K Fischer

University Medical Center Utrecht, Utrecht, Utrecht, Netherlands

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Publications (150)546.58 Total impact

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    ABSTRACT: Inhibitor development represents the most serious side effect of haemophilia treatment. Any difference in risk of inhibitor formation depending on the product used might be of clinical relevance. It was this study's objective to assess inhibitor development according to clotting factor concentrate in severe haemophilia A and B. The European Haemophilia Safety Surveillance (EUHASS) was set up as a study monitoring adverse events overall and according to concentrate. Since October 2008, inhibitors were reported at least quarterly. Number of treated patients was reported annually, specifying the number of patients completing 50 exposure days (Previously Untreated Patients, PUPs) without inhibitor development. Cumulative incidence, incidence rates and 95 % confidence intervals (CI) were calculated. Data from October 1, 2008 to December 31, 2012 were analysed for 68 centres that validated their data. Inhibitors developed in 108/417 (26 %; CI 22-30 %) PUPs with severe haemophilia A and 5/72 (7 %; CI 2-16) PUPs with severe haemophilia B. For Previously Treated Patients (PTPs), 26 inhibitors developed in 17,667 treatment years [0.15/100 treatment years (CI 0.10-0.22)] for severe haemophilia A and 1/2836 (0.04/100;(CI 0.00-0.20) for severe haemophilia B. Differences between plasma-derived and recombinant concentrates, or among the different recombinant FVIII concentrates were investigated. In conclusion, while confirming the expected rates of inhibitors in PUPs and PTPs, no class or brand related differences were observed.
    Thrombosis and Haemostasis 01/2015; 113(4). DOI:10.1160/TH14-10-0826 · 5.76 Impact Factor
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    ABSTRACT: - Copyright © 2014, Ferrata Storti Foundation.
    Haematologica 12/2014; DOI:10.3324/haematol.2014.115709 · 5.94 Impact Factor
  • K Fischer
    Haemophilia 12/2014; 21(1). DOI:10.1111/hae.12586 · 2.47 Impact Factor
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    ABSTRACT: Health-related quality of life (HRQoL) assessment is recognized as an important outcome in the evaluation of different therapeutic regimens for persons with haemophilia. The Canadian Haemophilia Outcomes-Kids' Life Assessment Tool (CHO-KLAT) is a disease-specific measure of HRQoL for 4 to 18-year-old boys with haemophilia. The purpose of this study was to extend this disease-specific, child-centric, outcome measure for use in international clinical trials. We adapted the North American English CHO-KLAT version for use in five countries: France, Germany, the Netherlands, Spain and the United Kingdom (UK). The process included four stages: (i) translation; (ii) cognitive debriefing; (iii) validity assessment relative to the PedsQL (generic) and the Haemo-QoL (disease-specific) and (iv) assessment of inter and intra-rater reliability. Cognitive debriefing was performed in 57 boys (mean age 11.4 years), validation was performed in 144 boys (mean age 11.0 years) and reliability was assessed for a subgroup of 64 boys (mean age 12.0 years). Parents also participated. The mean scores reported by the boys were high: CHO-KLAT 77.0 (SD = 11.2); PedsQL 83.8 (SD = 11.9) and Haemo-QoL 79.6 (SD = 11.5). Correlations between the CHO-KLAT and PedsQL ranged from 0.63 in Germany to 0.39 in the Netherlands and Spain. Test-retest reliability (concordance) for child self-report was 0.67. Child-parent concordance was slightly lower at 0.57. The CHO-KLAT has been fully culturally adapted and validated for use in five different languages and cultures (in England, the Netherlands, France, Germany and Spain) where treatment is readily available either on demand or as prophylaxis. © 2014 John Wiley & Sons Ltd.
