Granulocyte-Macrophage Colony–Stimulating Factor and Lung Immunity in Pulmonary Alveolar Proteinosis

Tazawa, Ryushi; Hamano, Emi; Arai, Toru; Ohta, Hiromitsu; Ishimoto, Osamu; Uchida, Kanji; Watanabe, Masato; Saito, Junichi; Takeshita, Miki; Hirabayashi, Yasuhiko; Ishige, Ikuo; Eishi, Yoshinobu; Hagiwara, Koichi; Ebina, Masahito; Inoue, Yoshikazu; Nakata, Koh; Nukiwa, Toshihiro

doi:10.1164/rccm.200406-716oc

Cited by 106 publications

(87 citation statements)

References 28 publications

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“…132, 133 Lin et al found a significant correlation between BALF (but not the serum GM-CSF antibodies) and other severity indicators, and subsequent requirement of therapeutic lung lavage. 134 This could be related to compartmentalization leading to differences in BALF and serum antibody levels.…”

Section: Granulocyte Macrophage Colony Stimulating Factor Autoantibodmentioning

confidence: 99%

Pulmonary Alveolar Proteinosis

Khan

Agarwal

2011

Respir Care

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Section: Granulocyte Macrophage Colony Stimulating Factor Autoantibodmentioning

confidence: 99%

Pulmonary Alveolar Proteinosis

Khan

Agarwal

2011

Respir Care

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“…Continúan siendo motivo de estudio los beneficios del trasplante del pulmón en este tipo de pacientes, razón por la cual su manejo aún lo constituyen las medidas de soporte (17). Así mismo, se requieren estudios adicionales para determinar el beneficio potencial de otras terapias experimentales tales como la plasmaféresis y la inmunoterapia anti -linfocitos B (18). A su vez estos pacientes requieren seguimientos periódicos mínimo cada seis meses, con control radiológico y de LDH cada tres meses en el primer año para descartar recaí-das tempranas.…”

Section: Discussionunclassified

Proteinosis alveolar idiopática. La presentación común de una rara enfermedad

Chamorro

Ñáñez

Pinedo

2013

rev. colomb. neumol.

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Proteinosis alveolar idiopática. La presentación común de una rara enfermedadIdiopathic alveolar proteinosis. The common presentation of a rare disease ResumenLa proteinosis alveolar pulmonar es una rara entidad de etiología aún desconocida que se caracteriza por la acumulación anormal de material lipoproteináceo dentro de los alvéolos. En la actualidad se pueden identificar tres formas de presentación (congénita, secundaria y adquirida), las cuales tienen como común denominador el extenso compromiso pulmonar. Las alteraciones primarias incluyen una disminución en la depuración del surfactante o un incremento en la producción del mismo, alteraciones que implican defectos genéticos que alteran las proteínas que componen al surfactante o al receptor del GM-CSF (por su sigla en inglés, Granulocyte-macrophage colony-stimulating factor). Su presentación adquirida cursa con la presencia de anticuerpos anti GM/CSF, resaltando así el rol de esta sustancia en la homeostasis del surfactante. En esta ocasión, se presenta el caso de una paciente joven con un cuadro de sintomatología respiratoria baja en quien se destaca la escasa correlación entre su apariencia clínica y su extenso compromiso parenquimatoso pulmonar. Palabras clave: proteinosis alveolar pulmonar, fosfolipoproteinosis alveolar pulmonar. AbstractPulmonary alveolar proteinosis (PAP) is a rare condition of unknown etiology that is characterized by an abnormal accumulation of lipoproteinaceus material within the alveoli. Today, three types of presentations have been identified (congenital, secondary and acquired) which have, as a common denominator, an extensive lung involvement. Primary alterations include a decrease in the clearance of surfactant or an increase in the production thereof, alterations involving genetic defects that alter the proteins that comprise the surfactant or the receptor of GM -CSF. The acquired presentation is characterized by the presence of anti -GM-CSF antibodies, thus highlighting the role of this substance in surfactant homeostasis.On this occasion, we present the case of a young female patient with lower respiratory symptoms, in whom, the low correlation between clinical appearance and extensive pulmonary parenchymal compromise is a remarkable feature.

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“…These findings suggested that aerosolized GM-CSF might be a suitable alternative to whole lung lavage, and results from a phase-1 pilot study, one clinical trial, and one report of a retrospective case series of subjects who were treated with aerosolized GM-CSF support this notion. [124][125][126] In the larger study performed by Tazawa et al 125 (35 subjects), subjects inhaled high (125 g twice daily) and low (125 g once daily) doses of recombinant human GM-CSF every other week over 2 sequential 12- Fig. 7.…”

Section: Granulocyte-macrophage Colony-stimulating Factormentioning

confidence: 99%

Aerosolized Medications for Gene and Peptide Therapy

Laube

2015

Respir Care

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Inhalation therapy has matured to include drugs that: (1) deliver nucleic acids that either lead to the restoration of a gene construct or protein coding sequence in a population of cells or suppress or disrupt production of an abnormal gene product (gene therapy); (2) deliver peptides that target lung diseases such as asthma, sarcoidosis, pulmonary hypertension, and cystic fibrosis; and (3) deliver peptides to treat diseases outside the lung whose target is the systemic circulation (systemic drug delivery). These newer applications for aerosol therapy are the focus of this paper, and I discuss the status of each and the challenges that remain to their successful development. Drugs that are highlighted include: small interfering ribonucleic acid to treat lung cancer and Mycobacterium tuberculosis; vectors carrying the normal alpha-1 antitrypsin gene to treat alpha-1 antitrypsin deficiency; vectors carrying the normal cystic fibrosis transmembrane conductance regulator gene to treat cystic fibrosis; vasoactive intestinal peptide to treat asthma, pulmonary hypertension, and sarcoidosis; glutathione to treat cystic fibrosis; granulocyte-macrophage colony-stimulating factor to treat pulmonary alveolar proteinosis; calcitonin for postmenopausal osteoporosis; and insulin to treat diabetes. The success of these new aerosol applications will depend on many factors, such as: (1) developing gene therapy formulations that are safe for acute and chronic administrations to the lung, (2) improving the delivery of the genetic material beyond the airway mucus barrier and cell membrane and transferring the material to the cell cytoplasm or the cell nucleus, (3) developing aerosol devices that efficiently deliver genetic material and peptides to their lung targets over a short period of time, (4) developing devices that increase aerosol delivery to the lungs of infants, (5) optimizing the bioavailability of systemically delivered peptides, and (6) developing peptide formulations for systemic delivery that do not cause persistent cough or changes in lung function.

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Granulocyte-Macrophage Colony–Stimulating Factor and Lung Immunity in Pulmonary Alveolar Proteinosis

Cited by 106 publications

References 28 publications

Pulmonary Alveolar Proteinosis

Pulmonary Alveolar Proteinosis

Proteinosis alveolar idiopática. La presentación común de una rara enfermedad

Aerosolized Medications for Gene and Peptide Therapy

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