CAR T cell therapy for children with rheumatic disease: the time is now.

Initial success with B cell-targeted chimeric antigen receptor (CAR) T cells for the treatment of systemic lupus erythematosus and other rheumatic diseases has generated enthusiasm for the broad application of this technology outside of the field of oncology. Paediatric patients with severe rheumatic diseases require lifelong therapy with a substantial toxicity burden and a high cost of care. Paradigm-shifting treatments, including CAR T cells, are desperately needed.

Relationship between hydroxychloroquine blood levels and lupus activity through the lens of the type 1 and type 2 lupus model: a cross-sectional study.

Introduction: In the type 1 and 2 SLE model, inflammation mediates type 1 manifestations, but its role in type 2 manifestations (eg, fatigue, myalgias, mood disturbance, cognitive dysfunction) is less clear. Therapeutic hydroxychloroquine (HCQ) levels reduce type 1 activity, but their relationship with type 2 activity is unknown. Exploring this relationship may illuminate type 2 SLE pathophysiology.

Development of CARRA/PReS-endorsed consensus Core and Expanded Datasets in childhood-onset systemic lupus erythematosus for international registry-based research.

Objectives: Childhood-onset systemic lupus erythematosus (cSLE), representing 15%-20% of individuals with SLE, has been difficult to study globally due to differences between registries. This initiative, supported by Childhood Arthritis Rheumatology Research Alliance (CARRA) and Paediatric Rheumatology European Society (PReS), aims to create Core and Expanded cSLE Datasets to standardise and enhance research worldwide.

Methods: 21 international cSLE experts and 4 patients participated in a Delphi process (questionnaires, 2 topic-specific focus groups and 3 virtual consensus meetings) to create 2 standardised cSLE datasets.

Bridging the Gap: A Pilot Study of a Health Care Transition Podcast Curriculum and Standardized Patient Case for Graduate Medical Education Trainees.

Introduction Pediatric to adult health care transition (HCT) is critical to maintaining the health and wellness of patients, and pediatric and adult providers often do not feel prepared to shepherd patients through this process. Methods  We designed an HCT curriculum consisting of nine podcasts paired with existing ambulatory experiential learning opportunities for internal medicine-pediatric residents (n=6). Before and after the curriculum we evaluated resident HCT self-assessment and resident performance working with a standardized patient (SP) and standardized parent in a novel objective structured clinical examination (OSCE) station designed to assess HCT skills.

A randomized controlled educational study to evaluate an e-learning module to teach the physical examination of the temporomandibular joint in juvenile idiopathic arthritis.

Background: The aim of the study was to evaluate the effectiveness of a novel e-learning module in teaching the physical exam of the temporomandibular joint (TMJ) in Juvenile idiopathic arthritis (JIA.).

Methods: An e-learning module was developed to convey the TMJ physical examination maneuvers that are considered to be best practice in JIA.

Implementation of a Clinician-led Medication Adherence Intervention Among Patients With Systemic Lupus Erythematosus.

Objective: Medication nonadherence in systemic lupus erythematosus (SLE) leads to poor clinical outcomes. We developed a clinician-led adherence intervention that involves reviewing real-time pharmacy refill data and using effective communication to address nonadherence. Prior pilot testing showed promising effects on medication adherence.

Interpreting hydroxychloroquine blood levels for medication non-adherence: a pharmacokinetic study.

Objective: Characterise the relationship between hydroxychloroquine (HCQ) blood levels and the number of missed doses, accounting for dosage, dose timing and the large variability in pharmacokinetics (PK) between patients.

Methods: We externally validated a published PK model and then conducted dosing simulations. We developed a virtual population of 1000 patients for each dosage across a range of body weights and PK variability.

What the SARS-CoV-2 Pandemic Has Taught Us About Immunosuppression, Vaccinations, and Immune Dysregulation: The Rheumatology Experience.

Purpose Of Review: This review reflects on the impact of the COVID-19 pandemic on the field of rheumatology, emphasizing resulting insights related to the risks of viral infections in immunosuppressed patients, vaccine immunogenicity in immunocompromised patients, and immune dysregulation in the setting of viral infection.

Recent Findings: During the pandemic, global patient registries provided real-time insights into the risk factors associated with severe COVID-19 outcomes in rheumatology patients. Updated evidence-based recommendations from the American College of Rheumatology (ACR) guided rheumatology practice during a time of considerable uncertainty.

Transition to Adult Rheumatology Care: A Disease-Specific Guide.

Young adults with childhood-onset rheumatic diseases are more frequently establishing and continuing care with adult rheumatologists. The transfer of care can be challenging for both the young adult patients and their adult rheumatologists, in large part due to differences between pediatric-onset rheumatic diseases and their adult-onset counterparts, or due to the rarity of some pediatric-onset rheumatic conditions. Other challenges are due to cultural differences between pediatric and adult medical care and to the young adult needing to increasingly perform self-management skills that were previously managed by parents or other caregivers.

The Type 1 & 2 systemic lupus erythematosus model: Perspectives of people living with systemic lupus erythematosus.

