Health status and health-related quality of life of children with haemophilia from six West European countries.

A multicentre, international, cross-sectional study was carried out in the frame of field testing of the first haemophilia-specific quality-of-life (QoL) questionnaire (Haemo-QoL). The aim of this paper is to describe health status and health care and their impact on QoL in haemophilic children in Western Europe. Children aged 4-16 years with severe haemophilia without inhibitors were enrolled by 20 centres in France, Germany, Italy, the Netherlands, Spain and the United Kingdom.

[Are the indications and choice of labile blood products adapted? National survey on prescriber's knowledge].

Objective: There is little data available on current practice related to prescription of labile blood products (LBP) by French physicians. The aim of this study was to assess whether prescriptions were conform to Anaes (French Medicine's agency) guidelines, with regard not only to indications but also quality of the products, so as to define the improvements that could be made.

Method: Thirty-four clinical case reports, classified by specialties were sent to prescribing physicians working in the regional health centers, from 17 different blood banks, from October 1997 to February 1998.

Pilot testing of the 'Haemo-QoL' quality of life questionnaire for haemophiliac children in six European countries.

In a multinational working group, an instrument (Haemo-QoL) to assess quality of life in children/adolescents with haemophilia and their parents has been developed. In co-operation with haemophilia treatment centres in six European countries, approximately 10 children/adolescents with haemophilia per country and their parents were asked to participate in the pilot-testing. Both self-reported and parent-reported questionnaires were provided for two age-groups of children (4-16 years).

European data of a clinical trial with a sucrose formulated recombinant factor VIII in previously treated haemophilia A patients.

To increase the safety of antihaemophilic treatment, the production process of full-length recombinant factor VIII (FVIII) KOGENATE Bayer (Kogenate FS) has been modified. Human albumin is no longer added as stabilizer during purification and in final formulation. Instead, the new KOGENATE Bayer production process uses sucrose as a stabilizer in the formulation and adds solvent/detergent virus inactivation step.

Use of recombinant factor VIIa (NovoSeven) in patients with Glanzmann thrombasthenia.

Recombinant factor VIIa (rFVIIa; NovoSeven, Novo Nordisk, Bagsvaerd, Denmark) appears effective and relatively safe for the treatment of bleeding and for surgical prophylaxis in patients with Glanzmann thrombasthenia as reported to the International Registry on rFVIIa and Congenital Platelet Disorders. One of the shortcomings of the Registry data is the heterogeneity of treatment protocol, including dosage, number of doses used, duration of treatment before declaration of failure, and mode of rFVIIa administration (bolus v continuous infusion). The data are not yet sufficient to define optimal regimens for various indications such as the type of bleeding or the type of procedures.

The use of recombinant factor VIIa (NovoSeven) in a patient with a factor XI deficiency and a circulating anticoagulant.

A 75-year-old female known to have a chronic myelomonocytic leukaemia and an acquired FXI deficiency (FXI level, 5%) related to a FXI inhibitor (38 Bethesda units) was admitted to the hospital for acute pneumonia associated with a bulky pleural effusion. A therapeutic puncture was found to be essential for the patient. But, such a procedure is a haemostatic challenge which requires adequate preparation.

[Evaluation of the immediate transfusion reaction incident reporting system at the Brest University Hospital Center].

The Haemovigilance Unit of Brest University Hospital has had a reporting system of transfusion reactions since october 1994. Reporting "any unexpected or undesirable effect due or likely to be due to the administering of blood cell components" must be done on an answering machine immediately or in the next eight hours. The main goal of the evaluation of this epidemiological surveillance system was to assess its sensitivity, its positive predictive value, its acceptability, its timeliness and its simplicity, according to the Centers for Disease Control criteria.

French previously untreated patients with severe hemophilia A after exposure to recombinant factor VIII : incidence of inhibitor and evaluation of immune tolerance.

