Practice Analysis and Determining the Knowledge and Skills Expected of a Pediatric Rheumatologist.

OBJECTIVE: The scope of clinical practice of pediatric rheumatology has been difficult to define. The lack of definition prevents an accurate understanding of the knowledge and skills required of practicing pediatric rheumatologists. A practice analysis process was used with the goal of establishing a precise definition of clinical pediatric rheumatology practice. The definition of practice will improve training and the creation of relevant certification examinations. METHODS: A practice analysis approach used meetings with a representative panel of pediatric rheumatologists to create a practice analysis document (PAD) and a test content outline (TCO). Panel experience, entrustable professional activities, and the current TCO were used to guide the process. Surveys were administered to fellowship program directors (PDs) and a broader group of practicing pediatric rheumatologists to revise and validate the content of the documents. RESULTS: A PAD was created, including 14 categories of conditions diagnosed or managed by pediatric rheumatologists and eight domains of practice, with the tasks, knowledge, and skills required to perform these tasks. The survey of PDs (n = 10) indicated that the PAD content is important and useful. A TCO was created and consists of 18 domains used to define content areas to be assessed on certifying examinations. The survey of practicing pediatric rheumatologists (n = 127) indicated that the TCO domains are relevant. CONCLUSION: A practice analysis process produced valuable resources for defining the clinical practice of pediatric rheumatology. The PAD and TCO can be used to develop more specific training curricula and to create relevant certification examinations.

Biologic Agents and Other Emerging Therapies for Childhood SLE.

Systemic lupus erythematosus (SLE) is a multisystem autoimmune disease characterized by remissions and flares. Twenty percent of SLE presents in childhood where the course of SLE is often more severe with significant morbidity and mortality. Several biologic agents have been developed recently for the treatment of lupus, and although some have proven to be safe and efficacious, many have failed to demonstrate significant benefit in clinical trials. There continues to be a desperate need for safe, effective medications that target specific pathway abnormalities seen in SLE. This is an area of intense research that is changing clinical practice in the treatment of childhood SLE. In this article, we discuss the use of B-cell inhibitors, including belimumab and rituximab, as well as the anti-complement drug eculizumab. Promising treatments on the horizon include the jak-stat inhibitors as well as anifrolumab, which targets interferon. [Pediatr Ann. 2022;51(2):e63-e71.].

Pediatric APS: State of the Art.

PURPOSE OF REVIEW: The purpose of this report is to review recent research findings on APS in children and neonates. RECENT FINDINGS: European evidence-based recommendations for diagnosis and treatment of pediatric APS has recently been published by the SHARE Initiative. Recent studies have shown a high prevalence of non-thrombotic manifestations in children with aPL, domains 4/5 specificity of 'innocent' anti-ß2GPI antibodies in infants, and a higher risk for developmental delays and learning disabilities, hence, the need for neurodevelopmental monitoring in children born to mothers with APS. An International effort on creating a new diagnostic criteria for APS is underway. Pediatric APS is a rare disease with significant differences from the APS in adults. Majority of the children with persistently positive aPL do not develop thrombotic events; however, relatively higher proportion of thrombosis in children is related to aPL positivity compared to adults; this may partly be due to the absence of common pro-thrombotic "second-hit" risk factors of adults such as atherosclerosis and cigarette smoking. Diagnosis of APS in children may be delayed or missed when adult APS criteria are used, because in pediatric APS, non-thrombotic clinical manifestations such as thrombocytopenia, hemolytic anemia, and neurological disorders such as migraine, epilepsy, and chorea may precede thrombotic manifestations. Around 20% of the children initially diagnosed with primary APS eventually develop SLE. Neonatal APS is rare; however, the offspring of mothers with APS are at a higher risk for developmental delays and learning disabilities; prematurity and IUGR may increase this risk. Regular assessment of neurodevelopmental status of these children should be performed. Thrombosis is a rare event in the offspring of mothers with APS; anticoagulation of such infants is not recommended. An international effort to create a new diagnostic criteria for APS is underway.

Identifying Targets for Improving Mental Healthcare of Adolescents with Systemic Lupus Erythematosus: Perspectives from Pediatric Rheumatology Clinicians in the United States and Canada.

OBJECTIVE: To identify targets for improving mental healthcare of adolescents with systemic lupus erythematosus (SLE) by assessing current practices and perceived barriers for mental health intervention by pediatric rheumatology clinicians. METHODS: Members of the Childhood Arthritis and Rheumatology Research Alliance (CARRA) completed a Web-based survey assessing current mental health practices, beliefs, and barriers. We examined associations between provider characteristics and the frequency of barriers to mental health screening and treatment using multivariable linear regression. RESULTS: Of the 375 eligible CARRA members, 130 responded (35%) and 119 completed the survey. Fifty-two percent described identification of depression/anxiety in adolescents with SLE at their practice as inadequate, and 45% described treatment as inadequate. Seventy-seven percent stated that routine screening for depression/anxiety in pediatric rheumatology should be conducted, but only 2% routinely used a standardized instrument. Limited staff resources and time were the most frequent barriers to screening. Respondents with formal postgraduate mental health training, experience treating young adults, and practicing at sites with very accessible mental health staff, in urban locations, and in Canada reported fewer barriers to screening. Long waitlists and limited availability of mental health providers were the most frequent barriers to treatment. Male clinicians and those practicing in the Midwest and Canada reported fewer barriers to treatment. CONCLUSION: Pediatric rheumatology clinicians perceive a need for improved mental healthcare of adolescents with SLE. Potential strategies to overcome barriers include enhanced mental health training for pediatric rheumatologists, standardized rheumatology-based mental health practices, and better integration of medical and mental health services.