    Haemophilia 12/2014; DOI:10.1111/hae.12597 · 2.47 Impact Factor
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    ABSTRACT: To facilitate early prophylaxis, step-up regimens starting prophylaxis with infusions 1× week−1 were introduced. Choice of initial regimen may affect outcome. This study aims to classify initial prophylactic regimens and compare them on short-term outcome. From the ‘European Paediatric Network for Haemophilia Management' (PedNet) registry, patients with severe haemophilia A without inhibitors, born 2000–2012, receiving prophylaxis were included. Treatment centres were classified according to the initial frequency of prophylactic infusions and the age at reaching infusions ≥3× week−1. Bleeding, and central venous access device (CVAD) use were compared at age 4 years. In 21 centres with 363 patients, three regimens were identified: (i) start prophylaxis with ≥3× week−1 infusions before age three (full: 19% of centres, 18% of patients); (ii) start 1–2× week−1, increasing frequency as soon as possible (asap), reaching ≥3× week−1 before age three (43% of centres, 36% of patients); (iii) start 1–2× week−1, increasing frequency according to bleeding (phenotype), reaching ≥3× week−1 after age three (38% of centres, 46% of patients). Prophylaxis was started at median 1.2 years on the full and asap regimen vs 1.8 years on the phenotype regimen. Complete prevention of joint bleeds was most effective on the full regimen (32% full vs. 27% asap and 8% phenotype), though at the cost of using most CVADs (88% full vs. 34% asap and 22% phenotype). The three prophylaxis regimens identified had different effects on early bleeding and CVAD use. This classification provides the first step towards establishing the optimum prophylactic regimen.
    Haemophilia 12/2014; DOI:10.1111/hae.12613 · 2.47 Impact Factor
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    ABSTRACT: Treatment of previously untreated patients (PUPs) with severe haemophilia A is complicated by the formation of inhibitors. Prediction of PUPs with high risk is important to allow altering treatment with the intention to reduce the occurrence of inhibitors. An unselected multicentre cohort of 825 PUPs with severe haemophilia A (FVIII<0.01 IU mL−1) was used. Patients were followed until 50 exposure days (EDs) or inhibitor development. All predictors of the existing prediction model including three new potential predictors were studied using multivariable logistic regression. Model performance was quantified [area under the curve (AUC), calibration plot] and internal validation (bootstrapping) was performed. A nomogram for clinical application was developed. Of the 825 patients, 225 (28%) developed inhibitors. The predictors family history of inhibitors, F8 gene mutation and an interaction variable of dose and number of EDs of intensive treatment were independently associated with inhibitor development. Age and reason for first treatment were not associated with inhibitor development. The AUC was 0.69 (95% CI 0.65–0.72) and calibration was good. An improved prediction model for inhibitor development and a nomogram for clinical use were developed in a cohort of 825 PUPs with severe haemophilia A. Clinical applicability was improved by combining dose and duration of intensive treatment, allowing the assessment of the effects of treatment decisions on inhibitor risk and potentially modify treatment.
    Haemophilia 12/2014; 21(2). DOI:10.1111/hae.12566 · 2.47 Impact Factor
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    ABSTRACT: Introduction and hypothesis To predict who will undergo midurethral sling surgery (surgery) after initial pelvic floor muscle training (physiotherapy) for stress urinary incontinence in women. Methods This was a cohort study including women with moderate to severe stress incontinence who were allocated to the physiotherapy arm from a previously reported multicentre trial comparing initial surgery or initial physiotherapy in treating stress urinary incontinence. Crossover to surgery was allowed. Results Data from 198/230 women who were randomized to physiotherapy was available for analysis, of whom 97/198 (49 %) crossed over to surgery. Prognostic factors for undergoing surgery after physiotherapy were age Conclusion In women with moderate to severe stress incontinence, individual prediction for surgery after initial physiotherapy is possible, thus enabling shared decision making for the choice between initial conservative or invasive management of stress urinary incontinence.
