Clinical Trial: Early Detection and Management of Bronchiolitis Obliterans Syndrome Following Pediatric Hematopoietic Stem Cell Transplantation

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Early Detection and Management of Bronchiolitis Obliterans Syndrome Following Pediatric Hematopoietic Stem Cell Transplantation

Brief Summary: This study aims to determine whether or not early spirometric detection and management of obstructive lung disease with combined fluticasone/azithromycin/montelukast therapy (FAM) can attenuate declining lung function, prevent the development of bronchiolitis obliterans, and improve patient outcomes following hematopoietic stem cell transplant.

Detailed Summary:

Bronchiolitis obliterans syndrome (BOS) is an inflammatory condition of the lungs that leads to obstructive physiology, irreversible fibrosis of terminal bronchioles, and obliteration of the small airways. In both children and adults, the prevalence of BOS is approximately 6% in those with chronic graft-vs-host disease (cGVHD), although this may be a gross underestimation given current diagnostic guidelines. Once diagnosed, the prognosis is extremely unfavorable. BOS carries a mortality rate of approximately 40-60%, with a five year survival rate of 13%.

Early on, BOS is symptomatically silent. Once symptoms are present, there is a high likelihood of irreversible disease regardless of the combination of immune suppression used. Given these circumstances, early diagnosis is of utmost importance, and can be characterized by an isolated and subclinical decline in lung function. Recent studies have suggested that early decline in lung function on pulmonary function testing (PFT) may be representative of developing BOS.

Due to the lack of consistent screening and diagnostic criteria, many patients with evolving BOS elude a timely diagnosis, thereby jeopardizing their chance of survival. In response, several experts have recommended frequent PFT screening and a modified, less stringent set of diagnostic criteria with the goal of establishing earlier diagnosis and timely intervention.

Traditionally, treatment of BOS has included aggressive immunosuppression, leaving patients at risk for life-threatening invasive infections, multi-system co-morbidities, and the threat of lung transplantation. Recent studies have demonstrated that early management with agents such as inhaled corticosteroids (ICS), macrolides, and leukotriene receptor antagonists (LTRA) can lead to improvements in bo
Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago

Current Primary Outcome:

  • Lung function [ Time Frame: 2 years ]
    Change in lung function at 12 and 24 months post-HSCT
  • Survival [ Time Frame: 2-4 years ]
    Overall survival at 2 years post-HSCT


Original Primary Outcome: Same as current

Current Secondary Outcome: Risk factor assessment [ Time Frame: 2 years ]

To identify risk factors for the development of BOS


Original Secondary Outcome: Same as current

Information By: Ann & Robert H Lurie Children's Hospital of Chicago

Dates:
Date Received: March 2, 2017
Date Started: April 2015
Date Completion: April 2019
Last Updated: March 2, 2017
Last Verified: March 2017