The study that we’ll be discussing at EBMT was performed by the Center for International Blood and Marrow Transplant Research, or CIBMTR for short. CIBMTR is a federally supported research program that was formed as a partnership between NMDP and the Medical College of Wisconsin. And so we collect, analyze, and report outcomes data in virtually all the patients who receive donor transplants in the United States, work with other global registries, share the data, and accelerate progress for the benefit of patients...
The study that we’ll be discussing at EBMT was performed by the Center for International Blood and Marrow Transplant Research, or CIBMTR for short. CIBMTR is a federally supported research program that was formed as a partnership between NMDP and the Medical College of Wisconsin. And so we collect, analyze, and report outcomes data in virtually all the patients who receive donor transplants in the United States, work with other global registries, share the data, and accelerate progress for the benefit of patients.
And so the study was, again, using the outcomes database from the CIBMTR, where we’ve noticed that there’s been a profound shift in how transplant programs prevent graft-versus-host disease. So there’s a method that’s been around now about 20 years called post-transplant cyclophosphamide, or PTCy for short, and it’s been associated with lower risk of severe acute and lower risk of moderate to severe chronic graft-versus-host disease. And over the last six years, we’ve seen it go from being used in maybe a third of patients now to two-thirds of patients in the United States. And so we asked whether now with this shift in the use of PTCy use, whether outcomes differ based on the donor types. And the reason that’s important is PTCy was originally developed for use in HLA mismatched related, what we call haploidentical transplant recipients, and suggested that outcomes might be quite similar between matched and mismatched patients, but the numbers were lower. So now with larger numbers, we thought we should look again.
So with that background, we asked how were the outcomes in patients who received PTCy-based GvHD prophylaxis in the United States. And we looked at overall survival and other outcomes like survival without relapse or bad graft versus host disease.
So this is one of the largest studies we’ve ever performed. So there are over 10,000 patients who are involved in the study that I’ll be presenting. The majority of them received PTCy from living donors and included matched related and matched unrelated donors as well as mismatched unrelated donors and haploidentical donors. We did also include recipients of umbilical cord blood grafts because that’s a viable donor option as well. And so we really wanted to look across all the different donor options.
And so what we found is that matching still does matter with a little bit of an exception. So the matched related donors were associated with the best overall survival and survival free of relapse or bad graft versus host disease, but the matched unrelated donors had outcomes similar to the matched related donors. Now, among the mismatched donors, the mismatched unrelated donors actually had outcomes similar to the matched unrelated donors, which is something that we had seen in a prior study. But when we compared the mismatched unrelated donors to the other, the cord blood type, or the haploidentical-related donors, overall survival was better in mismatched unrelated donors compared to cord, and the survival without relapse or GvHD was better in the MMUDs or mismatched unrelated donors compared to haploidentical and the cord blood.
So it does look like HLA still matters in these patients, But I think the good news is that now in 2025, we can find donors for virtually all patients. So lack of a donor is no longer a reason not to have a transplant. And the outcomes comparing matched unrelated to mismatched unrelated donors are quite comparable.
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