    International Urogynecology Journal 07/2014; 26(3). DOI:10.1007/s00192-014-2473-z · 2.16 Impact Factor
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    Blood transfusion = Trasfusione del sangue 05/2014; 12(Suppl 3):s515-s518. DOI:10.2450/2014.0008-14s · 1.90 Impact Factor
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    ABSTRACT: Haemophilia is a rare disease. To improve knowledge, prospective studies of large numbers of subjects are needed. To establish a large well-documented birth cohort of patients with haemophilia enabling studies on early presentation, side effects and outcome of treatment. Twenty-one haemophilia treatment centres have been collecting data on all children with haemophilia with FVIII/IX levels up to 25% born from 2000 onwards. Another eight centres collected data on severe haemophilia A only. At baseline, details on delivery and diagnosis, gene mutation, family history of haemophilia and inhibitors are collected. For the first 75 exposure days, date, reason, dose and product are recorded for each infusion. Clinically relevant inhibitors are defined as follows: at least two positive inhibitor titres and a FVIII/IX recovery <66% of expected. For inhibitor patients, results of all inhibitor- and recovery tests are collected. For continued treatment, data on bleeding, surgery, prophylaxis and clotting factor consumption are collected annually. Data are downloaded for analysis annually. In May 2013, a total of 1094 patients were included: 701 with severe, 146 with moderate and 247 with mild haemophilia. Gene defect data were available for 87.6% of patients with severe haemophilia A. The first analysis, performed in May 2011, lead to two landmark publications. The outcome of this large collaborative research confirms its value for the improvement of haemophilia care. High-quality prospective observational cohorts form an ideal source to study natural history and treatment in rare diseases such as haemophilia.
    Haemophilia 05/2014; 20(4). DOI:10.1111/hae.12448 · 2.47 Impact Factor
  • Frank W G Leebeek, Kathelijn Fischer
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    ABSTRACT: Background. In the Netherlands, the first formal haemophilia comprehensive care centre was established in 1964, and Dutch haemophilia doctors have been organised since 1972. Although several steps were taken to centralise haemophilia care and maintain quality of care, treatment was still delivered in many hospitals, and formal criteria for haemophilia treatment centres as well as a national haemophilia registry were lacking. Material and methods. In collaboration with patients and other stakeholders, Dutch haemophilia doctors have undertaken a formal process to draft new quality standards for the haemophilia treatment centres. First a project group including doctors, nurses, patients and the institute for harmonisation of quality standards undertook a literature study on quality standards and performed explorative visits to several haemophilia treatment centres in the Netherlands. Afterwards concept standards were defined and validated in two treatment centres. Next, the concept standards were evaluated by haemophilia doctors, patients, health insurance representatives and regulators. Finally, the final version of the standards of care was approved by Central body of Experts on quality standards in clinical care and the Dutch Ministry of Health. Results. A team of expert auditors have been trained and, together with an independent auditor, will perform audits in haemophilia centres applying for formal certification. Concomitantly, a national registry for haemophilia and allied disorders is being set up. Discussion. It is expected that these processes will lead to further concentration and improved quality of haemophilia care in the Netherlands.
