Every family whose child receives a diagnosis of a rheumatic disease (such as juvenile idiopathic arthritis (JIA), lupus, dermatomyositis and scleroderma) wants the best treatment and care. The doctors who treat your child also want to provide the best treatment and care for their patients. Over the past 20 years, new discoveries and medicines have greatly improved the lives of children with rheumatic diseases. Many new treatment choices are good, but how can we know which medication will work the best for each child? What if we can learn the answer to this question by simply collecting information on how usual care and treatment work for a lot of children with the same disease?
The usual way of deciding whether a treatment works for a disease is through large research studies called randomized controlled trials (RCTs). In RCTs, many patients must be recruited to be assigned to groups receiving the treatments being studied. These assignments are “randomized” (using a program which is like flipping a coin). The patients and the doctors usually don’t know which treatment the patient is being given. The patients in the different treatment groups are then studied over time to see if the one getting one treatment does better than the ones on the comparison treatment. The comparison treatment is often a placebo (a “sugar pill” that doesn’t contain any active drug). This research method is very costly, often takes a long time to get answers and is very difficult to enroll the large numbers of patients needed when a disease is rare (such as most pediatric diseases).
Because there are no clear-cut studies to help doctors, patients and their families choose one treatment over the other. The Childhood Arthritis & Rheumatology Research Alliance (CARRA) has developed a new way of studying how treatments work called Consensus Treatment Plans (CTPs). CTPs standardize these treatments, so that current treatment approaches can be studied and compared to each other in a new and different way.
In 2009, Pediatric Rheumatologists from CARRA had the idea to develop and then compare standardized CTPs for JIA, lupus, dermatomyositis and localized scleroderma. STOP-JIA and FROST are some examples of studies that are currently ongoing and use CARRA CTPs.
So, how does this work for patients? When a patient is diagnosed with a disease, the doctor discusses the different CTPs available with the patient and family. Then together they may select the one they feel will work best for the disease and the family. Each patient is followed regularly over time and information is gathered at each visit about the patient’s disease activity, and how the patient and the family are feeling.
CARRA created a secure, reliable, and uniform way to collect patient data called the CARRA Registry. The registry collects information from many doctors and patients to help doctors learn which CTP works best for patients. Patients can sign up in their doctor’s office and agree to add their identity-protected data to the registry and answer some questionnaires about how they feel.
When enough patients who are on the standardized CTP treatments have added data to the Registry, doctors can compare which of these treatments work best for a particular disease, and which treatments are safest. Until then, having standardized CTPs will help to guide all doctors to give standardized care for their patients, which will improve how patients do overall. If you’re interested in CTP treatments, please talk to your pediatric rheumatologist.
The Arthritis Foundation provides funding to CARRA and is a partner on this project.