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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Cyclophosphamide is Effective GVHD Prophylaxis in Matched Related and Unrelated HCT
November 2021Ruggeri 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. -
Haploidentical HCT is viable alternative to matched sibling HCT in patients with AML in CR1
July 2019Rashidi A, et al., Blood Advances – This large-scale retrospective study compared outcomes of adult patients with acute myeloid leukemia (AML) in first complete remission (CR1) undergoing haploidentical hematopoietic cell transplantation (HCT) (n=336) versus 869 patients who underwent matched sibling donor (MSD) HCT. Results indicate that both cohorts had comparable overall survival, leukemia-free survival, non-relapse mortality, relapse, and grade II-IV acute GVHD. -
Evaluation of new GVHD prevention regimens shows promising results
April 2019Bolaños-Meade J, et al., The Lancet Haematology – In a prospective, phase II trial, researchers found that tacrolimus, mycophenolate mofetil, with post-hematopoietic cell transplant (HCT) cyclophosphamide (TMMCy) produced the best GVHD-free, relapse-free survival (GRFS) for patients who received an allogeneic HCT when compared to the current standard of care.
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Steroid-free treatment rivals prednisone as initial GVHD therapy
March 2019Pidala JA, et al., Bio Blood Marrow Transplant – Examining the feasibility of clinical- and biomarker-based risk-adapted therapy for acute graft-versus-host disease (GVHD), a recent phase II trial revealed similar complete response/partial response rates for sirolimus compared to current standard of care, prednisone.
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Continued vigilance needed to prevent infections for HCT recipients
January 2019A recent study found late fatal infections (LFI) contributed to one-third of the deaths of both adult and pediatric patients who had survived more than 2 years after allogeneic hematopoietic cell transplantation (HCT). The results highlight the importance of ongoing monitoring and preventive measures for infect
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Experts Highlight Latest Advances in Immunotherapies for Hematologic Diseases
July 2018Paczesny S, et al. Blood – This series in the journal Blood outlines both current obstacles and new opportunities for the future on a selection of emerging immunotherapies now available to patients with hematologic diseases. -
Ruxolitinib Can Prevent Acute GVHD in Patients with Myelofibrosis
July 2018Kröger N, et al. Biol Blood Marrow Transplant – Ruxolitinib is approved for treating myelofibrosis and also has shown efficacy in treating steroid-resistant acute and chronic graft-versus-host disease (GVHD). -
Spotlight on Novel Therapies to Improve Post-HCT Graft-Versus-Leukemia Effects
April 2018Zeiser R, et al. Blood – The latest research in cellular therapies, antibody-based treatments, and tyrosine kinase inhibitor (TKI)-based approaches to enhance GVL effects are outlined in articles published in a special edition of Blood. -
Lower Relapse and Improved Survival with Mild Acute GVHD after HCT for ALL
December 2017Yeshurun M, et al. ASH abstract oral presentation, December 2017 – A large-scale multicenter study of pediatric and adult patients with acute lymphoblastic leukemia (ALL) has confirmed that HCT imparts a potent graft-versus-leukemia effect in ALL with improved survival and lower relapse in patients who experience grade I-II acute GVHD. -
Ibrutinib Leads to Durable Responses in Steroid-Resistant Chronic GVHD
November 2017Miklos D, et al. Blood – Using the tyrosine kinase inhibitor ibrutinib to treat steroid-resistant chronic graft-versus-host disease (GVHD) can result in overall response rates of 67%, according to results of a multicenter, open-label phase 2 study of 42 transplant recipients. -
Review: Emerging Therapeutics to Treat Chronic GVHD
