Advancing Stem Cell Transplant Outcomes: Leveraging Alternative Donors in Clinical Practice

Stephanie Lee, MD, MPH, professor and associate director, Clinical Research Division, Fred Hutch Cancer Center, discusses a study published in the Journal of Clinical Oncology assessing how to improve outcomes for patients undergoing bone marrow transplantation.

Watch part 1, part 2, and part 3 of the interview with Dr Lee.

The research in question was conducted as an observational study, a design that required acknowledging the inherent variability in clinical practice across participating centers. While all centers were committed to following the core study protocol, they retained the flexibility to implement specific procedures and patient management strategies based on their own judgment and institutional standards. This real-world setting, though challenging for achieving complete procedural uniformity, accurately reflected how the new transplant approaches were being integrated into diverse clinical environments.

A crucial finding of this work was the strong evidence establishing that patients can achieve excellent results using alternative donors, specifically the haploidentical (half-matched family) and mismatched unrelated donors. This realization fundamentally shifts the paradigm for hematopoietic stem cell transplantation (HSCT). With this positive outcome established as a new clinical baseline, the research now opens a broader landscape of inquiry. The next critical step is no longer just demonstrating viability but actively working to determine and select the best alternative donor within that expanded pool. Clinicians can now focus on refining the selection criteria to further optimize patient outcomes.

The ultimate goal remains the continuous improvement of results across all types of transplants, regardless of the initial donor match—fully matched or severely mismatched. By having established a successful baseline for alternative donor transplants, the research community is now empowered to innovate and improve upon it, driving higher success rates and lower complication rates for every patient.

This significant study was a collaborative effort, organized and conducted by the Bone Marrow Transplant Clinical Trials Network (BMT CTN). Its success was a direct result of the collective commitment from a vast network of investigators, transplant centers, and patients. The scale of the study, involving numerous participants and institutions, serves as a powerful demonstration of the groundbreaking research that can be achieved when the entire transplantation community unites to address a pressing clinical question. The investigators were careful to acknowledge the vital financial support provided by NIH funding, which made the ambitious scope of the study possible, and they expressed sincere gratitude to all the patients and collaborators whose participation was essential to the successful completion and impact of the work.