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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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. -
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. -
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. -
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). -
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. -
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). -
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.