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1.
Res Pract Thromb Haemost ; 7(4): 100183, 2023 May.
Article in English | MEDLINE | ID: mdl-37538495

ABSTRACT

Background: Glanzmann thrombasthenia (GT) is a rare congenital platelet function disorder associated with a severe bleeding diathesis. Thrombotic manifestations remain a rare condition. We report here the first case of recurrent venous thromboembolism (VTE) successfully treated with apixaban in a patient with GT. Our patient's morbid obesity was an additional challenge. Key Clinical Question: The Key Clinical Question was to determine if direct oral anticoagulants are suitable for patients with both obesity and GT. Clinical Approach: In our patient, the first episode of VTE occurred after the use of a low dose of activated recombinant factor VII for a minor procedure, whereas the second was unprovoked. Administration of rivaroxaban very quickly led to the appearance of bleeding symptoms and subsequently led to poor compliance and extension of deep vein thrombosis. The patient was switched to apixaban, with very good efficacy and safety over the cumulative 18 months of use. Conclusion: The last updated guidelines now recommend the use of rivaroxaban and apixaban for management of VTE in patients with obesity. Regarding patients with GT, there is still insufficient data on the use of direct oral anticoagulants. Management of thrombotic manifestations in these patients remains a rare and complex condition and could be improved by the creation of a specific international registry.

3.
Eur J Pediatr ; 180(3): 877-884, 2021 Mar.
Article in English | MEDLINE | ID: mdl-32959075

ABSTRACT

Myocarditis and Kawasaki disease are common but usually distinct diseases in children. During the coronavirus pandemic (COVID-19), reports of a new form of myocarditis with clinical features of Kawasaki appeared. We investigated the place of this new disease in the spectrum encompassing Kawasaki disease and myocarditis.Thirty two consecutive children referred to our centre for a suspicion of Kawasaki or a diagnosis of myocarditis were included and eventually divided into four groups: 11 Kawasaki diseases, 6 Kawasaki syndromes (children with another diagnosis), 7 myocarditis without Kawasaki clinical feature and 7 myocarditis with incomplete Kawasaki clinical features. All were treated with immunoglobulins except those of the myocarditis group. The survival rate was 91%. The 7 children with myocarditis and clinical features of incomplete Kawasaki were all positive for SARS-CoV-2. They had a transient myocardial failure with a favourable course and none had coronary artery disease.Conclusion: Every COVID-19 child within our population had a mild to severe myocarditis and presented with fever plus two or three Kawasaki clinical features. Short-term evolution was good for these children. This new disease seems to fill the gap between isolated myocarditis and Kawasaki disease. What is Known: • A new paediatric disease close to Kawasaki disease appeared during the COVID-19 pandemic What is New: • In our population, children presented with fever, vivid Kawasaki clinical features (although the Kawasaki syndrome was always incomplete) and a myocarditis without coronary abnormalities. • The new disease fills the gap between paediatric myocarditis and Kawasaki disease but its prognosis is much better.


Subject(s)
COVID-19/diagnosis , Mucocutaneous Lymph Node Syndrome/virology , Myocarditis/virology , Systemic Inflammatory Response Syndrome/diagnosis , Adolescent , COVID-19/complications , Child , Child, Preschool , Diagnosis, Differential , Female , Humans , Infant , Infant, Newborn , Male , Mucocutaneous Lymph Node Syndrome/diagnosis , Myocarditis/diagnosis , Retrospective Studies , Systemic Inflammatory Response Syndrome/complications
4.
Ann Biol Clin (Paris) ; 78(1): 35-46, 2020 02 01.
Article in French | MEDLINE | ID: mdl-32108578

