Inhaled Granulocyte/Macrophage-Colony Stimulating Factor as Therapy for Pulmonary Alveolar Proteinosis

American Journal of Respiratory and Critical Care Medicine

Volumn 181, Issue 12, 2010, Pages 1345-1354

  Tazawa, Ryushi ^a,o   Trapnell, Bruce C ^b   Inoue, Yoshikazu ^c   Arai, Toru ^c   Takada, Toshinori ^d   Nasuhara, Yasuyuki ^e   Hizawa, Nobuyuki ^e,f   Kasahara, Yasunori ^g   Tatsumi, Koichiro ^g   Hojo, Masayuki ^h   Ishii, Haruyuki ⁱ   Yokoba, Masanori ^j   Tanaka, Naohiko ^j,k   Yamaguchi, Etsuro ^l   Eda, Ryosuke ^m   Tsuchihashi, Yoshiko ⁿ   Morimoto, Konosuke ⁿ   Akira, Masanori ^c   Terada, Masaki ^d   Otsuka, Junji ^c   Ebina, Masahito ^o   Kaneko, Chinatsu ^a   Nukiwa, Toshihiro ^o   Krischer, Jeffrey P ^p   Akazawa, Kohei ^d   Nakata, Koh ^a,h   more..

^a NIIGATA UNIVERSITY MEDICAL AND DENTAL HOSPITAL   (Japan)

^b CINCINNATI CHILDREN S HOSPITAL RESEARCH FOUNDATION   (United States)

^c NATIONAL HOSPITAL ORGANIZATION KINKI CHUO CHEST MEDICAL CENTER   (Japan)

^d NIIGATA UNIVERSITY GRADUATE SCHOOL OF MEDICAL AND DENTAL SCIENCES   (Japan)

^e HOKKAIDO UNIVERSITY   (Japan)

^f UNIVERSITY OF TSUKUBA   (Japan)

^g CHIBA UNIVERSITY   (Japan)

^h NATIONAL CENTER FOR GLOBAL HEALTH AND MEDICINE   (Japan)

ⁱ KYORIN UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^j KITASATO UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^k TOKYO ROSAI HOSPITAL   (Japan)

^l AICHI MEDICAL UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^m NATIONAL SANYO HOSPITAL   (Japan)

ⁿ NAGASAKI UNIVERSITY   (Japan)

^o TOHOKU UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^p UNIVERSITY OF SOUTH FLORIDA   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

AUTOANTIBODY; GRANULOCYTE MACROPHAGE COLONY STIMULATING FACTOR ANTIBODY; ISONIAZID; LEVOFLOXACIN; RECOMBINANT GRANULOCYTE MACROPHAGE COLONY STIMULATING FACTOR; RIFAMPICIN; BIOLOGICAL MARKER;

ABSENCE OF SIDE EFFECTS; ADULT; ARTERIAL OXYGEN TENSION; ARTICLE; CEREBROSPINAL FLUID; CLINICAL ARTICLE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; CYTOLOGY; DIARRHEA; DISEASE COURSE; DRUG EFFICACY; DRUG MEGADOSE; DRUG SAFETY; DRUG WITHDRAWAL; FEMALE; FEVER; FOLLOW UP; HUMAN; HUMAN TISSUE; JAPAN; LOW DRUG DOSE; LUNG ALVEOLUS PROTEINOSIS; LUNG BIOPSY; LUNG CAPACITY; LUNG TUBERCULOSIS; MALE; MULTICENTER STUDY; MULTIPLE CYCLE TREATMENT; OBSERVATIONAL STUDY; OTITIS MEDIA; PHASE 2 CLINICAL TRIAL; PNEUMONIA; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; STOMACH ULCER; THERAPY EFFECT; TREATMENT DURATION; TREATMENT RESPONSE; TUBERCULOUS LYMPHADENITIS; UPPER RESPIRATORY TRACT INFECTION; BLOOD; COHORT ANALYSIS; COMPUTER ASSISTED TOMOGRAPHY; DOSE RESPONSE; ENZYME LINKED IMMUNOSORBENT ASSAY; INHALATIONAL DRUG ADMINISTRATION; LUNG; METHODOLOGY; MIDDLE AGED; PROSPECTIVE STUDY; RADIOGRAPHY; TREATMENT OUTCOME;

