Global expert perspectives on the future of hematopoietic cell transplantation and cell therapies

Abstract

Settings: Hematopoietic cell transplantation (HCT) remains a cornerstone therapy for hematologic malignancies and several non-malignant disorders. At the same time, emerging therapies, particularly chimeric antigen receptor T cell (CAR-T) cells and other immunotherapies, are transforming the treatment landscape. Understanding how specialists anticipate these changes is critical for strategic planning in healthcare systems. Methodology: We conducted a global expert survey using a foresight informed framework to explore expected developments in HCT and related technologies over the next decade. Following a literature review of 4,312 publications indexed in Web of Science (2018–2022), a questionnaire was distributed to specialists in hematologic cancers and bone marrow transplantation identified through publication databases. Personalized email invitations were sent in April and May 2023. Descriptive analyses and non-parametric tests were used to examine differences according to self-reported knowledge levels. Results: A total of 1,711 valid responses were analyzed. Haploidentical transplantation was identified as the most promising transplantation modality for the next decade, particularly among respondents with higher knowledge levels. Severe combined immunodeficiency and sickle cell disease were the most frequently cited non-malignant indications. Knowledge gaps, regulatory barriers, and treatment costs were perceived as major obstacles to innovation, while limited health system infrastructure was identified as the main barrier to expanding access in middle-income countries. CAR-T cell therapy was considered the most promising emerging technology overall, although highly knowledgeable respondents more often identified bispecific antibodies as the most promising innovation. Conclusion: Experts anticipate continued growth in haploidentical transplantation alongside the expansion of advanced immunotherapies such as CAR-T cells. However, regulatory, financial, and infrastructure constraints may delay their widespread implementation, particularly in middle-income settings. Although several emerging therapies discussed in this study – such as CAR-T cell therapy and certain autologous cellular approaches – do not directly depend on unrelated donor registries, the anticipated persistence of allogeneic transplantation as a core therapeutic modality suggests that maintaining robust national donor registries will remain strategically important. In particular, donor registries are expected to retain substantial relevance for malignant and non-malignant indications in which allogeneic HCT remains clinically indicated despite the parallel expansion of non-registry-dependent advanced therapies.