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- Berntorp, Erik, et al.
(författare)
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Treatment of haemophilia A and B and von Willebrand's disease : summary and conclusions of a systematic review as part of a Swedish health-technology assessment
- 2012
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Ingår i: Haemophilia. - : Wiley. - 1351-8216 .- 1365-2516. ; 18:2, s. 158-165
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Forskningsöversikt (refereegranskat)abstract
- In an ongoing health-technology assessment of haemophilia treatment in Sweden, performed by the governmental agency Dental and Pharmaceutical Benefits Agency (TLV; tandvårds-och läkemedelsförmånsverket), the Swedish Council on Health Technology Assessment (SBU; statens beredning för medicinsk utvärdering) was called upon to evaluate treatment of haemophilia A and B and von Willebrand's disease (VWD) with clotting factor concentrates. To evaluate the following questions: What are the short-term and long-term effects of different treatment strategies? What methods are available to treat haemophilia patients that have developed inhibitors against factor concentrates? Based on the questions addressed by the project, a systematic database search was conducted in PubMed, NHSEED, Cochrane Library, EMBASE and other relevant databases. The literature search covered all studies in the field published from 1985 up to the spring of 2010. In most instances, the scientific evidence is insufficient for the questions raised in the review. Concentrates of coagulation factors have good haemostatic effects on acute bleeding and surgical intervention in haemophilia A and B and VWD, but conclusions cannot be drawn about possible differences in the effects of different dosing strategies for acute bleeding and surgery. Prophylaxis initiated at a young age can prevent future joint damage in persons with haemophilia. The available treatment options for inhibitors have been insufficiently assessed. The economic consequences of various treatment regimens have been insufficiently analysed. Introduction of national and international registries is important.
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- Brodin, Elisabeth, et al.
(författare)
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The Swedish version of the Haemophilia Activity List.
- 2011
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Ingår i: Haemophilia : the official journal of the World Federation of Hemophilia. - : Wiley. - 1365-2516. ; 17:4, s. 662-8
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Tidskriftsartikel (refereegranskat)abstract
- There has been increasing interest in the patient's perspective on outcome of treatment. The Haemophilia Activity List (HAL) has been developed as a disease-specific questionnaire for haemophilia patients and is a validated self-report measure of function developed according to WHO's International Classification of Functioning, Disability and Health. To validate HAL in Sweden. The Dutch and English versions of HAL were translated into Swedish using 'the forward-backward translation' method and merged into a final Swedish version. Validation was performed against the Swedish version of the questionnaires Arthritis Impact Measurement 2 (AIMS 2) and Impact on Participation and Autonomy (IPA). Two hundred and twenty-five patients with severe and moderate forms of haemophilia A and B from three centres were invited to participate in the study. Spearman's rank correlation test was used for validation, and internal consistency of the HAL was calculated with Cronbach's alpha. Eighty-four patients (39%) (18-80 years old) filled out the questionnaires. The internal consistency of the Swedish version of HAL was high, with Cronbach's alpha being 0.98-0.71. Function of the legs had the highest consistency and transportation had the lowest. The correlation was excellent between the HAL sum score and AIMS 2 physical (r = 0.84, P< 0.01), IPA autonomy indoors (r = 0.83, P < 0.01) and autonomy outdoors (r = 0.89, P < 0.01). The Swedish version of HAL has both internal consistency and convergent validity and may complement other functional tests to gather information on the patient's self-perceived ability.
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- Kihlberg, Kristina, et al.
(författare)
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Factor IX antibodies and tolerance in hemophilia B in the Nordic countries - The impact of F9 variants and complications
- 2022
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Ingår i: Thrombosis Research. - : Elsevier BV. - 0049-3848 .- 1879-2472. ; 217, s. 22-32
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Tidskriftsartikel (refereegranskat)abstract
- Introduction: The development of inhibitory antibodies (inhibitors) in persons with hemophilia B (PwHB) causes significant morbidity. Data on the impact of the F9 variant and immune tolerance induction (ITI) outcome are limited.The aim of this study was to investigate the presence of neutralizing and non-neutralizing antibodies (NNA) in severe hemophilia B (HB) and to evaluate ITI outcome and complications in relation to the pathogenic F9 variant.Materials and methods: Persons with severe HB in the Nordic countries were enrolled and information on F9 variants, inhibitors, ITI and complications were collected. Analyses of anti-FIX antibodies with a fluorescence -immunoassay (xFLI) and an ELISA method were conducted.Results: Seventy-nine PwHB were enrolled. Null variants were seen in 33 (42 %) PwHB and 12 (15 %) had a current or former inhibitor. Eleven (92 %) of the inhibitor patients had experienced allergic manifestations and three (25 %) nephrotic syndrome. Of 10 PwHB with at least one ITI attempt, eight (80 %) were considered tolerant at enrolment. Immunosuppression was included in seven of eight successful or partially successful at-tempts. Five PwHB had at least one ITI failure before a successful or partially successful ITI. No NNA could be identified.Conclusion: A high proportion of severe F9 gene defects among persons with severe HB in the Nordic countries may explain the observed relatively high prevalence of inhibitors. ITI success was independent of the F9 variant and attained despite allergic manifestations and previous ITI failures. Inclusion of immunosuppression tenta-tively enhances the chances of ITI success. No NNA were observed.
