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Observational Study
. 2023 Jun;29(6):346.e1-346.e10.
doi: 10.1016/j.jtct.2023.03.007. Epub 2023 Mar 15.

Real-World Data Showing Trends and Outcomes by Race and Ethnicity in Allogeneic Hematopoietic Cell Transplantation: A Report from the Center for International Blood and Marrow Transplant Research

Jeffery J Auletta  1 , Jianqun Kou  2 , Min Chen  2 , Yung-Tsi Bolon  3 , Larisa Broglie  4 , Caitrin Bupp  3 , Debra Christianson  3 , Rachel N Cusatis  2 , Steven M Devine  3 , Mary Eapen  2 , Mehdi Hamadani  5 , Mary Hengen  3 , Stephanie J Lee  6 , Amy Moskop  4 , Kristin M Page  4 , Marcelo C Pasquini  2 , Waleska S Perez  2 , Rachel Phelan  4 , Marcie L Riches  2 , J Douglas Rizzo  2 , Wael Saber  2 , Stephen R Spellman  3 , Heather E Stefanski  3 , Patricia Steinert  2 , Eileen Tuschl  2 , Rafeek Yusuf  3 , Mei-Jie Zhang  7 , Bronwen E Shaw  2
Affiliations
Observational Study

Real-World Data Showing Trends and Outcomes by Race and Ethnicity in Allogeneic Hematopoietic Cell Transplantation: A Report from the Center for International Blood and Marrow Transplant Research

Jeffery J Auletta et al. Transplant Cell Ther. 2023 Jun.

Abstract

The use of HLA-mismatched donors could enable more patients with ethnically diverse backgrounds to receive allogeneic hematopoietic cell transplantation (HCT) in the United States. However, real-world trends and outcomes following mismatched donor HCT for diverse patients remain largely undefined. We conducted this study to determine whether the use of mismatched donor platforms have increased the access to allogeneic HCT for ethnically diverse patients, particularly through the application of novel graft-versus-host disease (GVHD) prophylaxis regimens, and whether outcomes for diverse patients are comparable to those of non-Hispanic White patients. This observational cross-sectional study used real-world data from the Center for International Blood and Marrow Transplant Research (CIBMTR) registry. All patients receiving their first allogeneic HCT in the United States between 2009 and 2020 were included, with a focus on transplantations performed in 2020. Data from patients undergoing allogeneic HCT using bone marrow, peripheral blood, or cord blood from HLA-matched or mismatched related and unrelated donors were analyzed. Specifically, relative proportion of allogeneic HCT was generated as percentage of total for donor type and for patient age, disease indication, GVHD prophylaxis, and race and ethnicity. Causes of death were summarized using frequencies, and the Kaplan-Meier estimator was used for estimating overall survival. Compared to matched related donor and matched unrelated donor HCT, more ethnically diverse patients received mismatched unrelated donor, haploidentical donor, and cord blood HCT. Although matched unrelated donor remains the most common donor type, the use of haploidentical donors has increased significantly over the last 5 years. Paralleling this increase in haploidentical HCT is the increased use of post-transplantation cyclophosphamide (PTCy) as GVHD prophylaxis. Relative to previous transplantation eras, the most contemporary era is associated with the highest survival rates following allogeneic HCT irrespective of patient race and ethnicity. Nonetheless, disease relapse remains the primary cause of death for both adult and pediatric allogeneic HCT recipients by donor type and across all patient racial/ethnic groups. Ethnically diverse patients are undergoing allogeneic HCT at higher rates, largely through the use of alternative donor platforms incorporating PTCy. Maintaining access to potential life-saving allogeneic HCT using alternative donors and novel GVHD prophylaxis strategies and improving HCT outcomes, particularly disease relapse, remain urgent clinical needs.

Keywords: Bone marrow transplantation; Cord blood; Death; Ethnicity; Graft-versus-host disease; Haploidentical; Hematopoietic cell transplantation; Infection; Peripheral blood; Race; Relapse; Related donor; Survival; Unrelated donor.

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Conflict of interest statement

Dr. Jeffery Auletta reports Employee, National Marrow Donor Program and Advisory Committee for AscellaHealth.

Dr. Steven Devine reports providing paid advise to the following companies - Orca Bio, Vor, Magenta Inc. He states that this money is paid to NMDP, not to him directly.

Kathryn Flynn reports research grants from NIH and Novartis, consulting for ReFocus, Inhibikase Therapeutics, and Pfizer.

Dr. Mehdi Hamadani reports Compensation: Research Support/Funding: Takeda Pharmaceutical Company; ADC Therapeutics; Spectrum Pharmaceuticals; Astellas Pharma. Research Funding to Institution: Janssen R&D, Celgene Corporation, Merck, MedImmune, Seattle Genetics, Millennium Pharmaceuticals. Consultancy: Incyte Corporation (2021); ADC Therapeutics (2021); Pharmacyclics (2018), Omeros (2021), Verastem (2019), TeneoBio (2019), MorphoSys (2021), Kite (2021), Genmab (2021), SeaGen (2021), Gamida Cell (2021), Novartis (2021), Legend Biotech (2021). Speaker’s Bureau: Sanofi Genzyme (2021), AstraZeneca (2021), BeiGene (2021), ADC Therapeutics (2022). DMC: Myeloid Therapeutics, Inc.

