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Article ; Online: Nebulised sargramostim in pulmonary alveolar proteinosis.

Livingstone, Cameron / Corallo, Carmela / Siemienowicz, Miranda / Pilcher, David / Stirling, Robert G

British journal of clinical pharmacology

2022  Volume 88, Issue 7, Page(s) 3523–3528

Abstract: Five patients, comprising nine treatment courses of sargramostim use in pulmonary alveolar proteinosis, are described. The prevailing standard of treatment, whole lung lavage (WLL), is highly invasive, resource intensive and carries some procedural risk. ...

Abstract Five patients, comprising nine treatment courses of sargramostim use in pulmonary alveolar proteinosis, are described. The prevailing standard of treatment, whole lung lavage (WLL), is highly invasive, resource intensive and carries some procedural risk. Nebulised recombinant human GM-CSF (sargramostim) offers a pharmacological treatment option, allowing patients to be treated at home, possessing potential advantages in patient experience and wider health resourcing. The majority of reported patients described subjective improvement in symptoms along with radiographical improvement, although this did not translate into significant improvement in pulmonary function testing. Drug scarcity and high drug cost remain potential barriers to accessing this treatment, and so careful patient selection and treatment outcome assessment remain as challenging needs. Incorporating the routine assessment of validated patient symptom scores with objective physiological measures will allow prediction of response to treatment and help guide management. This report describes the largest published experience of sargramostim use in Australia.
MeSH term(s) Australia ; Bronchoalveolar Lavage ; Granulocyte-Macrophage Colony-Stimulating Factor/adverse effects ; Humans ; Pulmonary Alveolar Proteinosis/diagnosis ; Pulmonary Alveolar Proteinosis/drug therapy ; Recombinant Proteins
Chemical Substances Recombinant Proteins ; sargramostim (5TAA004E22) ; Granulocyte-Macrophage Colony-Stimulating Factor (83869-56-1)
Language English
Publishing date 2022-02-28
Publishing country England
Document type Journal Article
ZDB-ID 188974-6
ISSN 1365-2125 ; 0306-5251 ; 0264-3774
ISSN (online) 1365-2125
ISSN 0306-5251 ; 0264-3774
DOI 10.1111/bcp.15266
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