Objective: In the new Type 1 & 2 model for systemic lupus erythematosus (SLE), Type 1 SLE represents classic inflammatory manifestations, such as arthritis, while Type 2 SLE encompasses symptoms such as pain and fatigue where the relationship to inflammation is less clear. The objective of this study was to interview individuals living with SLE to determine the content and face validity of the Type 1 & 2 SLE model.

Methods: We conducted a qualitative study using semi-structured interviews with a purposeful sample of participants who met classification criteria for SLE.

A qualitative study of facilitators of medication adherence in systemic lupus erythematosus: Perspectives from rheumatology providers/staff and patients.

Objective: Systemic lupus erythematosus (SLE) disproportionately affects patients from racial and ethnic minority groups. Medication adherence is lower among these patient populations, and nonadherence is associated with worse health outcomes. We aimed to identify factors that enable adherence to immunosuppressive medications among patients with SLE from racial and ethnic minority groups.

Using PROMIS-29 to determine symptom burdens in the context of the Type 1 and 2 systemic lupus erythematosus (SLE) model: a cross sectional study.

Objective: To account for heterogeneity in systemic lupus erythematosus (SLE) and bridge discrepancies between patient- and physician-perceived SLE activity, we developed the Type 1 and 2 SLE model. We examined PROMIS-29 scores, a composite patient-reported outcome (PRO) measure, through the lens of the model.

Methods: Patients completed PROMIS-29 and the polysymptomatic distress scale (PSD).

Patient and Physician Perspectives of Systemic Lupus Erythematosus Flare: A Qualitative Study.

Objective: Systemic lupus erythematosus (SLE) flares are associated with increased damage and decreased health-related quality of life. We hypothesized that there is discordance between physicians' and patients' views of SLE flare. In this study, we aimed to explore patient and physician descriptions of SLE flares.

The impact of pregnancy planning and medical readiness on reproductive outcomes in women with systemic lupus erythematosus.

Women with systemic lupus erythematosus (SLE) who get pregnant while SLE is active or while on teratogens have higher risk of poor pregnancy outcomes. The American College of Rheumatology (ACR) Reproductive Health Guidelines recommend women conceive when SLE is well controlled and treated with pregnancy-compatible medications. The Healthy Outcomes in Pregnancy with SLE Through Education of Providers (HOP-STEP) Intervention was created to ascertain pregnancy interest and contraceptive use followed by a personalized pregnancy prevention and/or planning discussion (https://www.

Renal Response Outcomes of the EuroLupus and National Institutes of Health Cyclophosphamide Dosing Regimens in Childhood-Onset Proliferative Lupus Nephritis.

Objective: We compared clinical characteristics and renal response in patients with childhood-onset proliferative lupus nephritis (LN) treated with the EuroLupus versus National Institutes of Health (NIH) cyclophosphamide (CYC) regimen.

Methods: A retrospective cohort study was conducted at 11 pediatric centers in North America that reported using both CYC regimens. Data were extracted from the electronic medical record at baseline and 3, 6, and 12 months after treatment initiation with CYC.

Real-world use and outcomes of belimumab in childhood-onset lupus: A single-center retrospective study.

Background: Studies of real-world effectiveness of belimumab in adults with systemic lupus erythematosus have shown improved disease control and decreased oral glucocorticoid use. However, belimumab use outside of clinical trial settings has not been well studied in childhood-onset systemic lupus erythematosus (cSLE). We aimed to characterize indications for belimumab use and evaluate oral glucocorticoid doses and disease activity scores in the year following belimumab initiation at a single, large pediatric rheumatology center.

2022 American College of Rheumatology Guideline for Vaccinations in Patients With Rheumatic and Musculoskeletal Diseases.

Objective: To provide evidence-based recommendations on the use of vaccinations in children and adults with rheumatic and musculoskeletal diseases (RMDs).

Methods: This guideline follows American College of Rheumatology (ACR) policy guiding management of conflicts of interest and disclosures and the ACR guideline development process, which includes the Grading of Recommendations Assessment, Development and Evaluation (GRADE) methodology. It also adheres to the Appraisal of Guidelines for Research and Evaluation (AGREE) criteria.

2022 American College of Rheumatology Guideline for Vaccinations in Patients With Rheumatic and Musculoskeletal Diseases.

Objective: To provide evidence-based recommendations on the use of vaccinations in children and adults with rheumatic and musculoskeletal diseases (RMDs).

Methods: This guideline follows American College of Rheumatology (ACR) policy guiding management of conflicts of interest and disclosures and the ACR guideline development process, which includes the Grading of Recommendations Assessment, Development and Evaluation (GRADE) methodology. It also adheres to the Appraisal of Guidelines for Research and Evaluation (AGREE) criteria.

American College of Rheumatology Guidance for COVID-19 Vaccination in Patients With Rheumatic and Musculoskeletal Diseases: Version 5.

Objective: To provide guidance to rheumatology providers on the use of COVID-19 vaccines for patients with rheumatic and musculoskeletal diseases (RMDs).

Methods: A task force was assembled that included 9 rheumatologists/immunologists, 2 infectious diseases specialists, and 2 public health physicians. After agreeing on scoping questions, an evidence report was created that summarized the published literature and publicly available data regarding COVID-19 vaccine efficacy and safety, as well as literature for other vaccines in RMD patients.