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Papers by Antonin Vitek

Research paper thumbnail of Use of Post-Transplant Cyclophosphamide in One-Antigen Mismatched Unrelated Donor Transplantation Results in Similar Transplant Outcomes Than Haploidentical Hransplantation: A Retrospective Study on Behalf of the Acute Leukemia Working Party of the EBMT

Blood

Introduction. In the absence of an HLA-identical sibling or a matched unrelated donor, whether to... more Introduction. In the absence of an HLA-identical sibling or a matched unrelated donor, whether to prefer a Haploidentical (Haplo) or a one antigen mismatched unrelated donor (MMUD) for allogeneic hematopoietic cell transplantation (HCT) remains an unanswered question. Implementation of graft-versus-host disease (GVHD) prophylaxis with post-transplant cyclophosphamide (PTCY) was initially pioneered in the Haplo and then also extended to MMUD setting, resulting in low rates of GVHD. Methods. This was a retrospective study from the EBMT registry. Included were adults undergoing either Haplo- or MMUD-HCT for acute myeloid leukemia during the period 2010-2018 and who were in first or second complete remission at allo-HCT. Only patients receiving unmanipulated grafts with PTCY as GVHD prophylaxis were included. Ex vivo and in vivo T-cell depletion were exclusion criteria. Comparisons were made among three groups: MMUD-HCT with peripheral blood as stem cell source (PBSC; n=124); Haplo-HCT ...

Research paper thumbnail of The timing of post-transplant cyclophosphamide administration in haploidentical transplantation: a comparative study on behalf of the ALWP of the EBMT

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2020

Timing of immunosuppressive-drugs therapy used in combination with post-transplant cyclophosphami... more Timing of immunosuppressive-drugs therapy used in combination with post-transplant cyclophosphamide (PTCY) in haploidentical hematopoietic stem cell transplant (Haplo-HSCT) is not standardized. We evaluated the schedule of immunosuppression therapy after haplo-HSCT in 509 patients with acute leukemia receiving PTCY on day +3 and +4 along with tacrolimus (group 1, n=215) or cyclosporine A (CSA) and mycophenolate -mofetil (MMF) from day+5 (group 2, n=170) or CSA+MMF from day 0 or 1 with PTCY on day +3 and +5 (group 3, n=124). Patients in group 3 were younger (median age 46 years, p=0.02), received bone marrow (77%, p<0.01) and a regimen containing thiotepa, fludarabine and busulfan (84%, p<0.01) more frequently. At 2 years, OS was 44%, 48% and 59% (p=0.15), LFS was 43%, 46%, and 53%, (p=0.05) and refined-GRFS (rGRFS) was 33%, 39% and 36% (p=0.02), in groups 1, 2 and 3, respectively. Grade II-IV acute GVHD was 25%, 39% and 18%, respectively (p<0.01), and chronic GVHD was 25%, ...

Research paper thumbnail of Haploidentical stem cell transplantation abrogates the prognostic impact of FLT3-ITD in acute myeloid leukemia. A report from the Acute Leukemia Working Party of the EBMT

Acute myeloid leukemia (AML) patients harboring the Fms-like tyrosine kinase 3 internal tandem du... more Acute myeloid leukemia (AML) patients harboring the Fms-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD) mutation are considered a particularly high risk patient subset preferentially allocated for allogeneic stem cell transplantation in first remission. Whether FLT3-ITD retains a prognostic role in haploidentical stem cell transplantation (haplo-SCT) is currently unknown. Patients and methods Using the international multicenter registry of the acute leukemia working party of the European society for blood and marrow transplantation, we performed a retrospective analysis to determine whether FLT3-ITD was prognostically significant in T-cell replete haplo-SCT transplanted AML patients. Results We evaluated 293 de-novo AML patients (202 FLT3 wt and 91 FLT3-ITD mut) transplanted in first remission with T-cell replete haplo-SCT between 2005-2016. FLT3-ITD mut patients were more likely to be NPM1 mutated as well as be in the intermediate risk cytogenetic risk category. In multivariate analysis, patients with FLT3-ITD had comparable rates of relapse incidence [Hazard ratio (HR)=1.34, confidence interval (CI) 95%, 0.67-2.7; P=0.9] and leukemia-free survival (HR=0.99, CI 95%, 0.62-1.57; P=0.9) to those of FLT3 wt patients. Survival was not significantly impacted by FLT3-ITD status (HR=0.96, CI 95%, 0.58-1.59; P=0.8), nor were the incidence of non-relapse mortality (HR=0.78, CI 95%, 0.41-1.46; P=0.44), and graft versus host diseasefree/relapse-free survival (HR=0.84, CI 95%, 0.54-1.28; P=0.42). Focused subset analysis of patients with intermediate risk cytogenetics confirmed the absence of a prognostic impact of FLT3-ITD also for this group of patients. Conclusion In AML patients undergoing T-cell replete haplo-SCT, the FLT3-ITD mutation does not retain its prognostic significance, potentially indicative of haplo-SCT's capacity to overcome the negative prognostic consequences of FLT3-ITD in AML.

Research paper thumbnail of Measurable residual disease status and outcome of transplant in acute myeloid leukemia in second complete remission: a study by the acute leukemia working party of the EBMT

Blood Cancer Journal

Measurable residual disease (MRD) prior to hematopoietic cell transplant (HCT) for acute myeloid ... more Measurable residual disease (MRD) prior to hematopoietic cell transplant (HCT) for acute myeloid leukemia (AML) in first complete morphological remission (CR1) is an independent predictor of outcome, but few studies address CR2. This analysis by the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation registry assessed HCT outcomes by declared MRD status in a cohort of 1042 adult patients with AML CR2 at HCT. Patients were transplanted 2006–2016 from human leukocyte antigen (HLA) matched siblings (n = 719) or HLA 10/10 matched unrelated donors (n = 293). Conditioning was myeloablative (n = 610) or reduced-intensity (n = 432) and 566 patients (54%) had in-vivo T cell depletion. At HCT, 749 patients (72%) were MRD negative (MRD NEG) and 293 (28%) were MRD positive (MRD POS). Time from diagnosis to HCT was longer in MRD NEG than MRD POS patients (18 vs. 16 months (P

Research paper thumbnail of Haploidentical Stem Cell Transplantation Attenuates the Prognostic Impact of FLT3-ITD in Acute Myeloid Leukemia

Background Acute myeloid leukemia (AML) patients harboring the fms-like tyrosine kinase 3 interna... more Background Acute myeloid leukemia (AML) patients harboring the fms-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD) mutation are considered a particularly high risk patient subset preferentially allocated for allogeneic stem cell transplantation in first remission. Whether FLT3-ITD retains a prognostic role in haploidentical stem cell transplantation (haplo-SCT) is currently unknown. Patients and methods To determine whether FLT3-ITD is prognostically significant in T-cell replete haplo-SCT transplanted AML patients, we performed a comparative analysis between FLT3wt and FLT3-ITD using the multicenter registry of the acute leukemia working party of the European society for blood and marrow transplantation. Patients included in this analysis were AML patients over the age of 18 who underwent a first T-cell replete haplo-SCT in first remission. Results We evaluated 293 de-novo AML patients (202 FLT3wt and 91 FLT3-ITD) transplanted in first remission with T-cell replete ha...

