-Author name in bold denotes the presenting author
-Asterisk * with author name denotes a Non-ASH member
Clinically Relevant Abstract denotes an abstract that is clinically relevant.

PhD Trainee denotes that this is a recommended PHD Trainee Session.

Ticketed Session denotes that this is a ticketed session.

1516 Combination Chemotherapy in Patients with Newly Diagnosed Blastic Plasmacytoid Dendritic Cell Neoplasms (BPDCN): First Results of a Prospective French Trial (LpDessai)

Program: Oral and Poster Abstracts
Session: 615. Acute Myeloid Leukemias: Commercially Available Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster I
Hematology Disease Topics & Pathways:
Research, clinical trials, Acute Myeloid Malignancies, AML, Clinical Research, Combination therapy, Diseases, Therapies, therapy sequence, Adverse Events, Myeloid Malignancies, Minimal Residual Disease
Saturday, December 9, 2023, 5:30 PM-7:30 PM

Eric Deconinck1, Berengere Gruson, MD, PhD2*, Sabeha Biichle3*, Anne Roggy4*, Florian Renosi5*, Thomas Fournet6*, Maxime Desmarets, MD, PhD7*, Gregory Tio8*, Tony Marchand, MD9*, Mikael Roussel10*, Thibaut Leguay, MD11*, Jean-Philippe Vial, MD12*, Francoise Huguet, MD13*, Francois Vergez14*, Emilie Chalayer, MD, PhD15*, Khaoula Mahfoudi16*, Fressia Honeyman17*, Sebastien Maury18*, Nicolas Freynet19*, Caroline Bonmati, MD20*, Veronique Latger-Cannard21*, Eve Gehlkopf, MD22*, Caroline Bret23*, Damien Roos Weil, MD, PhD24*, Magali Le Garff-Tavernier25*, Sophie Rigaudeau, MD26*, Gandhi Laurent Damaj, MD, PhD27*, Edouard Cornet, MD28*, Remy Gressin, MD29*, Marie-Christine Jacob30*, Delphine Martineau, MD31*, Aguirre Mimoun32*, Alice Garnier, MD33*, Marion Eveillard34*, Alban Villate, MD35*, Emmanuelle Rault36*, Fanny Delettre, PhD3* and Francine Garnache Ottou, PhD37*

1Besançon University Hospital, Besançon, France
2Clinique de l'Europe, Amiens, FRA
3INSERM, UMR1098-RIGHT, EFS Bourgogne Franche-Comté, Université Bourgogne Franche-Comté, BESANCON, France
4CHU Besançon, Besancon, France
5Université de Franche-Comté, CHU Besançon, EFS, INSERM, UMR RIGHT, BESANCON, France
6CHU Besançon, Besancon, FRA
7Université de Franche-Comté, CHU Besançon, EFS, INSERM, UMR RIGHT, Besançon, France
8Université de Franche-Comté, CHU Besançon, Inserm CIC 1431, Besançon, France
9Hematology Department, CHU Rennes, Rennes, France
10CHU Rennes, Rennes, France
11Hematology, CHU Bordeaux, Hôpital du Haut-Lévêque, Pessac, France
12CHU Bordeaux, PESSAC, France
13IUCT-Oncopole, Toulouse, France
14Institut Universitaire du Cancer Toulouse Oncopole, Toulouse, France
15Institut de Cancerologie Lucien Neuwirth, CHU Saint Etienne, Saint Etienne, FRA
16Institut de cancérologie Loire, Saint Priest en Jarez, France
17Institut de cancérologie de la loire, ST PRIEST EN JAREZ CEDEX, FRA
18Hematology Department, Hôpitaux universitaires Henri Mondor AP-HP & Université Paris Est Créteil, Créteil, France
19Hôpitaux universitaires Henri Mondor AP-HP, Creteil, France
20Service D'Hématologie, Centre Hospitalier Universitaire De Nancy, Nancy, FRA
21Centre Hospitalier De Nancy, Vandoeuvre-lès-Nancy, FRA
22Hematology, CHU Montpellier, Montpellier, FRA
23Hôpital saint Eloi, CHU Montpellier, MONTPELLIER, France
24Clinical Hematology, APHP, La Pitié Salpétriere, Sorbonne Universite, Paris, FRA
25Hematology Laboratory, Assistance Publique-Hôpitaux de Paris, Pitie-Salpetriere Hospital, Paris, France
26CH Versailles, Le Chesnay Rocquencourt, FRA
27CHU Caen, Caen, Normandy, FRA
28CHU de Caen, Caen Cedex9, FRA
29HOPITAL ALBERT MICHALLON, Department of Hematology, University Hospital Grenoble, Grenoble, France
30CHU Grenoble Alpes, La Tronche, France
31CH Côte Basque, Bayonne, FRA
32Service D'hématologie Biologique, CHU De Bordeaux, Bordeaux, FRA
33Hematology clinic, Nantes University Hospital, Nantes, France
34Hematology biology, CHU Nantes, Nantes, FRA
35CHU Bretonneau, Tours, France
36CHU Tours, Tours, France
37CHU Besancon, Cellular immunology and hematology laboratory, Université de Franche-Comté, CHU Besançon, EFS, INSERM, UMR RIGHT, Besançon, France


Blastic plasmacytoid dendritic cell neoplasms (BPDCN) are now well-characterized diseases clearly referenced in the 5th edition of the World Health Organization (WHO) Classification of Tumors (2022)1 but treatment remains a real challenge. There is no consensus on the first line treatment even if CD123 targeted therapies are available in the USA and some European countries. Standard chemotherapy remains largely used as first line treatment with better results of leukemia-based regimens despite a high toxicity rate in this frail population2,3. To establish a reference chemotherapy scheme, we prospectively evaluated the efficacy and toxicity of a combination chemotherapy (idarubicin, methotrexate, L-asparaginase, and dexamethasone) in newly diagnosed BPDCN patients (pts) in France.

