Allogeneic hematopoietic cell transplantation for blastic plasmacytoid dendritic cell neoplasm: a CIBMTR analysis

• DOI: 10.1182/bloodadvances.2023011308
• Publication Date: 2023-10-04T17:40:28Z
• Authors: Hemant S. Murthy; Mei-Jie Zhang; Karen Chen; Sairah Ahmed; Uday Deotare; Siddhartha Ganguly; Ankit Kansagra; Fotios V. Michelis; Taiga Nishihori; Mrinal Patnaik; Muhammad Bilal Abid; Mahmoud Aljurf; Yasuyuki Arai; Ulrike Bacher; Talha Badar; Sherif M. Badawy; Karen Ballen; Minoo Battiwalla; Amer Beitinjaneh; Nelli Bejanyan; Vijaya Raj Bhatt; Valerie I. Brown; Rodrigo Martino; Jean-Yves Cahn; Paul Castillo; Jan Cerny; Saurabh Chhabra; Edward Copelan; Andrew Daly; Bhagirathbhai Dho...; Miguel Angel Diaz...; César O. Freytes; Michael R. Grunwald; Shahrukh Hashmi; Gerhard C. Hildeb...; Omer Jamy; Jacinth Joseph; Christopher G. Ka...; Nandita Khera; Maxwell M. Krem; Yachiyo Kuwatsuka; Hillard M. Lazarus; Lazaros J. Lekakis; Hongtao Liu; Dipenkumar Modi; Pashna N. Munshi; Alberto Mussetti; Neil Palmisiano; Sagar S. Patel; David A. Rizzieri; Sachiko Seo; Mithun Vinod Shah; Akshay Sharma; Melhm Sohl; Scott R. Solomon; Matthew Ulrickson; Celalettin Ustun; Marjolein van der...; Leo F. Verdonck; John L. Wagner; Trent Wang; Baldeep Wirk; Amer Zeidan; Mark Litzow; Partow Kebriaei; Christopher S. Ho...; Daniel J. Weisdorf; Wael Saber; Mohamed A. Kharfa...
• Keywords: Homologous; Transplantation; Myeloproliferative Disorders; Hematopoietic Stem Cell Transplantation; 610; 610 Medicine & health; Hematology; Dendritic Cells; Middle Aged; 3. Good health; Neoplasm Recurrence; Local; Oncology; Recurrence; 616; Acute Disease; Chronic Disease; Medicine and Health Sciences; Humans; Transplantation, Homologous; Neoplasm Recurrence, Local

Abstract Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematological malignancy with a poor prognosis and considered incurable with conventional chemotherapy. Small observational studies reported allogeneic hematopoietic cell transplantation (allo-HCT) offers durable remissions in patients with BPDCN. We report an analysis of patients with BPDCN who received an allo-HCT, using data reported to the Center for International Blood and Marrow Transplant Research (CIBMTR). We identified 164 patients with BPDCN from 78 centers who underwent allo-HCT between 2007 and 2018. The 5-year overall survival (OS), disease-free survival (DFS), relapse, and nonrelapse mortality (NRM) rates were 51.2% (95% confidence interval [CI], 42.5-59.8), 44.4% (95% CI, 36.2-52.8), 32.2% (95% CI, 24.7-40.3), and 23.3% (95% CI, 16.9-30.4), respectively. Disease relapse was the most common cause of death. On multivariate analyses, age of ≥60 years was predictive for inferior OS (hazard ratio [HR], 2.16; 95% CI, 1.35-3.46; P = .001), and higher NRM (HR, 2.19; 95% CI, 1.13-4.22; P = .02). Remission status at time of allo-HCT (CR2/primary induction failure/relapse vs CR1) was predictive of inferior OS (HR, 1.87; 95% CI, 1.14-3.06; P = .01) and DFS (HR, 1.75; 95% CI, 1.11-2.76; P = .02). Use of myeloablative conditioning with total body irradiation (MAC-TBI) was predictive of improved DFS and reduced relapse risk. Allo-HCT is effective in providing durable remissions and long-term survival in BPDCN. Younger age and allo-HCT in CR1 predicted for improved survival, whereas MAC-TBI predicted for less relapse and improved DFS. Novel strategies incorporating allo-HCT are needed to further improve outcomes.