Respiratory societies update clinical guidelines for rare lung disease

The American Thoracic Society and the Japanese Respiratory Society have updated the clinical practice guidelines for the diagnosis and treatment of lymphangioleiomyomatosis, according to a recent news release.

In patients with lymphangioleiomyomatosis (LAM) — a rare, systemic lung disease that affects approximately five per million women of child-bearing age — cystic changes occur in the lung caused by neoplastic smooth muscle-like cells infiltrating the lung. These patients experience a decline in lung function at two to four times the normal rate, as well as repeated lung collapses.

Diagnostic and therapeutic tools for LAM have rapidly improved since 2010 when the LAM guidelines have last been updated by the European Respiratory Society.

A 27-member multidisciplinary committee of clinicians and scientists chaired by Joel Moss, MD, PhD, deputy chief of the National Heart, Lung, and Blood Institute and Francis X. McCormack, MD, director of the division of pulmonary, critical care and sleep medicine at the University of Cincinnati, wrote the new guidelines. Information from a questionnaire by the LAM Foundation was used by the committee to gather input from patients with LAM.

Based on low to moderate quality of evidence, the committee made five essential recommendations for diagnosing and managing patients with LAM:

Patients with abnormal or declining lung function should be treated with sirolimus as opposed to observation.
Patients with problematic chylous effusions should be treated with sirolimus prior to invasive management.
Doxycyline should not be used as treatment.
Hormonal therapy should not be used as treatment.
Patients with LAM–like cystic abnormalities, but no other present characteristics of LAM, should be examined through vascular endothelial growth factor D (VEGF-D) testing first rather than lung biopsy to diagnosis LAM.

“No two patients are alike,” Moss said. “The guidelines cannot take into account all of the complexities that clinicians face when making treatment decisions for patients with a rare lung disease.” Therefore, clinicians must take into account individual patients’ unique clinical circumstances when implementing guideline recommendations.

“The sirolimus and VEGF-D recommendations were based on moderate quality evidence, but overall the evidence base for LAM is limited,” McCormack added. “The literature search only uncovered two randomized, controlled trials. As the field evolves, recommendations regarding hormonal therapies and other interventions are likely to change.”

Research opportunities for each recommendation are included within the guidelines. In a future update, the guidelines will include information about using computed tomography for diagnosis, pneumothorax and pulmonary transplantation in patients with LAM. These updates will be submitted for publication at the end of 2016.

“The LAM guidelines will need to be updated frequently because the pace of discovery in LAM continues to accelerate,” McCormack said. – by Alaina Tedesco

Reference: https://www.sciencedaily.com/releases/2016/09/160915090022.htm