Our study was designed to prospectively determine whether or not unrelated cord blood transplantation (CBT) can produce outcomes comparable to related donor transplantation for children with b-thalassaemia. In 35 patients, 40 transplants were performed between October 2003 and September 2009. HLA matching at enrolment was 6/6 (n ¼ 8), 5/6 (n ¼ 16), 4/6 (n ¼ 27), or 3/6 (n ¼ 1) by low-resolution HLA-A, -B, and high-resolution DRB1. These patients received non-manipulated grafts without ex vivo expansion or T-cell depletion. The median number of nucleated and CD34 þ cells infused was 7.8 Â 10 7 /kg (range, 2.8-14.7 Â 10 7 /kg) and 4.0 Â 10 5 /kg (range, 1.7-19.9 Â 10 5 /kg), respectively. The 5-year OS and thalassaemia-free survival after the first transplant were 88.3 and 73.9%, respectively. The cumulative incidence of TRM at 2 years was 11.7%. Fourteen patients developed chronic skin GVHD. Thirty patients were alive and transfusion-independent with a Lansky performance score X80% achieved between 6 and 76 months post transplant (median, 36 months). These data compare acceptably with the survival rates of related-donor BMT for thalassaemia and suggest that patients without an available HLAcompatible sibling but who have well-matched unrelated donors should also be considered for CBT.
The purpose of this report was to develop novel biodegradable occlusion devices for closure of atrial septal defects (ASD). To manufacture the biodegradable occluders, polycaprolactone (PCL) components were first fabricated by a lab-scale micro-injection molding machine. They were then assembled and hot-spot welded into double umbrella-like devices of 50 mm in diameter. A special mechanism at the axis of the occluder was designed to self-lock the occluder after the two umbrellas were expanded. Furthermore, a nanofibrous matrix of poly-D-L-lactide-glycolide (PLGA)/type I collagen blend was produced via electrospinning to develop biodegradable and biomimetic anti-shunt membranes for the occluders. Characterization of the biodegradable PCL occluders was carried out. PCL occluders exhibited mechanical properties comparable to that of commercially available Amplatzer occluders. The sealing capability of biodegradable occluders was found superior to that of Amplatzer occluders. In addition, the cell attachment and spreading of endothelial cells seeded on the PLGA/collagen nanofibrous matrix and the interaction between cells and PLGA/collagen nanofibers were studied. The nanofibrous membranes made of PLGA/collagen were very effective in promoting cell proliferation during culture.
Objective. To characterize the atherosclerotic risk factors in the progression of subclinical atherosclerosis in patients with juvenile-onset systemic lupus erythematosus (SLE).Methods. This was a longitudinal study of 76 patients with juvenile-onset SLE. Carotid arteries were evaluated using ultrasonography at baseline and at followup visits at 6-month intervals over the 6-year study period. Clinical and laboratory parameters, disease activity, treatment, and traditional risk factors for atherosclerosis were evaluated. Data were analyzed using generalized estimating equations.Results. The mean ؎ SD age of the patients at baseline was 15.01 ؎ 3.48 years and the mean ؎ SD disease duration was 2.65 ؎ 2.5 years. The mean ؎ SD duration of followup was 3.74 ؎ 1.24 years. The mean ؎ SD intima-media thickness (IMT) of the common carotid arteries differed significantly between the patient and control (n ؍ 38) groups (0.63 ؎ 0.08 mm versus 0.54 ؎ 0.06 mm; P < 0.001). The presence of lymphopenia at diagnosis and at baseline and higher levels of serum creatinine and C-reactive protein at baseline were positively associated with progression of carotid IMT (P ؍ 0.006, P ؍ 0.043, P ؍ 0.037, and P ؍ 0.049, respectively). In multivariate analysis, only lymphopenia at baseline and at diagnosis were consistently associated with progression of IMT (P ؍ 0.012 and P ؍ 0.045, respectively).Conclusion. In patients with juvenile-onset SLE, some nontraditional risk factors for the progression of subclinical atherosclerosis were identified. Lymphopenia was the only independent risk factor for the progression of IMT. The pathogenic mechanisms warrant further investigation.
Pediatric myocarditis symptoms can be mild or as extreme as sudden cardiac arrest. Early identification of the severity of illness and timely provision of critical care is helpful; however, the risk factors associated with mortality remain unclear and controversial. We undertook a retrospective review of the medical records of pediatric patients with myocarditis in a tertiary care referral hospital for over 12 years to identify the predictive factors of mortality. Demographics, presentation, laboratory test results, echocardiography findings, and treatment outcomes were obtained. Regression analyses revealed the clinical parameters for predicting mortality. During the 12-year period, 94 patients with myocarditis were included. Of these, 16 (17%) patients died, with 12 succumbing in the first 72 hours after admission. Fatal cases more commonly presented with arrhythmia, hypotension, acidosis, gastrointestinal symptoms, decreased left ventricular ejection fraction, and elevated isoenzyme of creatine kinase and troponin I levels than nonfatal cases. In multivariate analysis, troponin I > 45 ng/mL and left ventricular ejection fraction < 42% were significantly associated with mortality. Pediatric myocarditis had a high mortality rate, much of which was concentrated in the first 72 hours after hospitalization. Children with very high troponin levels or reduced ejection fraction in the first 24 hours were at higher risk of mortality, and targeting these individuals for more intensive therapies may be warranted.
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