Myeloid Leukemia In First Remission Research Articles

Trends in allogeneic transplantation for favorable risk acute myeloid leukemia in first remission: a longitudinal study of >15 years from the ALWP of the EBMT.

We assessed outcomes of allogeneic transplantation (HSCT) in favorable risk AML in CR1 over 3 time periods. 1850 patients were included, 2005 to 2009- 222, 2010 to 2014 -392, and 2015 to 2021-1236; 526 with t (8:21), 625 with inv (16), and 699 with NPM1mutFLT3WT. Patients transplanted in 2015-2021 were older (p < 0.0001) with more patients ≥60 years of age (p < 0.0001). The most frequent diagnosis in 2015-2021 was NPM1mutFLT3WT vs. t (8:21) in the 2 earlier periods, (p < 0001). Haploidentical transplants (Haplo) increased from 5.9% to 14.5% (p < 0.0001). Graft-versus-host disease (GVHD) prophylaxis with post-transplant cyclophosphamide (PTCy) was more frequent in 2015-2021 vs. the other 2 periods (p < 0.0001). On multivariate analysis, incidence of total chronic GVHD was reduced in HSCTs performed ≥2015 vs. those performed in 2005-2009, hazard ratio (HR) = 0.74 (95% CI 0.56-0.99, p = 0.046) and GVHD-free, relapse-free survival (GRFS) improved for patients transplanted from 2010-2014 vs. those transplanted in 2005-2009, HR = 0.74 (95% CI 0.56-0.98, p = 0.037). Other HSCT outcomes did not differ with no improvement ≥2015. LFS, OS, and GRFS were inferior in patients with t (8:21) with HR = 1.32 (95% CI 1.03-1.68, p = 0.026), HR = 1.38 (95% CI 1.04-1.83, p = 0.027) and HR = 01.25 (95% CI 1.02-1.53, p = 0.035), respectively. In conclusion, this retrospective analysis of HSCT in patients with favorable risk AML, transplanted over 16 years showed an increased number of transplants in patients ≥60 years, from Haplo donors with PTCy. Most importantly, 3-year GRFS improved ≥2010 and total chronic GVHD reduced ≥2015, with no significant change in other HSCT outcomes.

Young (35 years) Mismatched Unrelated Donors and Vice Versa for Allogeneic Stem Cell Transplantation with post-Transplant Cyclophosphamide in Patients with Acute Myeloid Leukemia in First Remission: a Study on Behalf of the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation

Young (<35 years) Haploidentical Versus Old (>35 years) Mismatched Unrelated Donors and Vice Versa for Allogeneic Stem Cell Transplantation with post-Transplant Cyclophosphamide in Patients with Acute Myeloid Leukemia in First Remission: a Study on Behalf of the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation

DNA Sequencing to Detect Residual Disease in Adults With Acute Myeloid Leukemia Prior to Hematopoietic Cell Transplant

Preventing relapse for adults with acute myeloid leukemia (AML) in first remission is the most common indication for allogeneic hematopoietic cell transplant. The presence of AML measurable residual disease (MRD) has been associated with higher relapse rates, but testing is not standardized. To determine whether DNA sequencing to identify residual variants in the blood of adults with AML in first remission before allogeneic hematopoietic cell transplant identifies patients at increased risk of relapse and poorer overall survival compared with those without these DNA variants. In this retrospective observational study, DNA sequencing was performed on pretransplant blood from patients aged 18 years or older who had undergone their first allogeneic hematopoietic cell transplant during first remission for AML associated with variants in FLT3, NPM1, IDH1, IDH2, or KIT at 1 of 111 treatment sites from 2013 through 2019. Clinical data were collected, through May 2022, by the Center for International Blood and Marrow Transplant Research. Centralized DNA sequencing of banked pretransplant remission blood samples. The primary outcomes were overall survival and relapse. Day of transplant was considered day 0. Hazard ratios were reported using Cox proportional hazards regression models. Of 1075 patients tested, 822 had FLT3 internal tandem duplication (FLT3-ITD) and/or NPM1 mutated AML (median age, 57.1 years, 54% female). Among 371 patients in the discovery cohort, the persistence of NPM1 and/or FLT3-ITD variants in the blood of 64 patients (17.3%) in remission before undergoing transplant was associated with worse outcomes after transplant (2013-2017). Similarly, of the 451 patients in the validation cohort who had undergone transplant in 2018-2019, 78 patients (17.3%) with residual NPM1 and/or FLT3-ITD variants had higher rates of relapse at 3 years (68% vs 21%; difference, 47% [95% CI, 26% to 69%]; HR, 4.32 [95% CI, 2.98 to 6.26]; P < .001) and decreased survival at 3 years (39% vs 63%; difference, -24% [2-sided 95% CI, -39% to -9%]; HR, 2.43 [95% CI, 1.71 to 3.45]; P < .001). Among patients with acute myeloid leukemia in first remission prior to allogeneic hematopoietic cell transplant, the persistence of FLT3 internal tandem duplication or NPM1 variants in the blood at an allele fraction of 0.01% or higher was associated with increased relapse and worse survival compared with those without these variants. Further study is needed to determine whether routine DNA-sequencing testing for residual variants can improve outcomes for patients with acute myeloid leukemia.

