NCT00005900

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 10 (planned)
• Sponsor: Fairview University Medical Center · Academic / Other
• Sex: All
• Age: 0 Years
• Healthy volunteers: Not accepted

Summary

OBJECTIVES: I. Evaluate bronchoalveolar lavage fluid and serum obtained from pediatric patients with storage disorders prior to allogeneic hematopoietic stem cell transplantation (HSCT) for the presence of proinflammatory cytokines and for the production of nitric oxide by alveolar macrophages to identify possible risk factors for pulmonary complications. II. Investigate the underlying mechanism for the development of significant pulmonary complications in these patients during HSCT. III. Evaluate bronchoalveolar lavage fluid and serum obtained from these same patients at the time a pulmonary complication develops post-HSCT, or at 60 days post-HSCT if there has been no pulmonary complications.

Detailed description

PROTOCOL OUTLINE: Patients undergo bronchoscopy with bronchoalveolar lavage (BAL) prior to allogeneic hematopoietic stem cell transplantation (HSCT). ELISA assays for cytokines are performed. Patients are followed post-HSCT for the development of transplant related pulmonary complications. A repeat bronchoscopy with BAL is performed at the time pulmonary complications develop or at day 60 post-HSCT if no complications develop. Cytokine assays are repeated.

Conditions

• I Cell Disease
• Fucosidosis
• Globoid Cell Leukodystrophy
• Adrenoleukodystrophy
• Mannosidosis
• Niemann-Pick Disease
• Pulmonary Complications
• Mucopolysaccharidosis I
• Mucopolysaccharidosis VI
• Metachromatic Leukodystrophy
• Gaucher's Disease
• Wolman Disease

Timeline

• Start date: 1999-08-01
• First posted: 2000-06-05
• Last updated: 2005-06-24

Locations

1 site across 1 country: United States