Dataset Information

The International Prospective Glanzmann Thrombasthenia Registry: Pediatric Treatment and Outcomes.

ABSTRACT: Background ?Standard treatment for Glanzmann thrombasthenia (GT), a severe inherited bleeding disorder, is platelet transfusion. Recombinant activated factor VII (rFVIIa) is reported to be effective in GT with platelet antibodies and/or refractoriness to platelet transfusions. Methods ?We evaluated rFVIIa effectiveness and safety for the treatment and prevention of surgical and nonsurgical bleeding in children <18?years old, with or without platelet antibodies and/or refractoriness, as reported in the GT Registry (GTR). Data were used from the GTR, an international, multicenter, observational, postmarketing study of rFVIIa that prospectively collected data on the treatment and outcomes of bleeds in patients with GT. Only patients with a diagnosis of congenital GT were included in the registry. Results ?Between 2007 and 2011, 27 children were treated for 44 surgical procedures (minor: 36; major: 8); nonsurgical bleeds occurred in 104 patients (599 episodes: severe, 145; moderate, 454; spontaneous, 423; posttraumatic, 176). The effectiveness of treatment for minor procedures, major procedures, nonsurgical bleeds was 6/6, 1/1, and 75/84 for rFVIIa, 6/6, 2/2, and 64/76 for rFVIIa?+?antifibrinolytics (AF), 11/12, 1/1, and 162/214 for platelets?±?AF, and 5/6, 0/3, and 33/45 for rFVIIa?+?platelets?±?AF. In all, 25 adverse events were reported in children; no thromboembolic events were reported. Conclusion ?For all patients, regardless of platelet antibody or refractoriness status, rFVIIa, administered with or without platelets (± AF), provided effective hemostasis with a low frequency of adverse events in surgical, as well as nonsurgical, bleeding in patients with GT. clinicaltrials.gov identifier: NCT01476423.

SUBMITTER: Zotz RB

PROVIDER: S-EPMC6742499 | biostudies-literature | 2019 Jul

REPOSITORIES: biostudies-literature

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The International Prospective Glanzmann Thrombasthenia Registry: Pediatric Treatment and Outcomes.

Zotz Rainer B RB Poon Man-Chiu MC Di Minno Giovanni G D'Oiron Roseline R

TH open : companion journal to thrombosis and haemostasis 20190701 3

<b>Background</b> Standard treatment for Glanzmann thrombasthenia (GT), a severe inherited bleeding disorder, is platelet transfusion. Recombinant activated factor VII (rFVIIa) is reported to be effective in GT with platelet antibodies and/or refractoriness to platelet transfusions. <b>Methods</b> We evaluated rFVIIa effectiveness and safety for the treatment and prevention of surgical and nonsurgical bleeding in children <18 years old, with or without platelet antibodies and/or refractoriness ...[more]

PMID: 31523745

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Project description:Standard treatment for Glanzmann thrombasthenia is platelet transfusion. Recombinant activated factor VII has been shown to be successful in patients with Glanzmann thrombasthenia with platelet antibodies or who are refractory to platelet transfusions. The Glanzmann Thrombasthenia Registry prospectively collected worldwide information on the effectiveness and safety of platelet transfusion, recombinant activated factor VII and/or antifibrinolytics for the treatment of bleeds in patients with Glanzmann thrombasthenia. Data relating to 829 non-surgical bleeding episodes were entered into the Glanzmann Thrombasthenia Registry (severe/moderate: 216/613; spontaneous/post-traumatic: 630/199). Recombinant activated factor VII alone was used in 124/829 bleeds, recombinant activated factor VII+antifibrinolytics in 107/829, platelets±antifibrinolytics in 312/829, antifibrinolytics alone in 219/829, and recombinant activated factor VII+platelets±antifibrinolytics in 67/829. The proportion of successful treatments to stop bleeding was 91.0% in cases treated with recombinant activated factor VII only, 82.7% for recombinant activated factor VII+antifibrinolytics, 72.7% for treatment with recombinant activated factor VII+platelets±antifibrinolytics, 78.8% for platelets±antifibrinolytics and 84.7% for antifibrinolytics alone. Treatment failure was documented in 18 bleeding events (2% of the total treatments), the majority of which were in patients receiving treatment with antifibrinolytics; bleeding re-started in 6% of bleeds after initial effective treatment. Thirty-five adverse events were reported, none of which was a thromboembolic event. Among treatments that included recombinant activated factor VII, only one patient reported three possibly drug-related non-serious adverse events (nausea, dyspnea and headache). To conclude, non-surgical bleeds were common and often severe in Glanzmann thrombasthenia; both platelets and recombinant activated factor VII appeared to be effective, and with good safety profiles, for the treatment of non-surgical bleeds. This trial was registered at clinicaltrials.gov identifier: NCT01476423.

Project description:Background and aimsCardiac arrest (CA) in children is a major public health problem. Thanks to advances in cardiopulmonary resuscitation (CPR) guidelines and teaching skills, results in children have improved. However, pediatric CA has a very high mortality. In the treatment of in-hospital CA there are still multiple controversies. The objective of this study is to develop a multicenter and international registry of in-hospital pediatric cardiac arrest including the diversity of management in different clinical and social contexts. Participation in this register will enable the evaluation of the diagnosis of CA, CPR and post-resuscitation care and its influence in survival and neurological prognosis.MethodsAn intrahospital CA data recording protocol has been designed following the Utstein model. Database is hosted according to European legislation regarding patient data protection. It is drafted in English and Spanish. Invitation to participate has been sent to Spanish, European and Latinamerican hospitals. Variables included, asses hospital characteristics, the resuscitation team, patient's demographics and background, CPR, post-resuscitation care, mortality, survival and long-term evolution. Survival at hospital discharge will be evaluated as a primary outcome and survival with good neurological status as a secondary outcome, analyzing the different factors involved in them. The study design is prospective, observational registry of a cohort of pediatric CA.ConclusionsThis study represents the development of a registry of in-hospital CA in childhood. Its development will provide access to CPR data in different hospital settings and will allow the analysis of current controversies in the treatment of pediatric CA and post-resuscitation care. The results may contribute to the development of further international recommendations. Trial register: ClinicalTrials.gov Identifier: NCT04675918. Registered 19 December 2020 - Retrospectively registered, https://clinicaltrials.gov/ct2/show/record/NCT04675918?cond=pediatric+cardiac+arrest&draw=2&rank=10.

Dataset Information

The International Prospective Glanzmann Thrombasthenia Registry: Pediatric Treatment and Outcomes.

Publications

The International Prospective Glanzmann Thrombasthenia Registry: Pediatric Treatment and Outcomes.

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