About Our Research

Because your patients count on you to apply the best therapies that research has identified, we never let up in our efforts to improve the science of hematopoietic cell transplantation (HCT). Our observational and prospective research has led to significant improvements in survival and quality of life for people with blood cancer and other diseases.

We conduct research through our research program, the Center for International Blood and Marrow Transplant Research® (CIBMTR®), in collaboration with the Medical College of Wisconsin (MCW). This global collaborative effort has resulted in more than 1,200 peer-reviewed publications — including more than 80 in 2017 alone — and more than 220 active studies, driving the quest to save more lives.

Research Activities

At the heart of our research efforts is the outcomes registry that contains information on more than 475,000 HCT recipients, made possible because of the collaboration of more than 420 centers in the United States and worldwide contributing data. Our charge is to collect data on all allogeneic transplants and most autologous transplants in the United States, plus significant data from outside the U.S.

In addition, we lead health outcomes research and worldwide efforts for immunobiology-focused research, through our Research Sample Repository, which contains more than 2.6 million sample aliquots from related and unrelated transplant donors, cord blood units, and recipients. The CIBMTR is a respected leader in HCT research, collaborating with investigators to provide data, statistical support, and expert analysis to advance the HCT field.

We apply this research to:

  • Study social and economic barriers to care and the influence of psychosocial factors on outcomes
  • Publish guidelines to develop effective strategies for selecting the best available donor or cord blood unit for a patient
  • Increase availability of unrelated donors by understanding donor and recipient genetic determinants
  • Contribute to the immunobiological basis of HapLogicTM, our advanced HLA matching algorithm
  • Improve HCT outcomes through a better understanding of immune response pathways

Prospective research is conducted through the CIBMTR's RCI BMT (Resource for Clinical Investigation in Blood and Marrow Transplantation) and BMT CTN (Blood and Marrow Transplant Clinical Trials Network), which is funded by the National Institutes of Health. RCI BMT generates data, so that innovative ideas can progress to Phase II or Phase III trials. BMT CTN has opened more than 46 multi-institutional Phase II and III trials, involving more than 100 transplant centers, and more than 9,900 patients.

Access research resources and activities here and through CIBMTR.org:

Impact of Our Research

Significant progress has been made in the HCT field through collaboration among transplant clinicians, researchers, and biostatisticians. These practice-changing findings have contributed to substantial advances in survival and quality of life for hundreds of thousands of transplant recipients around the world.

Research Highlights:

  • High-Resolution Donor-Recipient HLA Matching Improves Outcomes
    Clinical Relevance: Expeditious transplantation with the best available donor, even if mismatched, may offer the best chance for survival. [1]
  • Unrelated Cord Blood Transplantation in Adults with Acute Leukemia
    Clinical relevance: These data support the use of UCB for adults with acute leukemia when there is no HLA-matched unrelated adult donor available, and when a transplant is needed urgently. [2]
  • Effect of T-Cell-Epitope Matching at HLA-DPB1 in Unrelated-Donor HCT
    Clinical relevance: To lower the risk of non-relapse mortality after unrelated-donor HCT, a non-permissive T-cell epitope mis-match at HLA-DPB1 should be avoided if possible. [3]
  • Reduced-Intensity HCT Better Than Chemotherapy in Older Patients
    Clinical relevance: Reduced-intensity conditioning allogeneic HCT in patients age 60-70 with AML in CR1 reduces relapse and improves leukemia-free survival compared to chemotherapy. [4]
  • Effect of Race and Gender on Access to HCT
    Clinical relevance: While waiting for further research to better understand disparate access to transplantation, the medical community should work at all levels to eliminate these disparities. [5]
  • Reduced-Intensity HCT for AML/MDS Not Affected by Age
    Clinical relevance: Older age along should not be considered a contraindication to hematopoietic cell transplantation. [6]
  • Solid Cancers After Allogeneic HCT
    Clinical relevance: Allogeneic transplant survivors, particularly those irradiated at young ages, face increased risks of solid cancers and should receive lifelong surveillance. [7]
  • Secondary Solid Cancers After Allogeneic HCT Using Bu/Cy Conditioning
    Clinical relevance: Recipients of allogeneic HCT using Bu-CY conditioning are at risk for developing solid cancers, a risk that increases with time, and so lifelong cancer surveillance is warranted in this population. [8]
  • Comparable Outcomes After Matched Related and Unrelated HCT
    Clinical relevance: Transplantation from URD and MRD donors produces similar survival for patients with AML. [9]
  • HCT Outcomes Comparable: Umbilical Cord Blood vs. Bone Marrow in Pediatric Acute Leukemia
    Clinical relevance: These data support the use of HLA-matched and one- or two-antigen HLA-mismatched umbilical cord blood in children with acute leukemia who need transplantation. [10]
  • HLA-C Matching Improves Outcomes in Cord Blood Transplantation
    Clinical relevance: To minimize mortality risk, matching at HLA-C should be sought for units that are matched at HLA-A, -B, or -DRB1 and for units with a single locus mismatch at HLA-A, -B, or -DRB1. [11]
  • Recommended Screening and Preventive Practices for Long-Term HCT Survivors
    Clinical relevance: Because of the HCT recipient's risk of developing long-term complications due to pre-, peri-, and post-transplant exposures, these guidelines offer a systematic follow-up plan for caring for HCT recipients. [12]
  • Bone Marrow vs. Peripheral Blood Stem Cell Randomized Study
    Clinical relevance: No differences in patient survival rates between patients receiving transplantation with bone marrow or PBSC from unrelated donors, although GVHD developed more often and was more severe in patients who received PBSC compared with those receiving marrow. [13]
  • Adult Donor (Marrow and PBSC) and Cord Blood Matching Guidelines
    Clinical relevance: Those making decisions for selection of adult donor (marrow and PBSC) and cord blood units can apply matching guidelines based on clinical research into the effect of HLA matching on transplant outcomes. [14]
  • Cost and Cost-Effectiveness of HCT
    Clinical relevance: A review of contemporary literature on the costs and cost-effectiveness of HCT in the United States and worldwide. This consolidation of available data identifies when HCT is a cost-effective option and where further study is needed. [15]
  • Six-Locus, High-Resolution HLA Haplotype Frequencies of the Be The Match Registry
    Clinical relevance: By studying six-locus, high-resolution HLA A∼C∼B∼DRB3/4/5∼DRB1∼DQB1 haplotype frequencies of more than 6.5 million Be The Match® Registry volunteer donors typed by DNA methods at recruitment, haplotype frequencies can be used to improve match predictions for donor selection algorithms. [16]

