Rethinking 'Care after Cure' in allogeneic hematopoietic stem cell transplantation using e-Health and patient reported outcomes

Allogeneic hematopoietic stem cell transplantation (HCT) is a curative treatment for many hematological diseases. Thanks to advances in conditioning treatment, donor inventory, matching techniques, and supportive care, the number of survivors after HCT keeps increasing. The majority of these patients has an expected survival of 50% at one year and will enjoy an 85% chance of remaining alive long term if they reach the two years post transplantation milestone. However, although HCT has the potential to cure the original disease, patients will carry on the burden of specific co-morbidities induced by the treatment, and their life expectancy will remain about 30% lower than that expected for their age. HCT survivors differ from the healthy population, as their risk of developing a metabolic syndrome or subsequent malignancies is about twice of that expected. The largest cross-sectional HCT survivor self-reported study to date identified HCT patients to be more than five times more likely to develop life-threatening complications than their HLA matched sibling donor counterparts and to have a threefold risk for adverse psychological outcome, essentially linked to somatic distress. Long-term HCT patients also tend to display sub-optimal health behaviors, thereby increasing their risk of developing unnoticed complications. Specific HCT survivorship issues are therefore becoming a true concern of transplantation healthcare teams. In this doctoral project, we hypothesized that the characterization of HCT survivors could be improved by using e-Health and patient reported outcomes (PRO) measures.

One of the most frequent medical complication following HCT is graft versus host disease (GVHD), which refers to the response of transplanted donor allogeneic cells to histocompatibility antigens expressed on tissues of the transplantation recipient. This results in a clinical syndrome which typically affects up to ten different organs. We were surprised to notice that no single publication clearly identified how GVHD should be assessed. We therefore created an international task force of GVHD experts to come to a position statement to describe the optimal way to assess this disease and proposed a standardized language to describe its evolution.

To facilitate the implementation of these recommendations, we developed an electronic tool, the eGVHD App using a human centered design strategy. This tool was validated by a three-step process (first in a pilot project in our own center, then through a survey done at an international congress and finally in a national randomized trial). We could show that using the App improved the accuracy and inter-rater reliability of GVHD assessment in healthcare professionals. In the meantime, the eGVHD App has been downloaded over 3000 times and is currently being used in a number of scientific projects.

We also performed a comprehensive profiling of HCT recipients having survived at least 2 years after HCT using a case-control cross-sectional design. We combined a physician-led clinical evaluation (supported by the eGVHD App) with a psychosocial evaluation of the perceived health status and behavior of HCT survivors. HCT recipients were then compared to individuals from the general population (matched based on age, gender and province of residence), using data from the Belgian national health survey. HCT survivors were shown to have a high number of medical complications and a lower perceived health status, a lower employment rate and to be using more prescription drugs than their matched counterparts.

Considering the high prevalence of GVHD and the wide range of medical and non-medical long-term effects, we identify survivorship as a chronic condition. We therefore recommend to rethink HCT follow-up based on the ‘Chronic Care Model’. This requires an intense multidisciplinary collaboration to address all aspects of health and demands clear guidelines and objectives to evaluate our current practice. Communication between all stakeholders (other health care providers, policy makers, community individuals and the patients themselves) needs to be fostered to achieve optimal long-term outcome. In particular, active patient involvement is crucial to fully appreciate the true impact of HCT, manage complications and design future survivorship research projects.