LIVIVO - Search results -

Search results

Result 1 - 10 of total 340

Search options

Article ; Online: Heavy menstrual blood loss in patients with von Willebrand disease: an unsolved problem.

Leebeek, Frank W G

The Lancet. Haematology

2023 Volume 10, Issue 8, Page(s) e561–e562

MeSH term(s)	Female ; Humans ; von Willebrand Diseases/complications ; von Willebrand Factor ; Menorrhagia/etiology ; Patients
Chemical Substances	von Willebrand Factor
Language	English
Publishing date	2023-06-26
Publishing country	England
Document type	Journal Article ; Comment
ISSN	2352-3026
ISSN (online)	2352-3026
DOI	10.1016/S2352-3026(23)00165-5
Database	MEDical Literature Analysis and Retrieval System OnLINE

Order via subito

This service is chargeable due to the Delivery terms set by subito. Orders including an article and supplementary material will be classified as separate orders. In these cases, fees will be demanded for each order.

Inter-library loan at ZB MED

Your chosen title can be delivered directly to ZB MED Cologne location if you are registered as a user at ZB MED Cologne.

Article ; Online: A novel VWF-associated thrombolytic agent.

Leebeek, Frank W G

Blood

2022 Volume 140, Issue 26, Page(s) 2770–2771

MeSH term(s)	Humans ; Animals ; Mice ; Fibrinolytic Agents/therapeutic use ; von Willebrand Factor ; Ischemic Stroke ; Fibrinolysis ; Thrombolytic Therapy
Chemical Substances	Fibrinolytic Agents ; von Willebrand Factor
Language	English
Publishing date	2022-12-29
Publishing country	United States
Document type	Editorial ; Comment
ZDB-ID	80069-7
ISSN	1528-0020 ; 0006-4971
ISSN (online)	1528-0020
ISSN	0006-4971
DOI	10.1182/blood.2022018024
Database	MEDical Literature Analysis and Retrieval System OnLINE

In stock of ZB MED Cologne/Königswinter

Uh III Zs.140: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 2021: Bestellungen von Artikeln über das Online-Bestellformular ab Jg. 2022: Lesesaal (EG)
Zs.MO 364: Show issues

Order via subito

Details ▾
- See ZB MED holdings
- Order with fees

Article ; Online: Diagnosis and treatment of von Willebrand disease in 2024 and beyond.

James, Paula / Leebeek, Frank / Casari, Caterina / Lillicrap, David

Haemophilia : the official journal of the World Federation of Hemophilia

2024 Volume 30 Suppl 3, Page(s) 103–111

Abstract: Manuscript background and aim: The diagnosis and clinical care of patients with von Willebrand disease (VWD) has continued to evolve since the characterization of the von Willebrand factor (VWF) gene in 1985. This condition is almost certainly the most ... ...

Abstract	Manuscript background and aim: The diagnosis and clinical care of patients with von Willebrand disease (VWD) has continued to evolve since the characterization of the von Willebrand factor (VWF) gene in 1985. This condition is almost certainly the most common inherited bleeding disorder, and the major symptomatic burden of the disease is experienced by females during their reproductive years. Diagnosis relies on the identification of a personal and family history of excessive mucocutaneous bleeding, and laboratory features consistent with quantitative and/or qualitative abnormalities of VWF. This review focuses on three aspects of VWD management, with current updates and a look into the future. Manuscript themes: First, we will address the role of genetics in the diagnosis and possible therapies for VWD. With current technologies, VWD genetic diagnosis is usually confined to the confirmation of type 2 subtypes of the disease and type 3 VWD analysis for family planning. While type 3 VWD is a potential candidate for the application of gene therapy, no treatments are currently close to entering the clinic. Second, the peri-procedural management of patients with VWD remains an important element of care. The choice of product, its dose and schedule all require careful consideration depending upon the type and disruptive nature of the planned procedure. Lastly, in addition to gene therapy, several other novel therapeutic interventions are also being developed for bleeding and prophylaxis in VWD. These include a VWF aptamer interfering with VWF clearance and bioengineered forms of VWF.
MeSH term(s)	Female ; Humans ; von Willebrand Diseases/diagnosis ; von Willebrand Diseases/genetics ; von Willebrand Diseases/therapy ; von Willebrand Factor/genetics ; von Willebrand Factor/therapeutic use ; von Willebrand Disease, Type 3 ; Hemorrhage/diagnosis
Chemical Substances	von Willebrand Factor
Language	English
Publishing date	2024-03-13
Publishing country	England
Document type	Review ; Journal Article
ZDB-ID	1229713-6
ISSN	1365-2516 ; 1351-8216 ; 1355-0691
ISSN (online)	1365-2516
ISSN	1351-8216 ; 1355-0691
DOI	10.1111/hae.14970
Database	MEDical Literature Analysis and Retrieval System OnLINE

In stock of ZB MED Cologne/Königswinter

Zs.A 4249: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular Jg. 1995 - 2021: Lesesall (2.OG) ab Jg. 2022: Lesesaal (EG)
Zs.MO 450: Show issues

Order via subito

Details ▾
- See ZB MED holdings
- Order with fees

Article ; Online: New Developments in Diagnosis and Management of Acquired Hemophilia and Acquired von Willebrand Syndrome.