Patients and methods

This single stage phase II study enrolled consecutive adult pts with suspected BPDCN in participating French centers. BPDCN diagnosis was centrally reviewed for cytology and immunophenotype (IF) (Pr Garnache Ottou F, UMR RIGHT BESANCON) and if needed for histology (Dr Petrella T, Montréal, Canada) according to published recommendations1,4. After giving their informed consent, patients were evaluated for tumoral involvement by PET-scan, systematic lumbar puncture, and the mSWAT score was calculated for skin extension. Patients then received three 21 days-cycles of Ida/Metho/L-asp/Dex combination, before evaluation. Eligible patients with complete response (CR), complete response with incomplete bone marrow recovery (CRi) or partial response (PR) underwent allogeneic hematopoietic stem cell transplantation (HCT). Those not eligible received consolidation chemotherapy with 28 days cycles of Metho/L-asp/Dex (Figure 1). The primary objective was the proportion of patients with CR after 3 cycles of chemotherapy. Secondary objectives were the proportion of patients with an objective response (ORR) defined as CR, CRi or PR, the minimal residual disease (MRD) in responding patients evaluated by the presence of plasmacytoid dendritic cell blast measured by flow cytometry in the bone marrow, the incidence of severe adverse events and overall survival (OS).

The competent ethics committee (Comité de Protection des Personnes Île de France 8) approved the study. The Besançon University hospital promoted the trial, financed by a grant of the French National Cancer Institute (INCa-DGOS_11093).


Twenty-eight pts, originating from 16 centers, were screened between May 2019 and March 2023. Two patients with a misdiagnosis were screen failures. Two patients did not receive the planned chemotherapy due to clinical deterioration; 24 patients (21 male, 3 female) received at least one chemotherapy cycle and 23 are analyzed with a current end-point on June 30, 2023. Median age was 65y (21-79y). ECOG status was 0-1 in 20 cases, and 2 in 3 cases. A cutaneous involvement was identified in 16 pts (70%) and an extra medullary involvement was present in 13 (62%) pts: spleen, n= 6 (25%); lymph nodes, n= 7 (30%). Twenty-three pts (90%) had a bone marrow infiltration (identified only on IF in 4 cases), and 3 cases (14%) a documented central nervous system involvement.

Nine pts had to stop planned treatment due to severe adverse events (3 deaths, 4 acute renal failure and 2 grade 4 febrile neutropenia) and in 4 pts, at least one cycle had to be delayed.

Among the 15 pts receiving at least 3 cycles, 12 (80% and 50% of the whole cohort) were in ORR (CR = 8, CRi = 2, PR = 2) after the 3rd cycle of chemotherapy, 2 pts did not respond. Eight of ten (80%) CR/CRi pts had a MRD < 10-4 without any further relapse. Nine of 12 responding patients (75%) received an allogeneic HCT 1-2 months after the end of the 3rd cycle. Global OS at 6 months for responding or failing pts were 100% and 37% respectively (Figure 2).


In selected pts, an adapted chemotherapy could offer high CR rate in pts able to follow the planned treatment but remains toxic in frail pts, with only half of the them achieving a 3rd cycle of chemotherapy. We confirm that pts achieving ORR and receiving HCT obtain prolonged CR. MRD level seems to correlate with OS and could be a useful tool to manage treatment toxicity in BPDCN frail pts.


  1. Khoury JD et al., Leukemia (2022) Jul;36(7):1703-1719
  2. Laribi et al. Blood Adv (2020) 4 (19): 4838–4848.
  3. Garnache-Ottou et al., Blood Adv (2019) 3 (24): 4238
  4. Philippe L et al., Haematologica (2017) ; Nov;102(11):1861-1868

Disclosures: Deconinck: GILEAD KITE: Other: Hospitality, Research Funding; Immunogen Inc.: Honoraria; NOVARTIS: Research Funding; STEMLINE MENARINI: Consultancy. Marchand: Astellas: Consultancy; Sobi: Consultancy; Jazz Pharmaceuticals: Consultancy, Other: travel fees; Servier: Consultancy, Other: Travel fees. Huguet: PFIZER: Honoraria; INCYTE: Honoraria; NOVARTIS: Honoraria. Le Garff-Tavernier: Novartis: Honoraria; Janssen: Honoraria; Alexion: Consultancy, Honoraria. Damaj: Takeda, Blueprint Medicines Corporation, and Thermo Fisher: Consultancy, Other: Advisory Role; Takeda, AbbVie and Pfizer: Other: Travel and Accommodation Expenses. Delettre: Stemline Menarini: Research Funding; Immunogen Inc.: Research Funding. Garnache Ottou: Immunogen Inc.: Research Funding; Stemline Menarini: Research Funding.

*signifies non-member of ASH