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

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

• Registration Number: NCT00005900
• Lead Sponsor: Fairview University Medical Center

Brief 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.

Study Timeline

• Study Start: 1999-08
• Study First Submitted: 2000-06-02
• Study First Submitted QC: 2000-06-02
• Study First Posted: 2000-06-05
• Status Verified: 2003-10
• Last Update Submitted: 2005-06-23
• Last Update Posted: 2005-06-24

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Trial Locations

Locations (1)

Fairview University Medical Center; 🇺🇸 Minneapolis, Minnesota, United States