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Full-Text Articles in Medicine and Health Sciences

Development Of A Novel Renal Activity Index Of Lupus Nephritis In Children & Young Adults, H. I. Brunner, M. Bennett, K. Abulaban, M. Klein-Gitelman, K. O'Neil, L. Tucker, S. Ardoin, K. Rouster-Stevens, B. A. Eberhard, P. Devarajan, +8 Additional Authors Jan 2016

Development Of A Novel Renal Activity Index Of Lupus Nephritis In Children & Young Adults, H. I. Brunner, M. Bennett, K. Abulaban, M. Klein-Gitelman, K. O'Neil, L. Tucker, S. Ardoin, K. Rouster-Stevens, B. A. Eberhard, P. Devarajan, +8 Additional Authors

Journal Articles

BACKGROUND: Noninvasive estimation of the degree of inflammation seen on kidney biopsy with lupus nephritis (LN) remains difficult. The objective of this study was to develop a Renal Activity Index for Lupus (RAIL) that, based solely on laboratory measures, accurately reflects histological LN activity. METHODS: We assayed traditional LN laboratory tests and 16 urine biomarkers (UBMs) in children (n=47) at the time of kidney biopsy. Histological LN activity was measured by the NIH Activity Index (NIH-AI) and the Tubulointerstitial Activity Index (TIAI). High LN-activity status (vs. moderate/low) was defined as NIH-AI scores > 10 (vs.5 (vs.92% accuracy and LN-activityTIAI status with …


Impact Of The 2008 Beijing Olympics On The Risk Of Pregnancy Complications, V. Assibey-Mensah, K. Liu, S. W. Thurston, T. P. Stevens, J. Zhang, C. Kane, Y. Pan, B. Weinberger, D. Q. Rich, +2 Additional Authors Jan 2016

Impact Of The 2008 Beijing Olympics On The Risk Of Pregnancy Complications, V. Assibey-Mensah, K. Liu, S. W. Thurston, T. P. Stevens, J. Zhang, C. Kane, Y. Pan, B. Weinberger, D. Q. Rich, +2 Additional Authors

Journal Articles

Taking advantage of the natural experiment of the 2008 Beijing Olympics (August 8 to September 24), when air pollution levels decreased by 13% to 60%, the authors assessed whether having >/=1 pregnancy month during the Olympics was associated with decreased risks of hypertensive disorders (HDs) and/or fetal-placental conditions (FPCs). Singleton births to mothers with >/=1 pregnancy month in 2008 or 2009 (N = 56,155) were included. Using generalized additive models, the authors estimated the risk of HDs and FPCs associated with (1) the 2008 Olympics compared with the same dates in 2009, and (2) increased mean ambient PM10 (particulate matter …


Endocrine Dysfunction In Diamond-Blackfan Anemia (Dba): A Report From The Dba Registry (Dbar), A. Lahoti, Y. T. Harris, P. W. Speiser, E. Atsidaftos, J. M. Lipton, A. Vlachos Jan 2016

Endocrine Dysfunction In Diamond-Blackfan Anemia (Dba): A Report From The Dba Registry (Dbar), A. Lahoti, Y. T. Harris, P. W. Speiser, E. Atsidaftos, J. M. Lipton, A. Vlachos

Journal Articles

BACKGROUND: Diamond-Blackfan anemia (DBA) is a rare inherited bone marrow failure syndrome. The mainstays of treatment involve chronic red cell transfusions, long-term glucocorticoid therapy, and stem cell transplantation. Systematic data concerning endocrine function in DBA are limited. We studied patients in the DBA Registry (DBAR) of North America to assess the prevalence of various endocrinopathies. PROCEDURE: In a pilot study, retrospective data were collected for 12 patients with DBA. Subsequently, patients with DBA aged 1-39 years were recruited prospectively. Combined, 57 patients were studied; 38 chronically transfused, 12 glucocorticoid-dependent, and seven in remission. Data were collected on anthropometric measurements, systematic …


Hydroxyurea Therapy For Children With Sickle Cell Anemia In Sub-Saharan Africa: Rationale And Design Of The Reach Trial, P. T. Mcgann, L. Tshilolo, B. Santos, G. A. Tomlinson, S. Stuber, T. Latham, B. Aygun, S. K. Obaro, P. Olupot-Olupot, R. E. Ware, +2 Additional Authors Jan 2016

