Lung transplantation for treatment of infants with surfactant protein B deficiency.
OBJECTIVE
To evaluate lung transplantation for treatment of surfactant protein B (SP-B) deficiency.


METHODS
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.
Hamvas
A
A
Edward Mallinckrodt Department of Pediatrics, Washington University School of Medicine, St. Louis, Missouri, USA.
Nogee
L M
LM
Mallory
G B
GB
Spray
T L
TL
Huddleston
C B
CB
August
A
A
Dehner
L P
LP
deMello
D E
DE
Moxley
M
M
Nelson
R
R
Cole
F S
FS
Colten
H R
HR
D000906:Antibodies / Q000097:blood; D001992:Bronchoalveolar Lavage Fluid / Q000737:chemistry; D005260:Female; D006801:Humans; D007165:Immunosuppression Therapy / Q000379:methods; D007223:Infant; D007231:Infant, Newborn; D008168:Lung / Q000473:pathology / Q000503:physiopathology; D016040:Lung Transplantation / Q000276:immunology / Q000473:pathology / Q000502:physiology; D011183:Postoperative Complications / Q000453:epidemiology; D011510:Proteolipids / Q000032:analysis / Q000276:immunology; D011663:Pulmonary Surfactants / Q000032:analysis / Q000172:deficiency* / Q000276:immunology; D012127:Respiratory Distress Syndrome, Newborn / Q000276:immunology / Q000473:pathology / Q000601:surgery*; D017741:Survivors; D016896:Treatment Outcome
D000906:Antibodies; D011510:Proteolipids; D011663:Pulmonary Surfactants