LUNG TRANSPLANTATION FOR TREATMENT OF INFANTS WITH SURFACTANT PROTEIN-B DEFICIENCY

Objective: To evaluate lung transplantation for treatment of surfactan t protein B (SP-B) deficiency. Study design: We compared surfactant co mposition and function from pretransplantation and posttransplantation samples of bronchoalveolar lavage fluid, somatic and lung growth, neu rodevelopmental progress, pulmonary function, and pulmonary immunohist ology in 3 infants with SP-B deficiency who underwent bilateral lung t ransplantation at 2 months of age and 3 infants who underwent lung tra nsplantation for other reasons. Results: Two years after transplantati on, the 2 surviving infants with SP-B deficiency exhibited comparable somatic growth and cognitive development to the comparison infants. Al l infants had delays in gross motor development that improved with tim e. Both groups have exhibited normal gas exchange, lung growth, and pu lmonary function. The SP-B-deficient infants have also exhibited norma l SP-B expression and pulmonary surfactant function after lung transpl antation. In two SP-B-deficient infants antibody to SP-B developed. No pathologic consequences of this antibody were identified. Conclusions : Apart from the development of anti-SP-B antibody, the outcomes for S P-B-deficient infants after lung transplantation are similar to those of infants who undergo lung transplantation for other reasons. Lung tr ansplantation offers a successful interim therapy until gene replaceme nt for this disease is available.