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  • 1
    In: Thrombosis and Haemostasis, Georg Thieme Verlag KG, Vol. 105, No. 04 ( 2011), p. 597-604
    Abstract: In patients with von Willebrand disease (VWD) replacement therapy with factor VIII/von Willebrand (VWF) concentrates is increasingly applied as prophylactic regimen. Since 2000, 82 consecutively enrolled patients with clinically relevant bleeding episodes (spontaneous, peri- or postoperative) were diagnosed with VWD [type 1: 42/82; type 2: 24/82; type 3: 13/82; acquired: 3/82]. In all patients, decision for initiating prophylaxis was based on a bleeding score 〉 2 prior to diagnosis, concomitant with recurrent bleeds associated with anaemia in patients with on-demand VWD therapy. We report results on secondary prophylactic VWF replacement therapy applied in 32 patients [children n=13; adolescents n=7; adults n=12] with VWD [type 1: 4; type 2: 15; type 3: 13] , 15 of which were females, and nine of these at the reproductive period. Eight patients were treated with Humate P→ or Wilate→ (n=24). Median [min-max] dose [vWF:RCo] was 40 [20–47] IU/kg, 23 patients were given substitution therapy twice weekly, seven patients three times a week, and two children four times per week. Within a 12-month-period haemoglobin concentrations returned to normal values. Median duration of prophylaxis was three years. Recurrent bleeding episodes stopped in 31 of 32 patients, whereas inhibitors developed in one. Following a 12-month observation period the monthly bleeding frequency and the bleeding score was significantly reduced [3 vs. 0.07; 3 vs. 0: p 〈 0.001], compared to the preprophylaxis/pre-diagnostic values. The use of secondary prophylactic VWF replacement therapy is an effective tolerated treatment modality, highly beneficial for patients with VWD, who present with recurrent bleeding events during on-demand therapy.
    Type of Medium: Online Resource
    ISSN: 0340-6245 , 2567-689X
    Language: English
    Publisher: Georg Thieme Verlag KG
    Publication Date: 2011
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  • 2
    In: European Journal of Haematology, Wiley, Vol. 98, No. S85 ( 2017-01), p. 1-15
    Abstract: The first Team Haemophilia Education ( THE ) Meeting was held on 7–8 May 2015 in Amsterdam, The Netherlands. It aimed to promote the optimal care of patients with haemophilia through education of the multidisciplinary treatment team. This was achieved by reviewing the latest developments in haemophilia management, considering how these can be implemented in the clinic to improve patient care and providing a platform for networking and debate for all haemophilia treatment team members. The second THE Meeting was held on 19–20 May in Frankfurt, Germany, and participants included doctors, nurses, physiotherapists, patient representatives and data management staff from 20 different countries. Topics covered the role of the multidisciplinary team in delivering the best haemophilia care, challenges in the management of haemophilia across Europe, available clotting factor treatments, future treatments and the use of genetics in advising carriers of haemophilia. This report is a summary of the key developments in haemophilia care presented by various investigators and healthcare professionals at THE Meeting 2016.
    Type of Medium: Online Resource
    ISSN: 0902-4441 , 1600-0609
    URL: Issue
    Language: English
    Publisher: Wiley
    Publication Date: 2017
    detail.hit.zdb_id: 2027114-1
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  • 3
    Online Resource
    Online Resource
    Elsevier BV ; 2015
    In:  Thrombosis Research Vol. 135 ( 2015-02), p. S34-S37
    In: Thrombosis Research, Elsevier BV, Vol. 135 ( 2015-02), p. S34-S37
    Type of Medium: Online Resource
    ISSN: 0049-3848
    Language: English
    Publisher: Elsevier BV
    Publication Date: 2015
    detail.hit.zdb_id: 1500780-7
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  • 4
    Online Resource
    Online Resource
    Georg Thieme Verlag KG ; 2007
    In:  Thrombosis and Haemostasis Vol. 98, No. 10 ( 2007), p. 912-915
    In: Thrombosis and Haemostasis, Georg Thieme Verlag KG, Vol. 98, No. 10 ( 2007), p. 912-915
    Type of Medium: Online Resource
    ISSN: 0340-6245 , 2567-689X
    Language: English
    Publisher: Georg Thieme Verlag KG
    Publication Date: 2007
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  • 5
    Online Resource
    Online Resource
    Georg Thieme Verlag KG ; 2022
    In:  Hämostaseologie Vol. 42, No. 02 ( 2022-04), p. 123-130
    In: Hämostaseologie, Georg Thieme Verlag KG, Vol. 42, No. 02 ( 2022-04), p. 123-130
    Abstract: Thrombotic events are an increasing challenge in pediatrics. Standard-of-care anticoagulants for pediatric thrombosis have several disadvantages which could be overcome by using direct oral anticoagulants (DOACs). Until recently, there was not enough evidence from clinical trials to recommend for or against the use of any of the four DOACs in children with thrombosis. In this literature review, we looked at the latest clinical trials in this field. On clinicaltrials.gov, we found 13 current studies with published results. For two of the four DOACs, namely dabigatran and rivaroxaban, we found successful phase III studies which led to the approval for the use in children. The results of these pivotal phase III studies allow to finally recommend rivaroxaban and dabigatran for the prophylaxis and treatment of thrombotic events in children.
