Recent Research
Stay up-to-date on the advancing field of HCT with short summaries and links to the most relevant published research.
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Event-free survival improved with midostaurin for patients with FLT3-ITD-mutated AML
February 2019The addition of the multi-targeted kinase inhibitor, midostaurin, to chemotherapy followed by allogeneic hematopoietic cell transplantation (HCT) significantly improved event-free survival (EFS) for patients with acute myeloid leukemia (AML) with FLT 3 internal tandem duplication (ITD). That’s according to the
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Timing of molecular testing may enhance treatment decisions for patients with AML
January 2019For patients with acute myeloid leukemia (AML), the timing of molecular testing can enhance patient management decisions and clinical trials recommendations. Researchers highlighted evidence that supports molecular testing at diagnosis, complete remission and relapse. At diagnosis, gene panel sequencing
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Allogeneic HCT improves 5-year survival for patients over 60 with AML in CR1
December 2018Devillier R et al. ASH Annual Meeting 2018 - Results from a recent multicenter analysis support hematopoietic cell transplantation (HCT) as a curative option for patients over 60 with acute myeloid leukemia. Researchers found that HCT improved relapse-free survival and overall survival for older patients at 5 years when compared with patients who did not undergo HCT. (Devillier R et al. 2018 ASH Annual Meeting)
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Survival has significantly improved in recent years for patients with AML undergoing HCT
December 2018Canaani J, et al. Journal of Internal Medicine - Researchers analyzed outcomes of patients with acute myeloid leukemia who underwent hematopoietic cell transplantation (HCT) in the past 20 years. Patients who underwent HCT in more recent years experienced significantly improved overall survival and decreased non-relapse mortality. -
Geriatric assessment helps inform decision making and management for older adults with AML
November 2018Loh KP and Klepin HD. Blood Advances - The presenters of this Blood Advances Talk recommend geriatric assessment for older adults with acute myeloid leukemia (AML). They reason that adults of the same age can vary based on underlying health status and physiologic reserve, therefore, decision making and management cannot be optimized using strategies based on chronologic age.
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Emerging prognostic factors identify older adults with high-risk AML likely to benefit from HCT
November 2018Veltri L, et al. Bio Blood Marrow Transplant - In a recent study, researchers analyzed hematopoietic cell transplant (HCT) outcomes in 185 patients (age >65) with high-risk acute myeloid leukemia (AML) and found the strongest independent risk factor for overall survival (OS) and relapse was associated with achievement of measurable residual disease negative status prior to HCT. Additional prognostic factors identified in this study could help identify patients most likely to benefit from HCT treatment.
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Residual disease assessment using next gen-sequencing is predictive of outcomes for patients with AML
October 2018Felicitas T., et al. Blood Journal – In this study, researchers found that next-generation sequencing (NGS) molecular measurable residual disease (MRD) assessment in patients with acute myeloid leukemia (AML) is highly predictive of relapse and survival outcomes when tested in complete remission (CR) before allogeneic hematopoietic cell transplantation (HCT).
