The Experts below are selected from a list of 115041 Experts worldwide ranked by ideXlab platform
Kumanan Wilson - One of the best experts on this subject based on the ideXlab platform.
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evaluation of the quality of Clinical Data Collection for a pan canadian cohort of children affected by inherited metabolic diseases lessons learned from the canadian inherited metabolic diseases research network
Orphanet Journal of Rare Diseases, 2020Co-Authors: Kylie Tingley, Monica Lamoureux, Michael Pugliese, Michael T Geraghty, Jonathan B Kronick, Beth K Potter, Doug Coyle, Kumanan WilsonAbstract:The Canadian Inherited Metabolic Diseases Research Network (CIMDRN) is a pan-Canadian practice-based research network of 14 Hereditary Metabolic Disease Treatment Centres and over 50 investigators. CIMDRN aims to develop evidence to improve health outcomes for children with inherited metabolic diseases (IMD). We describe the development of our Clinical Data Collection platform, discuss our Data quality management plan, and present the findings to date from our Data quality assessment, highlighting key lessons that can serve as a resource for future Clinical research initiatives relating to rare diseases. At participating centres, children born from 2006 to 2015 who were diagnosed with one of 31 targeted IMD were eligible to participate in CIMDRN’s Clinical research stream. For all participants, we collected a minimum Data set that includes information about demographics and diagnosis. For children with five prioritized IMD, we collected longitudinal Data including interventions, Clinical outcomes, and indicators of disease management. The Data quality management plan included: design of user-friendly and intuitive Clinical Data Collection forms; validation measures at point of Data entry, designed to minimize Data entry errors; regular communications with each CIMDRN site; and routine review of aggregate Data. As of June 2019, CIMDRN has enrolled 798 participants of whom 764 (96%) have complete minimum Data set information. Results from our Data quality assessment revealed that potential Data quality issues were related to interpretation of definitions of some variables, participants who transferred care across institutions, and the organization of information within the patient charts (e.g., neuropsychological test results). Little information was missing regarding disease ascertainment and diagnosis (e.g., ascertainment method – 0% missing). Using several Data quality management strategies, we have established a comprehensive Clinical Database that provides information about care and outcomes for Canadian children affected by IMD. We describe quality issues and lessons for consideration in future Clinical research initiatives for rare diseases, including accurately accommodating different clinic workflows and balancing comprehensiveness of Data Collection with available resources. Integrating Data Collection within Clinical care, leveraging electronic medical records, and implementing core outcome sets will be essential for achieving sustainability.
Michael T Geraghty - One of the best experts on this subject based on the ideXlab platform.
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evaluation of the quality of Clinical Data Collection for a pan canadian cohort of children affected by inherited metabolic diseases lessons learned from the canadian inherited metabolic diseases research network
Orphanet Journal of Rare Diseases, 2020Co-Authors: Kylie Tingley, Monica Lamoureux, Michael Pugliese, Michael T Geraghty, Jonathan B Kronick, Beth K Potter, Doug Coyle, Kumanan WilsonAbstract:The Canadian Inherited Metabolic Diseases Research Network (CIMDRN) is a pan-Canadian practice-based research network of 14 Hereditary Metabolic Disease Treatment Centres and over 50 investigators. CIMDRN aims to develop evidence to improve health outcomes for children with inherited metabolic diseases (IMD). We describe the development of our Clinical Data Collection platform, discuss our Data quality management plan, and present the findings to date from our Data quality assessment, highlighting key lessons that can serve as a resource for future Clinical research initiatives relating to rare diseases. At participating centres, children born from 2006 to 2015 who were diagnosed with one of 31 targeted IMD were eligible to participate in CIMDRN’s Clinical research stream. For all participants, we collected a minimum Data set that includes information about demographics and diagnosis. For children with five prioritized IMD, we collected longitudinal Data including interventions, Clinical outcomes, and indicators of disease management. The Data quality management plan included: design of user-friendly and intuitive Clinical Data Collection forms; validation measures at point of Data entry, designed to minimize Data entry errors; regular communications with each CIMDRN site; and routine review of aggregate Data. As of June 2019, CIMDRN has enrolled 798 participants of whom 764 (96%) have complete minimum Data set information. Results from our Data quality assessment revealed that potential Data quality issues were related to interpretation of definitions of some variables, participants who transferred care across institutions, and the organization of information within the patient charts (e.g., neuropsychological test results). Little information was missing regarding disease ascertainment and diagnosis (e.g., ascertainment method – 0% missing). Using several Data quality management strategies, we have established a comprehensive Clinical Database that provides information about care and outcomes for Canadian children affected by IMD. We describe quality issues and lessons for consideration in future Clinical research initiatives for rare diseases, including accurately accommodating different clinic workflows and balancing comprehensiveness of Data Collection with available resources. Integrating Data Collection within Clinical care, leveraging electronic medical records, and implementing core outcome sets will be essential for achieving sustainability.
Andrew C Harris - One of the best experts on this subject based on the ideXlab platform.