Meta-analysis on allogeneic transplant for treating pediatric patients with acute myeloid leukemia in first remission: reanalysis of primary data.

Meta-analysis on allogeneic transplant for treating pediatric patients with acute myeloid leukemia in first remission: reanalysis of primary data.

Outcome of autologous stem cell transplantation in patients with favorable-risk acute myeloid leukemia in first remission

ObjectiveTo evaluate the efficacy of autologous hematopoietic stem cell transplantation (auto-HSCT) in patients with favorable-risk acute myeloid leukemia in first remission.MethodTwenty patients who received auto-HSCT at our center between January 2014 and January 2021 were retrospectively reviewed.ResultsUntil last follow-up, three patients in the cohort were dead due to relapse. The estimated 1-year and 5-year overall survival were 95.00% ± 4.87% and 83.82% ± 8.58%, respectively. The estimated 5-year RFS and CIR (cumulative incidence of relapse) were 85.00% ± 7.98% and 15.00% ±7.98%, respectively.ConclusionThe outcome of auto-HSCT in patients with favorable-risk acute myeloid leukemia in first remission was excellent and auto-HSCT could be an effective treatment for these patients.

Maintenance therapy with hypomethylating agents for patients with acute myeloid leukemia in first remission not eligible for allogeneic hematopoietic cell transplantation: A systematic review and meta-analysis

BackgroundOlder patients encompass about 75 % of patients with acute myeloid leukemia (AML). Today therapeutic options to prevent relapse in older patients who managed to achieve complete remission (CR) after intensive chemotherapy are scarce. Recent randomized controlled trials (RCTs) have aimed to reduce the risk of relapse by means of post-CR maintenance therapy. MethodsWe performed a systematic review and meta-analysis of RCTs comparing the efficacy and safety of maintenance with hypomethylating agents (HMA) vs. observation, conventional care or placebo in older patients with AML who are not candidates for allogeneic hematopoietic transplantation (allo−HCT). We searched Cochrane Library, PubMed and conference proceedings up to August 2021. ResultsSix trials were included. Treatment with hypomethylating agents significantly improved overall survival (HR 0.80, 95 % CI 0.70 to 0.91, I2 = 30 %), and disease control (HR 0.80, 95 % CI 0.70 to 0.91, I2 = 0). A significant decrease was seen in both one year mortality (Risk Ratio [RR] 0.61, 95 % CI 0.48 to 0.77, I2 = 0) and mortality at the end of follow-up (RR 0.77, 95 % CI 0.67 to 0.88, I2 = 0). The rate of relapse at 6 months and at one-two years was lower in the HMA arm vs. control (RR, 0.59; 95 % CI, 0.47−0.72; RR, 0.74, I2 = 0; 95 % CI 0.69 – 0.91, I2 = 41 %, respectively). HMA were associated with a statistically non-significant increase in the risk of serious adverse events (RR 3.44, 95 % CI 0.93–12.74, I2 = 80 %). ConclusionsOur meta-analysis shows that in older patients who are not candidates for allo−HCT, maintenance therapy with HMA improves OS and disease control without a statistically significant increase in adverse events.

