Elsevier

The Journal of Pediatrics

Volume 130, Issue 2, February 1997, Pages 231-239
The Journal of Pediatrics

Lung transplantation for treatment of infants with surfactant protein B deficiency,☆☆,,★★

Presented in abstract form at the Society for Pediatric Research meeting, May 7-11, 1995 (Pediatr Res 1995;37:335A), San Diego, Calif.
https://doi.org/10.1016/S0022-3476(97)70348-2Get rights and content

Abstract

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)

Section snippets

PATIENTS

The clinical presentations of three unrelated term female infants were similar to previous reports: respiratory distress with the need for mechanical ventilation at less than 24 hours of age (SP-B–deficient infants 1, 2, and 3, Table I)4, 5, 6, 11.

. Clinical course after transplantation

Empty CellSP-B–deficient infantsComparison infants
Empty CellInfant 1Infant 2Infant 3C1 *C2†C3†
Age at transplantation (wk)10 (first), 26 (second)99351312
Duration of mechanical ventilation after transplantation (wk)5, 615233
Duration of

Pulmonary function testing and neurodevelopmental progress

To evaluate lung compliance and small airway patency, functional residual capacity, specific respiratory system compliance, and flow at FRC were measured with the Sensormedics model 2600 Pulmonary Cart (Sensormedics, Yorba Linda, Calif.). 16 Lung volume was estimated from serial standard posteroanterior chest radiographs as previously described. 17, 18 Neurodevelopmental progress was assessed with the Bayley Scales of Infant Development, 19 and gross motor function was assessed with the Peabody

Somatic growth, neurodevelopmental progress, lung growth, and pulmonary function testing

At follow-up at least 10 months after operation, two comparison infants and one infant with SP-B deficiency were at or above the 5th percentile for weight, whereas the other two infants were well below the 5th percentile for weight. All five infants were at or below the 10th percentile for length. Head circumference growth was progressing at or above the 5th percentile for all infants (Table II).

. Growth, development, and pulmonary function after transplantation

Empty CellSP-B–deficient infantsComparison

DISCUSSION

Infants with SP-B deficiency can be successfully treated with lung transplantation. The outcomes at 2 years of age of two such infants suggest that somatic and lung growth and cognitive development are maintained despite severe respiratory failure in the pretransplantation period, pulmonary denervation, and immunosuppression. Motor and auditory development may be adversely affected, however, by long-term use of neuromuscular blockade in the pretransplantation interval or by chronic

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  • Cited by (0)

    From the Edward Mallinckrodt Department of Pediatrics, and the Departments of Surgery and Pathology, Washington University School of Medicine and St. Louis Children's Hospital, and the Department of Pathology and the E.A. Doisy Department of Biochemistry and Molecular Biology, St. Louis University School of Medicine, St. Louis, Missouri; the Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, Maryland; and the Department of Pediatrics, University of South Florida School of Medicine, Tampa, Florida.

    ☆☆

    Supported in part by the American Lung Association of Eastern Missouri (A.H.), the American Lung Association (D.E.dM.), National Institute of Health grant No. HL-34748 (D.E.dM.), National Institutes of Health grant No. HL-37591 (H.R.C.), National Institutes of Health grant No. 1P20NS3256801 (F.S.C.), March of Dimes Basil O'Connor Award (L.M.N.), and March of Dimes (A.H., F.S.C.).

    Reprint requests: Aaron Hamvas, MD St. Louis Children's Hospital, Division of Newborn Medicine, One Children's Place, St. Louis, MO 63110.

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