Marcelo C Pasquini

University of Wisconsin - Milwaukee, Milwaukee, Wisconsin, United States

Are you Marcelo C Pasquini?

Claim your profile

Publications (83)464.76 Total impact

  • [Show abstract] [Hide abstract]
    ABSTRACT: Recent studies support the use of bortezomib-based therapies in light chain amyloidosis (AL). We performed a retrospective analysis of the safety, efficacy and long-term survival (median follow-up 3 years) after bortezomib-based treatment in 28 consecutive patients with de novo AL deemed ineligible at initial presentation. The first 14 patients received bortezomib and dexamethasone (VD), and the second 14 patients received cyclophosphamide, bortezomib and dexamethasone (CVD; CyBorD). Both regimens were well tolerated with no treatment-related mortality. The overall hematological response (HR) rate was 93% in both the groups. Median time to response was shorter in the CVD group (39 days vs 96 days in the VD group; P=0.002). Hematological and organ responses induced with bortezomib-based therapy enabled 8 (33%) of initially transplant ineligible patients to undergo autologous hematopoietic stem cell transplantation (AHCT), including 4 patients with cardiac stage III or IV. Seven of the eight patients (88%) who underwent subsequent AHCT achieved sustained HR at a median of 33 months posttransplant. These data suggest that bortezomib-based induction followed by AHCT is a viable therapeutic strategy for transplant-ineligible AL. Larger, multicenter prospective trials are necessary to confirm our findings.Bone Marrow Transplantation advance online publication, 27 April 2015; doi:10.1038/bmt.2015.73.
    Bone marrow transplantation 04/2015; DOI:10.1038/bmt.2015.73 · 3.47 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: Prospective validation of the hematopoietic cell transplantation-comorbidity index (HCT-CI) using contemporary patients treated with HCT across the Unites States is necessary to confirm its widespread applicability. We performed a prospective observational study including all patients (8115 recipients of allogeneic and 11,652 recipients of autologous HCT) who underwent first HCT that was reported to the CIBMTR between 2007 and 2009. In proportional hazards models, increased HCT-CI scores were independently associated with increases in hazard ratios for NRM (p<0.0001) and overall mortality (p<0.0001) among recipients of allogeneic HCT. HCT-CI Scores of ≥3 were uniformly associated with higher risks for outcomes in both allogeneic and autologous HCT, and all subgroups regardless of diagnoses, age, and conditioning intensity. Recipients of allogeneic HCT with scores of 1-2 who were aged <18 or were treated with lower intensity conditioning regimens had similar outcomes compared to those with score 0. Higher risks for overall mortality, but not for NRM, were observed among recipients of autologous HCT with scores of 1-2 versus 0. Our results confirm the validity the HCT-CI in both allogeneic and autologous HCT. The index should be used as a valid standard-of-care health measure in counseling patients for HCT, in clinical trial design, and in adjusting outcome analyses. Copyright © 2015 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.
    Biology of blood and marrow transplantation: journal of the American Society for Blood and Marrow Transplantation 04/2015; DOI:10.1016/j.bbmt.2015.04.004 · 3.35 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: Limited clinical data are available to assess whether the sequencing of cyclophosphamide (Cy) and total body irradiation (TBI) changes outcomes. We evaluated the sequence in 1769 (CyTBI N=948, TBICy N=821) recipients of related or unrelated hematopoietic cell transplantation (HCT) who received TBI (1200-1500cGY) for acute leukemia from 2003 to 2010. The two cohorts were comparable for median age, performance score, type of leukemia, first complete remission, Ph+ ALL, HLA matched siblings, stem cell source, anti-thymocyte globulin use, TBI dose, and type of graft-versus-host disease (GVHD) prophylaxis. The sequence of TBI did not significantly affect TRM (24% vs. 23% at 3y, p=0.67; relative risk [RR] 1.01, p=0.91), leukemia relapse (27% vs. 29% at 3y, p=0.34; RR 0.89, p=0.18), leukemia-free survival (49% vs. 48% at3y, p=0.27; RR 0.93, p=0.29), chronic GVHD (45% vs. 47% at 1y, p=0.39; RR 0.9, p= 0.11) or overall survival (53% vs. 52% at 3y, p=0.62; RR 0.96, p=0.57) for CyTBI and TBICy respectively. Corresponding cumulative incidences of sinusoidal obstruction syndrome were 4% and 6% at 100 days (p=0.08). This study demonstrates that the sequence of Cy and TBI does not impact transplant outcomes and complications in patients with acute leukemia undergoing HCT with myeloablative conditioning. Copyright © 2015 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.
