Pediatric Experience Research Articles

Knowledge, attitude, and practice of physicians towards familial hypercholesterolaemia in north regions of Saudi Arabia.

To assess knowledge, attitude and practice of physicians regarding familial hypercholesterolaemia. The cross-sectional study was conducted from October to December 2022 in Aljouf, Tabouk, Hail and northern border regions of Saudi Arabia after approval from the ethics review committee of Qurayyat Health Affairs. Those included were physicians specialising in familial hypercholesterolaemia and having experience in family medicine, endocrinology, paediatrics, cardiology, internal medicine and obstetrics and gynaecology. Their knowledge, attitude and practice were assessed using a predesigned questionnaire. Data were analysed using SPSS 20. Of the 430 subjects, 220(51.2%) were males, 89(20.7%) were aged 36-40 years, 169(39.3%) were residents, and 146(34%) had experience >15 years. Overall, 270(62.8%), 286(66.5%) and 287(66.7%) subjects did not know about the clinical diagnostic algorithms of Simon Broom, Dutch Lipid Clinic Network and United States MedPed programme investigated criteria, respectively. There were, 149(34.7%) subjects with high awareness level, and the level of awareness was significantly affected by gender, level of training, medical specialty, and familiarity with familial hypercholesteremia (p<0.05). There were 343(79.8%) subjects who said they had never diagnosed familial hypercholesterolaemia. There were knowledge gaps, low awareness, and inappropriate practices related to familial hypercholesterolemia, indicating the need for targetted programmes.

Promoting Safe and Supportive Health Care Spaces for Youth Experiencing Racism

ObjectiveThis qualitative study applies a community-based participatory research approach to elicit formative data on pediatric patient experiences of racism in the health care setting and to explore clinic-based opportunities for supporting pediatric patients experiencing racism. MethodsThe study is situated within the outpatient practice of a large tertiary academic medical center in a midsize Midwestern city. Community partners were involved in all aspects of the research, including research protocol design, recruitment, data analysis, community dissemination, and manuscript preparation. Participants were youth between 11 and 18 years, in middle or high school, self-identifying as a person of color, Latinx or Indigenous who answered yes to the question “have you ever experienced race-related prejudice and discrimination?” Parent/guardians of youth meeting inclusion criteria participated in separate focus groups. Data were analyzed using an interpretative phenomenological analysis approach. ResultsMajor findings were divided into 2 categories: 1) racism-related experiences in the health care setting; and 2) patient and parent/guardian recommendations to support pediatric patients experiencing racism. Among health care setting experiences, primary emerging themes included racism experienced in the health care setting, patient-clinician communication around racism, patient-clinician concordance, and high-quality clinical care. Recommendations were presented within the 4 domains of racism: intrapersonal, interpersonal, structural, and institutional. ConclusionsRacism experiences worsen child biological, psychological, and behavioral functioning, yet research is lacking on how health care professionals may best support pediatric patients experiencing racism. Study findings suggest opportunities for providing safer and more supportive health care spaces for youth experiencing racism.

Association between red blood cell transfusion and mortality in critically ill children: a single-center pediatric intensive care experience

Aims: Our study aimed to evaluate the association of erythrocyte transfusion with mortality and morbidity in critically ill children and to emphasize the importance of the risk it carries. Methods: A retrospective evaluation of 524 pediatric patients aged 1 month to 18 years who had been admitted between February 2022 to March 2023 at Sancaktepe Şehit Prof. Dr. İlhan Varank Training and Research Hospital, were performed. Children with hemoglobinopathies, and chronic anemia (defined as the presence of anemia for more than 6 weeks) were excluded. Demographic data, clinical variables, and outcome data were analyzed. Results: The median age was 36.5 (1.0-272.0) months, and 56.1% of the patients were male. The median duration of a pediatric intensive care unit stay was 5 (1-114) days. Respiratory diseases (44.7%) were the most common reasons for admission to the pediatric intensive care unit, followed by, neurological diseases (12.8%)and sepsis (11.3%). Mortality rate was 5.7%. The median baseline hemoglobin level upon admission was 10.5 g/dl (3.2-18.8). Severe anemia (hemoglobin &amp;lt; 7 g/dl was present in 6.1% of all patients. One hundred and sixteen (22.1%) patients were transfused; 61 (11.6%) were transfused only once. A total of 292 packed red blood cells transfusions were administered. Transfused patients required prolonged hospital stays, support for inotropic agents, invasive mechanical ventilation, and extracorporeal treatment and had an excessive mortality rate (p&amp;lt;0.001, p&amp;lt;0.001, p&amp;lt;0.001, p&amp;lt;0.001, p&amp;lt;0.001). Conclusion: Clinicians should consider that the administration of packed red blood cells transfusions in critically ill patients may have the potential to both raise mortality and provide life-saving benefits during their pediatric intensive care unit stay. As with any treatment with potential side effects, it is essential to reduce the use of unnecessary blood products.

