Dataset Information

LongHest project: A prospective, observational study of extended half-life treatment in the musculoskeletal health of patients with severe haemophilia A.

ABSTRACT:

Background

Prophylactic treatment is the gold standard in the treatment of patients with haemophilia. Prophylaxis with extended half-life (EHL) treatment has shown long-term safety and efficacy in patients with haemophilia.

Aim

To evaluate the efficacy of prophylaxis with EHL treatment in the frequency of haemarthrosis and musculoskeletal health in adult patients with severe haemophilia A.

Methods

Prospective cohort study. Forty-six patients with severe haemophilia A were recruited. The frequency of haemarthrosis (self-reports), joint condition (Haemophilia Joint Health Score), pain intensity (visual analogue scale), range of motion (goniometry), and strength (dynamometry) and muscle activation (surface electromyography) were evaluated. Three assessments were carried out: at baseline (T0), at 6 months (T1) and at 12 months following treatment (T2).

Results

There were significant changes in the within-subject effect in the frequency of haemarthrosis in elbow (F(1.05;96.20) = 3.95; P < .001) and knee (F(1.73;157.99) = 9.96; P < .001). Significant within-subject effect in elbow pain intensity (F(2;182) = 63.51; P < .001) was found. The mean values of the frequency haemarthrosis in elbow (from .66±1.01 to .04±.20) and knees (from .55±.68 to .33±.53) decrease after the period study. The intensity of elbow pain and (from 3.08±1.69 to 2.67±1.73), decrease after the 12-month follow-up period.

Conclusions

Prophylaxis with extended half-life treatment reduces the frequency of haemarthrosis in elbow and knee in adult patients with haemophilia. EHL treatment reduces the intensity of elbow pain in patients with haemophilic arthropathy.

SUBMITTER: Cuesta-Barriuso R

PROVIDER: S-EPMC9541876 | biostudies-literature | 2022 Sep

REPOSITORIES: biostudies-literature

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Publications

LongHest project: A prospective, observational study of extended half-life treatment in the musculoskeletal health of patients with severe haemophilia A.

Cuesta-Barriuso Rubén R Pérez-Llanes Raúl R Donoso-Úbeda Elena E Ucero-Lozano Roberto R

Haemophilia : the official journal of the World Federation of Hemophilia 20220513 5

<h4>Background</h4>Prophylactic treatment is the gold standard in the treatment of patients with haemophilia. Prophylaxis with extended half-life (EHL) treatment has shown long-term safety and efficacy in patients with haemophilia.<h4>Aim</h4>To evaluate the efficacy of prophylaxis with EHL treatment in the frequency of haemarthrosis and musculoskeletal health in adult patients with severe haemophilia A.<h4>Methods</h4>Prospective cohort study. Forty-six patients with severe haemophilia A were r ...[more]

PMID: 35561281

Similar Datasets

Project description:Background and objectiveHaemophilia B is a rare genetic disease that is caused by a deficiency of coagulation factor IX (FIX) in the blood and leads to internal and external bleeding. Under the current standard of care, haemophilia is treated either prophylactically or on-demand via intravenous infusions of FIX. These treatment strategies impose a high burden on patients and health care systems as haemophilia B requires lifelong treatment, and FIX is costly. Etranacogene dezaparvovec (ED) is a gene therapy for haemophilia B that has been recently approved by the United States Food and Drug Administration and has received a recommendation for conditional marketing authorization by the European Medicines Agency. We aimed to examine the cost-effectiveness of ED versus extended half-life FIX (EHL-FIX) prophylaxis for moderate-to-severe haemophilia B from a German health care payer perspective.MethodsA microsimulation model was implemented in R. The model used data from the ED phase 3 clinical trial publication and further secondary data sources to simulate and compare patients receiving ED or EHL-FIX prophylaxis over a lifetime horizon, with the potential for ED patients to switch treatment to EHL-FIX prophylaxis when the effectiveness of ED waned. Primary outcomes of this analysis included discounted total costs, discounted quality-adjusted life years (QALYs), incremental cost-effectiveness, and the incremental net monetary benefit. The annual discount rate for costs and effects was 3%. Uncertainty was examined via probabilistic analysis and additional univariate sensitivity analyses.ResultsProbabilistic analysis indicated that patients treated with ED instead of EHL-FIX prophylaxis gained 0.50 QALYs and experienced cost savings of EUR 1,179,829 at a price of EUR 1,500,000 per ED treatment. ED was the dominant treatment strategy. At a willingness to pay of EUR 50,000/QALY, the incremental net monetary benefit amounted to EUR 1,204,840.DiscussionDepending on the price, ED can save costs and improve health outcomes of haemophilia patients compared with EHL-FIX prophylaxis, making it a potentially cost-effective alternative. These results are uncertain due to a lack of evidence regarding the long-term effectiveness of ED.

Dataset Information

LongHest project: A prospective, observational study of extended half-life treatment in the musculoskeletal health of patients with severe haemophilia A.

Background

Aim

Methods

Results

Conclusions

Publications

LongHest project: A prospective, observational study of extended half-life treatment in the musculoskeletal health of patients with severe haemophilia A.

Similar Datasets

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