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1.
Haemophilia ; 23(5): e403-e408, 2017 Sep.
Article in English | MEDLINE | ID: mdl-28758324

ABSTRACT

BACKGROUND: Persons with severe haemophilia require lifelong replacement therapy, prophylaxis, to prevent bleeding. Data describing long-term outcomes of prophylactic treatment are scarce. The aim of this study was to investigate joint surgery and survival among persons with severe haemophilia with special attention to access to prophylaxis in the early years of life. METHODS: Eligible participants had severe haemophilia A or B and were treated at the Malmö centre from the 1960s onward. Time from birth until joint surgery was analysed for participants negative for factor inhibitor and alive in 2000. We compared survival among the entire cohort with severe haemophilia treated at the Malmö centre with the general male population of Sweden and a sample of persons with severe haemophilia from the United Kingdom (UK). RESULTS: Overall, 167 participants were included, 106 (63.5%) of whom had complete data on joint surgery. Among those born before 1970, 1970-1979 and ≥1980 approximately 37%, 21% and 0% had their first joint surgery by age 30, respectively. There were no second joint surgeries reported in cohorts born ≥1970. Persons with severe haemophilia and negative for HIV treated in Malmö have attained approximately similar survival to that of the general male population in Sweden and live slightly longer than persons with severe haemophilia from the UK. DISCUSSION AND CONCLUSION: Prophylaxis in Sweden, although costly, has markedly improved survival and joint outcomes for persons with severe haemophilia. This study highlights the importance of early start of replacement therapy to prevent or postpone serious joint damage.


Subject(s)
Hemarthrosis/surgery , Hemophilia A/mortality , Hemophilia B/mortality , Adolescent , Adult , Aged , Aged, 80 and over , Child , Child, Preschool , Cohort Studies , Factor IX/drug effects , Factor VIII/drug effects , Hemarthrosis/etiology , Hemophilia A/complications , Hemophilia A/drug therapy , Hemophilia A/epidemiology , Hemophilia B/complications , Hemophilia B/drug therapy , Hemophilia B/epidemiology , Humans , Infant , Infant, Newborn , Kaplan-Meier Estimate , Male , Middle Aged , Registries , Severity of Illness Index , Sweden/epidemiology , Treatment Outcome , United Kingdom/epidemiology , Young Adult
2.
Haemophilia ; 23(3): e180-e187, 2017 May.
Article in English | MEDLINE | ID: mdl-28393468

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.


Subject(s)
Hemophilia A/complications , Joint Diseases/complications , Joint Diseases/prevention & control , Quality of Life , Registries , Adolescent , Adult , Child, Preschool , Cross-Sectional Studies , Female , Hemophilia A/epidemiology , Hemophilia A/therapy , Humans , Male , Middle Aged , Phenotype , Scandinavian and Nordic Countries/epidemiology , Young Adult
3.
Haemophilia ; 23(2): e79-e86, 2017 Mar.
Article in English | MEDLINE | ID: mdl-28155262

ABSTRACT

INTRODUCTION: Mild haemophilia is a congenital bleeding disorder affecting males. The burden of arthropathy in mild haemophilia has not been comprehensively described. AIM: The aim of this study was to compare the incidence, age at diagnosis and surgery for arthropathy and related hospitalizations between people with mild haemophilia and the general population in Sweden. METHODS: This was a register-based cohort study. Eligible participants were those with mild haemophilia born between 1941 and 2008 and a randomly selected, birthdate and sex-matched comparison group from the general population. Follow-up was from birth (or earliest 1984) until death, emigration or end of the study in 2008. Data on arthropathy were obtained from a national patient register. Negative binomial and competing risk regression and Kaplan-Meier estimate curves were used in the analysis. RESULTS: Overall, 315 people with haemophilia and 1529 people in the comparison group were included. Participants with haemophilia born between 1984 and 2008 had a ninefold (95% CI: 3.3-27.2) and 16-fold (95% CI: 6.7-36.5) increased incidence of arthropathy-related hospital admission and arthropathy diagnosis respectively. None in this cohort underwent surgery. Among participants with haemophilia born prior to 1984, the rates of arthropathy diagnosis and surgery of the index joints (knee, elbow, ankle) were increased twofold (95% CI: 1.0-3.2) and fivefold (95% CI: 1.7-17.8) respectively. CONCLUSION: Our data suggested a higher burden of arthropathy among individuals with mild haemophilia compared to the general population. Further research should investigate the need for targeted joint screening programmes among individuals with mild haemophilia.