ADMINISTRATION, INHALATION; ADULT; BIOLOGICAL MARKERS; COHORT STUDIES; DOSE-RESPONSE RELATIONSHIP, DRUG; ENZYME-LINKED IMMUNOSORBENT ASSAY; FEMALE; GRANULOCYTE MACROPHAGE COLONY-STIMULATING FACTORS, RECOMBINANT; HUMANS; JAPAN; LUNG; MALE; MIDDLE AGED; PROSPECTIVE STUDIES; PULMONARY ALVEOLAR PROTEINOSIS; TOMOGRAPHY, X-RAY COMPUTED; TREATMENT OUTCOME;

EID: 77950628896     PISSN: 1073449X     EISSN: 15354970     Source Type: Journal    
DOI: 10.1164/rccm.200906-0978OC     Document Type: Article

References (29)

1. Rosen SH, Castleman B, Liebow AA. Pulmonary alveolar proteinosis. N Engl J Med 1958;258:1123-1142.
2. Kitamura T, Tanaka N, Watanabe J, Uchida, Kanegasaki S, Yamada Y, Nakata K. Idiopathic pulmonary alveolar proteinosis as an autoimmune disease with neutralizing antibody against granulocyte/macrophage colony-stimulating factor. J Exp Med 1999;190:875-880.
3. Uchida K, Nakata K, Trapnell BC, Terakawa T, Hamano E, Mikami A, Matsushita I, Seymour JF, Oh-Eda M, Ishige I, 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.
4. Trapnell BC, Whitsett JA, Nakata K. Pulmonary alveolar proteinosis. N Engl J Med 2003;349:2527-2539.
5. Ikegami M, Ueda T, Hull W, Whitsett JA, Mulligan RC, Dranoff G, Jobe AH. Surfactant metabolism in transgenic mice after granulocyte macrophage-colony stimulating factor ablation. Am J Physiol 1996;270:L650-L658.
6. Dranoff G, Crawford AD, Sadelain M, Ream B, Rashid A, Bronson RT, Dickersin GR, Bachurski CJ, Mark EL, Whitsett JA, et al. Involvement of granulocyte-macrophage colony-stimulating factor in pulmonary homeostasis. Science 1994;264:713-716.
7. Stanley E, Lieschke GJ, Grail D, Metcalf D, Hodgson G, Gall JA, Maher DW, Cebon J, Sinickas V, Dunn AR. 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.
8. Suzuki T, Sakagami T, Rubin BK, Nogee LM, Wood RE, Zimmerman SL, Smolarek T, Dishop MK, Wert SE, Whitsett JA, et al. Familial pulmonary alveolar proteinosis caused by mutations in CSF2RA. J Exp Med 2008;205:2703-2710.
9. Inoue Y, Trapnell BC, Tazawa R, Arai T, Takada T, Hizawa N, Kasahara Y, Tatsumi K, Hojo M, Ichiwata T, et al. Characteristics of a large cohort of autoimmune pulmonary alveolar proteinosis patients in Japan. Am J Respir Crit Care Med 2008;177:752-762.
10. Wasserman K, Blank N, Fletcher G. Lung lavage (alveolar washing) in alveolar proteinosis. Am J Med 1968;44:611-617.
11. Beccaria M, Luisetti M, Rodi G, Corsico A, Zoia MC, Colato S, Pochetti P, Braschi A, Pozzi E, Cerveri I. Long-term durable benefit after whole lung lavage in pulmonary alveolar proteinosis. Eur Respir J 2004;23:526-531.
12. Seymour JF, Presneill JJ. Pulmonary alveolar proteinosis: progress in the first 44 years. Am J Respir Crit Care Med 2002;166:215-235. (Pubitemid 34765459)
13. Reed JA, Ikegami M, Cianciolo ER, Lu W, Cho PS, Hull W, Jobe AH, Whitsett JA. Aerosolized GM-CSF ameliorates pulmonary alveolar proteinosis in GM-CSF-deficient mice. Am J Physiol 1999;276:L556-L563.
14. Tazawa R, Hamano E, Arai T, Ohta H, Ishimoto O, Uchida K, Watanabe M, Saito J, Takeshita M, Hirabayashi Y, et al. Granulocyte-macrophage colony-stimulating factor and lung immunity in pulmonary alveolar proteinosis. Am J Respir Crit Care Med 2005;171:1142-1149.