Neuropsychiatric Systemic Lupus Erythematosus in Children.

Systemic lupus erythematosus (SLE) is a systemic autoimmune disease characterized by the presence of antinuclear antibodies and other autoantibodies, as well as a clinical course that is characterized by flares and remissions. The clinical presentation is diverse, ranging from a mild disease characterized by rash and arthritis to a severe life-threatening disease involving multiple organs. Approximately 25% of children with SLE have neuropsychiatric manifestations of SLE, which are a major cause of morbidity and mortality. Neuropsychiatric symptoms may be the initial presentation of SLE in children. The mortality rate is relatively low, but morbidity may be significant and permanent damage can occur. This article discusses the importance, known pathophysiologic mechanisms, clinical approach, and evidence-based therapeutic options for the diagnosis and management of neuropsychiatric lupus erythematosus in children and adolescents.

Pediatric antiphospholipid syndrome.

Antiphospholipid syndrome (APS) is a multisystem autoimmune condition characterized by vascular thromboses associated with persistently positive antiphospholipid antibodies. There is currently a paucity of data (incidence, prevalence, thrombosis risk, and effective treatment) in pediatric APS. The purpose of this report is to review the current literature on APS in children and neonates, identify the gaps in current knowledge, and suggest avenues for studies to fill those gaps.

A survey of steroid-related osteoporosis diagnosis, prevention and treatment practices of pediatric rheumatologists in North America.

BACKGROUND: The purpose of our study is to assess practices of North American pediatric rheumatologists regarding monitoring, prevention, and treatment of low bone mineral density (BMD) in children on long-term glucocorticoid treatment. Long-term glucocorticoid therapy is associated with accelerated bone loss. Children with JIA and lupus have low baseline BMD and incident vertebral fractures commonly occur in these groups of patients even after a relatively short period of time being on systemic glucocorticoids. There are no established guidelines for identification, prevention, and treatment of glucocorticoid-induced bone loss in children. METHODS: A cross-sectional online survey was conducted with 199 physicians who were listed in the ACR database as practicing pediatric rheumatology in North America. RESULTS: 86 physicians (43%) responded; 87% were board-certified in pediatric rheumatology. 95% used dual energy X-ray absorptiometry as their primary modality for assessing BMD. 79% "rarely" or "never" obtained a baseline BMD measurement prior to initiation of glucocorticoid therapy. 42% of respondents followed BMD annually. 93% "frequently" or "always" prescribed calcium for patients on long-term corticosteroid therapy; 81% "frequently" or "always" prescribed vitamin D. In patients diagnosed with osteoporosis, 35%-50 % of the practitioners "sometimes", "frequently" or "always" prescribed bisphosphonates. Bisphosphonates are prescribed at similar rates for male and female patients, and slightly more frequently for pubertal than for pre-pubertal patients. 96% of respondents "rarely" or "never" prescribed calcitonin for patients on long-term glucocorticoid therapy; 92% "rarely" or "never" prescribe this medication for patients with known osteopenia or osteoporosis. CONCLUSIONS: Utilization of DXA in children on long-term corticosteroid therapy varies greatly among North American pediatric rheumatologists. Most respondents do not screen for low BMD on a regular basis despite acknowledging the risks of bone loss in this population. Broad consensus appears to be present among practitioners favoring the prescription of calcium and vitamin D for patients receiving long-term corticosteroid therapy. Relatively few respondents consistently recommend bisphosphonate therapy, even for patients with known low bone density; calcitonin is rarely used. These data underscore the need for studies to acquire specific data on bone loss, and its prevention and treatment in young patients on long-term glucocorticoid therapy.

Safety and immunogenicity of the quadrivalent HPV vaccine in female Systemic Lupus Erythematosus patients aged 12 to 26 years.

BACKGROUND: Women with SLE have higher rates of persistent human papilloma virus (HPV) infections and precancerous lesions than healthy women. HPV vaccine is safe and effective in healthy females aged 9-26 years. There are limited data on the safety and immunogenicity of HPV vaccine in females with SLE, and none in adolescents with SLE. Our study evaluates the safety and immunogenicity of recombinant quadrivalent HPV vaccine, Gardasil, in adolescents and young women with SLE. METHODS: This is a prospective, open-label study. Exclusion criteria included disease exacerbation within past 30 days; rituximab or cyclophosphamide within 6 months; pregnancy. Vaccine was administered at months 0, 2, and 6. Physical examination, SLEDAI scores and laboratory studies were performed at months 0, 2, 4, 6 and 7. Each patient's SLEDAI scores and laboratory profile in the year prior to vaccine administration were used as controls for that patient. Primary outcome measures were change in SLEDAI and mean HPV antibody titers. RESULTS: 27 patients, 12 to 26 years, were enrolled; 20 completed the study. Nine had mild/moderate lupus flares. Mean SLEDAI scores decreased from 6.14 pre-vaccination to 4.49 post-vaccination (p = 0.01). Of 12 patients with lupus nephritis, two experienced worsening renal function during/after the study and progressed to renal failure within 18 months of the study. Both had Class IV lupus nephritis with high chronicity scores (≥ 8) on renal biopsies performed within one year prior to study entry. Seropositivity post-vaccine was >94% for HPV 6, 11, 16 and 18. CONCLUSIONS: Quadrivalent HPV vaccine seems generally safe and well tolerated in this series of adolescents and young women with SLE, with no increase in mean SLEDAI scores. Progression to renal failure in two patients was most likely secondary to pre-existing severe renal chronicity and not secondary to HPV vaccination. Immunogenicity to the quadrivalent HPV vaccine was excellent, with the seropositivity rate >94% in all four HPV types.