    Biology of blood and marrow transplantation: journal of the American Society for Blood and Marrow Transplantation 03/2015; DOI:10.1016/j.bbmt.2015.03.017 · 3.35 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: Clinical outcomes after primary graft failure (PGF) remain poor. Here we present a large retrospective analysis (n=23 272) which investigates means to prevent PGF and early detection of patients at high risk. In patients with hematologic malignancies, who underwent their first myeloablative allogeneic hematopoietic cell transplantation, PGF was reported in 1,278 (5.5%), and there was a marked difference in PGFs using peripheral blood stem cell compared to bone marrow grafts (2.5 vs 7.3%; P<0.001). A 4-fold increase of PGF was observed in myeloproliferative disorders compared to acute leukemia (P<0.001). Other risk factors for PGF included recipient age below 30, HLA-mismatch, male recipients of female donor grafts, ABO-incompatibility, busulfan/cyclophosphamide conditioning, and cryopreservation. In bone marrow transplants, total nucleated cell doses ⩽2.4 × 10(8)/kg were associated with PGF (OR 1.39; P<0.001). The use of tacrolimus-based immunosuppression and granulocyte colony-stimulating factor were associated with decreased PGF risk. These data, allow clinicians to do more informed choices with respect to graft source, donor selection, conditioning and immunosuppressive regimens to reduce the risk of PGF. Moreover, a novel risk score determined on day 21 post-transplant may provide the rationale for an early request for additional hematopoietic stem cells.Leukemia accepted article preview online, 16 March 2015. doi:10.1038/leu.2015.75.
    Leukemia 03/2015; DOI:10.1038/leu.2015.75 · 9.38 Impact Factor
  • Biology of Blood and Marrow Transplantation 02/2015; 21(2). DOI:10.1016/j.bbmt.2014.11.027 · 3.35 Impact Factor
  • Source
    [Show abstract] [Hide abstract]
    ABSTRACT: Overall survival following hematopoietic cell transplantation (HCT) has improved over the past two decades through better patient selection and advances in HLA typing, supportive care, and infection prophylaxis. Nonetheless, mortality rates are still unsatisfactory and transplant-related mortality remains a major cause of death after unrelated allogeneic HCT. Since there are no known pre-HCT, non-HLA biologic predictors of survival following transplant, for over a decade, scientists have been investigating the role of non-HLA germline genetic variation in survival and treatment-related mortality after HCT. Variation in single nucleotide polymorphisms (SNPs) has the potential to impact chemotherapy, radiation, and immune responses, leading to different post-HCT survival outcomes. In this paper, we address the current knowledge of the contribution of genetic variation to survival following HCT and discuss study design and methodology for investigating HCT survival on a genomic scale.
    Current Hematologic Malignancy Reports 02/2015; DOI:10.1007/s11899-014-0246-x · 2.29 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: There are limited data to guide the choice of high-dose therapy (HDT) regimen prior to autologous hematopoietic cell transplantation (AHCT) for patients with Hodgkin (HL) and non-Hodgkin lymphoma (NHL). We studied 4,917 patients (NHL n=3,905; HL n=1,012) who underwent AHCT from 1995-2008 using the most common HDT platforms: BEAM (n=1730), CBV (n=1853), BuCy (n=789), and TBI-containing (n=545). CBV was divided into CBV(high) and CBV(low) based on BCNU dose. We analyzed the impact of regimen on development of idiopathic pulmonary syndrome (IPS), transplant-related mortality (TRM), progression free and overall survival (PFS and OS). The 1-year incidence of IPS was 3-6% and was highest in recipients of CBV(high) (HR 1.9) and TBI (HR 2.0) compared to BEAM. 1-year TRM was 4-8% and was similar between regimens. Among patients with NHL, there was a significant interaction between histology, HDT regimen, and outcome. Compared to BEAM, CBV(low) (HR 0.63) was associated with lower mortality in follicular lymphoma (p<0.001), and CBV(high) (HR1.44) with higher mortality in diffuse large B-cell lymphoma (p=0.001). For patients with HL, CBV(high) (HR1.54), CBV(low) (HR1.53), BuCy (HR1.77) and TBI (HR 3.39) were associated with higher mortality compared to BEAM (p<0.001). The impact of specific AHCT regimen on post transplant survival is different depending on histology; therefore, further studies are required to define the best regimen for specific diseases. Copyright © 2015 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.
