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International Registry (international + registry)
Selected AbstractsThe International Quotidian Hemodialysis Registry: Rationale and challengesHEMODIALYSIS INTERNATIONAL, Issue 2008Robert M. LINDSAY Abstract Outcomes from conventional thrice-weekly hemodialysis (CHD) are disappointing for a life-saving therapy. The results of the HEMO Study show that the recommended minimum dose (Kt/V) for adequacy is also the optimum attainable with CHD. Interest is therefore turning to alternative therapies exploring the effects of increased frequency and time of hemodialysis (HD) treatment. The National Institutes of Health have sponsored 2 randomized prospective trials comparing short hours daily in-center HD and long hours slow nightly home HD with CHD. An International Registry has also been created to capture observational data on patients receiving short hours daily in-center HD, long hours slow nightly home HD, and other alternative therapies. Participation by individual centers, other registries and the major dialysis chains is growing and currently data from nearly 3000 patients have been collected. Pitfalls in data collection have been identified and are being corrected. A matched cohort (patients in other registries) study is planned to obtain information regarding hard outcomes expected from these therapies. The Registry may become the most important source of information required by governments, providers, and the nephrological community in assessing the utility of such therapies. [source] Arrhythmogenic Right Ventricular Dyspiasia/Cardiomyopathy:JOURNAL OF CARDIOVASCULAR ELECTROPHYSIOLOGY, Issue 7 2000Need for an International Registry. Arrhythmogenic Right Ventricular Dysplasia/Cardiomyopathy. Arrhythmogenic right ventricular dysplasia/cardiomyopathy (ARVD/C) is a heart muscle disease characterized by peculiar right ventricular involvement and electrical instability that precipitates ventricular arrhythmias and sudden death. The purpose of the present consensus report of the Study Group of the European Society of Cardiology and the Scientific Council on Cardiomyopathies of the World Heart Federation is to review the considerable progress in our understanding of the etiopathogenesis, morbid anatomy, and clinical presentation of ARVD/C since its first description in 1977. This article will focus on the important hut still unanswered issues, mostly regarding risk stratification, clinical outcome, and management of affected patients. Because ARVD/C is relatively uncommon and any one center may have experience with only a few patients, an international registry is being established to accumulate information and enhance the numbers of patients that can be analyzed to answer the pending questions. The registry also will facilitate pathologic, molecular, and genetics research on the etiology and pathogenesis of the disease. Furthermore, availability of an international database will enhance awareness of this largely unrecognized condition among the medical community. Physicians are encouraged to enroll patients in the International Registry of ARVD/C. [source] Antimitochondrial antibodies in patients with chronic hepatitis C virus infection: description of 18 cases and review of the literatureJOURNAL OF VIRAL HEPATITIS, Issue 6 2005M. Ramos-Casals Summary., To describe the clinical and immunologic patterns of disease expression of patients with chronic hepatitis C virus (HCV) infection and positive antimitochondrial antibodies (AMA). We investigated the presence of AMA in 237 consecutive HCV patients with extrahepatic manifestations from an International Registry. AMA were detected by indirect immunofluorescence in triple rat tissue (liver, stomach and kidney), aceton-fixed criosections and FITC-conjugated rabbit anti-human immunoglobulins. We found positive AMA in 18 (8%) out of 237 HCV patients. All patients were female with a mean age at protocol inclusion of 65.8 years (ranging from 37 to 87 years). Twelve (67%) patients fulfilled classification criteria for systemic autoimmune diseases (SAD), including Sjögren's syndrome (n = 7), systemic sclerosis (n = 3) and systemic lupus erythematosus (n = 2). Fourteen (78%) of the HCV-AMA patients presented at least one of the highly suggestive characteristics of primary biliary cirrhosis (PBC): 9 (50%) had a specific M2 pattern, 6 (33%) had more than twice normal levels of alkaline phosphatase, 5 (28%) had raised IgM levels and 4 (22%) a histological pattern compatible with PBC. Five (28%) patients developed neoplasia after detection of AMA. Seven (39%) patients died, due to neoplasia (n = 4), cirrhotic complications (n = 2) and hepatopulmonary syndrome (n = 1). We describe a subset of HCV patients with positive AMA who presented a broad spectrum of clinical features, including liver, autoimmune and neoplasic manifestations. Two-thirds of these patients presented an associated SAD, mainly Sjögren's syndrome or systemic sclerosis, together with a high