Social Responsibility and Commitment to Children; Pediatric Nurses' Experiences With Redeployment During the First Wave of COVID-19: A Qualitative Study.

COVID-19 represented a challenge for health care worldwide and led to new tasks and a rethinking of resource use. It was necessary to establish capacity within hospitals and to reassign critical resources between hospitals. This study aimed to explore pediatric nurses' experiences of redeployment, new tasks, and use of specialized competencies during the first wave of COVID-19. An exploratory design, involving qualitative individual interviews with 12 pediatric nurses was used. The analysis resulted in 3 main themes. Firstly, a feeling of social responsibility focused on how pediatric nurses felt committed to use their competencies during the crisis, whether they remained on the pediatric ward or were redeployed. Secondly, fewer children to counterbalance the shortage of pediatric nurses showed how redeployment was offset by fewer pediatric patients. Thirdly, adapting pediatric nursing competencies to new tasks described how the nurses adapted their skills to new tasks either in pediatric or adult wards. The results revealed that pediatric nurses had a social and ethical commitment to society in a crisis. They agreed to be redeployed and take on new tasks but were still concerned about the health and well-being of the children and their families, which led to a sense of ambivalence. They questioned whether their skills were being used appropriately in redeployment to adult wards. Fewer pediatric patients mitigated the workload of the remaining nurses. There is a risk of neglecting the needs of hospitalized children and their families during a pandemic. There was concern that "voluntary coercion" was a counterproductive strategy for reassignment.

Early phase clinical trials in pediatric oncology: Swedish pediatric oncologists' experiences of balancing hope and expectations in life-threatening illnesses.

To study Swedish pediatric oncologists' practical and emotional experiences of referring, including and/or treating children in early-phase clinical trials. A nationwide study was conducted using a mixed-method approach. Structured interviews based on a study-specific questionnaire and participants' personal reflections were utilized. Survey responses were analyzed using descriptive statistics, while participants' comments were analyzed using thematic analysis. All interviews were recorded and transcribed verbatim. In total, 29 physicians with 4 to 32 years of experience in pediatric oncology participated, with 19 (66%) having > 10 years of experience. Three themes appeared: 1) Optimization-based approach focused on finding the most suitable treatment and care for every child with a refractory/relapsed cancer eligible for an early-phase clinical trial; 2) Team-based approach aimed at establishing local and national consensus in decision-making for treatment options, including early-phase clinical trials and palliative care; 3) Family-based approach in which the physicians provided families with actionable information, listened to their desires, and endeavored to maintain hope in challenging circumstances. Several participants (40% with ≤ 10 years of experience and 58% with > 10 years of experience) viewed the early-phase clinical trial as a potential "chance of cure". A majority (80%) of physicians with ≤ 10 years of experience, reported that they often or always felt personally and emotionally affected by communication regarding early-phase clinical trials. Delivering difficult news in cases of uncertain prognosis was identified as the major challenge. None of the study participants felt adequately prepared in terms of sufficient knowledge and experience regarding early-phase clinical trials. The physicians expressed a need for guidance and training in communication to address these challenges. Working with early-phase clinical trials highlight a field where physicians cannot solely rely on their expertise or past experiences, and where they are likely to be deeply emotionally involved. Physicians who care for children eligible for such studies require targeted educational initiatives and supervision.

Learner Influence on Pediatric Patient Experience: Exploring Family Members’ Perspectives

Learner Influence on Pediatric Patient Experience: Exploring Family Members’ Perspectives

Investing in Child Health Through Alternative Payment Models: Lessons From North Carolina Integrated Care for Kids.