November 2017Betts BC, et al. Biol Blood Marrow Transplant – Preclinical and clinical studies have significantly advanced the understanding of the pathophysiology of chronic graft-versus-host disease (GVHD), resulting in new immunomodulatory agents targeting innate immunity, early T-cell activation and signaling, T-cell migration, regulatory T cells, and B cell dysregulation. -
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. -
Immunoglobulin Significantly Reduces Chronic GVHD Rates
July 2017Finke J, et al. Lancet Haematol – Anti-human-T-lymphocyte immunoglobulin (ATLG) significantly lowers chronic graft-versus-host disease (GVHD) rates and reduces risks associated with long-term immune suppression, according to a long-term randomized study of 201 patients undergoing hematopoietic cell transplantation (HCT). -
Depression Prior to HCT is Prognostic of Lower Survival, Higher Acute GVHD
March 2017El-Jawahri A, et al. Cancer – Pre-transplant depression requiring treatment significantly affected post-allogenic hematopoietic cell transplant (HCT) outcomes, including lower overall survival (OS) and higher risk of acute graft-versus-host disease (GVHD). -
Review: New Targeted Therapies to Treat or Prevent Chronic GVHD
February 2017Cutler CS, et al. Blood – In this review, the authors summarize the current preclinical studies and early-phase clinical trial results on the following new chronic graft-versus-host disease (GVHD) therapeutic techniques. Many of these new approaches target specific immunologic pathways now known to play a role in chronic GVHD. -
New NIH Report: Recommended Follow-Up for Long-Term Survivors after HCT
December 2016Battiwalla M, et al. Biol Blood Marrow Transplant – This report outlines the recommendations issued by the National Institutes of Health (NIH) Late Effects Consensus Conference to plan the next phase of hematopoietic cell transplantation (HCT) research “to ensure that no survivor is left behind.” -
Adding Sirolimus to Standard GVHD Prophylaxis Significantly Reduces Acute GVHD
December 2016Sandmaier B, et al. ASH abstract oral presentation, December 2016 – Adding sirolimus to a standard graft-versus-host disease (GVHD) prophylaxis of mycophenolate mofetil (MMF) and cyclosporine (CSP) can significantly reduce the risks of grades II-IV and III-IV acute GVHD and non-relapse mortality (NRM), according to results of a phase 3 multi-center randomized trial presented at ASH. -
Biomarker Panel Can Predict Risk of Chronic GVHD
August 2016Yu J, et al. J Clin Oncol – Researchers analyzing plasma samples of 53 HCT recipients identified 4 proteins that when used in a biomarker panel was prognostic of developing chronic graft-versus-host disease (cGVHD). -
A Biomarker-Guided Prophylaxis Strategy Reduces Acute GVHD
May 2016Chang Y-J, et al. J Clin Oncol – A randomized trial of 228 patients undergoing haploidentical HCT has demonstrated that low-dose corticosteroid prophylaxis administered to patients at high risk of developing acute graft-versus-host disease (GVHD) can significantly decrease the incidence of the disease. -
Reduced Risk of Chronic GVHD with Post-Transplant Cyclophosphamide
April 2016Mielcarek M, et al. Blood – High-dose cyclophosphamide administered after peripheral blood stem cell (PBSC) transplantation can reduce the risk of chronic graft-versus-host disease (GVHD), according to a single-center study of 43 patients. -
Haploidentical, Unrelated Donor HCT Outcomes Comparable in Lymphoma
March 2016Kanate AS, et al. Blood – Reduced-intensity HCT using haploidentical or matched unrelated donors (URD) yields comparable outcomes, according to a study of 917 adults transplanted for lymphoma. -
Higher Dose of Invariant Natural Killer T Cells Improves HCT Outcomes
March 2016Malard F, et al. Blood – Allogeneic HCT using peripheral blood stem cell (PBSC) grafts have better outcomes when the grafts contain large doses of invariant natural killer T (iNKT) cells, according to a single-center study of 80 consecutive patients transplanted between 2010 and 2013. -
Pre-HCT Depression Leads to Lower Survival, Higher Risk of Acute GVHD