Dynamic assessment of measurable residual disease in favorable-risk acute myeloid leukemia in first remission, treatment, and outcomes

We aimed to investigate outcomes of different post-remission treatment (PRT) choices based on dynamic measurable residual disease (MRD) by multiparameter flow cytometry in favorable-risk AML (FR-AML). Four hundred and three younger patients with FR-AML in first complete remission (CR1) were enrolled in this registry-based cohort study, including 173 who received chemotherapy (CMT), 92 autologous stem cell transplantation (auto-SCT), and 138 allogeneic SCT (allo-SCT). The primary endpoint was the 5-year overall survival (OS). Subgroup analyses were performed based on dynamic MRD after the 1st, 2nd, and 3rd courses of chemotherapy. In subgroups of patients with negative MRD after 1 or 2 course of chemotherapy, comparable OS was observed among the CMT, auto-SCT, and allo-SCT groups (p = 0.340; p = 0.627, respectively). But CMT and auto-SCT had better graft-versus-host-disease-free, relapse-free survival (GRFS) than allo-SCT in both subgroups. For patients with negative MRD after three courses of chemotherapy, allo-SCT had better disease-free-survival than CMT (p = 0.009). However, OS was comparable among the three groups (p = 0.656). For patients with persistently positive MRD after 3 courses of chemotherapy or recurrent MRD, allo-SCT had better OS than CMT and auto-SCT (p = 0.011; p = 0.029, respectively). Dynamic MRD might improve therapy stratification and optimize PRT selection for FR-AML in CR1.

Dynamic Assessment of Measurable Residual Disease in Favorable-Risk Acute Myeloid Leukemia in First Remission, Treatment, and Outcomes

Purpose:We aimed to investigate outcomes of different post-remission treatment (PRT) choices based on dynamic measurable residual disease (MRD) in favorable-risk AML (FR-AML).Methods: Four hundred and three younger patients with FR-AML in first complete remission (CR1) were enrolled in this registry-based cohort study, including 173 who received chemotherapy (CMT), 92 autologous stem cell transplantation (auto-SCT), and 138 allogeneic SCT (allo-SCT). Subgroup analyses were performed based on dynamic MRD after the first, second, and third courses of chemotherapy. The primary endpoint was the 5-year overall survival (OS).Results: For patients with negative MRD after 1 course of chemotherapy, comparable OS was observed among the CMT, auto-SCT and allo-SCT groups (p=.284). But CMT had better graft-versus-host-disease-free, relapse-free survival (GRFS) than allo-SCT (p=.027). For patients with negative MRD after 2 courses of chemotherapy, comparable OS was also observed among the three groups (p=.967). However, CMT and auto-SCT had better GRFS than allo-SCT (p=.045; p=.020, respectively). For patients with negative MRD after 3 courses of chemotherapy, allo-SCT had better disease-free-survival than CMT (p=.011). However, OS was comparable among the three groups (p=.177). For patients with persistently positive MRD after 3 courses of chemotherapy or recurrent MRD, allo-SCT had better OS than CMT and auto-SCT (p=.012; p=.046, respectively).Conclusions: Dynamic MRD might improve therapy stratification and optimize PRT selection for FR-AML in CR1. CMT might be preferable for patients with negative MRD within 3 cycles of chemotherapy while allo-SCT for patients with persistently positive MRD after 3 cycles of chemotherapy and recurrent MRD. DisclosuresNo relevant conflicts of interest to declare.

Allogeneic Hematopoietic Cell Transplantation in Patients ≤ 60 Years with Intermediate-Risk Acute Myeloid Leukemia in First Remission - Results of the Randomized Etal-1 Trial-

Allogeneic Hematopoietic Cell Transplantation in Patients ≤ 60 Years with Intermediate-Risk Acute Myeloid Leukemia in First Remission - Results of the Randomized Etal-1 Trial-

Outcomes of non-myeloablative allogeneic stem cell transplant in older patients with acute myeloid leukaemia in first remission.

The benefits of non-myeloablative stem cell transplant in older patients with acute myeloid leukaemia are unclear. We compare the long-term outcomes of this regimen in those aged 55-65 years in first remission with a chemotherapy only cohort that achieved durable morphologic remission. Five-year overall survival was similar (32% vs 33%, P = 0.90), as was relapse-free survival (23% vs 20%, P = 0.37). There was a trend for decreased relapse that was balanced against increased non-relapse mortality with transplantation.

