Recent Research

Stay up-to-date on the advancing field of HCT with short summaries and links to the most relevant published research.

Hourigan CS, et al.  – Research presented at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting explores a standardized approach to measurable residual disease (MRD) testing in patients with acute myeloid leukemia (AML) undergoing hematopoietic stem cell transplant (HCT). Results support MRD as a useful prognostic tool with the potential to identify relapse risk and provide opportunities to individualize treatment for optimal outcomes.
Chowdhury AS, et al. – Research presented at the 2022 Tandem Meetings of the ASTCT and CIBMTR shows that expanding donor registry models to include mismatched unrelated donors (MMUDs) down to a 5/8 HLA match expands access to nearly 100% of patients in need of HCT. Based on alternative graft sources and advances in graft-versus-host disease prophylaxis following allogeneic HCT, MMUD has the potential to expand HCT access, particularly among racially and ethnically diverse patients.
Horowitz MM, et al. – Research presented at the 2022 Tandem Meetings of the ASTCT and CIBMTR shows that racially and ethnically diverse patients considering hematopoietic stem cell transplantation (HCT) are underrepresented in clinical trials conducted by the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). Data suggest major barriers not only for enrollment in clinical trials, but for ensuring underrepresented groups are included in the overall pipeline for HCT consideration.
Fingerson S, et al. – Presented at the 2022 Tandem Meetings of the ASTCT and CIBMTR, researchers evaluated a new Search Summary Score tool to provide rapid assessment of donor search productivity, showcasing its ability to streamline the search process for HCT. This tool allows clinical teams to focus their search strategies on the most likely available donor sources, particularly for racially and ethnically diverse patients and others less likely to have an HLA fully matched donor.
Stefanski H, et al. – A report presented at the 2022 Tandem Meetings of the ASTCT and CIBMTR from the NMDP/Be The Match shows that despite the challenges of the COVID-19 pandemic, HCT products can be reliably sourced and preserved without negative impacts to donors or patients. Cryopreserved products and products from COVID-19 positive donors appear to pose no overt risk.
Riches ML, et al. – Early data in an ongoing study presented at the 2022 Tandem Meetings of the ASTCT and CIBMTR show COVID-19 vaccination is less effective in those who have undergone cellular transplants and therapies in the last year, with more data needed to determine the ideal timing and number of boosters needed for this population. Vaccination continues to be important following transplant due to higher risk of COVID-19 complications in these vulnerable patients.
Fuchs EJ, et al.  – An observational study conducted by the CIBMTR published in Blood evaluated the impact of HLA-locus specific matching in haploidentical HCT using post-transplant cyclophosphamide (PTCy). The study results were used to create a tool to help with optimal haploidentical HCT donor selection based on predicted disease-free survival.
Wren J, et al. – Research presented at the 2022 Tandem Meetings of the ASTCT and CIBMTR shows the effectiveness of the NMDP/Be The Match BioBank, collaborating with multiple transplant centers and cell therapy labs to ensure access to hematopoietic cell transplantation (HCT) during the COVID-19 pandemic through cryopreservation.
Shaw BE, et al.  – Research presented at the European Group for Blood and Marrow Transplantation (EBMT) Annual Meeting shows outcomes remain very good at 3-years for patients receiving a mismatched unrelated donor hematopoietic stem cell transplant (MMUD HCT) with post-transplant cyclophosphamide (PTCy). Patients receiving reduced-intensity conditioning (RIC) in particular have excellent survival and very low rates of chronic graft-versus-host disease (GVHD).

Qayed M, et al.  – Research published in Blood Advances shows reductions in acute graft-versus-host disease (GVHD) in both matched unrelated and mismatched unrelated donor hematopoietic stem cell transplantation (MUD and MMUD HCT) when abatacept is added to standard of care treatment. Effects were substantial in MMUD, revealing abatacept as another promising tool to expand HCT access to those without a fully matched donor, which is more common for racially and ethnically diverse patients. 