Research paper thumbnail of Allogeneic hematopoietic stem cell transplantation using HLA-compatible donors in younger high cytogenetic risk CLL patients results in very high long-term progression-free survival and may therefore favorably compete with sequential targeted therapy

Research paper thumbnail of Allogeneic hematopoietic stem cell transplantation in patients over 50: The single centre experience

Analyses of hematopoietic stem cell transplantation (SCT) results are of high importance for deci... more Analyses of hematopoietic stem cell transplantation (SCT) results are of high importance for decision-making on treatment strategy for patients with SCT as a possible therapeutic alternative. In this paper the Czech National Registry of SCT and Transplantation Centre in Pilsen present their joint retrospective analysis of the results of allogeneic SCT in patients with chronic myeloid leukemia (CML) performed in the Czech Republic from 1988 to spring 2005. 295 patients (179 men and 116 women) ranging in age from 6.9 to 59.5 years (median 37.3) underwent transplants. In most cases the donor was an HLA-identical sibling (164; 55.6%) or a voluntary unrelated donor from the register (110; 37.3%), in a minority of cases another relative of the patient (21; 7.1%). Myeloablative conditioning was used in 90% of patients. The source of hematopoietic stem cells was bone marrow in 57%, peripheral blood in 41% and combination of both in 2% of cases. 83.4% of patients underwent transplant in the chronic phase of the illness while 7.8% in the acceleration phase and 6.1% in the blastic phase respectively. The median interval from the diagnosis to SCT was 316 days. Median follow-up after SCT was 2 years. SCT was complicated by acute graft versus host disease of grade II-IV in 33.7% of patients and by chronic graft versus host disease in 36.3% of patients. Median survival was not reached, 18 (6.1%) of patients died due to the relapse of CML and the cause of 101 (34.2%) deaths was transplant-related. Significant trends were observed during the study period: SCT were performed more frequently in older patients, less than one year from the diagnosis, reduced-intensity conditioning was used more often and the source of hematopoietic stem cells was peripheral blood in the majority of patients (p = 0.188 - < 0.0001). Also, transplantation activity changed - the annual rate of SCT increased steadily until 1999, while there was no such an increase between 2000 and 2005. The use of peripheral stem cells was associated with chronic graft versus host disease (p = 0.007). In Cox multivariate analysis the EBMT risk score and the interval from the diagnosis to SCT were identified as independent factors in patient survival. An "ideal" patient, aged under 30, undergoing transplant in the chronic phase of CML within one year since the diagnosis after 2000 had a survival probability of 88% for three years after SCT. It can be concluded that results of allogeneic SCT in CML in the Czech Republic reflect current global trends, are comparable with results achieved in other countries and show significant improvements.

Research paper thumbnail of Timing of Post-Transplantation Cyclophosphamide Administration in Haploidentical Transplantation: A Comparative Study on Behalf of the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation

Biology of Blood and Marrow Transplantation

Research paper thumbnail of Determinants of survival in myelofibrosis patients undergoing allogeneic hematopoietic cell transplantation

Leukemia

We aimed to evaluate the determinants of survival in myelofibrosis patients undergoing allogeneic... more We aimed to evaluate the determinants of survival in myelofibrosis patients undergoing allogeneic hematopoietic cell transplantation (allo-HCT) and to describe factors predicting the main post-HCT complications. This retrospective study by the European Society for Blood and Marrow Transplantation included 2916 myelofibrosis patients who underwent first allo-HCT from an HLA-identical sibling or unrelated donor between 2000 and 2016. After a median follow-up of 4.7 years from transplant, projected median survival of the series was 5.3 years. Factors independently associated with increased mortality were age ≥ 60 years and Karnofsky Performance Status <90% at transplant, and occurrence of graft failure, grades III–IV acute graft-vs.-host disease (aGVHD), and disease progression/relapse during follow-up. The opposing effects of chronic graft-vs.-host disease (GVHD) on non-relapse mortality and relapse incidence resulted in a neutral influence on survival. Graft failure increased in unrelated donor recipients and decreased with myeloablative conditioning (MAC) and negative donor/recipient cytomegalovirus serostatus. Risk of grades III–IV aGVHD was higher with unrelated donors and decreased with MAC. Relapse incidence tended to be higher in patients with intermediate-2/high-risk DIPSS categories and to decrease in CALR -mutated patients. Acute and chronic GVHD reduced the subsequent risk of relapse. This information has potential implications for patient counseling and clinical decision-making.

Research paper thumbnail of Trends in Allogeneic Stem Cell Transplantation for Myelofibrosis in Europe between 1995-2018: An EBMT Retrospective Analysis

Blood

Aim: Dynamic assessment of trends over time in patient- and transplant-specific characteristics a... more Aim: Dynamic assessment of trends over time in patient- and transplant-specific characteristics and outcomes for patients undergoing 1st allogeneic haematopoietic cell transplant (allo-HCT) for Myelofibrosis (MF). Methods and Results: A total of 4142 MF patients were analysed who underwent allo-HCT between 1995-2018 (24-year period) across 278 centres based on data reported to the European Society for Blood and Marrow Transplantation. For analysis, 4 cohorts were considered based on year of allo-HCT: <2006 n=389 (9.4%), 2006-2010 n=910 (22%), 2011-2014 n=1148 (27.7%) and 2015-2018 n=1695 (40.9%). A steady increase in MF allo-HCT activity over time was apparent paralleled with increasing numbers of participating transplant centres. A total of 2603 (62.8%) patients were male, 3239 (78.2%) had Primary MF, 409 (9.9%) and 494 (11.9%) post-Polycythaemia Vera and post-Essential Thrombocythaemia MF, respectively. An increased median interval (lnterquartile range (IQR)) between diagnosis ...

