The DOI matches its Crossref record; 27 of the 27 references it cites were found in a registry or an index.
Efficacy and Safety of Sirolimus in Lymphangioleiomyomatosis
Open accessAbstract
BACKGROUND: Lymphangioleiomyomatosis (LAM) is a progressive, cystic lung disease in women; it is associated with inappropriate activation of mammalian target of rapamycin (mTOR) signaling, which regulates cellular growth and lymphangiogenesis. Sirolimus (also called rapamycin) inhibits mTOR and has shown promise in phase 1-2 trials involving patients with LAM. METHODS: We conducted a two-stage trial of sirolimus involving 89 patients with LAM who had moderate lung impairment--a 12-month randomized, double-blind comparison of sirolimus with placebo, followed by a 12-month observation period. The primary end point was the difference between the groups in the rate of change (slope) in forced expiratory volume in 1 second (FEV(1)). RESULTS: During the treatment period, the FEV(1) slope was -12±2 ml per month in the placebo group (43 patients) and 1±2 ml per month in the sirolimus group (46 patients) (P<0.001). The absolute between-group difference in the mean change in FEV(1) during the treatment period was 153 ml, or approximately 11% of the mean FEV(1) at enrollment. As compared with the placebo group, the sirolimus group had improvement from baseline to 12 months in measures of forced vital capacity, functional residual capacity, serum vascular endothelial growth factor D (VEGF-D), and quality of life and functional performance. There was no significant between-group difference in this interval in the change in 6-minute walk distance or diffusing capacity of the lung for carbon monoxide. After discontinuation of sirolimus, the decline in lung function resumed in the sirolimus group and paralleled that in the placebo group. Adverse events were more common with sirolimus, but the frequency of serious adverse events did not differ significantly between the groups. CONCLUSIONS: In patients with LAM, sirolimus stabilized lung function, reduced serum VEGF-D levels, and was associated with a reduction in symptoms and improvement in quality of life. Therapy with sirolimus may be useful in selected patients with LAM. (Funded by the National Institutes of Health and others; MILES ClinicalTrials.gov number, NCT00414648.).
Overview
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
The check wasn’t confident either way, so it gives no verdict — never disputed.
In the citing paper
“Although exposure to mTOR inhibitors in the immediate post-transplant period has been associated with delayed bronchial anastomotic healing or anastomotic dehiscence, mTOR inhibitors are now successfully continued up until the time transplant in many centers without complications.(305, 315, 320)”
From Consensus document for the selection of lung transplant candidates: An update from the International Society for Heart and Lung Transplantation. (2021) · cites this paper as [315]
Experimental — accuracy not yet measuredThe check wasn’t confident either way, so it gives no verdict — never disputed.
In the citing paper
“Although exposure to mTOR inhibitors in the immediate post-transplant period has been associated with delayed bronchial anastomotic healing or anastomotic dehiscence, mTOR inhibitors are now successfully continued up until the time transplant in many centers without complications.(305, 315, 320)”
From Consensus document for the selection of lung transplant candidates: An update from the International Society for Heart and Lung Transplantation. (2021) · cites this paper as [315]
Experimental — accuracy not yet measured
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Citations
Cites
Regulation of the mTOR Complex 1 Pathway by Nutrients, Growth Factors, and Stress