Fifty French previously untreated patients with severe hemophilia A (factor VIII < 1%), treated with only one brand of recombinant factor VIII (rFVIII), were evaluated for inhibitor development, assessment of risk factors and outcome of immune tolerance regimen. The median period on study was 32 months (range 9-74) since the first injection of rFVIII. Fourteen patients (28%) developed an inhibitor, four of whom (8%) with a high titer (> or = 10 BU).

[Feasibility of following up transfused patients].

The aim of this study was to assess the natural history of patients after transfusion and the acceptability of a standardized biological follow-up. In 1995, during 1 month, in 13 French hospitals, a follow-up at 3 and 6 months after blood transfusion was proposed to all blood recipients who had not received any blood transfusion within the past 6 months (eligible patients): screening for red cell antibodies, alanine aminotransferase (ALT) activity and specific viral markers of hepatitis B (hepatitis B surface antigen and antibody to hepatitis virus core antigen), of hepatitis C (antibodies) and of Human Immunodeficiency Virus (antibodies). At the beginning of the study, 296 patients were followed for 6 months.

[Analysis of transfusion incident reports filed at 15 blood transfusion centers and health facilities during 17 months. Groupe Receveurs de laSFTS].

The principal result of the development of hemovigilance since 1994 has been the declaration of undesirable effects likely to be due to transfusions of labile blood products. Using the 1,694 cases of undesirable effects registered, it seemed worthwhile to us to analyze the distribution of the signs noticed, their frequency and the types of blood products responsible. This analysis allowed us to observe that the majority of reactions were shivery-feverish (47%) or allergic (24%).

[Pilot study of the characteristics of transfused patients and utilized labile blood products].

The aim of this study was to describe blood recipients and blood components transfused during the first 24 hours in 13 French hospitals. We included all blood recipients who had not had any blood transfusion within the past six months. Recipients were screened for red cell alloantibodies, the alanine aminotransferase activity and specific viral markers (hepatitis B and C, Human Immunodeficiency Virus).

[Importance of flow cytometry in the detection of anti-erythrocyte autoantibodies].

Coombs test and flow cytometry have been compared in terms of specificity and sensibility in a population of hospitalized patients, for whom a Coombs test had been required. The Coombs test seems more sensible than flow cytometry to detect red cell-bound IgG. For a given patient and over the time, flow cytometry seems better correlated with the severity of haemolysis if erythrocytes are strongly sensitized by IgG.

Incidence of factor VIII inhibitor development in severe hemophilia A patients treated only with one brand of highly purified plasma-derived concentrate.

The incidence of factor VIII inhibitor was studied in a cohort of 56 previously untreated patients with severe hemophilia A (factor VIII below 1 U/dl). They received only one brand of highly purified factor VIII concentrate (HPSD-VIII) prepared by conventional chromatography with a solvent-detergent step for viral inactivation. Follow-up since the first infusion of HPSD-VIII was from 1 to 76 months (mean = 29) and cumulative exposure days (CED) from 1 to over 100 (median = 26).

Alloimmunization against Iy, a low-frequency antigen on platelet glycoprotein Ib/IX as a cause of severe neonatal alloimmune thrombocytopenic purpura.

Neonatal alloimmune thrombocytopenia (NAIT) is usually induced by platelet-specific antibodies against HPA-1a (Zwa) or HPA-5b (Bra). Recently, low-frequency alloantigens on the platelet glycoprotein (GP) IIb/IIIa complex have been discovered as a cause for NAIT. In this report, a new low-frequency platelet-specific alloantigen, Iy, is described which induced severe NAIT.

[Fresh frozen plasma: a pilot study of the context of utilization and the indications].

The aim of this pilot study was to assess the feasibility of a tolerance study of qualified (secured by quarantine or solvent-detergent-treated) fresh frozen plasma (FFP) in real conditions of use. We included all patients receiving qualified FFP during a one-month observation period in three french hospitals (Besançon, Brest, Lyon). The 192 FFP transfusion episodes corresponded to 111 patients.

