A5053513542- Hemoglobinopathies and Related Disorders
- Iron Metabolism and Disorders
- Pharmacological Effects and Toxicity Studies
- Bone and Joint Diseases
- Bone health and osteoporosis research
- Trace Elements in Health
- Lysosomal Storage Disorders Research
- Folate and B Vitamins Research
- Vitamin D Research Studies
- Blood groups and transfusion
- Cerebral Palsy and Movement Disorders
- Glycogen Storage Diseases and Myoclonus
- Body Composition Measurement Techniques
- Child Nutrition and Feeding Issues
- Bone fractures and treatments
- Family and Disability Support Research
- Erythrocyte Function and Pathophysiology
- Nutrition and Health in Aging
- Hip disorders and treatments
- Acute Lymphoblastic Leukemia research
- Child Nutrition and Water Access
- Metabolism and Genetic Disorders
- Vitamin C and Antioxidants Research
- Infant Development and Preterm Care
- Bone health and treatments
UCSF Benioff Children's Hospital
2015-2025
University of California, San Francisco
2016-2025
Boston Children's Hospital
2007-2013
UC Davis Children's Hospital
2012
University of California, Davis
2012
Oakland University
2002-2010
University of Alabama at Birmingham
2008
Cincinnati Children's Hospital Medical Center
2008
University of Cincinnati
2008
Toronto General Hospital
2008
Diminished bone density and a propensity to fracture with minimal trauma are common in children adolescents moderate severe cerebral palsy (CP). The purpose of this study was provide detailed evaluation mineral (BMD) metabolism population assess the relationship these measures multiple other clinical, growth, nutrition variables.The group consisted 117 subjects ages 2 19 years (mean: 9.7 years) CP as defined by Gross Motor Functional Classification scale. Population-based sampling used...
Blood transfusion therapy is life-saving for patients with beta-thalassaemia and sickle cell disease (SCD), but often results in severe iron overload. This pilot study examined whether the biomarkers of tissue injury or inflammation differ these two diseases. Plasma malondialdehyde (MDA) was significantly increased 1.8-fold thalassaemia relative to control patients. In contrast, MDA SCD not different from controls. multivariate analysis, strongest predictors elevated were liver concentration...
Summary Deferasirox is a once‐daily, oral iron chelator developed for treating transfusional overload. Preclinical studies indicated that the kidney was potential target organ of toxicity. As patients with sickle cell disease often have abnormal baseline renal function, primary objective this randomised, open‐label, phase II trial to evaluate safety and tolerability deferasirox in comparison deferoxamine population. Assessment efficacy, as measured by change liver concentration (LIC) using...
BACKGROUND. Children with cerebral palsy frequently grow poorly. The purpose of this study was to describe observed growth patterns and their relationship health social participation in a representative sample children moderate-severe palsy. METHODS. In 6-site, multicentered, region-based cross-sectional study, multiple sources were used identify moderate or severe There 273 enrolled, 58% male, 71% white, Gross Motor Function Classification System levels III (22%), IV (25%), V (53%)....
Adults with beta thalassemia major frequently have low BMD, fractures, and bone pain. The purpose of this study was to determine the prevalence pain in all syndromes childhood, adolescence, adulthood, associations BMD fractures pain, etiology disease thalassemia. Patients Thalassemia Clinical Research Network, > or =6 yr age, no preexisting medical condition affecting mass requiring steroids, participated. We measured spine femur whole body BMC by DXA assessed vertebral abnormalities...
The aim of the study was to evaluate health children with cerebral palsy (CP) using a global assessment quality life, condition-specific measures, and assessments care use. A multicenter population-based cross-sectional survey 235 children, aged 2 18 years, moderate severe impairment, carried out Gross Motor Function Classification System (GMFCS) levels III (n = 56), IV 55), V 122). This group scored significantly below mean on Child Health Questionnaire (CHQ) for Pain, General Health,...