Outcomes of Intermediate Risk Acute Myeloid Leukemia in First Remission Undergoing Autologous Stem Cell Transplantation Compared to Allogeneic Stem Cell Transplantation and Chemotherapy Consolidation: A Retrospective, Propensity-Score Adjusted Analysis

Introduction: Post-remission therapy (PRT) for acute myeloid leukemia (AML) remains an area of debate. Autologous stem cell transplantation (AutoSCT) is listed as a possible PRT in patients with intermediate risk AML in the 2017 European Leukemia Network Recommendations. Our center has frequently performed AutoSCT for patients with AML and we aimed to retrospectively compare this strategy with allogeneic SCT (AlloSCT) and consolidation chemotherapy (CMT).Methods: We compared overall survival (OS) and leukemia free survival (LFS) using propensity score (PS) adjusted analysis of patients receiving PRT with: AutoSCT, matched sibling (Sib) AlloSCT, unrelated/mismatch (UD/MM) AlloSCT and CMT. We included patients diagnosed between 1984-2003 (period of AutoSCT at our centre) in first complete remission (CR1) following induction CMT. The 2010 Medical Research Council cytogenetic classification [Grimwade D. Blood 2010;116(3):354-65] was used to define intermediate risk patients. PS-adjusted analysis was performed with marginal mean weighting through stratification (MMWS) method [Hong G. Psych Methods 2012;17(1):44-60.], accounting for sex, de-novo vs. secondary, year of diagnosis, age, chromosome, performance status and high risk features based on prior institutional guidelines (monocytic subtypes, white blood cell > 50x109/L and > 1 cycle to CR1). In brief, log(PS) were calculated from multinomial regression. We excluded cases with log(PS) outside common support area (20% standard deviation from minimum and maximum of each group). Log(PS) were then stratified into 6 strata. MMWS weight was the product of inverse proportion of treatment in each stratum multiplied by proportion of treatment in the whole population. Data was censored at failure or at 10 years. Unadjusted survival analysis was performed using Kaplan Meier method and multivariate logistic regression, and adjustment was also performed by MMWS weight.Results: We identified 259 patients (62 Sib-AlloSCT, 18 UD/MM-AlloSCT, 30 AutoSCT and 149 CMT). Baseline pre-PRT characteristics were used for PS calculation before and after weight adjustment (Table). All SCTs were performed with myeloablative conditioning regimens, most being busulfan-cyclophosphamide based. Median follow-up time for patients who survived beyond 1 year was 6.5 years. Unadjusted OS and LFS were as shown in Figure panel A and B. We excluded 38 patients (2 Sib-AlloSCT, 3 AutoSCT and 33 CMT) based on the PS calculation. The pre-PRT characteristics were well balanced after PS adjustment. Adjusted OS at 2 and 5 years were: Sib-AlloSCT 75.3% and 65.5%; UD/MM-AlloSCT 38.2% and 38.2%; AutoSCT 60.7% and 46.0%; CMT 48.0% and 30.4% (Figure panel C). Adjusted LFS were: Sib-AlloSCT 71.4% and 65.5%; UD/MM-AlloSCT 38.2% and 38.2%; AutoSCT 50.8% and 46.0%; CMT 21.9% and 17.4%, respectively (Figure panel D). Adjusted cumulative incidence of relapse and non-relapse mortality (considered as competing risks) at 5 years were: Sib-AlloSCT 17.2% and 20.9%; UD/MM-AlloSCT 32.6% and 33.3%; AutoSCT 48.4% and 10.8%; CMT 80.5% and 10.9%. Adjusted multivariate HR, 95% confidence interval (CI) and p-value for OS, considering CMT as reference, were: Sib-AlloSCT [0.34, 0.19-0.61, <0.001], UD/MM-AlloSCT [1.03, 0.40-2.66, 0.953] and AutoSCT [0.71, 0.33-1.51, 0.374], respectively. Adjusted multivariate HR, 95%CI and p-value for LFS were Sib-AlloSCT [0.22, 0.13-0.37, <0.001], UD/MM-AlloSCT [0.62, 0.27-1.43, 0.262] and AutoSCT [0.45, 0.21-0.96, 0.038], respectively.Conclusion: Our results show that intermediate risk AML patients who underwent Sib-AlloSCT in CR1 had the best outcomes. AutoSCT significantly improved LFS, in comparison to CMT. However, this benefit did not result in a significant improvement in OS, possibly due to subsequent SCT in the CMT group after relapse. Limitations of our findings include a lack of molecular data and the older time period of our cohort. In a more contemporary cohort, we anticipate that advancements in donor selection and supportive care might improve outcomes with AlloSCT, particularly for the unrelated donor group. Despite this, our results support AutoSCT as a valid option for intermediate risk AML patients who lack an HLA-matched sibling. Further studies integrating molecular genetics and minimal residual disease are needed to guide the selection of patients for AutoSCT who are likely to gain the greatest benefit. [Display omitted] DisclosuresHogge:Novartis, Roche, and Sanofi: Consultancy. Sutherland:Janssen: Honoraria. Gerrie:Seattle Genetics: Honoraria, Membership on an entity's Board of Directors or advisory committees; Lundbeck: Honoraria; Janssen: Membership on an entity's Board of Directors or advisory committees; Roche: Research Funding.