Nakamura R, et al. and Martens M, et al. – Outcomes of allogeneic hematopoietic cell transplantation (alloHCT) among older patients with advanced myelodysplastic syndrome (MDS) are similar to younger patients and result in no worse quality of life (QOL). Researchers shared these findings at the 62nd and 63rd American Society of Hematology (ASH) Annual Meeting and Exposition.
Zhang T, et al. – Two research studies presented at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition called attention to the National Marrow Donor Program®/Be The Match® precision medicine initiative by presenting novel prognostic signatures in allogeneic hematopoietic cell transplantation (HCT) patients with myelodysplastic syndrome (MDS) based on whole genome sequencing and examining the impact of genomic subgroups on survival.
Devine S, et al. – Planned cryopreservation of allogeneic hematopoietic cell transplantation (HCT) grafts during the COVID-19 pandemic did not adversely impact early post-HCT outcomes according to research presented at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition.
Kean LS, et al. – Patients treated with abatacept in combination with the current standard of care for acute graft-versus-host disease prophylaxis after mismatched unrelated donor (7/8 MMUD) allogeneic hematopoietic stem cell transplantation (HCT) had significantly better survival outcomes according to research presented at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition.
Mussetti A, et al. – More apples-to-apples research evidence that matched unrelated donor (MUD) hematopoietic cell transplant (HCT) results in superior patient outcomes than haploidentical related donor HCT when available was presented at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition.
Khera N, et al. – Research presented at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition shows that rates of autologous and allogeneic hematopoietic cell transplants (HCT) have increased more rapidly among African American, Hispanic, and other underserved populations than among non-Hispanic whites from 2009 to 2018. However, a challenge still exists to close the gaps in overall survival for African American and other pediatric patients.
Gooptu M, et al., Blood—An observational study from the CIBMTR Graft Sources Working Committee found that 8/8 matched unrelated donor hematopoietic cell transplantation (HCT) with PTCy based graft versus host disease (GVHD) prophylaxis resulted in superior overall survival and disease-free survival compared to haploidentical HCT with PTCy in the reduced intensity conditioning (RIC) setting for treatment of acute leukemia and myelodysplastic syndromes. It is the first known study that offers a direct comparison between the two donor sources using PTCy.

Use of MMUDs can expand HCT access for ethnically diverse populations.

John M. Pagel, MD, PhD, Megan Othus, PhD, et al – Barriers to human leukocyte antigen (HLA) typing and cytogenetic testing can significantly impact the overall survival of patients newly diagnosed with acute myeloid leukemia (AML).  In this landmark study, a concerted effort to address those issues and streamline the initiation of donor identification and referral for hematopoietic cell transplant (HCT) consultation in early disease state led to a higher CR1 transplantation rate and improved 2-year overall survival.

Hsu J, et. al., Blood—Researchers presented data at the 62nd ASH Annual Meeting and Exposition on the impact of using fresh versus cryopreserved allogeneic bone marrow (BM) and peripheral blood stem cells (PBSC) grafts on patient allogeneic hematopoietic cell transplant (alloHCT) outcomes.

Auletta J, et al., Transplantation and Cellular Therapy—A manuscript published in Transplantation and Cellular Therapy offered the first report on the impact of the COVID-19 pandemic on the ability of the National Marrow Donor Program® (NMDP)/Be The Match® and the Network to safely deliver unrelated donor products early in the pandemic. The authors found that the NMDP/Be The Match and Network partners continued to effectively deliver domestic and unrelated donor products to allogeneic transplant patients in similar or shorter timeframes than before COVID-19, despite the many unexpected challenges brought about by the pandemic.

Nakamura, R et al., ASH oral abstract presentation, December 2020—Patients ages 65 and older with MDS who received an allogeneic hematopoietic cell transplantation (alloHCT) saw a similar benefit to overall survival (OS) as younger patients (50-64) when compared to other treatments.

Maakaron J, et al., ASH abstract poster presentation, December 2020—The CIBMTR® (Center for International Blood and Marrow Transplant Research®) presented their study results suggesting that efforts should focus on minimizing residual disease and better donor selection as age alone is not a barrier to successful hematopoietic cell transplantation (HCT) for acute myeloid leukemia (AML) in CR1 and should not exclude patients from HCT.

Dehn J, et al., Blood – A panel of immunogeneticists and other experts in the field of hematopoietic cell transplantation (HCT) has published updated evidence-based guidelines for the optimal selection of unrelated donors and cord blood units used for transplants.

The updated guidelines make recommendations for both HLA and non-HLA factors to consider in graft selection, including donor availability, donor age, patient sensitivity to HLA antigens, natural killer (NK) cell alloreactivity, and minimum total nucleated cell doses and CD34+ cell doses for cord blood units.


Shimoni A, et al., Biol Blood Marrow Transplant – In an analysis of 1134 patients age 50 or older with acute myeloid leukemia (AML), two-year hematopoietic cell transplant (HCT) survivors experienced comparable 10-year leukemia-free survival (LFS) and overall survival (OS) with matched sibling or unre-lated donors.


Cappelli B, et al. Haematologica – The youngest of three age-based patient cohorts (age 0-5 years) in a study of 736 patients with sickle cell disease (SCD) undergoing HLA-identical sibling hematopoietic cell transplantation (HCT) experienced 4-year event-free survival of 93%, results the researchers characterized as “excellent.” 

Bolaños-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.

Zeiser 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.

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