The Experts below are selected from a list of 169719 Experts worldwide ranked by ideXlab platform
Dietmar Berger - One of the best experts on this subject based on the ideXlab platform.
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Romiplostim or Standard of Care in patients with immune thrombocytopenia.
The New England Journal of Medicine, 2010Co-Authors: David J Kuter, Mathias J Rummel, Ralph V Boccia, Ingrid Pabinger, Dominik Selleslag, Francesco Rodeghiero, Beng H Chong, Xuena Wang, B. Gail Macik, Dietmar BergerAbstract:Romiplostim, a thrombopoietin mimetic, increases platelet counts in patients with immune thrombocytopenia, with few adverse effects. Methods In this open-label, 52-week study, we randomly assigned 234 adult patients with immune thrombocytopenia, who had not undergone splenectomy, to receive the Standard of Care (77 patients) or weekly subcutaneous injections of romiplostim (157 patients). Primary end points were incidences of treatment failure and splenectomy. Secondary end points included the rate of a platelet response (a platelet count >50×10 9 per liter at any scheduled visit), safety outcomes, and the quality of life. Results The rate of a platelet response in the romiplostim group was 2.3 times that in the Standard-of-Care group (95% confidence interval [CI], 2.0 to 2.6; P
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romiplostim or Standard of Care in patients with immune thrombocytopenia
The New England Journal of Medicine, 2010Co-Authors: David J Kuter, Mathias J Rummel, Ralph V Boccia, Gail B Macik, Ingrid Pabinger, Dominik Selleslag, Francesco Rodeghiero, Beng H Chong, Xuena Wang, Dietmar BergerAbstract:Romiplostim, a thrombopoietin mimetic, increases platelet counts in patients with immune thrombocytopenia, with few adverse effects. Methods In this open-label, 52-week study, we randomly assigned 234 adult patients with immune thrombocytopenia, who had not undergone splenectomy, to receive the Standard of Care (77 patients) or weekly subcutaneous injections of romiplostim (157 patients). Primary end points were incidences of treatment failure and splenectomy. Secondary end points included the rate of a platelet response (a platelet count >50×10 9 per liter at any scheduled visit), safety outcomes, and the quality of life. Results The rate of a platelet response in the romiplostim group was 2.3 times that in the Standard-of-Care group (95% confidence interval [CI], 2.0 to 2.6; P<0.001). Patients receiving romiplostim had a significantly lower incidence of treatment failure (18 of 157 patients [11%]) than those receiving the Standard of Care (23 of 77 patients [30%], P<0.001) (odds ratio with romiplostim, 0.31; 95% CI, 0.15 to 0.61). Splenectomy also was performed less frequently in patients receiving romiplostim (14 of 157 patients [9%]) than in those receiving the Standard of Care (28 of 77 patients [36%], P<0.001) (odds ratio, 0.17; 95% CI, 0.08 to 0.35). The romiplostim group had a lower rate of bleeding events, fewer blood transfusions, and greater improvements in the quality of life than the Standard-of-Care group. Serious adverse events occurred in 23% of patients (35 of 154) receiving romiplostim and 37% of patients (28 of 75) receiving the Standard of Care. Conclusions Patients treated with romiplostim had a higher rate of a platelet response, lower incidence of treatment failure and splenectomy, less bleeding and fewer blood transfusions, and a higher quality of life than patients treated with the Standard of Care. (Funded by Amgen; ClinicalTrials.gov number, NCT00415532.)
Sapna Pradyuman Patel - One of the best experts on this subject based on the ideXlab platform.