Oral Azacitidine Maintenance Therapy for Acute Myeloid Leukemia in First Remission

BackgroundAlthough induction chemotherapy results in remission in many older patients with acute myeloid leukemia (AML), relapse is common and overall survival is poor.MethodsWe conducted a phase 3, randomized, double-blind, placebo-controlled trial of the oral formulation of azacitidine (CC-486, a hypomethylating agent that is not bioequivalent to injectable azacitidine), as maintenance therapy in patients with AML who were in first remission after intensive chemotherapy. Patients who were 55 years of age or older, were in complete remission with or without complete blood count recovery, and were not candidates for hematopoietic stem-cell transplantation were randomly assigned to receive CC-486 (300 mg) or placebo once daily for 14 days per 28-day cycle. The primary end point was overall survival. Secondary end points included relapse-free survival and health-related quality of life.ResultsA total of 472 patients underwent randomization; 238 were assigned to the CC-486 group and 234 were assigned to the placebo group. The median age was 68 years (range, 55 to 86). Median overall survival from the time of randomization was significantly longer with CC-486 than with placebo (24.7 months and 14.8 months, respectively; P<0.001). Median relapse-free survival was also significantly longer with CC-486 than with placebo (10.2 months and 4.8 months, respectively; P<0.001). Benefits of CC-486 with respect to overall and relapse-free survival were shown in most subgroups defined according to baseline characteristics. The most common adverse events in both groups were grade 1 or 2 gastrointestinal events. Common grade 3 or 4 adverse events were neutropenia (in 41% of patients in the CC-486 group and 24% of patients in the placebo group) and thrombocytopenia (in 22% and 21%, respectively). Overall health-related quality of life was preserved during CC-486 treatment.ConclusionsCC-486 maintenance therapy was associated with significantly longer overall and relapse-free survival than placebo among older patients with AML who were in remission after chemotherapy. Side effects were mainly gastrointestinal symptoms and neutropenia. Quality-of-life measures were maintained throughout treatment. (Supported by Celgene; QUAZAR AML-001 ClinicalTrials.gov number, NCT01757535.)

Allogeneic hematopoietic stem cell transplantation can improve the prognosis of high-risk pediatric t(8;21) acute myeloid leukemia in first remission based on MRD-guided treatment

BackgroundPediatric acute myeloid leukemia (AML) with t(8;21) (q22;q22) is classified as a low-risk group. However, relapse is still the main factor affecting survival. We aimed to investigate the effect of allogeneic hematopoietic stem cell transplantation (allo-HSCT) on reducing recurrence and improving the survival of high-risk pediatric t(8;21) AML based on minimal residual disease (MRD)-guided treatment, and to further explore the prognostic factors to guide risk stratification treatment and identify who will benefit from allo-HSCT.MethodsOverall, 129 newly diagnosed pediatric t(8;21) AML patients were included in this study. Patients were divided into high-risk and low-risk group according to RUNX1-RUNX1T1 transcript levels after 2 cycles of consolidation chemotherapy. High-risk patients were divided into HSCT group and chemotherapy group according to their treatment choices. The characteristics and outcomes of 125 patients were analyzed.ResultsFor high-risk patients, allo-HSCT could improve 5-year relapse-free survival (RFS) rate compared to chemotherapy (87.4% vs. 61.9%; P = 0.026). Five-year overall survival (OS) rate in high-risk HSCT group had a trend for better than that in high-risk chemotherapy group (82.8% vs. 71.4%; P = 0.260). The 5-year RFS rate of patients with a c-KIT mutation in high-risk HSCT group had a trend for better than that of patients with a c-KIT mutation in high-risk chemotherapy group (82.9% vs. 75%; P = 0.400). Extramedullary infiltration (EI) at diagnosis was associated with a high cumulative incidence of relapse for high-risk patients (50% vs. 18.4%; P = 0.004); allo-HSCT can improve the RFS (P = 0.009).Conclusionsallo-HSCT can improve the prognosis of high-risk pediatric t(8;21) AML based on MRD-guided treatment. Patients with a c-KIT mutation may benefit from allo-HSCT. EI is an independent prognostic factor for high-risk patients and allo-HSCT can improve the prognosis.

