Prophylaxis usage, bleeding rates, and joint outcomes of hemophilia, 1999 to 2010: a surveillance project

Johnson, Marilyn; Soucie, J. Michael; Gill, Joan Cox

doi:10.1182/blood-2016-02-683169

Cited by 141 publications

(145 citation statements)

References 18 publications

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“…Our results regarding the increased use of prophylactic treatment and a decreased risk of bleeding concur with large-scale, prospective studies in The Netherlands [20] and the USA [22]. These studies also found reduced joint mobility in older age groups.…”

Section: Discussionsupporting

confidence: 89%

“…However, little change was observed in self-reported impairment of joint function among PWH > 16 years [20]. Finally, a large register-based study evaluated trends from 1999 to 2010 in bleeding rates and joint outcomes among 6,196 PWH in the USA [22]. This impressive study found increased use of prophylactic treatment and a much lower frequency of bleeding in PWH receiving prophylactic rather than on-demand treatment, but also a decline in bleeding rate over time for both groups [22].…”

Section: Introductionmentioning

confidence: 99%

“…Finally, a large register-based study evaluated trends from 1999 to 2010 in bleeding rates and joint outcomes among 6,196 PWH in the USA [22]. This impressive study found increased use of prophylactic treatment and a much lower frequency of bleeding in PWH receiving prophylactic rather than on-demand treatment, but also a decline in bleeding rate over time for both groups [22]. Some improvement in joint range of motion was observed between 1999 and 2010 in the age group of 20–49 years, but no overall difference was seen between the group receiving prophylactic treatment and the group receiving treatment on demand.…”

Section: Introductionmentioning

confidence: 99%

“…Some improvement in joint range of motion was observed between 1999 and 2010 in the age group of 20–49 years, but no overall difference was seen between the group receiving prophylactic treatment and the group receiving treatment on demand. However, in a separate analysis of 2,908 PWH, for which repeated assessments were available, prophylactic treatment initiated before the age of 4 years predicted a lower rate of decline in the range of motion of the joints [22]. …”

Section: Introductionmentioning

confidence: 99%

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Joint Mobility and Physical Function of Danish Hemophilia Patients: A Three-Wave Panel Study Spanning 24 Years

et al. 2018

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Background: The positive effects of factor treatment of hemophilia are well established, but the long-term outcomes are not well documented. This panel study evaluated changes in bleeding frequency, joint mobility, physical function, and symptoms in Danish patients with moderate to severe hemophilia A or B over 24 years. Methods: Three anonymous surveys were conducted in 1988, 2001, and 2012 targeting Danish patients with moderate to severe hemophilia, and the study participants, respectively, were 128, 156, and 164 male patients with hemophilia (PWH). The number of bleeding episodes, the use of factor concentrate, comorbidities, joint mobility, physical function, and symptoms were evaluated by means of self-reporting. Trends over time were analyzed using ordinal and multinomial logistic-regression models controlling for age group. Results: The proportion of PWH in the oldest age group (55–88 years) increased from 4% in 1988 to 18% in 2012. In 1988, a high risk of bleeding episodes was primarily found in the age group of 16–34 years. In 2012, a high risk was primarily found in the age group of 35–54 years. Joint mobility and physical function increased significantly from 1988 to 2012 but showed a noticeable decrement in the older age groups, even in 2012. Pain in the extremities, anxiety, and depression decreased significantly, but back pain increased. No significant changes were found for 7 other symptoms. Conclusions: Significant improvements in joint mobility and physical function have occurred over the last 24 years, but PWH > 35 years still experience a decline in these areas with age. This decline underscores the importance of life-long treatment and continuous rehabilitation of PWH.

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Section: Discussionsupporting

confidence: 89%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Joint Mobility and Physical Function of Danish Hemophilia Patients: A Three-Wave Panel Study Spanning 24 Years

et al. 2018

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“…However, secondary prophylaxis (initiated post-target joint development) may not afford the same degree of benefit in terms of mobility. Consistent with this observation, a longitudinal US HTC study also showed that although prophylaxis significantly predicted decreased bleeding at any age, only prophylaxis initiation prior to age 4 (and absent obesity) predicted preservation of joint motion 32. Consistent with this observation, a longitudinal US HTC study also showed that although prophylaxis significantly predicted decreased bleeding at any age, only prophylaxis initiation prior to age 4 (and absent obesity) predicted preservation of joint motion 32.…”

supporting

confidence: 63%

Achieving the unimaginable: Health equity in haemophilia

et al. 2019

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Haemophilia. 2020;26:17-24.wileyonlinelibrary.com/journal/hae | 17 | INTRODUC TI ONCurrent prophylaxis protocols for haemophilia A are designed conservatively, with a minimal goal of achieving factor VIII (FVIII) trough levels of ≥1%. This threshold was originally selected based on generally favourable outcomes from early cryoprecipitate studies that targeted trough FVIII levels of 1%-3%, supportive evidence that moderate haemophilia was associated with fewer bleeds and better preserved joint function than severe haemophilia, and limitations in plasma resources at the time. 1-3 However, persons with haemophilia (PwH) who are maintained at 1% FVIII continue to experience spontaneous bleeding and remain at higher risk for injury with daily activities. 4 Furthermore, current prophylaxis regimens do not prevent haemophilic arthropathy over a PwH's lifetime. 5 With the growing availability of current therapies, the validity of the 1% threshold has come under scrutiny, accompanied by the proposal to target higher thresholds ≥15%. 6 Recent success in clinical development of haemophilia therapies opens an era of new possibilities and holds great promise for people Abstract Historically, treatment based on the availability of clotting factor replacement has resulted in an arcane guideline for the correction of factor deficiencies in people with haemophilia (PwH). While all other disease entities seek to restore function to a normal level, PwH are restricted to factor nadirs still equivalent to mild or moderate disease, resulting in continued risk of bleeding. A new treatment paradigm is needed based on the defined needs of PwH. A treatment model was developed by a panel of haemophilia providers, patient advocates and health economists to establish specific treatment milestones and targeted outcomes. The panel defined a series of treatment milestones to characterize the activity and outcomes linked to level of factor deficiency correction. All agreed that the ultimate goal should be 'functional cure' and 'health equity'. Seven levels to achieving a functional cure were identified, (a) Sustain life; (b) Minimal joint impairment; (c) Freedom from any spontaneous bleeds;(d) Attainment of 'normal' mobility; (e) Able to sustain minor trauma without additional intervention; (f) Ability to sustain major surgery or trauma; and (g) Normal haemostasis. A parallel set of patient-reported outcomes to achieve health equity was identified. These guidelines are now comparable with other disorders where the goal is to replace missing proteins to attain normal activity levels. As we are no longer limited by plasma supply due to the manufacture of recombinant factors, mimetics, and the early success of gene therapy, health equity is now achievable. K E Y W O R D Shaemophilia, outcomes, prophylaxis, replacement factor How to cite this article: Skinner MW, Nugent D, Wilton P, et al.Achieving the unimaginable: Health equity in haemophilia.

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Community counts: Evolution of a national surveillance system for bleeding disorders

et al. 2018

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Prophylaxis usage, bleeding rates, and joint outcomes of hemophilia, 1999 to 2010: a surveillance project

Cited by 141 publications

References 18 publications

Joint Mobility and Physical Function of Danish Hemophilia Patients: A Three-Wave Panel Study Spanning 24 Years

Joint Mobility and Physical Function of Danish Hemophilia Patients: A Three-Wave Panel Study Spanning 24 Years

Achieving the unimaginable: Health equity in haemophilia

Community counts: Evolution of a national surveillance system for bleeding disorders

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