[Immunization against a new, infrequent alloantigen (Iy) on the platelet glycoprotein Ib/IX as a cause of a serious case of neonatal alloimmune thrombocytopenia].

Neonatal alloimmune thrombocytopenia is the consequence of maternal alloimmunization against platelet-specific alloantigens, usually PIA1 or Br(a). The clinical picture is characterized by signs of haemorrhagic diathesis as a result of marked thrombocytopenia. In the last years, rare cases of immunization against 'low-frequency' or 'private' platelet alloantigens on the platelet glycoprotein (GP) complex IIb/IIIa have been found.

[HIV infection among type A and B hemophiliacs].

Evolution of HIV infection was studied in 480 hemophiliacs A and 78 hemophiliacs B treated in the "Centre-West" Region. 23.3% hemophiliacs A and 46.

Impaired fetal erythrocytes' filterability: relationship with cell size, membrane fluidity, and membrane lipid composition.

The lipid composition of erythrocytes (red blood cells [RBCs]) plays a significant role in determining certain membrane biophysical properties. We have found that fetal RBCs showed a dramatically low filterability compared with adult RBCs and questioned whether this could be a consequence of their membrane lipid composition. We therefore studied fetal RBCs at two different gestational ages, neonatal RBCs and adult RBCs.

[Efficacy in viral inactivation of the concentrates of factor VIII and IX by the solvent/detergent procedure. Evaluation in patients with hemophilia].

Human immunodeficiency virus (HIV) infection and hepatitis virus B or C (HBV, HCV) transmission are major risks following infusion of coagulation factor concentrates. Thus, several methods have been used to achieve viral inactivation of concentrates prepared from plasma collected from a large number of donors. In this study, 32 patients with haemophilia A or B (n = 31) or von Willebrand's disease (n = 1) were treated between 1987 and 1990 only with factor VIII or IX concentrates inactivated by the solvent-detergent procedure.

The antiperinuclear factor. II. Variability of the perinuclear antigen.

Since the antiperinuclear factor (APF) test on human buccal cells is rather unpredictable, we have investigated the possible factors determining the expression of appropriate antigens by the cells. We failed to find any relationship of the expression of perinuclear antigens to the donor's smoking habits, the degree of contamination with saprophytic bacteria, the presence or absence of blood group substances in saliva, or the titers of serum antibodies to Epstein-Barr virus. Family studies were also performed to further elucidate a genetic predisposition to the expression of the APF antigen.

[Autotransfusion and hemodilution in orthopedic surgery in children].

The risk of transmitting viral diseases is a constant worry for those who perform blood transfusions. In the last three years several techniques were used to avoid homologous blood transfusions. Four series of patients were evaluated: 1st series: Only homologous blood transfusions (27 cases); 2nd series: Acute normovolaemic haemodilution during surgery (20 cases); 3rd series: Autologous blood storage prior to surgery and haemodilution during surgery (20 cases); 4th series: Blood storage prior to surgery + haemodilution + Trinitine perfusion during surgery (44 cases).

[Drug-induced thrombopenias and neutropenias. An in vitro study by the indirect immunofluorescence test].

The sera from 38 patients with suspected drug-induced thrombocytopenia (22) or neutropenia (16) were tested with the indirect immunofluorescence test on platelets or granulocytes for the presence of drug-dependent antibodies. Three drug-induced antibodies with reactivity against platelets and 5 with reactivity against granulocytes were detected. In 3 sera antibodies were found which reacted already with target cells without adding the drug to the test system.

[Effect of in vitro carbamylation of erythrocytes on their rheological properties].

In order to assess the role of carbamylation of erythrocyte proteins in the modification of rheological parameters of red blood cells observed in uremic patients, and in vitro carbamylation of erythrocytes and hemoglobin was carried out using sodium cyanate. The carbamylation of hemoglobin was determined by observation of the increase of HbA1 fraction. The deformability of erythrocytes and the viscosity of erythrocyte suspensions and of hemolysate were measured.