Patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) have a poor prognosis with conventional chemotherapy. In the present study, we retrospectively analyzed the outcome of patients with ...BPDCN who underwent allogeneic stem cell transplantation (allo-SCT) or autologous stem cell transplantation (auto-SCT). A total of 39 patients (allo-SCT, n = 34; auto-SCT, n = 5) were identified in the European Group for Blood and Marrow Transplantation registry. The 34 allo-SCT patients had a median age of 41 years (range, 10-70) and received transplantations from sibling (n = 11) or unrelated donors (n = 23) between 2003 and 2009. MAC was used in 74% of patients. Nineteen allo-SCT patients (56%) received transplantations in first complete remission. The 3-year cumulative incidence of relapse, disease-free survival, and overall survival was 32%, 33%, and 41%, respectively. By univariate comparison, being in first remission at allo-SCT favorably influenced survival, whereas age, donor source, and chronic GVHD had no significant impact. We conclude that high-dose therapy followed by allo-SCT from related or unrelated donors can provide durable remission even in elderly patients with BPDCN. However, it remains to be shown if graft-versus-malignancy effects can contribute significantly to BPDCN control after allo-SCT.
•BPDCN is a rare hematopoietic malignancy characterized by a poor prognosis and unusual resistance to conventional chemotherapy.•This study indicates that high-dose therapy followed by allo-SCT can provide durable disease control in this otherwise inevitably fatal condition.
The possibility of performing syngeneic hematopoietic stem cell transplantation is rare and there are concerns about the absence of a graft-versus-leukemia effect following such a strategy. We report ...the outcomes of a large series of adult patients who underwent syngeneic hematopoietic stem cell transplantation for acute myeloblastic leukemia or acute lymphoblastic leukemia.
The outcomes of all syngeneic transplants for acute myeloblastic or lymphoblastic leukemia reported to the European Group for Blood and Marrow Transplantation registry were analyzed; a study of prognostic factors was performed for those transplanted in first complete remission.
One hundred and sixty-two patients, 109 with acute myeloblastic leukemia and 53 with acute lymphoblastic leukemia, were identified; 116 were in first complete remission. Most of the patients did not receive prophylaxis against graft-versus-host disease. Nineteen patients developed acute graft-versus-host disease and only three patients developed chronic graft-versus-host disease. The median follow-up was 60 months. At 5 years the non-relapse mortality was 8 +/- 5%, the relapse incidence 49 +/- 8% and the leukemia-free survival 43 +/- 3%. The corresponding figures for patients in first complete remission were 7 +/- 2%, 40 +/- 4% and 53 +/- 5% at 5 years. Analysis of patients in first complete remission showed that the number of courses of chemotherapy required to induce first complete remission was the main risk factor: the leukemia-free survival at 5 years was 66 +/- 6% when first complete remission was reached after one induction course of chemotherapy and was only 20 +/- 9% when first complete remission was reached after at least two induction courses of chemotherapy (p = 0.0001); the relapse incidence was 30 +/- 6% and 54 +/- 10%, respectively (p = 0.007).
Outcomes were better for patients transplanted in first complete remission than in second complete remission or a more advanced phase of the disease. When a syngeneic donor is available for patients with high risk acute leukemia, allotransplantation should be performed as soon as the first complete remission has been achieved, ideally with one course of chemotherapy.
Abstract 3077
Blastic plasmacytoid dendritic cell neoplasm (BPDC), formerly known as blastic NK cell lymphoma, is a rare hematopoietic malignancy preferentially involving the skin, bone marrow and ...lymph nodes. The overall prognosis of BPDC is dismal. Most patients show an initial response to acute leukemia-like chemotherapy, but relapses with subsequent drug resistance occur in virtually all patients resulting in a median overall survival of only 9–13 months. However, anecdotal long-term remissions have been reported in young patients who received early myeloablative allogeneic stem cell transplantation (alloSCT). We therefore performed a retrospective analysis of patients identified in the EBMT registry in order to evaluate the outcome of autologous stem cell transplantation (autoSCT) or alloSCT for BPDC. Eligible were all patients who had been registered with a diagnosis of BPDC or Blastic NK cell lymphoma and had received autologous stem cell transplantation (autoSCT) or alloSCT in 2000–2009. Centres were contacted to provide a written histopathology and immunophenotyping report and information about treatment and follow-up details. Patients who did not have a diagnostic score ≥ 2 as proposed by Garnache-Ottou et al. (BJH 2009) were excluded.
