Lung transplantation for treatment of infants with surfactant protein B deficiency

• Published in: The Journal of pediatrics Vol. 130; no. 2; pp. 231 - 239
• Main Authors: Hamvas, Aaron, Nogee, Lawrence M., Mallory, George B., Spray, Thomas L., Huddleston, Charles B., August, Anna, Dehner, Louis P., deMello, Daphne E., Moxley, Michael, Nelson, Robert, Cole, F.Sessions, Colten, Harvey R.
• Format: Journal Article
• Language: English
• Published: New York, NY Mosby, Inc 01-02-1997; Elsevier
• Subjects: Antibodies - blood; Biological and medical sciences; Bronchoalveolar Lavage Fluid - chemistry; Female; Humans; Immunosuppression - methods; Infant; Infant, Newborn; Lung - pathology; Lung - physiopathology; Lung Transplantation - immunology; Lung Transplantation - pathology; Lung Transplantation - physiology; Medical sciences; Pneumology; Postoperative Complications - epidemiology; Proteolipids - analysis; Proteolipids - immunology; Pulmonary Surfactants - analysis; Pulmonary Surfactants - deficiency; Pulmonary Surfactants - immunology; Respiratory Distress Syndrome, Newborn - immunology; Respiratory Distress Syndrome, Newborn - pathology; Respiratory Distress Syndrome, Newborn - surgery; Respiratory system : syndromes and miscellaneous diseases; Surgery (general aspects). Transplantations, organ and tissue grafts. Graft diseases; Surgery of the respiratory system; Survivors; Treatment Outcome; SP-B; BAL; FRC; Human; Lung disease; Respiratory disease; Indication; Deficiency; Treatment efficiency; Lung; Transplantation; Proteins; Lung function; Pulmonary surfactant; Surgery; Child
• ISSN: 0022-3476; 1097-6833
• DOI: 10.1016/S0022-3476(97)70348-2

Objective: To evaluate lung transplantation for treatment of surfactant protein B (SP-B) deficiency.
Study design: We compared surfactant composition and function from pretransplantation and posttransplantation samples of bronchoalveolar lavage fluid, somatic and lung growth, neurodevelopmental progress, pulmonary function, and pulmonary immunohistology in 3 infants with SP-B deficiency who underwent bilateral lung transplantation at 2 months of age and 3 infants who underwent lung transplantation for other reasons.
Results: Two years after transplantation, the 2 surviving infants with SP-B deficiency exhibited comparable somatic growth and cognitive development to the comparison infants. All infants had delays in gross motor development that improved with time. Both groups have exhibited normal gas exchange, lung growth, and pulmonary function. The SP-B–deficient infants have also exhibited normal SP-B expression and pulmonary surfactant function after lung transplantation. 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 SP-B–deficient infants after lung transplantation are similar to those of infants who undergo lung transplantation for other reasons. Lung transplantation offers a successful interim therapy until gene replacement for this disease is available. (J Pediatr 1997;130:231-9)