scholarly journals Blastic plasmacytoid dendritic cell neoplasms: results of an international survey on 398 adult patients

2020 ◽  
Vol 4 (19) ◽  
pp. 4838-4848
Author(s):  
Kamel Laribi ◽  
Alix Baugier de Materre ◽  
Mohamad Sobh ◽  
Lorenzo Cerroni ◽  
Caterina Giovanna Valentini ◽  
...  

Abstract The purpose of this study is to describe the clinical and prognostic features and to evaluate the outcome of different therapeutic approaches among patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) who have been diagnosed and treated in different institutions. A total of 398 patients from 75 centers were included in the study. Treatment consisted of non-Hodgkin lymphoma (NHL)–like regimens in 129 (32.8%) patients and acute leukemia (AL)–like regimens in 113 (23.5%) patients. In 61 (15.5%) and 16 (4.1%) patients, chemotherapy was followed by allogeneic and autologous hematopoietic stem cell transplantation (HSCT), respectively. Twenty-seven (6.9%) patients received radiotherapy, 6 (1.5%) received new agents, and 62 (15.7%) received palliative care. After a median follow-up of 12 months, median overall survival (OS) was 18 months. Patients who received NHL/AL-like regimens, followed by allogeneic HSCT, had the best outcome; median OS was not reached. OS was 65 months for patients who underwent autologous HSCT; 18 months and 14 months, respectively, for those treated with AL-like and NHL-like regimens without consolidation; and 4 months for those receiving palliative care (P < .001). In BPDCN, chemotherapy with lymphoma- or AL-like regimens, followed by transplantation, represents the therapeutic strategy associated with the best outcome. Consolidation with allogeneic HSCT, when feasible, appears superior to autologous HSCT.

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 3441-3441
Author(s):  
André Tichelli ◽  
Eric Beohou ◽  
Myriam Labopin ◽  
Gérard Socié ◽  
Alicia Rovó ◽  
...  

Abstract Background Hematopoietic stem cell transplantation (HSCT) is potentially a curative treatment for a number of life-threatening malignant and non-malignant disorders. Despite significant improvements in outcome over time, long-term survivors are at risk for late complications and their mortality remains higher than expected. Second cancers are well-known late complications, associated with substantial mortality. Although we know the incidence and risk factors for many second cancers there is a paucity of data on their outcome after HSCT. We aimed to estimate the outcome of second cancers after HSCT using a large cohort of patients from the EBMT registry, and to compare their survival to similar cancers from a general population. Method This retrospective observational study was based on the mandatory minimum data set centers report to the EBMT. We identified all patients with second cancers (excluding malignant hematopoietic and lymphoid neoplasms; non-melanoma skin cancers) treated with HSCT in Europe (including Turkey and Israel), between 1977-2015. Primary diagnoses were acute leukemia (23%), chronic leukemia (9%), lymphoma (34%), plasma cell disorders (23%), solid tumors (4%), myelodysplastic syndromes and myeloproliferative neoplasms (6%) and acquired marrow failure syndromes (1%). For the different types of solid tumors, median and overallsurvival werecalculated (from time of diagnosis of the second cancer). Results were analyzed separately for patients treated with autologous and allogeneic HSCT (table 1). For six prevalent second cancers (breast, lung, melanoma, oropharyngeal, colorectal, prostate; table 2), the age-standardized 5-year overall survival (according to the International Cancer Survival Standard; ICSS) of HSCT patients with second cancer diagnosed since the year 2000 was compared to cancer patients from a general population (EUROCARE, European Cancer Registry, period 2000-2007). Results From the EBMT registry 4152 second cancers out of 220617 (1.88%) HSCT patients were extracted: 1450 (1.79%) out of 80784 patients after allogeneic, and 2702 (1.93%) out of 139833 patients after autologous HSCT. The median age of the patients at HSCT and at diagnosis of second cancer was 53 years (range 1.2-86) and 59 years (3.2-88), respectively. For allogeneic HSCT is was 46 years (1.2-73) and 54 years (3.2-77), for autologous HSCT, 56 years (1.7-86) and 62 years (4.5-88), respectively. The median follow-up time since transplantation of all patients was 121 months (range 5-409); it was 146 months (12-409) for allogeneic, and 114 months (5-354) for autologous HSCT. The median follow-up time since diagnosis of second cancer was 35 months (range 0-240); it was 38 months (0-240) for allogeneic, and 33 months (0-228) for autologous HSCT. The distribution and survival outcomes of second cancers are shown on table 1. Overall survival following diagnosis of second cancers depends mostly on the type of cancer. No relevant differences in median survival and 5-year overall survival were seen between patients treated with autologous or allogeneic HSCT (table 1). For second breast, lung and prostate cancers no difference in 5-year overall survival wasobserved,compared to cancer patients from a general population (table 2). Melanoma and colorectal cancers had worse, and oropharyngeal cancer better overall survival, compared to the EUROCARE control group. Conclusion This large population-based analysis on second cancers among HSCT survivors showed that the outcome for patients developing a second cancer after HSCT is mainly dependent on the type of cancer. It seems that for a number of second cancer overall survival is comparable to cancer patients from a general population. A systematic comparison is now required for all post HSCT second cancers. Disclosures No relevant conflicts of interest to declare.


Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 3045-3045
Author(s):  
John D. Dickinson ◽  
Fausto Loberiza ◽  
Victoria Whalen ◽  
Philip J. Bierman ◽  
Julie M. Vose ◽  
...  

Abstract Considerable advancements have been made in our understanding of the biology and treatment of CLL/SLL. Despite these advances, CLL/SLL essentially remains an incurable illness. Hematopoietic stem cell transplantation (HSCT) has been used in an attempt to improve remission duration and survival. However little high level data exists on outcomes for patients (pts) undergoing HSCT for CLL/SLL. We evaluated the long-term survival of 65 CLL/SLL pts who underwent allogeneic or autologous HSCT from 1995 until 2006 at the University of Nebraska Medical Center. The median duration of follow-up for surviving pts is 8.8 years. The median age was 49 years and there was no significant difference in age between the autologous and allogeneic groups. Thirty nine pts underwent allogeneic HSCT (n=25 matched related donor, n=14 matched unrelated donor) and 26 pts underwent autologous HSCT. For the group undergoing allogeneic HSCT, the stem cell source was mobilized peripheral blood progenitor cells in 74% and bone marrow in 26%. In the autologous HSCT group, 81% of pts received peripheral blood and 19% received bone marrow as their stem cell source. In the autologous group there were 19 deaths (10 from progression) over the period of follow-up. In the allogeneic group there were 29 deaths (including 8 from acute regimen related toxicity, 9 from infection, 3 from complications of GVHD, 1 from late pulmonary toxicity, one from PTLD, one from MDS/AML) and 10 pts are alive at end of follow-up. One hundred day mortality was significantly higher in the allogeneic group (20% vs. 6%; p=0.05). For the allogeneic group the cumulative incidence of grade II-IV acute graft versus host disease (GHVD) was 64% (95% confidence interval [CI]=47–76) and the cumulative incidence of chronic extensive GHVD was 50% (95% CI=29–68). One-year progression free survival (PFS) was significantly better among autologous SCT when compared to allogeneic HSCT (77% versus 45%; P=0.006), but at 5 years these differences were no longer apparent. Similarly, one-year overall survival (OS) was significantly better for autologous SCT (81% versus 48%; P=0.003) but at 5 years these differences were no longer significant (49% versus 31%; p=0.15). Among all patients undergoing allogeneic HSCT, 5-year PFS was significantly higher for patients with SLL vs. CLL (36% vs. 25%; P=0.04). In addition, 5 year OS was better for pts with SLL compared to CLL (51% vs. 33%, P=0.06). There was no difference in PFS or OS following autologous SCT between patients with a diagnosis of CLL versus SLL. The group of pts undergoing autologous HSCT demonstrates no plateau on the PFS curve, whereas for pts undergoing allogeneic HSCT there is a suggestion of a plateau in PFS at approximately 25%. In conclusion, CLL/SLL patients undergoing allogeneic SCT had a higher incidence of early treatment related mortality, mainly from regimen related toxicity and infection. In a subgroup analysis pts with CLL appear to have an inferior PFS compared with pts with SLL. This difference may be due to a more prominent underlying immune deficiency in CLL patients that leads to a higher probability of treatment related mortality. Pts with CLL/SLL undergoing autologous HSCT had a lower incidence of treatment related mortality, but there is no evidence of a plateau in progression-free survival.


Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 5893-5893
Author(s):  
Dulcineia Pereira ◽  
Carolina Teixeira ◽  
Sofia Ramalheira ◽  
Patricia Rocha ◽  
Claudia Moreira ◽  
...  

Abstract BACKGROUND: The best treatment strategy in patients with relapsed Follicular Lymphoma (FL) remains controversial. The incorporation of rituximab (R) in the 1st line chemotherapy (CT) regimen and in treatment relapse resulted in better progression-free survival (PFS) but the benefit in overall survival (OS) was observed in only one trial (Hiddemann W. et al, Blood 2006). Hematopoietic stem cell transplant (HSCT) is the only treatment potentially curative, although the ideal time for its implementation remains undefined. AIM: Evaluation of the best treatment strategy and the impact of HSCT in PFS and OS in patients with relapsed FL. METHODS: Retrospective study including 85 patients with relapsed FL followed at a cancer care center between 2000-2012. Selection criteria: treatment naïve patients with the diagnosis of FL; absence of histological transformation at diagnosis and/or during the 1st line treatment. Survival analysis using the Kaplan-Meier method. Type of response defined according to NCCN criteria. RESULTS: Median follow-up of 64 months [4-158]. Disease progression after the 1st line CT was documented in 85 patients (median age 51 years [28-78], 42.4% male). 64 of the 85 patients had an Ann Arbor stage III-IV, of which 85.9% with follicular pattern, 95.3% grade 1/2 and 43.8% FLIPI ≥ 3. All patients underwent one or more CT regimens containing R, except in one case. In this study, 27.1% (n = 23) patients with age ≤ 60 years were submitted to HSCT (52.2% allogeneic HSCT from a related donor versus 47.8% autologous HSCT), almost all with ≥ 2 prior lines of CT (95.6%, n = 22). 78.3% (n = 18) had a CR or PR> 75% at the time of HSCT, and one death related to graft versus host disease was registered. Patients undergoing HSCT had a better PFS than those not transplanted (p = 0.022). A significant improvement in OS was observed in the HSCT subgroup (p = 0.007), especially in those with stage III-IV (p = 0.006). The type of HSCT had no impact on PFS and OS (p> 0.05), perhaps due to the small number of patients and short follow-up. By univariate Cox regression analysis, the number of regimens of CT before HSCT and the histological grade were independent predictors of PFS (p <0.05). The age and the histological grade were independent predictors of OS (p <0.05). CONCLUSION: In this study, HSCT improved PFS and also OS in patients with relapsed FL, especially in patients receiving less than 3 CT regimen, highlighting the importance of completing the HSCT earlier, during the disease’s chemosensitive phase. Our data suggest the curative potential of HSCT in these patients, due to the GVL effect in allogeneic HSCT and/or intensive high-dose CT in autologous HSCT. More studies are needed to validate these observations. Disclosures No relevant conflicts of interest to declare.


Blood ◽  
2007 ◽  
Vol 110 (9) ◽  
pp. 3463-3471 ◽  
Author(s):  
André Tichelli ◽  
Christoph Bucher ◽  
Alicia Rovó ◽  
Georg Stussi ◽  
Martin Stern ◽  
...  