Dr. Stephanie Lee reports: Compensation: Consultant: Mallinckrodt, Equillium. Spouse: Consultant: Almirall, Rain Therapeutics, EMD Serono/Pfizer Honoraria: Wolters Kluwer

Payments: Research funding: Amgen, AstraZeneca, Incyte, Kadmon, Novartis, Pfizer, Syndax, Takeda. Provision of study medication: Janssen. Spouse: Research funding: BMS, EMD Serono

Relationships: Non-profit leadership: Board of Directors, National Marrow Donor Program. Spouse - Non-profit leadership: Secretary/Treasurer, Society for Investigative Dermatology Proprietary Interests: Spouse - Patents: Immunotherapy for Merkel Cell Carcinoma.

Dr. Marcelo Pasquini reports compensation from Bristol Myers Squibb for consulting.

Dr. Rachel Phelan reports compensation - advisory board from both Blue Bird Bio and Bioline Rx; research funding from Amgen.

Dr. Marcie Riches reports: Compensation: IQVIA Biotech, employee (as of 1/10/22).

Dr. Douglas Rizzo reports: Compensation: I participate in Optum Stem Cell Expert Panel to provide input /information about the center specific survival analysis based on my expertise. Compensation for this effort (~$1000) is directed to MCW rather than to me personally.

Dr. Bronwen Shaw reports consulting to Orcabio and Mallinkrodt.

Figures

Figure 1.
Figure 1.. Relative proportion of allogeneic hematopoietic cell transplants (HCTs) in the United States by recipient race and ethnicity, disease indications, and donor types.
Panel A, Relative proportion of allogeneic HCTs in the U.S. by recipient race and ethnicity (2010–2020). For each year, proportions of transplants by indicated race and ethnicity group is shown as percentage of absolute number of transplants performed (N) for that year. “Other” race and ethnicity group includes non-Hispanic Native Hawaiian or other Pacific Islander, American Indian or Alaska Native, more than one race, and non-resident of the U.S. Panel B, Relative proportion of allogeneic HCTs by disease indications in the U.S. by race and ethnicity in 2020. For each year, proportions of transplants by indicated disease indication is shown as percentage of absolute number of transplants performed (N) for that year. Non-malignant diseases category excludes aplastic anemia. ALL, acute lymphoblastic leukemia; AML, acute myeloid leukemia; MDS, myelodysplastic syndromes. Panel C, Relative proportion of allogeneic HCT in the U.S. by donor type (2010–2020). For each year, proportions of transplants by indicated donor type is shown as percentage of absolute number of transplants performed (N) for that year. CB, cord blood; Haplo, haploidentical donor; MMUD, mismatched unrelated donor; MRD, matched related donor; MUD, matched unrelated donor.
Figure 1.
Figure 1.. Relative proportion of allogeneic hematopoietic cell transplants (HCTs) in the United States by recipient race and ethnicity, disease indications, and donor types.
Panel A, Relative proportion of allogeneic HCTs in the U.S. by recipient race and ethnicity (2010–2020). For each year, proportions of transplants by indicated race and ethnicity group is shown as percentage of absolute number of transplants performed (N) for that year. “Other” race and ethnicity group includes non-Hispanic Native Hawaiian or other Pacific Islander, American Indian or Alaska Native, more than one race, and non-resident of the U.S. Panel B, Relative proportion of allogeneic HCTs by disease indications in the U.S. by race and ethnicity in 2020. For each year, proportions of transplants by indicated disease indication is shown as percentage of absolute number of transplants performed (N) for that year. Non-malignant diseases category excludes aplastic anemia. ALL, acute lymphoblastic leukemia; AML, acute myeloid leukemia; MDS, myelodysplastic syndromes. Panel C, Relative proportion of allogeneic HCT in the U.S. by donor type (2010–2020). For each year, proportions of transplants by indicated donor type is shown as percentage of absolute number of transplants performed (N) for that year. CB, cord blood; Haplo, haploidentical donor; MMUD, mismatched unrelated donor; MRD, matched related donor; MUD, matched unrelated donor.
Figure 2.
Figure 2.. Relative proportion of allogeneic HCTs in the United States by recipient race and ethnicity for two transplant eras, 2009–2014 and 2015–2020.
For each transplant era, proportions of transplants by indicated donor type is shown as percentage of absolute number of transplants performed (N) for that year. “Other” patient race and ethnicity group includes non-Hispanic Native Hawaiian or other Pacific Islander, American Indian or Alaska Native, more than one race, and non-resident of the U.S.
Figure 3.
Figure 3.. Relative proportion of matched and mismatched donor HCT in the U.S. by graft-versus-host disease (GvHD) prophylaxis (2010–2020).
Panel A, Relative proportion of matched donor HCTs in the U.S. by GvHD prophylaxis. For each year, proportions of transplants by indicated GvHD prophylaxis is shown as percentage of absolute number of transplants performed (N) for that year. Panel B, Relative proportion of mismatched donor HCTs in the U.S. by GvHD prophylaxis. Ex vivo graft manipulation includes T-cell depletion/CD34 selection as well as other techniques. CNI, calcineurin inhibitor; PTCy, post-transplant cyclophosphamide.
Figure 4.
Figure 4.. Trends in recipient overall survival after allogeneic HCT for hematologic malignancies by transplant era.
Kaplan-Meier curves combining survival data for AML, ALL and MDS is shown for each indicated transplant era.
See this image and copyright information in PMC

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