    Biology of blood and marrow transplantation: journal of the American Society for Blood and Marrow Transplantation 02/2015; 20(2). DOI:10.1016/j.bbmt.2015.02.005 · 3.35 Impact Factor
  • Source
    [Show abstract] [Hide abstract]
    ABSTRACT: The Patient Protection and Affordable Care Act requires that healthcare insurers cover routine patient costs associated with participating in clinical trials for cancer and other life-threatening diseases. There is a need to better define 'routine costs' within the context of hematopoietic stem cell transplantation (HSCT) clinical trials. This white paper presents guidance on behalf of the American Society for Blood and Marrow Transplantation for defining a standard HSCT episode and delineates components that may be considered as routine patient costs versus research costs. The guidelines will assist investigators, trial sponsors and transplant centers in planning for clinical trials that are conducted as a part of the HSCT episode and will inform payers who provide coverage for transplantation. Copyright © 2015 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.
    Biology of blood and marrow transplantation: journal of the American Society for Blood and Marrow Transplantation 01/2015; 21(4). DOI:10.1016/j.bbmt.2014.12.030 · 3.35 Impact Factor
  • Bone Marrow Transplantation 01/2015; 50(4). DOI:10.1038/bmt.2014.318 · 3.47 Impact Factor
  • Source
    [Show abstract] [Hide abstract]
    ABSTRACT: To develop a novel acute graft-versus-host disease (GVHD) Risk Score, we examined the GVHD clinical stage and grade of 1723 patients at the onset of treatment with systemic steroids. Using clinical grouping, descriptive statistics and recursive partitioning, we identified poorly responsive, high-risk (HR) acute GVHD by the number of involved organs and severity of GVHD at onset. The overall response [(complete response/partial response (CR/PR)] rate 28 days after initiation of steroid therapy for acute GVHD was lower in the 269 patients with HR-GVHD than in the 1454 patients with standard risk (SR)-GVHD [44% (95% CI 38-50%) vs. 68% (95% CI 66-70%), p<0.001. Patients with HR-GVHD were less likely to respond at day 28 [odds ratio (OR), 0.3, 95% CI 0.2-0.4, p<0.001], and had higher risks of mortality [relative risk (RR) 2.1, 95% CI 1.7-2.6, P<0.001] and transplant-related mortality (RR 2.5, 95% CI 2.0-3.2%, p<0.001) compared to patients with SR-GVHD. This refined definition of acute GVHD risk is a better predictor of response, survival and transplant-related mortality than other published acute GVHD risk scores. Patients with HR-GVHD are candidates for studies investigating new treatment approaches. Likewise, patients with SR-GVHD are candidates for studies investigating less toxic therapy. Copyright © 2015 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.
    Biology of blood and marrow transplantation: journal of the American Society for Blood and Marrow Transplantation 01/2015; 21(4). DOI:10.1016/j.bbmt.2015.01.001 · 3.35 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: Choosing Wisely(®) is a medical stewardship initiative led by the American Board of Internal Medicine Foundation in collaboration with professional medical societies in the United States. The American Society of Hematology (ASH) released its first Choosing Wisely(®) list in 2013. Using the same evidence-based methodology as in 2013, ASH has identified 5 additional tests and treatments that should be questioned by clinicians and patients under specific, indicated circumstances. The ASH 2014 Choosing Wisely(®) recommendations include: (1) do not anticoagulate for more than 3 months in patients experiencing a first venous thromboembolic event in the setting of major, transient risk factors for venous thromboembolism; (2) do not routinely transfuse for chronic anemia or uncomplicated pain crises in patients with sickle cell disease; (3) do not perform baseline or surveillance computed tomography scans in patients with asymptomatic, early-stage chronic lymphocytic leukemia; (4) do not test or treat for heparin-induced thrombocytopenia if the clinical pretest probability of heparin-induced thrombocytopenia is low; and (5) do not treat patients with immune thrombocytopenia unless they are bleeding or have very low platelet counts. © 2014 by The American Society of Hematology. All rights reserved.