ABSTRACT

Only a few studies on real-world clinical use of recombinant factor VIII -fusionned with Fc (rFVIIIFc, efmoroctocog alpha) have been performed to date, with data on the annual bleeding rate (ABR), the prophylaxis regimen, and FVIII consumption. The aim of our study was to report the real-world clinical application of rFVIIIFc with additional elements, both biological and clinical. A prospective monocentric study has been conducted in the Haemophilia treatment center (HTC) of the Strasbourg university hospital among the severe haemophilia A patients. Thirty male patients were enrolled in the study. After injection of rFVIIIFc, the average time spent above 5%, 2% and 1% of FVIII was respectively almost 3, 4 and 5 days. The average half-life was 15.8 hours. A strong linear correlation between incremental recovery of rFVIIIFc and weight and between rFVIIIFc half-life and basal VWF:Ag level was observed. FVIII activity measurement for rFVIIIFc showed similar results than those previously published. In the follow-up, residual FVIII activity was on average the one of a mild haemophilia patient, corroborated by the results of endogenous thrombin potential of the thrombin generation assay. In clinical practice, rFVIIIFc was well tolerated and patients were mostly satisfied or indifferent of the switch. A single failure was however noticed. No FVIII inhibitor has been detected. Decrease in FVIII consumption was observed, with reduced or unchanged ABR. The switch was an actual success for almost all of the 30 patients, corroborated by satisfactory clinical and biological results.


Subject(s)
Drug Substitution , Factor VIII/therapeutic use , Hemophilia A/drug therapy , Immunoglobulin Fc Fragments/therapeutic use , Recombinant Fusion Proteins/therapeutic use , Adolescent , Adult , Aged , Blood Coagulation Tests , Factor VIII/analysis , Factor VIII/pharmacokinetics , Half-Life , Hemophilia A/blood , Hemophilia A/metabolism , Hemophilia A/pathology , Humans , Male , Middle Aged , Patient Satisfaction , Recombinant Fusion Proteins/pharmacokinetics , Severity of Illness Index , Thrombin/metabolism , Young Adult
5.
Anticancer Res ; 30(3): 963-5, 2010 Mar.
Article in English | MEDLINE | ID: mdl-20393020

ABSTRACT

BACKGROUND: Delayed elimination of methotrexate associated with serious side-effects has been attributed to the co-administration of benzimidazole proton pump inhibitors. PATIENTS AND METHODS: We have retrospectively analyzed the causes of delayed methotrexate elimination in patients who had received the rescue agent glucarpidase to evaluate the potential implication of benzimidazoles. RESULTS: Between 2002 and 2008, six patients (mean age: 30 years; range: 4-74 years) were treated with glucarpidase. Delayed elimination associated with impaired renal function occured after the first cycle except in 2 patients (2nd and 8th administration of high-dose methotrexate). The possible causes of delayed elimination identified were: insufficient hydration (n=1) and drug-drug interactions (n=5). The potential drug-drug interactions included the co-administration of piperacillin/tazobactam (n=1) and proton pump inhibitors (omeprazole, n=3; esomeprazole, n=2). Impaired elimination of methotrexate was not observed either in the 3 patients who were treated further or during the previous cycles of the 2 pretreated patients in relation to the absence of co-prescription of proton pump inhibitors. CONCLUSION: In line with the recent literature and given the prohibitive cost of glucarpidase, we have advocated the cessation of proton pump inhibitors administration during methotrexate treatment.


Subject(s)
Antimetabolites, Antineoplastic/pharmacokinetics , Antimetabolites, Antineoplastic/poisoning , Antineoplastic Combined Chemotherapy Protocols/pharmacokinetics , Benzimidazoles/pharmacology , Methotrexate/pharmacokinetics , Methotrexate/poisoning , Proton Pump Inhibitors/pharmacology , Adolescent , Adult , Aged , Antimetabolites, Antineoplastic/administration & dosage , Antineoplastic Combined Chemotherapy Protocols/therapeutic use , Benzimidazoles/administration & dosage , Bone Neoplasms/drug therapy , Bone Neoplasms/metabolism , Child , Child, Preschool , Drug Interactions , Humans , Lymphoma/drug therapy , Lymphoma/metabolism , Methotrexate/administration & dosage , Middle Aged , Neuroblastoma/drug therapy , Neuroblastoma/metabolism , Osteosarcoma/drug therapy , Osteosarcoma/metabolism , Proton Pump Inhibitors/administration & dosage , Retrospective Studies , Young Adult , gamma-Glutamyl Hydrolase/therapeutic use
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