Abstract We assessed incidence and risk factors of cardiovascular events in 265 patients undergoing allogeneic hematopoietic stem-cell transplantation (HSCT) between 1980 and 2000 and who survived at least 2 years. Results were compared with a cohort of 145 patients treated during the same period with autologous HSCT. The median age of patients with allogeneic HSCT at last follow-up was 39 years, and median follow-up was 9 years. Eighteen (6.8%) patients after allogeneic and 3 (2.1%) patients after autologous HSCT experienced an arterial event. The cumulative incidence of first arterial event after allogeneic HSCT was 22.1% (95% CI, 12.0-40.9) at 25 years. The cumulative incidence 15 years after allogeneic HSCT was 7.5% as compared with 2.3% after autologous HSCT. Adjusting for age, risk of an arterial event was significantly higher after allogeneic HSCT (RR 6.92; P =.009). In multivariate analysis, allogeneic HSCT (RR: 14.5; P =.003), and at least 2 of 4 cardiovascular risk factors (hypertension, dyslipidemia, diabetes, obesity) (RR: 12.4; P =.02) were associated with a higher incidence of arterial events after HSCT. Thus, long-term survivors after allogeneic HSCT are at high risk for premature arterial vascular disease. HSCT might favor the emergence of established risk factors, such as hypertension, diabetes, and dyslipidemia.


2017 ◽  
Vol 2017 ◽  
pp. 1-6
Author(s):  
Martina Pennisi ◽  
Clara Cesana ◽  
Micol Giulia Cittone ◽  
Laura Bandiera ◽  
Barbara Scarpati ◽  
...  

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy with aggressive clinical course and poor prognosis. Diagnosis is based on detection of CD4+CD56+,CD123high, TCL-1+, and blood dendritic cell antigen-2/CD303+blasts, together with the absence of lineage specific antigens on tumour cells. In this report we present a case of BPDCN presenting with extramedullary and bone marrow involvement, extensively studied by flow cytometry and immunohistochemistry, who achieved complete remission after acute lymphoblastic leukemia like chemotherapy and allogeneic hematopoietic stem cell transplantation.


Author(s):  
Sadik Husian ◽  
Preethi Jeyaraman ◽  
S. K. Gupta ◽  
Reeta Rai ◽  
Sangeeta Pathak ◽  
...  

Abstract Methods This is a retrospective study. G-CSF was administered in the dose of 10 μg/kg subcutaneous as a single dose for 4 days. On day 5, peripheral blood stem cell (PBSC) apheresis was performed using Haemonetics MCS plus or COBE Spectra apheresis machine through a double-lumen central venous catheter. Primary outcome parameters were the total number of CD34+ HSCs/kg of recipient weight mobilized in peripheral blood and the number of days required for neutrophil and platelets engraftment, respectively. Objective We compared the effectiveness and safety of innovator filgrastim versus generic filgrastim in patients who underwent hematopoietic stem cell transplantation (HSCT). Results A total of 91 stem cell mobilizations was analyzed. There were 58 normal healthy donors for allogeneic HSCT and 33 patients for autologous HSCT. There was no statistically significant difference among groups in terms of total collected CD34+ cells value (p = 0.609). The mean time to neutrophil engraftment was 13.7 days in the innovator group and 13.2 days in the Grafeel group (p = 0.518). The mean time to platelet engraftment was 16.2 days in the innovator group and 14.8 days in the generic group (p = 0.435). The patient who received generic filgrastim had more febrile episodes during the course of transplantation (p = 0.020). Conclusion Generic filgrastim was found to be comparable to original filgrastim for peripheral blood stem cell mobilization in normal healthy donors for allogeneic HSCT and patients for autologous HSCT.


2013 ◽  
Vol 88 (1) ◽  
pp. 131-133 ◽  
Author(s):  
Paula Maio ◽  
Candida Fernandes ◽  
Ana Afonso ◽  
Fernanda Sachse ◽  
José Cabeçadas ◽  
...  

