Clinical Predictors of Mortality and Cause of Death in Lymphangioleiomyomatosis: A Population-based Registry

Oprescu, Nicoleta; McCormack, Francis X.; Byrnes, S.; Kinder, Brent W.

doi:10.1007/s00408-012-9419-3

Cited by 74 publications

(78 citation statements)

References 22 publications

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“…oxygen use). Even if the results may be biased by the lack of data from pulmonary function tests, the conclusions of the study about survival are consistent with estimates from previous recent cohorts [103]. Although LAM has a heterogeneous course and often unpredictable outcome, in the past few years some clinical characteristics have been found to be associated with prognosis.…”

Section: Clinical Presentation Natural History and Prognosissupporting

confidence: 77%

“…A report from Japan suggests that presentation with pneumothorax is associated with younger age and a more favourable prognosis than presentation with dyspnoea (10-year survival rate of 89% and 47%, respectively) [91]. In accordance with these results, the recent study by OPRESCU et al [103] showed that patients who presented with dyspnoea or cough had a worse prognosis compared with the subgroup who presented with pleural disease (pneumothorax or pleural effusion) or other presentations. In this report, age at presentation, the need for supplemental oxygen therapy and reported weight loss were independent predictors of survival; older age at presentation was associated with improved survival, whereas the need for supplemental oxygen therapy and reported weight loss were associated with a shorter survival.…”

Section: Clinical Presentation Natural History and Prognosismentioning

confidence: 78%

“…Mortality at 10 years has been estimated to be 30% from the time of lung biopsy [94] and ∼10-20% from the onset of symptoms [1,74,94]. Recently, in a large cohort of patients (n=401) with long-term follow-up [103], an estimated 10-year transplant-free survival of 86% was observed; the median transplant-free survival for the overall cohort was 29 years from the time of onset of symptoms, and 23 years from the time of diagnosis. No statistically significant difference between the hormonally treated and untreated subgroup was found when adjusted for the severity of disease (i.e.…”

Section: Clinical Presentation Natural History and Prognosismentioning

confidence: 99%

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The changing face of a rare disease: lymphangioleiomyomatosis

et al. 2015

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Lymphangioleiomyomatosis is a rare disease characterised by cystic destruction of the lung, lymphatic abnormalities and abdominal tumours. It affects almost exclusively females and can occur sporadically or in patients with tuberous sclerosis complex.In the past decade remarkable progress has been made in understanding of the pathogenesis of this disease leading to a new therapeutic approach. This review summarises recent advances regarding pathogenic mechanisms and clinical manifestations, and highlights the current and the most promising future therapeutic strategies. @ERSpublications Recent advances in pathogenesis, clinical manifestations and therapeutic strategies of lymphangioleiomyomatosis http://ow.ly/Rbmlh

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Section: Clinical Presentation Natural History and Prognosissupporting

confidence: 77%

Section: Clinical Presentation Natural History and Prognosismentioning

confidence: 78%

Section: Clinical Presentation Natural History and Prognosismentioning

confidence: 99%

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The changing face of a rare disease: lymphangioleiomyomatosis

et al. 2015

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“…The natural history of LAM is characterized by a progressive decline in lung function, with a reported median survival time of 29 years (26). These data were used to determine annual mortality rates for patients with LAM.…”

Section: Original Researchmentioning

confidence: 99%

Chest Computed Tomographic Image Screening for Cystic Lung Diseases in Patients with Spontaneous Pneumothorax Is Cost Effective

et al. 2017

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Rationale: Patients without a known history of lung disease presenting with a spontaneous pneumothorax are generally diagnosed as having primary spontaneous pneumothorax. However, occult diffuse cystic lung diseases such as Birt-Hogg-Dubé syndrome (BHD), lymphangioleiomyomatosis (LAM), and pulmonary Langerhans cell histiocytosis (PLCH) can also first present with a spontaneous pneumothorax, and their early identification by high-resolution computed tomographic (HRCT) chest imaging has implications for subsequent management.Objectives: The objective of our study was to evaluate the costeffectiveness of HRCT chest imaging to facilitate early diagnosis of LAM, BHD, and PLCH.Methods: We constructed a Markov state-transition model to assess the cost-effectiveness of screening HRCT to facilitate early diagnosis of diffuse cystic lung diseases in patients presenting with an apparent primary spontaneous pneumothorax. Baseline data for prevalence of BHD, LAM, and PLCH and rates of recurrent pneumothoraces in each of these diseases were derived from the literature. Costs were extracted from 2014 Medicare data. We compared a strategy of HRCT screening followed by pleurodesis in patients with LAM, BHD, or PLCH versus conventional management with no HRCT screening. Measurements and Main Results:In our base case analysis, screening for the presence of BHD, LAM, or PLCH in patients presenting with a spontaneous pneumothorax was cost effective, with a marginal cost-effectiveness ratio of $1,427 per quality-adjusted lifeyear gained. Sensitivity analysis showed that screening HRCT remained cost effective for diffuse cystic lung diseases prevalence as low as 0.01%. Conclusions:HRCT image screening for BHD, LAM, and PLCH in patients with apparent primary spontaneous pneumothorax is cost effective. Clinicians should consider performing a screening HRCT in patients presenting with apparent primary spontaneous pneumothorax.