Sengupta, S., Peterson, T. R., & Sabatini, D. M. (2010) · Molecular Cell · doi:10.1016/j.molcel.2010.09.026 · Cited by 1,275
Sirolimus for Angiomyolipoma in Tuberous Sclerosis Complex or Lymphangioleiomyomatosis
Bissler, J. J., McCormack, F. X., Young, L. R., Elwing, J., Chuck, G., Leonard, J. M., Schmithorst, V. J., Laor, T., Brody, A. S., Bean, J. A., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa063564 · Cited by 1,250
Self-citationKwiatkowski, D. J. (2002) · Human Molecular Genetics · doi:10.1093/hmg/11.5.525 · Cited by 674
Carsillo, T., Astrinidis, A., & Henske, E. P. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.97.11.6085 · Cited by 648
Minimal Clinically Important Differences in COPD Lung Function
Donohue, J. F. (2005) · COPD Journal of Chronic Obstructive Pulmonary Disease · doi:10.1081/copd-200053377 · Cited by 525
The truly remarkable universality of half a standard deviation: confirmation through another look
Norman, G. R., Sloan, J. A., & Wyrwich, K. W. (2004) · Expert Review of Pharmacoeconomics & Outcomes Research · doi:10.1586/14737167.4.5.581 · Cited by 490
The NHLBI Lymphangioleiomyomatosis Registry
Ryu, J. H., Moss, J., Beck, G. J., Lee, J.-C., Brown, K. K., Chapman, J. T., Finlay, G. A., Olson, E. J., Ruoss, S. J., MAURER, J. R., et al. (2005) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.200409-1298oc · Cited by 466
Self-citationTuberin Regulates p70 S6 Kinase Activation and Ribosomal Protein S6 Phosphorylation
Goncharova, E., Goncharov, D. A., Eszterhas, A., Hunter, D., Glassberg, M., Yeung, R. S., Walker, C., Noonan, D., Kwiatkowski, D., Chou, M. M., et al. (2002) · Journal of Biological Chemistry · doi:10.1074/jbc.m202678200 · Cited by 433
Pulmonary Lymphangioleiomyomatosis: A Study of 69 Patients
Urban, T., Lazor, R., Lacronique, J., Murris, M., Labrune, S., & Valeyre, D. (1999) · Medicine · doi:10.1097/00005792-199909000-00004 · Cited by 327
McCormack, F. X. (2008) · CHEST Journal · doi:10.1378/chest.07-0898 · Cited by 303
Self-citation
Research works printed but not linked: 1
- Khanna D, 2010, Clin Exp Rheumatol, 28, S55
Cited by
Sorted by citations
Items 601–700 of 999
Risk of breast cancer in patients with lymphangioleiomyomatosis
Nunez, O. G., Baldi, B. G., Radzikowska, E., de Carvalho, C. R. R., Herranz, C., Sobiecka, M., Torre, O., Harari, S. A., Vergeer, M., Kolbe, J., et al. (2019) · Cancer Epidemiology · doi:10.1016/j.canep.2019.06.004 · Cited by 7
Shigeta, K., Kikuchi, M., Tanaka, M., Takasaki, S., Oishi, H., Sado, T., Matsuda, Y., Noda, M., Okada, Y., Mano, N., et al. (2020) · Biomedical Chromatography · doi:10.1002/bmc.4853 · Cited by 7
Mariño‐Enríquez, A., Guillou, L., & Hornick, J. L. (2013) · Elsevier eBooks · doi:10.1016/b978-1-4160-5455-9.00003-x · Cited by 7
Interstitial lung diseases—can pathologists arrive at an etiology-based diagnosis? A critical update
Popper, H. (2012) · Archiv für Pathologische Anatomie und Physiologie und für Klinische Medicin · doi:10.1007/s00428-012-1305-0 · Cited by 7
Bassi, I., Hollis, G., Cottin, V., Harari, S. A., Zwanenburg, E., Veltkamp, M., Casanova, Á., Fletcher, M. J., Masefield, S. C., Powell, P., et al. (2016) · ERJ Open Research · doi:10.1183/23120541.00102-2015 · Cited by 7
Raghu, G. (2012) · Elsevier eBooks · doi:10.1016/b978-1-4377-1604-7.00092-0 · Cited by 7
Perricone, G., Airoldi, A., & Vangeli, M. (2018) · New England Journal of Medicine · doi:10.1056/nejmc1803858 · Cited by 7
Taveira‐DaSilva, A. M., & Moss, J. (2019) · Elsevier eBooks · doi:10.1016/b978-0-12-801238-3.11475-8 · Cited by 7
Self-citationReal-world evidence couples genomic biomarkers with therapeutic outcomes for canine hemangiosarcoma
Rodrigues, L., Wu, K., Harvey, G., Post, G. S., Miller, A., Hull, A., Lambert, L., Lopes, C., & Zou, J. (2025) · Scientific Reports · doi:10.1038/s41598-025-89862-9 · Cited by 6