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Risk-Adapted Treatment Strategies for Older Patients with AML
July 2018Arellano M, et al. Cancer – In this review, the authors argue that the current treatment algorithm for older patients with acute myeloid leukemia (AML) ─ induction, followed by consolidation (with or without transplantation) versus hospice ─ is no longer sufficient. -
Minimal Residual Disease Identifies Patients Who May Benefit from HCT in First Complete Remission
June 2018Freeman SD, et al. J Clin Oncol – A large-scale, long-term study of adults with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) has shown that measurable or minimal residual disease (MRD) status can be used for risk stratification to determine which patients may benefit from hematopoietic cell transplantation (HCT). -
Sorafenib Improves HCT Outcomes in Patients with FLT3-ITD AML
April 2018Xuan L, et al. Cancer – Three-year relapse rates were cut in half when allogeneic hematopoietic cell transplantation (HCT) patients with FLT3‐ITD acute myeloid leukemia (AML) received pre-transplant sorafenib, according to results of a study of 144 patients. -
Second HCT Effective for Subset of Relapsing Children with Acute Leukemia
April 2018Yaniv I, et al. Biol Blood Marrow Transplant – This multi-center study of 373 children who relapsed after allogeneic hematopoietic cell transplantation (HCT) revealed several prognostic factors predicting overall survival and leukemia-free survival in a subset of these patients. -
Risk-Stratification Tools for Treating Patients with AML in CR1
February 2018Menghrajani KN, et al. Blood Advances – This podcast discusses the state-of-the-art tools available to physicians to risk-stratify patients with acute myeloid leukemia (AML) in first complete remission (CR1). -
Cyclophosphamide is Effective GVHD Prophylaxis in Matched Related and Unrelated HCT
January 2018Ruggeri A, et al. ASH abstract oral presentation, December 2017 – In patients with acute leukemia, post-transplant cyclophosphamide (PT-Cy) can effectively control GVHD after matched related and unrelated donor HCT, and is even more effective when combined with 1 or 2 immunosuppressive drugs. -
Personalized HCT Prognostic Risk Scores Identified for Children with Acute Leukemias
January 2018Bitan M, et al. Biol Blood Marrow Transplant – Researchers analyzed outcomes of children with acute myeloid (AML, n=790) and acute lymphoblastic leukemia (ALL, n=1,096) who survived for at least 1 year in remission after hematopoietic cell transplantation (HCT) have developed personalized long-term prognostic scoring systems for these patients. -
Early Detection of WT1 Minimal Residual Disease Predicts Outcomes in AML
December 2017Lambert J, et al. ASH abstract oral presentation, December 2017 – Using Wilms’ tumor gene (WT1) expression as a marker for minimal residual disease (MRD) in patients with acute myeloid leukemia (AML) is a powerful prognostic factor in treatment selection, according to a study of 713 patients with AML. -
Molecular MRD Detection by NGS Predicts Relapse and Survival in Adults with AML
December 2017Jongen-Lavrencic M, et al. ASH abstract oral presentation, December 2017 – A study of 482 adults with newly diagnosed AML has demonstrated that prospective molecular minimal residual disease (MRD) detection by next-generation sequencing (NGS) of residual leukemia defined by non-DTA mutations in complete remission (CR) is an independent predictor for relapse and survival. -
New Composite Model Predicts Mortality Risk in AML
December 2017Sorror ML, et al. JAMA Oncol – Researchers have developed and validated a novel, risk-stratifying acute myeloid leukemia (AML) composite model (CM) incorporating comorbidities, age, and cytogenetic and molecular risk factors to predict mortality after initial therapy, which can guide clinicians in determining appropriate therapies for their patients with AML. -
The Current Therapeutic Landscape of FLT3 Inhibitors
November 2017Levis M. Blood Advances – In this 15-minute audio presentation, Dr. Mark Levis of Johns Hopkins University reviews the current clinical status of the FLT3 inhibitors midostaurin, quizartinib, gilteritinib, sorafenib, and crenolanib in treating patients with FLT3-mutated acute myeloid leukemia. -
Intermediate-Risk ‘Triple-Negative’ AML at CR1: Improved Outcomes with HCT
October 2017Heidrich K, et al. Ann Oncol – For patients with intermediate-risk acute myeloid leukemia (AML) without FLT3-ITD, biallelic CEBPA, and NPM1 mutations (triple-negative AML) who achieve complete remission, hematopoietic cell transplantation (HCT) significantly improved treatment outcomes. -
