The influence of assistance in home-based exercise programmes for individuals with intellectual disabilities.

Background: Regular physical activity (PA) decreases the risk of comorbidities associated with a sedentary lifestyle in individuals with intellectual disabilities (IDs). They also may experience additional barriers that may prevent PA, including access, proper instruction and support. At-home PA programming is a feasible alternative to long-term adherence.

A simple in situ method for optimizing settings for the Einzel lens elements in a focused ion beam.

Ion beams have had an incredible impact on research in the past couple of decades. One major reason for this is the continued development of systems having optimal beam currents that allows one to image more clearly at different spot sizes to include higher currents that allow for faster milling. The advancements for Focused ion beam (FIB) columns have developed rapidly due to the computational optimization of lens designs.

Extracorporeal membrane oxygenation in children receiving haematopoietic cell transplantation and immune effector cell therapy: an international and multidisciplinary consensus statement.

Use of extracorporeal membrane oxygenation (ECMO) in children receiving haematopoietic cell transplantation (HCT) and immune effector cell therapy is controversial and evidence-based guidelines have not been established. Remarkable advancements in HCT and immune effector cell therapies have changed expectations around reversibility of organ dysfunction and survival for affected patients. Herein, members of the Extracorporeal Life Support Organization (ELSO), Pediatric Acute Lung Injury and Sepsis Investigators (PALISI) Network (HCT and cancer immunotherapy subgroup), the Pediatric Diseases Working Party of the European Society for Blood and Marrow Transplantation (EBMT), the supportive care committee of the Pediatric Transplantation and Cellular Therapy Consortium (PTCTC), and the Pediatric Intensive Care Oncology Kids in Europe Research (POKER) group of the European Society of Pediatric and Neonatal Intensive Care (ESPNIC) provide consensus recommendations on the use of ECMO in children receiving HCT and immune effector cell therapy.

Case Discussion and Literature Review: Cancer Immunotherapy, Severe Immune-Related Adverse Events, Multi-Inflammatory Syndrome, and Severe Acute Respiratory Syndrome Coronavirus 2.

Pediatric, adolescent and young adult (AYA) patients receiving novel cancer immunotherapies may develop associated toxicities with overlapping signs and symptoms that are not always easily distinguished from severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection/clinical sequelae. We describe 2 diagnostically challenging cases of SARS-CoV-2 and Multi-Inflammatory Syndrome-Adult (MIS-A), in patients with a history of acute lymphoblastic leukemia following cellular therapy administration and review evolving characterization of both the natural course of SARS-CoV-2 infection and toxicities experienced in younger cancer immunotherapy patients. Vigilant monitoring for unique presentations and epidemiologic surveillance to promptly detect changes in incidence of either condition may be warranted.

Diagnosis, grading and management of toxicities from immunotherapies in children, adolescents and young adults with cancer.

Cancer immunotherapies are associated with remarkable therapeutic response rates but also with unique and severe toxicities, which potentially result in rapid deterioration in health. The number of clinical applications for novel immune effector-cell therapies, including chimeric antigen receptor (CAR)-expressing cells, and other immunotherapies, such as immune-checkpoint inhibitors, is increasing. In this Consensus Statement, members of the Pediatric Acute Lung Injury and Sepsis Investigators (PALISI) Network Hematopoietic Cell Transplantation-Cancer Immunotherapy (HCT-CI) Subgroup, Paediatric Diseases Working Party (PDWP) of the European Society of Blood and Marrow Transplantation (EBMT), Supportive Care Committee of the Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) and MD Anderson Cancer Center CAR T Cell Therapy-Associated Toxicity (CARTOX) Program collaborated to provide updated comprehensive recommendations for the care of children, adolescents and young adults receiving cancer immunotherapies.

The Organ Trail: A Review of Biomarkers of Organ Failure.

Pediatric organ failure and transplant populations face significant risks of morbidity and mortality. The risk of organ failure itself may be disproportionately higher among pediatric oncology patients, as cancer may originate within and/or metastasize to organs and adversely affect their function. Additionally, cancer directed therapies are frequently toxic to organs and may contribute to failure.