February 2016El-Jawahri A, et al. BMT Tandem Meetings abstract oral presentation, February 2016 – Results of a large-scale study comparing adults with and without diagnosed depression prior to hematopoietic cell transplantation (HCT) found that those with pre-HCT depression had significantly lower survival and a higher incidence of grade II-IV acute GVHD. -
Improved Outcomes in HCT Recipients with Acute GVHD Over Time
January 2016El-Jawahri A, et al. Biol Blood Marrow Transplant – A retrospective analysis of 427 hematopoietic cell transplantation (HCT) recipients who develop grades III or IV acute GVHD has found that outcomes have improved over time, with significantly lower transplant-related mortality (TRM) and increased overall survival (OS) in more recent transplants. -
Comparable Outcomes in Unrelated Donor vs. Haploidentical HCT with Cyclophosphamide
December 2015Mussetti A, et al. ASH abstract oral presentation, December 2015 – Lower-intensity hematopoietic cell transplantation (HCT) using haploidentical donors and post-transplant cyclophosphamide for GVHD prophylaxis can achieve outcomes comparable to HLA-matched unrelated donor transplants, according to results of a retrospective study of 917 adults with Hodgkin or Non-Hodgkin lymphoma. -
Quality of Life Better After Marrow Transplant Than After PBSC Transplant
December 2015Lee SJ, et al. ASH oral presentation, December 2015 – A secondary analysis of the large-scale (n=551) Blood and Marrow Transplant Clinical Trials Network (BMT CTN) randomized study of unrelated donor bone marrow (BM) vs. peripheral blood stem cell (PBSC) transplant has found that BM recipients have better psychological well-being and less burdensome chronic GVHD symptoms than PBSC recipients. -
Non-Myeloablative Haploidentical HCT in Older Patients
September 2015Kasamon YL, et al. J Clin Oncol – This retrospective, single-center study found that age had no significant effect on survival of 271 patients age 50-75 years undergoing non-myeloablative haploidentical hematopoietic cell transplantation (HCT). -
Ruxolitinib Effective in Treating Steroid-Refractory GVHD
September 2015Zeiser R, et al. Leukemia – Ruxolitinib therapy to treat corticosteroid-refractory acute and chronic graft-versus-host disease (GVHD) in transplant recipients can achieve response rates >80%, according to a multi-center study of 95 patients treated with this JAK1/2 inhibitor. -
A Refined Acute GVHD Risk Score Can Predict HCT Outcomes
April 2015MacMillan ML, et al. Biol Blood Marrow Transplant – Researchers examining the clinical stage and grade of acute graft-versus-host disease (GVHD) in 1,723 patients at the onset of treatment with systemic steroids have developed a new GVHD risk score that predicts response to initial therapy, survival, and transplant-related mortality. -
Outcomes of Extracorporeal Photopheresis Better in Early Stage GVHD
April 2015Berger M, et al. Transfusion — A study of extracorporeal photopheresis (ECP) in 71 patients has shown that the procedure is effective in treating steroid-refractory or -dependent acute GVHD or chronic GVHD with lower severity in the early stages. -
Validated Chronic GVHD Risk Score Can Predict Major HCT Outcomes
March 2015Arora M, et al. Biol Blood Marrow Transplant — A chronic graft-versus-host disease (GVHD) risk score, used to predict mortality in patients with GVHD, has now been validated by additional analysis of 1,128 patients with chronic GVHD transplanted between 2005 and 2007. -
Review: Post-Transplant High-Dose Cyclophosphamide to Prevent GVHD
March 2015Al-Homsi AS, et al. Bone Marrow Transplant — This review article focuses on the mechanism of action of post-transplant cyclophosphamide, with a discussion of the clinical data on its efficacy in the prevention of GVHD. -
Biomarkers Predict Severity of GVHD after HCT
January 2015Levine JE, et al. Lancet Haematol – A prognostic score based on the blood plasma concentration of three biomarkers can guide risk-adapted therapy at the onset of graft-versus-host disease (GVHD) after allogeneic hematopoietic cell transplantation (HCT). -