Hydroxyurea Therapy For Children With Sickle Cell Anemia In Sub-Saharan Africa: Rationale And Design Of The Reach Trial, P. T. Mcgann, L. Tshilolo, B. Santos, G. A. Tomlinson, S. Stuber, T. Latham, B. Aygun, S. K. Obaro, P. Olupot-Olupot, R. E. Ware, +2 Additional Authors

Journal Articles

BACKGROUND: Sickle cell anemia (SCA) is an inherited hematological disorder that causes a large but neglected global health burden, particularly in Africa. Hydroxyurea represents the only available disease-modifying therapy for SCA, and has proven safety and efficacy in high-resource countries. In sub-Saharan Africa, there is minimal use of hydroxyurea, due to lack of data, absence of evidence-based guidelines, and inexperience among healthcare providers. PROCEDURE: A partnership was established between investigators in North America and sub-Saharan Africa, to develop a prospective multicenter research protocol designed to provide data on the safety, feasibility, and benefits of hydroxyurea for children with SCA. RESULTS: …


Hydroxycarbamide Versus Chronic Transfusion For Maintenance Of Transcranial Doppler Flow Velocities In Children With Sickle Cell Anaemia-Tcd With Transfusions Changing To Hydroxyurea (Twitch): A Multicentre, Open-Label, Phase 3, Non-Inferiority Trial, R. E. Ware, B. R. Davis, W. H. Schultz, R. C. Brown, B. Aygun, S. Sarnaik, I. Odame, B. Fuh, R. J. Adams, +36 Additional Authors Jan 2016

Hydroxycarbamide Versus Chronic Transfusion For Maintenance Of Transcranial Doppler Flow Velocities In Children With Sickle Cell Anaemia-Tcd With Transfusions Changing To Hydroxyurea (Twitch): A Multicentre, Open-Label, Phase 3, Non-Inferiority Trial, R. E. Ware, B. R. Davis, W. H. Schultz, R. C. Brown, B. Aygun, S. Sarnaik, I. Odame, B. Fuh, R. J. Adams, +36 Additional Authors

Journal Articles

BACKGROUND: For children with sickle cell anaemia and high transcranial doppler (TCD) flow velocities, regular blood transfusions can effectively prevent primary stroke, but must be continued indefinitely. The efficacy of hydroxycarbamide (hydroxyurea) in this setting is unknown; we performed the TWiTCH trial to compare hydroxyurea with standard transfusions. METHODS: TWiTCH was a multicentre, phase 3, randomised, open-label, non-inferiority trial done at 26 paediatric hospitals and health centres in the USA and Canada. We enrolled children with sickle cell anaemia who were aged 4-16 years and had abnormal TCD flow velocities (>/=200 cm/s) but no severe vasculopathy. After screening, eligible …


Increased Hepcidin In Transferrin-Treated Thalassemic Mice Correlates With Increased Liver Bmp2 Expression And Decreased Hepatocyte Erk Activation, H. Chen, T. Choesang, H. Li, S. Sun, P. Pham, W. Bao, M. Feola, M. Westerman, L. Blanc, Y. Z. Ginzburg, +4 Additional Authors Jan 2016

Increased Hepcidin In Transferrin-Treated Thalassemic Mice Correlates With Increased Liver Bmp2 Expression And Decreased Hepatocyte Erk Activation, H. Chen, T. Choesang, H. Li, S. Sun, P. Pham, W. Bao, M. Feola, M. Westerman, L. Blanc, Y. Z. Ginzburg, +4 Additional Authors

Journal Articles

Iron overload results in significant morbidity and mortality in beta-thalassemic patients. Insufficient hepcidin is implicated in parenchymal iron overload in beta-thalassemia and approaches to increase hepcidin have therapeutic potential. We have previously shown that exogenous apo-transferrin markedly ameliorates ineffective erythropoiesis and increases hepcidin expression in Hbb(th1/th1) (thalassemic) mice. We utilize in vivo and in vitro systems to investigate effects of exogenous apo-transferrin on Smad and ERK1/2 signaling, pathways that participate in hepcidin regulation. Our results demonstrate that apo-transferrin increases hepcidin expression in vivo despite decreased circulating and parenchymal iron concentrations and unchanged liver Bmp6 mRNA expression in thalassemic mice. Hepatocytes …