Pediatric value-based payment reform has been hindered by limited return on investment (ROI) for child-focused measures and the accrual of financial benefits to non-health care sectors. States participating in the federally-funded Integrated Care for Kids (InCK) models are required to design child-centered alternative payment models (APMs) for Medicaid-enrolled children. The North Carolina InCK pediatric APM launched in January 2023 and includes innovative measures focused on school readiness and social needs. We interviewed experts at NC Medicaid managed care organizations, NC Medicaid, and actuaries with pediatric value-based payment experience to assess the NC InCK APM design process and develop strategies for future child-focused value-based payment reform. Key principles emerging from conversations included: accounting for payer priorities and readiness to implement measures; impact of data uncertainty on investment in novel measures; misalignment of a short-term ROI framework with whole child health measures; and state levers like mandates and financial incentives to promote implementation.

Advances in the diagnostics and treatment of juvenile dermatomyositis

Juvenile dermatomyositis (JDM) is by far the most frequent inflammatory myopathy in childhood and adolescence. It is clinically characterized by inflammatory changes of the skin and muscles but as amultisystemic disease can also affect the skeletal system, the gastrointestinal tract, lungs and heart. Intrinsic (multigenetic risk) and extrinsic factors (triggers) are involved in the pathogenesis resulting in endothelial damage, involvement of fascies, activation of the interferon system and autoimmune reactions including formation of myositis-specific autoantibodies (MSA). In contrast to dermatomyositis in adults, in children and adolescents there are no associations with malignant diseases. The variable expression, the rarity of the disease and the risk of long-term damage and complications necessitate pediatric rheumatological experience in the diagnostics and treatment. Recently, new approaches in drug treatment have substantially improved the outcome and prognosis but amultidisciplinary treatment (including physicians, physiotherapists, psychologists, social workers) is mandatory, especially in the first phases of the disease. Particularly important is aprofessionally correct treatment of the functional sequelae, which are aparticular focus of this article.

The STAMPPed Initiative for Pediatric Nurse Practitioner Students.

As the demand for nurse practitioners increases to meet health care needs, pathways to facilitate the transition into specialty advanced practice nursing education must be developed. The STudent Advisor/Mentorship Program for Pediatrics (STAMPPed) program was an innovative strategy established to support graduate nursing students with little to no pediatric clinical experience who demonstrated an interest in becoming pediatric nurse practitioners. The program provided structured guidance and support from experienced pediatric nursing faculty through monthly group meetings and the formulation of one-to-one student-faculty mentoring dyads. Mentees found the program to be helpful and foster supportive relationships.

Second infant spinal anesthetic: Incidence, dose modification, and adverse events after initial failure.

Infant spinal anesthesia is an important technique in premature and ex-premature infants undergoing lower abdominal surgery. Previous studies of infant spinal anesthesia report high failure rates, but fail to adequately identify contributing factors. The aim of this study is to retrospectively review spinal anesthetics from a quaternary anesthetic centre to determine overall spinal failure rate, incidence of second spinal attempts and adverse events associated with a second spinal anesthetic. A retrospective review of infant spinal anesthetics performed between May 2016 and June 2023. Five hundred and fifty-one infants (mean postmenstrual age 42.9 weeks and weight 3873 g) were included. The overall success rate on first attempt was 86.5% with a further 5.1% requiring a successful second spinal anesthetic after initial failure. Spinal anesthetic failure requiring conversion to general anesthesia occurred in 9.4% of cases The causes of failed spinal anesthetic were inability to access the subdural space (dry tap 4.2%), inadequate motor blockade (2.9%), and repeated bloody taps (2.2%). Spinal anesthetic failure was significantly increased in cases where the anesthetist was routinely performing less than 5 spinal anesthetics per year [OR 2.21 (95% CI 1.28, 3.83, p = .004)] but only weakly associated with years of pediatric anesthetic experience. Failure rates were 21.4% with styletted spinal needles and 9.2% for non styletted [OR 2.68 (95% CI 1.23-5.86, p = .012)]. The incidence of perioperative apnoea was 6.7% with the highest rate in infants in which failed spinal anesthesia required conversion to general anesthesia (25%). There were 28 cases where initial spinal anesthetic failed to produce adequate anesthesia and a repeat spinal anesthetic was performed. Repeat spinal anesthesia was successful in 92.8% of cases with awake caudal anesthesia successful in 7.2% of cases. In three cases high spinal blockade occurred, one after a single spinal and two after a repeat spinal. Both repeat spinal high block cases required intubation and brief resuscitation. Infant spinal anesthesia is associated with high success rates if experienced anesthetists are present or performing the block. Repeat spinal anesthesia may be associated with an increased incidence of high spinal block. Greater awareness of the slow onset of high block should promote techniques aimed at minimizing cephalad spread of local anesthetic including slight head up positioning during surgery.

