OF DISCUSSION Lymphangioleiomyomatosis (LAM) is a rare pulmonary disease of unknown etiology which occurs in women, primarily in their reproductive years. The goal of this study is to establish a registry of individuals with LAM (about 200 of whom are currently known) by forming a consortium of clinical centers and referring physicians who treat LAM patients. The LAM Registry will be coordinated by a Data Coordinating Center (DCC) at The Cleveland Clinic Foundation. The cohort of identified individuals with LAM will be used to characterize the clinical features of subjects with LAM and provide some information on the natural course of the disease. Another goal will be to examine the clinical features of LAM patients who undergo lung transplantation and to assess the efficacy of this therapy. Lung tissue will be collected from participants to allow future studies into the molecular basis of LAM. The assembled cohort will facilitate future research in therapeutic trials. The concept of a registry to record baseline information about these patients, follow their progress regularly, and assess the natural progression of the illness is a positive one. Furthermore, a substantial strength of this application is the experience and talent of the investigators at the There is a general lack of focus in this application concerning the data to be collected. While an application proposing a registry would not be expected to formally present hypotheses, especially for a disease as little understood as LAM, a framework which would guide the data collection is essential. There is a need to define better what data will be collected, how it will be collected and standardized, and for what purposes. For example, the routine archiving of sera and other tissue specimens should be addressed in more detail. Finally, there is some question whether our current knowledge of the disease is sufficiently advanced to warrant a prospective study, or whether comprehensive cross-sectional data would be more appropriate to the questions at hand. Overall, however, this is considered to be an excellent proposal from a very strong group, although somewhat lacking in focus, and is recommended with a decreased budget.
| Maurer, Janet R; Ryu, Jay; Beck, Gerald et al. (2007) Lung transplantation in the management of patients with lymphangioleiomyomatosis: baseline data from the NHLBI LAM Registry. J Heart Lung Transplant 26:1293-9 |
| Ryu, Jay H; Moss, Joel; Beck, Gerald J et al. (2006) The NHLBI lymphangioleiomyomatosis registry: characteristics of 230 patients at enrollment. Am J Respir Crit Care Med 173:105-11 |
| Sullivan, E J (1998) Lymphangioleiomyomatosis: a review. Chest 114:1689-703 |
