Background The Childhood Arthritis and Rheumatology Research Alliance (CARRA) developed consensus treatment plans (CTP) for childhood proliferative lupus nephritis (LN) induction therapy to reduce treatment variability and support comparative effectiveness research comparing efficacy and tolerability of Mycophenolate mofetil (MMF) and intravenous cyclophosphamide (IV CYC). The CTPs specify two immunosuppression regimens (IV CYC, oral MMF) and three corticosteroid regimens (primarily oral, primarily IV, and mixed oral/IV). In addition to provider-reported outcomes, we assessed patient-reported outcomes in a pilot observational study.
Methods We enrolled 41 subjects with childhood SLE from 10 CARRA sites. Subjects had new-onset biopsy proven proliferative LN and were starting MMF or IV CYC. We collected baseline demographics, disease-related features and patient-reported outcomes, including functional disability (Childhood Health Assessment Questionnaire (CHAQ), range 0–3), Health-related quality of life (HRQOL) (Child Health Questionnaire (CHQ), excellent, very good, good, poor, very poor), well-being (CHQ, range 0–10), and pain (CHAQ, 0–10). With the exception of pain, parents reported outcomes for subjects<10 years of age. We report baseline and 12 month follow-up results.
Results The majority of participants were female (83%), mean age was 14 years (SD 2.6). At baseline, SLEDAI ranged from 2–34 (median 13, IQR 10–21) and glomerular filtration rate ranged from 41–151 ml/min/1.73 m2 (median 94, IQR 70–107). Baseline functional ability (CHAQ) ranged from 0–1.75 (n=38) and was abnormal (CHAQ >0) in 55.3% of subjects. By 12 months, there was improvement with 14.8% reporting abnormal functional ability. HRQOL at baseline ranged from excellent to poor (13% excellent, 37% very good, 45% good, 5% poor, n=38) and was improved at 12 months (32% excellent, 48% very good, 16%, good, 4% poor, n=25, see figure 1). Baseline median parent-reported overall wellbeing was 3.0 (IQR 2–5), with 21% reporting doing ‘very well’ (score=0). At 12 months, overall wellbeing had improved (median 0.5, IQR 0–3) with 50% reporting doing ‘very well’. Baseline patient-reported pain ranged from 0 (no pain) to 10 (very severe pain) (median 1, IQR 0–5, n=39). Pain was improved at 12 months (range 0–7, median 0, IQR 0–3, n=27).
Conclusions Although diagnosed with a severe medical condition and experiencing functional disability, most children reported good to excellent QOL and only minimal pain. After 12 months, the proportion of patients reporting excellent QOL increased, however reduced QOL persisted for many. Further study is needed to elucidate the factors impacting overall QOL in children with SLE, and association with different treatment strategies.
Acknowledgements We would like to acknowledge the CARRA Registry Investigators, Dr. Marilyn Punaro for her leadership in the CARRA lupus nephritis CTP development process and Thomas Phillips for his data management assistance. This study was supported by funding from the Arthritis Foundation, Lupus Foundation of America, CARRA and NIAMS.
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