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neoadjuvant plus adjuvant dabrafenib and trametinib versus Standard of Care in patients with high risk surgically resectable melanoma a single centre open label randomised phase 2 trial
Lancet Oncology, 2018Co-Authors: Rodabe N Amaria, Peter A Prieto, Michael T Tetzlaff, Alexandre Reuben, Miles C Andrews, Merrick I Ross, Isabella C Glitza, Janice N Cormier, Hussein Abdulhassan Tawbi, Sapna Pradyuman PatelAbstract:Summary Background Dual BRAF and MEK inhibition produces a response in a large number of patients with stage IV BRAF-mutant melanoma. The existing Standard of Care for patients with clinical stage III melanoma is upfront surgery and consideration for adjuvant therapy, which is insufficient to cure most patients. Neoadjuvant targeted therapy with BRAF and MEK inhibitors (such as dabrafenib and trametinib) might provide clinical benefit in this high-risk p opulation. Methods We undertook this single-centre, open-label, randomised phase 2 trial at the University of Texas MD Anderson Cancer Center (Houston, TX, USA). Eligible participants were adult patients (aged ≥18 years) with histologically or cytologically confirmed surgically resectable clinical stage III or oligometastatic stage IV BRAFV600E or BRAFV600K (ie, Val600Glu or Val600Lys)-mutated melanoma. Eligible patients had to have an Eastern Cooperative Oncology Group performance status of 0 or 1, a life expectancy of more than 3 years, and no previous exposure to BRAF or MEK inhibitors. Exclusion criteria included metastases to bone, brain, or other sites where complete surgical excision was in doubt. We randomly assigned patients (1:2) to either upfront surgery and consideration for adjuvant therapy (Standard of Care group) or neoadjuvant plus adjuvant dabrafenib and trametinib (8 weeks of neoadjuvant oral dabrafenib 150 mg twice per day and oral trametinib 2 mg per day followed by surgery, then up to 44 weeks of adjuvant dabrafenib plus trametinib starting 1 week after surgery for a total of 52 weeks of treatment). Randomisation was not masked and was implemented by the clinical trial conduct website maintained by the trial centre. Patients were stratified by disease stage. The primary endpoint was investigator-assessed event-free survival (ie, patients who were alive without disease progression) at 12 months in the intent-to-treat population. This trial is registered at ClinicalTrials.gov, number NCT02231775. Findings Between Oct 23, 2014, and April 13, 2016, we randomly assigned seven patients to Standard of Care, and 14 to neoadjuvant plus adjuvant dabrafenib and trametinib. The trial was stopped early after a prespecified interim safety analysis that occurred after a quarter of the participants had been accrued revealed significantly longer event-free survival with neoadjuvant plus adjuvant dabrafenib and trametinib than with Standard of Care. After a median follow-up of 18·6 months (IQR 14·6–23·1), significantly more patients receiving neoadjuvant plus adjuvant dabrafenib and trametinib were alive without disease progression than those receiving Standard of Care (ten [71%] of 14 patients vs none of seven in the Standard of Care group; median event-free survival was 19·7 months [16·2–not estimable] vs 2·9 months [95% CI 1·7–not estimable]; hazard ratio 0·016, 95% CI 0·00012–0·14, p Interpretation Neoadjuvant plus adjuvant dabrafenib and trametinib significantly improved event-free survival versus Standard of Care in patients with high-risk, surgically resectable, clinical stage III–IV melanoma. Although the trial finished early, limiting generalisability of the results, the findings provide proof-of-concept and support the rationale for further investigation of neoadjuvant approaches in this disease. This trial is currently continuing accrual as a single-arm study of neoadjuvant plus adjuvant dabrafenib and trametinib. Funding Novartis Pharmaceuticals Corporation.
David J Kuter - One of the best experts on this subject based on the ideXlab platform.
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Romiplostim or Standard of Care in patients with immune thrombocytopenia.