A Content Analysis of Transgender Health and Wellness Themes Shared Through Social Media.

Transgender and gender-diverse (TGD) adolescents experience limited access to gender-affirming care (GAC) and information and use social media platforms for informational and social support. We conducted conventional content analysis of posts on the platform, Tumblr and applied univariate statistics to characterize health and wellness themes represented by this content. Content was predominantly transmasculine-relevant. Posts addressing the trans health care paradigm often co-occurred with content referencing affirmation logs, the physical and emotional impact of affirmation, surgery, and unspecified medical interventions. Gender-affirming hormone therapy was the most prevalent intervention referenced in posts related to non-professional/non-licensed medical care and advice requests. Transgender and gender-diverse youth-serving individuals can use this information to mitigate harm, enhance patient education, and improve the overall well-being of TGD youth. Further research is needed to characterize the effect of content encountered on social media on pediatric patient experiences and on outcomes of GAC.

Gemtuzumab ozogamicin for relapsed or primary refractory acute myeloid leukemia in children-the Polish Pediatric Leukemia and Lymphoma Study Group experience.

Gemtuzumab ozogamicin (GO), one of the first targeted drugs used in oncology, consists of an anti-cluster of differentiation 33 (CD33) monoclonal antibody bound to a derivative of cytotoxic calicheamicin. After the drug withdrawn in 2010 due to a significantly higher rate of early deaths, GO regained approval in 2017 for the treatment of newly diagnosed, refractory, or relapsed acute myeloid leukemia (AML) in adults and children over 15 years of age. The objective of the study was a retrospective analysis of clinical characteristics, treatment outcomes, and GO toxicity profile in children with primary refractory or relapsed (R/R) AML treated in Poland from 2008 to 2022. Data were collected through the Polish Registry of Acute Myeloid Leukemia. From January 2008 to December 2022, 35 children with R/R AML were treated with GO in seven centers of the Polish Pediatric Leukemia and Lymphoma Study Group. Most of the children (30 of 35) received only one GO cycle in combination with various chemotherapy cycles (IDA-FLA, DOXO-FLA, FLA, FLAG, and others). Eighteen children (51%) achieved complete remission (CR), 14 did not respond to treatment, and three progressed. GO therapy was followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT) in 18 children in CR. The 5-year overall survival (OS) after GO therapy was 37.1% ± 8.7% for the total cohort. There was a trend toward a superior outcome in patients with strong expression of CD33 expression (over 50% positive cells) compared with that in patients with lower expression of CD33 (OS, 41.2% ± 11.9% versus 27.8% ± 13.2%; p = 0.5; 5-year event-free survival, 35.4% ± 11.6% versus 25.7% ± 12.3%; p = 0.5, respectively). Children under 15 years have better outcome (OS, 34.9% ± 10.4% versus 30% ± 14.5%, p = 0.3). The most common adverse events were bone marrow aplasia, fever of unknown origin, infections, and elevated liver enzyme elevation. Sinusoidal obstruction syndrome occurred in two children. The use of GO in severely pretreated children, including those under 15 years of age, with previous failure of AML treatment is a feasible and effective bridging therapy to allo-HSCT with an acceptable toxicity profile.

