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  • 1
    Electronic Resource
    Electronic Resource
    Immune tolerance induction in the treatment of paediatric haemophilia A patients with factor VIII inhibitors (2000)
    Oxford UK : Blackwell Science Ltd
    Haemophilia 6 (2000), S. 0 
    Details
    ISSN: 1365-2516
    Source: Blackwell Publishing Journal Backfiles 1879-2005
    Topics: Medicine
    Notes: The development of an inhibitor to transfused factor VIII (FVIII) is a serious treatment-related problem in haemophiliac children. The management of patients with high titre FVIII inhibitors is difficult, and immune tolerance induction (ITI) is the only method available for the eradication of these inhibitors. The results of the ITI regimen used at the Children’s Hospital of Michigan Haemophilia Treatment Center are described and discussed. ITI was attempted in 14 children with severe haemophilia A (13 high responders, one low responder), with daily doses of FVIII alone. FVIII dosage was chosen according to the patient’s historical peak inhibitor titre. ITI included three phases; induction phase, dose reduction phase and maintenance phase. During the first phase, the starting dose was 50 or 100 U kg–1 d–1; during the second phase the FVIII dosage was reduced gradually to 25 U kg–1 every other day according to the inhibitor titre, FVIII recovery and/or half-life study. In the third (maintenance) phase, the children received either prophylactic therapy or episodic therapy for 12 months. The inhibitor elimination was defined as the time taken to achieve a negative inhibitor assay with no anamnestic response and normal FVIII recovery and/or normal half-life. Immune tolerance was achieved in 11 of 14 patients (79%) patients within a median time of 6 months; two children are still on therapy, three failed ITI. We observed either failure or prolongation of immune tolerance if the historical peak titre or the maximum titre during ITI was 〉200 BU. The success rate of our low dose ITI regimen is not different from that reported by other investigators and the inhibitor elimination time is similar to some of the studies reported previously.
    Type of Medium: Electronic Resource
    URL: http://dx.doi.org/10.1046/j.1365-2516.2000.00379.x
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  • 2
    Electronic Resource
    Electronic Resource
    Use of central venous catheters in children with haemophilia: one haemophilia treatment centre experience (1997)
    Oxford, UK : Blackwell Science Ltd
    Haemophilia 3 (1997), S. 0 
    Details
    ISSN: 1365-2516
    Source: Blackwell Publishing Journal Backfiles 1879-2005
    Topics: Medicine
    Notes: Summary. We report our clinic experience with central venous catheters (CVCs) in 23 children with haemophilia, who, in total, had 35 catheters. Seventeen of the 23 children had Broviac catheters (external), 6/23 had an implantable device (Port-A-Cath) only, while four had Broviac and Port-A-Cath at different times. The age of the patients at the time of initial catheter placement ranged from 4 months to 18 years; 11 were under age 3 years. Indications for CVC placement included induction of immune tolerance, treatment of HIV-related complications, prophylactic therapy following intracranial haemorrhage, primary prophylaxis and secondary prophylaxis. Catheter-related infection was the most common complication, occurring in 84% of the external catheters. Of these infections 79% occurred in HIV-negative subjects. Staph and strep species were the most common infectious organisms isolated. Minor bleeding around the catheter occurred in 20% of cases even with adequate correction of haemophilia. Three children accidentally removed the external catheter (12%). Thrombosis of the catheter was uncommon (8%), occurring in only two patients, one of whom had a small newborn size external catheter. When presenting with line sepsis, haemophilic children who were HIV negative had transient temperature spikes approximately 1 h after catheter flushing. This presentation was different from our oncology or HIV-positive patients with CVC-related sepsis. Our experience with ports (Port-A-Cath) in haemophilic children is limited, but catheter-related infection (40%) seems to be less of a problem with the implantable device and the ports have been well accepted by our haemophilic patients and their parents.
