GM-CSF pathway correction in pulmonary alveolar proteinosis

Expert Opinion on Biological Therapy

Volumn 10, Issue 9, 2010, Pages 1357-1365

  Antoniu, Sabina A ^a  

^a GRIGORE T POPA UNIVERSITY OF MEDICINE AND PHARMACY   (Romania)

Author keywords

Alveolar macrophages; Inhaled GM CSF; Pulmonary alveolar proteinosis; Surfactant abnormalities

Indexed keywords

AUTOANTIBODY; GRANULOCYTE MACROPHAGE COLONY STIMULATING FACTOR; RECOMBINANT GRANULOCYTE MACROPHAGE COLONY STIMULATING FACTOR;

ABSENCE OF SIDE EFFECTS; AUTOIMMUNE DISEASE; CLINICAL TRIAL; DRUG DOSE ESCALATION; GENE THERAPY; HISTOPATHOLOGY; HUMAN; LUNG ALVEOLUS MACROPHAGE; LUNG ALVEOLUS PROTEINOSIS; MULTIPLE CYCLE TREATMENT; NEUTROPHIL; NONHUMAN; PROTEIN DEFECT; REVIEW;

ADMINISTRATION, INHALATION; ANIMALS; AUTOANTIBODIES; GRANULOCYTE-MACROPHAGE COLONY-STIMULATING FACTOR; HUMANS; IMMUNOLOGIC FACTORS; PULMONARY ALVEOLAR PROTEINOSIS; SIGNAL TRANSDUCTION; TREATMENT OUTCOME;

EID: 77955541510     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.2010.510507     Document Type: Review

References (68)