Blastic plasmacytoid dendritic cell tumor is a rare, highly aggressive systemic neoplasm for which effective therapies have not yet been established. We describe a 73-year-old man with multiple nodules and patches emerging on the trunk and limbs. Lesional skin biopsy revealed a plasmacytoid dendritic cell tumor with dense dermal infiltrate of tumor cells with blastoid features. No apparent systemic involvement was identified in the initial stage. The patient was treated with prednisone daily, with notorious improvement of the skin lesions, although no complete remission was obtained. During the six-month follow-up period, no disease progression was documented, but fatal systemic progression occurred after that period of time.


Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 319-319 ◽  
Author(s):  
B. Lioure ◽  
J. Delaunay ◽  
D. Blaise ◽  
N. Milpied ◽  
P. Guardiola ◽  
...  

Abstract From 11/01 until 04/05, 832 patients (median age 46, 18–60) with AML (previous diagnosis of myelodysplasia or myeloproliferative disorder, and M3 excluded) were included in prospective phase III AML 2001 trial. After achieving CR, research to identify an HLA-identical sibling was performed for all patients as they received low dose consolidation (Daunorubicin (D): 60 mg/m2 × 2d OR Idarubicin (I): 12 mg/m2 × 2d plus SC ARAC 100 mg/m2 ×7d). 33 % patients had a donor then could proceed to a T-replete alloSCT: either conventional if age ≤ 50 (bone marrow graft; conditioning regimen: TBI (12 Gy 6 fractions over 3d) - cyclophosphamide (60 mg/kg × 2d); GvHD prophylaxis: ciclosporin-methotrexate d1+3+6) = arm M; or NST if age 51–60 (peripheral blood; Busulfan (oral Bu 4–8 mg/kg over 2d) - Fludarabin (30 mg/m2 × 4d) – Thymoglobulin® (2,5 mg/kg × 2d); ciclosporin alone), AFTER intensive consolidation (D: 60 mg/m2 × 2d OR I: 12 mg/m2 × 2d plus ARAC 3 g/m2 × 8 doses over 4d) = arm m. A small group of patients with a donor but low-risk prognostic features (favorable cytogenetics, no hyperleucocytosis, CR after 1 induction = 3% CR1 patients) didn’t receive 1st line alloSCT but intensive consolidation then a 2nd HD ARAC course; alloSCT was therefore considered at relapse = arm C. Patients without donor proceed to intensive consolidation then 1 or 2 autoSCT (1st after HDM 200 mg/m2 according to randomization, then Bu 16 mg/kg over 4d + HDM 140 mg/m2 for all patients) = arms A + B; they were combined for analysis as no difference was observed for DFS and OS. Actual results concern 532 patients with 15 months follow-up (A + B = 367; M = 111; m = 54). Median age was different between 3 groups (A + B = 46; M = 40; m = 54) as no difference was observed regarding leukocytosis or cytogenetic subgroups: favorable (t8;21) or inv16: A + B = 15%; M = 11%; m = 11%), intermediate (A + B = 72%; M = 78%; m = 67%), defavorable (5, 7, complex, 11q23 except t(9;11) or 3q; A + B = 13%; M = 11%; m = 22%). Conventional alloSCT results in better 2y DFS than autoSCT arms (M 71% vs A+B 52%, p=0,007) thought 2y OS advantage remains non significant (M 77% vs A+B 68%, p=0,06) as toxic death rate is higher (36% all deaths in arm M vs 14 % in arms A + B). No significant difference was observed between conventional alloSCT and NST (2y DFS 62%, OS 68%). Advantage for NST over autoSCT arms was non significant for DFS (p=0,24) and OS. Same results are obtained if considering only patients aged > 50: 2y EFS (m 62% vs A+B 50%, p=0,27) and OS (m 68% vs A+B 65%). After NST, toxicity accounts for 25% deaths, as relapse rate is 40% at 2y with no late relapse thereafter (vs 48 % at 2y and 61 % at 4y in arms A + B). In conclusion: 1) conventional alloSCT remains the best consolidation treatment for patients ≤ 50 with AML in CR1 despite higher toxicity; 2) NST after intensive consolidation seems promising: for older patients as toxicity is lower than conventional alloSCT, as few late relapse are observed in comparison with chemotherapy or autoSCT approaches; 3) NST may extend use of alternative sources of allogeneic hematopoietic stem cells to propose alloSCT approach for the majority of patients ≤ 60 with AML in CR1. Data with more than 2 years follow-up will be presented.


Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 4962-4962
Author(s):  
Pia Raanani ◽  
Anat Gafter-Gvili ◽  
Mical Paul ◽  
Isaac Ben-Bassat ◽  
Leonard Leibovici ◽  
...  

Abstract Background: Many studies have evaluated the role of polyvalent immunoglobulins (IVIG) and CMV-hyperimmune IVIG (CMV-IVIG) prophylaxis in patients undergoing hematopoietic stem cell transplantation (HSCT), with inconsistent results and implications for practice. Objectives: To evaluate the role of IVIG and CMV- IVIG prophylaxis in HSCT. Methods: Systematic review and meta-analysis of randomized controlled trials comparing IVIG or CMV- IVIG with placebo or no intervention (control) for prophylaxis in HSCT. The Cochrane Library, MEDLINE, conference proceedings and references were searched until 2007. Primary outcome: all-cause-mortality ; Secondary outcomes: CMV infections, acute graft versus host disease (GVHD), interstitial pneumonitis (IP), veno-occlusive disease (VOD) and adverse events. Relative risks (RR) with 95% confidence intervals (CIs) were estimated and pooled. Results: Nineteen trials met inclusion criteria. IVIG was compared to control in 10 trials (7 IVIG in allogeneic HSCT, 2 allogeneic/autologous HSCT and 1 autologous HSCT). When IVIG was compared to control, there was no difference in all-cause mortality (RR 1.03; 95% CI 0.89–1.18, 6 trials, Fig.). There was a reduction in the number of CMV infections (RR 0.70; 95% CI 0.50–0.99, 4 trials) and in the number of episodes of IP (RR 0.61; 95% CI 0.43–0.87, 6 trials). There was no significant difference in the number of episodes of acute GVHD (RR 0.93; 95% CI 0.83–1.05, 6 trials). The risk for VOD was increased (RR 2.71; 95% CI 1.06–6.94, 3 trials) as were adverse events in the IVIG group (RR 8.12; 95% CI 3.15–20.9, 5 trials). CMV-IVIG was compared to control in 7 trials, all allogeneic HSCT. There was no difference in all-cause mortality (RR 0.86; 95% CI 0.63–1.16, 4 trials). In addition, there were no differences in the risk for developing CMV infections (RR 1.07; 95% CI 0.86–1.33, 7 trials), acute GVHD (RR 1.02; 95% CI 0.72–1.44, 5 trials) or IP (0.95; 95% CI 0.58–1.56, 5 trials). Conclusions: Our review demonstrates that IVIG prophylaxis for HSCT reduces the risk for CMV infections and IP without a significant influence on mortality. The beneficial effects should be weighed against the higher incidence of adverse events, VOD, and cost associated with the use of prophylactic IVIG in HSCT patients. Conversely, the use of CMV-IVIG was not associated with a change in any of the parameters. Outcomes Summary Outcome IVIG vs. control CMV-IVIG vs. control All Cause Mortality RR 1.03; 95% CI 0.89–1.18 RR 0.86; 95% CI 0.63–1.16 CMV infections RR 0.70; 95% CI 0.50–0.99 RR 1.07; 95% CI 0.86–1.33 acute GVHD RR 0.93; 95% CI 0.83–1.05 RR 1.02; 95% CI 0.72–1.44 IP RR 0.61; 95% CI 0.43–0.87 RR 0.95; 95% CI 0.58–1.56 VOD RR 2.71; 95% CI 1.06–6.94 No data Adverse events RR 8.12; 95% CI 3.15–20.9 RR 7.0; 95% CI 0.38–129.34 Figure Figure


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