Overall, 139 patients could be identified in the database who fulfilled the inclusion criteria (alloSCT 100, autoSCT 39). Of 74 patients for whom the requested additional information could be obtained, central review confirmed the diagnosis of BPDC in 39 patients (34 alloSCT, 5 autoSCT). The 34 patients who had undergone alloSCT had a median age of 41 years (range: 10–70 years), were transplanted from a related (n=11) or unrelated donor (n=23); received peripheral blood stem cells (n=9), bone marrow stem cells (n=19) or cord blood (n=6); and had been treated with a reduced intensity conditioning regimen (RIC, n=9) or myeloablative conditioning (MAC, n=25). Nineteen of 34 patients were transplanted in CR1. After a median follow up time of 28 months (range: 4–77+ months), 11 patients relapsed (median time to relapse: 8 months, range: 2–27 months) of whom 8 died due to disease progression. 9 patients died in the absence of relapse. No relapse occurred later than 27 months after transplant. Median disease free survival (DFS) was 15 months (range: 4–77+ months) and median overall survival (OS) was 22 months (range: 8–77+ months; Figure 1a). However, long-term remissions of up to 77 months after alloSCT could be observed. Patients allografted in CR1 tended to have a superior DFS (p=0.119) and OS (p=0.057; Figure 1b). MAC was associated with a better OS (p=0.001) which was attributable to the significantly higher non-relapse mortality (NRM) rate of patients after RIC (p=0.014), who had been significantly older (age RIC: 56 years, age MAC: 36 years, p=0.0014). The relapse rate was not different in patients after RIC and MAC, respectively. However, there was no survivor after RIC. Median age in the autoSCT group was 47 years (range: 14–62 years). Three of 5 patients were transplanted in CR1 of whom 1 patient relapsed after 8 months, 1 patient experienced treatment related mortality and 1 patient remained in CR for 28 months. The 2 remaining patients had more advanced disease at autoSCT and relapsed 4 and 8 months thereafter. Display omitted
AlloSCT is effective in BPDC and might provide curative potential in this otherwise incurable disease, especially when performed in CR1. However, it remains to be shown by prospective studies if the potential benefit of alloSCT in BPDC is largely due to conditioning intensity, or if there is a relevant contribution of graft-versus-leukemia activity.
Tilly:Seattle Genetics, Inc.: Membership on an entity's Board of Directors or advisory committees, Research Funding; Celgene: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau, Travel/accommodations/meeting expenses; Genentech: Membership on an entity's Board of Directors or advisory committees; Roche: Membership on an entity's Board of Directors or advisory committees, Research Funding; Amgen: Research Funding, Speakers Bureau; Pfizer: Speakers Bureau; Janssen Cilag: Speakers Bureau.
