The Researcher’s Summary:
Children with systemic lupus erythematosus SLE (cSLE) often develop more severe organ involvement than adults. And this frequently includes the development of renal disease, called lupus nephritis (LN). Optimal control of lupus nephritis in children (cLN) is one of the most critical problems facing these children. This is because uncontrolled cLN can lead to end-stage renal disease, the need for dialysis and increased likelihood of death.
Unfortunately, the best treatment for these children is currently not known. Traditional research study designs used to determine optimal treatments in adult diseases, such as randomized controlled trials, are often not feasible in children due to fewer patient numbers and ethical concerns. And comparing the effects of treatments provided by various physicians in practice is often not reliable because physicians use so many different treatment approaches and track patients differently.
Thus, historically, many medical providers have simply applied what is learned in adult research studies to the treatment of their children. However, that may not always be valid due to differences related to the underlying disease or to the way children respond to medications.
Another approach for comparing the effects of different treatments is to standardize treatments that are used in routine practice so that they can be reliably compared. Thus, the pediatric rheumatology community, working through the Childhood Arthritis and Rheumatic Disease Alliance (CARRA), recently completed the development of Consensus Treatment Plans (CTPs) for cLN. These provide a standardized approach for the induction and maintenance treatment of proliferative cLN, with the goal of comparing the effectiveness and safety of cyclophosphamide, mycophenolate mofetil and azathioprine, the most commonly used medications for cLN. The CTPs also provide standardized steroid regimens. At many centers, patients with cSLE enroll on a registry (CARRAnet), which allows for data collection.
With the use of these new CTPs, we will be able to collect data to determine how these medications compare for the treatment of cLN. However, prior to conducting a large large-scale comparison, it is necessary to assess the usability of these CTPs in the community. Thus, this proposal aims to demonstrate that the standardization of treatment using CTPs for cSLE therapies among pediatric rheumatologists is feasible, and to obtain initial information about how these treatments compare.