    Blood transfusion = Trasfusione del sangue 04/2014; 12(Suppl 3):s501-s504. DOI:10.2450/2014.0041-14s · 1.90 Impact Factor
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    ABSTRACT: In elderly people with haemophilia (PWH), surgery of more than one joint of the lower extremities might be needed. Multiple joint procedures (MJP) were introduced in 1995, defined as any combination of Total Knee or Total Hip Arthroplasty or Ankle Arthrodesis during one in-hospital stay. The expectation is that by means of such procedures this specific population is able to physically function better for an extended period of time. Thus, they will participate in their society in an optimal way. In this study, we tried to describe an outcome after MJP, including pre- and post-operative pain and range of motion (ROM), and recommend measurement tools. 22 of 37 PWH who underwent MJP between 1995 and 2012 were available for assessment. Pain (WFH score) and range of motion were compared pre and postoperatively. Current outcome was described by VAS per joint, nocturnal and overall pain, MACTAR, Hemophilia Activity List, SF36, and EQ-5D. Mean age at surgery was 50, 3 years (SD 8, 3); mean follow-up 12 years (1-18 years). Pain (VAS) decreased post-surgery (Median 1 - 1, 5), but moderate pain remained. Extension of knees slightly increased, but both knee flexion and ankle plantar and dorsal flexion decreased. PWH reported the ability to stand longer but also pointed at specific problems, e.g. riding a bike (MACTAR). The HAL showed limited activities (functional domains), especially in the 'complex lower extremity' (22, 8/100). The SF36 and EQ-5D showed a mix of physical problems of our population, while experiencing moderate pain and reasonable physical functioning. This led us to the conclusion that adequate follow-up is needed: ROM of all joints, VAS of all joints as well as nocturnal and overall pain, HAL, SF36 and EQ5D. Performance based activities and participation need further attention.
    Haemophilia 03/2014; 20(2):276-81. DOI:10.1111/hae.12285 · 2.47 Impact Factor
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    ABSTRACT: Recently we reported data suggesting that platelets could compensate for the bleeding phenotype in severe haemophilia A (HA). The aim of this study was to confirm these results in a larger population with a detailed characterisation of clinical phenotype. Patients with diagnostic severe HA (FVIII:C <1%) were scored for clinical phenotype by integrating data on age at first joint bleed, joint damage, bleeding frequency and FVIII consumption. Phenotype was defined as onset of joint bleeding-score + arthropathy-score + joint bleeding-score + (2* treatment intensity-score). After a washout period of three days, blood was collected for measurement of basal level of platelet activation, platelet reactivity, endothelial cell activation and presence of procoagulant phospholipids in plasma. Thirty-three patients with severe HA were included, 13 patients with a mild, 12 patients with an average and eight patients with a severe clinical phenotype. No relevant differences in basal level of platelet activation, platelet reactivity, endothelial cell activation and procoagulant phospholipids between all three groups were observed. The mean annual FVIII consumption per kg did not correlate with the platelet P-selectin expression and glycoprotein (GP)IIbIIIa activation on platelets. In conclusion, variability in clinical phenotype in patients with diagnostic severe HA is not related to platelet activation or reactivity, measured as platelet degranulation and platelet GPIIbIIIa opening.
    Thrombosis and Haemostasis 01/2014; 111(6). DOI:10.1160/TH13-07-0546 · 5.76 Impact Factor
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    ABSTRACT: An adequate use of coping strategies could help patients to deal with disease-related stress. The study aim was to explore coping behaviour in adult patients with severe haemophilia and its possible determinants. Coping was assessed through three basic dimensions (task-oriented, emotion-oriented and avoidance coping), using the short version of the Coping Inventory for Stressful Situations (CISS-21). Patients' scores were compared with Dutch working men (N = 374), according to three categories: low use (<P25 of normal), average use (P25-P75) and high use (>P75). Determinants were measured using questionnaires on activities (Haemophilia Activities List), participation (Impact on Participation and Autonomy Questionnaire), physical functioning [physical component of the Dutch Arthritis Impact Measurement Scales-2 (D-AIMS2)] and socio-psychological health (psychological component of the D-AIMS2). In total, 86 adults with severe haemophilia (FVIII/IX<1%) were included. The median age was 38 years (range: 18-68) with 85% affected with haemophilia A and 75% using prophylaxis. Patients with haemophilia used task-oriented coping as frequently as the control group (P = 0.13); but used significantly less emotion-oriented coping (57% vs. 25%, P < 0.05) and avoidance coping (P < 0.05). Emotion-oriented coping showed a strong correlation with socio-psychological health (r = 0.67) and weak correlations with participation (r = 0.32) and social interaction (r = 0.29). Other associations of coping strategies with patient characteristics of health status could not be demonstrated. Overall, patients predominantly used the task-oriented approach to deal with their disease; the use of this strategy was comparable to the control group. Having a poor psychological health, less social interaction and/or less participation in daily life was associated with an increased use of emotion-oriented coping.