Research paper thumbnail of Post-transplantation cyclophosphamide GvHD prophylaxis after hematopoietic stem cell transplantation from 9/10 or 10/10 HLA-matched unrelated donors for acute leukemia

Leukemia

HLA-matching largely contributes to unrelated donor hematopoietic cell transplantation (UD-HCT) s... more HLA-matching largely contributes to unrelated donor hematopoietic cell transplantation (UD-HCT) success but, due to the selective deletion of alloreactive T-cells, post-transplantation cyclophosphamide (PTCy) could modulate its negative impact on outcomes. We retrospectively compared acute leukemia patients receiving 10/10 or 9/10 HLA allele-matched UD-HCT with PTCy-GvHD prophylaxis between 2010 and 2017, reported to EBMT registry. The 100-day incidence of grade ≥2 and grade ≥3 aGvHD were comparable for 10/10 and 9/10 UD (28% versus 28%, p = 0.8 and 10% versus 8%, p = 0.5, respectively). The 2-year cGvHD and extensive cGvHD were similar between 10/10 and 9/10 UD (35% versus 44%, p = 0.2 and 21% versus 20%, p = 0.6, respectively). The 2-year nonrelapse mortality was 20% after 10/10 and 16% after 9/10 UD-HCT ( p = 0.1). Relapse incidence at 2-year was 24% for 10/10 and 28% for 9/10 UD-HCT ( p = 0.4). Leukemia-free survival at 2-year was the same for 10/10 and 9/10 UD (56 and 56%, p = 0.6, respectively), with comparable overall survival (62 and 59%, p = 0.9, respectively). Multivariate analysis showed no effect of HLA-matching on outcomes. An advanced disease status and patient disability remained the most important factors portending a worse survival. PTCy could alleviate the detrimental effect of HLA-allele mismatching in UD-HCT, potentially expanding the donor pool for acute leukemia patients.

Research paper thumbnail of Haploidentical transplant with post-transplant cyclophosphamide for T-cell acute lymphoblastic leukemia: a report from the EBMT acute leukemia working party

Biology of Blood and Marrow Transplantation

Allogeneic hematopoietic cell transplantation (HCT) is recommended in high-risk patients with T c... more Allogeneic hematopoietic cell transplantation (HCT) is recommended in high-risk patients with T cell acute lymphoblastic leukemia (T-ALL). For patients with no HLA identical donor, haploidentical transplantation (haplo-HCT) is becoming the leading source of stem cell donation. However, data is scarce on predictive factors for outcome in that setting. We identified 122 adults (20% female; median age 31 years; range 18-68) with T-ALL who received a haplo-HCT with post-transplant cyclophosphamide (ptCy) between 2010 and 2017. Median follow-up of living patients was 23 months. The 2-year relapse incidence and non-relapse mortality were 45% and 21%, respectively. The 2-year leukemia-free survival (LFS), overall survival (OS) and GVHD-free, relapse-free survival (GRFS) were 34%, 42% and 27%, respectively. The 2-year LFS and OS were highly influenced by disease status at transplant, being 49% and 55% respectively for first complete remission (CR1), 34% and 50% respectively for CR2, 8% and 12% respectively for patients with active disease. On multivariate analysis, only disease status affected LFS and OS. Transplantation in CR2 negatively affected LFS, whereas active disease at haplo-HCT negatively affected LFS and OS. In conclusion, haplo-HCT with ptCy produced encouraging results in this challenging disease, particularly when performed in CR. Despite the limitation of the small sample size, results were not affected by the type of conditioning, questioning the need for total body irradiation based-myeloablative conditioning (TBI-MAC) in that setting.

Research paper thumbnail of Long-Term Follow-up Data Support the Curative Potential of Allogeneic Hematopoietic Cell Transplantation in Patients with Chronic Lymphocytic Leukemia: A Retrospective Analysis from the Chronic Malignancies Working Party of the EBMT

Blood

Objectives: In patients with refractory chronic lymphocytic leukemia (CLL), the potential to cure... more Objectives: In patients with refractory chronic lymphocytic leukemia (CLL), the potential to cure is unique to allogeneic stem cell transplantation (HSCT). Clearance of minimal residual disease by suspected graft-versus-leukemia effects has been described elegantly and documented in several independent patient cohorts. Yet data from large numbers of patients which support the concept of cure by allogeneic transplantation have not been published. With the advent of new targeted therapies for patients with advanced chemo-refractory CLL, this information becomes crucial for clinical decision making and patient counselling. Therefore, we aimed at the description of long-term survival outcomes, and the estimation of excess mortality compared to the age- and sex-matched general population. Patients and Methods: Data from patients with CLL who had received a first allogeneic HSCT from an HLA-identical sibling (SIB) or alternative donor between January 2000 and December 2010, and who were r...

Research paper thumbnail of Post-Transplant Cyclophosphamide Versus Antithymocyte Globulin in Patients with Acute Myeloid Leukemia Undergoing Allogeneic Stem Cell Transplantation from One Antigen HLA-Mismatched Donors: A Retrospective Analysis from the Acute Leukemia Working Party of the EBMT

Blood

Introduction. Several alternative donor sources are currently available for patients who lack an ... more Introduction. Several alternative donor sources are currently available for patients who lack an HLA-matched related or unrelated donor, including haploidentical, cord blood and one antigen HLA-mismatched donors (9/10 mMUD). Use of post-transplant (allo-HSCT) cyclophosphamide (PTCY) as graft-versus-host disease (GVHD) prophylaxis allowed outcome improvements in the haploidentical setting. Its use has also been reported as a safe and feasible option in 9/10 mMUD transplants. However, to date, the main strategy used as GVHD prophylaxis in 9/10 mMUD allo-HSCT is in vivo T-cell depletion with antithymocite globulin (ATG). Data comparing these two different anti GVHD prophylaxis strategies in 9/10 mMUD allo-HSCT are limited. Methods. We compared PTCY versus ATG as GVHD prophylaxis in patients undergoing 9/10 mMUD allo-HSCT for which high-resolution HLA-allele typing was available in the ALWP/EBMT data registry. Included were adult patients (age>18 years) undergoing their first allo-HS...