Research Grants

We offer grants to support research that advances the medical science of hematopoietic cell transplantation:

Ensuring Research Integrity

Our research is conducted and reviewed with the highest integrity, ensuring there is no actual or apparent financial conflict of interest that could influence the conduct or review of the research. View our Conflict of Interest Policy (PDF) that describes the requirements of researchers participating in studies with us.


  1. Lee SJ, Klein J, Haagenson M, et al. High-resolution donor-recipient HLA matching contributes to the success of unrelated donor marrow transplantation. Blood. 2007; 110(13): 4576-4583. Access
  2. Eapen M, Rocha V, Sanz G, et al. Effect of graft source on unrelated donor haematopoietic stem-cell transplantation in adults with acute leukaemia: A retrospective analysis. Lancet Oncol. 2010; 11(7): 653-660. Access
  3. Fleischhauer K, Shaw BE, Gooley T, et al. Effect of T-cell-epitope matching at HLA-DPB1 in recipients of unrelated-donor haemopoietic-cell transplantation: A retrospective study. Lancet Oncol. 2012; 13(4): 366-374. Access
  4. Farag SS, Maharry K, Zhang MJ, et al. Comparison of reduced-intensity hematopoietic cell transplantation with chemotherapy in patients age 60-70 years with acute myelogenous leukemia in first remission. Biol Blood Marrow Transplant. 2011; 17(12): 1796-1803. Access
  5. Joshua TV, Rizzo JD, Zhang MJ, et al. Access to hematopoietic stem cell transplantation: Effect of race and sex. Cancer. 2010; 116(14): 3469-3476. Access
  6. McClune BL, Weisdorf DJ, Pedersen TL, et al. Effect of age on outcome of reduced-intensity hematopoietic cell transplantation for older patients with acute myeloid leukemia in first complete remission or with Myelodysplastic syndrome. J Clin Oncol. 2010; 28(11): 1878-1887. Access
  7. Rizzo JD, Curtis RE, Socié G, et al. Solid cancers after allogeneic hematopoietic cell transplantation. Blood. 2009; 113(5): 1175-1183. Access
  8. Majhail NS, Brazauskas R, Rizzo JD, et al. Secondary solid cancers after allogeneic hematopoietic cell transplantation using busulfan cyclophosphamide conditioning. Blood. 2011; 117(1): 316-322. Access
  9. Saber W, Opie S, Rizzo JD, et al. Outcomes after matched unrelated donor vs. identical sibling hematopoietic cell transplantation (HCT) in adults with acute myelogenous leukemia (AML). Blood. 2012; 119(17): 3908-3916. Access
  10. Eapen M, Rubinstein P, Zhang MJ, et al. Outcomes of transplantation of unrelated umbilical cord blood and bone marrow in children with acute leukemia: A comparison study. Lancet. 2007; 369(9577): 1947-1954. Access
  11. Eapen M, Klein JP, Sanz GF, et al. Effect of donor-recipient HLA matching at HLA A, B, C, and DRB1 on outcomes after umbilical-cord blood transplantation for leukaemia and myelodysplastic syndrome: a retrospective analysis. Lancet Oncol. 2011; 12(13): 1214-1221. Access
  12. Majhail NS, Rizzo JD, Lee SJ, et al. Recommended screening and preventive practices for long-term survivors after hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2012; 18(3): 348-371. Access
  13. Anasetti C, Logan BR, Lee SJ, et al. Peripheral-blood stem cells versus bone marrow from unrelated donors. N Engl J Med. 2012; 367(16): 1487-1496. Access
  14. Spellman SR, Eapen M, Logan BR, et al. A perspective on the selection of unrelated donors and cord blood units for transplantation. Blood. 2012; 120(2): 259-265. Access
  15. Preussler JM, Denzen EM, Majhail NS. Costs and cost-effectiveness of hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2012; 18(11): 1620-1628. Access
  16. Gragert L, Madbouly A, Freeman J, Maiers M. Six-locus high resolution HLA haplotype frequencies derived from mixed-resolution DNA typing for the entire US donor registry. Hum Immunol. 2013; 74(10): 1313-1320. Access