Autologous versus unrelated donor stem cell transplantation for adults with primary acute myeloid leukemia in first remission

目的分析自体造血干细胞移植（auto-HSCT）与无关供者造血干细胞移植（URD-HSCT）治疗首次完全缓解期（CR1）成人原发性急性髓系白血病（AML）的疗效，比较并探讨两种移植方式的差异及适应人群。方法对2008年3月至2018年11月在中国医学科学院血液病医院接受auto-HSCT及URD-HSCT的成人原发性AML患者进行回顾性分析。结果共纳入147例患者，其中auto-HSCT组87例，URD-HSCT组60例，两组病例基线特征基本一致。auto-HSCT组、URD-HSCT组+30 d粒细胞植活率差异无统计学意义［92.6%（95% CI 86.9%～98.3%）对98.3%（95% CI 95.0%～100.0%），P＝0.270］，auto-HSCT组+60 d血小板累积植活率低于URD-HSCT组［83.6%（95% CI 75.8%～91.4%）对93.3%（95% CI 87.0%～99.6%），P<0.001］。URD-HSCT组急性GVHD累积发生率为56.7%（95% CI 43.0%～68.2%），Ⅱ～Ⅳ度急性GVHD累积发生率为16.7%（95% CI 8.5%～27.2%），慢性GVHD累积发生率为33.3%（95% CI 21.7%～45.4%），广泛型慢性GVHD累积发生率为15.0%（95% CI 7.3%～25.2%）。中位随访53.8（0.8～127.8）个月，auto-HSCT组、URD-HSCT组5年总生存（OS）率分别为71.7%（95% CI 61.7%～81.7%）、67.8%（95% CI 55.8%～79.8%）（P＝0.556），无白血病生存（LFS）率分别为64.6%（95% CI 54.4%～74.8%）、68.1%（95% CI 56.3%～79.9%）（P＝0.642）。auto-HSCT组5年累积复发率高于URD-HSCT组［31.9%（95% CI 22.2%～42.1%）对15.1%（95% CI 7.4%～25.6%），P＝0.015］，移植相关死亡率（TRM）低于URD-HSCT组［3.4%（95% CI 0.9%～8.9%）对16.7%（95% CI 8.5%～27.2%），P＝0.006］。HLA相合及不全相合的无关供者移植在造血重建、GVHD、OS、LFS、复发及TRM方面差异均无统计学意义。遗传学低、中危患者auto-HSCT、URD-HSCT组移植后OS率、LFS率差异均无统计学意义，高危患者auto-HSCT后累积复发率高于URD-HSCT，LFS率低于URD-HSCT。结论对于成人原发性AML-CR1患者，auto-HSCT与URD-HSCT具有相似的OS率和LFS率，auto-HSCT组复发率较高而TRM较低。缺乏同胞供者的遗传学高危组患者应选择URD-HSCT以减少移植后复发，提高生存率。

Allogeneic Stem Cell Transplantation in Patients with Acute Myeloid Leukemia and Poor Karnofsky Performance Status Score. a Study from the Acute Leukemia Working Party (ALWP) of the European Society for Blood and Marrow Transplantation (EBMT)

We report here the results of a retrospective study designed to evaluate outcome of patients with AML with KPS score ≤80% following allo-SCT. The analysis included adult AML patients undergoing allo-SCT in CR1 between 2000 and 2018, with a KPS score at the time of transplant between 50% and 80%. A total of 2,963 patients were identified. Median age at transplant was 55 years (18-77 years). The KPS score was 80% in 85% of the patients and On multivariate analysis, transplant from a MSD was associated with a reduced risk of aGvHD (10/10 UD HR 1.8, 9/10 UD HR 2.4, haplo HR 1.9, CB HR 3.4, p In order to compare outcome following MAC and RIC conditioning regimens the analysis was restricted to patients receiving transplant from MSD or UD. Patients with a KPS score of 80% or In conclusion, allo-SCT is feasible in patients with acute myeloid leukemia in first remission and KPS score ≤80%, with acceptable NRM and survival rates. In patients with a KPS score of 80% a MAC regimen was associated with lower relapse rate, similar NRM and better GRFS as compared to RIC, while in patients with a KPS score

Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Acute Myeloid Leukemia and Karnofsky Performance Status Score Equal or Lower Than 80%. a Study from the Acute Leukemia Working Party (ALWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Acute Myeloid Leukemia and Karnofsky Performance Status Score Equal or Lower Than 80%. a Study from the Acute Leukemia Working Party (ALWP) of the European Society for Blood and Marrow Transplantation (EBMT)

A Safety and Efficacy Study of Oral Venetoclax Tablets and Injectable Azacitidine Versus Best Supportive Care as Maintenance Therapy in Adult Participants With Acute Myeloid Leukemia in First Remission After Conventional Chemotherapy to Evaluate Improvement in Relapse-Free Survival

A Safety and Efficacy Study of Oral Venetoclax Tablets and Injectable Azacitidine Versus Best Supportive Care as Maintenance Therapy in Adult Participants With Acute Myeloid Leukemia in First Remission After Conventional Chemotherapy to Evaluate Improvement in Relapse-Free Survival

Impact of the Number of Consolidation Chemotherapy Cycles Prior to Allogeneic Stem Cell Transplant for Adults with Acute Myeloid Leukemia in First Remission

Impact of the Number of Consolidation Chemotherapy Cycles Prior to Allogeneic Stem Cell Transplant for Adults with Acute Myeloid Leukemia in First Remission

Cord blood transplantation is associated with good outcomes in secondary Acute Myeloid Leukaemia in first remission.

We conducted a retrospective survey within the European Society for Blood and Marrow Transplantation (EBMT) registry to assess the outcomes of cord blood transplantation (CBT) in secondary acute myeloid leukaemia (sAML). Inclusion criteria consisted of ≥18years of age, sAML, first CBT between 2002 and 2016, and either first complete remission (CR) or active disease at CBT. One hundred forty-six patients met the study inclusion criteria. Status at transplantation was first CR (n=97), primary refractory sAML (n=30) or relapsed (n=19) sAML. Neutrophil engraftment was achieved in 118 patients while the remaining 25 patients (17%) failed to engraft. This includes 13% of patients transplanted in first CR versus 30% of those transplanted with active disease (P=0.008). Two-year incidences of relapse were 25% in first CR patients versus 36% in those with advanced disease (P=0.06) while 2-year incidences of nonrelapse mortality were 35% and 49% (P=0.03), respectively. At 2-year overall survival, leukaemia-free survival and graft-versus-host disease (GVHD)-free relapse-free survival were 42% vs. 19% (P<0.001), 40% vs. 16% (P<0.001), and 26% vs. 12% (P=0.002) in first CR patients versus those with advanced disease, respectively. We report here the first study of CBT in a large cohort of sAML patients. Main observation was that CBT rescued approximately 40% of patients with sAML in first CR.

Hematopoietic stem cell transplantation for adult patients with isolated NPM1 mutated acute myeloid leukemia in first remission.

Acute myeloid leukemia (AML) in first remission (CR1) with isolated NPM1 mutation (iNPM1m) is considered a good prognosis genotype, although up to one-third relapse. To evaluate the best transplant strategy, we retrospectively compared autologous stem cell transplantation (auto-SCT), related (MSD), and fully matched unrelated (MUD) allogeneic stem cell transplantation (allo-SCT). We identified 256 adult patients including 125 auto-SCT, 72 MSD, and 59 MUD. The 2-year leukemia-free survival (LFS) was 62% in auto-SCT, 69% in MUD, and 81% in MSD (P = .02 for MSD vs others). The 2-year overall survival (OS) was not different among auto-SCT, MUD, and MSD, reaching 83% (P = .88). The 2-year non-relapse mortality (NRM) was 2.5% in auto-SCT and 7.5% in allo-SCT (P = .04). The 2-year cumulative incidence of relapse (RI) was higher after auto-SCT (30%) than after MUD (22%) and MSD (12%, P = .01). In multivariate analysis, MSD versus auto-SCT but not MUD versus auto-SCT was associated with lower RI (P < .01 and P = .13, respectively) and better LFS (P = .01 and P = .31, respectively). Age correlated with higher NRM (P < .01). Allo-SCT using MSD appears as a reasonable transplant option for young patients with iNPM1m AML in CR1. Auto-SCT was followed by worse RI and LFS, but similar OS to both allo-SCT modalities.