    Haemophilia 01/2014; DOI:10.1111/hae.12366 · 2.47 Impact Factor
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    ABSTRACT: Patients with inherited bleeding disorders are an interesting group to study the long-term course of chronic hepatitis C virus (HCV) infection, because of their uniform mode of infection and reliable follow-up. Our aim was to assess the long-term occurrence of adverse liver-related events in these patients. The occurrence and determinants of end-stage liver disease (ESLD) were assessed using retrospective data of 863 HCV infected patients with inherited bleeding disorders from the Netherlands and the UK. Median follow-up since HCV infection was 31 years, while 30% of patients had >35 follow-up years. Nineteen percent of patients spontaneously cleared the virus and 81% developed chronic HCV infection. Of the 700 patients with chronic HCV, 90 (13%) developed ESLD. Hepatocellular carcinoma (HCC) was diagnosed in 3% of patients with chronic HCV, 41% of which occurred in the last six years. Determinants of ESLD development were age at infection (hazard ratio (HR) 1.09 per year increase), HIV co-infection (HR 10.85), history of alcohol abuse (HR 4.34) and successful antiviral treatment (HR 0.14). Of the 487 patients who were still alive at the end of follow-up, 49% did not undergo optimal conventional antiviral treatment. After over 30 years of HCV infection, ESLD occurred in a significant proportion of patients with inherited bleeding disorders. HCC appears to be an increasing problem. There is a significant potential for both conventional and new antiviral treatment regimens to try and limit ESLD occurrence in the future.
    Journal of Hepatology 01/2014; 60(1):39-45. DOI:10.1016/j.jhep.2013.08.010 · 9.86 Impact Factor
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    ABSTRACT: To compare midurethral sling surgery and pelvic floor physiotherapy as initial treatment in women with moderate to severe urinary stress incontinence. Multicentre randomised trial. The study population was made up of women aged 35 to 80 years with moderate to severe stress incontinence. They received surgical treatment or pelvic floor physiotherapy, having been stratified according to incontinence severity. Crossover between groups was allowed during the follow-up period. Improvement at 12 months follow-up measured with the Patient Global Impression of Improvement score was the primary outcome measure (Dutch Trial Register number: NTR1248). We randomised 230 women to the sling surgery group and 230 to pelvic floor physiotherapy group. A total of 49% of women in the physiotherapy group and 11.2% of women in the surgery group crossed over to the alternative treatment. An improvement was reported by 90.8% of women in the surgery group and 64.4% of women in the physiotherapy group (absolute difference: 26.4 percentage points; 95% confidence interval: 18.1 to 34.5). A post hoc per-protocol analysis showed that women who underwent additional surgery after physiotherapy had similar outcomes to women initially assigned to surgery. The symptoms of women with moderate to severe stress incontinence were improved more with midurethral sling surgery than with pelvic floor exercises at 1 year.
    Nederlands tijdschrift voor geneeskunde 01/2014; 158:A7233.
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    ABSTRACT: Magnetic resonance imaging (MRI) scores for haemophilic arthropathy are useful for evaluation of early and moderate arthropathy. The most recent additive International Prophylaxis Study Group (IPSG) MRI scale for haemophilic arthropathy includes joint effusion. However, it is unknown whether joint effusion is haemophilia specific. Correct interpretation of joint effusion is needed for outcome assessment of prophylactic therapies in haemophilia care. The aim of this study was to compare joint effusion on MRI between young adults with haemophilia and healthy controls. MRI's of both knees and ankles of 26 haemophilic patients (104 joints) and 30 healthy active men (120 joints) were assessed. Scans in both groups were performed in 2009/2010 and 2012 respectively. Joint effusion was measured and scored according to the MRI atlas referred by the IPSG MRI scale for haemophilic arthropathy. Median age of haemophilic patients and healthy controls was 21 and 24 years respectively. In haemophilic patients 23% of knees and 22% of ankles showed joint effusion. Healthy controls had significantly more positive scores for knee effusion (67%, P < 0.01) and a comparable scores for effusion in the ankle (17%). Joint effusion according to criteria of the IPSG MRI scale was observed significantly more often in knees of healthy controls, while findings in ankles were similar. These data suggest that joint effusion in knees and ankles is not haemophilia specific. Inclusion of joint effusion in the MRI scale is expected to reduce its specificity for haemophilic arthropathy.