Leebeek, Frank W G

HemaSphere

2021 Volume 5, Issue 6, Page(s) e586

Abstract: Acquired hemophilia A and acquired von Willebrand syndrome are rare, but life-threatening bleeding disorders that require prompt diagnosis and treatment by hematologists. Acquired hemophilia A is defined as an acquired severe bleeding tendency caused by ... ...

Abstract	Acquired hemophilia A and acquired von Willebrand syndrome are rare, but life-threatening bleeding disorders that require prompt diagnosis and treatment by hematologists. Acquired hemophilia A is defined as an acquired severe bleeding tendency caused by autoantibody formation against coagulation factor VIII. Acquired von Willebrand syndrome is characterized by a new onset bleeding tendency caused by a reduced concentration and/or function of von Willebrand factor. These disorders are associated with a variety of underlying disorders, including various hematological malignancies, for example, plasma cell disorders, lymphoproliferative disorders, monoclonal gammopathy of undetermined significance, and myeloproliferative neoplasms. It is of utmost important to recognize these acquired bleeding disorders in these patients who are at risk for severe bleeding, and to perform additional diagnostic hemostasis laboratory evaluation. This will enable immediate diagnosis of the acquired bleeding disorder and management of both the bleeding episodes and the causative underlying disorder. In recent years, several new etiological factors for acquired hemophilia A, such as treatment with immune checkpoint inhibitors or DPP-4 inhibitors and SARS-CoV2 infection, and for acquired von Willebrand syndrome, for example, left ventricular assist devices, have been identified and also new treatment options have become available. In this concise review, the most recent data on etiology, diagnosis, and treatment of acquired bleeding disorders are presented and discussed.
Language	English
Publishing date	2021-06-01
Publishing country	United States
Document type	Journal Article ; Review
ISSN	2572-9241
ISSN (online)	2572-9241
DOI	10.1097/HS9.0000000000000586
Database	MEDical Literature Analysis and Retrieval System OnLINE

Order via subito

Inter-library loan at ZB MED

Your chosen title can be delivered directly to ZB MED Cologne location if you are registered as a user at ZB MED Cologne.

Article ; Online: Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues.

Leebeek, Frank W G / Miesbach, Wolfgang

Blood

2021 Volume 138, Issue 11, Page(s) 923–931

Abstract: In the last decade, enormous progress has been made in the development of gene therapy for hemophilia A and B. After the first encouraging results of intravenously administered adeno-associated virus (AAV)-based liver-directed gene therapy in patients ... ...

Abstract	In the last decade, enormous progress has been made in the development of gene therapy for hemophilia A and B. After the first encouraging results of intravenously administered adeno-associated virus (AAV)-based liver-directed gene therapy in patients with severe hemophilia B were reported in 2011, many gene therapy studies have been initiated. Most of these studies, using AAV vectors with various gene constructs, showed sufficient factor VIII and IX expression in patients to significantly reduce the number of bleeds and the need for prophylaxis in most patients with severe hemophilia. This resulted in great clinical benefit for nearly all patients. In this review, we will summarize the most recent findings of reported and ongoing gene therapy trials. We will highlight the successful outcome of trials with focus on the results of recently reported phase 1 trials and preliminary results of phase 2b/3 trials for hemophilia A and B. These new reports also reveal the impact of side effects and drawbacks associated with gene therapy. We will therefore also discuss the limitations and remaining issues of the current gene therapy approaches. These issues must be resolved before gene therapy will be widely available for the hemophilia patient population.
MeSH term(s)	Animals ; Clinical Trials as Topic ; Dependovirus/genetics ; Factor IX/genetics ; Factor VIII/genetics ; Genetic Therapy/methods ; Genetic Vectors/genetics ; Genetic Vectors/therapeutic use ; Hemophilia A/genetics ; Hemophilia A/therapy ; Hemophilia B/genetics ; Hemophilia B/therapy ; Humans
Chemical Substances	Factor VIII (9001-27-8) ; Factor IX (9001-28-9)
Language	English
Publishing date	2021-11-23
Publishing country	United States
Document type	Journal Article ; Review
ZDB-ID	80069-7
ISSN	1528-0020 ; 0006-4971
ISSN (online)	1528-0020
ISSN	0006-4971
DOI	10.1182/blood.2019003777
Database	MEDical Literature Analysis and Retrieval System OnLINE