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| 11. |
- Kihlberg, Kristina, et al.
(författare)
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No difference in quality of life between persons with severe haemophilia A and B
- 2023
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Ingår i: Haemophilia. - : Wiley. - 1351-8216 .- 1365-2516. ; 29:4, s. 987-996
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Tidskriftsartikel (refereegranskat)abstract
- IntroductionGood health-related quality of life (HRQoL) is an important goal in the treatment of persons with haemophilia B (PwHB). Studies focusing on this population are limited, however, and data are insufficient. AimTo assess the HRQoL in PwHB and to compare this to data on persons with haemophilia A (PwHA), as well as to evaluate the impact of joint health on HRQoL and to identify areas of insufficient care. MethodsThe B-NORD study enrolled persons with severe haemophilia B and matched controls with haemophilia A. HRQoL was assessed using the EQ-5D-3L questionnaire and joint health using Haemophilia Joint Health Score 2.1 (HJHS). ResultsThe EQ-5D-3L was completed by 63 PwHB and 63 PwHA. Mobility problems were reported by 46% of PwHB and 44% of PwHA, pain/discomfort by 62% and 56%, and anxiety/depression by 33% and 17%, respectively. No significant difference was observed between PwHA and PwHB in EQ-5D profiles, level sum score, EQ-5D index (PwHB mean .80, PwHA mean .83, p = .24), or EQ VAS score (PwHB: mean 70, PwHA: mean 77, p = .061). Linear regression adjusted for age demonstrated that an increase in HJHS score was associated with a significant decrease in both EQ-5D index (B -.003, R-2 .22) and EQ VAS score (B -.37, R-2 .17). ConclusionDespite the majority of patients being treated with prophylaxis, impaired HRQoL was reported in both PwHB and PwHA. No differences in HRQoL were found between the two groups. Impaired joint health had a significant negative impact on HRQoL.
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- Lassila, Riitta, et al.
(författare)
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Practical Viewpoints on the Diagnosis and Management of Heparin-Induced Thrombocytopenia
- 2011
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Ingår i: Seminars in Thrombosis and Hemostasis. - : Georg Thieme Verlag KG. - 1098-9064 .- 0094-6176. ; 37:3, s. 328-335
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Tidskriftsartikel (refereegranskat)abstract
- Heparin-induced thrombocytopenia (HIT, type II) is an immune-mediated disorder due to antibodies formed against heparin platelet factor 4 complexes, usually appearing at days 5 to 14 after initiation of heparin. It is important to recognize HIT because heparin prophylaxis or treatment paradoxically associates with new venous and/or arterial thrombosis. Early clinical suspicion and diagnosis together with proper pharmacotherapy and close laboratory monitoring are the cornerstones for successful management. This includes monitoring of Thrombocytopenia, its Timing to heparin administration, appearance of new Thrombosis or resistance to treatment, and differential diagnosis by exclusion of oTher causes (the 4T's). Specific attention should be paid to the absence or presence of thrombosis and to tailoring thromboprophylaxis or anticoagulant therapy with a nonheparin alternative. Even in the absence of HIT-associated thrombosis, an active policy for prolonged thromboprophylaxis is demanded. Rapid and reliable assays should be developed for diagnosis and anticoagulation monitoring to secure safe management with nonheparins. Semiquantitative testing for on-call hours should be available and later confirmed as clinically needed. Alternative therapeutic options are available, but because their use is infrequent, experienced coagulation treatment centers should provide guidance in the treatment and in laboratory monitoring. Most of the evidence in HIT is grade IC, and thus the best evidence is provided by clinical experience. New anticoagulants and platelet inhibitors may offer future alternatives in the management of HIT, but the current treatment options provide the best experience and benefit. The joint clinical and laboratory guidelines provided in this article along with two practical case scenarios were prepared by a Nordic expert panel. They will be valuable for hematologists and colleagues who do not routinely encounter HIT.
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- Lehtinen, A. E., et al.
