Pediatric hematopoietic stem cell transplantation (HCT) is performed for an increasing number of life-threatening malignant and non-malignant conditions with the potential to provide definitive cure. However, its efficacy is limited by an inordinate number of complications, of which pulmonary complications constitute the leading cause of death during the initial year after transplantation. Approximately 2,500 pediatric HCTs are performed every year in the United States of which 1,600 are allogeneic. Allogeneic HCTs are associated with more severe pulmonary complications such as pneumonitis (infectious and non-infectious) and pulmonary hemorrhage during the first year after transplant. Predictors of these life-threatening conditions are unknown. The incidence of pulmonary complications plateaus after the first two post-transplant years. Thus, the first year post-HCT offers a crucial window of opportunity for risk mitigation to improve outcome.
This Workshop, co-sponsored by NHLBI, NICHD and NCI, was a multidisciplinary effort to describe the status of pulmonary complications occurring within a year after HCT in children, to identify critical gaps in existing knowledge, and to explore avenues for research to address these knowledge gaps to advance care and optimize outcomes.
Recognizing the need for coordinated basic, translational and clinical future research endeavors in this vulnerable and distinctive cohort, Workshop participants arrived at a consensus on the following important focus areas:
The Workshop succeeded in highlighting both the magnitude and complexity of the problem and the pressing need for research in this field, which has the potential to improve outcomes both in children currently receiving HCT as therapy, and in children suffering from a host of other conditions for which HCT holds the promise of lasting cure.