1/15. tympanoplasty in a patient with hemophilia b.We report the case of a 36-year-old hemophilia b who suffered from cholesteatoma and underwent tympanoplasty. Though the factor ix activity was less than 1% of normal before surgery, adequate replacement of factor ix led to the achievement of hemostasis during and after surgery. The cholesteatoma was completely extirpated with matrix, and a type I canal-up tympanoplasty was subsequently performed. Careful preoperative evaluation and close cooperation with the hematologist are required if surgery is to be successful. We also present here the use of continuous administration of monoclonal antibody-purified factor ix concentrate, Christmassin M.- - - - - - - - - - ranking = 1keywords = administration (Clic here for more details about this article) |
2/15. Management of haemophilia in patients with high-titre inhibitors: focus on the evolution of activated prothrombin complex concentrate AUTOPLEX T.Numerous therapeutic strategies have been applied to the management of patients with inhibitors to factors VIII or IX. Different treatment approaches are analysed including prothrombin complex concentrates (PCCs), activated prothrombin complex concentrates (aPCCs), porcine factor viii concentrate, inhibitor neutralization, immune tolerance therapy, immunosuppressive regimens and recombinant factor viia. Clinical data are reported in the analysis of several treatments. PCCs and aPCCs have gained widespread acceptance as the standard first-line approach for patients with inhibitors. The aPCC AUTOPLEX T has achieved a high response rate with a low level of thrombotic events. Four case studies are presented in which AUTOPLEX T has been used successfully. Administration of platelet concentrate or, in elective surgery, waiting for inhibitor levels to decline are useful adjuncts to some treatments. The optimal treatment depends on the patient's inhibitor status--low responder (minimal or no increase in inhibitor levels upon administration of replacement clotting factor) or high responder (replacement clotting factor generates inhibitor production). A suggested algorithm for treating high-responder inhibitor patients is presented.- - - - - - - - - - ranking = 1keywords = administration (Clic here for more details about this article) |
3/15. Successful induction of immune tolerance with FIX recombinant in a patient with haemophilia B with inhibitor.We describe successful induction of immune tolerance in a 12-month-old patient with severe haemophilia B and low-titre and low response antifactor IX inhibitor at 5 Bethesda Unit (BU). immune tolerance induction (ITI) was started with recombinant factor (rFIX) at 40-50 U kg(-1) day(-1) (1000 U three times per week) and 2 years after the beginning of therapy the inhibitor had been reduced to undetectable values. As complications, which appeared during the ITI, the patient presented minor haemorrhagic complications, which remitted following the administration of rFVIIa, and recurring infections of the catheter, requiring the withdrawal and subsequent placement of a new catheter.- - - - - - - - - - ranking = 1keywords = administration (Clic here for more details about this article) |
4/15. Haemophilia B Leyden arising de novo by point mutation in the putative factor ix promoter region.Haemophilia B Leyden is characterized by severe factor ix deficiency during childhood with partial resolution at puberty or following the administration of anabolic steroids. The disorder has recently been associated with point mutations in the putative factor ix promoter region, which contains an imperfect direct repeat spanning a possible start site of transcription. We have identified a T to C transition at position 8 in the promoter region of a patient with the haemophilia B Leyden phenotype. A mutation at this site has not been previously reported and occurs within the repeat consensus sequence in the transcribed but untranslated portion of the gene. There is no family history of haemophilia and sequence analysis of his mother and other family members indicates that the mutation has arisen de novo in this patient. This observation provides further support for a causal relationship between point mutations in the presumptive promoter region of the factor ix gene and the Leyden phenotype.- - - - - - - - - - ranking = 1keywords = administration (Clic here for more details about this article) |
5/15. Disappearance of inhibitor to factor ix in a patient with severe haemophilia B and immunological characterization of the inhibitor.Disappearance of an inhibitor to factor ix in an 11-year-old boy with haemophilia B is described. He had been given a total of 14,200 units of a prothrombin complex concentrate (PCC) before an inhibitor to factor ix developed. He subsequently received four separate infusions of PCC and his inhibitor titre rose in response to the treatment for the following 4 years. No inhibitor is presently detected despite repeated administration of PCC. Immunological characterization of the inhibitor by inhibitor neutralization assays, modified crossed-immunoelectrophoresis and enzyme-linked immunosorbent assay demonstrated that it contained IgG2 and IgG4 heavy chains and kappa and lambda light chains. No large deletion of the factor ix gene in the patient was observed using cDNA (cVII).- - - - - - - - - - ranking = 1keywords = administration (Clic here for more details about this article) |
