Pulmonary Alveolar Proteinosis in Greece-Türkiye-Cyprus: Answers in a Real-Life Comparison.
Pulmonary alveolar proteinosis (PAP) is a rare disease of inappropriate alveolar surfactant accumulation. The study objective was to compare Greece-Türkiye-Cyprus cohorts, followed-up for 20 years.
Data were retrieved retrospectively by chart review.
One hundred and thirty patients, Greece 39, Türkiye 87, Cyprus 4, were included; 115 (89%) autoimmune (a)PAP, 13 (10%) secondary, 1 (1%) hereditary. Abnormal anti-GM-CSF antibody titre was available in 86/115 patients, further analyzed for aPAP. Forty (46%) were male, with median (IQR) age at diagnosis of 40 (31-50) years. At median (IQR) duration of clinical follow-up of 39 (18-78) months, three patients died. Non-survivors had higher rates of cardiovascular disease [67% vs. 5%, p < 0.001], LTOT (67% vs. 13%, p = 0.001), pulmonary fibrosis [67% vs. 5%] and worse functional status at follow-up [FVC% pred 49 (29) vs. 87 (74-97); p = 0.026, DLCO% pred 21 (46) vs. 72 (58-82); p = 0.016]. aPAP patients were stratified by therapy group (no WLL-no i-GM-CSF, WLL alone, i-GMCSF alone and both WLL and iGM-CSF). WLL was more frequently performed in Türkiye and i-GMCSF alone in Greece (p = 0.006). All therapies were effective. No difference was detected on outcome when sole iGM-CSF was compared to WLL alone or in combination with iGM-CSF [deceased/alive 0%/100% vs. 7%/93%, p = 0.491]. Sole iGM-CSF significantly ameliorated clinical and functional parameters of disease severity [DLCO% (p = 0.013), SatO2 (p = 0.002), 6MWT (p = 0.026)]. Across all therapy groups, pulmonary fibrosis eliminated beneficial response to treatment.
This first real-life comparison of aPAP cohorts originating from three countries showed the non-inferiority of sole iGM-CSF treatment. Fibrosis negatively affects response to treatment and survival.
Data were retrieved retrospectively by chart review.
One hundred and thirty patients, Greece 39, Türkiye 87, Cyprus 4, were included; 115 (89%) autoimmune (a)PAP, 13 (10%) secondary, 1 (1%) hereditary. Abnormal anti-GM-CSF antibody titre was available in 86/115 patients, further analyzed for aPAP. Forty (46%) were male, with median (IQR) age at diagnosis of 40 (31-50) years. At median (IQR) duration of clinical follow-up of 39 (18-78) months, three patients died. Non-survivors had higher rates of cardiovascular disease [67% vs. 5%, p < 0.001], LTOT (67% vs. 13%, p = 0.001), pulmonary fibrosis [67% vs. 5%] and worse functional status at follow-up [FVC% pred 49 (29) vs. 87 (74-97); p = 0.026, DLCO% pred 21 (46) vs. 72 (58-82); p = 0.016]. aPAP patients were stratified by therapy group (no WLL-no i-GM-CSF, WLL alone, i-GMCSF alone and both WLL and iGM-CSF). WLL was more frequently performed in Türkiye and i-GMCSF alone in Greece (p = 0.006). All therapies were effective. No difference was detected on outcome when sole iGM-CSF was compared to WLL alone or in combination with iGM-CSF [deceased/alive 0%/100% vs. 7%/93%, p = 0.491]. Sole iGM-CSF significantly ameliorated clinical and functional parameters of disease severity [DLCO% (p = 0.013), SatO2 (p = 0.002), 6MWT (p = 0.026)]. Across all therapy groups, pulmonary fibrosis eliminated beneficial response to treatment.
This first real-life comparison of aPAP cohorts originating from three countries showed the non-inferiority of sole iGM-CSF treatment. Fibrosis negatively affects response to treatment and survival.
Authors
Papiris Papiris, Chousein Chousein, Kallieri Kallieri, Mogulkoc Mogulkoc, Papaioannou Papaioannou, Frangopoulos Frangopoulos, Adamide Adamide, Kolilekas Kolilekas, Gökçe Gökçe, Ocal Ocal, Papakosta Papakosta, Özyürek Özyürek, Tzilas Tzilas, Yalnız Yalnız, Kontonasiou Kontonasiou, Demirdöğen Demirdöğen, Verykakis Verykakis, Ursavaş Ursavaş, Antonogiannaki Antonogiannaki, Deniz Deniz, Apollonatou Apollonatou, Hanta Hanta, Chatzis Chatzis, Levounets Levounets, Olympiou Olympiou, Vandorou Vandorou, Dimeas Dimeas, Daniil Daniil, Roussakis Roussakis, Schams Schams, Monnier Monnier, Rampiadou Rampiadou, Steiropoulos Steiropoulos, Markopoulou Markopoulou, Tzanakis Tzanakis, Gourgoulianis Gourgoulianis, Prountzos Prountzos, Bouros Bouros, Carey Carey, Trapnell Trapnell, Griese Griese, Çetinkaya Çetinkaya, Manali Manali
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