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international multicenter standardization of acute graft versus host disease Clinical Data Collection a report from the mount sinai acute gvhd international consortium
Biology of Blood and Marrow Transplantation, 2016Co-Authors: Andrew C Harris, Rachel Young, Steven M Devine, William J Hogan, Francis Ayuk, Udomsak Bunworasate, Chantiya Chanswangphuwana, Yvonne A EfeberaAbstract:Acute graft-versus-host disease (GVHD) remains a leading cause of morbidity and nonrelapse mortality after allogeneic hematopoietic cell transplantation. The Clinical staging of GVHD varies greatly between transplant centers and is frequently not agreed on by independent reviewers. The lack of standardized approaches to handle common sources of discrepancy in GVHD grading likely contributes to why promising GVHD treatments reported from single centers have failed to show benefit in randomized multicenter Clinical trials. We developed guidelines through international expert consensus opinion to standardize the diagnosis and Clinical staging of GVHD for use in a large international GVHD research consortium. During the first year of use, the guidance followed discussion of complex Clinical phenotypes by experienced transplant physicians and Data managers. These guidelines increase the uniformity of GVHD symptom capture, which may improve the reproducibility of GVHD Clinical trials after further prospective validation.
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guidelines for the standardization of acute graft versus host disease Clinical Data Collection an international consensus report
Blood, 2015Co-Authors: Andrew C Harris, Rachel Young, Steven M Devine, William J Hogan, Francis Ayuk, Udomsak Bunworasate, Chantiya Chanswangphuwana, Yvonne A Efebera, Ernst HollerAbstract:Clinical GVHD staging varies between centers and is not agreed upon by independent reviewers (Weisdorf, BBMT 2003). These inconsistencies help explain why promising GVHD treatments from single center studies have not reproduced in multicenter trials. To address this issue, our international GVHD research consortium has developed guidance that has been refined through consensus following case discussions, resulting in uniform and reproducible GVHD Clinical symptom reporting. We record all raw target organ symptom Data and apply staging based upon this Data (Table 1). Only areas of erythema, bullae and desquamation are quantified because other skin changes are not typical of active GVHD rash. Upper GI symptoms must meet thresholds to diagnose GVHD: anorexia with associated weight loss, nausea and/or 2+ vomiting episodes/day lasting 3+ days. For lower GI GVHD we collect stool volumes excluding formed/mostly-formed stools. Unquantified episodes are counted at 200 ml per episode (3 ml/kg for children We classify biopsy report interpretation into four standardized categories: Positive (unequivocal presence of GVHD), Equivocal (findings suggestive of GVHD, but the pathologist cannot confirm the diagnosis), Non-diagnostic (no abnormalities or subtle findings insufficient to determine etiology), and Non-GVHD etiology (definitive evidence of another diagnosis without concomitant features suggestive of GVHD). We then combine the biopsy interpretation with Clinical decision making to assign an overall confidence level to the diagnosis of GVHD in each target organ (Table 2): Confirmed (positive biopsy, regardless of clinician treatment decision), Probable (GVHD diagnosis is sufficiently favored that treatment is given without a positive biopsy), Possible (symptoms present without a positive biopsy; not treated as GVHD), Negative (no symptoms or symptoms are present but a GVHD diagnosis is not entertained and a non-GVHD etiology is identified). Uniform Clinical Data Collection is essential for future GVHD trials and requires application of clear guidance. While still subject to refinement, these guidelines, in use for 2 years by an international research consortium, are designed to be clear, easy to apply, and for Clinical trial use. Disclosures Devine: Genzyme: Research Funding. Chen: Bayer: Consultancy, Research Funding. Porter: Novartis: Patents & Royalties, Research Funding. Levine: Novartis: Consultancy.
Kylie Tingley - One of the best experts on this subject based on the ideXlab platform.