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“…Patients with LAM frequently present with dyspnea on exertion; recurrent pneumothoraces; chylous effusions; incidental detection of lung cysts on imaging studies; and, less frequently, bleeding angiomyolipomas (1,2). The clinical course of LAM is variable but LAM is generally considered to be a chronic disease with a median transplant-free survival time spanning more than a decade (5). LAM lesions are characterized by proliferation of a neoplastic LAM cell, which has features both of smooth muscle and melanocytic cells (4).…”

mentioning

confidence: 99%

Sustained Effects of Sirolimus on Lung Function and Cystic Lung Lesions in Lymphangioleiomyomatosis

Yao

Taveira-DaSilva²,

Jones³

et al. 2014

Am J Respir Crit Care Med

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Rationale: Sirolimus therapy stabilizes lung function and reduces the size of chylous effusions and lymphangioleiomyomas in patients with lymphangioleiomyomatosis.Objectives: To determine whether sirolimus has beneficial effects on lung function, cystic areas, and adjacent lung parenchyma; whether these effects are sustained; and whether sirolimus is well tolerated by patients.Methods: Lung function decline over time, lung volume occupied by cysts (cyst score), and lung tissue texture in the vicinity of the cysts were quantified with a computer-aided diagnosis system in 38 patients. Then we compared cyst scores from the last study on sirolimus with studies done on sirolimus therapy. In 12 patients, we evaluated rates of change in lung function and cyst scores off and on sirolimus.Measurements and Main Results: Sirolimus reduced yearly declines in FEV 1 (22.3 6 0.1 vs. 1.0 6 0.3% predicted; P , 0.001) and diffusing capacity of carbon monoxide (22.6 6 0.1 vs. 0.9 6 0.2% predicted; P , 0.001). Cyst scores 1.2 6 0.8 years (30.5 6 11.9%) and 2.5 6 2 years (29.7 6 12.1%) after initiating sirolimus were not significantly different from pretreatment values (28.4 6 12.5%). In 12 patients followed for 5 years, a significant reduction in rates of yearly decline in FEV 1 (21.4 6 0.2 vs. 0.3 6 0.4% predicted; P = 0.025) was observed. Analyses of 104 computed tomography scans showed a nonsignificant (P = 0.23) reduction in yearly rates of change of cyst scores (1.8 6 0.2 vs. 0.3 6 0.3%; P = 0.23) and lung texture features. Despite adverse events, most patients were able to continue sirolimus therapy.Conclusions: Sirolimus therapy slowed down lung function decline and increase in cystic lesions. Most patients were able to tolerate sirolimus therapy.Keywords: sirolimus; lung cystic destruction; LAM Lymphangioleiomyomatosis (LAM) is a multisystem disease affecting almost exclusively women, which is characterized by cystic lung destruction, renal angiomyolipomas, and lymphangioleiomyomas (1-4). Patients with LAM frequently present with dyspnea on exertion; recurrent pneumothoraces; chylous effusions; incidental detection of lung cysts on imaging studies; and, less frequently, bleeding angiomyolipomas (1, 2). The clinical course of LAM is variable but LAM is generally considered to be a chronic disease with a median transplant-free survival time spanning more than a decade (5). LAM lesions are characterized by proliferation of a neoplastic LAM cell, which has features both of smooth muscle and melanocytic cells (4). The histologic features of LAM consist of cysts and LAM cells in the walls of cysts and along blood vessels, lymphatics, and bronchioles, causing airways narrowing, vascular wall thickening, lymphatic disruption, and venous occlusion (4).The inherited form of LAM associated with tuberous sclerosis complex (TSC) is caused by mutations in the TSC1 or TSC2 suppressor genes (6-8). Sporadic LAM has been associated only with mutations of TSC2 (6-8). TSC1 and TSC2 encode, respectively, hamartin and tuberin, two Corres...

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Clinical Predictors of Mortality and Cause of Death in Lymphangioleiomyomatosis: A Population-based Registry

Cited by 74 publications

References 22 publications

The changing face of a rare disease: lymphangioleiomyomatosis

The changing face of a rare disease: lymphangioleiomyomatosis

Chest Computed Tomographic Image Screening for Cystic Lung Diseases in Patients with Spontaneous Pneumothorax Is Cost Effective

Sustained Effects of Sirolimus on Lung Function and Cystic Lung Lesions in Lymphangioleiomyomatosis

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