Donor-derived cell-free DNA (dd-cfDNA) for detection of allograft rejection in pediatric kidney transplants.

In pediatric transplantation, acute rejection is a major contributor of graft failure. Current approaches include kidney biopsy in response to graft dysfunction and/or the emergence of donor-specific HLA antibodies (DSA). However, biopsy is associated with potential complications.

Safety and efficacy of sucroferric oxyhydroxide in pediatric patients with chronic kidney disease.

Background: Pediatric patients with advanced chronic kidney disease (CKD) are often prescribed oral phosphate binders (PBs) for the management of hyperphosphatemia. However, available PBs have limitations, including unfavorable tolerability and safety.

Methods: This phase 3, multicenter, randomized, open-label study investigated safety and efficacy of sucroferric oxyhydroxide (SFOH) in pediatric and adolescent subjects with CKD and hyperphosphatemia.

Functional Assessment of Fatigue and Other Patient-Reported Outcomes in Patients Enrolled in the Global aHUS Registry.

Introduction: Atypical hemolytic uremic syndrome (aHUS) is a progressive and potentially life-threatening disease characterized by complement-mediated thrombotic microangiopathy. Patients with aHUS may experience fatigue, which can negatively impact their lives, but there is a knowledge gap regarding disease burden in these patients.

Methods: In this longitudinal study, patients with aHUS from the Global aHUS Registry who completed patient-reported outcome assessments (Functional Assessment of Chronic Illness Therapy-Fatigue scale [FACIT-Fatigue], general health status, and work status) at ≥2 time points were assessed relative to treatment status: (i) never treated with eculizumab; (ii) on eculizumab at registry enrollment and continued therapy; and (iii) started eculizumab after registry enrollment.

National Cooperative Growth Study: 25 Years of Growth Hormone Data, Insights, and Lessons for Future Registries.

Background: The National Cooperative Growth Study (NCGS) data are reviewed from 1985-2010 to report on final demographic, efficacy, and safety findings, and to illustrate the value of long-term, real-world follow-up to physicians and patients.

Methods: The NCGS was a multicenter, open-label, observational, postmarketing surveillance study of Genentech growth hormone (GH) products for the treatment of children with growth failure in North America.

Findings: Data from 65,205 patients representing 240,951 patient-years of experience were collected.

Special Considerations in Pediatric Kidney Transplantation.

Universally accepted as the treatment of choice for children needing renal replacement therapy, kidney transplantation affords children the opportunity for an improved quality of life over dialysis therapy. Immunologic and surgical advances over the last 15 years have improved the pediatric patient and kidney graft survival. Unique to pediatrics, congenital genitourinary anomalies are the most common primary diseases leading to kidney failure, many with urological issues.

Cefepime-induced neurotoxicity in a pediatric patient on chronic hemodialysis: a case report.

Impaired renal function increases the risk for cefepime-induced neurotoxicity. Symptoms include disorientation, myoclonus, status epilepticus, ataxia, gait disturbance, coma, and death. A high index of suspicion and early recognition of symptoms can minimize the risk of progression of symptoms to permanent neurologic impairment or death.

Thrombotic microangiopathy associated with Valproic acid toxicity.

Background: Thrombotic microangiopathy (TMA) is a serious, sometimes life-threatening disorder marked by the presence of endothelial injury and microvascular thrombi. Drug-induced thrombotic microangiopathy (DI-TMA) is one specific TMA syndrome that occurs following drug exposure via drug-dependent antibodies or direct tissue toxicity. Common examples include calcineurin inhibitors Tacrolimus and Cyclosporine and antineoplastics Gemcitabine and Mitomycin.

Management of Hypertension in Children and Adolescents.

Hypertension in children and adolescents is becoming a greater problem in the developed world. Although traditionally thought of as usually secondary to renal, vascular, or endocrine causes, primary hypertension is becoming the most common form seen in childhood. This changing epidemiology is related to the recent obesity epidemic.

The utility of the IGF-I generation test in children with chronic kidney disease.