6/15. Suppression of secondary antibody response by intravenous immunoglobulin in a patient with haemophilia b and antibodies.A 39-year-old patient, suffering from severe haemophilia B and antibodies against factor ix, has twice been treated with extracorporeal protein A-sepharose adsorption followed by conventional substitution therapy in combination with immunosuppression (cyclophosphamide). On both occasions, separated by a 2-year interval, the same procedure was followed except that, on the second, administration of i.v. immunoglobulin (Gammonativ. KabiVitrum) was added. Within a week of the first treatment the patient developed a 15-fold increase in the antibody titre. Following the second treatment described here, no secondary antibody response could be detected, and after a further 12 weeks only traces of antibodies are demonstrable. It seems that antibody synthesis was suppressed by the i.v. immunoglobulin. No evidence was found to demonstrate that the effect was due either to a non-specific suppression of the immune and reticuloendothelial systems or to the action of interfering antibodies. It has not yet been established whether or not the protein A-sepharose adsorption technique, or the immunosuppressive treatment, contributed in any way to the result. The observations suggest a new approach to the treatment of haemophiliacs with antibodies of the high-responding type.- - - - - - - - - - ranking = 1keywords = administration (Clic here for more details about this article) |
7/15. Haemorrhage and factor ix deficiency in pituitary insufficiency.Acquired isolated factor ix deficiency is rare. A case presenting with haemorrhage after dental extraction is described. The factor ix deficiency was associated with pituitary insufficiency and was completely corrected following the administration of thyroxine. This contrasts with a previously reported case in which both thyroxine and cortisone were required for complete correction of the factor ix deficiency. The underlying mechanism and the consequences are briefly discussed.- - - - - - - - - - ranking = 1keywords = administration (Clic here for more details about this article) |
8/15. Spontaneous disappearance of an IgA anti-factor ix inhibitor in a child with Christmas disease.The few inhibitors to blood coagulation factor ix in patients with Christmas disease (hemophilia b) that have been studied have been shown to belong to the IgG class of immunoglobulins. We report the first case in which a factor ix inhibitor was of the IgA class. Additionally, he appears to be the youngest patient with hemophilia b to have developed an inhibitor. His inhibitor complicated treatment of the patient for several years because of its anamnestic rise following factor ix concentrate administration. It then spontaneously vanished and has not returned in spite of repeated factor ix complex administration.- - - - - - - - - - ranking = 2keywords = administration (Clic here for more details about this article) |
9/15. gastrectomy in siblings with Christmas disease (hemophilia b).gastrointestinal hemorrhage is regarded as one of the commonest clinical manifestations of classic hemophilia and Christmas disease. Although major surgery is usually avoided in such cases, recently we had 2 cases of Christmas disease in siblings who had undergone a successful gastrectomy and concomitant administration of a concentrated preparation of factor ix. In addition, findings in 7 other Japanese patients with hemophilia who underwent gastrectomy are discussed.- - - - - - - - - - ranking = 1keywords = administration (Clic here for more details about this article) |
10/15. Studies on a family with combined functional deficiencies of vitamin k-dependent coagulation factors.Two siblings with m ild hemorrhagic symptoms had combined functional deficiencies of vitamin k-dependent clotting factors. prothrombin (0.18-0.20 U/ml) and Stuart factor (factor x, 0.18-0.20 U/ml) and Stuart factor (factor x, 0.18-0.20 U/ml) were most severely affected. Antigenic amounts of affected coagulation factors were normal and normal generation of thrombin activity occurred in the patients' plasmas after treatment with nonophysiologic activators that do not require calcium for prothrombin activation. Hepatobilary disease, malabsorptive disorders, and plasma warfarin were not present. Both parents had normal levels of all coagulation factors. The patients' plasmas contained prothrombin that reacted both with antibody directed against des-gamma-carboxyprothrombin and native prothrombin. Crossed immunoelectrophoresis of patients' plasmas and studies of partially purified patient prothrombin suggested the presence of a relatively homogeneous species of dysfunctional prothrombin, distinct from the heterologous species found in the plasma of warfarin-treated persons. These studies are most consistent with a posttranslational defect in hepatic carboxylation of vitamin k-dependent factors. This kindred uniquely possesses an autosomal recessive disorder of vitamin k-dependent factor formation that causes production of an apparently homogeneous species of dysfunctional prothrombin; the functional deficiencies in clotting factors are totally corrected by oral or parenteral administration of vitamin K1.- - - - - - - - - - ranking = 1keywords = administration (Clic here for more details about this article) |
| | Next -> |