1. Rosen SH, Castleman B, Liebow AA. Pulmonary alveolar proteinosis. N Engl J Med 1958;258:1123-1142
2. Trapnell BC, Whitsett JA, Nakata K. Pulmonary alveolar proteinosis. N Engl J Med 2003;349:2527-2539
3. Inoue Y, Trapnell BC, Tazawa R, et al. Characteristics of a large cohort of patients with autoimmune pulmonary alveolar proteinosis in Japan. Am J Respir Crit Care Med 2008;177:752-762
4. De La Fuente AA, Voorhout WF, Demello DE. Congenital alveolar proteinosis in the Netherlands: a report of five cases with immunohistochemical and genetic studies on surfactant apoproteins Pediatr Pathol Lab Med, 17, 221-231
5. Stevens PA, Pettenazzo A, Brasch F, et al. Nonspecific interstitial pneumonia, alveolar proteinosis, and abnormal proprotein trafficking resulting from a spontaneous mutation in the surfactant protein C gene. Pediatr Res 2005;57:89-98
6. Suzuki T, Sakagami T, Rubin BK, et al. Familial pulmonary alveolar proteinosis caused by mutations in CSF2RA. J Exp Med 2008;205:2703-2710
7. Tredano M, Griese M, Brasch F, et al. Mutation of SFTPC in infantile pulmonary alveolar proteinosis with or without fibrosing lung disease. Am J Med Genet A 2004;126A:18-26
8. Buechner HA, Ansari A. Acute silico-proteinosis. A new pathologic variant of acute silicosis in sandblasters, characterized by histologic features resembling alveolar proteinosis. Dis Chest, 1969, 55, 274-278
9. Ruben FL, Talamo TS. Secondary pulmonary alveolar proteinosis occurring in two patients with acquired immune deficiency syndrome. Am J Med 1986;80:1187-1190
10. Cordonnier C, Fleury-Feith J, Escudier E, et al. Secondary alveolar proteinosis is a reversible cause of respiratory failure in leukemic patients. Am J Respir Crit Care Med 1994;149:788-794
11. Tran Van Nhieu J, Vojtek AA, Bernaudin JF, et al. Pulmonary alveolar proteinosis associated with Pneumocystis carinii. Ultrastructural identification in bronchoalveolar lavage in AIDS and immunocompromised non-AIDS patients. Chest 1990;98:801-805
12. Cummings KJ, Donat WE, Ettensohn DB, et al. Pulmonary alveolar proteinosis in workers at an indium processing facility. Am J Respir Crit Care Med 2010;181:458-464
13. Uchida K, Beck DC, Yamamoto T, et al. GM-CSF autoantibodies and neutrophil dysfunction in pulmonary alveolar proteinosis. N Engl J Med 2007;356:567-579
14. Michaud G, Reddy C, Ernst A. Whole-lung lavage for pulmonary alveolar proteinosis. Chest 2009;136:1678-1681
15. Ceruti M, Rodi G, Stella GM, et al. Successful whole lung lavage in pulmonary alveolar proteinosis secondary to lysinuric protein intolerance: a case report. Orphanet J Rare Dis 2007;2:14
16. Wardwell NR Jr, Miller R, Ware LB. Pulmonary alveolar proteinosis associated with a disease-modifying antirheumatoid arthritis drug. Respirology 2006;11:663-665
17. Hodges O, Zar HJ, Mamathuba R, Thomas J. Bilateral partial lung lavage in an infant with pulmonary alveolar proteinosis. Br J Anaesth 2010;104:228-230
18. Nishinakamura R, Wiler R, Dirksen U, et al. The pulmonary alveolar proteinosis in granulocyte macrophage colony-stimulating factor/interleukins 3/5 betac receptor-deficient mice is reversed by bone marrow transplantation. J Exp Med 1996;183:2657-2662
19. Borie R, Debray MP, Laine C, et al. Rituximab therapy in autoimmune pulmonary alveolar proteinosis. Eur Respir J 2009;33:1503-1506
20. Cho K, Nakata K, Ariga T, et al. Successful treatment of congenital pulmonary alveolar proteinosis with intravenous immunoglobulin G administration. Respirology 2006;11(Suppl):S74-7
21. Luisetti M, Rodi G, Perotti C, et al. Plasmapheresis for treatment of pulmonary alveolar proteinosis. Eur Respir J 2009;33:1220-1222
22. Larson RK, Gordinier R. Pulmonary alveolar proteinosis. report of six cases, review of the literature, and formulation of a new theory. Ann Intern Med 1965;62:292-312
23. Singh G, Katyal SL, Bedrossian CW, Rogers RM. Pulmonary alveolar proteinosis. Staining for surfactant apoprotein in alveolar proteinosis and in conditions simulating it. Chest 1983;83:82-86
24. Perez-Gil J. Structure of pulmonary surfactant membranes and films: the role of proteins and lipid-protein interactions. Biochim Biophys Acta 2008;1778(7-8):1676-1695
25. Rugonyi S, Biswas SC, Hall SB. The biophysical function of pulmonary surfactant. Respir Physiol Neurobiol 2008;163:244-255