Haploidentical HCT is Safe and Effective for Older Patients with AML/MDS
October 2017Ciurea SO, et al. Biol Blood Marrow Transplant – Haploidentical hematopoietic cell transplantation (HCT) is an appropriate therapy for older adults with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS), and can result in a 2-year progression-free survival (PFS) of 74% in selected patients. -
Vorinostat Can Lead to Lower Incidence of Acute GVHD
September 2017Choi SW, et al. Blood – Adding vorinostat to a standard pre-transplant graft-versus-host disease (GVHD) prophylaxis can significantly lower the incidence of acute GVHD, according to results of a prospective, phase II study. -
Study Identifies Optimal Therapies for Relapsed Pediatric AML
September 2017Karlsson L, et al. Br J Haematol – A large-scale study of children with relapsed acute myeloid leukemia (AML) has found that intensive re-induction therapy followed by hematopoietic cell transplantation (HCT) in second complete remission (CR2) is the best available treatment for the majority of these patients. -
Comorbidity Scores are Prognostic of Overall Survival in Relapsed/Refractory AML
July 2017Middeke JM, et al. Biol Blood Marrow Transplant – Comorbidity evaluations can provide clinically useful prognostic information at the time of diagnosis of relapsed or refractory acute myeloid leukemia (AML), according to results of a phase II trial. -
Cytogenetic Risk Prognostic of HCT Outcomes in Older Patients with AML in CR2
May 2017Michelis FV, et al. Cancer – Allogeneic hematopoietic cell transplantation (HCT) is a curative treatment option for older patients with acute myeloid leukemia (AML) in second complete remission (CR2), particularly for those with favorable or intermediate cytogenetic risk, according to researchers who studied 169 transplants at 78 centers. -
Updated ELN Recommendations on the Diagnosis and Management of AML in Adults
March 2017Döhner H, et al. Blood – The 2017 European LeukemiaNet (ELN) recommendations for diagnosis and management of acute myelogenous leukemia (AML) in adults includes updates based on several recent advances in AML, most notably advances in knowledge of the genomic landscape of the disease. -
Improved HCT Outcomes for Fit Patients with MDS/AML After Standard Conditioning
March 2017Scott BL, et al. J Clin Oncol – This randomized phase III trial of 272 adults with myelodysplastic syndromes (MDS) and acute myelogenous leukemia (AML) found that patients with hematopoietic cell transplantation (HCT) comorbidity index scores ≤4 and with <5% marrow myeloblasts had better relapse-free survival (RFS) after HCT with myeloablative conditioning (MAC), than after reduced-intensity conditioning (RIC). -
Emerging Trends in Managing FLT3-Mutated AML
January 2017Pratz KW, et al. Blood – In this installment of the “How I treat …” series in Blood, the authors present four clinical case studies of patients with FLT3-mutated acute myeloid leukemia (AML) to illustrate their strategies to treat both newly diagnosed patients and those with relapsed or refractory disease. -
Tyrosine Kinase Inhibitor Improves Post-HCT Outcomes in Patients with AML
December 2016Brunner AM, et al. Br J Haematol – In a single-center study of consecutive adults with FLT3 - internal tandem duplication (ITD) acute myeloid leukemia (AML), 29 patients transplanted in first complete remission (CR1) who received post-transplant sorafenib had significantly better outcomes than 55 patients in a control cohort. -
High-Risk Patients with AML Undergoing Early HCT Have Improved Outcomes
December 2016Pagel JM, et al. ASH abstract oral presentation, December 2016 – In newly diagnosed adults with acute myeloid leukemia (AML), early identification of high-risk cytogenetics and HLA testing with an organized effort to identify a suitable allogeneic hematopoietic cell transplant (HCT) donor can lead to a higher transplant rate and better transplant outcomes, according to research presented at ASH. -
Somatic Mutations Improve Transplant Prognostics in MDS
November 2016Della Porta MG, et al. J Clin Oncol – In a study of 401 patients with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) evolving from MDS (MDS/AML), researchers found that ASXL1, RUNX1, and TP53 mutations were independent predictors of higher rates of relapse and lower overall survival (p values <0.001 and p=0.017, respectively) after hematopoietic cell transplantation (HCT) in both patients with MDS and MDS/AML. -
Is Cord Blood the Best Alternative Graft Source for Patients with Minimal Residual Disease?