In stock of ZB MED Cologne/Königswinter

Uh III Zs.140: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 2021: Bestellungen von Artikeln über das Online-Bestellformular ab Jg. 2022: Lesesaal (EG)
Zs.MO 364: Show issues

Order via subito

Details ▾
- See ZB MED holdings
- Order with fees

Article ; Online: A prothrombotic von Willebrand factor variant.

Leebeek, Frank W G

Blood

2019 Volume 133, Issue 4, Page(s) 288–289

MeSH term(s)	Blood Platelets ; Factor VIII ; Humans ; Integrins ; von Willebrand Diseases ; von Willebrand Factor
Chemical Substances	Integrins ; von Willebrand Factor ; Factor VIII (9001-27-8)
Language	English
Publishing date	2019-01-21
Publishing country	United States
Document type	Journal Article ; Comment
ZDB-ID	80069-7
ISSN	1528-0020 ; 0006-4971
ISSN (online)	1528-0020
ISSN	0006-4971
DOI	10.1182/blood-2018-11-883488
Database	MEDical Literature Analysis and Retrieval System OnLINE

In stock of ZB MED Cologne/Königswinter

Uh III Zs.140: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 2021: Bestellungen von Artikeln über das Online-Bestellformular ab Jg. 2022: Lesesaal (EG)
Zs.MO 364: Show issues

Order via subito

Details ▾
- See ZB MED holdings
- Order with fees

Article ; Online: The state of oral health in patients with haemophilia in the Netherlands.

Mulders, Greta / van Verseveld, Hanneke / van der Geer, Joyce / Wolvius, Eppo / Leebeek, Frank

Haemophilia : the official journal of the World Federation of Hemophilia

2023 Volume 29, Issue 2, Page(s) 466–478

Abstract: Introduction: Previous studies show contradictory outcomes regarding dental, gingival and periodontal status in persons with haemophilia (PWH) compared to healthy controls. PWH may experience disease-specific barriers to access dental care due to their ... ...

Abstract	Introduction: Previous studies show contradictory outcomes regarding dental, gingival and periodontal status in persons with haemophilia (PWH) compared to healthy controls. PWH may experience disease-specific barriers to access dental care due to their bleeding tendency, which may lead to delays in oral care and severe dental problems. Aim: To determine the current subjective and objective oral health status in adult PWH. Methods: Randomly selected PWH of the Erasmus MC Haemophilia Treatment Center (HTC), Rotterdam, the Netherlands, were invited to participate. Data was collected using the Oral Health Impact Profile (OHIP-14NL) and personal interviews. A dentist used the DMFT index, the Dutch Periodontal Screening index (DPSI), plaque and bleeding index to score the dental status. Results: Forty-eight adult PWH were included in this study, 20 mild, 15 moderate-severe and 13 severe haemophilia with a mean age of 44.7. PWH scored low on the OHIP-14 questionnaire (median total score 1.0; IQR .0-3.0), indicating a high self-rating oral health status. The number of bleeding events, bleeding- and plaque index score was not statistically significant between patients with mild, moderate or severe haemophilia. The mean number of decayed, missing, and filled teeth (DMFT-score) was significantly lower in the group of patients with severe haemophilia (median 2.0) compared to mild haemophilia (median 16.0) (p = .04). Twenty-five patients (52.1%) reported to have encountered bleeding problems during or after dental interventions during their lifetime. Conclusion: Dutch adult PWH A/B have good dental status and oral health status.
MeSH term(s)	Adult ; Humans ; Hemophilia A/complications ; Oral Health ; Netherlands/epidemiology ; Hemophilia B ; Surveys and Questionnaires
Language	English
Publishing date	2023-01-10
Publishing country	England
Document type	Journal Article
ZDB-ID	1229713-6
ISSN	1365-2516 ; 1351-8216 ; 1355-0691
ISSN (online)	1365-2516
ISSN	1351-8216 ; 1355-0691
DOI	10.1111/hae.14719
Database	MEDical Literature Analysis and Retrieval System OnLINE

In stock of ZB MED Cologne/Königswinter

Zs.A 4249: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular Jg. 1995 - 2021: Lesesall (2.OG) ab Jg. 2022: Lesesaal (EG)
Zs.MO 450: Show issues

Order via subito

Details ▾
- See ZB MED holdings
- Order with fees

Article ; Online: How I manage severe von Willebrand disease.

