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Review

The next breakthrough in LAM clinical trials may be their design: challenges in design and execution of future LAM clinical trials

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References

  • El-Chemaly S, Henske EP. Towards personalised therapy for lymphangioleiomyomatosis: lessons from cancer. Eur Respir Rev 2014;23:30-5
  • Taveira-DaSilva AM, Moss J. Optimizing treatments for lymphangioleiomyomatosis. Expert Rev Respir Med 2012;6:267-76
  • Carsillo T, Astrinidis A, Henske EP. Mutations in the tuberous sclerosis complex gene TSC2 are a cause of sporadic pulmonary lymphangioleiomyomatosis. Proc Natl Acad Sci USA 2000;97:6085-90
  • Yu J, Astrinidis A, Henske EP. Chromosome 16 loss of heterozygosity in tuberous sclerosis and sporadic lymphangiomyomatosis. Am J Respir Crit Care Med 2001;164:1537-40
  • Henske EP, McCormack FX. Lymphangioleiomyomatosis - a wolf in sheep’s clothing. J Clin Invest 2012;122:3807-16
  • Goncharova EA, Goncharov DA, Eszterhas A, et al. Tuberin regulates p70 S6 kinase activation and ribosomal protein S6 phosphorylation. A role for the TSC2 tumor suppressor gene in pulmonary lymphangioleiomyomatosis (LAM). J Biol Chem 2002;277:30958-67
  • McCormack FX, Inoue Y, Moss J, et al. Efficacy and safety of sirolimus in lymphangioleiomyomatosis. N Engl J Med 2011;364:1595-606
  • Seyama K, Kumasaka T, Souma S, et al. Vascular endothelial growth factor-D is increased in serum of patients with lymphangioleiomyomatosis. Lymphat Res Biol 2006;4:143-52
  • Young LR, Vandyke R, Gulleman PM, et al. Serum vascular endothelial growth factor-D prospectively distinguishes lymphangioleiomyomatosis from other diseases. Chest 2010;138:674-81
  • Young LR, Lee HS, Inoue Y, et al. Serum VEGF-D concentration as a biomarker of lymphangioleiomyomatosis severity and treatment response: a prospective analysis of the Multicenter International Lymphangioleiomyomatosis Efficacy of Sirolimus (MILES) trial. Lancet Respir Med 2013;1:445-52
  • El-Chemaly S, Goldberg HJ, Glanville AR. Should mammalian target of rapamycin inhibitors be stopped in women with lymphangioleiomyomatosis awaiting lung transplantation? Expert Rev Respir Med 2014;8:657-60
  • Ando K, Kurihara M, Kataoka H, et al. Efficacy and safety of low-dose sirolimus for treatment of lymphangioleiomyomatosis. Respir Investig 2013;51:175-83
  • Taveira-DaSilva AM, Hathaway O, Stylianou M, Moss J. Changes in lung function and chylous effusions in patients with lymphangioleiomyomatosis treated with sirolimus. Ann Intern Med 2011;154:797-805. W-292-793
  • Yao J, Taveira-DaSilva AM, Jones AM, et al. Sustained effects of sirolimus on lung function and cystic lung lesions in lymphangioleiomyomatosis. Am J Respir Crit Care Med 2014;190:1273-82
  • Yao J, Taveira-DaSilva AM, Jones AM, et al. Sustained effects of sirolimus on lung function and cystic lung lesions in lymphangioleiomyomatosis. Am J Respir Crit Care Med 2014;190(11):1273-82
  • Cottin V, Harari S, Humbert M, et al. Pulmonary hypertension in lymphangioleiomyomatosis: characteristics in 20 patients. Eur Respir J 2012;40:630-40
  • Taveira-DaSilva AM, Hathaway OM, Sachdev V, et al. Pulmonary artery pressure in lymphangioleiomyomatosis: an echocardiographic study. Chest 2007;132:1573-8
  • Zafar MA, McCormack FX, Rahman S, et al. Pulmonary vascular shunts in exercise-intolerant patients with lymphangioleiomyomatosis. Am J Respir Crit Care Med 2013;188:1167-70
  • Davies DM, de Vries PJ, Johnson SR, et al. Sirolimus therapy for angiomyolipoma in tuberous sclerosis and sporadic lymphangioleiomyomatosis: a phase 2 trial. Clin Cancer Res 2011;17:4071-81
