Leucèmia Josep Carreras, Universitat Autònoma de Barcelona, Badalona, Spain
10Hematology Department, Vall d'Hebron University Hospital, University Autònoma of Barcelona (UAB), Barcelona, Spain
11Department of Hematology, Hospital Universitario 12 de Octubre, Madrid, Spain
12Department of Hematology, Hospital Fundación Jiménez Díaz, Madrid, Spain
13Department of Hematology, Hospital General de Albacete, Albacete, Spain
14Department of Hematology, Hospital Universitario Virgen De La Victoria, Málaga, Spain
15Department of Hematology, Hospital Universitario de Guadalajara, Guadalajara, ESP
16Department of Clinical Hematology, Hospital S João-Porto, Porto, Portugal
17Department of Hematology, Instituto Português Oncología do Porto Francisco Gentil, Porto, Portugal
18Department of Hematology., Hospital Universitario Quirónsalud, Madrid, Spain
19Department of Hematology, Hospital Universitario Infanta Leonor, Madrid, ESP
20Complejo Hospitalario Universitario de A Coruña, A Coruña, ESP
21Department of Hematology, Hospital Clínico Universitario Lozano Blesa, Zaragoza, Spain
22Department of Hematology, Hospital Universitario Central de Asturias, Instituto Universitario (IUOPA), Instituto de Investigación del Principado de Asturias (ISPA), Oviedo, Spain
23Department of Hematology, Hospital San Pedro de Alcántara, Caceres, Spain
24Department of Hematology, Hospital Universitario Gregorio Maranon, Madrid, Spain
25Department of Hematology, Hospital Universitario Virgen del Rocío, Instituto de Biomedicina de Sevilla (IBIS/CSIC), University of Seville, Sevilla, Spain
26Department of Hematology, Hospital Virgen de Valme, Sevilla, ESP
27Hospital Universitario De Donostia, Donostia-San Sebastián, ESP
28Hospital San Pedro, Logroño, ESP
29Department of Hematology, Centro Hospitalar Universitario Lisboa Norte - Hospital de Santa Maria, Lisboa, Portugal
30Hematology Division, Hospital Guillermo Grant Benavente, Concepcion, CHL
31Department of Hematology, Hospital Universitari Joan XXIII. ICO-Tarragona, Tarragona, Spain
32National Research Institute of Oncology, Warsaw, Poland
33Department of Laboratory Medicine, Medical University of Gdansk, Gdansk, Poland
34Department of Hematology and Transplantology, Medical University of Gdansk, Gdansk, Poland
35Department of Hematology and Transplantology, Medical University in Katowice, Katowice, Poland
36Department of Hematology and Transplantology, Medical University in Szczecin, Szczecin, Poland
37National Research Institute of Oncology, Gliwice, Poland
38Department of Hematology and Bone Marrow Transplantation, Poznan University of Medical Sciences, Poznan, Poland
39Department of Hematology, Hospital Universitario Miguel Servet, Zaragoza, Spain
40Hospital Universitario Lucus Agusti, Lugo, Spain
41Hospital Universitario Puerta del Mar, Cádiz, Spain
42Department of Hematology, Hospital Rio Carrión - Complejo Asistencial de Palencia, Palencia, Spain
43Department of Hematology, Hospital Povisa, VIgo, Spain
44Department of Hematology, Hospital Universitario Virgen Macarena, Sevilla, ESP
45Department of Hematology, Hospital Santa Ana de Motril, Motril, Spain
46Department of Hematology and Medical Oncology,, Hospital Clínico Universitario de Valencia. INCLIVA Biomedical Research Institute, Valencia, Spain
47Department of Hematology, Hospital General Universitario De Alicante, Alicante, Spain
48Department of Hematology, Hospital Universitario Marqués De Valdecilla, Santander, Spain
49Department of Hematology, Hospital Regional Universitario de Málaga, Málaga, Spain
50Department of Hematology, Instituto de Investigación Sanitaria La Fe, Valencia, Spain

Background

BPDCN (Blastic Plasmacytoid Dendritic Cell Neoplasm) is a rare and aggressive hematological malignancy, with heterogeneous therapeutic approaches. Few data are available regarding efficacy of treatment strategies in real world. The study aims to elucidate the optimal management strategies for BPDCN and identify patients who could benefit from curative treatments and novel therapies. This includes the CD33 or CD123 targeted molecules, as tagraxofusp, which has demonstrated activity as a front-line therapy.

Material This multinational retrospective study performed by PALG and PETHEMA (EPI-BLAS study) groups registered BPDCN patients (WHO 2008 or 2016) from June 1999, onwards. We categorized patients into three groups based on the therapeutic approaches: intensive, non-intensive, and supportive care only treatments. Patients receiving intensive schedules were subclassified into 3 groups: treated with Acute Myeloid Leukemia (AML)-like regimens, Acute Lymphocytic Leukemia (ALL)-like regimens, and lymphoma-like regimens. Non-intensive treatments were divided into 4 groups: AML-like, ALL-like, lymphoma-like (including local radiotherapy or intralesional chemotherapy), and targeted agents (anti-CD33 or anti-CD123). We analyzed composite complete remission (CRc) after first-line and overall survival (OS). CRc was defined as the sum of full CR, clinical CR with cutaneous sequelae, clinical CR with hematologic partial response, and clinical CR with incomplete recovery. The different demographic variables of the various treatment subgroups were compared to identify differences.