Leebeek, Frank W G / Atiq, Ferdows

British journal of haematology

2019 Volume 187, Issue 4, Page(s) 418–430

Abstract: Von Willebrand disease (VWD) is the most common inherited bleeding disorder. Most patients with mild and moderate VWD can be treated effectively with desmopressin. The management of severe VWD patients, mostly affected by type 2 and type 3 disease, can ... ...

Abstract	Von Willebrand disease (VWD) is the most common inherited bleeding disorder. Most patients with mild and moderate VWD can be treated effectively with desmopressin. The management of severe VWD patients, mostly affected by type 2 and type 3 disease, can be challenging. In this article we review the current diagnosis and treatment of severe VWD patients. We will also discuss the management of severe VWD patients in specific situations, such as pregnancy, delivery, patients developing alloantibodies against von Willebrand factor and VWD patients with recurrent gastrointestinal bleeding. Moreover, we review emerging treatments that may be applied in future management of patients with severe VWD.
MeSH term(s)	Disease Management ; Female ; Forecasting ; Humans ; Male ; Pregnancy ; von Willebrand Disease, Type 2/therapy ; von Willebrand Disease, Type 3/surgery ; von Willebrand Diseases/therapy
Language	English
Publishing date	2019-09-09
Publishing country	England
Document type	Journal Article ; Review
ZDB-ID	80077-6
ISSN	1365-2141 ; 0007-1048
ISSN (online)	1365-2141
ISSN	0007-1048
DOI	10.1111/bjh.16186
Database	MEDical Literature Analysis and Retrieval System OnLINE

Full text online

Accessible to users with ZB MED library card

In stock of ZB MED Cologne/Königswinter

Uh III Zs.182: Show issues

Location:
Je nach Verfügbarkeit (siehe Angabe bei Bestand)
bis Jg. 2021: Bestellungen von Artikeln über das Online-Bestellformular
ab Jg. 2022: Lesesaal (EG)

Order via subito

Details ▾

Article ; Online: Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE-B trial 2 years after gene therapy.

Itzler, Robbin / Buckner, Tyler W / Leebeek, Frank W G / Miller, Joel / Recht, Michael / Drelich, Douglass / Monahan, Paul E / Pipe, Steven W

Haemophilia : the official journal of the World Federation of Hemophilia

2024

Abstract: Introduction: For people with haemophilia B (PwHB), bleeding may occur despite prophylaxis, negatively affecting health-related quality of life (HRQoL). The pivotal phase 3 HOPE-B trial investigating the adeno-associated virus gene transfer product, ... ...

Abstract	Introduction: For people with haemophilia B (PwHB), bleeding may occur despite prophylaxis, negatively affecting health-related quality of life (HRQoL). The pivotal phase 3 HOPE-B trial investigating the adeno-associated virus gene transfer product, etranacogene dezaparvovec (EDZ), demonstrated sustained factor IX (FIX) activity and bleed protection in PwHB with baseline FIX levels ≤2%. Aim: Assess how EDZ affects HRQoL in HOPE-B trial participants. Methods: HRQoL was evaluated using generic and disease-specific patient reported outcomes (PROs) including the EQ-5D-5L and the Hem-A-QoL questionnaires. Mean domain and total scores were compared 6 months pre- and the first 2 years post-EDZ administration using repeated measures linear mixed models. The percentage of participants with minimal clinically important improvements in HRQoL was also evaluated. Results: Two years post-EDZ, there were nominally significant increases in the least squares (LS) mean score for the EQ-5D-5L Index Value (.04; p = .0129), reflecting better HRQoL. Nominally significant decreases in the LS mean scores, reflecting better HRQoL, were also found for the Hem-A-QoL total score (-6.0; p < .0001) and the Treatment (-13.94; p < .0001), Feelings (-9.01; p < .0001), Future (-6.45; p = .0004) and Work/School (-5.21; p = .0098) domains. The percentage of participants with ≥15-point improvement ranged from 45.83% (95% CI: 31.37%, 60.83%) for Treatment to 13.89% (95% CI: 4.67%, 29.50%) for Family Planning. Results were similar for Year 1. Conclusion: In conclusion, gene therapy with EDZ improved HRQoL in the first and second years in several Hem-A-QoL domains, including Treatment, Feelings, Work/School and Future domains, whereas improvement in other aspects of HRQoL were not demonstrated.
Language	English
Publishing date	2024-03-10
Publishing country	England
Document type	Journal Article
ZDB-ID	1229713-6
ISSN	1365-2516 ; 1351-8216 ; 1355-0691
ISSN (online)	1365-2516
ISSN	1351-8216 ; 1355-0691
DOI	10.1111/hae.14977
Database	MEDical Literature Analysis and Retrieval System OnLINE

