5PSQ-012 Case report: Inhaled granulocyte-macrophage colony-stimulating factor for mild-to-moderate autoimmune pulmonary alveolar proteinosis – 24 months of follow-up
Background and ImportanceAutoimmune pulmonary alveolar proteinosis (aPAP) is a disease caused by IgG antibodies against granulocyte-macrophage colony-stimulating factor (GM-CSF). The treatment of choice is bronchoalveolar lavage (BAL).Aim and ObjectivesTo present aPAP evolution during 24 months of t...
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Veröffentlicht in: | European journal of hospital pharmacy. Science and practice 2023-03, Vol.30 (Suppl 1), p.A114-A115 |
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Zusammenfassung: | Background and ImportanceAutoimmune pulmonary alveolar proteinosis (aPAP) is a disease caused by IgG antibodies against granulocyte-macrophage colony-stimulating factor (GM-CSF). The treatment of choice is bronchoalveolar lavage (BAL).Aim and ObjectivesTo present aPAP evolution during 24 months of treatment with inhaled GM-CSF.Material and MethodsA 37-year-old male diagnosed with aPAP in April 2014 has required 3 BAL (October 2014, February 2016 and March 2019). In the last BAL, he developed major complications that required admission to the intensive care unit. In October 2019, he presented a new worsening, so off-label treatment with sargramostim (250µg inhaled every 12 hours for 7 days every other week) was chosen.The pharmacy service prepared a favorable report on off-label treatment and requested drug from regulatory agency. In the pharmaceutical care consultation, the administration technique, stability, dosage regimen, storage (2-8°C) were explained and doubts were resolved.ResultsThe clinical and functional evolution of the ventilatory parameters and the six-minute walk test are shown in the table 1.Abstract 5PSQ-012 Table 1 7 months after third BAL 3 months treatment 6 months treatment 18 months treatment 24 months treatment Pulmonary function test Forced expiratory volume in one second (FEV1) ml (%) 2630(63) 2690(72) 3080(75) 2900(71) 3030(75) Transfer factor of the lung for carbon monoxide, corrected (cTLCO) (%) 37 53 52 74 68 Transfer coefficient of the lung for carbon monoxide, corrected (cKCO) (%) 59 69 64 85 74 Six-minute walk test Theoretical distance (m) 614.52 435.71 627.06 619.17 Heart rate minute 6 (beats per minute) 136 108 108 105 Oxygen saturation minute 6 (%) 89 89 93 91 The figure 1 shows the radiological evolution (chest X-ray) from the situation before third BAL (1), further worsening after 7 months after third BAL (2), improvement after 3 months of treatment with inhaled GM-CSF (3) and stability after 18 months of treatment (4).Abstract 5PSQ-012 Figure 1After 24 months of treatment, the patient has not presented any adverse events and maintains an excellent response with significant improvement in gas exchange, which has allowed home oxygen therapy to be withdrawn.Conclusion and RelevanceIn conclusion, our case supports that inhaled GM-CSF has been safe and effective in the treatment of aPAP and represents a therapeutic option after resistance or contraindication to BAL.References and/or AcknowledgementsConflict of InterestNo co |
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ISSN: | 2047-9956 2047-9964 |
DOI: | 10.1136/ejhpharm-2023-eahp.239 |