An Open-Label Trial of Granulocyte Macrophage Colony Stimulating Factor Therapy for Moderate Symptomatic Pulmonary Alveolar Proteinosis: Study Outcomes
The primary end point was an improvement in oxygenation as assessed by a > 10 mm Hg decrease in the room air alveolar-arterial oxygen gradient (P[A-a]O2). For the patients who received GM-CSF therapy after hospital admission for bilateral lavage (history of two or more WLLs in the prior 4 months), the primary outcome variable was the need for subsequent therapeutic lavage or further hospital admission days (rather than oxygenation). Secondary end points were as follows: (1) improvement in symptoms as assessed by a dyspnea questionnaire, pulmonary function tests, 6-min walk distance with oxygen saturation, and CXR abnormalities; (2) overall tolerability of therapy; (3) requirement for WLL and sustainability of a response as assessed by follow-up telephone interview; and (4) quality of life as measured by the SF-36 survey.
Single frontal CXRs were graded as to the presence and extent of lung opacification using a visual scoring system similar to one described by Remy-Jardin et al. Scores were determined for degree of opacification, extent of opacification, and severity of opacification.