Pulmonary Complications of Pediatric Hematopoietic Cell Transplantation. A National Institutes of Health Workshop Summary

Abstract

Approximately 2,500 pediatric hematopoietic cell transplants (HCTs), most of which are allogeneic, are performed annually in the United States for life-threatening malignant and nonmalignant conditions. Although HCT is undertaken with curative intent, post-HCT complications limit successful outcomes, with pulmonary dysfunction representing the leading cause of nonrelapse mortality. To better understand, predict, prevent, and/or treat pulmonary complications after HCT, a multidisciplinary group of 33 experts met in a 2-day National Institutes of Health Workshop to identify knowledge gaps and research strategies most likely to improve outcomes. This summary of Workshop deliberations outlines the consensus focus areas for future research.

Keywords: acute respiratory distress syndrome; hematopoietic cell transplantation; pediatrics; pulmonary complications; respiratory failure.

Figure 1.

Geographic distribution of Workshop participants at the 2-day cross-disciplinary trans–National Institutes of Health (NIH) Workshop to elucidate pulmonary complications presented by the pediatric hematopoietic cell transplant recipient. Sponsored by the National, Heart, Lung and Blood Institute, the Eunice Kennedy Shriver National Institute of Child Health and Human Development, and the National Cancer Institute of the NIH in Bethesda, Maryland, the Workshop was attended by 33 speakers and participants from across the United States and from Vancouver, Canada, and 15 federal participants from across the NIH.

Figure 2.

Organization of Workshop sessions. This figure represents the organization of the 2-day Workshop. Led by a session chair, experts in the various fields succinctly summarized the current state of the art, identified key knowledge gaps, and suggested future research direction for the listed topics addressed in sessions 1–5. In session 6, the chairs presented the summary of their individual sessions, and the group reached consensus on essential knowledge gaps and research strategies. This sixth session was facilitated by the National Heart, Lung, and Blood Institute Program Official, and consensus was attained through candid discussion and informal voting without the use of a more formal consensus building process (e.g., Delphi process). HCT = hematopoietic cell transplant.

Figure 3.

Organization of content centered around the patient receiving hematopoietic cell transplant. This figure represents the broad cross-cutting areas covered during the Workshop, integrated around the transplant recipient. BOOP = bronchiolitis obliterans organizing pneumonia; BOS = bronchiolitis obliterans syndrome; DAH = diffuse alveolar hemorrhage; GVHD = graft-versus-host disease; HCT = hematopoietic cell transplant; IPS = idiopathic pneumonia syndrome; PARDS = pediatric acute respiratory distress syndrome; PERDS = periengraftment respiratory distress syndrome; TMA = thrombotic microangiopathy.

Figure 4.

Knowledge gaps and research opportunities in pulmonary dysfunction after pediatric hematopoietic cell transplantation. *Hematopoietic cell transplant–pediatric acute respiratory distress syndrome bundle includes 1) lung protective strategies, 2) prone positioning, 3) sedation/neuromuscular blockade, 4) aggressive screening and management of cardiac dysfunction, 5) monitoring of renal function and restricted fluid management, 6) antiinflammatory therapies, 7) nutritional supplementation, 8) indications of extracorporeal membrane oxygenation, and 9) palliative care. HCT = hematopoietic cell transplant; PARDS = pediatric acute respiratory distress syndrome.