Genetics of interstitial lung diseases: a state-of-the-art review
Ng, N., Molina‐Molina, M., Adegunsoye, A., Borie, R., Newton, C. A., Raby, B. A., Zhang, D., Padilla, M. L., Crestani, B., Horwitz, M. S., et al. (2025) · European Respiratory Journal · doi:10.1183/13993003.00788-2025 · Cited by 8
Functional Measures in Non-COPD Chronic Respiratory Diseases: A Systematic Review
Zamboti, C. L., Pimpão, H. A., Bertin, L. D., Krinski, G. G., Garcia, T., Filho, S. L. S. D. S., Cavalheri, V., Pitta, F. D. O., & Camillo, C. A. (2024) · Journal of Clinical Medicine · doi:10.3390/jcm13226887 · Cited by 7
Progress in the treatment of lymphangioleiomyomatosis: From bench to bedside
Taveira‐DaSilva, A. M., & Moss, J. (2012) · Revista Portuguesa de Pneumologia · doi:10.1016/j.rppneu.2012.02.005 · Cited by 6
Self-citationLaboratory models for the investigation of lymphangiomatosis
Rockson, S. G. (2014) · Microvascular Research · doi:10.1016/j.mvr.2014.08.007 · Cited by 6
Ito, T., Suno, M., Sakamoto, K., Yoshizaki, Y., Yamamoto, K., Nakanishi, R., Hirano, Y., Irie, M., Kurosaki, T., Otani, S., et al. (2016) · Transplantation Proceedings · doi:10.1016/j.transproceed.2015.12.021 · Cited by 6
Sirolimus in lymphangioleiomyomatosis: A case in point for research in ‘orphan’ diseases
Koul, P. A., & Mehfooz, N. (2019) · Lung India · doi:10.4103/lungindia.lungindia_280_19 · Cited by 6
Low-dose sirolimus in retroperitoneal lymphangioleiomyomas
Ussavarungsi, K., Laroia, A. T., & Burger, C. (2019) · Lung India · doi:10.4103/lungindia.lungindia_433_18 · Cited by 6
Initial presentation of lymphangioleiomyomatosis in third trimester of pregnancy
Alkemade, L., Berghuis, M. A. T., Koopman, B., & van Pampus, M. G. (2021) · BMJ Case Reports · doi:10.1136/bcr-2020-237824 · Cited by 6
Criner, R. N., Al-Abcha, A. K., Lambert, A., & Han, M. K. (2021) · Clinics in Chest Medicine · doi:10.1016/j.ccm.2021.04.014 · Cited by 8
Humoral response to inactivated SARS-CoV-2 vaccines in patients on sirolimus alone
Cheng, C., Wang, Y., Hu, D., Zhou, W., Liu, C., Tian, X., Zhang, H., Xu, Y.-C., & Xu, K. (2022) · Science China Life Sciences · doi:10.1007/s11427-022-2102-3 · Cited by 6
McNamara, M. C., Hosios, A. M., Torrence, M. E., Zhao, T., Fraser, C. S., Wilkinson, M. E., Kwiatkowski, D. J., Henske, E. P., Wu, C., Sarosiek, K. A., et al. (2022) · iScience · doi:10.1016/j.isci.2022.105458 · Cited by 6
Wang, Z., Liu, X., Wang, W., Xu, J., Sun, H., Wei, J., Yu, Y., Zhao, Y., Wang, X., Liao, Z., et al. (2023) · Frontiers in Molecular Biosciences · doi:10.3389/fmolb.2023.1000248 · Cited by 5
Tuberous Sclerosis Complex Kidney Lesion Pathogenesis: A Developmental Perspective
Pietrobon, A., & Stanford, W. L. (2023) · Journal of the American Society of Nephrology · doi:10.1681/asn.0000000000000146 · Cited by 6
Lymphangioleiomyomatosis in patients with tuberous sclerosis: a national centre audit
Johnson, J., Somerfield, W., & Johnson, S. R. (2024) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-024-03115-y · Cited by 11
Mammalian Target of Rapamycin Inhibitors in Organ Transplantation
Langone, A. J., & Helderman, J. H. (2012) · CHEST Journal · doi:10.1378/chest.12-1247 · Cited by 7
A case of pulmonary lymphangioleiomyomatosis complicated with uterine and retroperitoneal tumors
Numata, T., Araya, J., Mikami, J., Hara, H., Harada, T., Takahashi, H., Nakayama, K., & Kuwano, K. (2015) · Respiratory Medicine Case Reports · doi:10.1016/j.rmcr.2015.02.001 · Cited by 6
Kristof, A. S., Petrof, B. J., Hamid, Q. A., Kolb, M. R. J., Landry, J., MacKenzie, A., McCormack, F. X., Murawski, I. J., Moss, J., Rauch, F., et al. (2017) · Annals of the American Thoracic Society · doi:10.1513/annalsats.201705-406ws · Cited by 6