Research paper thumbnail of Prognostic Pre-Transplant Factors in Myelodysplastic Syndromes Primarily Treated by Allogeneic Hematopoietic Stem Cell Transplantation: A Retrospective Study on Behalf of the MDS Subcommittee of the Chronic Leukaemia Working Party of the EBMT

Blood

3014FN2 Background: Allogeneic hematopoietic stem cell transplantation (SCT) is associated with i... more 3014FN2 Background: Allogeneic hematopoietic stem cell transplantation (SCT) is associated with iron overload and iron toxicity, especially in patients with myelodysplastic syndromes (MDS). Causes of this increased iron load are transfusions, ineffective erythropoiesis and, myelosuppressive therapy. Investigators found that iron toxicity (often using serum ferritin as a surrogate marker of iron burden) was associated with an adverse effect on non-relapse mortality (NRM) and overall survival (OS). As pre-transplant anti-leukemic treatment may lead to organ damage which may influence outcome after SCT, our study aimed to minimize the role of previous treatment toxicity by only including MDS patients who had not received intensive anti-leukemic treatment prior to SCT conditioning, allowing more insight in the role of iron toxicity in allogeneic SCT. A retrospective analysis of data from the EBMT registry was carried out after an additional survey within selected EBMT centers. Objective...

Research paper thumbnail of Significance of Minimal Residual Disease Monitored by Quantitative Assessment of WT1gene in Patients in First Remission of Acute Myeloid Leukemia Before Allogeneic Stem Cell Transplantation

Blood

4485 Purpose Thanks to the development of knowledge in the field of molecular biology, the great ... more 4485 Purpose Thanks to the development of knowledge in the field of molecular biology, the great progress has been done in risk stratification of patients with acute myeloid leukemia (AML) at diagnosis, in recent years. Based on the recommendations of international expert groups there were identified the patients who may benefit from the allogeneic stem cell transplantation (allo-SCT) as a consolidation of first complete remission (CR). In the absence of an universal marker for minimal residual disease (MRD) measurements, there is still little information about the importance of MRD prior to allo-SCT. Our department has a very good experience with quantitative monitoring of WT1 gene expression as a marker of MRD during treatment of AML. The aim was to retrospectively evaluate the significance of MRD in patients indicated for allo-SCT in 1.CR. Patients and methods Overall 35 patients (pts) in the first morphological CR were transplanted from April 2005 - July 2011. Median age was 46 ...

Research paper thumbnail of Combination Chemotherapy Leading In Advanced MDS Patients to a Rapid Clearence of Bone Marrow Blasts Prior Stem Cell transplantation (SCT) Is Superior to up-Front SCT Even with Intensified Conditioning for Long-Term Survival

Blood

4020 A retrospective analysis of influence of different clinical and laboratory parameters on dis... more 4020 A retrospective analysis of influence of different clinical and laboratory parameters on disease outcome was performed in a cohort of 43 patients with advanced myelodysplasia (MDS)(RAEB…

Research paper thumbnail of Center Characteristics and Procedure-Related Factors Have an Impact on Outcomes of Allogeneic Transplantation for Patients with CLL: A Retrospective Analysis from the European Society for Blood and Marrow Transplantation (EBMT)

Blood

Introduction:Even in the era of novel targeted therapies for the treatment of Chronic Lymphocytic... more Introduction:Even in the era of novel targeted therapies for the treatment of Chronic Lymphocytic Leukemia (CLL) patients, such as BTK, PI3K and BCL2 inhibitors, allogeneic hematopoietic stem cell transplantations (alloHCT) will remain an important treatment option for a subset of patients with very high risk CLL. The current study focused on the impact of center and procedure-related factors on outcomes after alloHCT, taking into account the impact of patient- and disease-related risk factors. Patients and Methods:Data of 684 CLL patients who received a first alloHCT between 2000 and 2011 were analyzed. Their data were collected as part of the EBMT CLL Data Quality Initiative. Outcomes of interest were Event-Free Survival (EFS) up to 5 years after transplantation and mortality in the first 100 days after alloHCT. Outcomes were analyzed by means of the Kaplan-Meier method and Cox proportional hazards models with a frailty (random effects) component to take into account unexplained c...

Research paper thumbnail of Comparison of Matched-Sibling Donors Versus Unrelated Donors in Allogeneic Stem Cell Transplantation (allo-SCT) for Primary Refractory Acute Myeloid Leukemia (PRF AML): A Report of 1041 Patients from the Acute Leukemia Working Party of the EBMT

Blood

Background: PRF-AML is associated with a dismal prognosis. Approximately one third of patients yo... more Background: PRF-AML is associated with a dismal prognosis. Approximately one third of patients younger than 60 years, and 50 % of older patients, with newly diagnosed AML, fail to achieve complete remission (CR) with standard induction chemotherapy. Allo-SCT in the setting of active disease is an alternative strategy. The increased availability of unrelated donors (UD) together with the use of reduced-intensity conditioning (RIC) regimens have opened the possibility for transplantation to a larger number of patients in comparison to standard myeloablative regimens (MAC). Because of the high risk of allo-SCT in this setting, there are still questions on the patient outcome depending on the donor type. Aims: The current study aimed to compare the outcomes of allo-SCT from matched sibling donors (MSD) (n=660) vs UDs (n=381), for patients with PRF AML. Methods: The major endpoints were to assess overall survival (OS), leukemia-free survival (LFS), relapse incidence (RI), and non relapse...

Research paper thumbnail of Outcome of Patients with Myelofibrosis Relapsing after Allogeneic Stem Cell Transplant: A Retrospective Study By the Chronic Malignancies Working Party of EBMT

Blood

Background: Over the last decade, there has been a significant increase in the number of patients... more Background: Over the last decade, there has been a significant increase in the number of patients with Myelofibrosis (MF) undergoing allogeneic stem cell transplantation (SCT). However, scarce information exists on the outcome and management of those patients who relapse following SCT. Moreover, the management of relapse occurring post-SCT is often heterogeneous and ranges from palliation to intensive salvage approaches. We therefore conducted a retrospective EBMT registry analysis of adult MF patients who relapsed following first SCT episode. Results: A total of 1216 adult patients (997 (82%) with Primary MF (PMF) and 219 (18%) with secondary MF (sMF)) underwent 1st allogeneic SCT between 2000 and 2010. A total of 251 patients from this cohort (206 with PMF and 45 with sMF) had conformed relapse ≥ day 30 after HSCT and were included in the analysis. Within this relapse cohort, there were 163 males and 88 females; median age was 55 years old (range 21.5-70 years). A total of 84 pati...

