Remission the primary T2T goal in childhood-onset lupus, say new points to consider
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Remission should be the primary goal for patients with childhood-onset systemic lupus erythematosus, according to new points to consider for treat-to-target endorsed by the Pediatric Rheumatology European Society.
The points to consider have been published in the Annals of the Rheumatic Diseases.
“New treatments are not yielding as many positive results in lupus clinical trials as compared to other rheumatic diseases like rheumatoid arthritis,” Eve Mary Dorothy Smith, MD, of the Institute of Life Course and Medical Sciences, at the University of Liverpool, in the United Kingdom, told Healio. “This has made us particularly interested in using treat-to-target (T2T) as a tool to try and standardize treatment and monitoring, optimize use of currently available treatments, and get disease under control more quickly to try and avoid persistence of active disease, flares and longer-term damage.”
To examine and reconcile the existing data and expert opinion on T2T for childhood-onset SLE, and develop points to consider for treatment goals, Smith and colleagues formed an international task force. Initially established in July 2021, this group included two patient representatives, one parent representative and 20 pediatric rheumatologists with experience childhood-onset SLE. Two pediatric and adult rheumatologists, four nephrologists, an adult rheumatologist and members from the task force steering committee were also included.
The task force developed preliminary overarching principles and points to be investigated following a literature review. A virtual consensus meeting was conducted in November 2021, during which the proposed principles and recommendations were shared, along with the survey outcomes evaluating them and any relevant pieces of literature. Following an open comment period, an online poll was conducted. Any recommendation to reach more than 80% consensus on the first vote was adopted. In cases where 80% was not achieved, points were discussed and voted on again until consensus was achieved.
The task force ultimately adopted four overarching principles and 14 points to consider. The overarching principles are:
- All treatment decisions should match the disease manifestations and be agreed upon by provider, caregivers and patients;
- The first treatment goal should be to maintain long-term survival and limit organ damage;
- Disease management requires thorough understanding of potential manifestations; and
- Patients with child-onset SLE require “regular and long-term follow-up.”
Meanwhile, according to the points to consider, the primary treatment target should be remission. However, if patients cannot achieve remission, low disease activity is an acceptable goal. Additional treatment goals should be the prevention of flares, the curbing and prevention of organ damage, the treatment and prevention of antiphospholipid antibody-related morbidity, and the control of comorbidity.
Patients with lupus with clinically inactive disease should be closely monitored, the document continues. Disease factors impacting quality of life should be addressed in a multidisciplinary fashion. The task force recommends that providers monitor kidney function early. Meanwhile, in patients with lupus nephritis following induction therapy, immunomodulatory therapy lasting at least 3 years is recommended, Smith and colleagues wrote. Maintenance therapy should use as low a dose of glucocorticoids as possible.
Unless contraindications exist, all patients should routinely receive hydroxychloroquine, according to the task force. Patients should undergo regular assessments and close monitoring to maintain the target after it is achieved.
“Remission has been emphasized as the main T2T target, with low disease activity state representing an acceptable alternative when this cannot be achieved,” Smith said.
“We have highlighted that both the treatment targets and therapeutic strategies should be decided upon through shared decision making with the patient/caregivers,” she added. “Further goals emphasized in the manuscript include aiming for the prevention of flares and organ damage, glucocorticoid sparing, proactively addressing factors that impact health-related quality of life — fatigue, pain, mental health, educational challenges, medication side effects — and aiming for on-going longer-term maintenance of the target.”
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