Self-citationNamba, M., Masuda, T., Nakamura, T., Horimasu, Y., Miyamoto, S., Nakashima, T., Iwamoto, H., Fujitaka, K., Hamada, H., & Hattori, N. (2017) · Internal Medicine · doi:10.2169/internalmedicine.8645-16 · Cited by 6
A Case Report of Lymphangioleiomyomatosis Presenting as Spontaneous Pneumothorax
Riojas, R. A., Bahr, B. A., Thomas, D. B., Perciballi, J., & Noyes, L. (2012) · Military Medicine · doi:10.7205/milmed-d-11-00333 · Cited by 6
Gupta, N., Hagner, M., Wu, H., Young, L. R., Palipana, A., Szczesniak, R. D., & McCormack, F. X. (2020) · Annals of the American Thoracic Society · doi:10.1513/annalsats.202006-702rl · Cited by 6
Self-citationEffect of everolimus on multifocal micronodular pneumocyte hyperplasia in tuberous sclerosis complex
Daccord, C., Nicolas, A., Demicheli, R., Chehade, H., Hottinger, A. F., Beigelman‐Aubry, C., & Lazor, R. (2020) · Respiratory Medicine Case Reports · doi:10.1016/j.rmcr.2020.101310 · Cited by 6
El‐Chemaly, S., & Henske, E. P. (2015) · Expert Review of Respiratory Medicine · doi:10.1586/17476348.2015.1024663 · Cited by 6
DiMario, F. J. (2019) · Wiley eBooks · doi:10.1002/9781119142812.ch143 · Cited by 6
Novel treatment strategies for lymphangioleiomyomatosis: a narrative review
Élia, D., Harari, S., Fan, L., Diesler, R., & Henske, E. (2025) · European Respiratory Review · doi:10.1183/16000617.0019-2025 · Cited by 5
Szalai, F., Pápay, J., Dezső, K., Kuthi, L., Sebestyén, A. R., Khoór, A., & Krencz, I. (2025) · Histopathology · doi:10.1111/his.70026 · Cited by 5
Seo, J., Park, S., Lee, D. C., Yoo, W., Yang, Y. R., Seo, H. W., Park, J. L., Joo, J., Park, K. C., & Byun, S. (2026) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-026-02688-3 · Cited by 5
The Challenges of Clinical Research in Orphan Diseases
Spagnolo, P., & du Bois, R. M. (2014) · Springer eBooks · doi:10.1007/978-1-4471-2401-6_2 · Cited by 5
Sauter, M., Sigl, J., Schotten, K., Günthner-Biller, M., Knabl, J., & Fischereder, M. (2013) · The European Journal of Contraception & Reproductive Health Care · doi:10.3109/13625187.2013.859667 · Cited by 5
Abdominal ultrasonographic manifestations in pediatric patients with tuberous sclerosis complex
Xu, Z., Wu, J., Gui-min, X., & Luo, H. (2020) · Translational Pediatrics · doi:10.21037/tp-20-150 · Cited by 5
Fang, F., Tamura, R., Braun, T., & Kidwell, K. (2022) · Statistics in Biopharmaceutical Research · doi:10.1080/19466315.2022.2118162 · Cited by 5
Wan, L., He, W., Wang, Y., Xu, Y., Lu, Q., Zhang, M., Wang, Q., Dun, S., Liu, L., Shi, X., et al. (2022) · Therapeutic Advances in Neurological Disorders · doi:10.1177/17562864221138148 · Cited by 5
Jobu, Y., Nishigawa, M., Furihata, K., Furihata, M., Uchida, K., & Taniuchi, K. (2025) · Human Cell · doi:10.1007/s13577-024-01165-9 · Cited by 5
Successful treatment of progressive diffuse PEComatosis
Lawson, K., Maher, T. M., Hansell, D. M., & Nicholson, A. G. (2012) · European Respiratory Journal · doi:10.1183/09031936.00076411 · Cited by 5
Emerging Therapies for Soft-Tissue Sarcomas
Bonneville-Levard, A., Tassy, L., & Cassier, P. A. (2013) · Hematology/Oncology Clinics of North America · doi:10.1016/j.hoc.2013.07.011 · Cited by 5
Development of ATAQ-LAM: a tool to assess quality of life in Lymphangioleiomyomatosis
Walker, T., Desserich, J. A., Albright, K., Wamboldt, F. S., Belkin, A., Fier, K., & Swigris, J. J. (2015) · Health and Quality of Life Outcomes · doi:10.1186/s12955-015-0294-5 · Cited by 5
An extrapulmonary manifestation of lymphangioleiomyomatosis: A rare case report
Tümay, V., Güner, O. S., & Zorluoğlu, A. (2017) · International Journal of Surgery Case Reports · doi:10.1016/j.ijscr.2017.10.057 · Cited by 5
Chebib, N., Archer, F., Erny, A., Leroux, C., & Cottin, V. (2018) · Scientific Reports · doi:10.1038/s41598-018-32795-3 · Cited by 5
Sirolimus in der Therapie der LAM – ein neuer MILEStone erreicht?