Non-Invasive Assessment Of Endothelial Function In Children With Obesity And Lipid Disorders, L. C. Hudgins, V. Annavajjhala, A. Kovanlikaya, M. D. Frank, A. Solomon, T. S. Parker, R. S. Cooper Jan 2016

Non-Invasive Assessment Of Endothelial Function In Children With Obesity And Lipid Disorders, L. C. Hudgins, V. Annavajjhala, A. Kovanlikaya, M. D. Frank, A. Solomon, T. S. Parker, R. S. Cooper

Journal Articles

BACKGROUND: Digital tonometry is designed to non-invasively screen for endothelial dysfunction by the detection of impaired flow-induced reactive hyperaemia in the fingertip. We determined whether digital reactive hyperaemia correlated with risk factors for atherosclerosis in two groups of children at increased risk for endothelial dysfunction. METHODS: A total of 15 obese children and 23 non-obese, dyslipidaemic children, 8-21 years of age, were enrolled, and their medical histories, anthropometric measurements, carotid wall thickness by means of ultrasonography, and fasting blood samples for cardiovascular risk factors were obtained. The standard endoPAT index of digital reactive hyperaemia was modified to reflect the true …


Organ Iron Accumulation In Chronically Transfused Children With Sickle Cell Anaemia: Baseline Results From The Twitch Trial, J. C. Wood, A. R. Cohen, S. L. Pressel, B. Aygun, H. Imran, L. Luchtman-Jones, A. A. Thompson, B. Fuh, W. H. Schultz, R. E. Ware, +1 Additional Author Jan 2016

Organ Iron Accumulation In Chronically Transfused Children With Sickle Cell Anaemia: Baseline Results From The Twitch Trial, J. C. Wood, A. R. Cohen, S. L. Pressel, B. Aygun, H. Imran, L. Luchtman-Jones, A. A. Thompson, B. Fuh, W. H. Schultz, R. E. Ware, +1 Additional Author

Journal Articles

Transcranial Doppler (TCD) With Transfusions Changing to Hydroxyurea (TWiTCH) trial is a randomized, open-label comparison of hydroxycarbamide (also termed hydroxyurea) versus continued chronic transfusion therapy for primary stroke prevention in patients with sickle cell anaemia (SCA) and abnormal TCD. Severity and location of iron overload is an important secondary outcome measure. We report the baseline findings of abdominal organ iron burden in 121 participants. At enrollment, patients were young (9.8 +/- 2.9 years), predominantly female (60:40), and previously treated with transfusions (4.1 +/- 2.4 years) and iron chelation (3.1 +/- 2.1 years). Liver iron concentration (LIC; 9.0 +/- 6.6 mg/g …


Validation Of The Lupus Nephritis Clinical Indices In Childhood-Onset Systemic Lupus Erythematosus, R. Mina, K. Abulaban, M. Klein-Gitelman, A. Eberhard, S. Ardoin, N. Singer, K. Onel, L. Tucker, K. O'Neil, H. I. Brunner, +8 Additional Authors Jan 2016

Validation Of The Lupus Nephritis Clinical Indices In Childhood-Onset Systemic Lupus Erythematosus, R. Mina, K. Abulaban, M. Klein-Gitelman, A. Eberhard, S. Ardoin, N. Singer, K. Onel, L. Tucker, K. O'Neil, H. I. Brunner, +8 Additional Authors

Journal Articles

OBJECTIVE: To validate clinical indices of lupus nephritis (LN) activity and damage when used in children against the criterion standard of kidney biopsy findings. METHODS: In 83 children requiring kidney biopsy the SLE Disease Activity Index Renal Domain (SLEDAI-R); British Isles Lupus Assessment Group index Renal Domain (BILAG-R), Systemic Lupus International Collaborating Clinics Renal Activity (SLICC-RAS) and Damage Index Renal Domain (SDI-R) were measured. Fixed effect and logistic models were done to predict International Society of Nephrology/Renal Pathology Society (ISN/RPS) class; low/moderate vs. high LN-activity [NIH Activity Index (NIH-AI) score:10; Tubulointerstitial Activity Index (TIAI) score:5) or the absence vs. presence …