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evaluation of the quality of Clinical Data Collection for a pan canadian cohort of children affected by inherited metabolic diseases lessons learned from the canadian inherited metabolic diseases research network
Orphanet Journal of Rare Diseases, 2020Co-Authors: Kylie Tingley, Monica Lamoureux, Michael Pugliese, Michael T Geraghty, Jonathan B Kronick, Beth K Potter, Doug Coyle, Kumanan WilsonAbstract:The Canadian Inherited Metabolic Diseases Research Network (CIMDRN) is a pan-Canadian practice-based research network of 14 Hereditary Metabolic Disease Treatment Centres and over 50 investigators. CIMDRN aims to develop evidence to improve health outcomes for children with inherited metabolic diseases (IMD). We describe the development of our Clinical Data Collection platform, discuss our Data quality management plan, and present the findings to date from our Data quality assessment, highlighting key lessons that can serve as a resource for future Clinical research initiatives relating to rare diseases. At participating centres, children born from 2006 to 2015 who were diagnosed with one of 31 targeted IMD were eligible to participate in CIMDRN’s Clinical research stream. For all participants, we collected a minimum Data set that includes information about demographics and diagnosis. For children with five prioritized IMD, we collected longitudinal Data including interventions, Clinical outcomes, and indicators of disease management. The Data quality management plan included: design of user-friendly and intuitive Clinical Data Collection forms; validation measures at point of Data entry, designed to minimize Data entry errors; regular communications with each CIMDRN site; and routine review of aggregate Data. As of June 2019, CIMDRN has enrolled 798 participants of whom 764 (96%) have complete minimum Data set information. Results from our Data quality assessment revealed that potential Data quality issues were related to interpretation of definitions of some variables, participants who transferred care across institutions, and the organization of information within the patient charts (e.g., neuropsychological test results). Little information was missing regarding disease ascertainment and diagnosis (e.g., ascertainment method – 0% missing). Using several Data quality management strategies, we have established a comprehensive Clinical Database that provides information about care and outcomes for Canadian children affected by IMD. We describe quality issues and lessons for consideration in future Clinical research initiatives for rare diseases, including accurately accommodating different clinic workflows and balancing comprehensiveness of Data Collection with available resources. Integrating Data Collection within Clinical care, leveraging electronic medical records, and implementing core outcome sets will be essential for achieving sustainability.
Yvonne A Efebera - One of the best experts on this subject based on the ideXlab platform.
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international multicenter standardization of acute graft versus host disease Clinical Data Collection a report from the mount sinai acute gvhd international consortium
Biology of Blood and Marrow Transplantation, 2016Co-Authors: Andrew C Harris, Rachel Young, Steven M Devine, William J Hogan, Francis Ayuk, Udomsak Bunworasate, Chantiya Chanswangphuwana, Yvonne A EfeberaAbstract:Acute graft-versus-host disease (GVHD) remains a leading cause of morbidity and nonrelapse mortality after allogeneic hematopoietic cell transplantation. The Clinical staging of GVHD varies greatly between transplant centers and is frequently not agreed on by independent reviewers. The lack of standardized approaches to handle common sources of discrepancy in GVHD grading likely contributes to why promising GVHD treatments reported from single centers have failed to show benefit in randomized multicenter Clinical trials. We developed guidelines through international expert consensus opinion to standardize the diagnosis and Clinical staging of GVHD for use in a large international GVHD research consortium. During the first year of use, the guidance followed discussion of complex Clinical phenotypes by experienced transplant physicians and Data managers. These guidelines increase the uniformity of GVHD symptom capture, which may improve the reproducibility of GVHD Clinical trials after further prospective validation.
-
guidelines for the standardization of acute graft versus host disease Clinical Data Collection an international consensus report
Blood, 2015Co-Authors: Andrew C Harris, Rachel Young, Steven M Devine, William J Hogan, Francis Ayuk, Udomsak Bunworasate, Chantiya Chanswangphuwana, Yvonne A Efebera, Ernst HollerAbstract:Clinical GVHD staging varies between centers and is not agreed upon by independent reviewers (Weisdorf, BBMT 2003). These inconsistencies help explain why promising GVHD treatments from single center studies have not reproduced in multicenter trials. To address this issue, our international GVHD research consortium has developed guidance that has been refined through consensus following case discussions, resulting in uniform and reproducible GVHD Clinical symptom reporting. We record all raw target organ symptom Data and apply staging based upon this Data (Table 1). Only areas of erythema, bullae and desquamation are quantified because other skin changes are not typical of active GVHD rash. Upper GI symptoms must meet thresholds to diagnose GVHD: anorexia with associated weight loss, nausea and/or 2+ vomiting episodes/day lasting 3+ days. For lower GI GVHD we collect stool volumes excluding formed/mostly-formed stools. Unquantified episodes are counted at 200 ml per episode (3 ml/kg for children We classify biopsy report interpretation into four standardized categories: Positive (unequivocal presence of GVHD), Equivocal (findings suggestive of GVHD, but the pathologist cannot confirm the diagnosis), Non-diagnostic (no abnormalities or subtle findings insufficient to determine etiology), and Non-GVHD etiology (definitive evidence of another diagnosis without concomitant features suggestive of GVHD). We then combine the biopsy interpretation with Clinical decision making to assign an overall confidence level to the diagnosis of GVHD in each target organ (Table 2): Confirmed (positive biopsy, regardless of clinician treatment decision), Probable (GVHD diagnosis is sufficiently favored that treatment is given without a positive biopsy), Possible (symptoms present without a positive biopsy; not treated as GVHD), Negative (no symptoms or symptoms are present but a GVHD diagnosis is not entertained and a non-GVHD etiology is identified). Uniform Clinical Data Collection is essential for future GVHD trials and requires application of clear guidance. While still subject to refinement, these guidelines, in use for 2 years by an international research consortium, are designed to be clear, easy to apply, and for Clinical trial use. Disclosures Devine: Genzyme: Research Funding. Chen: Bayer: Consultancy, Research Funding. Porter: Novartis: Patents & Royalties, Research Funding. Levine: Novartis: Consultancy.