Kreuter, M., & Wirtz, H. R. W. (2018) · Pneumologie · doi:10.1055/a-0802-3633 · Cited by 5
Sensory Signaling Pathways in Inflammatory and Neuropathic Pain
Willemen, H. L. D. M., & Eijkelkamp, N. (2019) · Oxford University Press eBooks · doi:10.1093/oxfordhb/9780190860509.013.30 · Cited by 5
Pulmonary Lymphangioleiomyomatosis originates in the pleural mesothelial cell population
Clements, D., Miller, S., & Johnson, S. R. (2020) · Medical Hypotheses · doi:10.1016/j.mehy.2020.109703 · Cited by 5
Ishida, N., Miyazu, T., Tamura, S., Suzuki, S., Tani, S., Yamade, M., Iwaizumi, M., Osawa, S., Hamaya, Y., Shinmura, K., et al. (2020) · World Journal of Gastroenterology · doi:10.3748/wjg.v26.i45.7263 · Cited by 5
Bakhtiary, H., Barzegar, M., Shiva, S., Poorshiri, B., Hajalioghli, P., & Ghadim, H. H. (2021) · Iranian journal of child neurology. · doi:10.22037/ijcn.v15i4.30591 · Cited by 5
Mariño‐Enríquez, A., & Hornick, J. L. (2019) · Elsevier eBooks · doi:10.1016/b978-0-323-49714-5.00003-x · Cited by 5
Doubková, M., Štefániková, M., Čan, V., Merta, Z., & Svoboda, M. (2019) · Klinicka onkologie · doi:10.14735/amko2019367 · Cited by 5
Upregulation of acid ceramidase contributes to tumor progression in tuberous sclerosis complex
Astrinidis, A., Li, C., Zhang, E. Y., Zhao, X., Zhao, S., Guo, M., Olatoke, T., Mattam, U., Huang, R., Zhang, A. G., et al. (2023) · JCI Insight · doi:10.1172/jci.insight.166850 · Cited by 5
Self-citationTarasewicz, A., Dębska-Ślizień, A. M., Rutkowska, B., Szurowska, E., & Matuszewski, M. (2018) · Transplantation Proceedings · doi:10.1016/j.transproceed.2018.03.028 · Cited by 5
Ben‐Ami, E., Hornick, J. L., & Wagner, A. J. (2018) · Expert Opinion on Orphan Drugs · doi:10.1080/21678707.2018.1493376 · Cited by 5
Highly effective sirolimus therapy for abdominal lymphangioleiomyoma
Lecuelle, D., Basille, D., Renard, C., Saint, F., & Jounieaux, V. (2019) · Respiratory Medicine and Research · doi:10.1016/j.resmer.2019.02.002 · Cited by 5
The emergence of targetable MEKanisms in sporadic lymphatic disorders
Dellinger, M. T., & McCormack, F. X. (2020) · EMBO Molecular Medicine · doi:10.15252/emmm.202012822 · Cited by 5
Self-citationEfficacy of hormonal suppression in a patient with chyluria due to lymphangioleiomyomatosis
Contini, P., Schiavina, M., Schiavina, R., Tavalazzi, F., Fabiani, A., Scioscio, V. D., Spagnolo, P., & Richeldi, L. (2011) · Multidisciplinary Respiratory Medicine · doi:10.1186/2049-6958-6-5-313 · Cited by 5
Tuberous Sclerosis Complex and Lymphangioleiomyomatosis: Miles to Go, Promises to Keep
Henske, E. P. (2011) · Annals of Internal Medicine · doi:10.7326/0003-4819-154-12-201106210-00015 · Cited by 5
Suina, K., Hayashi, T., Mitani, K., Suzuki, K., Takahashi, K., & Seyama, K. (2014) · Respiratory Investigation · doi:10.1016/j.resinv.2014.02.004 · Cited by 5
Identification of the Lymphangioleiomyomatosis Cell and Its Uterine OriginPreprint
Guo, M., Yu, J. J., Perl, A. T., Wikenheiser‐Brokamp, K. A., Riccetti, M., Zhang, E. Y., Sudha, P., Adam, M., Potter, A., Kopras, E. J., et al. (2019) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/784199 · Cited by 5
Self-citationZhou, B., Guo, Q., Zhou, H., XIE, W., XUE, T., Li, M., Yang, L., & Shi, Z. (2016) · Biomedical Reports · doi:10.3892/br.2016.652 · Cited by 4
Chen, J., He, W., Wang, Y., Zhang, M., Lu, Q., Wang, Q., Luo, X., Wang, B., & Zou, L. (2022) · Child s Nervous System · doi:10.1007/s00381-022-05446-2 · Cited by 4
Shrestha, S., Adib, E., Imani, J., Aguiar, D. J., Lamattina, A. M., Tassew, D., Henske, E. P., Perrella, M. A., Priolo, C., & El‐Chemaly, S. (2022) · Journal of Biological Chemistry · doi:10.1016/j.jbc.2022.102580 · Cited by 4
Sporadic Lymphangioleiomyomatosis Disease: A Case Report.