frequency of multiple autoantibodies and an increased prevalence of cirrhosis and neoplasia. [source] Current challenges of pharmacovigilance in bleeding disorders: converting the burden to benefitHAEMOPHILIA, Issue 2 2010R. LASSILA Summary., Safety surveillance studies have proven essential in research and development of new biological therapies for bleeding disorders as well as other diseases. Although product safety regarding HIV, hepatitis, and other blood-borne infections is currently excellent, potential new infectious agents require continued vigilant monitoring. Inhibitor development is the most common serious side effect of haemophilia replacement therapy. Several aetiological factors associated with inhibitors have been identified, but their true impact is still largely unknown. Moreover, whether plasma-derived and recombinant factor products differ in their immunogenic profiles is an unresolved issue. Coagulation factor products under development and those currently on the market require uniform, long-term surveillance. The European Haemophilia Safety Surveillance (EUHASS) project was recently established to meet these goals. The pharmaceutical industry and clinicians face common challenges complying with these requirements. In rare diseases like haemophilia, obtaining adequate patient numbers poses a challenge. Another challenge is a lack of methods for assessing disease severity, a surprising deficiency in the era of modern medical and laboratory technology. National and international registries can be used to gather required safety surveillance information. Simultaneously, clinicians benefit from well-organized registry data in their daily practice and harmonize the quality of comprehensive haemophilia care by homogeneous follow-up platforms. Experience with such registries comes, for example, from Europe (PEDNET), the USA (CDC/UDC), the UK (UKHCDO), and Sweden (Malmö). It is important to commit to future pharmacovigilance efforts, aiming at high-quality safety surveillance programmes at both the pharmaceutical research community and clinical levels. [source] Worldwide childhood type 1 diabetes incidence , what can we learn from epidemiology?PEDIATRIC DIABETES, Issue 2007G Soltesz Abstract:, Type 1 diabetes is the most common form of diabetes in most part of the world, although reliable data are still unavailable in several countries. Wide variations exist between the incidence rates of different populations, incidence is lowest in China and Venezuela (0.1 per 100 000 per year) and highest in Finland and Sardinia (37 per 100 000 per year). In most populations girls and boys are equally affected. In general, the incidence increases with age, the incidence peak is at puberty. After the pubertal years, the incidence rate significantly drops in young women, but remains relatively high in young adult males up to the age 29,35 years. Prospective national and large international registries (DIAMOND and EURODIAB) demonstrated an increasing trend in incidence in most regions of the world over the last few decades and increases seem to be the highest in the youngest age group. Analytical epidemiological studies have identified environmental risk factors operating early in life which might have contributed to the increasing trend in incidence. These include enteroviral infections in pregnant women, older maternal age (39,42 years), preeclampsia, cesarean section delivery, increased birthweight, early introduction of cow's milk proteins and an increased rate of postnatal growth (weight and height). Optimal vitamin D supplementation during early life has been shown to be protective. Some of these environmental risk factors such as viruses may initiate autoimmunity toward the beta cell, other exposures may put on overload on the already affected beta cell and thus accelerate the disease process. [source] Bernard Soulier syndrome in pregnancy: a systematic reviewHAEMOPHILIA, Issue 4 2010P. PEITSIDIS Summary., Bernard Soulier syndrome (BSS) is a rare disorder of platelets, inherited mainly as an autosomal recessive trait. It is characterised by qualitative and quantitative defects of the platelet membrane glycoprotein (GP) Ib-IX-V complex. The main clinical characteristics are thrombocytopenia, prolonged bleeding time and the presence of giant platelets. Data on the clinical course and outcome of pregnancy in women with Bernard Soulier syndrome is scattered in individual case reports. In this paper, we performed a systematic review of literature and identified 16 relevant articles; all case reports that included 30 pregnancies among 18 women. Primary postpartum haemorrhage was reported in 10 (33%) and secondary in 12 (40%) of pregnancies, requiring blood transfusion in 15 pregnancies. Two women had an emergency obstetric hysterectomy. Alloimmune thrombocytopenia was reported in 6 neonates, with one intrauterine death and one neonatal death. Bernard Soulier syndrome in pregnancy is associated with a high risk of serious bleeding for the