    Type of Medium: Online Resource
    ISSN: 0720-9355 , 2567-5761
    Language: English
    Publisher: Georg Thieme Verlag KG
    Publication Date: 2022
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  • 6
    Online Resource
    Online Resource
    OMICS Publishing Group ; 2015
    In:  Health Care : Current Reviews Vol. 02, No. 03 ( 2015)
    In: Health Care : Current Reviews, OMICS Publishing Group, Vol. 02, No. 03 ( 2015)
    Type of Medium: Online Resource
    ISSN: 2375-4273
    Language: Unknown
    Publisher: OMICS Publishing Group
    Publication Date: 2015
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  • 7
    In: BioMed Research International, Hindawi Limited, Vol. 2013 ( 2013), p. 1-7
    Abstract: Among the discussed risk factors for high-titre inhibitor (HRI) development in patients with hemophilia A (HA) are high dose FVIII replacement therapy and use of recombinant FVIII concentrates (rFVIII). The aim of this study was to evaluate the aforementioned risk factors for HRI development in children with hemophilia A ≤2%. About 288 ascertained PUPs (Israel and Germany) were followed after initial HA diagnosis over 200 exposure days. Inhibitor-free survival, hazard ratios (HR), and 95% confidence intervals (CIs) were calculated. Adjustment was performed for factor VIII concentrates, median single dose over the first three months of treatment, first FVIII administration before the age of three months, presence of risk HA gene mutations, “intensive treatment moments” and “year of birth” (proxy for different treatment periods). HRI occurred in 71/288 children (24.7%). In multivariate analysis adjusted for “year of birth”, underlying risk gene mutations (HR/CI: 2.37/1.40–3.99), FVIII dose, measured per one IU increase per kgbw (HR/CI: 1.05/1.04–1.07), and first FVIII administration before the age of three months showed a significant impact on HR development. The risk of HRI development was similar for recombinant or plasmatic FVIII products. Children at risk should be treated with carefully calculated lower dose regimens, adapted to individual bleeding situations.
    Type of Medium: Online Resource
    ISSN: 2314-6133 , 2314-6141
    Language: English
    Publisher: Hindawi Limited
    Publication Date: 2013
    detail.hit.zdb_id: 2698540-8
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  • 8
    In: Blood, American Society of Hematology, Vol. 138, No. Supplement 1 ( 2021-11-05), p. 1043-1043
    Abstract: Background: Heavy menstrual bleeding (HMB) is a common gynaecological problem and is the reason for 18-30% of gynaecological visits. In a European study in 4,502 women, 27.2% were diagnosed with HMB (Fraser et al., 2015). On the other hand, HMB is often associated with bleeding disorders (Shankar et al., 2004). The definition of HMB has different perspectives; from a subjective perspective HMB is defined as e excessive menstrual blood loss impacting on women's physical, social, emotional and/or mental quality of life, whereas from an objective perspective it is defined as excessive blood loss & gt;80 ml per cycle (Munro et al., 2012). There are different approaches for treating HMB such as the administration of non-steroidal drugs, Desmopressin, herbal Vitex Agnus Castus (VAC), Tranexamic acid (TXA) or a hormonal therapy; moreover, HMB can also be treated surgically. The aim of this study was to test the efficacy of the specific HMB management used in our center: Women with HMB are treated usually with VAC, which optimizes the relation of estrogen to progesterone in the female body (Yavarikia et al., 2013). VAC can be used without using any hormones (Shahnazi et al, 2016), providing a treatment with a low impact on women's bodies. Additionally, TXA is given during the menstruation due to its antifibrinolytic effect. Furthermore, the "Pictural Blood-Loss-Assessment- Chart" (PBAC Score) is administered to analyze the menstrual blood loss. Methods: Two ad hoc patient-reported questionnaires were developed to test the efficacy of the HMB management in our center The baseline questionnaire includes the following aspects: menarche, duration, regularity, number of sanitary products, pain level, medical background and family history. During the first visit in our center women with HMB filled in the baseline questionnaire. In addition, laboratory tests are done including a comprehensive coagulation test and the examination of iron and hemoglobin levels. Moreover, we presented an application called "My Flow Score" to the patients, which calculates the PBAC score as a result of all entered sanitary products women use during their period. Women