Research paper thumbnail of Use of Post-Transplant Cyclophosphamide in One-Antigen Mismatched Unrelated Donor Transplantation Results in Similar Transplant Outcomes Than Haploidentical Hransplantation: A Retrospective Study on Behalf of the Acute Leukemia Working Party of the EBMT

Blood

Introduction. In the absence of an HLA-identical sibling or a matched unrelated donor, whether to... more Introduction. In the absence of an HLA-identical sibling or a matched unrelated donor, whether to prefer a Haploidentical (Haplo) or a one antigen mismatched unrelated donor (MMUD) for allogeneic hematopoietic cell transplantation (HCT) remains an unanswered question. Implementation of graft-versus-host disease (GVHD) prophylaxis with post-transplant cyclophosphamide (PTCY) was initially pioneered in the Haplo and then also extended to MMUD setting, resulting in low rates of GVHD. Methods. This was a retrospective study from the EBMT registry. Included were adults undergoing either Haplo- or MMUD-HCT for acute myeloid leukemia during the period 2010-2018 and who were in first or second complete remission at allo-HCT. Only patients receiving unmanipulated grafts with PTCY as GVHD prophylaxis were included. Ex vivo and in vivo T-cell depletion were exclusion criteria. Comparisons were made among three groups: MMUD-HCT with peripheral blood as stem cell source (PBSC; n=124); Haplo-HCT ...

Research paper thumbnail of The timing of post-transplant cyclophosphamide administration in haploidentical transplantation: a comparative study on behalf of the ALWP of the EBMT

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2020

Timing of immunosuppressive-drugs therapy used in combination with post-transplant cyclophosphami... more Timing of immunosuppressive-drugs therapy used in combination with post-transplant cyclophosphamide (PTCY) in haploidentical hematopoietic stem cell transplant (Haplo-HSCT) is not standardized. We evaluated the schedule of immunosuppression therapy after haplo-HSCT in 509 patients with acute leukemia receiving PTCY on day +3 and +4 along with tacrolimus (group 1, n=215) or cyclosporine A (CSA) and mycophenolate -mofetil (MMF) from day+5 (group 2, n=170) or CSA+MMF from day 0 or 1 with PTCY on day +3 and +5 (group 3, n=124). Patients in group 3 were younger (median age 46 years, p=0.02), received bone marrow (77%, p<0.01) and a regimen containing thiotepa, fludarabine and busulfan (84%, p<0.01) more frequently. At 2 years, OS was 44%, 48% and 59% (p=0.15), LFS was 43%, 46%, and 53%, (p=0.05) and refined-GRFS (rGRFS) was 33%, 39% and 36% (p=0.02), in groups 1, 2 and 3, respectively. Grade II-IV acute GVHD was 25%, 39% and 18%, respectively (p<0.01), and chronic GVHD was 25%, ...

Research paper thumbnail of Haploidentical stem cell transplantation abrogates the prognostic impact of FLT3-ITD in acute myeloid leukemia. A report from the Acute Leukemia Working Party of the EBMT

Acute myeloid leukemia (AML) patients harboring the Fms-like tyrosine kinase 3 internal tandem du... more Acute myeloid leukemia (AML) patients harboring the Fms-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD) mutation are considered a particularly high risk patient subset preferentially allocated for allogeneic stem cell transplantation in first remission. Whether FLT3-ITD retains a prognostic role in haploidentical stem cell transplantation (haplo-SCT) is currently unknown. Patients and methods Using the international multicenter registry of the acute leukemia working party of the European society for blood and marrow transplantation, we performed a retrospective analysis to determine whether FLT3-ITD was prognostically significant in T-cell replete haplo-SCT transplanted AML patients. Results We evaluated 293 de-novo AML patients (202 FLT3 wt and 91 FLT3-ITD mut) transplanted in first remission with T-cell replete haplo-SCT between 2005-2016. FLT3-ITD mut patients were more likely to be NPM1 mutated as well as be in the intermediate risk cytogenetic risk category. In multivariate analysis, patients with FLT3-ITD had comparable rates of relapse incidence [Hazard ratio (HR)=1.34, confidence interval (CI) 95%, 0.67-2.7; P=0.9] and leukemia-free survival (HR=0.99, CI 95%, 0.62-1.57; P=0.9) to those of FLT3 wt patients. Survival was not significantly impacted by FLT3-ITD status (HR=0.96, CI 95%, 0.58-1.59; P=0.8), nor were the incidence of non-relapse mortality (HR=0.78, CI 95%, 0.41-1.46; P=0.44), and graft versus host diseasefree/relapse-free survival (HR=0.84, CI 95%, 0.54-1.28; P=0.42). Focused subset analysis of patients with intermediate risk cytogenetics confirmed the absence of a prognostic impact of FLT3-ITD also for this group of patients. Conclusion In AML patients undergoing T-cell replete haplo-SCT, the FLT3-ITD mutation does not retain its prognostic significance, potentially indicative of haplo-SCT's capacity to overcome the negative prognostic consequences of FLT3-ITD in AML.

Research paper thumbnail of Measurable residual disease status and outcome of transplant in acute myeloid leukemia in second complete remission: a study by the acute leukemia working party of the EBMT

Blood Cancer Journal

Measurable residual disease (MRD) prior to hematopoietic cell transplant (HCT) for acute myeloid ... more Measurable residual disease (MRD) prior to hematopoietic cell transplant (HCT) for acute myeloid leukemia (AML) in first complete morphological remission (CR1) is an independent predictor of outcome, but few studies address CR2. This analysis by the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation registry assessed HCT outcomes by declared MRD status in a cohort of 1042 adult patients with AML CR2 at HCT. Patients were transplanted 2006–2016 from human leukocyte antigen (HLA) matched siblings (n = 719) or HLA 10/10 matched unrelated donors (n = 293). Conditioning was myeloablative (n = 610) or reduced-intensity (n = 432) and 566 patients (54%) had in-vivo T cell depletion. At HCT, 749 patients (72%) were MRD negative (MRD NEG) and 293 (28%) were MRD positive (MRD POS). Time from diagnosis to HCT was longer in MRD NEG than MRD POS patients (18 vs. 16 months (P

Research paper thumbnail of Haploidentical Stem Cell Transplantation Attenuates the Prognostic Impact of FLT3-ITD in Acute Myeloid Leukemia

Background Acute myeloid leukemia (AML) patients harboring the fms-like tyrosine kinase 3 interna... more Background Acute myeloid leukemia (AML) patients harboring the fms-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD) mutation are considered a particularly high risk patient subset preferentially allocated for allogeneic stem cell transplantation in first remission. Whether FLT3-ITD retains a prognostic role in haploidentical stem cell transplantation (haplo-SCT) is currently unknown. Patients and methods To determine whether FLT3-ITD is prognostically significant in T-cell replete haplo-SCT transplanted AML patients, we performed a comparative analysis between FLT3wt and FLT3-ITD using the multicenter registry of the acute leukemia working party of the European society for blood and marrow transplantation. Patients included in this analysis were AML patients over the age of 18 who underwent a first T-cell replete haplo-SCT in first remission. Results We evaluated 293 de-novo AML patients (202 FLT3wt and 91 FLT3-ITD) transplanted in first remission with T-cell replete ha...