    Type of Medium: Electronic Resource
    URL: http://dx.doi.org/10.1046/j.1365-2516.1997.00117.x
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  • 3
    Electronic Resource
    Electronic Resource
    The safety and efficacy of B-domain deleted recombinant factor VIII concentrates in patients with severe haemophilia A: an update (2005)
    Oxford, UK : Blackwell Science Ltd
    Haemophilia 11 (2005), S. 0 
    Details
    ISSN: 1365-2516
    Source: Blackwell Publishing Journal Backfiles 1879-2005
    Topics: Medicine
    Type of Medium: Electronic Resource
    URL: http://dx.doi.org/10.1111/j.1365-2516.2005.01099.x
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  • 4
    Electronic Resource
    Electronic Resource
    In vivo recovery with products of very high purity — assay discrepancies (1998)
    Oxford, UK : Blackwell Science Ltd
    Haemophilia 4 (1998), S. 0 
    Details
    ISSN: 1365-2516
    Source: Blackwell Publishing Journal Backfiles 1879-2005
    Topics: Medicine
    Notes: Summary. In view of reports of FVIII assay discrepancies in post-infusion plasma samples depending on methods used, we compared FVIII results run by each of four different methods following infusion of rFVIII (Kogenate®). Nine persons with haemophilia A were infused with each of two lots of product. Plasma samples were obtained at baseline, and at 10 min, 30 min, 1, 2, 4, 8, 12, 14, 30 and 48 h post-infusion for measurement of FVIII. FVIII assay methods were chromogenic, and one-stage APTT using three different types of activators: micronized, silica, ellagic acid, and kaolin. The same reference plasma standard was used throughout. Results demonstrated a consistent difference in FVIII values, with chromogenic assays being considerably higher than those run by one-stage assays. The discrepancy was greatest when kaolin was the activator. These results point out the problems in attempting to determine the “correct” FVIII level in patient plasma samples following infusion of high purity FVIII preparations. Potential “pitfalls” include the standard used for defining product potency, the methods, reagents, instrumentation and standards used in assaying plasma samples and, in some instances, the characteristics of the product itself. This situation has considerable cost implications, potential impact on patient care, and makes it difficult to compare results between laboratories.
    Type of Medium: Electronic Resource
    URL: http://dx.doi.org/10.1046/j.1365-2516.1998.440641.x
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  • 5
    Electronic Resource
    Electronic Resource
    Factor IX inhibitors and anaphylaxis in haemophilia B (1996)
    Oxford, UK : Blackwell Publishing Ltd
    Haemophilia 2 (1996), S. 0 
    Details
    ISSN: 1365-2516
    Source: Blackwell Publishing Journal Backfiles 1879-2005
    Topics: Medicine
    Type of Medium: Electronic Resource
    URL: http://dx.doi.org/10.1111/j.1365-2516.1996.tb00149.x
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  • 6
    Electronic Resource
    Electronic Resource
    Types of bleeding seen during the first 30 months of life in children with severe haemophilia A and B (1996)
    Oxford, UK : Blackwell Publishing Ltd
    Haemophilia 2 (1996), S. 0 
    Details
    ISSN: 1365-2516
    Source: Blackwell Publishing Journal Backfiles 1879-2005
    Topics: Medicine
    Notes: Summary. In view of excellent preservation of joint structure and function in Swedish patients started on primary prophylaxis at 1–2 years of age, it has recently been recommended that primary prophylaxis be considered optimal management for U.S. children with severe haemophilia (〈 1% FVIII or FIX). In order to determine the age at which joint bleeding began, we reviewed (49) patients with severe haemophilia who were seen at our haemophilia centre for all bleeding episodes from birth on. Eight of the 49 children were first treated during the first few days of life for bleeding related to circumcision (6), spontaneous splenic rupture (1) or soft tissue haematoma (1). None had intracranial haemorrhage (ICH) in the immediate neonatal period. Other bleeding episodes for which children were treated during the first 30 months of life included soft tissue haematoma (42 episodes), tongue and mouth bleeding (37 episodes), ICH (four children) or for surgery (five children). Joint bleeding occurred in 16/49 children with severe haemophilia. Four of the 16 had haemophilia B, while 13 had haemophilia A. A single episode of severe joint bleeding occurred in each of the five patients before 12 months of age. Eight patients had 12 episodes of joint bleeding between 12 and 18 months of age and three patients accounted for 15 episodes between 18 and 24 months of age. Three children had a total of four joint haemorrhages between 25 and 30 months, and 33/49 children did not have any joint bleeding before 30 months of age. It is noteworthy that two children had recurrent bleeding into the same joint. One with haemophilia A had recurrent bleeding into the knees (at 14, 18, 20, 21 months), while one with haemophilia B had recurrent bleeding into one ankle starting at age 16 months. It would thus seem appropriate to begin primary prophylaxis in children with severe haemophilia once joint bleeding has begun (one or two episodes), rather than at a standard age.
