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Sargramostim Therapy Demonstrates Significant Improvements in Quality-of-Life for Patients With Crohn's Disease

Posted on: Monday, 31 October 2005, 09:00 CST

HONOLULU, Oct. 31 /PRNewswire/ -- Sargramostim therapy was associated with improved and maintained quality-of-life (QOL) in patients with moderately to severely active Crohn's disease, according to data presented at the 70th annual American College of Gastroenterology (ACG) Meeting.

These data from N.O.V.E.L. 1, a multi-center, randomized, double-blind, placebo-controlled Phase 2 trial, demonstrated treatment with sargramostim resulted in significant improvements in quality-of-life from baseline to Day 57 versus placebo, as measured by the Inflammatory Bowel Disease Questionnaire (IBDQ) and the Short Form 36 (SF-36) health surveys. Improvements in QOL ranging from moderate to large occurred early and were maintained throughout the treatment.

"Crohn's disease severely impacts both physical and emotional quality-of- life, and current treatments are often as debilitating as the disease itself," said Brian K. Dieckgraefe, M,D., Ph.D., co-investigator and assistant professor of medicine, Division of Gastroenterology, Washington University School of Medicine, St. Louis. "This study demonstrates that sargramostim offers significant benefits to Crohn's disease patients in terms of improving patient-reported outcomes and facilitating a more active and productive quality-of-life."

Evolving research suggests a primary defect in Crohn's disease may arise from a breakdown in intestinal barrier function composed of several layers of cells that protect the gastrointestinal (GI) tract. This breakdown may precede secondary inflammatory processes associated with Crohn's disease. Sargramostim may address a primary defect of Crohn's disease by helping to improve immune cell function within the intestinal barrier, unlike other therapies that broadly suppress immune response and inflammatory symptoms.

In the United States, sargramostim is marketed as LEUKINE(R) by Berlex, Inc., a U.S. affiliate of Schering AG, Germany.

About the Study

This study was a randomized, placebo-controlled Phase 2 trial evaluating the efficacy and safety of sargramostim 6 µg/kg/day for 8 weeks in patients (N=124) with moderately to severely active Crohn's disease.

The IBDQ, the standard instrument for assessment of health-related quality-of-life (HRQOL) in patients with inflammatory bowel diseases, and the SF-36, a multi-purpose, short-form health survey that is often used as a generic instrument, were used to measure physical, emotional and social outcomes from the patients' point-of-view.

Patients who received sargramostim had a 20% to 22% improvement from baseline total IBDQ Day 29 and thereafter versus 7% to 13% in the placebo group (P = 0.0059 to 0.0397). This was supported by 12% to 20% improvements in the Physical Component Summary score from Day 29 and thereafter, 35% improvements in the General Health subscore of the SF-36 on Day 29 and follow up Day 30 (P = 0.0366 and 0.0133), and 71% to 73% improvements in the Vitality subscore of the SF-36 on Day 57 and follow-up Day 30 (P = 0.0131 and 0.0121, respectively) for patients who received sargramostim. Improvements ranging from 24% to 34% were also observed in the Bowel and Systemic subscores of the IBDQ on Day 29 and thereafter (P = 0.0001 to 0.0384). Bodily Pain was also improved by 39% to 46% on Day 57 and follow-up Day 30 (P = 0.0272 and 0.0077, respectively). The Social subscore of the IBDQ was improved by 17% on follow- up Day 30 versus 4% in the placebo group (P = 0.0288), while the Social score of the SF-36 was improved by 20% to 36% from Day 29 and thereafter (P = 0.0102 to 0.0471). These are considered moderate to large improvements in QOL as indicated by the effect size that ranged from 0.45 to 1.21.

The IBDQ and the SF-36 were completed at baseline; Days 15, 29, 43, and 57 of treatment; and 30 days after treatment. Differences between groups were analyzed by the Van Elteren Test.

About Crohn's Disease

There are approximately 400,000-500,000 cases of diagnosed Crohn's disease in the United States and an estimated one million diagnosed cases worldwide. Crohn's disease affects men and women equally and seems to be more common within families. About 20 percent of people with Crohn's disease have a blood relative, most often a brother or sister, with some form of inflammatory bowel disease (IBD).

The most common symptoms of Crohn's disease are abdominal pain and diarrhea. Other symptoms include fatigue, rectal bleeding, fever, loss of appetite and weight loss.

Crohn's disease can be difficult to diagnose because its symptoms are similar to other intestinal disorders such as irritable bowel syndrome (IBS) and ulcerative colitis. Crohn's disease causes inflammation in the GI tract and usually occurs as an inflammation of the small bowel or colon; however, it can affect any part of the digestive tract from the mouth to the anus. The inflammation from Crohn's disease can extend deeply into the lining of the affected organ, causing pain, diarrhea and frequent bowel movements.

Contact: Marybeth Quane 212-299-8972 Marybeth.Quane@zenogroup.com

Dr. Brian K. Dieckgraefe

CONTACT: Marybeth Quane, +1-212-299-8972, Marybeth.Quane@zenogroup.com

Source: PRNewswire

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