Rare ILD/DPLD最新文献

{"title":"Perception of the caregivers of patients with idiopathic pulmonary fibrosis: results of the French national survey, RESPIR","authors":"S. Marchand-Adam, A. Bourdin, E. Bergot, H. Nunes, B. Wallaert, G. Prévôt, V. Cottin","doi":"10.1183/13993003.congress-2019.pa1402","DOIUrl":"https://doi.org/10.1183/13993003.congress-2019.pa1402","url":null,"abstract":"","PeriodicalId":267660,"journal":{"name":"Rare ILD/DPLD","volume":"78 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2019-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"131791554","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prognostic impact and clinical characteristics of Interstitial Pneumonia with Autoimmune features (IPAF) in a multidisciplinary setting","authors":"C. Carrubba, L. Cavagna, E. Marasco, G. Zanframundo, A. Valentini, V. Morandi, V. Codullo, Lorenzo Volpiano, F. Motta, C. Montecucco, F. Meloni","doi":"10.1183/13993003.congress-2019.pa3674","DOIUrl":"https://doi.org/10.1183/13993003.congress-2019.pa3674","url":null,"abstract":"Background: Clinical evolution and optimal management of IPAF are still undefined and need prospective assessment. Objectives: to define the characteristics and evolution of IPAF patients in a multidisciplinary setting. Methods: IPAF pts referring to multidisciplinary Rheumatology/Pneumology outpatient clinic. We excluded patients with diagnosis of Anti-synthetase Sd. Data were retrospectively collected. Results: we analyzed the first 25 pts (19 F, 76%, 6 M, 24%), with a median onset age of 67 years (IQR, 59-74) and follow-up of 32 months (IQR 22-69). ANA test was positive in 23 (92%) cases (cytoplasmic+ in 17:68%). Anti-ENA screen was + in 9: 36% pts. One pt was + for anti-PM-Scl, one for for anti-Mi2 and one for anti-Ku Ab. Pts had mainly a NSIP pattern (19, 76%, cases; with concomitant OP, in 3). Three patients had UIP-like and 3 OP pattern. The majority of patients (15, 60%) satysfied only the morphological and serological domains. Clinical domains satisfied were: arthritis (4) Raynaud’s ph. (5) palmar teleangectasias (2) mechanic’s hands (1) and Hikers feet (1). Clinical spectrum time course was variable in 5 pts: 3 pts developed arthritis after ILD, and 2 developed ILD respectively after arthritis and inflammatory miopathy. Three pts were admitted in ICU for Rapidly Progressive (RP) ILD and 2 died. All pts received low dose steroids in combination mainly with Azathioprine, Mycophenolate, Cyclosporin, Hydroxychloroquine. 92% of patients were alive at FU. Conclusions: A multidisciplinary approach is useful in the diagnosis and the management of IPAF. RP-ILD was common, prognosis of IPAF was highly variable.","PeriodicalId":267660,"journal":{"name":"Rare ILD/DPLD","volume":"13 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2019-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"115752972","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Tolerance and efficacy of antifibrotic treatments in IPF patients carriying telomere related gene mutations","authors":"A. Justet, Dymph Klay, V. Cottin, M. M. Molina, H. Nunes, M. Reynaud‐Gaubert, J. Naccache, E. Manali, A. Froidure, S. Jouneau, L. Wémeau, C. Andrejak, A. Gondouin, S. Hirschi, B. Bondue, P. Bonniaud, C. Tromeur, G. Prévost, S. Marchand-Adam, M. Funke-Chambour, A. Gamez, C. Kanengiesser, C. V. Moorsel, B. Crestani, R. Borie","doi":"10.1183/13993003.congress-2019.oa2140","DOIUrl":"https://doi.org/10.1183/13993003.congress-2019.oa2140","url":null,"abstract":"Telomere related gene (TRG) mutations are found in approximately 20% of patients with familial pulmonary fibrosis and are associated with hepatic, cutaneous and hematologic manifestations. Pirfenidone and nintedanib have been shown to slow the decline of FVC of patients with idiopathic pulmonary fibrosis (IPF). There is limited evidence of efficacy and safety of these treatments in IPF patients carrying a TRG mutation. The objective of this retrospective multicenter study was to analyze the efficacy and safety of antifibrotic drugs in IPF patients carrying a TRG mutation. We identified 103 IPF patients carrying a mutation in TERT (n=74), TERC (n=17), RTEL1 (n=10) or PARN (n=3). from 6 countries (France, Netherlands, Belgium, Spain, Greece and Switzerland) Forty-four patients received nintedanib, 59 received pirfenidone. The mean age at diagnosis was 57.9 ± 13.6 years and the median delay between diagnosis and treatment initiation was 6.0 months [3.0-15.1]. At initiation of treatment, the FVC was 80.8% ± 20.2% and the DLCO was 44.0% ± 13.7%. The median duration of treatment was 10.8 months [6.5-18.1]. Antifibrotic treatment was terminated in 18 patients because of progression of the disease and in 15 patients due to intolerable side effects, mainly digestive (nintedanib, n = 9, pirfenidone, n = 6). The median decline in FVC prior to initiation of treatment was 24.5 mL [10.5-30.1] per month. After initiation of