Emerging approaches to improve allogeneic hematopoietic cell transplantation outcomes for non-malignant diseases.
Zachariah DeFilippMehrdad HefaziYi-Bin ChenBruce R BlazarPublished in: Blood (2021)
Many congenital or acquired non-malignant diseases (NMD) of the hematopoietic system can be potentially cured by allogeneic hematopoietic cell transplantation (HCT) with varying types of donor grafts, degrees of HLA matching, and intensity of conditioning regimens. Unique features that distinguish the use of allogeneic HCT in this population include higher rates of graft failure, immune-mediated cytopenias, and the potential to achieve long-term disease-free survival in a mixed chimerism state. Additionally, in contrast to patients with hematologic malignancies, a priority is to completely avoid graft-versus-host disease in patients with NMD, as there is no theoretical beneficial graft-versus-leukemia effect that can accompany graft-versus-host responses. In this review, we discuss the current approach to each of these clinical issues and how emerging novel therapeutics hold promise to advance transplant care for patients with NMD.
Keyphrases
- bone marrow
- stem cell transplantation
- free survival
- hematopoietic stem cell
- high dose
- healthcare
- acute myeloid leukemia
- palliative care
- quality improvement
- small molecule
- cell cycle arrest
- type diabetes
- adipose tissue
- magnetic resonance imaging
- big data
- allogeneic hematopoietic stem cell transplantation
- low dose
- pain management
- cell death