Aerosolized GM-CSF Therapy of Pulmonary Alveolar Proteinosis (PAP).

Tazawa, Ryushi; Inoue, Yoshiya; Takada, Toshinori; Arai, Tatsuo; Nasuhara, Yasuyuki; Hizawa, Nobuyuki; Kasahara, Yasunori; Hojo, Masaaki; Yokoba, Masanori; Tanaka, Naomichi; Yamaguchi, Etsuro; Eda, Ryosuke; Tsuchihashi, Yoshiko; Oishi, Kazunori; Tomita, Masaki; Kaneko, Chinatsu; Nukiwa, Toshihiro; Krischer, J.; Trapnell, B.C.; Nakata, Koh

doi:10.1164/ajrccm-conference.2009.179.1_meetingabstracts.a3031

Cited by 1 publication

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“…CT scoring systems have been proposed to enable more quantitative and reproducible evaluation, and may also serve as endpoints in clinical trials. [36][37][38] Several serum biomarkers, including KL-6, LDH, YKL40, CCL-2, CCL-18, CEA, CA-19.9, CYFRA 21-1, NSE, have been shown to correlate variably with disease severity but are not specific and are therefore not used in daily clinical practice. 39…”

Section: Pap: Disease Monitoringmentioning

confidence: 99%

Pulmonary alveolar proteinosis

2020

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PAP is an ultra-rare disease in which surfactant components, that impair gas exchange, accumulate in the alveolae. There are three types of PAP. The most frequent form, primary PAP, includes autoimmune PAP which accounts for over 90% of all PAP, defined by the presence of circulating anti-GM-CSF antibodies. Secondary PAP is mainly due to haematological disease, infections or inhaling toxic substances, while genetic PAP affects almost exclusively children. PAP is suspected if investigation for interstitial lung disease reveals a crazy-paving pattern on chest CT-scan, and is confirmed by a milky looking bronchoalveolar lavage that gives a positive periodic acid-Schiff (PAS) reaction indicating extracellular proteinaceous material. PAP is now rarely confirmed by surgical lung biopsy.Whole lung lavage (WLL) is still the first line treatment, with an inhaled GM-CSF as second line treatment. Inhalation has been found to be better than subcutaneous injections. Other treatments, such as rituximab or plasmapheresis, seem to be less efficient or ineffective. The main complications of PAP are due to infections by standard pathogens (Streptococcus, Haemophilus, and Enterobacteria) or opportunistic pathogens like mycobacteria, Nocardia, Actinomyces, Aspergillus or Cryptococcus are. The clinical course of PAP is unpredictable and spontaneous improvement can occur. The 5-year actuarial survival rate is 95%.

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Section: Pap: Disease Monitoringmentioning

confidence: 99%