Towards development of treat to target (T2T) in childhood-onset systemic lupus erythematosus: PReS-endorsed overarching principles and points-to-consider from an international task force

Abstract

ObjectivesApplication of ‘treat-to-target’ (T2T) in childhood-onset systemic lupus erythematosus (cSLE) may improve care and health outcomes. This initiative aimed to harmonise existing evidence and expert opinion regarding T2T for cSLE.MethodsAn international T2T Task Force was formed of specialists in paediatric rheumatology, paediatric nephrology, adult rheumatology, patient and parent representatives. A steering committee formulated a set of draft overarching principles and points-to-consider, based on evidence from systematic literature review. Two on-line preconsensus meeting Delphi surveys explored healthcare professionals’ views on these provisional overarching principles and points-to-consider. A virtual consensus meeting employed a modified nominal group technique to discuss, modify and vote on each overarching principle/point-to-consider. Agreement of >80% of Task Force members was considered consensus.ResultsThe Task Force agreed on four overarching principles and fourteen points-to-consider. It was agreed that both treatment targets and therapeutic strategies should be subject to shared decision making with the patient/caregivers, with full remission the preferred target, and low disease activity acceptable where remission cannot be achieved. Important elements of the points-to-consider included: aiming for prevention of flare 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 maintenance of the target over the long-term. An extensive research agenda was also formulated.ConclusionsThese international, consensus agreed overarching principles and points-to-consider for T2T in cSLE lay the foundation for future T2T approaches in cSLE, endorsed by the Paediatric Rheumatology European Society.

Impact of puberty, sex determinants and chronic inflammation on cardiovascular risk in young people

“…Assessment of disease activity in young people with chronic inflammation usually pertain to composite scores aiming to combine objective and subjective assessment of disease manifestations, as well as laboratory parameters which are relevant for systemic inflammation or immune system abnormalities contributing to chronic inflammation ( 151 – 153 ). Despite improvement in quantifying inflammation and improved guidelines to ensure tighter control of disease activity in young people with chronic inflammatory and autoimmune conditions ( 154 – 156 ), there is still evidence of increased CVD-risk in children and young people which is not adequately captured by the CVD-risk assessment tools routinely used for primary care prevention strategies in general population( 157 , 158 ).…”

Therapierefraktäre Verläufe beim juvenilen systemischen Lupus erythematodes

“…Wenn man von einer therapierefraktären Erkrankung spricht, muss man das Behandlungsziel der Therapie mit ins Auge fassen. Dieses Behandlungsziel wurde vor Kurzem von einer PReS-endorsed International Childhood Lupus T2T Task Force [1] erarbeitet und publiziert. Dabei werden patientenbezogene Behandlungsziele festgelegt und in regelmäßigen Abständen überprüft.…”

Rhupus syndrome in the pediatric population: A comprehensive systematic literature review.

“…In its childhood (cSLE) or juvenile (jSLE) expressions, there is a higher disease activity, worsening involvement of several organs and systems that, in turn, give an increased burden of medication and complications to the pediatric and adolescent patients with SLE, resulting in worst standardized mortality rates for cSLE of 18.3, than for aSLE with 3.1. [7] In aSLE the diagnosis uses criteria from the American College of Rheumatology (ACR) or the Systemic Lupus International Collaborating Clinics (SLICC) classification criteria. The SLICC gives more weight to immunologic laboratory findings than the ACR criteria and seems better in pediatrics.…”

“…In its childhood (cSLE) or juvenile (jSLE) expressions, there is a higher disease activity, worsening involvement of several organs and systems that, in turn, give an increased burden of medication and complications to the pediatric and adolescent patients with SLE, resulting in worst standardized mortality rates for cSLE of 18.3, than for aSLE with 3.1. [ 7 ]…”