Abstract
Background: A patient with autoimmune PAP with persistent disease underwent 3 WLL, 10 plasmapheresis cycles and further 3 WLL, from October 2004 to May 2007. Despite a substantial clinical and functional benefit, the radiographic appearance showed a partial resolution of lung infiltrates(1). At the beginning of 2010, after a worsening of respiratory conditions, the patient was admitted to inhaled GM-CSF (Sagramostin) therapy as compassionate treatment.
Methods: GM-CSF, dispensed byAkita 2 nebulizer (Vectura),was administered following:250 mcg/day every other week for 12 weeks, then 250 mcg/day on 2 consecutive days every 2 weeks for 6 months. Follow up visits were scheduled at 3, 10, 18, 30 months and after that once a year. Functional and HRCT data and PaO2 were collected.
Results: From the start of the inhalatory therapy the patient no more required WLL. Furthermore we found a significant increase in DLCO%(p=0.013) and FVC%(p=0.023) while FEV1% show a positive trend(Fig.1). No substantial differences in blood gas analysis. The pulmonary involvement at HRCT shows a significant decrease of lung infiltrates(p=0.039) in terms of pathological segments.
Conclusion: These data underscore the utility of inhaled GM-CSF not only in case of progressing disease but also in case of persistence/stabilization, in order to increase response rate.
1.Luisetti et al. Eur Respir J 2009; 33: 1220–1222.
- Copyright ©the authors 2016