Haploidentical transplantation combined with unrelated cord blood resulted in superior one-year disease-free survival compared with the same transplant using bone marrow in patients with hematological malignancies, according to a study published in The Lancet aHematology.
Researchers led by Sijian Yu, MD, from the Department of Hematology at Nanfang Hospital in Guangzhou, China, assessed whether disease-free survival rates were higher when haploidentical peripheral blood stem cell (PBSC) transplants were combined with unrelated cord blood versus bone marrow.
The open-label, randomized, phase 3 trial was conducted across seven hospitals in China and enrolled patients 18–65 years old undergoing their first transplant with a Eastern Cooperative Oncology Group performance status of 0–2 and transplant comorbidity index of 0–2. At the time of study, patients were randomized 1:1 to receive haploidentical PBSC transplant with either unrelated cord blood (157 patients) or bone marrow (157 patients). The trial’s primary endpoint was one-year disease-free survival.
The median follow-up was 17.2 months (IQR, 10.0-20.8). One-year disease-free survival was 82.2% (95% CI, 75.2-87.3) in the group receiving haploidentical PBSCs plus unrelated cord blood and 65.6% (95% CI, 57.6-72.5) in the group receiving haploidentical PBSCs plus bone marrow (HR, 0.47; 95% CI, 0.30-0.74; P=.001).
The most common grade 3-5 adverse events within 100 days of transplantation were infections (37% in the PBSCs plus cord blood group versus 49% in the PBSCs plus bone marrow group; P=.03), acute graft-versus-host disease (31% vs 39%), and gastrointestinal disorders (24% in both groups).
Transplantation-related deaths within 100 days occurred in 4% of patients in the PBSCs plus cord blood group and 11% in the bone marrow group.
“Transplantation of haploidentical PBSCs plus unrelated cord blood achieves superior disease-free survival compared with transplantation of haploidentical PBSCs plus bone marrow in patients with hematological malignancies, with lower incidence of relapse and transplantation-related mortality,” the authors concluded. “Including unrelated cord blood rather than bone marrow in a mixed graft for transplantation could be a better option for this population.”
Reference
Yu S, Huang F, Xu N. Haploidentical peripheral blood stem cells combined with bone marrow or unrelated cord blood as grafts for haematological malignancies: an open-label, multicentre, randomised, phase 3 trial. 2025. Lancet Haematol. doi.org/10.1016/S2352-3026(24)00372-7