  • Bissler JJ, Kingswood JC, Radzikowska E, et al. Everolimus for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis (EXIST-2): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet 2013;381:817-24
  • Mohammadieh AM, Bowler SD, Silverstone EJ, et al. Everolimus treatment of abdominal lymphangioleiomyoma in five women with sporadic lymphangioleiomyomatosis. Med J Aust 2013;199:121-3
  • A study to determine the effectiveness of escalating doses of RAD001 (Everolimus) in patients with lymphangioleiomyomatosis. Available from: https://clinicaltrials.gov/ct2/show/NCT01059318
  • Moses MA, Harper J, Folkman J. Doxycycline treatment for lymphangioleiomyomatosis with urinary monitoring for MMPs. N Engl J Med 2006;354:2621-2
  • Chang WY, Cane JL, Kumaran M, et al. A 2-year randomised placebo-controlled trial of doxycycline for lymphangioleiomyomatosis. Eur Respir J 2014;43:1114-23
  • Pimenta SP, Baldi BG, Kairalla RA, Carvalho CR. Doxycycline use in patients with lymphangioleiomyomatosis: biomarkers and pulmonary function response. J Bras Pneumol 2013;39:5-15
  • Taveira-DaSilva AM, Stylianou MP, Hedin CJ, et al. Decline in lung function in patients with lymphangioleiomyomatosis treated with or without progesterone. Chest 2004;126:1867-74
  • Johnson SR, Whale CI, Hubbard RB, et al. Survival and disease progression in UK patients with lymphangioleiomyomatosis. Thorax 2004;59:800-3
  • Glassberg MK, Elliot SJ, Fritz J, et al. Activation of the estrogen receptor contributes to the progression of pulmonary lymphangioleiomyomatosis via matrix metalloproteinase-induced cell invasiveness. J Clin Endocrinol Metab 2008;93:1625-33
  • Li C, Zhou X, Sun Y, et al. Faslodex inhibits estradiol-induced extracellular matrix dynamics and lung metastasis in a model of lymphangioleiomyomatosis. Am J Respir Cell Mol Biol 2013;49:135-42
  • Baldi BG, Ribeiro Carvalho CR. Doxycycline in lymphangioleiomyomatosis: not all questions are answered. Eur Respir J 2014;43:1536-7
  • Johnson SR, Chang WY, Tattersfield AE, et al. Doxycycline in lymphangioleiomyomatosis: not all questions are answered. Eur Respir J 2014;43:1538
  • Brunelli A, Catalini G, Fianchini A. Pregnancy exacerbating unsuspected mediastinal lymphangioleiomyomatosis and chylothorax. Int J Gynaecol Obstet 1996;52:289-90
  • Ryu JH, Moss J, Beck GJ, et al. The NHLBI lymphangioleiomyomatosis registry: characteristics of 230 patients at enrollment. Am J Respir Crit Care Med 2006;173:105-11
  • Yu JJ, Robb VA, Morrison TA, et al. Estrogen promotes the survival and pulmonary metastasis of tuberin-null cells. Proc Natl Acad Sci USA 2009;106:2635-40
  • Harari S, Cassandro R, Chiodini I, et al. Effect of a gonadotrophin-releasing hormone analogue on lung function in lymphangioleiomyomatosis. Chest 2008;133:448-54
  • Trial of aromatase inhibition in lymphangioleiomyomatosis (TRAIL). Available from: https://clinicaltrials.gov/ct2/show/NCT01353209
  • Taveira-DaSilva AM, Steagall WK, Rabel A, et al. Reversible airflow obstruction in lymphangioleiomyomatosis. Chest 2009;136:1596-603
  • Yen KT, Putzke JD, Staats BA, Burger CD. The prevalence of acute response to bronchodilator in pulmonary lymphangioleiomyomatosis. Respirology 2005;10:643-8
  • Nebulized or inhaled albuterol for lymphangioleiomyomatosis. Available from: https://clinicaltrials.gov/ct2/show/NCT01799538
  • Baldi BG, Albuquerque AL, Pimenta SP, et al. Exercise performance and dynamic hyperinflation in lymphangioleiomyomatosis. Am J Respir Crit Care Med 2012;186:341-8
  • Baldi BG, de Albuquerque AL, Pimenta SP, et al. A pilot study assessing the effect of bronchodilator on dynamic hyperinflation in LAM. Respir Med 2013;107:1773-80
