Mucopolysaccharidosis I Clinical Trial
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.
;
Primary Purpose: Screening
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