Recent Research - New NIH Report: Recommended Follow-Up for Long-Term Survivors after HCT

This report outlines the recommendations issued by the National Institutes of Health (NIH) Late Effects Consensus Conference, which was tasked with planning the next phase of hematopoietic cell transplantation (HCT) research “to ensure that no survivor is left behind.”

The table below highlights key recommendations from the consensus report.

Area of research	Selected recommendations based on post-HCT risks
Cardiac, vascular and metabolic	HCT survivors have approximately 4 times the risk of developing cardiovascular risk than the general population. Arterial disease Establish HCT-specific risk prediction models Evaluate novel imaging and blood biomarkers for screening Test interventions in high-risk survivors Cardiac dysfunction Determine contribution of pre-HCT exposures and comorbidities Examine mechanisms of enhanced cardiotoxicity Test preventive interventions in high-risk survivors
Subsequent neoplasms	Subsequent solid tumor neoplasms occur at more than a two-fold risk in HCT survivors compared to the general population, with a high incidence in skin malignancies. Define magnitude of risks for specific subsequent neoplasms Assess genetic risk factors Validate cancer prevention interventions (e.g., HPV vaccination)
Immune dysregulation	There is a growing awareness of very late lethal infections in the absence of active GVHD. Late infections Establish a long-term multicenter registry to identify serious infections, types of pathogens and risk factors Evaluate immunologic correlates using banked samples Evaluate early and late microbiota changes with late infections and immune reconstitution Immune reconstitution Identify the molecular mechanisms of late dysfunctional adaptive immunity Investigate adaptive immune system neogenesis, maturation and exhaustion Assess late functional pathogen-specific T- and B-cell responses Prevention of infections Correlate immune reconstitution markers with vaccine responses to standardize thresholds for initiating vaccination Conduct vaccination specific prospective multicenter trials Assess role of other therapies such as IVIG
Quality of life and psychosocial outcomes	HCT recipients are at a long-term risk for psychological distress that can affect survival. Design and test risk-targeted interventions that address resource utilization and costs, as well as process measures, including feasibility, treatment fidelity, sustainability and dissemination potential. Priority domains are sexual dysfunction, fatigue/sleep disruption, non-adherence, health behaviors such as physical inactivity, and psychological dysfunction
Research methodology and study design	To better understand post-transplant late effects, there is a critical need to establish new cohorts or expand existing cohorts that capture comprehensive and complete follow-up of HCT recipients. High priority areas for data collection are late effects that have high incidence of morbidity, impairment, disability and/or premature mortality, that have excess risk compared to general population and have potentially modifiable risk factors
Health care delivery	HCT survivor care needs to be responsive to the preferences of the patient and caregivers with close coordination between the referring physician and transplant center staff. Health care delivery models Evaluate patient self-management and information technology tools for enhancing patient involvement Evaluate healthcare disparities and issues in special populations including caregivers Evaluate role of supportive therapies Coverage and value Establish infrastructure and databases to conduct studies on costs and value of HCT Identify patient-centered coverage models for preventive care and late complications Evaluate patient reported outcomes to inform value and coverage models

Battiwalla M, et al. Biol Blood Marrow Transplant

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New NIH Report: Recommended Follow-Up for Long-Term Survivors after HCT

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