Haemophilia : the official journal of the World Federation of Hemophilia
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Desmopressin (l-deamino-8-D-arginine vasopressin, DDAVP) is a synthetic analogue of the antidiuretic hormone vasopressin. Like the natural antidiuretic hormone, desmopressin increases the plasma levels of factor VIII and von Willebrand factor (vWF), with the advantage, compared to vasopressin, that it produces little or no vasoconstriction, no increase in blood pressure, and no contraction of the uterus or gastrointestinal tract, so that it is well tolerated when administered to humans. ⋯ The compound was shown to be efficacious even in bleeding disorders not involving a deficiency or dysfunction of factor VIII or vWF, including congenital and acquired defects of platelet function and such frequent abnormalities of hemostasis as those associated with chronic kidney and liver diseases. Desmopressin has also been used prophylactically in patients undergoing surgical operations characterized by large blood loss and transfusion requirements.
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The haemostatic system has a central role in controlling the amount and the duration of menstrual bleeding, thus abnormally prolonged or profuse bleeding does occur in most women affected by inherited bleeding disorders. Whereas irregular, premenarchal or postmenopausal uterine bleeding is unusual in inherited or acquired heamorrhagic disorders, severe acute bleeding and menorrhagia at menarche and chronic menorrhagia during the entire reproductive life are common manifestations. Prevalence and morbidity of menorrhagia in inherited bleeding disorders have been poorly investigated. ⋯ In case of failure or contraindication (COC and LNG-IUS are best avoided in adolescents), before considering surgical options, treatment with desmopressin becomes the preferred choice in patients known to be responsive. The availability of desmopressin preparations for self-administration makes home treatment feasible in well selected cases. The treatment is efficacious and safe provided that patients are instructed to self-administer the agent only during the first two or three more heavy days of menstrual period, for a maximum of three to four doses and no more than two consecutive administrations at a 12-h interval.
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The aim of the study was to review the complications, management and outcome of pregnancy in carriers of haemophilia over a 10-year period following the introduction of a multidisciplinary management guideline. Comparison was made to a 10-year cohort prior to implementation of the guidelines. A retrospective review of case notes of carriers of haemophilia (41 haemophilia A, 12 haemophilia B) who had received obstetric care at the Royal Free Hospital between 1995 and 2005 was conducted. ⋯ The incidence of primary and secondary postpartum haemorrhage was 19% and 2%, respectively. There were two neonatal head bleeding complications associated with prolonged labour or instrumental delivery. Availability of management guideline and care provided in a multidisciplinary approach can help to minimize bleeding complications in carriers of haemophilia and their newborns.
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Glanzmann thrombasthenia (GT) is a rare platelet function disorder characterized by a defect in fibrinogen binding to platelet membrane glycoprotein (GP) IIb/IIIa. Recombinant FVIIa (rFVIIa) is a haemostatic agent approved for the treatment of haemophilia patients with inhibitors, patients with acquired haemophilia and in EU also for treatment of factor VII (FVII)-deficient patients and GT patients with antibodies to GPIIb-IIIa. The present study was conducted to evaluate the use of the whole blood test system, rotational thrombelastometry (ROTEM), in measuring the overall haemostasis potential of rFVIIa in 28 GT patients treated with rFVIIa. ⋯ Both ex vivo and in vivo data showed beneficial effects on CT in the presence of rFVIIa, but no effect of added rFVIIa was seen on CFT and MCF. In conclusion, the use of thrombelastography at least in the modified form of ROTEM seems to be of limited use in predicting an adequate dose of rFVIIa in GT patients. A good clinical haemostatic response was recorded in spite of the limited changes in the ROTEM pattern supporting the conclusion that ROTEM should not be the method of choice for monitoring rFVIIa therapy in Glanzmann patients.
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Factor VIII (FVIII) inhibitors remain a serious complication of treatment for patients with haemophilia A. Immune tolerance induction (ITI) can eliminate inhibitors in the majority of patients, but there are major concerns related with this therapy. Investigators have raised the possibility that the use of FVIII/von Willebrand factor (FVIII/VWF) concentrates may improve the success rate of ITI and may shorten the duration of therapy necessary to attain tolerance. ⋯ Eight percent of patients failed to attain either partial or complete tolerization and discontinued ITI. Another 24% are continuing with ITI but have titres of >10 BU. This study adds further retrospective data to the information regarding the use of FVIII/VWF concentrate in ITI.