Haematopoietic cell transplantation for blastic plasmacytoid dendritic cell neoplasm: a North American multicentre collaborative study

Abstract

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is incurable with conventional therapies. Limited retrospective data have shown durable remissions after haematopoietic cell transplantation (HCT) [allogeneic (allo) or autologous (auto)]. We conducted a multicentre retrospective study in BPDCN patients treated with allo-HCT and auto-HCT at 8 centres in the United States and Canada. Primary endpoint was overall survival (OS). The population consisted of 45 consecutive patients who received an allo-HCT (n = 37) or an auto-HCT (n = 8) regardless of age, pre-transplant therapies, or remission status at transplantation. Allo-HCT recipients were younger (50 (14-74) vs. 67 (45-72) years, P = 0·01) and had 1-year and 3-year OS of 68% [95% confidence interval (CI) = 49-81%] and 58% (95% CI = 38-75%), respectively. Allo-HCT in first complete remission (CR1) yielded superior 3-year OS (versus not in CR1) [74% (95% CI = 48-89%) vs. 0, P < 0·0001]. Allo-HCT outcomes were not impacted by regimen intensity [3-year OS for myeloablative conditioning = 61% (95% CI = 28-83%) vs. reduced-intensity conditioning = 55% (95% CI = 28-76%)]. One-year OS for auto-HCT recipients was 11% (95% CI = 8-50%). These results demonstrate efficacy of allo-HCT in BPDCN, especially in patients in CR1. Pertaining to auto-HCT, our results suggest lack of efficacy against BPDCN, but this observation is limited by the small sample size. Larger prospective studies are needed to better define the role of HCT in BPDCN.

Cladribine‐based salvage regimen‐induced deep remission of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in leukemia phase

“…Although several reports have revealed that patients with BPDCN normally responded well with initial chemotherapy, unfortunately, the patient of our case responded the other way. Even after several cycles of intensive chemotherapies.…”

<p>Case Report of Anti-CD123 Chimeric Antigen Receptor T-Cell Therapy Followed by Radiotherapy for a Recurrence of Blastic Plasmacytoid Dendritic Cell Neoplasm After Allogeneic Hematopoietic Stem Cell Transplantation</p>

“…6,7 Allo-HSCT therapy is more effective than autologous HSCT therapy. 8 In recent years, other treatments have been used for BPDCN, especially for recurrent/refractory (R/R) patients. These new treatments and clinical trials for R/R BPDCN patients included Elzonris (tagraxofusp SL-401), which targets CD123 on tumor cells regardless of primary patients or R/R patients, 9 daratumumab, which targets CD38 on tumor cells for only a few patients, 10 pralatrexate, which is used for progression of BPDCN, 11 venetoclax, which promotes tumor cell apoptosis, 12 bortezomib, which downregulates NF-kB activity, 13 and 5ʹ-azacytidine, which reduces tumor cell methylation levels.…”

Blastic Plasmacytoid Dendritic Cell Neoplasm in a pediatric Peruvian patient

“…Protocol for ALL includes prophylaxis of the nervous system with intrathecal chemotherapy, since it is considered one of the main causes of morbidity and mortality in patients with this disease 7 . Bone marrow transplantation is usually reserved for cases with one or multiple relapses; in elderly patients is also used as a consolidation therapy after chemotherapy 8,11,13 . However, in pediatric patients it does not improve survival 2 .…”