Pediatric Alveolar Proteinosis Medication

Updated: Jan 16, 2021
  • Author: Danielle M Goetz, MD; Chief Editor: Girish D Sharma, MD, FCCP, FAAP  more...
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Medication Summary

Numerous investigators report the successful use of GM-CSF in adults with PAP. [1] Evidence suggests that GM-CSF leads to therapeutic responses in pediatric patients as well. [46]

A meta-analysis by Khan et al demonstrated effectiveness of GM-CSF therapy. [24]

One case report described successful therapy with intravenous immunoglobulin (IVIG) for a single case of CAP. [47]


Colony-stimulating factors

Class Summary

Seymour et al (2001) reported their experience in treating PAP with GM-CSF. [32] Fourteen patients received GM-CSF at an initial dosage of 5 mcg/kg/d for 6-12 weeks. The alveolar-arterial oxygen gradient, carbon monoxide diffusing capacity, chest CT scans, and exercise test results were serially monitored. Nonresponders underwent a stepwise dose increase; 5 of 14 patients (34%) responded to a dosage of 5 mcg/kg/d, and 1 patient responded to 20 mcg/kg/d. Overall, 43% of patients responded. Among responders, the mean improvement in the difference in partial pressures of oxygen in mixed alveolar gas and mixed arterial blood (A-a DO2) was 23.2 mm Hg. Responses lasted a median of 39 weeks, and the effects were reproducible with the resumption of therapy. No serious adverse effects were noted. GM-CSF–related eosinophilia was predictive of a successful response to therapy.

Another prospective phase II trial of subcutaneous GM-CSF showed that A-a DO2 values improved in 3 of 4 patients treated with 5-9 mcg/kg/d. Values changed from 48.3 ± 20.1 mm Hg at baseline to 18.3 ± 4.2 mm Hg at week 16. [33]

One case report described the successful treatment of a 13-year-old patient who was given inhaled GM-CSF after whole-lung lavage failed. [46]

Sargramostim (Leukine)

GM-CSF stimulates division and maturation of early myeloid and macrophage precursor cells. Reported to increase granulocytes in 48-91% of patients.