Results

From 200 patients with follow-up and therapy records, 132 patients (66%) received intensive treatment, 53 (26%) received non-intensive (including anti-CD33 or CD123 targeted agents), and 15 (8%) received supportive care. Among intensive treatment group, 45 (34%) received AML-like (34 with 3+7, 9 FLAG-IDA, and 2 HAM); 60 (46%) ALL-like (55 HyperCVAD and 5 with PETHEMA-ALL), and 27 (20%) lymphoma-like (17 CHOP, 7 etoposide-based, 1 ABVD, and 2 bortezomib-based). The non-intensive group included 21 (40%) AML-like regimens (10 AZA, 5 AZA-VEN, 4 LDAC, and 2 fludarabine+LDAC); 18 (34%) CD123 or CD33 targeted molecules, 9 (16%) ALL-like therapies (vincristine and dexamethasone-based), and 5 (10%) lymphoma-like, including 3 with radiotherapy or intralesional chemotherapy.

As expected, significant difference was found between intensive vs. non-intensive subgroups in age (57 vs. 71 years, p<0.001). There were significant differences in some demographic variables among AML-like vs. ALL-like vs. lymphoma-like vs. targeted agents subgroups: age (63 vs. 56 vs. 63 vs. 66 years; p=0.03), cutaneous involvement (71 vs. 81 vs. 89 vs. 100 %; p=0.04) and CD123 positive (90 vs. 96 vs. 70 vs. 100 %; p=0.02).

Median OS in patients receiving intensive therapy was 15.8 (95% CI 9.7-22.0) months (n=132). From them, 91 patients reached CRc (79%) vs 24 who did not (21%), with a median OS of 32.7 (95% CI 19.0-46.4) vs 7.9 (95% CI 6.2-9.6) months, respectively (p=0.02). Regarding non-intensive treatment subgroup (n=53), median OS was 8.0 (95% CI 4.7-11.4) months. It was longer in the 27 patients who achieved CRc (60%) than in 18 who did not (40%): 17.2 (95% CI 11.2-23.1) vs 7.0 (95% CI 5.6-8.5) months (p=0.005).

There were no statistically significant differences in OS among patients treated with intensive AML-like, ALL-like, and lymphoma-like approaches (10.1 vs. 34.2 vs. 19.1 months, respectively; p=0.08). Regarding non-intensive schedules, median OS were 10.1, 7.3, 3.6 and 6.9 months in the AML-like, ALL-like, lymphoma-like and targeted therapies groups, respectively. No statistically significant differences were observed (p=0.28).

Conclusion

Our findings emphasize the importance of achieving CRc to improve the prognosis of these difficult-to-treat patients. Our study also shows that patients treated with intensive treatment regimens for BPDCN had better OS than those treated with non-intensive therapies. The differences between treatment subgroups, such as age at diagnosis, among others, could influence the differences in OS. Larger studies and observational protocols are needed to identify best upfront regimens for BPDCN.

Disclosures

This study was partially funded by Menarini.

Disclosures: Salamero: Astellas, Jazz, BMS: Consultancy; Jazz, Abbvie: Honoraria. Rodriguez Macias: Astellas: Consultancy, Honoraria, Speakers Bureau; Servier: Consultancy, Honoraria, Speakers Bureau; Jazz Pharmaceuticals: Consultancy, Honoraria, Speakers Bureau; Otsuka: Consultancy, Honoraria, Speakers Bureau; BMS-Celgene: Consultancy, Honoraria, Speakers Bureau; Abbvie: Consultancy, Honoraria, Speakers Bureau. Tormo: AbbVie, Gilead, Pfizer, Astellas, BMS: Honoraria; Janssen, AbbVie, Jazz: Other: Travel grant for attending meetings; SOBI: Other: Data Safety Monitoring Board. Montesinos: Novartis: Consultancy, Research Funding, Speakers Bureau; Jazzpharma: Consultancy, Research Funding, Speakers Bureau; Astellas: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other: research support, Speakers Bureau; Janssen: Membership on an entity's Board of Directors or advisory committees, Other: research support, Speakers Bureau; AbbVie: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other: research support, Research Funding, Speakers Bureau; Daiichi Sankyo, Inc.: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other: research support, Research Funding, Speakers Bureau; Pfizer: Consultancy, Research Funding, Speakers Bureau; Servier: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other: research support, Research Funding, Speakers Bureau; Kura Oncology: Consultancy; Syndax: Consultancy; Glycomimetics: Consultancy.

*signifies non-member of ASH