Search Results - "DiMichele, Donna"
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The principal results of the International Immune Tolerance Study: a randomized dose comparison
Published in Blood (09-02-2012)“…The International Immune Tolerance Study was a multicenter, prospective, randomized comparison of high-dose (HD; 200 IU/kg/d) and low-dose (LD; 50 IU/kg 3…”
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2
Immune tolerance in haemophilia: the long journey to the fork in the road
Published in British journal of haematology (01-10-2012)“…Summary Antibody eradication is the ultimate goal of inhibitor management. The only clinically proven strategy for achieving antigen‐specific tolerance to…”
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3
Hemophilia Therapy — Navigating Speed Bumps on the Innovation Highway
Published in The New England journal of medicine (26-05-2016)“…Hemophilia A is an X-linked hemorrhagic disorder characterized by a congenital absence or decrease in plasma clotting factor VIII, a procoagulation cofactor…”
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A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) Study
Published in Blood (15-01-2007)“…The development of inhibitory antibodies to factor VIII is a serious complication of hemophilia. FEIBA (factor VIII inhibitor-bypassing activity), an activated…”
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5
The polygenic nature of inhibitors in hemophilia A: results from the Hemophilia Inhibitor Genetics Study (HIGS) Combined Cohort
Published in Blood (21-02-2013)“…Studies of determinants of development of inhibitory Abs to factor VIII in people with hemophilia A indicate a complex process involving multiple factors. The…”
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Prior Publication Productivity, Grant Percentile Ranking, and Topic-Normalized Citation Impact of NHLBI Cardiovascular R01 Grants
Published in Circulation research (12-09-2014)“…RATIONALE:We previously demonstrated absence of association between peer-review–derived percentile ranking and raw citation impact in a large cohort of…”
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7
Navigating Speed Bumps on the Innovation Highway in Hemophilia Therapeutics
Published in HemaSphere (01-10-2018)“…The unprecedented emergence of novel therapeutics for both hemophilia A and B during the last half decade has been accompanied by the promise of even more…”
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8
Inhibitors in childhood hemophilia A: Genetic and treatment-related risk factors for development and eradication
Published in Pediatric blood & cancer (2013)“…The development of neutralizing antibodies remains a serious complication of hemophilia replacement therapy. Factor VIII inhibiting antibodies (inhibitors)…”
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Citation Impact of NHLBI R01 Grants Funded Through the American Recovery and Reinvestment Act as Compared to R01 Grants Funded Through a Standard Payline
Published in Circulation research (27-02-2015)“…RATIONALE:The American Recovery and Reinvestment Act (ARRA) allowed National Heart, Lung, and Blood Institute to fund R01 grants that fared less well on peer…”
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10
Planning for the future workforce in hematology research
Published in Blood (30-04-2015)“…The medical research and training enterprise in the United States is complex in both its scope and implementation. Accordingly, adaptations to the associated…”
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Catalyzing innovation in clinical trial design and analysis at NHLBI: a funding opportunity for clinical trials in blood disorders
Published in Blood advances (12-12-2017)Get full text
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12
Executive summary of the NHLBI State of the Science (SOS) Workshop: Overview and next steps in generating a national blueprint for future research on factor VIII inhibitors
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01-07-2019)Get full text
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Origins and organization of the NHLBI State of the Science Workshop: Generating a national blueprint for future research on factor VIII inhibitors
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01-07-2019)“…Introduction The major complication of protein replacement therapy for haemophilia A is the development of anti‐FVIII antibodies or inhibitors that occur in…”
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14
End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings
Published in Blood advances (10-12-2019)“…To address the global burden of sickle cell disease and the need for novel therapies, the American Society of Hematology partnered with the US Food and Drug…”
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15
Management of factor VIII inhibitors
Published in International journal of hematology (01-02-2006)“…The development of inhibitory alloantibodies to factor VIII (FVIII) is a major complication of clotting factor replacement therapy for hemophilia A. Inhibitor…”
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16
Knowledge and therapeutic gaps: a public health problem in the rare coagulation disorders population
Published in American journal of preventive medicine (01-12-2011)“…Rare coagulation disorders (RCDs) present a considerable and multifaceted public health risk. Although inherited RCDs affect a minor segment of any local…”
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17
Inhibitor development in haemophilia B: an orphan disease in need of attention
Published in British journal of haematology (01-08-2007)“…Summary Factor IX (FIX) inhibitors develop in 1·5–3% of haemophilia B patients. Due to its low incidence compared with that in haemophilia A, few comparable…”
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18
Paroxysmal nocturnal hemoglobinuria in pediatric patients
Published in Pediatric blood & cancer (01-09-2012)“…Background Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease in children. The most significant clinical features of PNH include: bone marrow failure,…”
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19
Immune tolerance therapy dose as an outcome predictor
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01-07-2003)“…The question of dose as a successful ITT outcome predictor in haemophilia A and B is an important one on many levels. Increased morbidity associated with…”
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20
Mutation in the factor VII hepatocyte nuclear factor 4α-binding site contributes to factor VII deficiency
Published in Blood coagulation & fibrinolysis (01-10-2011)“…Severe coagulant factor VII (FVII) deficiency in postpubertal dizygotic twin males results from two point mutations in the FVII gene, a promoter region T→C…”
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