    Haemophilia 12/2013; DOI:10.1111/hae.12338 · 2.47 Impact Factor
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    ABSTRACT: To evaluate outcome of prophylactic clotting factor replacement in children with haemophilia, the Haemophilia Joint Health Score (HJHS) was developed aiming at scoring early joint changes in children aged 4-18. The HJHS has been used for adults on long-term prophylaxis but interpretation of small changes remains difficult. Some changes in these patients may be due to sports-related injuries. Evaluation of HJHS score in healthy adults playing sports could improve the interpretation of this score in haemophilic patients. The aim of this study was to evaluate the HJHS scores in a cohort of young, healthy men participating in sports. Concomitant with a project collecting MRI images of ankles and knees in normal young adults, HJHS scores were assessed in 30 healthy men aged 18-26, participating in sports one to three times per week. One physiotherapist assessed their clinical function using the HJHS 2.1. History of joint injuries was documented. MRI images were scored by a single radiologist, using the International Prophylaxis Study Group additive MRI score. Median age of the study group was 24.3 years (range 19.0-26.4) and median frequency of sports activities was three times per week (range 1-4). Six joints (five knees, one ankle) had a history of sports-related injury. The median overall HJHS score was 0 out of 124 (range 0-3), with 60% of subjects showing no abnormalities on HJHS. All joints were normal on MRI. These results suggest that frequent sports participation and related injuries are not related with abnormalities in HJHS scores.
    Haemophilia 10/2013; DOI:10.1111/hae.12290 · 2.47 Impact Factor
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    ABSTRACT: Physiotherapy involving pelvic-floor muscle training is advocated as first-line treatment for stress urinary incontinence; midurethral-sling surgery is generally recommended when physiotherapy is unsuccessful. Data are lacking from randomized trials comparing these two options as initial therapy. We performed a multicenter, randomized trial to compare physiotherapy and midurethral-sling surgery in women with stress urinary incontinence. Crossover between groups was allowed. The primary outcome was subjective improvement, measured by means of the Patient Global Impression of Improvement at 12 months. We randomly assigned 230 women to the surgery group and 230 women to the physiotherapy group. A total of 49.0% of women in the physiotherapy group and 11.2% of women in the surgery group crossed over to the alternative treatment. In an intention-to-treat analysis, subjective improvement was reported by 90.8% of women in the surgery group and 64.4% of women in the physiotherapy group (absolute difference, 26.4 percentage points; 95% confidence interval [CI], 18.1 to 34.5). The rates of subjective cure were 85.2% in the surgery group and 53.4% in the physiotherapy group (absolute difference, 31.8 percentage points; 95% CI, 22.6 to 40.3); rates of objective cure were 76.5% and 58.8%, respectively (absolute difference, 17.8 percentage points; 95% CI, 7.9 to 27.3). A post hoc per-protocol analysis showed that women who crossed over to the surgery group had outcomes similar to those of women initially assigned to surgery and that both these groups had outcomes superior to those of women who did not cross over to surgery. For women with stress urinary incontinence, initial midurethral-sling surgery, as compared with initial physiotherapy, results in higher rates of subjective improvement and subjective and objective cure at 1 year. (Funded by ZonMw, the Netherlands Organization for Health Research and Development; Dutch Trial Register number, NTR1248.).