The New England Journal of Medicine, 2010Co-Authors: David J Kuter, Mathias J Rummel, Ralph V Boccia, Ingrid Pabinger, Dominik Selleslag, Francesco Rodeghiero, Beng H Chong, Xuena Wang, B. Gail Macik, Dietmar BergerAbstract:Romiplostim, a thrombopoietin mimetic, increases platelet counts in patients with immune thrombocytopenia, with few adverse effects. Methods In this open-label, 52-week study, we randomly assigned 234 adult patients with immune thrombocytopenia, who had not undergone splenectomy, to receive the Standard of Care (77 patients) or weekly subcutaneous injections of romiplostim (157 patients). Primary end points were incidences of treatment failure and splenectomy. Secondary end points included the rate of a platelet response (a platelet count >50×10 9 per liter at any scheduled visit), safety outcomes, and the quality of life. Results The rate of a platelet response in the romiplostim group was 2.3 times that in the Standard-of-Care group (95% confidence interval [CI], 2.0 to 2.6; P
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romiplostim or Standard of Care in patients with immune thrombocytopenia
The New England Journal of Medicine, 2010Co-Authors: David J Kuter, Mathias J Rummel, Ralph V Boccia, Gail B Macik, Ingrid Pabinger, Dominik Selleslag, Francesco Rodeghiero, Beng H Chong, Xuena Wang, Dietmar BergerAbstract:Romiplostim, a thrombopoietin mimetic, increases platelet counts in patients with immune thrombocytopenia, with few adverse effects. Methods In this open-label, 52-week study, we randomly assigned 234 adult patients with immune thrombocytopenia, who had not undergone splenectomy, to receive the Standard of Care (77 patients) or weekly subcutaneous injections of romiplostim (157 patients). Primary end points were incidences of treatment failure and splenectomy. Secondary end points included the rate of a platelet response (a platelet count >50×10 9 per liter at any scheduled visit), safety outcomes, and the quality of life. Results The rate of a platelet response in the romiplostim group was 2.3 times that in the Standard-of-Care group (95% confidence interval [CI], 2.0 to 2.6; P<0.001). Patients receiving romiplostim had a significantly lower incidence of treatment failure (18 of 157 patients [11%]) than those receiving the Standard of Care (23 of 77 patients [30%], P<0.001) (odds ratio with romiplostim, 0.31; 95% CI, 0.15 to 0.61). Splenectomy also was performed less frequently in patients receiving romiplostim (14 of 157 patients [9%]) than in those receiving the Standard of Care (28 of 77 patients [36%], P<0.001) (odds ratio, 0.17; 95% CI, 0.08 to 0.35). The romiplostim group had a lower rate of bleeding events, fewer blood transfusions, and greater improvements in the quality of life than the Standard-of-Care group. Serious adverse events occurred in 23% of patients (35 of 154) receiving romiplostim and 37% of patients (28 of 75) receiving the Standard of Care. Conclusions Patients treated with romiplostim had a higher rate of a platelet response, lower incidence of treatment failure and splenectomy, less bleeding and fewer blood transfusions, and a higher quality of life than patients treated with the Standard of Care. (Funded by Amgen; ClinicalTrials.gov number, NCT00415532.)
Gerhard Leitz - One of the best experts on this subject based on the ideXlab platform.
