Blood stem cell transplant using novel
treatment modality improves outcomes and reduces risk of
life-threatening graft-versus-host disease, NMDP and CIBMTR
research shows
Results show nearly three-fold increased
likelihood of identifying a suitable donor for blood cancer
patients from ethnically diverse backgrounds
Research results from NMDP℠ and CIBMTR® (Center for
International Blood and Marrow Transplant Research®)—published
today in the peer-reviewed Journal of Clinical Oncology—found no
discernable difference in overall survival (OS) among patients with
blood cancer receiving blood stem cell transplant when
investigators used an unrelated donor matched at 7/8 human
leukocyte antigen (HLA) markers, compared to a fully matched 8/8
unrelated donor when using a post-transplant cyclophosphamide-based
(PTCy) graft-versus-host disease (GVHD) prevention strategy.
Conducted by CIBMTR—a research collaboration between the Medical
College of Wisconsin® and NMDP—the observational study also showed
no discernable differences in GVHD-free, relapse-free survival
(GRFS) for adults with blood cancers who had a 7/8 or an 8/8
unrelated donor transplant when using PTCy.
The study, “Post-Transplant Cyclophosphamide–Based
Graft-Versus-Host Disease Prophylaxis Attenuates Disparity in
Outcomes Between Use of Matched or Mismatched Unrelated Donors,”
evaluated 10,025 adult patients at 153 U.S. transplant centers who
underwent initial unrelated donor blood stem cell transplant for
acute leukemia or myelodysplastic syndromes from 2017–2021 and
reported outcomes to CIBMTR. The most clinically significant
outcome observed showed that when compared to 8/8 unrelated donor
transplant using PTCy, 7/8 unrelated donor transplant with PTCy had
similar OS (hazard ratio [HR], 0.96 [95% Confidence Interval,
0.823-1.11]; P = .60) and similar GRFS (HR, 0.90 [0.79-1.02]; P =
.1).
Previously established methods to prevent GVHD involved use of
immunosuppressants called calcineurin inhibitors, yet outcomes were
traditionally worse with mismatched donors. In this study, when
compared to 8/8 unrelated donor transplant using traditional
calcineurin-inhibitor GVHD prevention methods, OS was improved
after 8/8 PTCy transplant (HR, 0.88 [0.80-0.96]; P = .004) and
similar with 7/8 PTCy transplant (HR, 0.92 [95% CI, 0.80 to 1.05];
P = .2062). GRFS was improved after 8/8 (HR, 0.61 [0.57-0.66]; P
< .0001) or 7/8 PTCy transplant (HR, 0.68 [0.60-0.76]; P <
.0001).
Investigators showed adoption of novel treatment modality PTCy
can eliminate disparities in survival, greatly reduce GVHD risk,
and expand safe and effective transplant access to people for whom
a matched unrelated donor is unavailable, which disproportionally
impacts patients who are racially and ethnically diverse.
“These data confirm we are at the forefront of transplant
practice change. As our global population becomes more diverse and
geographically disparate, patients facing blood cancer are
increasingly looking for a safe and effective method to increase
their survival and longevity,” Brian Shaffer, MD, co-first author;
bone marrow transplant specialist & cellular therapist,
Memorial Sloan Kettering Cancer Center, said. “Achieving
recognition from one the most prestigious, peer-reviewed medical
publications demonstrates the credibility of these data and builds
the evidential case for using mismatched unrelated donors to expand
access to blood stem cell therapy for all patients.”
In the U.S., patients with blood cancers from Asian/Pacific
Islander backgrounds currently have a 47% chance of finding a fully
matched, 8/8 unrelated donor on the NMDP Registry℠ of potential
volunteer donors. However, those odds increase to 92% when
searching for a 7/8 match or greater. African American and Black
patients’ chances jump from 29% with an 8/8 match, to as high as
84% with a 7/8 match—while patients with Latino / Hispanic or
non-Hispanic White ancestry improve their odds from 48% (8/8) to
90% (7/8) and 79% (8/8) to 99% (7/8), respectively.1
“This novel approach transforms the landscape for physicians,
researchers, patients and families, expanding the definition of a
‘suitable donor,’” said Steven Devine, MD, chief medical officer,
NMDP and senior scientific director, CIBMTR. “Not only does
achieving significantly greater outcomes give patients another
chance at life, it breaks down the barriers to transplant for all
patients, regardless of their ancestry.”
These results are part of NMDP’s ongoing research commitment to
expand access to cell therapy to all patients and close the donor
availability gap. NMDP’s network of transplant centers, many of
which participate in CIBMTR trials, are collaborating to bring new
research to light that is challenging previously established blood
stem cell transplantation science. NMDP’s Donor for All initiative
includes several research efforts, including this published study,
that aim to establish a new, safe and effective approach for using
mismatched unrelated donor transplants in the U.S. and abroad.
Operational data from NMDP-facilitated transplants show 60%
year-over-year growth in mismatched unrelated donor transplant—the
majority of which are from unrelated donors matched at 7/8 HLA
markers, indicating how the U.S. medical community is seeking other
safe and effective, evidence-based methods to prolong patients’
ability to survive and thrive.2
“This is scientific evidence showing we are addressing an unmet
need,” Dr. Devine added.
CIBMTR presented findings from this study at the American
Society for Transplantation and Cellular Therapy’s scientific
congress, Tandem 2024, in February 2024.
About CIBMTR®
CIBMTR® (Center for International Blood and Marrow Transplant
Research®) is a nonprofit research collaboration between NMDP℠, in
Minneapolis, and the Medical College of Wisconsin, in Milwaukee.
CIBMTR collaborates with the global scientific community to
increase survival and enrich quality of life for patients. CIBMTR
facilitates critical observational and interventional research
through scientific and statistical expertise, a large network of
centers, and a unique database of long-term clinical data for more
than 675,000 people who have received hematopoietic cell
transplantation and other cellular therapies. Learn more at
cibmtr.org.
About NMDP℠
At NMDP℠, we believe each of us holds the key to curing blood
cancers and disorders. As a global nonprofit leader in cell
therapy, NMDP creates essential connections between researchers and
supporters to inspire action and accelerate innovation to find
life-saving cures. With the help of blood stem cell donors from the
world’s most diverse registry and our extensive network of
transplant partners, physicians and caregivers, we’re expanding
access to treatment so that every patient can receive their
life-saving cell therapy. NMDP. Find cures. Save lives. Learn more
at nmdp.org.
1
Chowdhury AS, Maiers M, Spellman SR,
Deshpande T, Bolon YT, Devine SM. Existence of HLA-mismatched
unrelated donors closes the gap in donor availability regardless of
recipient ancestry. Transplant Cell Ther.
2023:29(11):686.e1-686.e8. doi: 10.1016/j.jtct.2023.08.014.
2
Internal data on file, 2021-2023.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240717205240/en/
Media contact: Jess Ayers media@nmdp.org