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

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

Gaucher's disease, I cell disease, Niemann-Pick disease, Wolman disease, adrenoleukodystrophy, disease-related problem/condition, fucosidosis, genetic diseases and dysmorphic syndromes, globoid cell leukodystrophy, inborn errors of metabolism, mannosidosis, metachromatic leukodystrophy, mucopolysaccharidosis, mucopolysaccharidosis I, mucopolysaccharidosis VI, oncologic disorders, pulmonary complications, rare disease, sphingolipidoses

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.

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.

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United States