Review: Collaborative Partnerships to Manage Patients with Chronic GVHD
January 2015Flowers MED, et al. Blood — In this review titled “How we treat chronic graft-versus-host disease” the authors outline their strategies for treating chronic GVHD, emphasizing that transplant center physicians and the hematology and oncology specialists who offer ongoing supportive care need to be collaborative partners to best manage their patients who develop chronic GVHD. -
Post-HCT Cyclophosphamide is Effective as Sole GVHD Prophylaxis
December 2014Kanakry CG, et al. Blood — High-dose, post-transplant cyclophosphamide can successfully reduce severe graft-versus-host disease (GVHD) after T-cell-replete allogeneic transplantation for AML, ALL, and MDS, using related and unrelated donors. -
Review: Next Generation Treatment of Acute GVHD
December 2014Magenau J, et al. Leukemia — This review describes the recent advances in the understanding of the underlying molecular basis of acute graft-versus-host disease (GVHD). -
Post-Transplant Cyclophosphamide Yields Comparable Outcomes Across Donor Types and Conditioning Regimens
December 2014McCurdy SR, et al. ASH abstract oral presentation, December 2014 — Adults transplanted for advanced or poor-risk hematologic malignancies can have comparable outcomes using haploidentical, related donor, or unrelated donor grafts when high-dose post-transplant cyclophosphamide is administered to reduce graft-versus-host-disease (GVHD), according to study results presented at ASH. -
MMF Plus Corticosteroids Does Not Improve Acute GVHD Treatment Over Corticosteroids Alone
November 2014Bolaños-Meade J, et al. Blood – This phase 3 multi-center randomized double-blinded trial examined whether mycophenolate mofetil (MMF) plus corticosteroids was superior to corticosteroids alone as initial therapy for acute GVHD. -
Review Series in Blood Journal: Advances in HCT
August 2014Negrin RS, et al. Blood — Scientific and clinical translational research highlighting treatment advances in the field of HCT are available in five review articles in Blood. -
Tacrolimus-Sirolimus Comparable to Tacrolimus-Methotrexate as GVHD Prophylaxis
August 2014Cutler C, et al. Blood – A phase 3 Blood and Marrow Transplant Clinical Trials Network (BMT CTN) study of 304 randomized patients demonstrated that tacrolimus and sirolimus (Tac/Sir) was comparable to tacrolimus and methotrexate (Tac/Mtx) in preventing acute GVHD and early mortality after related donor hematopoietic cell transplantation (HCT). -
Review: Novel Approaches to Treat Acute GVHD
June 2014Holtan SG, et al. Blood — This review highlights novel approaches to early diagnosis, prevention, and treatment of acute graft-versus-host disease, focusing on those now available in the clinical care setting. -
Pre-Transplant Comorbidities Predict Severity of Acute GVHD
May 2014Sorror ML, et al. Blood — A multi-center study of 2,985 allogeneic transplants has demonstrated that the hematopoietic cell transplantation comorbidity index (HCT-CI) can predict the severity of graft-versus-host disease (GVHD) and subsequent mortality. -
Extracorporeal Photopheresis Induces Responses in Chronic GVHD
March 2014Dignan FL, et al. Bone Marrow Transplant — Extracorporeal photopheresis (ECP) to treat chronic GVHD induced complete or partial responses in 50% of transplant recipients in an intention-to-treat-analysis of 38 patients with moderate to severe GVHD. -
Imatinib to Treat Steroid-Refractory Chronic GVHD
December 2013Olivieri A, et al. Blood - In this prospective trial, researchers administered imatinib to treat steroid-refractory chronic GVHD in 39 adult transplant recipients aged 28-73 years. -
Review: Strategies to Improve Outcomes of Unrelated Donor PBSC Transplants
December 2013Sohn SK, et al. Transfusion - The authors of this review note that because of the higher incidence of graft-versus-host disease (GVHD) in peripheral blood stem cell (PBSC) transplant compared to marrow transplant, strategies need to be different for the two stem cell grafts. -