ABO Incompatible Living Donor Liver Transplantation in Children: A Single Centre Experience from India

BackgroundIn recent years, pediatric ABO incompatible (ABOi) living donor liver transplant (LT) has shown promising outcomes and can potentially eliminate organ shortage. This study aims to report pediatric ABOi LT experience including short- and long-term outcomes. MethodsIt is a single-centre retrospective study. Out of 108 LTs, 20 were done in children. We compared the outcomes between ABOi (n=20) and non-ABOi (n=220) pediatric living donor liver transplantation (LDLT) performed during the study period. All the children received pre-LT desensitization therapy comprising rituximab and plasmapheresis targeting pre-LT isohemagglutinin (IHA) titres of ≤1:16. ResultsOut of 239 pediatric LDLT from 2017-2022, 19 children (11 females) underwent 20 ABOi LT (including one retransplant with ABOi domino allograft) at a median age of 12(12,51) months with majority being biliary atresia (60%). The median change in CD19 cell%, CD20 cell%, and IHA titres after rituximab from day -14 to day -1 (before LT) was satisfactory. In the first 3 months following LT, acute cellular rejection, culture proven sepsis, biliary and vascular complications were seen in 10%, 20%, 20%, and 15% respectively. None of the ABOi LT recipients developed antibody mediated rejection. ABOi LT recipients as compared to non ABOi LT had higher incidence of bile leak and prolonged hospital stay with rest of the complications including biliary strictures and long-term outcome being comparable. At a median follow up of 21(14,33) months, 4 children expired (21%). ConclusionABOi LT in children shows excellent outcomes and can be performed safely with prior desensitization when compatible liver is unavailable.

Paediatric nurses' experiences of success and failure in first-time peripheral intravenous catheter insertion: A qualitative study

PurposeTo explore in-depth the thoughts, anxiety factors, and experiences of paediatric nurses regarding the success and failure of first-time peripheral intravenous catheter insertion. Design and methodsThe study employed a qualitative research method using the phenomenology design. Face-to-face semi-structured in-depth interviews were conducted with nurses (N = 12) working in the paediatric service and neonatal intensive care unit of a teaching and research hospital. The transcripts were analysed using a phenomenological approach and an inductive process. ResultsFour themes were identified: Peripheral Intravenous Catheter Insertion, Experience of Success on the First Attempt, Experience of Failure on the First Attempt, and Proposed Solutions. Nurses felt a sense of happiness when they successfully inserted a peripheral intravenous catheter on their first attempt in paediatric patients. Conversely, they experienced emotions such as sadness, worry, stress, and anxiety when they failed and had to make repeated attempts. ConclusionUnsuccessful peripheral intravenous catheter insertions cause distress and anxiety for nurses, patients, and parents alike. Therefore, nurses' experiences of peripheral intravenous catheter insertions in healthcare settings should not be ignored and require improvement. Implications for clinical practiceUnsuccessful peripheral intravenous catheter insertions affect children, parents, and nurses negatively, thus reducing the success rate of repeated peripheral intravenous catheter insertions. Therefore, it is advisable for nurses conducting paediatric peripheral intravenous catheterisations to undergo periodic training. A specialised intervention team should be established for cases of initial failure, and a mentoring system between seasoned and new nurses is recommended.

Questions to promote child-centered care in racially discordant interactions in pediatric oncology

ObjectiveTo examine questioning practices in racially discordant interactions and describe how these practices engendered child-centered care. MethodsWe used applied conversation analysis to analyze a collection of 300 questions directed to children across 10 cases involving children of color and their families in disease reevaluation appointments in pediatric oncology. ResultsOur analysis generated two patterns: 1) both the pediatric oncologists’ and caregivers built upon one another’s talk to enable the child’s conversational turn, and 2) the oncologists’ reformulated requests as questions to invite the child’s permission and cooperation for completing exams and understanding symptoms. ConclusionChildren, pediatric oncologists, and caregivers coordinated their actions to enable children to participate as recipients of and respondents to questions. The analysis of real-time interactions illuminates practices for centering children in clinical encounters and the benefits of doing so. Practical implicationsThis study’s findings have implications for defining competencies and practices for fostering child-centered communication, creating training materials based on real-time encounters, and identifying strategies for humanizing pediatric patient experiences.

SARS-CoV-2 reinfections in the pediatric cohort-a single-center experience.