are prescribed VAC and/or TXA for the management of their HMB. After four months patients are scheduled for a follow up appointment. At that time they complete the follow-up questionnaire including the following aspects: compliance with the medication or the reason for the non-compliance, health complaints due to the medication, duration of the period, improvement, pain level, PBAC score if the patient did use the APP "My Flow Score" or if not the number of sanitary products. A blood test was taken and the HMB management was adapted to the patients' needs. Results: So far, 100 women with HMB with a median age of 14 years (range 9-50) were enrolled in our study. They had their menarche with a median age of 12 years (range 8-17). Diagnosis of women ranged from iron or folic acid deficiency to different forms of bleeding disorders; with the majority suffering from von Willebrand disease (43/100) followed by iron deficiency (14/100). One fifth of the patients had more than one diagnosis. 1/3 of patients received a treatment for the HMB previously, mainly contraceptives, TXA or VAC. 49% reported anomalies with regard to previous other bleeding; 25.8% had increased hematomas and 19.6% recurrent epistaxis. Most of them reported anomalies already in the family (60.2%). Before treatment women had a median PBAC Score of 169 (range 77-800) and reported a median pain level of 6.5 during menstruation on a scale ranging from 1 (low pain) to 10 (extreme pain). 87/100 women received VCA, of these 75.4% regularly and 85/100 women received TXA, of these 84.6% regularly. 81% of them received a combination of both medicines. Compared to before 28.8% of patients reported shorter duration of menstruation and reduced bleeding with treatment, 11.9% did not experience any improvement; the PBAC score decreased significantly (p & lt;.0001). Conclusions: Although the type of diagnosis in women suffering from HMB had a great variation, these patients had a high disease burden with a relatively high level of pain and a high loss of blood assessed with the PBAC score. Thanks to a combination of VAC and TXA the disease burden in these women could be reduced, especially for the time of menstruation and amount of bleeding. VAC proved to be highly accepted by women due to its low treatment burden. Disclosures Von Mackensen: University Medical Centre Hamburg-Eppendorf: Current Employment; Sobi: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Sanofi: Consultancy; Novo Nordisk: Consultancy; Biomarin: Speakers Bureau; CSL Behring: Speakers Bureau; Chugai/Roche: Membership on an entity's Board of Directors or advisory committees.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    RVK:
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    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2021
    detail.hit.zdb_id: 1468538-3
    detail.hit.zdb_id: 80069-7
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  • 9
    In: Blood, American Society of Hematology, Vol. 122, No. 21 ( 2013-11-15), p. 4773-4773
    Abstract: Hereditary bleeding disorders (HBD) affect children from birth during their life course. Medical treatment and the overall prognosis of children with HBD have significantly progressed. But even in patients cared for in specialized treatment centers it remains unclear whether survival till adulthood goes along with health-related quality of life [Hr-QoL] comparable to other patients groups respectively comparable to population children. Therefore aim of the present study was to assess self- as well as parents/proxies reported Hr-QoL in children and adolescents with HBD and to compare the results with findings from children affected by another chronic medical condition respectively with healthy siblings and peers. Methods 91 patients with HBD (thrombosis / deep venous thrombosis, DVT, respectively Haemophilia A and B) aged 8 to 16 years from 6 Hemophilia study centers were investigated regarding Hr-QoL. Hr-QoL was assessed in patients and parents/proxies with the generic KINDL-R questionnaire exploring overall well-being and 6 sub-dimensions (physical well-being, psychological well-being, self-esteem, family-related well-being, friend-related well-being, and school-related well-being). Findings were compared with 70 children with stroke / TIA respectively with healthy controls (45 healthy siblings and 106 healthy peers). Results Overall well-being in children with thrombosis / DVT (77.6 ± 9.7 points) was comparable to healthy controls but in children with Haemophilia A or B (76.0 ± 9.6) lower compared with healthy peers (80.2 ± 9.7, p = 0.005). No differences occurred between