57 research outputs found
Desmopressin in moderate hemophilia a patients: A treatment worth considering
Desmopressin increases endogenous factor VIII levels in hemophilia A. Large inter-individual variation in the response to desmopressin is observed. Patients with a lower baseline factor VIII activity tend to show a reduced response, therefore, desmopressin is less frequently used in moderate hemophilia A patients (baseline factor VIII activity 1-5 international units/deciliter), even though factor VIII levels may rise substantially in some of them. We aim to describe the response to desmopressin in moderate hemophilia A patients and to identify predictors. We selected data on 169 patients with moderate hemophilia from the multicenter Response to DDAVP In non-severe hemophilia A patients: in Search for dEterminants (RISE) cohort study. Adequate response to desmopressin was defined as a peak factor VIII level ≥ 30, and excellent response as ≥ 50 international units/deciliter after desmopressin administration. We used univariate and multiple linear regression techniques to analyze predictors of the peak factor VIII level. Response was considered adequate in 68 patients (40%), of whom 25 showed excellent response (15%). Intravenous administration, age, pre-desmopressin factor VIII activity and von Willebrand factor antigen, peak von Willebrand factor activity and desmopressin-induced rise in von Willebrand factor antigen were significant predictors of peak factor VIII level and explained 65% of the inter-individual variation. In 40% of moderate hemophilia A patients, desmopressin response was adequate, thus it is important not to withhold this group of patients from desmopressin responsiveness. Among the six predictors that we identified for desmopressin-induced factor VIII rise, factor VIII activity and desmopressin-induced rise in von Willebrand factor antigen had the strongest effect
Replacement therapy for bleeding episodes in factor VII deficiency: A prospective evaluation
Patients with inherited factor VII (FVII) deficiency display different clinical phenotypes requiring ad hoc management. This study evaluated treatments for spontaneous and traumatic bleeding using data from the Seven Treatment Evaluation Registry (STER). One-hundred one bleeds were analysed in 75 patients (41 females; FVII coagulant activity <1-20%). Bleeds were grouped as haemarthroses (n=30), muscle/subcutaneous haematomas (n=16), epistaxis (n=12), gum bleeding (n=13), menorrhagia (n=16), central nervous system (CNS; n=9), gastrointestinal (GI; n=2) and other (n=3). Of 93 evaluable episodes, 76 were treated with recombinant, activated FVII (rFVIIa), eight with fresh frozen plasma (FFP), seven with plasma-derived FVII (pdFVII) and two with prothrombin-complex concentrates. One-day replacement therapy resulted in very favourable outcomes in haemarthroses, and was successful in muscle/subcutaneous haematomas, epistaxis and gum bleeding. For menorrhagia, single- or multiple-dose schedules led to favourable outcomes. No thrombosis occurred; two inhibitors were detected in two repeatedly treated patients (one post-rFVIIa, one post-pdFVII). In FVII deficiency, most bleeds were successfully treated with single 'intermediate' doses (median 60 µg/kg) of rFVIIa. For the most severe bleeds (CNS, GI) short- or long-term prophylaxis may be optimalPatients with inherited factor VII (FVII) deficiency display different clinical phenotypes requiring ad hoc management. This study evaluated treatments for spontaneous and traumatic bleeding using data from the Seven Treatment Evaluation Registry (STER). One-hundred one bleeds were analysed in 75 patients (41 females; FVII coagulant activity <1-20%). Bleeds were grouped as haemarthroses (n=30), muscle/subcutaneous haematomas (n=16), epistaxis (n=12), gum bleeding (n=13), menorrhagia (n=16), central nervous system (CNS; n=9), gastrointestinal (GI; n=2) and other (n=3). Of 93 evaluable episodes, 76 were treated with recombinant, activated FVII (rFVIIa), eight with fresh frozen plasma (FFP), seven with plasma-derived FVII (pdFVII) and two with prothrombin-complex concentrates. One-day replacement therapy resulted in very favourable outcomes in haemarthroses, and was successful in muscle/subcutaneous haematomas, epistaxis and gum bleeding. For menorrhagia, single- or multiple-dose schedules led to favourable outcomes. No thrombosis occurred; two inhibitors were detected in two repeatedly treated patients (one postrFVIIa, one post-pdFVII). In FVII deficiency, most bleeds were successfully treated with single 'intermediate' doses (median 60 μg/kg) of rFVIIa. For the most severe bleeds (CNS, GI) short- or long-term prophylaxis may be optimal. © Schattauer 2013
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An open-label, multi-centre, post-marketing study to assess the efficacy and safety of a plasma-derived VWF/FVIII concentrate in patients with von Willebrand disease.
