OBJECTIVES: Allogeneic (allo) haematopoietic stem cell transplantation (HSCT) is indicated for various malignant and non-malignant disorders. In the absence of a matched sibling donor, technique choice is influenced by multiple clinical and non-clinical factors. Our objective is to review the literature on donor availability (DA), quality of life (QoL) and economic implications.

METHODS: Systematic literature review identified recent (last 10 years, no geographical restriction) literature on clinical outcomes, DA, QoL and economic (E) analyses in paediatric allo-HSCT. Study types included clinical trials, observational studies and economic evaluations. Electronic searches (MEDLINE, EMBASE) were supplemented with hand-searching of key grey literature. We identified 4,932 unique citations, after title/abstract screen 237 full-text articles were reviewed and 121 articles (94 clinical outcomes, 16 DA, 5 QoL and 6 E) were included. We report findings from DA, QoL and E sub-sets.

RESULTS: Available literature was sparse and heterogenous, and interpretation is challenged by the variety of practices regardless of donor source – e.g. pre-/post-HSCT treatments, definition of match. DA studies were all retrospective, primarily from US/Canada (n=7/16), and most examined securing a matched familial donor, or unrelated donor from registry. Studies reported the importance of a ‘common’ HLA type in finding an unrelated donor.

QoL studies were from 4 different countries (patients/study: US, n=23 and n=80; Sweden, n=75; China, n=350; France, n=341). Generalisable themes are difficult to draw, though infection and GvHD were noted as significant QoL drivers.

E studies consisted of 2 economic evaluations (Australia, India) and 4 costing studies (US, n=3; Italy). The Australian study compared cost-effectiveness of allo-HSCT techniques; the Indian study compared allo-HSCT vs transfusion chelation in thalassaemia.

CONCLUSIONS: Although limited, literature indicates locating unrelated donors can be challenging, especially for mixed/non-Caucasian ethnic groups. Considering the complexity of decision-making in the area, sparsity and heterogeneity of QoL and E studies were striking.