    Blood 12/2014; 124(24):3524-8. DOI:10.1182/blood-2014-09-599399 · 9.78 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: Cytokine-based mobilization in light chain (AL ) amyloidosis is frequently complicated by fluid overload, weight gain, cardiac arrhythmias, and peri-mobilization mortality. We analyzed hematopoietic progenitor cells (HPC) mobilization outcomes in 49 consecutive AL amyloidosis patients at our institution between 2004-2013 with G-CSF (G) (10μg/kg/d) (n=25) vs. an institutional protocol to limit G exposure using plerixafor (P) (P at 0.24mg/kg SC starting day 3 of G 10μg/kg) (n=24). G+P strategy yielded higher total CD34+ cells/kg (12.8 ×106 vs. 6.3 ×106; p<0.001), and CD34+ cells/kg collected on day 1(10.8 ×106 vs. 4.9 ×106, p=0.004) compared with the G cohort. More G+P patients collected ≥5 ×106 CD34+HPCs/kg (22 vs.16, p=0.02) and ≥10 ×106 CD34+ HPCs/kg (13 vs. 5,p=0.01). Four patients (16%) had mobilization failure with G; none with G+P. Peri-mobilization weight gain was lower with G+P strategy (median weight gain 1 vs. 7 lbs., p=0.009). Numbers of apheresis sessions (median 1 vs. 1, p=0.52), number of hospitalization days (median 1.1 vs. 1.6, p=0.52), transfusions, use of intravenous antibiotics and cardiac arrhythmias were similar. In conclusion our study demonstrates that upfront use of G+P as a mobilization strategy results in superior HPC collection, no mobilization failures, and less weight gain than G alone.
    Biology of Blood and Marrow Transplantation 12/2014; 20(12). DOI:10.1016/j.bbmt.2014.08.002 · 3.35 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: Hematopoietic cell transplantation (HCT) with non-myeloablative (NMA) conditioning for lymphoproliferative diseases (LD) includes fludarabine with and without low-dose TBI. Transplant outcomes were compared among patients aged ⩾40 years with LD who received a HCT with TBI (N=382) or no-TBI (N=515) NMA from 2001 to 2011. The groups were comparable except for donor, graft, prophylaxis for GVHD, disease status and year of HCT. Cumulative incidences of grades II-IV GVHD at 100 days were 29% and 20% (P=0.001) and of chronic GVHD at 1 year were 54% and 44% (P=0.004) for TBI and no-TBI, respectively. Multivariate analysis of progression/relapse, treatment failure and mortality showed no outcome differences by conditioning. Full donor chimerism at day 100 was observed in 82% vs 64% in the TBI and no-TBI groups, respectively (P=0.006). Subsets of the four most common conditioning/GVHD prophylaxis combinations demonstrated higher rates of grades II-IV acute (P<0.001) and chronic GVHD (P<0.001) among recipients of TBI-mycophenolate mofetil (MMF) compared with other combinations. TBI-based NMA conditioning induces faster full donor chimerism, but overall survival outcomes are comparable to no-TBI regimens. Combinations of TBI and MMF are associated with higher rates of GVHD without impact on survival outcomes in patients with LD.Bone Marrow Transplantation advance online publication, 1 December 2014; doi:10.1038/bmt.2014.269.
    Bone Marrow Transplantation 12/2014; 50(3). DOI:10.1038/bmt.2014.269 · 3.47 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: The Blood and Marrow Transplant Clinical Trials Network conducts large, multi-institutional clinical trials with the goal of improving the outcomes of hematopoietic cell transplantation (HCT) for patients with life-threatening disorders. Well designed HCT trials benefit from standardized criteria for defining diagnoses, treatment plans and graft source selection. In this perspective, we summarize evidence supporting criteria for the selection of related and unrelated adult volunteer progenitor cell donors or umbilical cord blood units. These standardized criteria for graft source selection have been adopted by the BMT CTN to enhance the interpretation of clinical findings within and among future clinical protocols.
