NMDP and CIBMTR to Present New, Promising Stem Cell Transplantation Trial Data using Mismatched, Unrelated Donors at 2024 ASCO Annual Meeting
May 23 2024 - 5:01PM
Business Wire
Adults with blood cancers receiving peripheral
blood stem cell transplant exhibited excellent survival rates and
low incidence of life-threatening graft-versus-host-disease
Additional NMDP presentation on
population-level access barriers to bone marrow transplant
NMDPSM, a global nonprofit leader in cell therapy, and the
CIBMTR® (Center for International Blood and Marrow Transplant
Research®), announced that interim results from the ACCESS trial
will be presented as an oral abstract on Friday, May 31 at the
American Society of Clinical Oncology (ASCO) Annual Meeting in
Chicago, Ill., demonstrating that adults with hematologic
malignancies who received peripheral blood stem cell (PBSC)
transplant from HLA-mismatched unrelated donors (MMUD) followed by
post-transplant cyclophosphamide (PTCy) graft-versus-host-disease
(GvHD) prophylaxis exhibited a 79% overall survival, with a
significant 51% GvHD-free, relapse-free survival (GRFS) probability
at one-year post transplant. Notably, ASCO also selected this
abstract to be presented at its 2024 Best of ASCO program in
July.
“We want more patients to survive and thrive — results from
ACCESS to-date have shown positive, significant transplant outcomes
and good quality of life for patients,” said Monzr M. Al Malki,
M.D., Associate Professor, Department of Hematology &
Hematopoietic Cell Transplantation, City of Hope; presenting author
and ACCESS study co-chair. “These preliminary data provide strong
support for inclusion of this novel treatment approach for patients
receiving PBSC from partially matched, unrelated donors and advance
current evidence demonstrating how more patients can benefit from
the potentially curative therapeutic effects of transplant.”
In addition to achieving very good OS and GRFS clinical
endpoints, adult participants also exhibited low rates of severe
acute and chronic GvHD, both at 9%. The NMDP-sponsored ACCESS
trial, conducted through the CIBMTR — a research collaboration
between the Medical College of Wisconsin and NMDP — enrolled 70
adult patients with blood cancers and disorders, including acute
myeloid leukemia (AML), myelodysplastic syndrome (MDS), and acute
lymphoblastic leukemia (ALL), from 13 participating transplant
centers, like City of Hope. All patients in this stratum received
reduced intensity conditioning, most often fludarabine/melphalan
(63%) or fludarabine/busulfan (20%), and PBSCs from donors matched
at 5-7 of 8 HLA loci, predominantly at the 7/8 match level (67%).
Notably, half of patients enrolled in the trial were people of
color.
“Historically, barriers in access to transplant have been
inhibited by two factors: the low availability of matched, related
sibling donors; and the substantial variance of matched, unrelated
donor availability on international registries, particularly for
patients with diverse ancestry — many of whom already face
significant challenges,” said study co-author Steven M. Devine,
M.D., Chief Medical Officer, NMDP; Senior Scientific Director,
CIBMTR. “Our research findings advance our ability to offer more
options to patients without a fully matched donor, many of whom are
ethnically diverse and have been underserved in receiving
potentially life-saving cell therapy.”
Addressing Equitable Care: NMDP-led Donor for All
Research
Traditionally, finding an available matched, unrelated donor on
international registries has been limited for patients with
ethnically diverse ancestry – from as low as 29% for Black or
African American patients and 48% for Latino and Hispanic patients
versus 79% for non-Hispanic Whites. NMDP’s network of transplant
centers, many of which participate in CIBMTR trials, are bringing
new research to light that is challenging previously established
stem cell transplantation science.
ACCESS builds upon findings from the first “Donor for All”
trial, a groundbreaking NMDP-sponsored 15-MMUD study, which showed
that PTCy was effective in decreasing risk for GvHD in adults with
hematologic malignancies receiving bone marrow (BM) transplants
from MMUD. Similar to ACCESS, 15-MMUD patients — 48% of whom were
ethnically diverse — showed a one-year overall survival (OS) rate
of 76% and exhibited a low rate of moderate / severe GvHD.
Enrolling now, the OPTIMIZE trial is evaluating whether a reduced
dose of PTCy will safely and effectively prevent GvHD while
reducing infection risk in patients with hematologic malignancies
receiving PBSC HCT from MMUDs. Finally, a recent observational
study by the CIBMTR presented at the 2024 Tandem Meetings, reported
no discernable differences in OS or GRFS for adult patients with
hematologic malignancies using MMUD HCT at an 8/8 or 7/8 HLA match
level using PTCy GvHD prophylaxis — increasing the likelihood for
patients of all ethnicities of finding a suitable donor to at least
84% and up to 99%.
“Our Donor for All research is the foundational future upon
which we are building a new platform protocol using innovative
strategies for preventing and treating GvHD, decreasing risk for
post-transplant relapse of hematologic malignancies, and in the
future, applying MMUD transplant to cure non-malignant conditions,
such as sickle cell disease,” said Dr. Devine. “Through CIBMTR, we
are showing that science can solve the gap in equitable access to
transplant, giving new hope to patients worldwide.”
2024 ASCO Presentation Details
Oral Presentation (Abstract
#6503)
Friday, May 31; 2:45-5:45 p.m. CDT; Room
S100bc
Post-transplant cyclophosphamide-based
graft-versus-host disease prophylaxis following mismatched
unrelated donor peripheral blood stem cell (PBSC)
transplantation
Monzr M. Al Malki, M.D.
Poster Discussion (Abstract
#1528)
Saturday, June 1; 9 a.m. – noon CDT; Hall
A
Identifying states for targeted alloHCT
access initiatives using social vulnerability, physician density,
and unmet need
Samantha Watters, MPH
About CIBMTR®
CIBMTR® (Center for International Blood and Marrow Transplant
Research®) is a nonprofit research collaboration between NMDPSM, 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 NMDPSM
At NMDPSM, 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.
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