Response to Inhaled Granulocyte-Macrophage Colony-Stimulating Factor in a Patient With Alveolar Proteinosis

Response to inhaled granulocyte‐macrophage colony‐stimulating factor in patient with mild‐to‐moderate autoimmune pulmonary alveolar proteinosis—24 months of follow‐up

“…In the case presented by Rodríguez Portal et al, a 32-year-old woman who started therapy with inhaled GM-CSF after two whole lung lavages, no adverse effects appeared after 12 months of treatment, and dyspnea went from grade 3 to 1 on the Medical Research Council scale with improvement in FVC from 37% to 51% and in DLCO from 23% to 51%, with withdrawal of home oxygen therapy. 6 Gajewska et al also reported a case of a 14-year-old boy treated with inhaled GM-CSF after three whole lung lavages with clinical improvement after 17 months of treatment: FVC went from 70% to 79% and DLCO from 54% to 74%, with no adverse effects. 8 As an alternative to inhaled GM-CSF therapy, after failure or contraindication of whole lung lavage, other treatments such as subcutaneous GM-CSF 250 mcg/day have been tried with similar response rates; however, unlike inhaled therapy, some mild adverse effects such as fever, fatigue, headache, and injection site complications are included.…”

“…In the case presented by Rodríguez Portal et al, a 32‐year‐old woman who started therapy with inhaled GM‐CSF after two whole lung lavages, no adverse effects appeared after 12 months of treatment, and dyspnea went from grade 3 to 1 on the Medical Research Council scale with improvement in FVC from 37% to 51% and in DLCO from 23% to 51%, with withdrawal of home oxygen therapy 6 . Gajewska et al also reported a case of a 14‐year‐old boy treated with inhaled GM‐CSF after three whole lung lavages with clinical improvement after 17 months of treatment: FVC went from 70% to 79% and DLCO from 54% to 74%, with no adverse effects 8 …”

“…3 There are currently no randomized clinical trials investigating the use of inhaled recombinant human GM-CSF in aPAP, 5 so evidence is supported by some observational studies and meta-analyses. 3,[6][7][8] However, the duration of treatment in the literature is variable, and an optimal guideline has not yet been established.…”