The factor VIII treatment history of non-severe hemophilia A
Background: In patients with non-severe hemophilia A, we lack detailed knowledge
on the timing of treatment with factor VIII (FVIII) concentrates. This knowledge could
provide information about the expected treatment timing in patients with severe hemophilia A treated with non-replacement therapies.
Objective: To assess the FVIII treatment history in patients with non-severe hemophilia A.
Methods: Patients with non-severe hemophilia (baseline FVIII activity [FVIII:C]
2-40 IU/dL) were included from the INSIGHT study. The primary outcome was median age at first FVIII exposure (ED1). In a subgroup of patients for whom more detailed information was available, we analyzed the secondary outcomes: median age at first 20 EDs, annualized bleeding rate for all bleeds (ABR), joint bleeds (AJBR), and
major spontaneous bleeds (ASmBR).
Results: In the total cohort (n = 1013), median baseline FVIII activity was 8 IU/dL (interquartile range [IQR] 4-15) and the median age at ED1 was 3.7 years (IQR 1.4-7.7). Median
age at ED1 rose from 2.5 years (IQR 1.2-5.7) in patients with FVIII:C 2-5 IU/dL to 9.7 years
(IQR 4.8-16.0) in patients with FVIII:C 25-40 IU/dL. In the subgroup (n = 104), median
age at ED1, ED5, ED10, and ED20 was 4.0 years (IQR 1.4-7.6), 5.6 years (IQR 2.9-9.3),
7.5 years (IQR 4.4-11.3), and 10.2 years (IQR 6.5-14.2), respectively. Median ABR, AJBR,
and ASmBR were 1.1 (IQR 0.5-2.6), 0.3 (IQR 0.1-0.7), and 0 (IQR 0-0), respectively.
Conclusion: This study demonstrates that in non-severe hemophilia A, the age at first
FVIII exposure increases with baseline FVIII:C and that major spontaneous bleeds
rarely occur
Diffuse splenic and visceral hemangiomas complicated by chronic consumption coagulopathy
Abstract The case of a 7-year-old girl with a 2 year history of easy bruising associated with thrombocytopenia is reported. On admission she presented with ecchymoses, abdominal distention and splenomegaly. Hemostasis investigation revealed a consumption coagulopathy. Several radiological studies failed to confirm the diagnosis of diffuse splenic and visceral hemangiomatosis, which was eventually estasblished by an explorative laparotomy. Platelet count and the other coagulation abnormalities progressively returned to normal after splenectomy, although the remaining hemangiomas were extensive. © 1998 Wiley. All rights reserved
Clinical remission following monoclonal anti-CD20 therapy in two children with chronic refractory idiopathic thrombocytopenic purpura
Two patients, a 4-year-old boy and a 6-year-old girl who had a 2-year
and a 3-year history of idiopathic thrombocytopenic purpura,
respectively, were referred to our Department. Both patients had
frequent haemorrhagic events. They received i.v. immunoglobulin,
corticosteroids, cyclosporine, interferon alpha-2b and azathioprine, but
no clinical remission was established. The girl also underwent
splenectomy. Anti-CD20 antibody was administered to both patients at a
dose of 375 mg/m(2) once weekly for 4 weeks. No side-effects were
detected. During the 18-month follow-up period the patients received no
other drug and remained in clinical remission. The B lymphocytes
remained undetectable in peripheral blood for 3 months and they
progressively increased during the following 4 months. Rituximab is a
novel, quite effective, safe treatment of chronic refractory idiopathic
thrombocytopenic purpura in childhood. More studies and follow up of
patients for longer periods are necessary