    Type of Medium: Electronic Resource
    URL: http://dx.doi.org/10.1111/j.1365-2516.1996.tb00156.x
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  • 7
    Electronic Resource
    Electronic Resource
    The safety and efficacy of B-domain deleted recombinant factor VIII concentrate in patients with severe haemophilia A (2003)
    Oxford, UK : Blackwell Science Ltd
    Haemophilia 9 (2003), S. 0 
    Details
    ISSN: 1365-2516
    Source: Blackwell Publishing Journal Backfiles 1879-2005
    Topics: Medicine
    Notes: Background: B-domain-deleted recombinant factor VIII (BDDrFVIII) was developed when the B-domain was found to be redundant for maintaining haemostasis. This allows formulation of the final product without albumin added as a stabilizer.Methods: Three multicentre clinical studies and one pharmacokinetic study were conducted in 218 patients to evaluate the safety and haemostatic efficacy of BDDrFVIII.Results: Previously treated patients (n = 113; median duration, 1711 days; median exposure days, 385; total 98 096 287 IU infused) rated 97–99% of all infusions as good or excellent efficacy. FVIII inhibitor was noted in one patient in the previously treated patient cohort after 113 exposure days. Among 101 previously untreated patients, responses to BDDrFVIII were rated as excellent or good in 92–95% of infusions (median duration, 1413 days; median exposure days, 148; total 12 636 458 IU infused). Thirty-two previously untreated patients developed inhibitors after a median duration of 12 exposure days (range, 3–49). Sixteen of 32 (50%) patients had low levels (≤ 5 Bethesda units) and 16 had high levels of inhibitors. Inhibitors disappeared in six of 14 (43%) of the high-level and six of eight (75%) of the low-level patients who underwent immune tolerance induction therapy. A total of 42 patients underwent surgery and the overall efficacy of BDDrFVIII was rated as excellent or good for 99.6% of infusions.Conclusions: The results of these clinical studies indicate that BBDrFVIII is safe and effective and has haemostatic activity similar to that of full-length FVIII concentrates.
    Type of Medium: Electronic Resource
    URL: http://dx.doi.org/10.1046/j.1365-2516.2003.00708.x
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  • 8
    Electronic Resource
    Electronic Resource
    The overall effectiveness of prophylaxis in severe haemophilia (2003)
    Oxford, UK : Blackwell Science Ltd
    Haemophilia 9 (2003), S. 0 
    Details
    ISSN: 1365-2516
    Source: Blackwell Publishing Journal Backfiles 1879-2005
    Topics: Medicine
    Notes: Summary.  The aim of this retrospective review was to assess the overall effectiveness of prophylaxis when compared with on-demand treatment of haemophilic patients. Twenty-five children (22 with severe haemophilia A and three with severe haemophilia B) were evaluated. Five haemophilia A patients received primary prophylaxis (instituted before the onset of any joint bleed) while the other 17 haemophilia A and all three haemophilia B patients were on secondary prophylaxis. We compared factor usage, number of bleeding episodes, emergency room (ER) visits and hospitalizations while on prophylaxis to those while on demand therapy. All subjects were male, the median age at time of review was 11.4 years and at start of prophylaxis was 4.5 years. Thirteen of the 25 patients (52%) required indwelling venous catheters for access, seven of these had one or more (one–six) episodes of line sepsis. Haemophilia A patients received an average of 23.8 U kg−1 (20–30 U kg−1) of recombinant factor VIII three times a week while haemophilia B patients received 50 U kg−1 recombinant FIX twice weekly. There was a significant reduction in the mean number of major bleeds on prophylaxis from 15.5 to 1.9 per year and a significant decrease in target joints, ER visits and hospitalizations. Although factor usage per year was higher on prophylaxis, there was an overall reduction in number of bleeds and resultant decrease in hospitalizations and ER visits. By preventing new target joints, prophylaxis can lead to reduction in long-term morbidity and a better quality of life despite increased central lines and higher factor usage.
