OBJECTIVES: Health policy makers and payers have a limited knowledge and understanding of allogeneic haematopoietic stem cell transplantation (alloHSCT) and in particular the impact of graft-versus-host disease (GvHD) on patient outcomes (quality of life (QoL) and their long-term survival) and costs. The purpose of this analysis is to raise awareness of the GvHD economic burden and the need for new and scientifically proven and cost-effective treatments to maximize successful outcomes.

METHODS: A targeted literature review studied GvHD in alloHSCT, to examine patient burden and especially the impact of GvHD on QoL. The economic burden, considering the impact on healthcare resource utilization and costs, and the current and potential treatments were analyzed.

RESULTS: AlloHSCT is complex and expensive but increasingly used as a potentially curative therapy. Following alloHSCT, GvHD is the most life-threatening complication, with between 39% and 59% of patients developing acute GvHD; treating GvHD places a significant burden on patients and healthcare resources, and increases costs. A US study showed 1-year post alloHSCT costs increasing from $263,568 to $466,720 for acute GvHD patients. Other studies showed costs up to four times higher depending on the severity. Systemic steroids are used first-line for GvHD but response decreases with disease severity. There is no standard for second-line and subsequent treatments and many therapies are used for GvHD based on individual physician experience rather than clinical and economic evidence.

CONCLUSIONS: To ensure optimal benefit from alloHSCT, the long-term goal is to prevent or control GvHD, preserving the beneficial effects of the graft and minimising the costs of an expensive but life-saving therapy. Treatments to improve long-term post-transplant outcomes remain an unmet need. Improving and expanding the clinical and economic evidence base should be a high priority when investigating new therapeutic agents.