Pediatric Alveolar Proteinosis Treatment & Management

  • Author: Anne E Stone, MD; Chief Editor: Michael R Bye, MD   more...
 
Updated: Nov 23, 2011
 

Medical Care

The appropriate management depends on the patient's age at presentation, the severity of symptoms, and the anticipated course of the disease. Any predisposing conditions (eg, malignancy, infection) should be treated because resolution of the primary condition may lead to remission of pulmonary alveolar proteinosis (PAP). Reports describe spontaneous remission of primary PAP without medical intervention.[1, 10] Treatment of congenital PAP is often difficult, particularly in the long run.[21]

  • Mechanical ventilation: Mechanical ventilation is necessary in children with congenital PAP. No reports show any benefit from the use of high-frequency oscillatory ventilation (HFOV) or other unconventional forms of mechanical ventilation.[3]
  • Gene therapy: Because congenital PAP is a single-gene defect, it may be a candidate disease for gene therapy.
  • GM-CSF therapy: In addition, several trials of GM-CSF therapy for acquired PAP have shown encouraging results.[22, 23]
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Surgical Care

The mainstay of therapy in PAP is whole-lung lavage. Patients who undergo lavage during the course of their illness have improved survival rates. In addition, 84% of the published cases reported clinical, physiologic, and radiographic improvements after initial therapeutic lavage.[10, 24] Response rates substantially differ when patients are divided into cohorts by age. Patients younger than 20 years old have a 58% response compared with 90% patients older than 40 years.[10]

The mechanism of improvement is unknown but is presumed to be the removal of surfactant buildup or minimizing the effect of macrophage dysfunction. Successful treatment with lobar lavage with fiberoptic bronchoscopy is also reported.

Other surgical options include extracorporeal membrane oxygenation (ECMO) and lung transplantation.[2, 25]

  • Lung lavage
    • In brief, the procedure involves single-lung ventilation while the contralateral lung is lavaged with sodium chloride solution. A double-lumen endotracheal tube (ETT) is used in older children to allow for simultaneous ventilation of one lung and lavage of the contralateral lung with the patient under general anesthesia.[26]
    • Isotonic sodium chloride solution with or without heparin is generally instilled into the lungs. The patient is ventilated with 100% oxygen, and the dependent lung is filled with 3-5 mL/kg of fluid and drained. Lavage is repeated until no sediment material is obtained.
    • The lungs retain variable amounts of fluid. Chest percussion is reported to improve the yield of material. The patient should be intermittently suctioned through the ETT after the procedure to remove any residual fluid.
    • Serum electrolyte levels should be monitored because fluid fluxes may cause electrolyte imbalances.
    • The use of whole-lung lavage is less well established in young infants and newborns than in others primarily because of the technical difficulties associated with passing a necessarily large ETT through a small glottis. However, the success rate of this procedure is described in infants as small as 5 kg. In smaller infants, whole-lung lavage performed while the infant is receiving cardiopulmonary bypass or ECMO is reported.
  • ECMO: ECMO may provide a bridge to lung transplantation or definitive lung lavage in patients who are either too critically ill or too small to undergo lavage.
  • Lung transplantation
    • At this time, the only definitive therapy for CAP is bilateral lung transplantation.[2]
    • PAP has recurred in lungs transplanted into adults.[27]
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Consultations

  • CAP
    • Consult a neonatologist and a pulmonologist when a patient has congenital PAP. An opinion from or a referral to a center with expertise in neonatal lung transplantation is required when this option is being considered.
    • Consultation with a geneticist should be offered to parents of a child with congenital PAP. Antenatal screening for this condition is now possible.
  • Acquired PAP
    • For the older child, consultation with a pulmonologist is mandatory.
    • The opinions of an immunologist, an infectious disease specialist, or a hematologist may also be necessary, depending on clinical findings and suspicion for secondary PAP.
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Diet

No specific diet is necessary. However, as in any chronic disease, attention should be paid to the provision of sufficient calories to maintain adequate growth. Young infants with feeding difficulties due to dyspnea may require feeding through a nasogastric or gastrostomy tube. When prescribing a high-calorie diet, ensure that the carbohydrate load is not excessive because this may exacerbate respiratory difficulties due to a high respiratory quotient and subsequent high CO2 burden.

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Activity

In general, the patient's degree of dyspnea limits his or her activity. No limitations on activity are necessary.

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Contributor Information and Disclosures
Author

Anne E Stone, MD  Fellow, Division of Pediatric Pulmonary Medicine, Columbia University Medical Center, Children's Hospital of New York-Presbyterian

Anne E Stone, MD is a member of the following medical societies: American Academy of Pediatrics, American College of Chest Physicians, and American Thoracic Society

Disclosure: Nothing to disclose.

Coauthor(s)

Michael R Bye, MD  Professor of Clinical Pediatrics, Division of Pulmonary Medicine, Columbia University College of Physicians and Surgeons; Attending Physician, Pediatric Pulmonary Medicine, Morgan Stanley Children's Hospital of New York Presbyterian, Columbia University Medical Center

Michael R Bye, MD is a member of the following medical societies: American Academy of Pediatrics, American College of Chest Physicians, and American Thoracic Society

Disclosure: Nothing to disclose.

Specialty Editor Board

Susanna A McColley, MD  Professor of Pediatrics, Northwestern University, The Feinberg School of Medicine; Director of Cystic Fibrosis Center, Head, Division of Pulmonary Medicine, Children's Memorial Medical Center of Chicago

Susanna A McColley, MD is a member of the following medical societies: American Academy of Pediatrics, American College of Chest Physicians, American Sleep Disorders Association, and American Thoracic Society

Disclosure: Genentech Honoraria Speaking and teaching; Genentech Honoraria Consulting; Boston Scientific Consulting fee Consulting; Gilead Honoraria Speaking and teaching; Caremark Consulting fee Consulting; Vertex Pharmaceuticals Honoraria Speaking and teaching

Mary L Windle, PharmD  Adjunct Associate Professor, University of Nebraska Medical Center College of Pharmacy; Editor-in-Chief, Medscape Drug Reference

Disclosure: Nothing to disclose.

Charles Callahan, DO  Professor, Deputy Chief of Clinical Services, Walter Reed Army Medical Center

Charles Callahan, DO is a member of the following medical societies: American Academy of Pediatrics, American College of Chest Physicians, American College of Osteopathic Pediatricians, American Thoracic Society, Association of Military Surgeons of the US, and Christian Medical & Dental Society

Disclosure: Nothing to disclose.

Mary E Cataletto, MD  Director of Children's Sleep Services, Winthrop Sleep Disorders Center, Mineola, NY; Professor of Clinical Pediatrics, State University of New York at Stony Brook, Stony Brook, NY

Mary E Cataletto, MD is a member of the following medical societies: American Academy of Pediatrics and American College of Chest Physicians

Disclosure: Shering Plough Pharmaceuticals Honoraria Consulting

Chief Editor

Michael R Bye, MD  Professor of Clinical Pediatrics, Division of Pulmonary Medicine, Columbia University College of Physicians and Surgeons; Attending Physician, Pediatric Pulmonary Medicine, Morgan Stanley Children's Hospital of New York Presbyterian, Columbia University Medical Center

Michael R Bye, MD is a member of the following medical societies: American Academy of Pediatrics, American College of Chest Physicians, and American Thoracic Society

Disclosure: Nothing to disclose.

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