Abstract The efficacy and safety of GM-CSF inhalation therapy for aPAP have been demonstrated in the following clinical trials: (1) a phase 2 trial in Japan (Tazawa et al., Sargramostim, 2010, AJRCCM), (2) PAGE trial (Tazawa et al., Sargramostim, 2019, NEJM), (3) IMPALA (Trapnell et al., Molgramostim, 2020, NEJM), and (4) IMPALA2 (Trapnell et al., Molgramostim, 2025, NEJM). The primary endpoints were the AaDO2 difference or %DLCO change in patients with relative moderate disease after 24 or 48 weeks of inhalation. The former was approximately 5-10 mmHg, and the latter was approximately 10%, indicating a modest therapeutic effect. Cases of relapse have also been observed after clinical trials. In fact, better outcomes can be achieved by combining this treatment with whole lung lavage or by administering it for a longer period. Going forward, it will be necessary to optimize this treatment in terms of: 1) the inhalation period, amount, and device; 2) patient selection for whom therapeutic effects are expected; 3) the relative merits of intermittent versus continuous administration; and 4) treatment goals (conditions with a low likelihood of relapse). These points will be considered using 28 cases of aPAP at our hospital who have been followed for over 30 months. This abstract is funded by: None
Ohkouchi et al. (Fri,) studied this question.
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