15. Nakata T, Inoue Y, Nukiwa T, Tazawa R, Tsuchihashi K, Takada T, Terada M, Kanazawa H, Hizawa N, Trapnell BC. Multicenter phase II trial of inhaled aerosolized granulocyte-macrophage colony-stimulating factor for patients with idiopathic alveolar proteinosis; the prognosis of patients [abstract]. Am J Respir Crit Care Med 2007;175:A497.
16. Uchida K, Beck DC, Yamamoto T, Berclaz PY, Abe S, Staudt MK, Carey BC, Filippi MD, Wert SE, Denson LA, et al. GM-CSF autoantibodies and neutrophil dysfunction in pulmonary alveolar proteinosis. N Engl J Med 2007;356:567-579.
17. Seymour JF, Presneill JJ, Schoch OD, Downie GH, Moore PE, Doyle IR, Vincent JM, Nakata K, Kitamura T, Langton D, 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.
18. Venkateshiah SB, Yan TD, Bonfield TL, Thomassen MJ, Meziane M, Czich C, Kavuru MS. An open-label trial of granulocyte macrophage colony stimulating factor therapy for moderate symptomatic pulmonary alveolar proteinosis. Chest 2006;130:227-237.
19. Coates AL, Dinh L, MacNeish CF, Rollin T, Gagnon S, Ho SL, Lands LC. Accounting for radioactivity before and after nebulization of tobramycin to insure accuracy of quantification of lung deposition. J Aerosol Med 2000;13:169-178.
20. National Cancer Institute. Common Terminology Criteria for Adverse Events, (CTCAE) v3.0. Bethesda: National Cancer Institute; 2006.
21. Akira M, Inoue Y, Yamamoto S, Sakatani M. Non-specific interstitial pneumonia: findings on sequential CT scans of nine patients. Thorax 2000;55:854-859.
22. Wylam ME, Ten R, Prakash UB, Nadrous HF, Clawson ML, Anderson PM. Aerosol granulocyte-macrophage colony stimulating factor for pulmonary alveolar proteinosis. Eur Respir J 2006;27:585-593.
23. Shibata Y, Berclaz PY, Chroneos ZC, Yoshida M, Whitsett JA, Trapnell BC. GM-CSF regulates alveolar macrophage differentiation and innate immunity in the lung through PU.1. Immunity 2001;15:557-567.
24. Yamamoto H, Yamaguchi E, Agata H, Kandatsu N, Komatsu T, Kawai S, Baba K, Awaya T, Nishikomori R, Tsurusawa M, et al. A combination therapy of whole lung lavage and GM-CSF inhalation in pulmonary alveolar proteinosis. Pediatr Pulmonol 2008;43:828-830.
25. Tazawa R, Nakata K, Inoue Y, Nukiwa T. Granulocyte-macrophage colony-stimulating factor inhalation therapy for patients with idiopathic pulmonary alveolar proteinosis: a pilot study; and long-term treatment with aerosolized granulocyte-macrophage colony-stimulating factor: a case report. Respirology 2006;41:S61-S64.
26. Robinson TE, Trapnell BC, Goris ML, Quittell LM, Cornfield DN. 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.
27. Murayama J, Fukuda K, Sato T, Yano H, Ohtsuka M, Yoshizawa Y, Hasegawa S. Pulmonary alveolar proteinosis. Xe-133 scintigraphic findings before and after bronchopulmonary lavage. Clin Nucl Med 1993;18:123-125.
28. Anderson PM, Markovic SN, Sloan JA, Clawson ML, Wylam M, Arndt CA, Smithson WA, Burch P, Gornet M, Rahman E. 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.
29. Markovic SN, Suman VJ, Nevala WK, Geeraerts L, Creagan ET, Erickson LA, Rowland KM Jr, Morton RF, Horvath WL, Pittelkow MR. A dose-escalation study of aerosolized sargramostim in the treatment of metastatic melanoma: an NCCTG Study. Am J Clin Oncol 2008;31:573-579.