This study focused on timelines of infection episodes and dominant variants and aims to determine disease severity and outcome of pediatric patients with reinfection. This study retrospectively evaluated the medical records of the hospitalized patients and/or outpatients aged 0-18 with a positive severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) polymerase chain reaction between March 2020 and September 2022 at Ege University Children's Hospital. Ninety-one pediatric patients reinfected with SARS-CoV-2 were included in the study. There was an underlying disease in 26.4% of the patients. The median time between the two infection episodes was 184 (90-662) days. There were 24 patients (26.3%) with the first infection in pre-Delta period; 17 (18.6%) of them were reinfected in Omicron BA.1 period, while 7 (7.6%) in Omicron BA.4/BA.5 period. Forty-five patients (49.4%) were infected initially in the Delta period; 35 patients (38.4%) were reinfected in the Omicron BA.1 period, while 10 patients (10.9%) were reinfected in the Omicron BA.4/BA.5 period. Twenty-two patients (24.1%) had the first infection in the Omicron BA.1 period and then reinfected in the Omicron BA.4/BA.5 period. Patients with reinfection more frequently displayed a symptom (84.6% vs. 94.5%, p = 0.03). The hospitalization rate significantly declined in reinfection (15.3% vs. 7.6%, p = 0.03). Severe disease, treatment needs and steroid use were decreased in reinfections without a significant difference (p > 0.05). Intensive care unit admission was not altered. This study revealed that reinfections frequently develop in previously healthy children but do not cause more severe outcomes. The risk of symptomatic reinfections is still high due to the effect of the Omicron variant.

Virtual Reality and 3D Simulation in the Treatment of Pediatric Patients with Central Nervous System Tumors

Pediatric central nervous system tumors are the primary solid malignancies in children and remain a leading cause of mortality in infancy. Advances in pediatric neuro-oncology, driven by molecular oncology research, emphasize the critical need for high-quality pathological tissue to support advanced molecular investigations. However, the vast heterogeneity of these tumors requires precise discrimination of collection sites, aligning with preoperative imaging data. Surgical resection, a pivotal step in diagnosis and treatment, could result in potential morbidities influencing children's neurological status. This, in turn, affects the feasibility of subsequent oncological treatments, influencing overall prognosis and quality of life. To address these challenges, technological tools enhance neurosurgeon orientation in pre-surgical planning and resection. While stereotactic navigation systems reduce morbidity, limitations persist in providing only two-dimensional anatomical information. Recent developments in 3D surgical simulation and virtual reality revolutionize procedural planning, offering real-time integration with intraoperative navigation systems. Beyond surgery, virtual reality has potential in case discussions, preoperative planning, and operative guidance, aiming to improve care and patient outcomes. The virtual reality experience, coupled with detailed anatomical visualization, facilitates meticulous surgical strategy planning for minimal invasiveness. Despite expanding literature on virtual reality applications in neurosurgery, pediatric neurosurgical oncology experiences remain limited. Scientific evaluation of simulation systems' impact on techniques and outcomes, combined with advances in neuroimaging, offers promise for adapting surgical approaches based on neoplastic brain lesion behavior.&#x0D; In conclusion, incorporating 3D surgical simulation and virtual reality technologies in pediatric neurosurgical oncology holds substantial benefits, offering improved procedural planning, enhanced precision, and patient-specific adaptation. Despite limited reported experiences, the compelling advantages underscore the need for further exploration and consideration in the evolving landscape of pediatric neuro-oncology.

Clinical Characteristics and Treatment Outcomes of Idiopathic Multicentric Castleman Disease: Data from a Pediatric Cohort