Research paper thumbnail of Allogeneic hematopoietic stem cell transplantation using HLA-compatible donors in younger high cytogenetic risk CLL patients results in very high long-term progression-free survival and may therefore favorably compete with sequential targeted therapy

Research paper thumbnail of Allogeneic hematopoietic stem cell transplantation in patients over 50: The single centre experience

Analyses of hematopoietic stem cell transplantation (SCT) results are of high importance for deci... more Analyses of hematopoietic stem cell transplantation (SCT) results are of high importance for decision-making on treatment strategy for patients with SCT as a possible therapeutic alternative. In this paper the Czech National Registry of SCT and Transplantation Centre in Pilsen present their joint retrospective analysis of the results of allogeneic SCT in patients with chronic myeloid leukemia (CML) performed in the Czech Republic from 1988 to spring 2005. 295 patients (179 men and 116 women) ranging in age from 6.9 to 59.5 years (median 37.3) underwent transplants. In most cases the donor was an HLA-identical sibling (164; 55.6%) or a voluntary unrelated donor from the register (110; 37.3%), in a minority of cases another relative of the patient (21; 7.1%). Myeloablative conditioning was used in 90% of patients. The source of hematopoietic stem cells was bone marrow in 57%, peripheral blood in 41% and combination of both in 2% of cases. 83.4% of patients underwent transplant in the chronic phase of the illness while 7.8% in the acceleration phase and 6.1% in the blastic phase respectively. The median interval from the diagnosis to SCT was 316 days. Median follow-up after SCT was 2 years. SCT was complicated by acute graft versus host disease of grade II-IV in 33.7% of patients and by chronic graft versus host disease in 36.3% of patients. Median survival was not reached, 18 (6.1%) of patients died due to the relapse of CML and the cause of 101 (34.2%) deaths was transplant-related. Significant trends were observed during the study period: SCT were performed more frequently in older patients, less than one year from the diagnosis, reduced-intensity conditioning was used more often and the source of hematopoietic stem cells was peripheral blood in the majority of patients (p = 0.188 - < 0.0001). Also, transplantation activity changed - the annual rate of SCT increased steadily until 1999, while there was no such an increase between 2000 and 2005. The use of peripheral stem cells was associated with chronic graft versus host disease (p = 0.007). In Cox multivariate analysis the EBMT risk score and the interval from the diagnosis to SCT were identified as independent factors in patient survival. An "ideal" patient, aged under 30, undergoing transplant in the chronic phase of CML within one year since the diagnosis after 2000 had a survival probability of 88% for three years after SCT. It can be concluded that results of allogeneic SCT in CML in the Czech Republic reflect current global trends, are comparable with results achieved in other countries and show significant improvements.

Research paper thumbnail of Timing of Post-Transplantation Cyclophosphamide Administration in Haploidentical Transplantation: A Comparative Study on Behalf of the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation

Biology of Blood and Marrow Transplantation

Research paper thumbnail of Determinants of survival in myelofibrosis patients undergoing allogeneic hematopoietic cell transplantation

Leukemia

We aimed to evaluate the determinants of survival in myelofibrosis patients undergoing allogeneic... more We aimed to evaluate the determinants of survival in myelofibrosis patients undergoing allogeneic hematopoietic cell transplantation (allo-HCT) and to describe factors predicting the main post-HCT complications. This retrospective study by the European Society for Blood and Marrow Transplantation included 2916 myelofibrosis patients who underwent first allo-HCT from an HLA-identical sibling or unrelated donor between 2000 and 2016. After a median follow-up of 4.7 years from transplant, projected median survival of the series was 5.3 years. Factors independently associated with increased mortality were age ≥ 60 years and Karnofsky Performance Status <90% at transplant, and occurrence of graft failure, grades III–IV acute graft-vs.-host disease (aGVHD), and disease progression/relapse during follow-up. The opposing effects of chronic graft-vs.-host disease (GVHD) on non-relapse mortality and relapse incidence resulted in a neutral influence on survival. Graft failure increased in unrelated donor recipients and decreased with myeloablative conditioning (MAC) and negative donor/recipient cytomegalovirus serostatus. Risk of grades III–IV aGVHD was higher with unrelated donors and decreased with MAC. Relapse incidence tended to be higher in patients with intermediate-2/high-risk DIPSS categories and to decrease in CALR -mutated patients. Acute and chronic GVHD reduced the subsequent risk of relapse. This information has potential implications for patient counseling and clinical decision-making.

Research paper thumbnail of Trends in Allogeneic Stem Cell Transplantation for Myelofibrosis in Europe between 1995-2018: An EBMT Retrospective Analysis

Blood

Aim: Dynamic assessment of trends over time in patient- and transplant-specific characteristics a... more Aim: Dynamic assessment of trends over time in patient- and transplant-specific characteristics and outcomes for patients undergoing 1st allogeneic haematopoietic cell transplant (allo-HCT) for Myelofibrosis (MF). Methods and Results: A total of 4142 MF patients were analysed who underwent allo-HCT between 1995-2018 (24-year period) across 278 centres based on data reported to the European Society for Blood and Marrow Transplantation. For analysis, 4 cohorts were considered based on year of allo-HCT: <2006 n=389 (9.4%), 2006-2010 n=910 (22%), 2011-2014 n=1148 (27.7%) and 2015-2018 n=1695 (40.9%). A steady increase in MF allo-HCT activity over time was apparent paralleled with increasing numbers of participating transplant centres. A total of 2603 (62.8%) patients were male, 3239 (78.2%) had Primary MF, 409 (9.9%) and 494 (11.9%) post-Polycythaemia Vera and post-Essential Thrombocythaemia MF, respectively. An increased median interval (lnterquartile range (IQR)) between diagnosis ...