Iron-overload associated endocrinopathy is the most frequently reported complication of chronic transfusion therapy in patients with thalassaemia (Thal). This study compared iron-overloaded subjects Thal (n = 142; 54%M; age 25.8 +/- 8.1 years) and transfused sickle-cell disease (Tx-SCD; n 199; 43%M, 24.9 13.2 to non-transfused SCD (non-Tx-SCD; 64, 50%M, 25.3 11.3 years), explore whether underlying haemoglobinopathy influences development endocrinopathy. Subjects were recruited from 31...
A natural history study was conducted in 142 Thalassemic (Thal), 199 transfused Sickle Cell Disease (Tx-SCD, n = 199), and 64 non-Tx-SCD subjects to describe the frequency of iron-related morbidity mortality. Subjects recruited from 31 centers US, Canada or UK were similar with respect age (overall: 25 +/- 11 years, mean SD) gender (52% female). We found that Tx-SCD hospitalized more frequently compared Thal (P < 0.001). Among those hospitalized, adult likely be unemployed (RR 1.6, 95% CI...
This study aimed to determine differences in the rates of growth, endocrine- and calcium-related abnormalities various thalassemia syndromes North America treated with current therapies. Medical history, physical examinations blood urine collections were obtained from patients all age 6 years older Thalassemia Clinical Research Network. 361 subjects, 49% male, mean 23.2 (range 6.1-75 years) studied. Approximately 25% children adults, regardless syndrome, had short stature. Overall growth was...
Abstract Although it is life saving, transfusion therapy has resulted in the majority of sickle cell anemia and thalassemia patients being at risk for hemosiderosis‐induced organ damage. It unknown whether complications iron overload are affected by underlying disease. In order to address this problem, we compared prevalence dysfunction both groups receiving chronic (β thalassemia, N = 30; anemia, 43). Both had similar quantitative liver iron. Thalassemia greater cardiac disease (20% vs....
Abstract Serum ferritin (SF) and liver iron concentration (LIC), as measured by SQUID biosusceptometry, were assessed in a convenience sample of transfusion independent thalassemia patients (nTx‐Thal, n = 26), regularly transfused (Tx‐Thal, 89), or sickle cell (SCD, 45) to investigate the severity overload relationship between SF LIC nTx‐Thal compared SCD Tx‐Thal. correlated with (R S 0.53, P < 0.001), but was found be poor predictor for LIC. significantly lower ( 0.001) than other...
Summary Little is known about the effects of thalassaemia on kidney. Characterization underlying renal function abnormalities in timely because newer iron chelator, deferasirox, can be nephrotoxic. We aimed to determine prevalence and correlates patients, treated before deferasirox was widely available, using 24‐h collections urine. calculated creatinine clearance urine calcium‐to‐creatinine ratio measured urinary β 2 ‐microglobulin, albumin, protein. used multivariate modelling identify...
Chronic transfusion therapy has played a central role in extending life expectancy for patients with hemoglobinopathies such as thalassemia. However, this life-saving is associated numerous complications that now comprise the bulk of management considerations This review reports on experience Thalassemia Longitudinal Cohort and reviews available literature to establish guidelines
Objectives. To compare the development of secondary sexual characteristics in children with cerebral palsy (CP) moderate to severe motor impairment general population and relate their maturation a measure body fat. Methods. A multicenter, cross-sectional survey 207 who were 3 18 years age had CP (Gross Motor Functional Classification System [GMFCS] levels 3, 4, 5) was conducted at 6 geographic sites; attempts made identify all eligible through multiple methods enroll them study. Trained...
Pediatric Reference Data for Dual X-Ray Absorptiometric Measures of Normal Bone Density in the Distal FemurRichard C. Henderson1 2, Robert K. Lark1, Jamie E. Newman3, Heidi Kecskemethy4, Ellen B. Fung5 6, Jordan Renner7 8 and H. Theodore Harcke9 10Audio Available | Share
Abstract Purpose We evaluated bone mineral density (BMD) and risk factors for poor mineralization in children with sickle cell anemia (SCA). Patients Methods Twenty‐five severe manifestations of SCA (frequent hospitalizations, growth delay, or need chronic red transfusions) were enrolled. Bone was assessed at lumbar spine proximal femur dual‐energy X‐ray absorptiometry (DXA), Z‐scores calculated by comparison age, sex, ethnicity‐specific reference data. Results The median age the study...