mother and the neonate. A multidisciplinary team approach and individualised management plan for such women are required to minimise these risks. An international registry is recommended to obtain further knowledge in managing women with this rare disorder. [source] The International Quotidian Dialysis Registry: Annual Report 2007HEMODIALYSIS INTERNATIONAL, Issue 3 2007Gihad E. NESRALLAH Abstract In view of the need to study both intermediate and definitive outcomes associated with daily and extended-hours hemodialysis (HD), our group has undertaken the design and implementation of an international registry to collect data describing the treatments and outcomes of patients treated with these regimens. The International Quotidian Dialysis Registry began recruiting patients in June 2004. There are currently 229 patients enrolled in the registry, up from 199 last year. The projected growth is 2000 patients by 2008. This paper constitutes the third annual report of progress of patient and center recruitment, and includes descriptive data drawn from the 3 primary patient groups currently tracked by the registry: home nocturnal, home short-daily, and in-center short-daily HD. As the cohort grows, patients will be compared with control subjects drawn from their respective national registries, and comparative analyses will follow. [source] Arrhythmogenic Right Ventricular Dyspiasia/Cardiomyopathy:JOURNAL OF CARDIOVASCULAR ELECTROPHYSIOLOGY, Issue 7 2000Need for an International Registry. Arrhythmogenic Right Ventricular Dysplasia/Cardiomyopathy. Arrhythmogenic right ventricular dysplasia/cardiomyopathy (ARVD/C) is a heart muscle disease characterized by peculiar right ventricular involvement and electrical instability that precipitates ventricular arrhythmias and sudden death. The purpose of the present consensus report of the Study Group of the European Society of Cardiology and the Scientific Council on Cardiomyopathies of the World Heart Federation is to review the considerable progress in our understanding of the etiopathogenesis, morbid anatomy, and clinical presentation of ARVD/C since its first description in 1977. This article will focus on the important hut still unanswered issues, mostly regarding risk stratification, clinical outcome, and management of affected patients. Because ARVD/C is relatively uncommon and any one center may have experience with only a few patients, an international registry is being established to accumulate information and enhance the numbers of patients that can be analyzed to answer the pending questions. The registry also will facilitate pathologic, molecular, and genetics research on the etiology and pathogenesis of the disease. Furthermore, availability of an international database will enhance awareness of this largely unrecognized condition among the medical community. Physicians are encouraged to enroll patients in the International Registry of ARVD/C. [source] NOD2-Associated pediatric granulomatous arthritis, an expanding phenotype: Study of an international registry and a national cohort in spainARTHRITIS & RHEUMATISM, Issue 6 2009Carlos D. Rosé Objective To study the phenotype characteristics of the largest to date cohort of patients with pediatric granulomatous arthritis (PGA) and documented mutations in the NOD2 gene. Methods We analyzed merged data from 2 prospective cohorts of PGA patients, the International PGA Registry and a Spanish cohort. A systematic review of the medical records of interest was performed to identify phenotype characteristics. Results Forty-five patients with PGA (23 sporadic cases and 22 from familial pedigrees) and documented NOD2 mutations were identified and formed the basis of the study. Of these 45 patients, 18 had the R334W-encoding mutation, 18 had R334Q, 4 had E383K, 3 had R587C, 1 had C495Y, and 1 had W490L. The majority of patients manifested the typical triad of dermatitis, uveitis, and arthritis. In contrast, in 13 patients, the following "atypical" manifestations were noted: fever, sialadenitis, lymphadenopathy, erythema nodosum, leukocytoclastic vasculitis, transient neuropathy, granulomatous glomerular and interstitial nephritis, interstitial lung disease, arterial hypertension, hypertrophic cardiomyopathy, pericarditis, pulmonary embolism, hepatic granulomatous infiltration, splenic involvement, and chronic renal failure. In addition, 4 individuals who were asymptomatic carriers of a disease-causing mutation were documented. Conclusion NOD2 -associated PGA can be a multisystem disorder with significant visceral involvement. Treating physicians should be aware of the systemic nature of this condition, since some of these manifestations may entail long-term morbidity. [source] Second malignancies in children with neuroblastoma after combined treatment with 131I-metaiodobenzylguanidineCANCER, Issue 5 2003Alberto Garaventa M.D. Abstract BACKGROUND 131I-metaiodobenzylguanidine (131I-MIBG) is selectively taken up by cells of neural crest origin, allowing targeted