    Biology of blood and marrow transplantation: journal of the American Society for Blood and Marrow Transplantation 09/2014; 21(1). DOI:10.1016/j.bbmt.2014.09.017 · 3.35 Impact Factor
  • Source
    A D'Souza, M Pasquini, R Spellecy
    [Show abstract] [Hide abstract]
    ABSTRACT: Hematopoietic cell transplantation (HCT) is a complex and highly specialized medical treatment that is associated with significant risks, including death. Furthermore, transplantation is offered to patients who often have no other curative treatment alternatives. The routine-consent process for HCT typically occurs before HCT and is influenced by many factors related to patients, physicians and the transplant per se. These factors can impede the consent process and subsequently result in a failure of proper engagement in and an understanding of the procedure with resultant adverse consequences influencing patients and even the patient-physician relationship. We contend that informed consent is a dynamic and ongoing process and that better patient education can assist in the decision making, fulfill the ethical principle of respect for autonomy and engage the patient to maximize compliance and adherence to therapy. This manuscript reviews the key literature pertaining to the decision-making and consent process in HCT and proposes guidelines for improving the consent process. Strategies for improving patient comprehension, engagement and enhancing consent forms are discussed.Bone Marrow Transplantation advance online publication, 22 September 2014; doi:10.1038/bmt.2014.207.
    Bone Marrow Transplantation 09/2014; 50(1). DOI:10.1038/bmt.2014.207 · 3.47 Impact Factor
  • Source
    [Show abstract] [Hide abstract]
    ABSTRACT: Corticosteroids are the accepted primary therapy for acute graft-versus-host disease (GvHD), but durable responses are seen in only about half the patients. BMT-CTN 0802, a phase III multi-center randomized double blinded trial, was designed to test whether mycophenolate mofetil (MMF) plus corticosteroids was superior to corticosteroids alone as initial therapy for acute GvHD. Patients with newly diagnosed acute GvHD were eligible if required systemic therapy. Patients were randomized to receive prednisone with either MMF or placebo. The primary endpoint was acute or chronic GvHD-free survival at day 56 after initiation of therapy. A futility rule for GvHD free survival at day 56 was met at a planned interim analysis after 235 eligible patients (out of 372) were enrolled: 116 to MMF, 119 to placebo. Baseline characteristics were well balanced between treatment groups including grade and organ distribution of GvHD. GvHD free survival at day 56, cumulative incidence of chronic GvHD at 12 months, overall survival, EBV reactivation, cumulative incidence of severe, life threatening infections, cumulative incidence of relapse at 12 months, quality of severe infections were similar. The addition of MMF to corticosteroids as initial therapy of acute GvHD does not improve GvHD-free survival compared with treatment with corticosteroids alone. The study was registered at NCT01002742.
    Blood 08/2014; DOI:10.1182/blood-2014-06-577023 · 9.78 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: Grade II-IV acute graft-vs.-host disease (GVHD) occurs in approximately 35% of matched, related donor (MRD) allogeneic hematopoietic cell transplantation (HCT) recipients. We sought to determine if the combination of tacrolimus and sirolimus (Tac/Sir) was more effective than tacrolimus and methotrexate (Tac/Mtx) in preventing acute GVHD and early mortality after allogeneic MRD HCT in a phase 3, multicenter trial. The primary endpoint of the trial was to compare 114 day grade II-IV acute GVHD-free survival using an intention-to-treat analysis of 304 randomized subjects. There was no difference in the probability of day 114 grade II-IV acute GVHD-free survival (67% vs. 62%, p=0.38). Grade II-IV GVHD was similar in the Tac/Sir and Tac/Mtx arms (26% vs. 34%, p=0.48). Neutrophil and platelet engraftment were more rapid in the Tac/Sir arm (14 vs. 16 days, p<0.001; 16 vs. 19 days, p=0.03). Oropharyngeal mucositis was less severe in Tac/Sir arm (peak OMAS score 0.70 vs. 0.96, p<0.001), but otherwise toxicity was similar. Chronic GVHD, relapse-free survival and overall survival at 2 years were no different between study arms (53% vs. 45%, p=0.06; 53% vs. 54%, p=0.77; 59% vs. 63%, p=0.36). Based on similar long-term outcomes, more rapid engraftment and less oropharyngeal mucositis, the combination of Tac/Sir is an acceptable alternative to Tac/Mtx after MRD HCT. (Funded by the National Heart, Lung, and Blood Institute and National Cancer Institute; number, NCT00406393).