Subject(s)
Hemophilia A/complications , Hemophilia B/complications , Joint Diseases/etiology , Cohort Studies , Female , Hemophilia A/mortality , Hemophilia A/pathology , Hemophilia B/mortality , Hemophilia B/pathology , Humans , Male , Sweden
4.
J Intern Med ; 279(6): 502-14, 2016 Jun.
Article in English | MEDLINE | ID: mdl-26999367

ABSTRACT

Haemophilia is a congenital disorder with bleeding episodes as its primary symptom. These episodes can result in negative outcomes including joint damage, loss of active days due to hospitalization and reduced quality of life. Effective treatment, however, can improve the outcome. Registries have been used as a valuable source of information regarding the monitoring of treatment and outcome. The two main aims of this exploratory study were to establish which haemophilia registries publish peer-reviewed outcome assessment research and then to extract, classify and report the treatment outcomes and their extent of use in the retrieved registries. Using relevant keywords, we searched PubMed and Web of Science databases for publications during the period 1990-2015. Retrieved references were screened in a stepwise process. Eligible papers were original full articles on haemophilia outcomes that used data from a computerized patient database. Descriptive results were summarized. Of 2352 references reviewed, 25 full texts were eligible for inclusion in the study. These papers were published by 11 registries ranging from local to international in coverage. It is still relatively rare for registries to produce peer-reviewed publications about outcomes, and most that currently do produce such papers are located in Europe and North America. More information is available on traditional outcomes such as comorbidities and arthropathy than on health-related quality of life or the social and developmental impact of haemophilia on patients. Inhibitors, HIV and viral hepatitis are amongst the most commonly reported comorbidities. Research has focused more on factor consumption and less on hospitalization or time lost at school or work due to haemophilia. Haemophilia registries, especially those at the national level, are valuable resources for the delivery of effective health care to patients. Validated outcome measurement instruments are essential for the production of reliable and accurate evidence. Finally, such evidence should be communicated to physicians, patients, the public and health policymakers.


Subject(s)
Evidence-Based Medicine , Hemophilia A/therapy , Hemophilia B/therapy , Registries/standards , Comorbidity , Cost of Illness , Hemophilia A/complications , Hemophilia A/mortality , Hemophilia B/complications , Hemophilia B/mortality , Humans , Joint Diseases/etiology , Peer Review , Quality of Life , Treatment Outcome
5.
J Intern Med ; 277(1): 1-15, 2015 Jan.
Article in English | MEDLINE | ID: mdl-25169114

ABSTRACT

Congenital haemophilia A and B are genetic disorders affecting factor VIII and factor IX production, respectively. Factor replacement is the only effective treatment for these deficiencies, but a patient's immune system can develop inhibitory antibodies which bind and interfere with the function of the replaced factor in a variety of ways. The main treatment goal for patients with inhibitors is to induce immune tolerance to the injected factor. If not successful, a different treatment termed bypass therapy is needed to treat bleeds. The goal of this review is to demonstrate the usefulness of haemophilia registries as information sources to supplement available evidence regarding predictors of inhibitor development and immune tolerance induction (ITI) outcomes. In this systematic review, relevant keywords were used to search online academic databases during February 2014. Inclusion criteria were original publication and data obtained from a haemophilia or ITI registry with a minimum of 30 patients. A data collection form was created to extract information from selected manuscripts. Titles, abstracts and then full texts were screened to determine the eligibility of reports for this review. Eleven manuscripts from nine registries were determined eligible and included in the study. Registries have reported on some core variables, but are inconsistent in reporting less practiced predicting variables. Variables that may affect inhibitor and ITI outcomes were each divided into two categories: patient characteristics (such as age and family history) and treatment-related variables (including exposure days, treatment duration and dose). It is recommended that, in addition to exploratory hypothesis testing, a minimum set of variables should be collected and reported by registries. International collaboration and well-designed prospective registries are of major importance to advance this field in order to determine inhibitor risks and ITI outcomes and facilitate the development of new treatments.


Subject(s)
Factor VIII/antagonists & inhibitors , Factor VIII/therapeutic use , Hemophilia A/drug therapy , Hemophilia B/drug therapy , Registries , Age Factors , Child , Evidence-Based Medicine , Female , Follow-Up Studies , Hemophilia A/genetics , Hemophilia A/immunology , Hemophilia B/genetics , Hemophilia B/immunology , Humans , Immune Tolerance/drug effects , Infant , Infant, Newborn , International Cooperation , Male , Risk Assessment , Severity of Illness Index , Sex Factors , Time Factors , Treatment Outcome
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