healthy controls and children with stroke/TIA (74.2 ± 10.3). In children with HBD none of the KINDL-R sub-dimensions showed values below healthy siblings. Two KINDL-R sub-dimensions showed lower values compared with healthy peers. Parents/proxies rated the Hr-QoL of their children with HBD similar to their healthy children. The internal consistency of overall well-being in all study subgroups was acceptable ( 〉 0.7) but showed poor results ( 〈 0.5) in two KINDL-R sub-dimensions. Discussion The application of a generic Hr-QoL questionnaire in a sample of children and adults with HBD seems feasible. Their Hr-QoL seems comparable to their siblings but below their healthy peers. Disclosures: Halimeh: Octapharma AG: Investigator Other, Research Funding.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    RVK:
    RVK:
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2013
    detail.hit.zdb_id: 1468538-3
    detail.hit.zdb_id: 80069-7
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  • 10
    Online Resource
    Online Resource
    American Society of Hematology ; 2014
    In:  Blood Vol. 124, No. 21 ( 2014-12-06), p. 5068-5068
    In: Blood, American Society of Hematology, Vol. 124, No. 21 ( 2014-12-06), p. 5068-5068
    Abstract: Introduction: It is well know that most of the coagulation factor increase during pregnancy in healthy women. Nevertheless the uninterrupted course of coagulation parameters from the beginning until the end of a pregnancy in healthy women has not been described yet. Only reference ranges for the third month and the sixth month of pregnancy are evaluated. There aren't any data available for the course of coagulation parameters during pregnancy in women with known coagulation disorders. In 2012 we started a study to investigate reference ranges during pregnancy for all coagulation factors, anticoagulants and activation markers of coagulation in 100 healthy pregnant women and 100 pregnant women with a previously known mild bleeding disorders. The study has been approved by the Ethics Committee Nordrhein. Samples and Methods: We analysed samples of pregnant women by conducting the following tests: Blood count, VWF:RCo, VWF:Ag, VWF:CB, Fibrinogen (Clauss), activities of FII, FV, FVII, FVIII (clotting and chromogenic), FIX, FX, FXI, FXII, FXIII. d-dimer, prothrombin fragment 1.2, Quick, partial thromboplastin time, plasma thrombin time , CRP, proteine S, proteine C, antithrombin, Lupus antigoaculant, ACA, ß2-GP in week 10, 16, 22, 28, 34, 40 and 6 weeks post partum (max. +/- 1) Interim Results: Currently 21 pregnant women were included in our study. 16 obviously healthy women were used to calculate the reference ranges for pregnancy. Because of strict inclusion and exclusion criteria (no previous spontaneous abortion, no previous placenta haematoma, no previous pre-eclampsia and only natural pregnancies) most of the women are in the group of the no known coagulation disorder so far. Discussion/Conclusion: There are signs that defects in the coagulation system can be associated to complications during pregnancy like child loss, intrauterine haematoma and genital bleeding. The evaluation of reference ranges helps to detect and to value coagulation disorders during pregnancy. It might be possible to explain the higher abortion rate in women with mild bleeding disorders by determination of reference values of all pro- and anticoagulants during pregnancy. If a treatment with coagulation factor concentrates can help to prevent miscarriage is still subject of ongoing studies. Determination of reference ranges for coagulation factors in the third trimenon might help to determine the peripartum bleeding risk of women with mild coagulation disorders and to help to decide whether a women needs coagulation factor concentrate during labour. Limitation: Based on the strict inclusion and exclusion criteria the number of patients is small. Disclosures Halimeh: Biotest: The authors declare that they receive research grant from Biotest AG Other. Rott:Biotest: The authors declare that they receive research grant from Biotest AG Other. Osseiran:Biotest: The authors declare that they receive research grant from Biotest AG Other. Kappert:Biotest: The authors declare that they receive research grant from Biotest AG Other. Siebert:Biotest: The authors declare that they receive research grant from Biotest AG Other.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
    RVK:
    RVK:
    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2014
    detail.hit.zdb_id: 1468538-3
    detail.hit.zdb_id: 80069-7
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