    Blood 06/2014; 124(8). DOI:10.1182/blood-2014-04-567164 · 9.78 Impact Factor
  • Shernan G Holtan, Marcelo Pasquini, Daniel J Weisdorf
    [Show abstract] [Hide abstract]
    ABSTRACT: Over the past five years, many novel approaches to early diagnosis, prevention, and treatment of acute graft-versus-host disease (aGVHD) have been translated from the bench to the bedside. In this review, we highlight recent discoveries in the context of current aGVHD care. The most significant innovations that have already reached the clinic are prophylaxis strategies based upon a refinement of our understanding of key sensors, effectors, suppressors of the immune alloreactive response and the resultant tissue damage from the aGVHD inflammatory cascade. In the near future, aGVHD prevention and treatment will likely involve multiple modalities, including small molecules regulating immunologic checkpoints, enhancement of suppressor cytokines and cellular subsets, modulation of the microbiota, graft manipulation, and other donor-based prophylaxis strategies. Despite long-term efforts, major challenges in treatment of established aGVHD still remain. Resolution of inflammation and facilitation of rapid immune reconstitution in those with only a limited response to corticosteroids is a research arena that remains rife with opportunity and urgent clinical need.
    Blood 06/2014; DOI:10.1182/blood-2014-01-514786 · 9.78 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: Patient registries, frequently referred to as outcome registries, are 'organized systems' that use observational study methods to collect uniform data. Registries are used to evaluate specified outcomes for a population defined by a particular disease, condition or exposure that serves one or more predetermined scientific, clinical or policy purposes. Outcome registries were established very early in the development of hematopoietic SCT (HSCT). Currently, myriads of national and international HSCT registries collect information about HSCT activities and outcomes. These registries have contributed significantly to determining trends, patterns, treatment practices and outcomes. There are many different HSCT registries, each with different aims and goals; some are led by professional organizations, others by government authorities, health care providers or third parties. Some registries simply assess activity and others study outcomes. These registries are complementary and are gradually developing interoperability with each other to expand future collaborative research activities. A key development in the last few years was the incorporation of recommendations into the World Health Organization guiding principles on cell, tissue and organ transplantation. The data collection and analysis should be an integral part of therapy and an obligation rather than a choice for transplant programs. This article examines challenges in ensuring data quality and functions of outcome registries, using HSCT registries as an example. It applies to all HSCT-related data, but is predominantly focused on HSCT registries of professional organizations.Bone Marrow Transplantation advance online publication, 28 April 2014; doi:10.1038/bmt.2014.78.
    Bone marrow transplantation 04/2014; 49(8). DOI:10.1038/bmt.2014.78 · 3.47 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: Acute myeloid leukemia (AML) represents a heterogeneous group of high-grade myeloid neoplasms of the elderly with variable outcomes. Though remission-induction is an important first step in the management of AML, additional treatment strategies are essential to ensure long-term disease-free survival. Recent pivotal advances in understanding the genetics and molecular biology of AML have allowed for a risk-adapted approach in its management based on relapse-risk. Allogeneic hematopoietic cell transplantation (allo-HCT) represents an effective therapeutic strategy in AML providing the possibility of cure with potent graft-versus-leukemia reactions, with a demonstrable survival advantage in younger patients with intermediate- or poor-risk cytogenetics. Herein we review the published data regarding the role of allo-HCT in adults with AML. We searched MEDLINE/PubMed and EMBASE/Ovid. In addition, we searched reference lists of relevant articles, conference proceedings and ongoing trial databases. We discuss the role of allo-HCT in AML patients stratified by cytogenetic- and molecular-risk in first complete remission, as well as allo-HCT as an option in relapsed/refractory AML. Besides the conventional sibling and unrelated donor allografts, we review the available data and recent advances for alternative donor sources such as haploidentical grafts and umbilical cord blood. We also discuss conditioning regimens, including reduced intensity conditioning which has broadened the applicability of allo-HCT. Finally we explore recent advances and future possibilities and directions of allo-HCT in AML. Practical therapeutic recommendations have been made where possible based on available data and expert opinion.
    04/2014; 6(2):69-81. DOI:10.4252/wjsc.v6.i2.69

Publication Stats

1k Citations
464.76 Total Impact Points


  • 2007–2015
    • University of Wisconsin - Milwaukee
      Milwaukee, Wisconsin, United States
  • 2005–2015
    • Medical College of Wisconsin
      • • Division of Hematology/Oncology
      • • Center for International Blood & Marrow Transplant Research
      Milwaukee, Wisconsin, United States
  • 2013
    • Ottawa Hospital Research Institute
      Ottawa, Ontario, Canada
    • Nationwide Children's Hospital
      Columbus, Ohio, United States
  • 2009
    • Roswell Park Cancer Institute
      Buffalo, New York, United States