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an open label randomized parallel group study of perioperative epoetin alfa versus Standard of Care for blood conservation in major elective spinal surgery safety analysis
Spine, 2009Co-Authors: Christopher P Stowell, Stanley C Jones, Christopher Enny, Wayne Langholff, Gerhard LeitzAbstract:Study Design. Prospective, open-label, randomized, parallel-group study at 80 centers. Objective. To demonstrate there is no clinically impor - tant additional risk for deep vein thrombosis with perioperative use of epoetin alfa versus Standard of Care in spine surgery without prophylactic anticoagulation. Summary of Background Data. Trials of epoetin alfa in orthopedic surgery that demonstrated no additional risk of thrombovascular events included perioperative pharmacologic anticoagulation. Methods. Subjects received epoe t in alfa 600 U/kg subcutaneously once weekly starting 3 weeks before spinal surgery plus Standard of Care for blood conservation, or Standard of Care alone. Perioperative anticoagulation therapy was not permitted; mechanical deep vein thrombosis prophylaxis was allowed. Doppler imaging for deep vein thrombosis was done on postoperative day 4 (or day of discharge), or for suspected deep vein thrombosis. Deep vein thrombosis was diagnosed by Doppler result or adverse event report. The criterion for no additional risk of deep vein thrombosis was a 1-sided 97.5% upper confidence limit ≤4% between groups. Results. of the 680 subjects analyzed (340 in each treatment group), 16 (4.7%) in the epoetin alfa group and 7 (2.1%) in the Standard of Care group had a diagnosis of deep vein thrombosis either by Doppler or by adverse event report with normal Doppler. The between-group difference was 2.6% (97.5% upper confidence limit, 5.4%). Deep vein thrombosis confirmed by Doppler (4.1% vs. 2.1%), other clinically relevant thrombovascular events (1.5% vs. 0.9%), and all adverse events combined (76.5% vs. 73.2%) occurred with similar frequency in the 2 treatment groups. Conclusion. This study documented a higher incidence of deep vein thrombosis and similar rates of other clinically relevant thrombovascular events with epoetin alfa versus Standard of Care for blood conservation in subjects who did not receive prophylactic anticoagulation before spinal surgery. Antithrombotic prophylaxis should be considered when erythropoietin is used in the surgical setting,
Thomas O Crawford - One of the best experts on this subject based on the ideXlab platform.
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Consensus Statement for Standard of Care in Spinal Muscular Atrophy
Journal of Child Neurology, 2020Co-Authors: Ching H Wang, Mary K. Schroth, Anita K. Simonds, Annie Aloysius, Leslie Morrison, Thomas O Crawford, Richard S Finkel, Brenda Wong, Enrico Bertini, Anthony TrelaAbstract:Spinal muscular atrophy is a neurodegenerative disease that requires multidisciplinary medical Care. Recent progress in the understanding of molecular pathogenesis of spinal muscular atrophy and advances in medical technology have not been matched by similar developments in the Care for spinal muscular atrophy patients. Variations in medical practice coupled with differences in family resources and values have resulted in variable clinical outcomes that are likely to compromise valid measure of treatment effects during clinical trials. The International Standard of Care Committee for Spinal Muscular Atrophy was formed in 2005, with a goal of establishing practice guidelines for clinical Care of these patients. The 12 core committee members worked with more than 60 spinal muscular atrophy experts in the field through conference calls, e-mail communications, a Delphi survey, and 2 in-person meetings to achieve consensus on 5 Care areas: diagnostic/new interventions, pulmonary, gastrointestinal/nutrition, orthopedics/rehabilitation, and palliative Care. Consensus was achieved on several topics related to common medical problems in spinal muscular atrophy, diagnostic strategies, recommendations for assessment and monitoring, and therapeutic interventions in each Care area. A consensus statement was drafted to address the 5 Care areas according to 3 functional levels of the patients: nonsitter, sitter, and walker. The committee also identified several medical practices lacking consensus and warranting further investigation. It is the authors' intention that this document be used as a guideline, not as a practice Standard for their Care. A practice Standard for spinal muscular atrophy is urgently needed to help with the multidisciplinary Care of these patients.
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Standard of Care for Spinal Muscular Atrophy
Spinal Muscular Atrophy, 2016Co-Authors: Thomas O CrawfordAbstract:While spinal muscular atrophy primarily manifests with weakness of skeletal muscle, secondary complications regularly accompany that weakness and further compromise overall functioning. Difficulties with breathing, progressive skeletal deformity, impaired nutrition, and other problems often cause early death, may be discomforting or painful, and limit access to a normal life. Much that is the experience of spinal muscular atrophy is the experience of its complications. Complications also greatly confound clinical trial design, necessitating more expensive trials that have less statistical power to identify a treatment effect. Development and refinement of a current Standard of Care is an ongoing process that aspires to lessen the burden of disease and level of weakness at every age. Advancement of understanding about the best possible Standard of Care aims both to benefit clinical research and to improve the life of affected individuals and their families.