Background: To determine if the insulin-like-growth factor (IGF-I) generation test is a marker for growth hormone (GH) sensitivity in children with chronic kidney disease (CKD).

Methods: This was a randomized cross-over study in which children with CKD received low-dose (0.025 mg/kg/day) and high-dose (0.

Evaluation of serum creatinine concentration-based glomerular filtration rate equations in pediatric patients with chronic kidney disease.

Study Objective: To evaluate the accuracy of four equations based on serum creatinine concentration-the original Schwartz equation and the Leger, Bedside Chronic Kidney Disease in Children (CKiD), and Counahan-Barratt equations-for determining glomerular filtration rate (GFR) in pediatric patients with chronic kidney disease.

Design: Retrospective, observational, cross-sectional study.

Setting: Single-center, academic, outpatient pediatric nephrology clinic.

C1q nephropathy in the pediatric population: pathology and pathogenesis.

C1q nephropathy was originally described nearly 25 years ago by Jennette and Hipp. Since that time there have been a limited number of publications on C1q nephropathy, most of them in the pediatric literature. Despite reported incidences as high as 16% in some pediatric biopsy series, a consensus definition on the diagnosis of C1q nephropathy is lacking and its existence as a distinct clinical disease entity remains controversial.

First-year response to rhGH therapy in children with CKD: a National Cooperative Growth Study Report.

A clear definition of the appropriate growth response during recombinant human growth hormone (rhGH) treatment has never been established in the pediatric chronic kidney disease (CKD) population. We present here data from Genentech's National Cooperative Growth Study (NCGS) on the first-year growth response in prepubertal children with CKD. Using NCGS data, we constructed response curves for the first year of rhGH therapy in 270 (186 males, 84 females) naïve-to-treatment, prepubertal children with CKD prior to transplant or dialysis.

Factors affecting short-term precision of musculoskeletal measures using peripheral quantitative computed tomography (pQCT).

Unlabelled: Few studies have investigated factors influencing the precision of peripheral quantitative computed tomography (pQCT) measures. This study found time between repeat scans and subject anthropometric characteristics to influence short-term precision of pQCT-derived measures in the lower leg. These findings have implications for both investigators and clinicians utilizing pQCT outcomes.

Long-term safety of recombinant human growth hormone in children.

Background: Between 1985 and 2006, the National Cooperative Growth Study (NCGS) monitored the safety and efficacy of recombinant human growth hormone (rhGH) in 54,996 children.

Methods: Enrolled patients were followed until rhGH discontinuation. Investigators submitted adverse event reports for targeted events or those potentially rhGH-related.

A randomized, prospective study of 3 minimally invasive surgical approaches in total hip arthroplasty: comprehensive gait analysis.

Purported advantages of total hip arthroplasty performed with minimally invasive surgical (MIS) approaches are less muscle damage and faster recovery. There are little data scientifically evaluating these claims. Twenty-four consecutive hips were randomized to total hip arthroplasty through 1 of 3 MIS approaches (2-incision, mini-posterior, and mini-anterolateral).

Mycophenolate mofetil in children with frequently relapsing nephrotic syndrome: a report from the Southwest Pediatric Nephrology Study Group.

Children with frequently relapsing nephrotic syndrome (FRNS) often develop adverse effects from prednisone. Attempts to induce long-term remission in such patients have had varying levels of success. In this multicenter, prospective, open-label study, 14 centers enrolled 33 patients with FRNS, all of whom were in remission at the time of entry.

Living-donor nerve transplantation for global obstetric brachial plexus palsy.

The first reported case of live-donor nerve transplantation is presented, performed in an 8-month-old infant with global obstetric brachial plexus palsy (OBPP) and four root avulsions who had undergone prior sural nerve autografting at 3 months. Cross-chest C7 nerve transfer and temporary tacrolimus/prednisone immunosuppression were utilized. Acute rejection was prevented, with no observable complications from the immunosuppressive medications, ipsilateral deficits resulting from the use of the contralateral C7 root as a donor nerve, or untoward effects on growth and development occurring over a 2-year follow-up period.