Nikmanesh, Y., Shokripour, M., Mokhtari, M., Khazayi, M., Monabati, A., Rezayi, R., & Bahtouee, M. (2023) · PubMed Central · doi:10.30476/ijms.2022.95521.2689 · Cited by 4
Nelson, N. C., Kogan, R., Condos, R., & Hena, K. (2023) · Journal of Clinical Medicine · doi:10.3390/jcm13010015 · Cited by 4
Brown, J. A., Faley, S. L., Judge, M., Ward, P., Ihrie, R. A., Carson, R., Armstrong, L. C., Şahin, M., Wikswo, J. P., Ess, K. C., et al. (2024) · Journal of Neurodevelopmental Disorders · doi:10.1186/s11689-024-09543-y · Cited by 5
Surgical management of pleural complications in lymphangioleiomyomatosis
Stanifer, B. P., Harris, S., Nguyen, S. N., Vinogradsky, A., Trivedi, N., Fonseca, L., Salvatore, M. M., Sonett, J. R., D'Armiento, J. M., & Goldklang, M. P. (2024) · Journal of Thoracic and Cardiovascular Surgery · doi:10.1016/j.jtcvs.2024.08.038 · Cited by 4
Pifithrin-μ sensitizes mTOR-activated liver cancer to sorafenib treatment
Lv, J., Wang, Y., Lv, J., Zheng, C., Zhang, X., Wan, L., Zhang, J., Liu, F., & Zhang, H. (2025) · Cell Death and Disease · doi:10.1038/s41419-025-07332-6 · Cited by 4
Insights into Tuberous Sclerosis Complex : From Genes to Clinics
Kim, S. Y. (2025) · Journal of Korean Neurosurgical Society · doi:10.3340/jkns.2025.0035 · Cited by 4
A Pregnant Woman Presenting With Progressively Worsening Dyspnea and Pneumothorax
Agrawal, A., Karle, E. M., Patel, T. P., Wilson, G., Hofmann, H., Sunna, R., & Krvavac, A. (2020) · CHEST Journal · doi:10.1016/j.chest.2019.11.035 · Cited by 4
High-Intensity Exercise Improves Pulmonary Function and Exercise Tolerance in a Patient with TSC-LAM
Lowder, T. (2020) · Advances in respiratory medicine · doi:10.5603/arm.a2020.0129 · Cited by 4
Advancement in translational respiratory research using nanotechnology
Pandey, P., Purohit, D., Chellappan, D. K., Gupta, G., Tambuwala, M. M., Aljabali, A. A. A., Satija, S., & Dureja, H. (2020) · Elsevier eBooks · doi:10.1016/b978-0-12-820658-4.00010-8 · Cited by 4
Taveira‐DaSilva, A. M., Gopalakrishnan, V., Yao, J., Chen, M. Y., Julien-Williams, P. A., Jones, A. M., Pacheco–Rodriguez, G., & Moss, J. (2022) · BMC Pulmonary Medicine · doi:10.1186/s12890-022-02123-7 · Cited by 4
Self-citationHarun, N., Gupta, N., McCormack, F. X., & Macaluso, M. (2023) · Clinical Trials · doi:10.1177/17407745231158906 · Cited by 5
Self-citationThe role of moesin in diagnosing and assessing severity of lymphangioleiomyomatosis
Song, X., Cai, H., Peng, W., Chen, K., Abuduxukuer, Z., Zeng, Y., Zhu, G., Lu, C., Chen, Y., Wang, J., et al. (2024) · Respiratory Research · doi:10.1186/s12931-024-02685-6 · Cited by 4
Direct modulation of TRPM8 ion channels by rapamycin and analog macrolide immunosuppressants
Tóth, B. I., Bazeli, B., Janssens, A., Lisztes, E., Racskó, M., Kelemen, B., Herczeg, M., Nagy, T. M., Kövér, K. E., Mitra, A., et al. (2025) · eLife · doi:10.7554/elife.97341.3 · Cited by 4
A chylothorax in a young woman: The difficulties of medical management
Barton, E. C., Johnson, S. R., Collin, N., Bhatt, N., & Maskell, N. A. (2024) · Respirology Case Reports · doi:10.1002/rcr2.1303 · Cited by 4
Therapeutic strategies for treatment of pulmonary lymphangioleiomyomatosis
Krymskaya, V. P. (2014) · Expert Opinion on Orphan Drugs · doi:10.1517/21678707.2014.953481 · Cited by 4
Mou, Y., Ye, L., Wang, J., Yee, M., Song, Y.-Q., Zhu, L.-F., & Jin, M. (2016) · PubMed · Cited by 4