In stock of ZB MED Cologne/Königswinter

Zs.A 4249: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular Jg. 1995 - 2021: Lesesall (2.OG) ab Jg. 2022: Lesesaal (EG)
Zs.MO 450: Show issues

Order via subito

Details ▾
- See ZB MED holdings
- Order with fees

Article ; Online: Update of thrombosis in multiple myeloma.

Leebeek, Frank W G

Thrombosis research

2016 Volume 140 Suppl 1, Page(s) S76–80

Abstract: With the introduction of thalidomide and multi-agent chemotherapy in the treatment of multiple myeloma around 15years ago a strongly increased risk of venous thrombosis was observed. The occurrence of venous thrombosis in multiple myeloma is not only ... ...

Abstract	With the introduction of thalidomide and multi-agent chemotherapy in the treatment of multiple myeloma around 15years ago a strongly increased risk of venous thrombosis was observed. The occurrence of venous thrombosis in multiple myeloma is not only determined by the kind of treatment, but also by several other factors, including disease specific factors, patient-specific factors, changes in pro-and anticoagulant factors and fibrinolysis. Studies showed a prevalence of up to 25% in patients with newly diagnosed multiple myeloma. Therefore these patients nowadays receive prophylaxis with aspirin, low molecular weight heparin or warfarin in order to reduce the risk of venous thrombosis. It is however still debatable which patients should receive prophylaxis and what the best kind of prophylaxis is, considering both the risk of thrombosis and the risk of bleeding. In recent years several new anti-myeloma agents have been developed and investigated in large clinical studies. The risk of thrombosis using these new drugs seems less than with thalidomide and lenalidomide-based regimens. In this article an update on prevention and management of thrombotic events in patients with multiple myeloma is given.
MeSH term(s)	Anticoagulants/therapeutic use ; Antineoplastic Agents/adverse effects ; Antineoplastic Agents/therapeutic use ; Aspirin/therapeutic use ; Heparin, Low-Molecular-Weight/therapeutic use ; Humans ; Multiple Myeloma/complications ; Multiple Myeloma/drug therapy ; Thalidomide/adverse effects ; Thalidomide/analogs & derivatives ; Thalidomide/therapeutic use ; Thrombosis/chemically induced ; Thrombosis/complications ; Thrombosis/drug therapy ; Thrombosis/prevention & control ; Warfarin/therapeutic use
Chemical Substances	Anticoagulants ; Antineoplastic Agents ; Heparin, Low-Molecular-Weight ; Thalidomide (4Z8R6ORS6L) ; Warfarin (5Q7ZVV76EI) ; lenalidomide (F0P408N6V4) ; Aspirin (R16CO5Y76E)
Language	English
Publishing date	2016-04
Publishing country	United States
Document type	Journal Article ; Review
ZDB-ID	121852-9
ISSN	1879-2472 ; 0049-3848
ISSN (online)	1879-2472
ISSN	0049-3848
DOI	10.1016/S0049-3848(16)30103-7
Database	MEDical Literature Analysis and Retrieval System OnLINE

In stock of ZB MED Cologne/Königswinter

Zs.A 863: Show issues

Location:
Je nach Verfügbarkeit (siehe Angabe bei Bestand)
bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular
Jg. 1995 - 2021: Lesesall (1.OG)
ab Jg. 2022: Lesesaal (EG)

Order via subito

Details ▾
- See ZB MED holdings
- Order with fees

To top

More links

Kategorien

Order via subito

Inter-library loan at ZB MED

More links

Kategorien

In stock of ZB MED Cologne/Königswinter

Order via subito

More links

Kategorien

In stock of ZB MED Cologne/Königswinter

Order via subito

More links

Kategorien

Order via subito

Inter-library loan at ZB MED

More links

Kategorien

In stock of ZB MED Cologne/Königswinter

Order via subito

More links

Kategorien

In stock of ZB MED Cologne/Königswinter

Order via subito

More links

Kategorien

In stock of ZB MED Cologne/Königswinter

Order via subito

Full text online

More links

Kategorien

In stock of ZB MED Cologne/Königswinter

Order via subito

More links

Kategorien

In stock of ZB MED Cologne/Königswinter

Order via subito

More links

Kategorien

In stock of ZB MED Cologne/Königswinter

Order via subito