Research paper thumbnail of Post-transplantation cyclophosphamide GvHD prophylaxis after hematopoietic stem cell transplantation from 9/10 or 10/10 HLA-matched unrelated donors for acute leukemia

Leukemia

HLA-matching largely contributes to unrelated donor hematopoietic cell transplantation (UD-HCT) s... more HLA-matching largely contributes to unrelated donor hematopoietic cell transplantation (UD-HCT) success but, due to the selective deletion of alloreactive T-cells, post-transplantation cyclophosphamide (PTCy) could modulate its negative impact on outcomes. We retrospectively compared acute leukemia patients receiving 10/10 or 9/10 HLA allele-matched UD-HCT with PTCy-GvHD prophylaxis between 2010 and 2017, reported to EBMT registry. The 100-day incidence of grade ≥2 and grade ≥3 aGvHD were comparable for 10/10 and 9/10 UD (28% versus 28%, p = 0.8 and 10% versus 8%, p = 0.5, respectively). The 2-year cGvHD and extensive cGvHD were similar between 10/10 and 9/10 UD (35% versus 44%, p = 0.2 and 21% versus 20%, p = 0.6, respectively). The 2-year nonrelapse mortality was 20% after 10/10 and 16% after 9/10 UD-HCT ( p = 0.1). Relapse incidence at 2-year was 24% for 10/10 and 28% for 9/10 UD-HCT ( p = 0.4). Leukemia-free survival at 2-year was the same for 10/10 and 9/10 UD (56 and 56%, p = 0.6, respectively), with comparable overall survival (62 and 59%, p = 0.9, respectively). Multivariate analysis showed no effect of HLA-matching on outcomes. An advanced disease status and patient disability remained the most important factors portending a worse survival. PTCy could alleviate the detrimental effect of HLA-allele mismatching in UD-HCT, potentially expanding the donor pool for acute leukemia patients.

Research paper thumbnail of Haploidentical transplant with post-transplant cyclophosphamide for T-cell acute lymphoblastic leukemia: a report from the EBMT acute leukemia working party

Biology of Blood and Marrow Transplantation

Allogeneic hematopoietic cell transplantation (HCT) is recommended in high-risk patients with T c... more Allogeneic hematopoietic cell transplantation (HCT) is recommended in high-risk patients with T cell acute lymphoblastic leukemia (T-ALL). For patients with no HLA identical donor, haploidentical transplantation (haplo-HCT) is becoming the leading source of stem cell donation. However, data is scarce on predictive factors for outcome in that setting. We identified 122 adults (20% female; median age 31 years; range 18-68) with T-ALL who received a haplo-HCT with post-transplant cyclophosphamide (ptCy) between 2010 and 2017. Median follow-up of living patients was 23 months. The 2-year relapse incidence and non-relapse mortality were 45% and 21%, respectively. The 2-year leukemia-free survival (LFS), overall survival (OS) and GVHD-free, relapse-free survival (GRFS) were 34%, 42% and 27%, respectively. The 2-year LFS and OS were highly influenced by disease status at transplant, being 49% and 55% respectively for first complete remission (CR1), 34% and 50% respectively for CR2, 8% and 12% respectively for patients with active disease. On multivariate analysis, only disease status affected LFS and OS. Transplantation in CR2 negatively affected LFS, whereas active disease at haplo-HCT negatively affected LFS and OS. In conclusion, haplo-HCT with ptCy produced encouraging results in this challenging disease, particularly when performed in CR. Despite the limitation of the small sample size, results were not affected by the type of conditioning, questioning the need for total body irradiation based-myeloablative conditioning (TBI-MAC) in that setting.

Research paper thumbnail of Long-Term Follow-up Data Support the Curative Potential of Allogeneic Hematopoietic Cell Transplantation in Patients with Chronic Lymphocytic Leukemia: A Retrospective Analysis from the Chronic Malignancies Working Party of the EBMT

Blood

Objectives: In patients with refractory chronic lymphocytic leukemia (CLL), the potential to cure... more Objectives: In patients with refractory chronic lymphocytic leukemia (CLL), the potential to cure is unique to allogeneic stem cell transplantation (HSCT). Clearance of minimal residual disease by suspected graft-versus-leukemia effects has been described elegantly and documented in several independent patient cohorts. Yet data from large numbers of patients which support the concept of cure by allogeneic transplantation have not been published. With the advent of new targeted therapies for patients with advanced chemo-refractory CLL, this information becomes crucial for clinical decision making and patient counselling. Therefore, we aimed at the description of long-term survival outcomes, and the estimation of excess mortality compared to the age- and sex-matched general population. Patients and Methods: Data from patients with CLL who had received a first allogeneic HSCT from an HLA-identical sibling (SIB) or alternative donor between January 2000 and December 2010, and who were r...

Research paper thumbnail of Post-Transplant Cyclophosphamide Versus Antithymocyte Globulin in Patients with Acute Myeloid Leukemia Undergoing Allogeneic Stem Cell Transplantation from One Antigen HLA-Mismatched Donors: A Retrospective Analysis from the Acute Leukemia Working Party of the EBMT

Blood

Introduction. Several alternative donor sources are currently available for patients who lack an ... more Introduction. Several alternative donor sources are currently available for patients who lack an HLA-matched related or unrelated donor, including haploidentical, cord blood and one antigen HLA-mismatched donors (9/10 mMUD). Use of post-transplant (allo-HSCT) cyclophosphamide (PTCY) as graft-versus-host disease (GVHD) prophylaxis allowed outcome improvements in the haploidentical setting. Its use has also been reported as a safe and feasible option in 9/10 mMUD transplants. However, to date, the main strategy used as GVHD prophylaxis in 9/10 mMUD allo-HSCT is in vivo T-cell depletion with antithymocite globulin (ATG). Data comparing these two different anti GVHD prophylaxis strategies in 9/10 mMUD allo-HSCT are limited. Methods. We compared PTCY versus ATG as GVHD prophylaxis in patients undergoing 9/10 mMUD allo-HSCT for which high-resolution HLA-allele typing was available in the ALWP/EBMT data registry. Included were adult patients (age>18 years) undergoing their first allo-HS...

