Children's Memorial Hospital

About: Children's Memorial Hospital is a based out in . It is known for research contribution in the topics: Population & Transplantation. The organization has 5652 authors who have published 8967 publications receiving 283837 citations.

Mental health functioning among children and adolescents with perinatal HIV infection and perinatal HIV exposure

Abstract: Mental health problems (MHPs) among children with perinatal HIV infection have been described prior to and during the highly active antiretroviral therapy (HAART) era. Yet child, caregiver and socio-demographic factors associated with MHPs are not fully understood. We examined the prevalence of MHPs among older children and adolescents with perinatal HIV exposure, including both perinatally HIV-infected (PHIV + ) and perinatally HIV-exposed but uninfected (PHEU) youth. Our aims were to identify the impact of HIV infection by comparing PHIV+ and PHEU youth and to delineate risk factors associated with MHPs, in order to inform development of appropriate prevention and intervention strategies. Youth and their caregivers were interviewed with the Behavior Assessment System for Children, 2nd edition (BASC-2) to estimate rates of at-risk and clinically significant MHPs, including caregiver-reported behavioral problems and youth-reported emotional problems. The prevalence of MHPs at the time of study entry was c...

Challenges of alloimmunization in patients with haemoglobinopathies.

Abstract: Red blood cell (RBC) transfusions can be life-sustaining in chronic inherited anaemias, such as thalassaemia, and the indications for blood transfusions in patients with sickle cell disease continue to expand. Complications of transfusions, such as allosensitization, can create significant medical challenges in the management of patients with haemoglobinopathies. This review summarizes key findings from the medical literature related to alloimmunization in haemoglobinopathies and examines potential measures to mitigate these risks. Areas where future studies are needed are also addressed.

Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial.

Abstract: Importance An unmet need remains for safe and efficacious treatments for Duchenne muscular dystrophy (DMD). To date, there are limited agents available that address the underlying cause of the disease. Objective To evaluate the safety, tolerability, and efficacy of viltolarsen, a novel antisense oligonucleotide, in participants with DMD amenable to exon 53 skipping. Design, Setting, and Participants This phase 2 study was a 4-week randomized clinical trial for safety followed by a 20-week open-label treatment period of patients aged 4 to 9 years with DMD amenable to exon 53 skipping. To enroll 16 participants, with 8 participants in each of the 2 dose cohorts, 17 participants were screened. Study enrollment occurred between December 16, 2016, and August 17, 2017, at sites in the US and Canada. Data were collected from December 2016 to February 2018, and data were analyzed from April 2018 to May 2019. Interventions Participants received 40 mg/kg (low dose) or 80 mg/kg (high dose) of viltolarsen administered by weekly intravenous infusion. Main Outcomes and Measures Primary outcomes of the trial included safety, tolerability, and de novo dystrophin protein production measured by Western blot in participants’ biceps muscles. Secondary outcomes included additional assessments of dystrophin mRNA and protein production as well as clinical muscle strength and function. Results Of the 16 included boys with DMD, 15 (94%) were white, and the mean (SD) age was 7.4 (1.8) years. After 20 to 24 weeks of treatment, significant drug-induced dystrophin production was seen in both viltolarsen dose cohorts (40 mg/kg per week: mean [range] 5.7% [3.2-10.3] of normal; 80 mg/kg per week: mean [range] 5.9% [1.1-14.4] of normal). Viltolarsen was well tolerated; no treatment-emergent adverse events required dose reduction, interruption, or discontinuation of the study drug. No serious adverse events or deaths occurred during the study. Compared with 65 age-matched and treatment-matched natural history controls, all 16 participants treated with viltolarsen showed significant improvements in timed function tests from baseline, including time to stand from supine (viltolarsen: −0.19 s; control: 0.66 s), time to run/walk 10 m (viltolarsen: 0.23 m/s; control: −0.04 m/s), and 6-minute walk test (viltolarsen: 28.9 m; control: −65.3 m) at the week 25 visit. Conclusions and Relevance Systemic treatment of participants with DMD with viltolarsen induced de novo dystrophin production, and clinical improvement of timed function tests was observed. Trial Registration ClinicalTrials.gov Identifier:NCT02740972

Masses of the salivary gland region in children.

Abstract: Background Noninflammatory masses of the salivary gland region in children are extremely rare. Therefore, very few published individual and institution-based experiences exist. Design Retrospective chart review from 1990 through 1997. Setting University-based children's hospital. Design Patients 18 years of age or younger with a tumor in the salivary gland region. Masses of infectious origin were excluded. Hemangiomas and lymphangiomas were tallied for relative incidences only. Results Three hundred twenty-four consecutive cases of salivary gland masses were found: 192 hemangiomas (59.2%), 89 lymphangiomas (27.5%), and 43 (13.3%) solid masses. No significant difference was found between the age at presentation of the patients with benign solid tumors and the patients with malignant solid tumors (mean + SEM age, 7.2 + 0.7 years). Sixty-one percent of the masses were found in the parotid region; 18% were localized to the submandibular gland region; and the remaining 21% were located in a minor salivary gland site. The most common benign perisalivary masses were pilomatrixomas (20.9%), followed by pleomorphic adenomas (11.6%). The most common malignant masses were mucoepidermoid carcinomas (9.3%), followed by rhabdomyosarcomas (7.0%). Treatment was individualized to the disease. Twenty-two patients had adequate data for follow-up analysis (mean + SEM follow-up, 30.0 + 8.4 months). Four patients (18.2%) experienced recurrent or residual disease and were alive with disease at last follow-up, and 100% of our population demonstrated disease-specific survival at last follow-up. Conclusions Vascular lesions outnumber solid tumors of the salivary gland region. The most common salivary tumors were pleomorphic adenomas, followed by mucoepidermoid carcinomas. Although certain solid salivary masses may demonstrate locally aggressive behavior, the overall prognosis is favorable.