    Type of Medium: Electronic Resource
    URL: http://dx.doi.org/10.1046/j.1365-2516.2003.00757.x
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  • 9
    Electronic Resource
    Electronic Resource
    B-domain deleted recombinant factor VIII preparations are bioequivalent to a monoclonal antibody purified plasma-derived factor VIII concentrate: a randomized, three-way crossover study (2005)
    Oxford, UK : Blackwell Science Ltd
    Haemophilia 11 (2005), S. 0 
    Details
    ISSN: 1365-2516
    Source: Blackwell Publishing Journal Backfiles 1879-2005
    Topics: Medicine
    Notes: Summary.  Background:  Deletion of the B-domain of recombinant blood coagulation factor VIII (BDDrFVIII) increases the manufacturing yield of the product but does not impair in vitro or in vivo functionality. BDDrFVIII (ReFacto®) has been developed with the additional benefit of being formulated without human albumin.Objective:  The primary objective of this three-way crossover-design study was to compare the pharmacokinetic (PK) parameters of two BDDrFVIII formulations (one reconstituted with 5 mL of sterile water, the other reconstituted with 4 mL sodium chloride 0.9% USP) with those of a plasma-derived, full-length FVIII preparation (Hemofil® M) in patients with haemophilia A to determine bioequivalence.Methods:  A series of blood samples were collected over a period of 48 h after i.v. administration of each of the FVIII preparations. Plasma FVIII activity was determined using a validated chromogenic substrate assay. Plasma FVIII activity vs. time curves was characterized for a standard set of PK parameter estimates. Two parameter estimates, the maximum plasma concentration (Cmax) and the area under plasma concentration vs. time curves (AUCs), were used to evaluate bioequivalence. The two preparations were considered bioequivalent if the 90% confidence intervals for the ratio of geometric means for Cmax and AUCs fell within the bioequivalence window of 80% to 125%.Results/Conclusion:  Results show that each BDDrFVIII formulation is bioequivalent to Hemofil M and the two formulations of BDDrFVIII are bioequivalent to each other.
    Type of Medium: Electronic Resource
    URL: http://dx.doi.org/10.1111/j.1365-2516.2005.01068.x
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  • 10
    Electronic Resource
    Electronic Resource
    Anaphylactic response to factor IX replacement therapy in haemophilia B patients: complete gene deletions confer the highest risk (1999)
    Oxford UK : Blackwell Science Ltd
    Haemophilia 5 (1999), S. 0 
    Details
    ISSN: 1365-2516
    Source: Blackwell Publishing Journal Backfiles 1879-2005
    Topics: Medicine
    Notes: Haemophilia B is an X-linked recessive coagulopathy due to mutations in the factor IX gene. Occasionally, patients receiving factor IX replacement therapy develop inhibiting antibodies to the factor IX protein, and it has been recently documented that a subset of these patients have had anaphylactic responses to factor IX replacement therapy in association with the development of inhibiting antibodies. To determine the relationship between mutation type and the risk of anaphylaxis, eight unrelated patients from families in whom anaphylaxis had occurred were genotyped. The mutations were compared to those in 550 haemophilia B patients and to those in 276 patients with clinically severe disease. Individuals with complete gene deletions were found to be at greatest risk for anaphylaxis, with an estimated risk of 26% or greater. Anaphylaxis was less likely to occur in patients with protein truncation mutations or partial gene deletions and least likely to occur with missense mutations. Genotypes can help physicians and patients anticipate the likelihood of anaphylaxis, a potentially life-threatening complication of factor IX replacement therapy. The very high risk of anaphylaxis associated with a complete gene deletion suggests that the lack of expression of a partial protein product may predispose to anaphylaxis and/or that the absence of a closely linked, codeleted gene enhances the anaphylactic immune response.
    Type of Medium: Electronic Resource
    URL: http://dx.doi.org/10.1046/j.1365-2516.1999.t01-1-00303.x
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