    New England Journal of Medicine 09/2013; 369(12):1124-33. DOI:10.1056/NEJMoa1210627 · 54.42 Impact Factor
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    ABSTRACT: Prophylactic treatment in severe hemophilia is very effective but limited by cost-issues. The implementation of two different prophylactic regimens in the Netherlands and Sweden since the 1970s may be considered as a 'natural experiment'. We compared costs and outcome of Dutch intermediate- and Swedish high-dose prophylactic regimens for patients with severe hemophilia (FVIII/IX <1IU/dl) born 1970-1994 using prospective standardised outcome assessment and retrospective collection of cost data. 78 Dutch and 50 Swedish patients, median age 24 (range14-37) were included. Intermediate-dose prophylaxis used less factor concentrates [median (IQR) Netherlands 2100 IU/kg/yr (1400-2900) vs Sweden 4000 IU/kg/yr (3000-4900); p<0.01]. Clinical outcome was slightly inferior for the intermediate-dose regimen (p<0.01): 5-year bleeding [median 1.3 (0.8-2.7) vs 0 (0.0-2.0) joint bleeds/yr] and joint health [HJHS score over 10/144 points in 46% vs 11%], while social participation and quality of life (EQ-5D) were similar. Annual total costs were 66% higher for high-dose prophylaxis: mean 180 (95%CI 163-196)x1000 USD for Dutch vs 298 (95%CI 271-325)X1000 USD for Swedish patients (p<0.01). At group level, the incremental benefits of high dose prophylaxis appear limited. At patient level, prophylaxis should be tailored individually and many patients may do well on lower doses of concentrate without compromising safety.
    Blood 06/2013; DOI:10.1182/blood-2012-12-470898 · 9.78 Impact Factor
  • K Fischer, P de Kleijn
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    ABSTRACT: Outcome assessment in haemophilia is important to assess results of prophylactic treatment. Recently, the Haemophilia Joint Health Score (HJHS) was developed to assess early joint damage in children with haemophilia. Thus, the aim of this study was to assess reliability and explore validity of the HJHS in teenagers and young adults with haemophilia. Twenty-two patients with haemophilia (mean age 20.4, range 14-30, including 15 severe) were assessed by the HJHS1.0, Haemophilia Activities List (HAL), SF36 and self-evaluation was performed using a Visual Analogue Scale (VAS) scale. A subset of 12 patients were assessed by three physiotherapists to establish interobserver reliability (intraclass correlation coefficient: ICC). Total HJHS1.0 scores were calculated without overall global gait. Validity was explored by the assessment of Pearson's correlation with all outcome parameters and recent Pettersson scores. Overall outcome was good, with median HJHS score of 5.5 of a maximum 144 (range 0-34), median patients' VAS of 96.5 and maximum scores for HAL and SF36 physical functioning for the majority of patients. Pettersson scores were low (median 3.5 of 78, N = 18). Interobserver reliability was good (ICC 0.84), with limits of agreement of ±7.2 points. ICC was unaffected by different score calculation methods. Exploration of validity in 22 patients showed weak correlations of HJHS scores with patients' VAS (0.33) and HAL (-0.40) and strong correlations with SF36-PF (-0.66) and Pettersson scores (0.86). These results suggest that interobserver reliability of the HJHS1.0 in teenagers and young adults with limited joint damage is excellent. Preliminary data on validity were similar or better than those in children.
    Haemophilia 06/2013; DOI:10.1111/hae.12197 · 2.47 Impact Factor

Publication Stats

3k Citations
546.58 Total Impact Points


  • 2001–2014
    • University Medical Center Utrecht
      • • Department of Hematology
      • • Julius Center for Health Sciences and Primary Care
      Utrecht, Utrecht, Netherlands
  • 2007
    • University of Texas MD Anderson Cancer Center
      Houston, Texas, United States
  • 2002
    • Utrecht University
      Utrecht, Utrecht, Netherlands