Inhibition of IL-6 Can Offer “Profound Protection” from Acute GVHD
December 2013Kennedy GA, et al. ASH abstract oral presentation, December 2013 – Grade II-IV and grade III-IV acute graft-versus-host disease (GVHD) is significantly reduced in transplant recipients receiving a monoclonal antibody inhibiting interleukin-6 (IL-6), according to research results presented at ASH. -
Personalized Prognostic Information for Adult Leukemia Survivors after HCT
November 2013Lee SJ, et al. Biol Blood Marrow Transplant - By analyzing outcomes data of 3,339 adults with AML and 1,434 adults with ALL submitted to the CIBMTR, researchers have developed formulas to estimate future leukemia-free survival (LFS) of transplant recipients surviving one year or more post-transplant. -
Post-HCT Rituximab Prophylaxis Can Reduce Chronic GVHD
September 2013Cutler C, et al. Blood. - In this trial of 65 patients, administering post-transplant rituximab (375 mg/m2 IV) at 3, 6, 9, and 12 months after transplantation resulted in significantly lower two-year cumulative incidence of systemic corticosteroid-requiring chronic graft-versus-host disease (GVHD) compared to a concurrent control group: 31% vs. 48.5% respectively (p=0.015).
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Report: Prophylaxis and Treatment of GVHD
August 2013Ruutu T, et al. Bone Marrow Transplant - A working group formed by the European Group for Blood and Marrow Transplantation (EBMT) and European LeukemiaNet has developed recommendations for prophylaxis and treatment of acute and chronic GVHD.
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Lower Chronic GVHD in Double Cord Blood HCT
August 2013Malard F, et al. Leukemia - Double cord blood and 9/10 matched unrelated donor reduced-intensity transplants have similar outcomes, but using cord blood results in a significantly lower incidence of chronic graft-versus-host disease (GVHD), according to a study of 152 patients (median age 53 years) transplanted for hematological malignancies. -
Outcomes for HCT in Patients Over Age 70
July 2013Brunner AM, et al. Biol Blood Marrow Transplant - Select older patients can undergo HCT, according to results of a study of 54 consecutive patients >70 years undergoing reduced-intensity transplantation between 2007 and 2012 at Massachusetts General Hospital (n=14) or the Dana-Farber Cancer Institute (n=40). -
Review: Cord Blood Transplant Supported by Third-Party Donor Cells
July 2013Van Besien K, et al. Biol Blood Marrow Transplant - This article reviews umbilical cord blood transplantation (CBT) supported by co-infusion of adult CD34+ stem cells from mismatched unrelated donors.
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Emerging Therapies for Chronic Cutaneous GVHD
April 2013Rogers CJ, et al. Bone Marrow Transplant - A review article discussing current and emerging therapies for chronic cutaneous GVHD, including extracorporeal photopheresis, and second-line drugs targeting T cells, B cells, and inflammatory cytokines.
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Graft-Versus-Tumor Effects in Advanced Malignancies
March 2013Storb R, et al. J Clin Oncol - A study of a minimal-intensity pre-transplant conditioning regimen consisting of low-dose total body irradiation with or without fludarabine has shown that it can induce strong graft-versus-tumor (GVT) effects leading to lasting remissions in 45% to 75% of patients.
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Review: The Role of Sirolimus in GVHD Management
January 2013Abouelnasr A, et al. Biol Blood Marrow Transplant - This review summarizes the mechanisms of action of sirolimus in preventing and treating graft-versus-host disease (GVHD). -
Marrow, PBSC Have Comparable Survival; More cGVHD with PBSC
November 2012Anasetti C, et al. N Eng J Med - A phase III, multi-center, randomized trial comparing peripheral blood stem cells (PBSC) and bone marrow transplant outcomes has found comparable 2-year survival.
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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.