Changes in health‐related quality of life with treatment of longer‐acting clotting factors: results in the A‐<scp>LONG</scp> and B‐<scp>LONG</scp> clinical studies

Wyrwich, Kathleen W.; Krishnan, Sangeeta; Auguste, Priscilla; Poon, Jiat Ling; Maltzahn, Robyn von; Yu, R.; Pierce, Glenn F.; Mei, Baisong; Mahlangu, Johnny; Mackensen, Sylvia von

doi:10.1111/hae.12987

Cited by 40 publications

(60 citation statements)

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“…When we compare haemophilia A and B, we can see that adult patients have similar bleeding rates and HRQoL . Similar to results in our study, paediatric patients with haemophilia A that switched to BAX 855, a glycopegylated rFVIII, saw an improvement in HRQoL in all domains of the Paediatric Quality of Life Inventory.…”

Section: Discussionsupporting

confidence: 86%

“…patients have similar bleeding rates 9 and HRQoL. 27 Similar to results in our study, paediatric patients with haemophilia A that switched to BAX 855, a glycopegylated rFVIII, saw an improvement in HRQoL in all domains of the Paediatric Quality of Life Inventory. However, this did not reach significance in the youngest cohort.…”

Section: Hrqol Was Assessed Using the Haem-a-qol And Hrqol Respondersupporting

confidence: 88%

“…Greater improvements were seen in patients switching from on-demand to prophylactic treatment. 27 Although this study is in adults, and not directly comparable, it will be interesting to see if the improvement in HRQoL seen with rIX-FP is maintained in adult and adolescent patients to a similar or greater degree than seen with other long-acting factors.…”

Section: Hrqol Was Assessed Using the Haem-a-qol And Hrqol Respondermentioning

confidence: 91%

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Health‐related quality of life in paediatric haemophilia B patients treated with rIX‐FP

et al. 2018

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IntroductionFrequent infusions and bleeds can impact on the health‐related quality of life (HRQoL) of paediatric haemophilia B patients. rIX‐FP (IDELVION®) is a fusion protein linking recombinant factor IX with recombinant albumin, and is associated with low bleeding rates with a weekly regimen, which could improve HRQoL.AimsTo measure the effect of rIX‐FP prophylaxis on the HRQoL of paediatric patients and treatment satisfaction in their caregivers using the Haemo‐QoL and Hemo‐SATP questionnaires, respectively.MethodsAt baseline and end‐of‐study (EOS), patients 4‐11 years old participating in the PROLONG‐9FP program answered the Haemo‐QoL questionnaire and gave information on their socio‐demographic data and physical activity. Caregivers completed the Hemo‐SatP. Minimal important differences (MID) (|Cohen's d| > 0.5) between baseline and EOS and the number of responders (patients with meaningful subject‐level improvements over time) at EOS were calculated.ResultsTwenty patients (age group I: 4‐7 years old [n = 12]; age group II: 8‐12 years old [n = 8]) completed the Haemo‐QoL questionnaire at baseline. MIDs were found in age group I representing improvement for “physical health” (d = −0.547) domain; 60% of patients were responders for “physical health.” In age group II, MIDs were seen in most domains; 71.4% patients were responders in “total score.” In caregivers, improvements were seen for most domains of the Hemo‐SatP with a small effect size. Fewer patients missed school when treated with rIX‐FP and 94.1% patients maintained their physical activity level.ConclusionProphylaxis with rIX‐FP led to substantial improvements in HRQoL in paediatric patients and treatment satisfaction in caregivers.

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Section: Discussionsupporting

confidence: 86%

Section: Hrqol Was Assessed Using the Haem-a-qol And Hrqol Respondersupporting

confidence: 88%

Section: Hrqol Was Assessed Using the Haem-a-qol And Hrqol Respondermentioning

confidence: 91%

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Health‐related quality of life in paediatric haemophilia B patients treated with rIX‐FP

et al. 2018

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“…12 Clinical trial outcomes have shown for both EHL recombinant FVIII and FIX drugs that prophylaxis regimens are associated with very low annualized bleeding rates (ABRs) compared to on-demand treatment. 17,18 EHL products have been licensed at different times in the USA and Europe. 17,18 EHL products have been licensed at different times in the USA and Europe.…”

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confidence: 99%

Real‐life experience in switching to new extended half‐life products at European haemophilia centres

et al. 2019

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The concept of replacement therapy in haemophilia is changing significantly thanks to the switch from standard products to extended half‐life products. These novel drugs are showing beneficial effects overcoming current prophylaxis limitations by reducing the infusion frequency, maintaining a higher trough level to ensure a lower risk of bleeding, and making treatment significantly less distressing to patients by improving the quality of life. Real‐life data on the efficacy of novel drugs and their impact on routine management of haemophilia A and B patients are still limited. This manuscript reports the results of a European survey conducted by the European Association for Haemophilia and Allied Disorders (EAHAD) at the beginning of 2018 on the clinical management of patients using extended half‐life recombinant FVIII and FIX fusion products, since at the time of the survey none of the PEGylated products were available yet. We report data on the efficacy of these novel drugs by 33 European haemophilia centres that have already switched to extended half‐life fusion products, showing a significant reduction in the number of infusions and a satisfactory trough levels in the clinical care of haemophilia patients, with a greater impact for haemophilia B.

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“…37 Studies of primary prophylaxis have shown that normal or nearnormal activity with respect to joint motion and school or work attendance is achievable. Patients on prophylaxis maintain or improve physical activity levels, compared with pretreatment levels [38][39][40][41][42] and achieve similar activity levels as those with mild or moderate haemophilia. 43 Prophylaxis also enables sports participation, which in turn contributes to improved fitness, health satisfaction and psychosocial wellness.…”

Section: Comparison Between Workers With Vs Without Haemophilia Hasmentioning

confidence: 99%

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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Changes in health‐related quality of life with treatment of longer‐acting clotting factors: results in the A‐LONG and B‐LONG clinical studies

Abstract: Changes in Haem-A-QoL key domains and 'Total Score' suggest that prophylaxis with long-acting rFVIIIFc or rFIXFc resulted in meaningful HRQoL improvements.

Cited by 40 publications

References 21 publications

Health‐related quality of life in paediatric haemophilia B patients treated with rIX‐FP

Health‐related quality of life in paediatric haemophilia B patients treated with rIX‐FP

Real‐life experience in switching to new extended half‐life products at European haemophilia centres

Achieving the unimaginable: Health equity in haemophilia

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