17 YEARS OF EXPERIENCE WITH CHRONIC IDIOPATHIC THROMBOCYTOPENIC PURPURA IN CHILDHOOD - IS THERAPY ALWAYS BETTER
Between 1975 and 1992 450 children with idiopathic thrombocytopenic
purpura (ITP) were diagnosed, and of those 100 (22%) developed the
chronic form of the disease. Approximately half the patients with
chronic ITP presented with mild to moderate hemorrhagic manifestations
at the onset of purpura (30 cases) and/or later during the course of the
disease (25 cases). The incidence of intracranial hemorrhage was 1%,
and the mortality rate due to overwhelming septicemia after splenectomy
was also 1%. Overall one-third of the patients received no therapy;
two-thirds of them went into spontaneous remission within 8 months to 8
years from the onset of ITP. Steroids given in conventional or high
doses (51 cases) achieved a transient (if any) rise in platelet count,
but in no case were steriods curative. Remission related to intravenous
immune globulin (IVIG) therapy was noticed in 38.5% of the children (10
of 26) after variable courses. The response rate to splenectomy was
95.0%. Ultimately the long-term outcome in children with chronic ITP
was as follows: remission, 58 cases (spontaneous, 30; after IVIG
therapy, 10; after splenectomy, 18); hemostatic platelet values, 22
cases (spontaneous, 16; after IVIG, 5; after splenectomy, 1). Thirteen
children were lost in follow-up, and 7 remain thrombocytopenic but
asymptomatic. These data indicate that chronic ITP in childhood runs a
benign course in most cases and may remit with or without therapy even
several years from onset. Therefore, therapeutic intervention has to be
individualized, and splenectomy, which is not always safe, should be
reserved for problematic cases that fail to respond to conventional
therapeutic modalities
Assessment of bone mineral density and markers of bone turnover in children under long-term oral anticoagulant therapy
Oral anticoagulants antagonize vitamin K action and potentially impair the carboxylation of osteocalcin, a protein essential for normal bone matrix formation. In the present study, bone mineral density (BMD) and bone turnover markers were evaluated in 23 children under long-term oral anticoagulant therapy. BMD of the lumbar spine was assessed (Dual Energy x-ray Absorptiometry) and reported as z score. Osteoblast [bone alkaline phosphatase, osteocalcin (Gla-Oc), amino-terminal procollagen 1 extension peptide] and osteoclast (urinary calcium and deoxypyridinoline, serum cross-linked C telopeptide) activity markers were measured. Vitamin D ([25(OH) D], parathormone, calcium, phosphorus, magnesium) and vitamin K status [factors II, VII, IX, X, protein C, protein S, undercarboxylated osteocalcin (Glu-Oc)] were determined. The above parameters were also evaluated in 25 healthy controls. Patients presented with higher levels in Glu-Oc, parathormone, and bone resorption markers, lower levels in bone formation markers and 25(OH) D, whereas 52% of them showed signs of osteopenia (-1> BMD z score > -2.5). Statistical analysis demonstrated that anticoagulant therapy was an independent predictor of alterations in Glu-Oc, Gla-Oc, bone alkaline phosphatase, amino-terminal procollagen 1 extension peptide, and serum cross-linked C telopeptide levels. It seems that long-term use of coumarin derivatives may cause osteopenia in children with the risk of developing osteoporosis later in life. © 2008 by Lippincott Williams & Wilkins
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