AJRCCM : 100-Y ear A nniversary . Progress in Interstitial Lung Disease
Kaner, R. J., Brown, K. K., & Martínez, F. J. (2017) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201703-0584ed · Cited by 4
Self-citationRoles of human epidermal growth factor receptor family in pulmonary lymphangioleiomyomatosis
Kobayashi, K., Miki, Y., Saito, R., Adachi, K., Seyama, K., Okada, Y., & Sasano, H. (2018) · Human Pathology · doi:10.1016/j.humpath.2018.07.002 · Cited by 4
Courtwright, A., Baldi, B. G., Kidambi, P., Cui, Y., Lamattina, A. M., Villalba, J. A., Bagwe, S., Goldberg, H. J., Rosas, I. O., Henske, E. P., et al. (2018) · Sarcoidosis vasculitis and diffuse lung diseases · doi:10.36141/svdld.v35i3.6321 · Cited by 4
Self-citationCyst‐based measurements for assessing lymphangioleiomyomatosis in computed tomography
Lo, P., Brown, M. S., Kim, H., Kim, H., Argula, R. G., Strange, C., & Goldin, J. G. (2015) · Medical Physics · doi:10.1118/1.4916655 · Cited by 4
Self-citationMedhat, M. A., Malek, M. A. Y. A., Zaki, S., Helmy, A., & Driscoll, J. J. (2014) · Case Reports in Oncological Medicine · doi:10.1155/2014/904581 · Cited by 4
The mTOR inhibitor revolution rolls on
Northrup, H. (2014) · The Lancet Oncology · doi:10.1016/s1470-2045(14)71101-5 · Cited by 4
Seizures in tuberous sclerosis complex: hitting the target
Roach, E. S., & Kwiatkowski, D. J. (2016) · The Lancet · doi:10.1016/s0140-6736(16)31576-8 · Cited by 4
Novotný, R., Chlupáč, J., Marada, T., Bloudíčková, S. R., Vavrinova, H., Janoušek, L., & Froněk, J. (2019) · Case Reports in Transplantation · doi:10.1155/2019/2172163 · Cited by 4
Lymphangioleiomyomatosis: A Review
Konkol, S. (2022) · American Journal of Biomedical Science & Research · doi:10.34297/ajbsr.2022.17.002320 · Cited by 4
Pleural Fibrosis: Now That’s What mTORC(ing) About
Mutsaers, S. E., & Krymskaya, V. P. (2023) · American Journal of Respiratory Cell and Molecular Biology · doi:10.1165/rcmb.2023-0327ed · Cited by 4
Inhibition of NAMPT as a Therapeutic Strategy to Suppress Tumor Growth in Lymphangioleiomyomatosis
Fard, S. S., Kundu, N., Torres, A. S., Faltas, C., Martino, J. D. D., & Holz, M. K. (2025) · Biochimica et biophysica acta. Molecular cell research · doi:10.1016/j.bbamcr.2025.119986 · Cited by 3
Bai, W., Zhang, S., Hua, L., Wang, D., Guo, M., Wang, X., Zhou, Y., Cao, Y., Wang, Q., Zhang, N., et al. (2025) · Respiratory Research · doi:10.1186/s12931-025-03285-8 · Cited by 3
The role of Ephexin1 in translation and mTOR-targeted cancer therapy
Kim, J., Chang, I. Y., Lee, J.-H., Yong, J., Jeon, Y. J., & You, H. J. (2025) · Experimental & Molecular Medicine · doi:10.1038/s12276-025-01520-2 · Cited by 3
Patel, S. P., Othus, M. K. D., Chae, Y. K., Azenkot, T., Wagner, M. J., Threlkel, S. A., Farley, J. H., Magner, C. M., Chen, H. X., Sharon, E., et al. (2025) · Journal for ImmunoTherapy of Cancer · doi:10.1136/jitc-2025-013147 · Cited by 3
High-Throughput Proteomics in Lymphangioleiomyomatosis
Diesler, R., Han, T., Steagall, W. K., Salem, S. T., Khabibullin, D., Chami, J., Squitieri, B., Jones, A. M., Julien-Williams, P. A., Machado, T., et al. (2025) · CHEST Journal · doi:10.1016/j.chest.2025.12.026 · Cited by 3
Self-citationZou, C., Wang, Y. J., Chen, Q., Shi, J., Wu, X., & Luo, Q. (2026) · Frontiers in Oncology · doi:10.3389/fonc.2026.1745024 · Cited by 3
Pneumothorax: how to predict, prevent and cure
Hallifax, R., Walker, S. P., & Marciniak, S. J. (2020) · European Respiratory Society eBooks · doi:10.1183/2312508x.10023919 · Cited by 3