26. Arellano M, Lonial S. Clinical uses of GM-CSF, a critical appraisal and update. Biologics 2008;2:13-27
27. Dranoff G, Crawford AD, Sadelain M, et al. Involvement of granulocyte-macrophage colony-stimulating factor in pulmonary homeostasis. Science 1994;264:713-716
28. Stanley E, Lieschke GJ, Grail D, et al. Granulocyte/macrophage colony-stimulating factor-deficient mice show no major perturbation of hematopoiesis but develop a characteristic pulmonary pathology. Proc Natl Acad Sci USA 1994;91:5592-5596
29. Yoshida M, Ikegami M, Reed JA, et al. GM-CSF regulates protein and lipid catabolism by alveolar macrophages. Am J Physiol Lung Cell Mol Physiol 2001;280:L379-86
30. Robb L, Drinkwater CC, Metcalf D, et al. Hematopoietic and lung abnormalities in mice with a null mutation of the common beta subunit of the receptors for granulocyte-macrophage colony-stimulating factor and interleukins 3 and 5. Proc Natl Acad Sci USA 1995;92:9565-9569
31. Ikegami M, Ueda T, Hull W, et al. Surfactant metabolism in transgenic mice after granulocyte macrophage-colony stimulating factor ablation. Am J Physiol 1996;270:L650-8
32. Reed JA, Ikegami M, Robb L, et al. Distinct changes in pulmonary surfactant homeostasis in common beta-chain- and GM-CSF-deficient mice. Am J Physiol Lung Cell Mol Physiol 2000;278:L1164-71
33. Celada A, Borras FE, Soler C, et al. The transcription factor PU.1 is involved in macrophage proliferation. J Exp Med 1996;184(1):61-69
34. Iwabuchi H, Kawasaki T, Yamamoto T, et al. Expression of PU.1 and terminal differentiation of alveolar macrophages in newborn rats. Cell Tissue Res 2007;329:71-79
35. Berclaz PY, Shibata Y, Whitsett JA, Trapnell BC. GM-CSF, via PU.1, regulates alveolar macrophage Fcgamma R-mediated phagocytosis and the IL-18/IFN-gamma-mediated molecular connection between innate and adaptive immunity in the lung. Blood 2002;100:4193-4200
36. Shibata Y, Berclaz PY, Chroneos ZC, et al. GM-CSF regulates alveolar macrophage differentiation and innate immunity in the lung through PU.1. Immunity 2001;15:557-567
37. Kitamura T, Tanaka N, Watanabe J, et al. Idiopathic pulmonary alveolar proteinosis as an autoimmune disease with neutralizing antibody against granulocyte/macrophage colony-stimulating factor. J Exp Med 1999;190:875-880
38. Uchida K, Nakata K, Trapnell BC, et al. High-affinity autoantibodies specifically eliminate granulocyte-macrophage colony-stimulating factor activity in the lungs of patients with idiopathic pulmonary alveolar proteinosis. Blood 2004;103:1089-1098
39. Kitamura T, Uchida K, Tanaka N, et al. Serological diagnosis of idiopathic pulmonary alveolar proteinosis. Am J Respir Crit Care Med 2000;162:658-662
40. Bonfield TL, Farver CF, Barna BP, et al. Peroxisome proliferator- activated receptor-gamma is deficient in alveolar macrophages from patients with alveolar proteinosis. Am J Respir Cell Mol Biol 2003;29:677-682
41. Baker AD, Malur A, Barna BP, et al. Targeted PPARgamma deficiency in alveolar macrophages disrupts surfactant catabolism. J Lipid Res 2010;51:1325-1331
42. Barna BP, Malur A, Dalrymple H, et al. A novel 1,25-dihydroxyvitamin D-activin A pathway in human alveolar macrophages is dysfunctional in patients with pulmonary alveolar proteinosis (PAP). Autoimmunity 2009;42:56-62
43. Sakagami T, Beck D, Uchida K, et al. Patient-derived granulocyte/ macrophage colony-stimulating factor autoantibodies reproduce pulmonary alveolar proteinosis in nonhuman primates. Am J Respir Crit Care Med 2010;182:49-61
44. Bonfield TL, Barna BP, John N, et al. Suppression of activin A in autoimmune lung disease associated with anti-GM-CSF. J Autoimmun 2006;26:37-41
45. Trapnell BC, Whitsett JA. GM-CSF regulates pulmonary surfactant homeostasis and alveolar macrophage-mediated innate host defense. Ann Rev Physiol 2002;64:775-802
46. Sergeeva A, Ono Y, Rios R, Molldrem JJ. High titer autoantibodies to GM-CSF in patients with AML, CML and MDS are associated with active disease. Leukemia 2008;22:783-790
47. Ikegami M, Jobe AH, Huffman Reed JA, Whitsett JA. Surfactant metabolic consequences of overexpression of GM-CSF in the epithelium of GM-CSF-deficient mice. Am J Physiol 1997;273:L709-14
48. Huffman JA, Hull WM, Dranoff G, et al. Pulmonary epithelial cell expression of GM-CSF corrects the alveolar proteinosis in GM-CSF-deficient mice. J Clin Invest 1996;97:649-655
