NMDP and CIBMTR to Present Promising Stem Cell Transplant Data at 2024 ASCO Meeting

Adults with blood cancers who receive peripheral blood stem cell (PBSC) transplants show promising survival rates and a low incidence of severe graft-versus-host disease (GvHD), according to interim results from the ACCESS trial, announced by NMDP and CIBMTR. These findings will be presented at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago. The trial focused on adults with hematologic malignancies who received PBSC transplants from mismatched unrelated donors (MMUD) and post-transplant cyclophosphamide (PTCy) for GvHD prophylaxis.

Key outcomes included a 79% overall survival rate and a 51% GvHD-free, relapse-free survival (GRFS) rate at one year post-transplant. Additionally, severe acute and chronic GvHD incidences were both observed at a low rate of 9%. These promising results support the inclusion of this treatment approach for patients receiving PBSC transplants from partially matched unrelated donors, enhancing transplant options, especially for those who traditionally face challenges in finding matched donors.

Dr. Monzr M. Al Malki, the presenting author and ACCESS study co-chair, emphasized the positive transplant outcomes and improved quality of life for patients. He noted that these preliminary data provide strong support for integrating this new treatment approach into clinical practice, thereby benefiting more patients from the potentially curative effects of transplants.

The ACCESS trial, sponsored by NMDP and conducted through CIBMTR, enrolled 70 adult patients with various blood cancers and disorders, such as acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and acute lymphoblastic leukemia (ALL). These patients, from 13 transplant centers including City of Hope, received reduced intensity conditioning, predominantly fludarabine/melphalan or fludarabine/busulfan, and PBSCs from donors with a 5-7 out of 8 HLA loci match, mostly at the 7/8 match level. Notably, the trial's demographics included a 50% representation of people of color.

Dr. Steven M. Devine, a co-author of the study and Chief Medical Officer of NMDP, highlighted the historical barriers to transplant, such as the low availability of matched, related sibling donors and the significant variance in matched unrelated donor availability, particularly for patients from diverse ancestries. The findings from the ACCESS trial advance the ability to offer more transplant options to patients without a fully matched donor, many of whom are ethnically diverse.

The NMDP-led research addresses these barriers through initiatives like the "Donor for All" trial, which demonstrated that PTCy effectively reduces GvHD risk in adults receiving bone marrow transplants from MMUD. In this trial, nearly half of the participants were ethnically diverse, showing a one-year overall survival rate of 76% and a low rate of moderate to severe GvHD. Building on these findings, the ongoing OPTIMIZE trial is assessing whether a reduced dose of PTCy can safely prevent GvHD while minimizing infection risk in patients receiving PBSC transplants from MMUD.

Recent research presented by CIBMTR at the 2024 Tandem Meetings revealed no significant differences in overall survival or GRFS between patients using MMUD HCT at 8/8 or 7/8 HLA match levels with PTCy prophylaxis. This increases the likelihood of finding suitable donors for patients of all ethnicities to at least 84%, sometimes reaching up to 99%.

Dr. Devine reiterated that the "Donor for All" research underpins the future of equitable access to stem cell transplants, offering new hope to patients worldwide by bridging the gap in transplant availability. The ongoing efforts aim to prevent and treat GvHD, decrease post-transplant relapse risk for hematologic malignancies, and potentially apply MMUD transplants to cure non-malignant conditions like sickle cell disease.

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