Both autologous stem cell transplantation (autoSCT) and reduced intensity allogeneic stem cell transplantation (RIST) may be considered as treatment options in patients with relapsed follicular ...lymphoma (FL). It is currently unknown which transplant strategy is optimal in patients with FL. We have therefore conducted a retrospective comparison of patients who have undergone an autoSCT or a RIST. Adult patients undergoing a 1st transplant procedure and reported to the EBMT between Jan 1998 and Dec 2005 were included. 1394 patients underwent an autoSCT and 110 a RIST with median ages of 51 (20–73) and 50 (32–65) respectively. Patients undergoing a RIST had significantly more advanced disease (stage IV 74% vs 60% p=0.01) and B symptoms (39% vs 27% p=0.05) at diagnosis but there was no difference in the size of the largest mass or the LDH. The median time from diagnosis to transplant was 27 months for the whole group but was significantly longer for patients undergoing a RIST (34 months vs 26 months p=0.005). Patients undergoing a RIST had received more lines of prior therapy (61% 3 or more lines vs 34% for those undergoing autoSCT p<0.001) but there was no difference in the percentage of patients receiving rituximab prior to transplantation. 49% of the autoSCT were performed prior to 2002 compared to 26% of the RIST (p<0.001). At transplant the disease status for RIST and autoSCT patients was: CR 34% vs 43% ; PR 52% vs 56%; refractory disease 10% vs 5% respectively (p=0.02). Patients undergoing a RIST received stem cells from matched sibling donors in 94 cases and matched unrelated donors (MUD) in 16. All patients undergoing an autoSCT received unpurged bone marrow stem cells. With a median follow-up post transplant of 26 months (range 6–112) 84 patients are alive in the RIST group and 1154 patients in the autoSCT group. In multivariate analysis non-relapse mortality (NRM) was significantly worse for patients undergoing a RIST (relative risk (RR) 3.5, p<0.001) and for those older than 45 (RR 2.3, p=0.01) or with refractory disease at transplant (RR 3.3, p<0.001). The cumulative incidence of NRM at 100 days and 1 year was 5% and 15% respectively for those undergoing a RIST compared to 2% and 3% for those undergoing an autoSCT. Disease relapse was significantly worse for those receiving an autoSCT (RR 2.8, p<0.001), more lines of prior therapy (RR 1.3, p=0.02), stage IV disease at diagnosis (RR 1.4, p=0.004) and for those with refractory disease (RR 1.6, p<0.001) or poor performance status at transplant (RR=3.0, p=0.005). Patients undergoing a RIST had a 5 year relapse rate of 19% compared to 47% for those undergoing an autoSCT. For patients with follow-up beyond 30 months (RIST 26 patients, autoSCT 449 patients) there were no relapses beyond this time point in the RIST group and 87 relapses in the autoSCT group. Progression free survival was significantly worse for those with chemorefractory disease (RR 3.1, p<0.001), more lines of prior therapy (RR 1.3, p=0.004) or with a poor PS (RR 2.5, p=0.03). The PFS at 3 years and 5 years was 62% for patients receiving a RIST compared to 58% and 48% respectively for those receiving an autoSCT. The comparison of PFS between autoSCT and RIST was non-proportional over time with a significant advantage to RIST after 2 years (RR 9.0, p=0.002). In conclusion RIST is associated with a higher NRM but a lower relapse rate when compared to autoSCT resulting in an improved PFS following a RIST.
Aplastic anemia (AA) is rarely described after a diagnosis of autoimmune disease (aID).
To assess the prevalence of prior aID in patients with AA recorded in the registry of the European Group for ...Blood and Marrow Transplantation (EBMT) and to evaluate treatment and outcome.
1,251 AA patients from 18 EBMT centers were assessed.
Fifty patients (4%) were eligible: 22 males and 28 females with a median age of 46 years at the diagnosis of aID and of 51 years at the diagnosis of AA. Information on the treatment of AA was available in 49 patients: 38 received only immunosuppressive therapy (IST), 8 patients underwent hematopoietic stem cell transplantation (HSCT) - 6 as first-line therapy and 2 after failure of IST - whilst 3 patients had a spontaneous recovery. After a median follow-up of 3.19 years, 32 patients were alive, including 7 of the 8 patients who underwent HSCT. Only 6 of 32 patients who were alive at the last follow-up were receiving IST for AA.
Most cases of AA following aID benefitted from IST or HSCT if a matched donor was available. Further prospective investigation is needed to assess the effects of IST on the outcome of underlying aID.
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