treatment, the median decline in FVC was 18.0 mL [9.0-27.0] per month in patients treated with nintedanib and 25.4 mL per month [14.7-36.7] in patients treated with pirfenidone. This retrospective series shows that antifibrotic therapies are well tolerated in IPF patients with TRG mutations. Further analyses are needed to evaluate the efficacy.","PeriodicalId":267660,"journal":{"name":"Rare ILD/DPLD","volume":"22 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2019-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"131029805","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"KCa3.1 ion channel is expressed by component cells of lymphangioleiomyomatosis (LAM) nodules","authors":"S. Naveed, D. Clements, S. Johnson, K. Roach, P. Bradding","doi":"10.1183/13993003.congress-2019.pa3686","DOIUrl":"https://doi.org/10.1183/13993003.congress-2019.pa3686","url":null,"abstract":"Introduction: LAM is a rare lung disease characterised by progressive cystic destruction of the lung. There is no effective treatment. The hallmark lesion of LAM is the LAM nodule, a complex structure consisting of proliferating smooth muscle-like ‘LAM cells’, fibroblasts, lymphatic endothelial and inflammatory cells. KCa3.1 ion channels are implicated in several cell processes including proliferation, migration, differentiation and collagen secretion. We hypothesise that KCa3.1-dependent cell processes in LAM cells and LAM associated fibroblasts (LAFs) contribute to the development of LAM nodules and the accompanying lung damage. Methods: TSC null LAM cells and primary LAFs were obtained from the National Centre for LAM, Nottingham. We examined these cells for the presence of KCa3.1 channels using qRT-PCR and whole-cell patch clamp electrophysiology, and used immunohistochemistry to detect KCa3.1 in LAM tissue. We also examined the effects of KCa3.1 blockers on the formation of 3D LAM spheroids resulting from LAM cell and LAF co-culture. Results: Both LAM cells and LAFs (N=3 donors) expressed KCa3.1 mRNA. At rest, both LAM cells and LAFs displayed similar membrane currents with slight outward rectification at positive potentials. Both cell types responded to the KCa3.1 channel opener 1-EBIO with the development of characteristic KCa3.1 whole cell currents, which were blocked by the selective KCa3.1 blocker senicapoc. Immunoreactive KCa3.1 was present in LAM tissue, and inhibition of KCa3.1 channels inhibited the formation of LAM spheroids dose-dependently. Conclusion: LAM cells and primary LAFs express functional KCa3.1 channels which may contribute to the development of LAM nodules.","PeriodicalId":267660,"journal":{"name":"Rare ILD/DPLD","volume":"38 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2019-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"114642290","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effectiveness of various therapy regimens for patients with pulmonary Langerhans cell histiocytosis during long-term follow-up","authors":"A. Zinchenko, L. Novikova, M. Ilkovich","doi":"10.1183/13993003.congress-2019.oa2142","DOIUrl":"https://doi.org/10.1183/13993003.congress-2019.oa2142","url":null,"abstract":"","PeriodicalId":267660,"journal":{"name":"Rare ILD/DPLD","volume":"34 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2019-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"134647354","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Segment by segment lavage as an excellent alternative treatment in pulmonary alveolar proteinosis in a 3 years old boy","authors":"S. Yousefzadegan, M. Modaresi, Rouhollah Shirzadi, Safura Navaie, Mahdieh Ghaempanah","doi":"10.1183/13993003.congress-2019.pa1406","DOIUrl":"https://doi.org/10.1183/13993003.congress-2019.pa1406","url":null,"abstract":"","PeriodicalId":267660,"journal":{"name":"Rare ILD/DPLD","volume":"10 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2019-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"126057764","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinico-pathological patterns of the thoracic endometriosis","authors":"I. Dvorakovskaya, B. Ariel, L. Novikova, P. Yablonskiy","doi":"10.1183/13993003.congress-2019.pa1412","DOIUrl":"https://doi.org/10.1183/13993003.congress-2019.pa1412","url":null,"abstract":"","PeriodicalId":267660,"journal":{"name":"Rare ILD/DPLD","volume":"29 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2019-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"123970251","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical and pathological features of idiopathic and secondary pleuroparenchymal fibroelastosis in patients undergoing lung transplantation","authors":"Naoya Ikegami, N. Nakajima, K. Tanizawa, A. Yoshizawa, T. Handa, T. Chen-Yoshikawa, T. Kubo, A. Osumi, Y. Yamada, M. Hamaji, D. Nakajima, Y. Yutaka, S. Tanaka, Kizuku Watanabe, Y. Nakatsuka, Y. Murase, T. Nakanishi, Takafumi Niwamoto, Kazuo