November 2024 SOCRA Source Journal - Journal - Page 81
CLINICAL TRIALS
2024 POSTER
PROGRAM
SPECIAL RECOGNITION AWARD WINNER
Janelle A. Olson, PhD, NMDP
ACCESS Clinical Trial
Eliminates Disparity in
Identifying a Donor for all
Patients in Need of Stem
Cell Transplantation
Janelle A. Olson, Sarah Smith, Stephanie Bo-Subait,
Brent Logan, Erin Leckrone, Juan Wu, Heather E.
Stefanski, Jeffery J. Auletta, Stephen R. Spellman,
Craig Malmberg, Thomas Silverman, Deborah Mattila,
Michelle Kuxhausen, Caitrin Bupp, Rachel Cusatis, Larisa
Broglie, Bronwen E. Shaw, Antonio Martin Jimenez
Jimenez, Monzr M. Al Malki, Steven Michael Devine
BACKGROUND: For many patients with blood cancer, hematopoietic stem cell transplantation (HSCT) remains the
only option for a cure. The gold standard for HSCT is a related donor which is available for only 30% of patients, thus
matched unrelated (URD) (volunteer) donors are used. However, many patients cannot identify a fully matched URD,
which disproportionally impacts ethnically diverse (ED) patients due to diverse genetics and lack of matched donors
on registries. Using mismatched unrelated donors (MMUD) could alleviate this problem as modeling shows that all
patients should have an available MMUD, but outcomes are historically worse using MMUD donors.
Recently published results of a prospective, phase II multi-center trial (15-MMUD) assessed effectiveness of MMUD
HSCT using a post-transplant chemotherapy drug, cyclophosphamide (PTCy), to prevent graft versus host disease, a
serious complication where donor cells attack the patient. The study achieved its primary endpoint with 76% 1-year
overall survival (OS), showing this MMUD transplant approach is safe and effective.
Notably, 48% of 15-MMUD patients were ED, signi昀椀cantly higher than typical clinical trials. Such patients have long
faced disparities in access to transplant, worse post-HCT outcomes, and low clinical trial enrollment.
The current ACCESS study evaluates use of peripheral blood stem cells (PBSC), rather than bone marrow (BM) used
on 15-MMUD, as PBSC is most commonly used in transplant and less invasive for donors.
PURPOSE: We sought to determine whether OS in adults receiving PBSC from MMUD would be comparable or
superior to 15-MMUD results.
METHODS: We conducted a prospective, multi-center Phase II study (ACCESS; NCT04904588) to assess the impact of
PTCy-based GVHD prevention on OS following MMUD transplantation. We report results of a planned analysis of the
昀椀rst 70 adult patients completed. The study was designed to detect a primary endpoint of 1-year OS of 75%. Patientreported outcomes surveys were administered with questions about patient’s physical, emotional and social well-being
and 昀椀nancial impacts of disease and treatment.
RESULTS: Forty-two sites were open to enrollment, with 13 sites enrolling the 昀椀rst 70 patients. Patients had a median
age of 65 years. The primary endpoint of OS at 1-year post-HSCT was 79%.
Key secondary endpoints included a rate of severe acute GVHD of 9% at 6 months, and a disease relapse rate of 21%
at 1-year.
Looking at social determinants of health within PRO data, ED patients on ACCESS were signi昀椀cantly younger (55 v. 64
years), experienced more 昀椀nancial toxicity (score 22 v. 29.5) and higher social vulnerability (53% v. 18%)
CONCLUSION: Encouraging 1-year OS was observed in ACCESS. Rates of GVHD and other complications appear
comparable to other transplant settings, suggesting MMUD HSCT could expand access to a potentially life-saving
therapy and transform transplant options for patients.
Notably, half of enrolled patients were ED demonstrating that using MMUD expands access to HSCT for ED patients.
The PRO data showed access to HSCT is not only expanded to ED patients, but also to those with additional social
vulnerabilities.
Accrual was brisk and site participation broad, suggesting ACCESS addresses an unmet patient need.
SOCRA SOURCE © November 2024
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