  • Puhan MA, Lareau SC. Evidence-based outcomes from pulmonary rehabilitation in the chronic obstructive pulmonary disease patient. Clin Chest Med 2014;35:295-301
  • Dowman L, Hill CJ, Holland AE. Pulmonary rehabilitation for interstitial lung disease. Cochrane Database Syst Rev 2014;10:CD006322
  • Rochester CL, Fairburn C, Crouch RH. Pulmonary rehabilitation for respiratory disorders other than chronic obstructive pulmonary disease. Clin Chest Med 2014;35:369-89
  • Pulmonary Rehabilitation in Lymphangioleiomyomatosis. Available from: https://clinicaltrials.gov/ct2/show/NCT02009241
  • Atochina-Vasserman EN, Goncharov DA, Volgina AV, et al. Statins in Lymphangioleiomyomatosis. Simvastatin and atorvastatin induce differential effects on tuberous sclerosis complex 2-null cell growth and signaling. Am J Respir Cell Mol Biol 2013;49:704-9
  • Goncharova EA, Goncharov DA, Fehrenbach M, et al. Prevention of alveolar destruction and airspace enlargement in a mouse model of pulmonary lymphangioleiomyomatosis (LAM). Sci Transl Med 2012;4:154ra134
  • Safety of Simvastatin in LAM and TSC (SOS). Available from: https://clinicaltrials.gov/ct2/show/NCT02061397
  • Parkhitko A, Myachina F, Morrison TA, et al. Tumorigenesis in tuberous sclerosis complex is autophagy and p62/sequestosome 1 (SQSTM1)-dependent. Proc Natl Acad Sci USA 2011;108:12455-60
  • Safety study of sirolimus and hydroxychloroquine in women with lymphangioleiomyomatosis (SAIL). Available from: https://clinicaltrials.gov/ct2/show/NCT01687179
  • Gottenberg JE, Ravaud P, Puechal X, et al. Effects of hydroxychloroquine on symptomatic improvement in primary Sjogren syndrome: the JOQUER randomized clinical trial. JAMA 2014;312:249-58
  • Rosenfeld MR, Ye X, Supko JG, et al. A phase I/II trial of hydroxychloroquine in conjunction with radiation therapy and concurrent and adjuvant temozolomide in patients with newly diagnosed glioblastoma multiforme. Autophagy 2014;10:1359-68
  • Tyryshkin A, Bhattacharya A, Eissa NT. SRC kinase is a novel therapeutic target in lymphangioleiomyomatosis. Cancer Res 2014;74:1996-2005
  • The tolerability of saracatinib in subjects with lymphangioleiomyomatosis (LAM) (SLAM-1). Available from: https://clinicaltrials.gov/ct2/show/NCT02116712
  • McNeish IA, Ledermann JA, Webber L, et al. A randomised, placebo-controlled trial of weekly paclitaxel and saracatinib (AZD0530) in platinum-resistant ovarian, fallopian tube or primary peritoneal cancer dagger. Ann Oncol 2014;25:1988-95
  • Molina JR, Foster NR, Reungwetwattana T, et al. A phase II trial of the Src-kinase inhibitor saracatinib after four cycles of chemotherapy for patients with extensive stage small cell lung cancer: NCCTG trial N-0621. Lung Cancer 2014;85:245-50
  • Seyama K, Kumasaka T, Kurihara M, et al. Lymphangioleiomyomatosis: a disease involving the lymphatic system. Lymphat Res Biol 2010;8:21-31
  • Cui Y, Osorio JC, Risquez C, et al. Transforming growth factor-beta1 downregulates vascular endothelial growth factor-D expression in human lung fibroblasts via the Jun NH2-terminal kinase signaling pathway. Mol Med 2014;20:120-34
  • El-Chemaly S, Pacheco-Rodriguez G, Malide D, et al. Nuclear localization of vascular endothelial growth factor-D and regulation of c-Myc-dependent transcripts in human lung fibroblasts. Am J Respir Cell Mol Biol 2014;51:34-42
  • Pimenta SP, Baldi BG, Acencio MM, et al. Doxycycline use in patients with lymphangioleiomyomatosis: safety and efficacy in metalloproteinase blockade. J Bras Pneumol 2011;37:424-30

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