Pilot study testing a combination of immunosuppressive drugs holds promise for children with lupus

Lupus Foundation of America

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A small pilot study involving 12 individuals who developed lupus during childhood showed that treatment using a combination of the immunosuppressive drugs rituximab and cyclophosphamide, given over an 18-month period, can provide continued improvement for at least five years.

The combination therapy also allowed doctors to substantially reduce the total dosage of cyclophosphamide and eliminated the need for large doses of oral steroids, improving health to the point that all twelve study participants reported feeling “like I don’t have lupus anymore.”

Dr. Thomas Lehman and his colleagues at the Hospital for Special Surgery in New York City and the Robert Wood Johnson Medical School at Rutgers University in New Jersey administered the medications two weeks apart at the start of the study, and then again six and eighteen months later. The 12 participants were followed for a period of sixty months. 

Using the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI), a tool used by doctors to measure lupus disease activity, the investigators found that the combination therapy resulted in continued improvement in all areas, a dramatic reduction in mean (average) SLEDAI scores at one year and five years, and a substantial reduction in the mean daily dose of prednisone. Only four of the twelve participants remained on 10 milligrams of prednisone daily, with the others receiving less. 

This approach to treatment holds several major advantages including:

  • The ability to quickly reduce the use of steroids without triggering disease flares.
  • A reduction in the side effects commonly associated with steroid use, such as changes in appearance. 
  • A reduction in overall steroid use, which lowers the risk of serious complications from long term use, such as heart disease, diabetes, cataracts and bone loss. Bone loss is an especially critical issue for young people with lupus who are still developing physically. 

The results of this study suggest that doctors can suppress lupus disease activity using this combination therapy, which is good news for all children with a serious case of lupus.  But the investigators caution that this was a small study involving only a few patients. Larger numbers of participants are required to confirm the results of this study. However, conducting larger studies is a challenge because lupus is less common among children than adults.

Many more children with lupus have been treated using this approach, but the investigators say it will take additional time to make certain that these patients will continue to do well.  Studies of the combination therapy are just beginning in adults. 

Prolonged improvement of childhood onset SLE following systematic administration of rituximab and cyclophosphamide.
Thomas JA Lehman, Chahait Singh, Anusha Ramanathan, Risa Alperin, Alexa Adams, Laura Barinstein and Nandini Moorthy
Pediatric Rheumatology 2014, 12:3 doi: 10.1186/1546-0096-12-3.