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Modelling US health equity impacts of emicizumab for severe haemophilia A: aggregate distributional cost-effectiveness analysis

Por: Majda · T. · Lee · J. S. · Curtis · R. · Kowal · S. L.
Objectives

Emicizumab is the first bispecific antibody approved for prophylaxis in people with haemophilia A with or without factor VIII inhibitors. Aggregate distributional cost-effectiveness analysis assesses health equity impacts by evaluating how health effects and costs from funding an intervention are distributed among population subgroups. The objective was to evaluate how funding emicizumab for people with severe haemophilia A (PwSHA) impacts population health and health disparities in the USA.

Design

Population-level model of PwSHA from the perspective of the US healthcare system, using published sources and considering a lifetime time horizon.

Intervention

Emicizumab versus other haemophilia A prophylaxis treatments.

Primary outcome measures

Quality-adjusted life-years (QALYs) gained and change in Atkinson index of inequality in quality-adjusted life expectancy.

Results

When an estimated 6512 PwSHA in the USA were treated with emicizumab, the US healthcare system would save US$160 billion over those individuals’ lifetimes. If these cost savings fund additional healthcare interventions in the overall population, funding emicizumab would improve overall US population health (1 068 903 QALYs gained, using a threshold of US$150 000/QALY) and reduce existing overall US inequities (–0.01% on the Atkinson index).

In all scenarios tested for sensitivity, increased emicizumab and prophylaxis utilisation led to further reductions in health disparities and greater increases in population health. Results were robust to deterministic variations in the allocation of cost savings due to emicizumab use.

Conclusion

Funding emicizumab treatments for PwSHA improves overall population health and reduces overall health inequities in the USA. Cost savings from the use of emicizumab free up important resources that can be leveraged to support other healthcare interventions, but decisions on how these funds are used have large consequences for equity.

Effectiveness of Open Incision Negative Pressure Wound Therapy for Surgical Site Infection Prevention for Abdominal Surgery—A Systematic Review and Meta‐Analysis

ABSTRACT

Open incision negative pressure wound therapy (NPWT) combines delayed closure with negative pressure to prevent surgical site infection (SSI). Its effectiveness in preventing SSI remains unclear, complicating its risk–benefit assessment. PubMed and Web of Science databases were searched for relevant English studies. Two reviewers independently screened titles and abstracts using the ASReviewer tool. Full-text articles were assessed for eligibility. Eight studies were included in the systematic review and five were pooled in the meta-analysis. Data extraction followed the PRISMA guidelines, and the risk of bias was assessed. A meta-analysis was performed using a random-effects model for SSI occurrence. Eight studies (three RCTs and five cohort studies) with 1655 patients were included. Studies were pooled based on control interventions: primary closure (PC) or delayed primary closure (DPC). Pooled odds ratio (OR) estimates favoured NPWT over PC for SSI reduction (OR, 0.15; 95% CI, 0.02–0.87). No significant SSI risk difference was found between the NPWT and DPC groups (OR, 0.28; 95% CI, 0.06–1.27). Preventive NPWT is associated with a reduced risk of SSI in abdominal surgery compared to PC. Our findings indicate that standardising treatment and reporting protocols could improve future evaluations of NPWT effectiveness.

Trial Registration: PROSPERO identifier: CRD42024401669

Ten sessions of hyperbaric oxygen versus sham treatment in patients with long covid (HOT-LoCO): a randomised, placebo-controlled, double-blind, phase II trial

Por: Kjellberg · A. · Hassler · A. · Boström · E. · El Gharbi · S. · Al-Ezerjawi · S. · Schening · A. · Fischer · K. · Kowalski · J. H. · Rodriguez-Wallberg · K. A. · Bruchfeld · J. · Stahlberg · M. · Nygren-Bonnier · M. · Runold · M. · Lindholm · P.
Objectives

To evaluate if 10 sessions of hyperbaric oxygen treatments (HBOTs) improve short- and long-term health related quality of life, symptoms and physical performance in long covid patients compared with placebo.

Design

Parallel, randomised, placebo-controlled, double-blind trial.

Setting

Single-centre, university hospital, Sweden.

Participants

Previously healthy subjects aged 18–60 years, diagnosed with long covid were included. We excluded pregnant women, patients with RAND-36 (role limitations due to physical health (RP) and physical functioning (PF)) above 70, diabetes, hypertension and contraindications for HBOT.

Interventions

Subjects were randomly assigned to 10 sessions of HBOT or sham (placebo) treatments over 6 weeks. HBOT involved 100% oxygen, 2.4 bar, 90 min, placebo medical air, 1.34–1.2 bar. Randomisation (1:1) was done electronically, in blocks stratified by sex and disease severity. Subjects and investigators were blinded to allocation.

Primary and secondary outcome measures

Primary endpoints were changes from baseline in RAND-36 PF and RP at 13 weeks. Efficacy was analysed on an intention-to-treat basis. Harms were evaluated according to the actual treatment given.

Results

Between 15 September 2021 and 20 June 2023, 80 subjects (65 women, 15 men) were enrolled and randomised (40 in each group). The trial is completed. The primary endpoint analysis included 79 subjects (40 in HBOT and 39 in control). At 13 weeks, both groups showed improvement, with no significant difference between HBOT and placebo in PF (least square mean difference between groups (LSD), 0.63 (95% CI –7.04 to 8.29), p=0.87) and RP (LSD, 2.35 (95% CI –5.95 to 10.66), p=0.57). Harms: 43 adverse events (AEs), most commonly cough and chest pain/discomfort, occurred in 19 subjects (49%) of the HBOT group and 38 AEs in 18 subjects (44%) of the placebo group, one serious AE in HBOT and one death in the placebo group.

Conclusions

10 HBOT sessions did not show more short-term benefits than placebo for long covid patients. Both groups improved, with a notable sex difference. HBOT has a favourable harm profile.

Trial registration number

ClinicalTrials.gov (NCT04842448), EudraCT (2021-000764-30). The trial was funded by Vetenskapsrådet (2022-00834), Region Stockholm (2020-0731, 2022-0674), Hjärt-Lungfonden and OuraHealth Oy.

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