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.

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.

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.

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.

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.

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.

Measurement and treatment of elevated blood pressure in the pediatric patient with chronic kidney disease.

Hypertension, as in adults, is a frequent complication found in children with chronic kidney disease (CKD). Indeed, hypertension has now become one of the most prevalent chronic diseases of childhood. The most recent data available (2003) indicate that at least 38% of children with CKD in the United States are receiving antihypertensive therapy.

Monitoring changes in gene expression in renal ischemia-reperfusion in the rat.

Background: Although acute renal failure (ARF) is a relatively common disorder with major morbidity and mortality, its molecular basis remains incompletely defined. The present study examined global gene expression in the well-characterized ischemia-reperfusion model of ARF using DNA microarray technology.

Methods: Male Wistar rats underwent bilateral renal ischemia (30 min) or sham operation, followed by reperfusion for 1, 2, 3 or 4 days.