October 2016Milano F, et al. N Engl J Med – A retrospective study of 582 patients with acute leukemia or myelodysplastic syndromes has shown that umbilical cord blood may be a superior graft choice for the approximately 70% of patients who lack an HLA-matched related donor, especially if they are minimal residual disease positive (MRD+). -
Pre-HCT Genetic Profiling Can Identify AML Patients at Increased Risk for Relapse
September 2016Luskin MR, et al. Biol Blood Marrow Transplant – Researchers used next generation sequencing (NGS) of 26 genes and found mutations in TP53, WT1 and FLT3-ITD were associated with an increased risk of acute myeloid leukemia (AML) relapse after HCT (adjusted Hazard Ratio [aHR] 2.90, p=0.009; aHR 2.51, p=0.02; and aHR 1.83, p=0.07, respectively). -
FLT3 Mutation Has No Effect on Survival in HCT for AML
July 2016Deol A, et al. Cancer – This multi-center study of 511 adults with acute myeloid leukemia (AML) showed that FLT3 mutation status had no effect on non-relapse mortality (NRM), leukemia-free survival (LFS), or overall survival (OS) after hematopoietic cell transplantation (HCT). -
Comparable Survival, More GVHD Risk Using EBV-Seropositive HCT Donors
July 2016Styczynski J, et al. J Clin Oncol – A large-scale retrospective study of the effect of Epstein-Barr virus (EBV) serostatus on outcomes of allogeneic HCT in 11,364 patients with acute leukemia found that patients receiving grafts from EBV-seropositive donors had the same overall survival as patients who received grafts from EBV-seronegative donors. -
Early HCT Warranted in Therapy-Related MDS, AML
June 2016Finke J, et al. Bone Marrow Transplant – In this long-term study of 79 patients with therapy-related myelodysplasia (t-MDS) or acute myeloid leukemia (t-AML), 10-year disease-free survival (DFS) and OS rates in this high-risk patient cohort were 35% and 38%, respectively. -
Review: How I Treat AML Patients with Preexisting Comorbidities
June 2016Ofran Y, et al. Blood – In this installment of the “How I treat …” series in Blood, the authors present eight clinical vignettes of patients with acute myeloid leukemia (AML), and discuss practical approaches to comorbidities that affect clinical decision-making, especially in determining which patients are candidates for allogeneic HCT. -
Minimal Residual Disease a Powerful Prognosticator in Standard-Risk AML
February 2016Ivey A, et al. N Engl J Med – Minimal residual disease (MRD) detected by quantitation of NPM1-mutated transcripts is more predictive of relapse and survival than molecular profiling in patients with NPM1-mutated acute myeloid leukemia (AML), according to a study of 2,569 samples obtained from 346 patients with NPM1-mutated AML. -
Review: Maintenance with TKIs Post-Transplant in FLT3-ITD Positive AML
February 2016Schiller GJ, et al. Biol Blood Marrow Transplant – This review discusses the clinical evidence on FLT3-specific tyrosine kinase inhibitors (TKIs) being used as post-transplant maintenance in patients with FLT3-ITD acute myelogenous leukemia (AML) who are at high-risk for disease relapse. -
Review: HCT for Patients with AML in First Complete Remission
January 2016Cornelissen JJ, et al. Blood – In this review, part of a Blood journal special review series on acute myeloid leukemia (AML), the authors note that although the majority of patients AML enter remission upon induction chemotherapy, the risk of relapse is considerable and it varies greatly according to age and genetic subtype. -
AML Review Series in Blood Highlights Latest Advances
January 2016Löwenberg B, et al. Blood – The journal Blood has published a six-article series examining the current and future treatment of AML to guide the optimal individual treatment approach. -
ATG Significantly Lowers Rate of Chronic GVHD
January 2016Kröger N, et al. N Engl J Med – Adding anti-thymocyte globulin (ATG) to a myeloablative conditioning regimen can cut the 2-year rate of chronic GVHD in half, according to results of a multi-center, prospective randomized trial of 168 patients transplanted for acute leukemias. -
Minimal Residual Disease Best Method to Define Complete Remission in AML
January 2016Araki D, et al. J Clin Oncol – Results of a study of 359 adults transplanted for acute myeloid leukemia (AML) indicate there is clinical relevance to minimal residual disease (MRD) that may lead to a better estimate of expected treatment outcomes. -
Higher Relapse-Free Survival with Myeloablative HCT in Patients with AML and MDS
December 2015Scott BL, et al. ASH abstract oral presentation, December 2015 – A phase III randomized trial presented at the ASH Annual Meeting has shown that although reduced-intensity regimens have lower treatment-related mortality (TRM), they also incur higher relapse rates. -
