Abstract:
BACKGROUND:Treatment of patients with Cystic Fibrosis homozygous for the Phe508del gene, with Lumacaftor /Ivacaftor (LUM/IVA) improves outcomes in patients with FEV1 > 40% predicted. We set out to observe the most sensitive clinical measure that would change with treatment in terms of exercise capacity or lung function in adults with severe lung disease as defined by an FEV1 < 40% predicted when clinically stable. METHODS:10 adults homozygous for the Phe508del received LUM/IVA. We assessed; six minute walk test (6MWT), spirometry, gas transfer (DLCO), plethysmography, and nitrogen multiple breath washout (MBW) at baseline, 4, 12, 24 and 52 weeks. Comparison was made with 10 matched historical controls that had been observed over 12 months. RESULTS:There was a significant improvement in 6MWT by 4 weeks of treatment; with a mean increase of 78 m (SD 62.3) and this increased to 118.1 m (SD 80.9) (ANOVA p = 0.006) by 52 weeks. Significant improvements were also seen in the resting heart rate and the oxygen saturation (SaO2) after 6 min walking. A significant improvement was not seen in FEV1 though until 24 weeks, though this was maintained at 52 weeks (ANOVA, p = 0.0004). There were no significant differences seen in the MBW or DLCO. After 12 months treatment with LUM/IVA, in comparison to historical controls; the 6MWT increased by 118 m (SD 80.9), but fell in the controls - 61.3 m (SD 31.1). FEV1; LUM/IVA led to an increase of 0.398 L/min, compared to a fall in the controls - 0.18 (SD 0.2). CONCLUSION:In adults homozygous for Phe508del with severe disease, treatment with LUM/IVA results in a clinically significant improvement in 6MWT that was evident at 4 weeks and maintained at 52 weeks. Improvement in exercise tolerance is an important outcome to consider in those with more severe airways disease. TRIAL REGISTRATION:This was an observational trial conducted on individuals who became eligible to receive LUM/IVA. All investigations were carried out as part of routine clinical care. The trial was registered in retrospect on the 13/5/2019 on the Australian New Zealand Clinical Trials registry; ACTRN12619000708156 .
journal_name
BMC Pulm Medjournal_title
BMC pulmonary medicineauthors
Wark PAB,Cookson K,Thiruchelvam T,Brannan J,Dorahy DJdoi
10.1186/s12890-019-0866-ysubject
Has Abstractpub_date
2019-06-17 00:00:00pages
106issue
1issn
1471-2466pii
10.1186/s12890-019-0866-yjournal_volume
19pub_type
杂志文章abstract::Following publication of the original article [1], the author flagged aspects of the article that affected readability of some of the article's scientific content. ...
journal_title:BMC pulmonary medicine
pub_type: 杂志文章,已发布勘误
doi:10.1186/s12890-018-0717-2
更新日期:2018-11-07 00:00:00
abstract:BACKGROUND:Selection bias is a systematic error in epidemiologic studies that may seriously distort true measures of associations between exposure and disease. Observational studies are highly susceptible to selection bias, and researchers should therefore always examine to what extent selection bias may be present in ...
journal_title:BMC pulmonary medicine
pub_type: 杂志文章
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abstract:BACKGROUND:Sarcoidosis is a systemic granulomatous disease caused by CD4+ cell-dominant inflammation. Meanwhile, diffuse panbronchiolitis is a chronic inflammatory respiratory disease predominantly caused by CD8+ lymphocytes and neutrophils. Herein, we report a rare case of sarcoidosis in which the clinical presentatio...
journal_title:BMC pulmonary medicine
pub_type: 杂志文章
doi:10.1186/s12890-020-1117-y
更新日期:2020-03-30 00:00:00
abstract:BACKGROUND:Technology offers opportunities to improve healthcare, but little is known about Internet use by COPD patients. We tested two hypotheses: Internet access is associated with socio-demographic disparities and frequency of use is related to perceived needs. METHODS:We analyzed data from a 2007-2008 national co...
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pub_type: 杂志文章
doi:10.1186/1471-2466-14-66
更新日期:2014-04-22 00:00:00
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更新日期:2020-10-29 00:00:00
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journal_title:BMC pulmonary medicine
pub_type: 杂志文章,meta分析,评审
doi:10.1186/s12890-016-0315-0
更新日期:2016-11-24 00:00:00
abstract:BACKGROUND:The evidence regarding the association between lung cancer and occupational exposure to cement is controversial. This study investigated causes of deaths from cancer of respiratory tract among cement workers. METHODS:The deaths of the Greek Cement Workers Compensation Scheme were analyzed covering the perio...
journal_title:BMC pulmonary medicine
pub_type: 杂志文章
doi:10.1186/1471-2466-12-30
更新日期:2012-06-27 00:00:00
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journal_title:BMC pulmonary medicine
pub_type: 杂志文章
doi:10.1186/s12890-015-0004-4
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journal_title:BMC pulmonary medicine
pub_type: 杂志文章
doi:10.1186/s12890-019-0784-z
更新日期:2019-01-24 00:00:00
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pub_type: 杂志文章
doi:10.1186/1471-2466-14-122
更新日期:2014-07-29 00:00:00
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journal_title:BMC pulmonary medicine
pub_type: 杂志文章
doi:10.1186/1471-2466-14-177
更新日期:2014-11-15 00:00:00
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pub_type: 杂志文章
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更新日期:2018-05-18 00:00:00
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pub_type: 杂志文章
doi:10.1186/s12890-020-01361-x
更新日期:2020-12-07 00:00:00
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pub_type: 杂志文章,多中心研究
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更新日期:2017-04-26 00:00:00
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pub_type: 杂志文章
doi:10.1186/s12890-020-1143-9
更新日期:2020-04-20 00:00:00
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pub_type: 杂志文章
doi:10.1186/s12890-020-01254-z
更新日期:2020-08-12 00:00:00
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journal_title:BMC pulmonary medicine
pub_type: 杂志文章
doi:10.1186/1471-2466-9-35
更新日期:2009-07-19 00:00:00
abstract::An amendment to this paper has been published and can be accessed via the original article. ...
journal_title:BMC pulmonary medicine
pub_type: 杂志文章,已发布勘误
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更新日期:2018-07-31 00:00:00
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pub_type: 杂志文章
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journal_title:BMC pulmonary medicine
pub_type: 杂志文章,随机对照试验
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更新日期:2012-09-19 00:00:00
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pub_type: 杂志文章
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更新日期:2020-10-20 00:00:00
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journal_title:BMC pulmonary medicine
pub_type: 杂志文章
doi:10.1186/1471-2466-3-2
更新日期:2003-09-08 00:00:00
abstract:BACKGROUND:Improvement in health-related quality of life (HRQL) has been suggested as an alternative treatment goal of non-tuberculous mycobacterial pulmonary disease (NTM-PD). This study was performed to elucidate the longitudinal changes in HRQL using St. George's Respiratory Questionnaire (SGRQ) among patients with ...
journal_title:BMC pulmonary medicine
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更新日期:2020-05-07 00:00:00
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journal_title:BMC pulmonary medicine
pub_type: 杂志文章
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更新日期:2016-05-10 00:00:00
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journal_title:BMC pulmonary medicine
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更新日期:2014-03-11 00:00:00
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journal_title:BMC pulmonary medicine
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更新日期:2015-07-10 00:00:00
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journal_title:BMC pulmonary medicine
pub_type: 杂志文章
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更新日期:2018-04-03 00:00:00
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journal_title:BMC pulmonary medicine
pub_type: 杂志文章
doi:10.1186/s12890-019-1039-8
更新日期:2019-12-30 00:00:00