(författare)
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Surgical outcomes in patients with haemophilia A or B receiving extended half-life recombinant factor VIII and IX Fc fusion proteins: Real-world experience in the Nordic countries
- 2022
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Ingår i: Haemophilia. - : Wiley. - 1351-8216 .- 1365-2516. ; 28:5, s. 713-719
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Tidskriftsartikel (refereegranskat)abstract
- Introduction Perioperative dosing recommendations vary across Nordic haemophilia treatment centres (HTCs) for extended half-life (EHL) factor concentrates in haemophilia A/B (HA/HB) patients. Aim To summarise Nordic real-world surgical experiences with EHL recombinant factor VIII/IX Fc (rFVIIIFc/rFIXFc) fusion proteins using retrospective data from clinical records at four HTCs in Finland, Sweden and Norway. Methods Factor dosing and surgical outcomes were recorded from HA/HB patients who underwent surgery and were treated with rFVIIIFc/rFIXFc. Perioperative factor dosing regimens were clinician-determined based on local practises. Results Twenty five surgeries were performed on 20 patients, all covered by bolus injections except one minor HA surgery; eight minor surgeries were in paediatric patients. Median preoperative rFVIIIFc dose for major HA surgeries (n = 8) was 48 IU/kg (range: 35-57), with total consumption up to Day 14 of 427 IU/kg (196-568). For the two major HB surgeries (in one patient), preoperative rFIXFc doses were 50 IU/kg and 20 IU/kg; total consumption up to Day 14 was 130 IU/kg and 40 IU/kg. Median preoperative rFVIIIFc/rFIXFc bolus doses for minor HA (n = 10) and HB (n = 4) surgeries were 50 IU/kg (24-79) and 47 IU/kg (40-71), with total consumption up to Day 5 of 138 IU/kg (49-404) and 100 IU/kg (43-125), respectively. Intraoperative and postoperative haemostatic responses were rated as at least good/excellent for 24/25 surgeries, with bleeding episodes reported in only three surgeries. Conclusion Nordic real-world experiences suggest that EHL products can be used safely and effectively for peri-operative haemostasis. Further research is required to develop local dosing guidelines for optimised treatment schedules.
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- Osooli, M, et al.
(författare)
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The association between health utility and joint status among people with severe haemophilia A : findings from the KAPPA register
- 2017
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Ingår i: Haemophilia. - : Wiley. - 1351-8216. ; 23:3, s. E180-E187
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Tidskriftsartikel (refereegranskat)abstract
- INTRODUCTION: People with severe haemophilia A have reportedly impaired health related quality of life (utility) mainly due to recurrent bleeding, arthropathy and treatment burden.AIM: To estimate utilities and evaluate their potential correlates - most importantly the joint status - among people with severe haemophilia A.METHODS: In this cross-sectional study, eligible participants had severe haemophilia A, were aged ≥15, negative for factor VIII inhibitor and included in the KAPPA register of Denmark, Norway and Sweden. Data on demographics, treatment history, haemophilia joint health score, and EQ-5D utility were obtained from the register. We used box plots to present utilities and joint status and ordinary least squares regression to evaluate correlates of utilities. Participants were consecutively enrolled in the KAPPA register between April 2013 and June 2016.RESULTS: Overall, 173 participants with median age of 34 (interquartile range: 25-45) were included. Twelve (6.9%) participants were on episodic treatment while 161 (93.1%) were treated using prophylaxis. Concomitant diseases and positive inhibitor history were reported for 73 (43.2%) and 21 (12.1%) participants, respectively. The highest median utility (1.0) was observed among those aged <29 on prophylaxis and those aged 30-44 who had started prophylaxis by age 3. In the multi-variable regression, joint scores of 16-25 (Coef. -0.18, 95% CI: -0.30, -0.06), 26-35 (Coef. -0.21, 95% CI: -0.36, -0.06) and >35 (Coef. -0.37, 95% CI: -0.52, -0.23) were associated with lower utilities.CONCLUSION: Moderate to severe joint manifestations are associated with reduced utilities among persons with severe haemophilia A.
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- Steen Carlsson, K, et al.
(författare)
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People with Haemophilia and Female Carriers in Sweden have a Higher Risk of Developing Anxiety, Depression and Pain Based on Treatment Patterns as Compared to Matched Controls: Data from a Registry Study over a Period of 11 Years
- 2019
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Ingår i: ISTH 2019 abstract OC 32.3.
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Konferensbidrag (refereegranskat)abstract
- Background: People with haemophilia (PwH) have increased risk of bleeds associated with acute and chronic pain, and long-term disability. Anxiety and depression are other determinants negatively affecting quality of life in PwH. Despite the severity of these co-morbidities, they have not been extensively investigated and associations between them even less. Aims: The MIND study (NCT03276130) aims to identify patterns of prescribed pain, anti-depressive and anti-anxiety medication and management of pain, depression and anxiety in PwH in four Nordic countries, based on patient registry data (A) and surveys (B). Here we describe current and retrospective treatment patterns of prescribed medication (part A) in PwH in Sweden, explore associations with the PwH background and complication characteristics, and compare prescribed medication use between PwH and the general population. Methods: This 11-year (2007-2017) retrospective population-based registry study, uses international classification systems for diagnoses, surgeries and medications. The study population includes all ages and was identified by diagnosis of haemophilia A or B, or at least one prescription of any factor VIII, IX, or bypassing agent. For each study subject, five age and gender-matched controls were included. Results: Data were extracted for 1550 PwH. The study population is shown in figure 1. The probability of being prescribed with analgesics, neuroleptics and anti-depressants in PwH compared to controls are shown in Table 1. Conclusions : A substantially higher prescription of analgesics in Swedish PwH as compared to controls suggests a need for increased focus on pain management including improved bleed protection and prevention of, e.g. arthropathy. The increased prescription of neurolep-tics and anti- depressants demonstrates that anxiety and depression are overrepresented in PwH and underscores the importance of identifying and managing the broad PwH population including non- frequent health care users. A similar prescription pattern was seen in female carriers suggesting a need for high medical attention and further research to address knowledge gaps.
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