Background: Idiopathic multicentric Castleman disease (iMCD) is a rare, atypical hematologic disorder characterized by diffuse lymphadenopathy and systemic inflammation causing potentially fatal multi-organ system dysfunction. Though the etiology remains unknown, interleukin-6 (IL-6) has been implicated in disease pathogenesis and siltuximab, a monoclonal antibody that antagonizes IL-6, is the only FDA-approved iMCD treatment. Despite substantial morbidity for children with iMCD, the pediatric experience has not been well characterized. It is unclear whether insights gleaned from adults apply to children or whether siltuximab, which was never prospectively studied in a pediatric population, achieves similar response rates in children. We sought to correct this knowledge gap and better characterize pediatric iMCD. Methods: The ACCELERATE longitudinal Castleman disease (CD) natural history registry was interrogated for patients under 18 years of age at time of diagnosis. For each patient, complete medical history and lymph node biopsy slides were collected and extracted, and a panel of clinicians and pathologists performed independent adjudication of the diagnosis. Demographics - including age at diagnosis, histopathologic subtype, clinical subtype, clinical symptoms, and most severe laboratory parameters present within ± 90 days from date of pathological diagnosis were aggregated. Lastly, treatments administered to the cohort of pediatric patients were inventoried and both clinical response (defined as at least 50% reduction in abnormal clinical and laboratory criteria) and durable response (defined as a positive clinical response lasting ≥ 365 days with no other CD treating drug administered in between) were determined. Results: Nineteen pediatric patients were confirmed to have iMCD, of which 14 (74%) were male and 5 (26%) were female (Table 1A). The mean (standard deviation [SD]) age at diagnosis was 13.6 (4.3), with the youngest patient diagnosed at 2 years of age. Much of the cohort demonstrated hypervascular lymph node histology (89.5%). Clinically, 84% of the patients demonstrated the severe TAFRO (thrombocytopenia, anasarca, fever, renal dysfunction/reticulin fibrosis, organomegaly) subtype while 16% were not otherwise specified. 95% of patients presented with constitutional symptoms and all but one patient presented with fatigue and fevers. All 19 patients presented with organomegaly and fluid retention (anasarca, ascites, pleural effusions, and/or peripheral edema). No patients demonstrated CD-specific skin abnormalities (violaceous lymphocytic papules or cherry hemangiomata) or interstitial lymphocytic pneumonitis. Labs indicated significant systemic inflammation, with mean (SD) C-reactive protein, erythrocyte sedimentation rates, and albumin values of 128.3 (102.7) mg/L, 74.5 (42.5) mm/hr, and 2.7 (0.6) g/dL, respectively. Eighteen (95%) patients presented with anemia, with a mean (SD) hemoglobin value of 9.5 (2.2) g/dL. Sixteen (84%) patients demonstrated platelet abnormalities, with 13 patients presenting with thrombocytopenia and three with thrombocytosis. No patient demonstrated hypergammaglobulinemia. Nine patients received three unique siltuximab regimens: siltuximab ± CS (corticosteroids), siltuximab + sirolimus ± CS, and siltuximab + other (other being defined as all other treatments such as rituximab, eculizumab, and ravulizumab) (Table 1B). Siltuximab ± CS was administered to six patients; 83% (n=5) had a positive response, and 60% of responders achieved a durable response. Lastly, two patients with TAFRO died from their disease. Conclusions: Overall, real-world medical record data from a cohort of pediatric iMCD patients enrolled in ACCELERATE reveals high disease burden within this population. A notable degree of TAFRO, the most severe iMCD subtype, was observed within this pediatric cohort, with clinical manifestations and laboratory abnormalities commonly observed in adult iMCD. This makes this population particularly vulnerable and highlights the need for prompt and accurate treatment. We also observed a high response rate to siltuximab ± CS in this small cohort, supporting its use in pediatric iMCD. Further research examining the effectiveness of siltuximab in a larger cohort of pediatric patients as well as other treatments is needed to identify optimal treatment approaches.

The dual role of body mass index on Juvenile Idiopathic Arthritis course: a pediatric experience.

Despite the well-known role of obesity as risk factor for Juvenile Idiopathic Arthritis (JIA) severity, emerging but limited evidence suggested a similar role for underweight. We investigated the role of body mass index (BMI) across its full spectrum in a cohort of children with JIA.We retrospectively studied 113 children with JIA classified according to the International League of Association for Rheumatology (ILAR) criteria attending our Rheumatology Clinic. The patients underwent a comprehensive evaluation including both clinical and biochemical assessments. According to BMI Z-score, the cohort was divided into five groups as underweight, normal weight, overweight (OW), obesity (OB), and severe OB. Disease activity was calculated by Juvenile Arthritis Disease Activity Score 10 (JADAS-10) joint reduced count and relapses were defined according to Wallace criteria.The mean age of the cohort was 7.43 ± 4.03years. The prevalence of underweight, normal weight, OW, OB, and severe OB was 7.2%, 54.1%, 10.8%, 17.1%, and 10.8%, respectively. Significant higher ferritin levels and erythrocyte sedimentation rate values were found in patients with severe OB and underweight compared to subjects belonging to normal weight, OW, and OB groups. A greater JADAS-10 score was observed in underweight patients and in those with severe OB than other groups. The relapse rate was higher in patients with severe OB and underweight compared to other groups. Conclusions: Both underweight and OB might negatively affect JIA course. Weight control is fundamental in children with JIA to avoid a more unfavourable course of the disease. What is Known: • Obesity represents a well-known risk factor for JIA severity. • The role of underweight in children with JIA is still poorly explored. What is New: • As observed in children with obesity, underweight young patients with JIA seem to experience a more severe JIA course. • Healthy lifestyle promotion in children with JIA is a crucial step in the management of the disease.

