Public Release: 

Oral sirolimus alters the course of DIPNECH syndrome in three patients

American College of Physicians

1. Oral sirolimus alters the course of DIPNECH syndrome in three patients

Abstract: http://annals.org/aim/article/doi/10.7326/L17-0634

URLs go live when the embargo lifts

Sirolimus, which is used to prevent rejection after kidney transplants, has been used to successfully treat three cases of a rare disorder called diffuse idiopathic pulmonary neuroendocrine cell hyperplasia, or DIPNECH syndrome. The syndrome is so rare that there are no clinical recommendations to guide care and, therefore, no proven therapies. The brief case report is published in Annals of Internal Medicine.

DIPNECH syndrome is a precancerous condition that causes proliferation of neuroendocrine cells in the bronchial wall and the formation of tumors. A team of researchers from the University of Tours, France, saw three women with DIPNECH syndrome, all of whom were experiencing shortness of breath. The patients' symptoms were treated with bronchodilators and inhaled corticosteroids. Pulmonary function deteriorated in all three patients, while volume of the carcinoid tumors increased. The team began treating the patients with oral sirolimus (2 mg/day) while continuing the other treatments. However, this dose was lowered to 1 to 1.5 mg/day because the patients could not tolerate the side effects of the original dose. At the lower dose, side effects resolved. After 1 year of treatment, pulmonary function improved in three patients and CT scans showed reduction in both nodule volume and air trapping in two patients and progression in the third.

According to the researchers, these findings show that sirolimus could be a viable treatment for patients with this rare syndrome. To date, no other treatment has been shown to alter the course of DIPNECH syndrome. However, the researchers caution other clinicians with similar patients that further data on the safety and efficacy of drugs in the same class is required before they can be recommended for patients with this rare condition.

Media contact: For an embargoed PDF, please contact Lauren Evans at laevans@acponline.org. To interview with the lead author, Sylvain Marchand-Adam, please email s.marchandadam@univ-tours.fr.

2. FDA precertification pilot program for medical software raises questions about whether new health-related apps will be studied rigorously

Abstract: http://annals.org/aim/article/doi/10.7326/M17-2715

URLs go live when the embargo lifts

Under the U.S. Food and Drug Administration's (FDA) new Digital Health Software Precertification (Pre-Cert) Program seeks to alter the review process for certain marketed digital health products, including apps. The authors of an article in Annals of Internal Medicine outline the benefits and risks of the program.

The Pre-Cert Program was designed to expedite regulatory review for companies that demonstrate quality and organization excellence in software development. Nine companies were chosen to participate in the Pre-Cert pilot program: Apple; Samsung; Verily; Johnson & Johnson; Roche; Fitbit; Pear Therapeutics; Phosphorus; and Tidepool. Under the agreement, the companies are required to provide the FDA with information about their quality management, collect and report postmarket data, and allow FDA staff to visit their sites.

Pre-Cert will focus on free-standing software, such as apps that diagnose or treat disease, rather than medical devices. Since these apps can undergo frequent updating, regulatory flexibility is needed. But the Pre Cert Program may reduce incentives for developers to adequately study their software products before patients rely on them. Pre-Cert may also lead to confusion among physicians and patients, who often use FDA clearance or approval as a proxy for product safety and effectiveness.

The authors conclude that the Pre-Cert Program reduces the prospect of FDA oversight in this area, with unclear outcomes for patients.

Media contact: For an embargoed PDF, please contact Lauren Evans at laevans@acponline.org. To interview with the lead author, Aaron S. Kesselheim, MD, JD, MPH, please contact Elaine St. Peter at estpeter@bwh.harvard.edu or 617-525-6375.

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