Azacitidine Feasible as a Bridge to HCT in Patients with Higher-Risk MDS or Low-Blast Count AML
December 2015Voso MT, et al. ASH abstract oral presentation, December 2015 – A multi-center study of 93 patients with higher-risk MDS or low blast count AML demonstrated that induction with azacitidine (AZA) is a feasible “bridge to transplant,” with 52% of patients able to undergo HCT. -
Meta-Analysis: Age Not a Determining Factor in HCT Selection for AML
November 2015Rashidi A, et al. Biol Blood Marrow Transplant – A meta-analysis of 749 patients >60 years undergoing hematopoietic cell transplantation (HCT) for acute myeloid leukemia (AML) has shown that older age per se should not be the sole criterion against selecting HCT therapy. -
Low Non-Relapse Mortality, GVHD after HCT in Older Patients with AML in First Remission
November 2015Devine SM, et al. J Clin Oncol – A multi-center phase II study of 114 patients aged 60-74 years with AML in first remission has found that reduced-intensity HCT can lead to a low incidence of non-relapse mortality (NRM) and graft-versus-host disease (GVHD). -
HCT Yields Best Outcomes in Patients ≥60 Years with AML
October 2015Versluis J, et al. Lancet Haematol – A study analyzing a series of four multi-center trials has shown significantly better 5-year overall survival for allogeneic hematopoietic cell transplantation (HCT) in patients aged 60 years and older with acute myeloid leukemia (AML) when compared with no and other post-remission therapies. -
Review: Acute Myeloid Leukemia
October 2015Döhner H, et al. N Engl J Med – In this review, the authors describe recent advances in disease classification, identification of prognostic factors, understanding of the genomic landscape, current treatment, and new therapies under investigation in acute myeloid leukemia (AML) in adults. -
AML patients ≥70 Achieve 60% Estimated Survival with UCB HCT
October 2015Sandhu KS, et al. Biol Blood Marrow Transplant – In this single-center retrospective study of 22 transplant recipients 70 years and older, 10 patients undergoing umbilical cord blood (UCB) transplantation had an estimated 2-year overall survival of 60%. -
Review: Treating Early Relapsed and Refractory AML
August 2015Thol F, et al. Blood – In this installment of the “How I treat ...” series in Blood, the authors note that few patients with primary refractory or resistant acute myeloid leukemia (AML) can be cured with conventional salvage therapy, and they should therefore be evaluated for allogeneic hematopoietic cell transplantation (HCT). -
Haploidentical vs. Matched Unrelated HCT for AML
July 2015Ciurea SO, et al. Blood — This study of 2,174 adults with acute myeloid leukemia (AML) compared using haploidentical (n=192) and 8/8 HLA-matched unrelated donor (n=1,982) grafts. -
Extramedullary Disease in AML Does Not Affect HCT Outcomes
May 2015Goyal SD, et al. Bone Marrow Transplant — Researchers analyzing outcomes of 9,797 allogeneic transplants in patients with AML - including 814 patients with extramedullary disease (EMD) - have found that EMD does not affect transplant outcomes. -
Comparable Survival in Cord Blood, Mismatched Unrelated HCT for AML
May 2015Malard F, et al. Biol Blood Marrow Transplant — A long-term study of 651 adults with acute myeloid leukemia (AML) receiving reduced-intensity (RIC) or nonmyeloablative conditioning (NMA) transplantation has found comparable four-year survival with umbilical cord blood and mismatched unrelated donors: 47.9% vs. 50.5%, respectively (p=0.81). -
Comparable HCT Outcomes for Patients with de novo or Secondary AML in CR1
April 2015Michelis FV, et al. Bone Marrow Transplant — A single-center study of 264 patients with de novo acute myeloid leukemia (AML; n=180) and secondary AML (sAML; n=84) in first complete remission (CR1) undergoing allogeneic hematopoietic cell transplantation (HCT) has shown that both patient groups have comparable long-term outcomes. -
Review: How I Treat the Older Patient with AML
January 2015Ossenkopple G, et al. Blood – In this installment of the “How I treat” series in Blood, the authors discuss the highly heterogeneous clinical biology of acute myeloid leukemia (AML) among patients and how those variations are relevant for prognosis and treatment choice. -
T Cell-Depletion Yields Low Rates of Chronic GVHD, Relapse in Patients Transplanted for AML
September 2011Devine SM, et al. Biol Blood Marrow Transplant – Hematopoietic cell transplantation (HCT) using T cell-depleted grafts can yield low rates of chronic graft-versus-host disease (GVHD) and relapse in patients with acute myelogenous leukemia (AML) in first remission (CR1), according to the results of Blood and Marrow Transplant Clinical Trials Network (BMT CTN) trial 0303.