49. Zsengeller ZK, Reed JA, Bachurski CJ, et al. Adenovirus-mediated granulocyte-macrophage colony-stimulating factor improves lung pathology of pulmonary alveolar proteinosis in granulocyte-macrophage colony-stimulating factor-deficient mice. Hum Gene Ther 1998;9:2101-2109
50. Kleff V, Sorg UR, Bury C, et al. Gene therapy of betac-deficient pulmonary alveolar proteinosis (betac-PAP): studies in a murine in vivo model. Mol Ther 2008;16(4):757-764
51. Ferlazzo G, Klein J, Paliard X, et al. Dendritic cells generated from CD34+ progenitor cells with flt3 ligand, c-kit ligand, GM-CSF, IL-4, and TNF-alpha are functional antigen-presenting cells resembling mature monocyte-derived dendritic cells. J Immunother 2000;23:48-58
52. Eksioglu EA, Mahmood SS, Chang M, Reddy V. GM-CSF promotes differentiation of human dendritic cells and T lymphocytes toward a predominantly type 1 proinflammatory response. Exp Hematol 2007;35:1163-1171
53. Dranoff G, Jaffee E, Lazenby A, et al. Vaccination with irradiated tumor cells engineered to secrete murine granulocyte-macrophage colony-stimulating factor stimulates potent, specific, and long-lasting anti-tumor immunity. Proc Natl Acad Sci USA 1993;90:3539-3543
54. Fattorossi A, Battaglia A, Pierelli L, et al. Effects of granulocyte-colony-stimulating factor and granulocyte/ macrophage-colony- stimulating factor administration on T cell proliferation and phagocyte cell-surface molecules during hematopoietic reconstitution after autologous peripheral blood progenitor cell transplantation. Cancer Immunol Immunother 2001;49:641-648
55. Reed JA, Ikegami M, Cianciolo ER, et al. Aerosolized GM-CSF ameliorates pulmonary alveolar proteinosis in GM-CSF-deficient mice. Am J Physiol 1999;276:L556-63
56. Seymour JF, Dunn AR, Vincent JM, et al. Efficacy of granulocyte- macrophage colony-stimulating factor in acquired alveolar proteinosis. N Engl J Med 1996;335(25):1924-1925
57. Seymour JF, Begley CG, Dirksen U, et al. Attenuated hematopoietic response to granulocyte-macrophage colony-stimulating factor in patients with acquired pulmonary alveolar proteinosis. Blood 1998;92(8):2657-2667
58. Kavuru MS, Sullivan EJ, Piccin R, et al. Exogenous granulocyte-macrophage colony-stimulating factor administration for pulmonary alveolar proteinosis. Am J Respir Crit Care Med 2000;161:1143-1148
59. Venkateshiah SB, Yan TD, Bonfield TL, et al. An open-label trial of granulocyte macrophage colony stimulating factor therapy for moderate symptomatic pulmonary alveolar proteinosis. Chest 2006;130:227-237
60. Bonfield TL, Kavuru MS, Thomassen MJ. Anti-GM-CSF titer predicts response to GM-CSF therapy in pulmonary alveolar proteinosis. Clin Immunol 2002;105:342-350
61. Seymour JF, Presneill JJ, Schoch OD, et al. Therapeutic efficacy of granulocyte-macrophage colony-stimulating factor in patients with idiopathic acquired alveolar proteinosis. Am J Respir Crit Care Med 2001;163:524-531
62. Schoch OD, Schanz U, Koller M, et al. BAL findings in a patient with pulmonary alveolar proteinosis successfully treated with GM-CSF. Thorax 2002;57:277-280
63. Anderson PM, Markovic SN, Sloan JA, et al. Aerosol granulocyte macrophage-colony stimulating factor: a low toxicity, lung-specific biological therapy in patients with lung metastases. Clin Cancer Res 1999;5:2316-2323
64. Arai T, Hamano E, Inoue Y, et al. Serum neutralizing capacity of GM-CSF reflects disease severity in a patient with pulmonary alveolar proteinosis successfully treated with inhaled GM-CSF. Respir Med 2004;98:1227-1230
65. Tazawa R, Hamano E, Arai T, et al. Granulocyte-macrophage colony-stimulating factor and lung immunity in pulmonary alveolar proteinosis. Am J Respir Crit Care Med 2005;171:1142-1149
66. Wylam ME, Ten R, Prakash UB, et al. Aerosol granulocyte-macrophage colony-stimulating factor for pulmonary alveolar proteinosis. Eur Respir J 2006;27:585-593
67. Robinson TE, Trapnell BC, Goris ML, et al. Quantitative analysis of longitudinal response to aerosolized granulocyte-macrophage colony-stimulating factor in two adolescents with autoimmune pulmonary alveolar proteinosis. Chest 2009;135:842-848
68. Tazawa R, Trapnell BC, Inoue Y, et al. Inhaled granulocyte/ macrophage-colony stimulating factor as therapy of pulmonary alveolar proteinosis. Am J Respir Crit Care Med 2010;181:1345-1354