Nota clínicaProteinosis alveolar. Respuesta al tratamiento con factor estimulante de colonias de granulocitos y macrófagos por vía inhaladaResponse to Inhaled Granulocyte-Macrophage Colony-Stimulating Factor in a Patient With Alveolar Proteinosis
Section snippets
Introducción
La proteinosis alveolar pulmonar (PAP) es una rara enfermedad caracterizada por la acumulación en el alvéolo pulmonar de un material lipoproteico derivado del surfactante alveolar, lo que provoca el consiguiente deterioro del intercambio gaseoso. Su incidencia estimada es de 0,36 casos por millón de habitantes, con una prevalencia de 3,7 por millón de habitantes1. La forma de presentación más frecuente es la que ocurre en adultos jóvenes previamente sanos, que desarrollan disnea de esfuerzo
Observación clínica
Mujer de 31 años, exfumadora (consumo acumulado de 13 paquetes/año). Presentaba desde unos 4 meses antes tos seca persistente, disnea progresiva hasta hacerse de grado 2 en la escala del Medical Research Council (MRC)4, febrícula y pérdida de unos 2 kg de peso. En la exploración física se evidenciaban finos crepitantes inspiratorios en ambas bases pulmonares y acropaquías. El resto era normal.
Los resultados de los análisis realizados fueron los siguientes: hemoglobina de 16,9 g/dl, hematócrito
Discusión
Presentamos un caso tratado con GM-CSF por vía inhalada, después de 2 lavados pulmonares totales, con el que se obtuvo muy buena respuesta clínica y en el intercambio gaseoso, lo que permitió retirar la oxigenoterapia domiciliaria.
La PAP se caracteriza por la acumulación en el alvéolo pulmonar de un material lipoproteico derivado del surfactante alveolar. No hay inflamación en la vía aérea y la arquitectura pulmonar se encuentra conservada6. Se sabe que su patogenia está relacionada con una
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Cited by (14)
Assay system development to measure the concentration of sargramostim with high specificity in patients with autoimmune pulmonary alveolar proteinosis after single-dose inhalation
2018, Journal of Immunological MethodsCitation Excerpt :Based on the etiology, several clinical trials of GM-CSF inhalation have been conducted with variable response rates ranging from 40 to 62%. From 2004 to 2008, 35 patients have completed 24 weeks of GM-CSF inhalation, of whom 23 cases responded with a mean reduction in the alveolar–arterial gradient (AaDO2) of 12.3 mm Hg (Tazawa et al., 2010; Arai et al., 2004; Price et al., 2006; Yamamoto et al., 2008; Robinson et al., 2009; Rodriguez Portal et al., 2009). Currently, an investigator-initiated clinical trial of sargramostim inhalation therapy is being carried out for the purpose of pharmaceutical approval.
Treatment of Adult Primary Alveolar Proteinosis
2015, Archivos de BronconeumologiaRespiratory diseases registries in the national registry of rare diseases
2014, Archivos de BronconeumologiaCitation Excerpt :The total number of cases in Spain is unknown. The most significant results from national and international series are summarized in Table 3.25–34 The Spanish Alveolar Proteinosis Registry (REPA) is an integral part of the SEPAR PII-EPID and its consultant committee consists of 5 pulmonologists.
Emerging inhalation aerosol devices and strategies: Where are we headed?
2014, Advanced Drug Delivery ReviewsCitation Excerpt :In a single-patient case study, a patient received nebulised GM-CSF (sargramostim) treatment when marked deterioration in gas exchange was observed after two lavages. A positive clinical response and improvements in gas exchange were exhibited with no adverse effects after 1 year of treatment [176]. A multicentre, self-controlled phase II trial conducted in Japan also found that nebulised GM-CSF (leukine) therapy was safe and effective in treating pulmonary alveolar proteinosis [175].
Effectiveness of granulocyte-macrophage colony-stimulating factor therapy in autoimmune pulmonary alveolar proteinosis: A meta-analysis of observational studies
2012, ChestCitation Excerpt :Our initial database search retrieved 1,585 citations, of which 1,580 were excluded because they did not meet our inclusion criteria (Fig 1). Fourteen studies (17 patients) were single-patient case reports/small case series, and these were not included in the analysis (Table 1).10,18–30 Initially, six observational studies meeting our inclusion criteria were included in the analysis.31–36
The Clinical View through the Archives: The Clinical Notes of 2009
2010, Archivos de Bronconeumologia