Research paper thumbnail of Prognostic Pre-Transplant Factors in Myelodysplastic Syndromes Primarily Treated by Allogeneic Hematopoietic Stem Cell Transplantation: A Retrospective Study on Behalf of the MDS Subcommittee of the Chronic Leukaemia Working Party of the EBMT

Blood

3014FN2 Background: Allogeneic hematopoietic stem cell transplantation (SCT) is associated with i... more 3014FN2 Background: Allogeneic hematopoietic stem cell transplantation (SCT) is associated with iron overload and iron toxicity, especially in patients with myelodysplastic syndromes (MDS). Causes of this increased iron load are transfusions, ineffective erythropoiesis and, myelosuppressive therapy. Investigators found that iron toxicity (often using serum ferritin as a surrogate marker of iron burden) was associated with an adverse effect on non-relapse mortality (NRM) and overall survival (OS). As pre-transplant anti-leukemic treatment may lead to organ damage which may influence outcome after SCT, our study aimed to minimize the role of previous treatment toxicity by only including MDS patients who had not received intensive anti-leukemic treatment prior to SCT conditioning, allowing more insight in the role of iron toxicity in allogeneic SCT. A retrospective analysis of data from the EBMT registry was carried out after an additional survey within selected EBMT centers. Objective...

Research paper thumbnail of Significance of Minimal Residual Disease Monitored by Quantitative Assessment of WT1gene in Patients in First Remission of Acute Myeloid Leukemia Before Allogeneic Stem Cell Transplantation

Blood

4485 Purpose Thanks to the development of knowledge in the field of molecular biology, the great ... more 4485 Purpose Thanks to the development of knowledge in the field of molecular biology, the great progress has been done in risk stratification of patients with acute myeloid leukemia (AML) at diagnosis, in recent years. Based on the recommendations of international expert groups there were identified the patients who may benefit from the allogeneic stem cell transplantation (allo-SCT) as a consolidation of first complete remission (CR). In the absence of an universal marker for minimal residual disease (MRD) measurements, there is still little information about the importance of MRD prior to allo-SCT. Our department has a very good experience with quantitative monitoring of WT1 gene expression as a marker of MRD during treatment of AML. The aim was to retrospectively evaluate the significance of MRD in patients indicated for allo-SCT in 1.CR. Patients and methods Overall 35 patients (pts) in the first morphological CR were transplanted from April 2005 - July 2011. Median age was 46 ...

Research paper thumbnail of Combination Chemotherapy Leading In Advanced MDS Patients to a Rapid Clearence of Bone Marrow Blasts Prior Stem Cell transplantation (SCT) Is Superior to up-Front SCT Even with Intensified Conditioning for Long-Term Survival

Blood

4020 A retrospective analysis of influence of different clinical and laboratory parameters on dis... more 4020 A retrospective analysis of influence of different clinical and laboratory parameters on disease outcome was performed in a cohort of 43 patients with advanced myelodysplasia (MDS)(RAEB…

Research paper thumbnail of Center Characteristics and Procedure-Related Factors Have an Impact on Outcomes of Allogeneic Transplantation for Patients with CLL: A Retrospective Analysis from the European Society for Blood and Marrow Transplantation (EBMT)

Blood

Introduction:Even in the era of novel targeted therapies for the treatment of Chronic Lymphocytic... more Introduction:Even in the era of novel targeted therapies for the treatment of Chronic Lymphocytic Leukemia (CLL) patients, such as BTK, PI3K and BCL2 inhibitors, allogeneic hematopoietic stem cell transplantations (alloHCT) will remain an important treatment option for a subset of patients with very high risk CLL. The current study focused on the impact of center and procedure-related factors on outcomes after alloHCT, taking into account the impact of patient- and disease-related risk factors. Patients and Methods:Data of 684 CLL patients who received a first alloHCT between 2000 and 2011 were analyzed. Their data were collected as part of the EBMT CLL Data Quality Initiative. Outcomes of interest were Event-Free Survival (EFS) up to 5 years after transplantation and mortality in the first 100 days after alloHCT. Outcomes were analyzed by means of the Kaplan-Meier method and Cox proportional hazards models with a frailty (random effects) component to take into account unexplained c...

Research paper thumbnail of Comparison of Matched-Sibling Donors Versus Unrelated Donors in Allogeneic Stem Cell Transplantation (allo-SCT) for Primary Refractory Acute Myeloid Leukemia (PRF AML): A Report of 1041 Patients from the Acute Leukemia Working Party of the EBMT

Blood

Background: PRF-AML is associated with a dismal prognosis. Approximately one third of patients yo... more Background: PRF-AML is associated with a dismal prognosis. Approximately one third of patients younger than 60 years, and 50 % of older patients, with newly diagnosed AML, fail to achieve complete remission (CR) with standard induction chemotherapy. Allo-SCT in the setting of active disease is an alternative strategy. The increased availability of unrelated donors (UD) together with the use of reduced-intensity conditioning (RIC) regimens have opened the possibility for transplantation to a larger number of patients in comparison to standard myeloablative regimens (MAC). Because of the high risk of allo-SCT in this setting, there are still questions on the patient outcome depending on the donor type. Aims: The current study aimed to compare the outcomes of allo-SCT from matched sibling donors (MSD) (n=660) vs UDs (n=381), for patients with PRF AML. Methods: The major endpoints were to assess overall survival (OS), leukemia-free survival (LFS), relapse incidence (RI), and non relapse...

Research paper thumbnail of Outcome of Patients with Myelofibrosis Relapsing after Allogeneic Stem Cell Transplant: A Retrospective Study By the Chronic Malignancies Working Party of EBMT

Blood

Background: Over the last decade, there has been a significant increase in the number of patients... more Background: Over the last decade, there has been a significant increase in the number of patients with Myelofibrosis (MF) undergoing allogeneic stem cell transplantation (SCT). However, scarce information exists on the outcome and management of those patients who relapse following SCT. Moreover, the management of relapse occurring post-SCT is often heterogeneous and ranges from palliation to intensive salvage approaches. We therefore conducted a retrospective EBMT registry analysis of adult MF patients who relapsed following first SCT episode. Results: A total of 1216 adult patients (997 (82%) with Primary MF (PMF) and 219 (18%) with secondary MF (sMF)) underwent 1st allogeneic SCT between 2000 and 2010. A total of 251 patients from this cohort (206 with PMF and 45 with sMF) had conformed relapse ≥ day 30 after HSCT and were included in the analysis. Within this relapse cohort, there were 163 males and 88 females; median age was 55 years old (range 21.5-70 years). A total of 84 pati...