Chen, H. Date, T. Hirai","doi":"10.1183/13993003.congress-2019.pa3679","DOIUrl":"https://doi.org/10.1183/13993003.congress-2019.pa3679","url":null,"abstract":"Introduction: Idiopathic pleuroparenchymal fibroelastosis (IPPFE) has been recognized as a distinct entity. Similar PPFE pattern is also seen in secondary interstitial lung diseases (ILDs), which is categorized as secondary PPFE (SPPFE). IPPFE and SPPFE have not been comparatively studied. Aims and Objectives: To evaluate the clinical and pathological features of IPPFE and SPPFE. Methods: Patients with IPPFE and SPPFE were identified from consecutive recipients undergoing cadaveric lung transplantation (LT) at Kyoto University Hospital between 2010 and 2018. IPPFE was diagnosed through post-transplant multidisciplinary consensus. SPPFE was diagnosed when the diagnostic criteria for IPPFE were fulfilled in secondary ILDs. Clinical data and pathological features of explanted lungs were retrospectively evaluated. Results: Of 104 cadaveric LT recipients, nine were diagnosed as IPPFE and seven as SPPFE. Etiologies for SPPFE were late onset non-infectious pulmonary complication after hematopoietic stem-cell transplantation or chemotherapy in six, and dermatomyositis in one. At the time of LT, the median age of patients was 46 years and eleven were managed with long-time oxygen therapy or mechanical ventilation. Distribution of pathological PPFE was similar between IPPFE and SPPFE. Granulomas and peribronchiolar inflammation were more frequently observed with SPPFE than IPPFE, while the frequency of constrictive bronchiolitis obliterans was not significantly different. Estimated three-year survival rate after LT was 79.6% (IPPFE 75.0%, SPPFE 85.7%). Conclusions: IPPFE and SPPFE have similar PPFE lesions and different additional pathological findings.","PeriodicalId":267660,"journal":{"name":"Rare ILD/DPLD","volume":"70 2 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2019-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"115495033","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pulmonary Alveolar Microlithiasis ~ Epidemiology in Japan and Developing treatment strategies in a novel mouse model ~","authors":"A. Saito, R. Takamiya, N. Fujitani, S. Ariki, K. Kuronuma, H. Chiba, Motoko Takahashi, Hiroki Takahashi","doi":"10.1183/13993003.congress-2019.oa2145","DOIUrl":"https://doi.org/10.1183/13993003.congress-2019.oa2145","url":null,"abstract":"Pulmonary alveolar microlithiasis (PAM) is a rare, autosomal recessive lung disease associated with accumulation of calcium phosphate microliths in the alveolar space. #1 Epidemiology in Japan PAM is found worldwide, but it predominates in a few countries, particularly Japan, Turkey and Italy. However, it is unknown whether this accurately reflects the distribution of the patients in the world. The Japanese Ministry of Health, Labor and Welfare conducted a study to find the PAM patients in Japan. It was revealed that there are only seven patients with PAM currently alive in Japan. It has been thought that there are many PAM patients in Japan, but it suggested that the number of PAM patients is decreasing as a result of the decrease in consanguineous marriage after the world war ll. Based on these data, we will report on the current status of PAM in Japan. #2 New Biomarker and Treatment Strategy for PAM We have already reported that a low-phosphate diet prevented and/or diminished microlith formation in PAM mouse model. However, although phosphate dietary restriction is easily accomplished in mice, phosphate is ubiquitous in the human diet and attempts to limit intake are often unsuccessful. Phosphate binders are widely used in hyperphosphatemic patients with CKD. We found that oral administration of phosphate binders to PAM mice attenuated the microlith burden in the lung without appreciable adverse effects. Also, we found that COX-2 was clearly down-regulated under this treatment, which seems to be a useful biomarker for PAM. These results suggest that phosphate binders may be a useful adjunct to dietary phosphate restriction in the treatment of PAM.","PeriodicalId":267660,"journal":{"name":"Rare ILD/DPLD","volume":"65 2 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2019-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"132474016","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"BMS-986278, a lysophosphatidic acid 1 (LPA1) receptor antagonist, in healthy participants: A single/multiple ascending dose (SAD/MAD) phase 1 study","authors":"G. Tirucherai, Dale Yu, Ratna G. Revankar, G. Klinger, J. Lier, J. Taubel, Todd A Correll","doi":"10.1183/13993003.congress-2019.pa1398","DOIUrl":"https://doi.org/10.1183/13993003.congress-2019.pa1398","url":null,"abstract":"","PeriodicalId":267660,"journal":{"name":"Rare ILD/DPLD","volume":"150 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2019-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"123256469","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}