Francis X Mccormack

Publications (110) View all

• Article: St. George's Respiratory Questionnaire has Longitudinal Construct Validity in Lymphangioleiomyomatosis*

  Jeffrey J Swigris, Hye-Seung Lee, Marsha Cohen, Yoshikazu Inoue, Joel Moss, Lianne Singer, Lisa R Young, Francis X McCormack
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  ABSTRACT: ABSTRACT BACKGROUND: Lymphangioleiomyomatosis (LAM) is an uncommon, progressive, cystic lung disease that causes shortness of breath, hypoxemia and impaired health-related quality of life (HRQL). Whether St. George's Respiratory Questionnaire (SGRQ)-a respiratory-specific HRQL instrument-captures longitudinal changes in HRQL in LAM patients is unknown. METHODS: Using data from the Multicenter International Lymphangioleiomyomatosis Efficacy and Safety of Sirolimus (MILES) trial, we performed analyses to examine associations between SGRQ scores and values for four external measures (anchors). Anchors included (1) the one-second forced expiratory volume (FEV1), (2) diffusing capacity of the lung for carbon monoxide, (3) distance walked during the six-minute walk test, and (4) serum vascular endothelial growth factor-D. RESULTS: SGRQ scores correlated with the majority of anchor values at baseline, 6 and 12 months. Results from longitudinal analyses demonstrated that SGRQ change scores tracked changes over time in values for each of the four anchors. At 12 months, subjects with the greatest improvement from baseline in FEV1 experienced the greatest improvement in SGRQ scores (Symptoms domain -13.4±14.6 points, Activity domain -6.46±8.20 points, Impacts domain -6.25±12.8 points, SGRQ total -7.53±10.0 points). Plots of cumulative distribution functions further supported the longitudinal validity of the SGRQ in LAM. CONCLUSIONS: In LAM, SGRQ scores are associated with variables used to assess LAM severity. The SGRQ is sensitive to change in LAM severity, particularly when change is defined by FEV1, perhaps the most clinically relevant and prognostically important variable in LAM. The constellation of results here support the validity of the SGRQ as capable of assessing longitudinal change in HRQL in LAM.
  Chest 01/2013; · 5.25 Impact Factor
• Article: Lymphangioleiomyomatosis: calling it what it is: a low-grade, destructive, metastasizing neoplasm.

  Francis X McCormack, William D Travis, Thomas V Colby, Elizabeth P Henske, Joel Moss
  American Journal of Respiratory and Critical Care Medicine 12/2012; 186(12):1210-2. · 11.08 Impact Factor
• Article: The way forward in lymphangioleiomyomatosis: a trial for every patient, every patient in a trial.

  Francis X McCormack
  Jornal brasileiro de pneumologia: publicacao oficial da Sociedade Brasileira de Pneumologia e Tisilogia 08/2011; 37(4):422-3.
• Article: Diffuse cystic lung disease at high-resolution CT.

  Danielle M Seaman, Cristopher A Meyer, Matthew D Gilman, Francis X McCormack
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  ABSTRACT: OBJECTIVE: This article will illustrate and describe the spectrum of diseases associated with air cysts at high-resolution CT (HRCT). CONCLUSION: HRCT is an important modality in the evaluation of interstitial lung disease to include cystic lung disease. Although most commonly associated with lymphangioleiomyomatosis or Langerhans cell histiocytosis, cystic lung disease is increasingly being recognized as a feature of other entities. Awareness of the spectrum of HRCT findings associated with these diseases may help the trained observer narrow the differential diagnosis.
  American Journal of Roentgenology 06/2011; 196(6):1305-11. · 2.78 Impact Factor
• Article: Efficacy and safety of sirolimus in lymphangioleiomyomatosis.

  Francis X McCormack, Yoshikazu Inoue, Joel Moss, Lianne G Singer, Charlie Strange, Koh Nakata, Alan F Barker, Jeffrey T Chapman, Mark L Brantly, James M Stocks, [......], Gregory P Downey, Eugene J Sullivan, Thomas V Colby, Roy T McKay, Marsha M Cohen, Leslie Korbee, Angelo M Taveira-DaSilva, Hye-Seung Lee, Jeffrey P Krischer, Bruce C Trapnell
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  ABSTRACT: 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. 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)). 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. 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.).
  New England Journal of Medicine 03/2011; 364(17):1595-606. · 53.30 Impact Factor