radiotherapy of tumors such as neuroblastoma (NB) and pheochromocytoma. Radiotherapy may provide additional benefits in the treatment of NB, with moderate side effects such as hematologic and thyroid toxicity. However, with longer follow-up, other complications might occur. We describe our experience with second cancers occurring in children treated with 131I-MIBG and chemotherapy. METHODS The clinical records of 119 consecutive NB cases treated with 131I-MIBG at a single institution between 1984 and 2001 were reviewed for the occurrence of a second malignant neoplasm (SMN). RESULTS Overall, five cases of SMN occurred in the study patients. In particular, two cases of myeloid leukemia, one of angiomatous fibrous histiocytoma, one of malignant schwannoma, and one case of rhabdomyosarcoma were detected. The schwannoma and the rhabdomyosarcoma developed within the residual neuroblastic mass after first-line therapy. CONCLUSIONS Should 131I-MIBG treatment become more broadly employed in the therapeutic strategy for neuroblastoma, the risk of second cancer will have to be taken into consideration. The organization of an international registry of subjects treated with 131I-MIBG might better define the frequency and features of second malignancies following this radiometabolic approach. Cancer 2003;97:1332,38. © 2003 American Cancer Society. DOI 10.1002/cncr.11167 [source] Transcatheter closure of perimembranous ventricular septal defects using the amplatzer membranous VSD occluder: Immediate and midterm results of an international registryCATHETERIZATION AND CARDIOVASCULAR INTERVENTIONS, Issue 4 2006Ralf Holzer MD Abstract Objective: To report the immediate and midterm results of transcatheter closure of perimembranous ventricular septal defect (PmVSD) using the Amplatzer membranous VSD occluder (AMVSD). Methods: Between April 2002 and August 2004, 100 patients underwent an attempt of percutaneous device closure of PmVSD using the AMVSD in 24 international centers. The median age was 9.0 years (0.7,58 years) and the median weight was 27.5 kg (7,121 kg). Results: A device was successfully deployed in 93/100 (93%) patients. Reasons for procedural failure were an increased gradient across the left ventricle outflow tract in one patient, aortic regurgitation in 2 patients, and inability to securely position the device in 4 patients. The median VSD size by TEE was 7.0 mm (1.5,13 mm), median device size 10 mm (4,16 mm) and median fluoroscopy time 22.1 min (8.9,96.0 min). Weight below 10 kg (P = 0.0392), inlet extension of the VSD (P = 0.0139) and aortic cusp prolapse into the VSD (P = 0.0084) were significantly associated with a lower procedural success. Patients have been followed up for a median of 182 days (1,763 days). There were no procedure-related deaths. Complications were encountered in 29/100 (29%) patients, including rhythm or conduction anomalies in 13 patients (two with complete heart block requiring permanent pacemaker implantation), new or increased aortic (9 patients) or tricuspid (9 patients) regurgitation, most of which were classified as trivial or mild. Patients with a weight below 10 kg had a significantly higher incidence of adverse events than patients with a weight above 10 kg (58.3% versus 25.0%, P = 0.0285). Immediately after device release complete closure of the defect was present in 54/93 (58.1%) patients, increasing to 46/55 (83.6%) patients at 6-months follow-up (P = 0.0012). Left ventricle end-diastolic diameter decreased from a median of 44 mm prior to device closure to a median of 39 mm at 6-months postprocedure (P = 0.0015). Conclusion: Closure of PmVSDs using the AMVSD occluder is safe and effective. However, longer follow-up period is warranted prior to the wide spread use of this device. © 2006 Wiley-Liss, Inc. [source] Pheochromocytoma in childhood: implication for further diagnostic proceduresACTA PAEDIATRICA, Issue 12 2004O Beck We report on our experience with two patients with pheochromocytoma. One patient underwent surgery of pheochromocytoma at the age of 30 y; 18 y later, medullary thyroid carcinoma (MTC) was detected in his son. Subsequently, multiple endocrine neoplasia (MEN) type 2A was diagnosed by genetic examination in both father and son. Further diagnostic procedures also revealed an MTC in the father. The other patient suffered from bifocal pheochromocytoma of the left suprarenal gland. Diagnostic work-up revealed papillary thyroid carcinoma, which was also detected in the mother 8 mo later. Whereas a point mutation in SDHB gene was found in the son, no genetic abnormality was detected in the mother. Conclusion: Every pheochromocytoma in childhood warrants further diagnostic work-up, including genetic examination. In addition, clinical data of patients suffering from pheochromocytoma and papillary thyroid carcinoma should be collected by an international registry, and a joint effort should be undertaken in order to define possible underlying mutated genes in these patients. [source] | ||||||||||