Zhao, S., Hao, S., Zhou, J., Chen, X., Zhang, T., Qi, Z., Zhang, T., Jalal, S., Zhai, C., Lu, Y. Y., et al. (2024) · Cellular & Molecular Biology Letters · doi:10.1186/s11658-024-00638-x · Cited by 3
Tuberous sclerosis: a survey in the canton of Vaud, Switzerland
Hagon-Nicod, O., Fellmann, F., Nový, J., Lebon, S., Wider, C. W., Lazor, R., & Bonny, O. (2024) · Frontiers in Medicine · doi:10.3389/fmed.2024.1513619 · Cited by 3
Metadata
- Authors
- McCormack, F. X., Inoue, Y., Moss, J., Singer, L. G., Strange, C., Nakata, K., Barker, A. F., Chapman, J. T., Brantly, M. L., Stocks, J. M., Brown, K. K., Lynch, J. P., Goldberg, H. J., Young, L. R., Kinder, B. W., Downey, G. P., Sullivan, E. J., Colby, T. V., McKay, R. T., Cohen, M. M., Korbee, L., Taveira-DaSilva, A. M., Lee, H.-S., Krischer, J. P., & Trapnell, B. C.
- Affiliations
- University of Cincinnati (US)
- National Hospital Organization (JP)
- National Institutes of Health (US)
- University of Toronto (CA)
- Medical University of South Carolina (US)
- Niigata University Medical and Dental Hospital (JP)
- Oregon Health & Science University (US)
- Cleveland Clinic (US)
- University of Florida (US)
- The University of Texas Health Science Center at Tyler (US)
- National Jewish Health (US)
- Cincinnati Children's Hospital Medical Center (US)
- Harvard University (US)
- United Therapeutics (United States) (US)
- Mayo Clinic (US)
- University of South Florida (US)
- ORCID
- Venue
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Published
- 2011-04-28
- Volume
- 364
- Issue
- 17
- Pages
- 1595-1606
- ISSN
- 0028-4793 (ISSN-L)
- 1533-4406
- PMC
- PMC3118601
- OpenAlex
- W2147028601
- Semantic Scholar
- 0af4a52c455e626ae3c70aa2ba2d64208b19a305
- Cited by
- 1,099
- References
- 27
- Citations by year
- 2026: 38 · 2025: 54 · 2024: 77 · 2023: 70 · 2022: 80 · 2021: 70 · 2020: 72 · 2019: 78 · 2018: 77 · 2017: 65 · 2016: 78 · 2015: 87 · 2014: 71 · 2013: 81 · 2012: 68
- FWCI
- 54.96
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- New England Journal of Medicine (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Funders
- Tuberous Sclerosis Alliance
- Pfizer
- LAM Foundation
- Cincinnati Children's Hospital Medical Center
- National Institutes of Health (1ul1rr026314-01, 1UL1RR026314)
- U.S. Food and Drug Administration (FD003362, R01 FD003362)
- Canadian Institutes of Health Research
- National Heart, Lung, and Blood Institute (R01HL085453, ZIAHL002541)
- National Center for Research Resources (1UL1 RR-026314-01, U54RR019259, UL1-RR026314, U54RR019498, RR019498, RR019259)
- NHLBI Division of Intramural Research
- Topics
- Health Sciences › Medicine › Genetics (clinical) › Tuberous Sclerosis Complex Research
- Health Sciences › Medicine › Surgery › Vascular Malformations and Hemangiomas
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Pulmonary Hypertension Research and Treatments
- Keywords
- lymphangioleiomyomatosis, rapamycin, mTOR signaling, lung function, FEV1, VEGF-D, quality of life, adverse events, drug discontinuation, cystic lung disease
- MeSH terms
- Adult, Double-Blind Method, Female, Forced Expiratory Volume, Humans, Middle Aged, Quality of Life, Vital Capacity, Lymphangioleiomyomatosis, Observation, Sirolimus, Medication Adherence, Intention to Treat Analysis, TOR Serine-Threonine Kinases
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