Assessing Treatment Burden in Pediatric Hemophilia: Validation of the Observer-Reported Child Hemophilia Treatment Experience Measure (Child Hemo-TEM)

Background: Despite treatment safety and efficacy advancements for children with hemophilia (CwH), home treatment can be complicated and burdensome. Treatment burdens are major contributing factors to adherence issues. The Child Hemo-TEM is a patient-centric outcome measure developed following the United States (US) Food and Drug Administration guidance for patient-reported outcome measure development. Designed to assess the treatment burden for CwH, the measure is a caregiver observer-reported outcome (ObsRO) measure where questions are answered by the caregiver about the child's treatment experience based on what the caregiver has seen, heard, or been told by the child. Aim: The purpose of this study was to psychometrically validate the Child Hemo-TEM ObsRO. Methods: Data was collected through a cross-sectional, web survey from a population of caregivers of CwH aged 2 to &amp;lt; 12 years in the US. The survey consisted of the Child Hemo-TEM, sociodemographic items, child's hemophilia medical history, and supporting items/measures required to assess the measure's psychometric properties. The survey took up to 45 minutes to complete and was administered at a single time point. To be eligible for the study, caregivers needed to be: aged ≥ 18 years; a caregiver of a male child with a diagnosis of severe to moderate hemophilia A or B, with a factor level of &amp;lt; 2%, with or without an inhibitor, and currently receiving either prophylactic or on-demand treatment; and living in the same residence as the child over the past 2 weeks. Recruitment was conducted by a patient advocacy group and a professional market research firm. Recruitment quotas were targeted to ensure a diverse population and mix of frequency of prophylactic treatment(s) and treatment administration type. All psychometric analyses were conducted following an a-priori statistical analysis plan, which included examination of measurement characteristics, item reduction, factor analysis, measure scoring, internal consistency, reliability, and validity. Results: Caregivers ( n=187) of CwH completed the web survey. The respondents were, on average, 37.8 years of age, mothers (84.0%), and predominantly White/Caucasian (74.3%). Twelve items were dropped from the validation-ready, 19-item measure: 5 items due to high ceiling effects at baseline (&amp;gt;50%) mirrored by the Rasch results and 1 item as it was highly correlated with a retained item. Further, as there were only a small number of children who self-injected, 6 items designed to assess child self-injection were removed. The remaining items underwent further psychometric testing. An exploratory factor analysis generated a single, 7-item factor. The Child Hemo-TEM was found to be internally consistent with a Cronbach's α of 0.855. A-priori validity hypotheses were adapted to match the final factor structure. Hypothesized associations were predominantly confirmed with a few exceptions. For convergent validity (Pearson's r significance set at α = 0.05), a few of the associations, although significant, were below 0.30 (Table 1). The Child Hemo-TEM was able to discriminate between levels of self-complaining about their infusions (p &amp;lt; 0.001), psychological stress reported by parent (p &amp;lt; 0.001), and ease/difficulty of their medication in its current form, among other variables. Based on psychometric findings and taking into consideration learnings from the concept elicitation during measure development, the brief, 7-item Child Hemo-TEM was finalized with 1 domain capturing information from all the hypothesized concepts of treatment concerns (3 items), adherence (1 item), physical impact (1 item), and interference with daily life (2 items) (Figure 1). A five-point Likert response scale with ranges from “Never” to “Always”, or a “Don't know” option, is used. Conclusion: The Child Hemo-TEM ObsRO has been found to be conceptually sound with adequate evidence to support its reliability and validity. Incorporation of the measure into both clinical and research settings will help assess the pediatric patient's experience of living with hemophilia as well as aid clinicians in tailoring treatments to patient characteristics and situations. Future studies are needed to assess test-retest reproducibility, sensitivity to change, and meaningful change thresholds of this new measure.