Category: Pulmonary Function

An Open-Label Trial of Granulocyte Macrophage Colony Stimulating Factor Therapy for Moderate Symptomatic Pulmonary Alveolar Proteinosis: Conclusion

The other factor that would argue in favor of a treatment effect for the observed clinical response is that many of these patients required frequent WLL for symptom relief before enrolling in our study, suggesting that they did not have many quiescent periods in their disease activity. GM-CSF therapy was well tolerated, with minor side…

An Open-Label Trial of Granulocyte Macrophage Colony Stimulating Factor Therapy for Moderate Symptomatic Pulmonary Alveolar Proteinosis: Conclusion

Two plausible mechanisms for this observation in our patients are that both of these patients received escalating doses of GM-CSF for 26 weeks and 48 weeks, respectively, which may have overcome the inhibitory effect of the anti-GM-CSF antibody. The second mechanism could be that these two patients had a relatively lower anti-GM-CSF titer (1:3200 and…

An Open-Label Trial of Granulocyte Macrophage Colony Stimulating Factor Therapy for Moderate Symptomatic Pulmonary Alveolar Proteinosis: Long-term Follow-up

The patients were followed up for a mean (± SD) duration of 39 ± 17.3 months. Of the 12 responders, 8 patients (67%) did not require WLL or home oxygen, and 4 patients (33%) required WLL for a mean of two occasions (range, one to five occasions). Of the nine nonresponders, four patients (44%) did…

An Open-Label Trial of Granulocyte Macrophage Colony Stimulating Factor Therapy for Moderate Symptomatic Pulmonary Alveolar Proteinosis: Discussion

The improvement in clinical parameters was not apparent until 8 to 12 weeks after initiation of therapy in our study. This was similar to the study by Seymour et al, in which improvement in P(A-a)O2 was not evident until 4 to 6 weeks, with maximal improvement achieved at approximately 6 to 10 weeks of therapy….

An Open-Label Trial of Granulocyte Macrophage Colony Stimulating Factor Therapy for Moderate Symptomatic Pulmonary Alveolar Proteinosis: Results

Twenty-seven patients with adult idiopathic PAP were screened, and 25 patients were enrolled in the trial. Figure 1 is a flow chart showing the disposition of all screened patients. Circulating anti-GM-CSF antibodies were present in all patients, measured in pretreatment sera as previously described. Among the 25 patients enrolled in the trial, 18 were men…

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…

An Open-Label Trial of Granulocyte Macrophage Colony Stimulating Factor Therapy for Moderate Symptomatic Pulmonary Alveolar Proteinosis: Dosage and Dose Escalation

Patients were treated with recombinant human yeast-derived GM-CSF (Leukine; formerly Immunex Corporation and now Berlex; Seattle, WA) administered subcutaneously once daily for 3 months. Patients were trained to self-administer daily subcutaneous therapy. The starting dose was 250 Mg/d, which was progressively increased to 5 |xg/kg/d for the second month of study, and to 9 |xg/kg/d…

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