Challenges and perspectives in the treatment of patients with haemophilia in Brasil

Guedes, Vanessa Girotto; Thomas, Sylvia; Wachholz, Patrick Alexander; Souza, Sérgio Augusto Lopes de

doi:10.1590/1806-9282.64.10.872

Cited by 6 publications

(12 citation statements)

References 5 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…This study found that noncompliance with the treatment protocol remains high in adults with hemophilia, even in a country that has been implementing public health policies to improve the treatment and provides public healthcare assistance and free-of-charge distribution of coagulation factors to hemophilic patients, such as Brazil. 4,12,15,17 In other studies, the level of compliance ranged from 43% to 87%, but they evaluated different aspects of hemophilia treatment, mainly adherence to prophylactic treatment, and included children and adolescents. [17][18][19][20][21] The present study showed a much lower rate of compliance, but it evaluated other aspects of the treatment protocol in addition to the adherence to clotting factor treatment prescribed and included only adults that are associated with a higher chance of noncompliance than younger age patients.…”

Section: Discussionmentioning

confidence: 99%

“…22 However, our study showed low compliance with the protocol even in a country that has adopted a public healthcare system with free and universal coverage for its population, including treatment with high-cost products, which shows that other factors are also important. 4,12,16,17 Protocols are essential to achieve better physical and psychosocial health and quality of life, focusing on relevant aspects, such as monitoring pharmacological treatment, bleeding, and the presence of inhibitors. Additionally, scheduled appointments as part of the protocol are opportunities for health promotion and prevention of harm.…”

Section: Discussionmentioning

confidence: 99%

“…[1][2][3] Despite an increase in treatment advances in recent years, hemophilia remains associated with a high prevalence of disabilities and mortality in many countries. 2,4,5 One of the factors that may influence this finding is compliance with the hemophilia treatment protocol, which may be due to problems related either to the patient or healthcare professional adherence to the protocol recommendations. In addition to other chronic diseases, such as diabetes mellitus and systemic arterial hypertension, a challenge for healthcare professionals is ensuring that patients comply with their treatments.…”

Section: Introductionmentioning

confidence: 99%

“…12 Regarding hemophilia care, the Brazilian Ministry of Health has implemented public policies to improve treatment, especially by making higher investments mainly in the acquisition and the regular free-of-charge distribution of plasma-derived or recombinant coagulation factors, aiming to reduce the risks of complications. 4,[13][14][15][16] Thus, this study aimed (i) to identify compliance with a treatment protocol among adult patients with hemophilia and (ii) to evaluate the factors associated with and impact on mortality of noncompliance with a hemophilia treatment protocol.…”

Section: Introductionmentioning

confidence: 99%

See 3 more Smart Citations

<p>Factors Associated with Compliance with the Treatment Protocol and Mortality in Adults with Hemophilia</p>

Soares

Simeoni

Almeida

et al. 2020

PPA

View full text Add to dashboard Cite

Objective: Hemophilia is associated with a high prevalence of disabilities and mortality. This finding can be influenced by patient compliance with the treatment protocol. This study aims to identify compliance with a treatment protocol in adult patients with hemophilia and to evaluate the factors associated with and the impact on mortality of noncompliance with a hemophilia treatment protocol. Methods: This was a cross-sectional study that was performed between June 2015 and May 2016, followed by a cohort to evaluate mortality until July 2019 that included all adult patients with hemophilia registered in the Federal District, Brazil. Results: Among 138 patients enrolled in the study, 35 patients were compliant with all items of the treatment protocol (25.4%). Regarding each item, compliance with the medical consultations was 71.0% (98/138); the clotting factor regimen was 65.9% (91/138); and the serological tests were 51.4% (71/138). The mortality was 7.2% (10/138). Noncompliance with any aspect of the protocol was associated with mortality: medical consultations (p<0.001), clotting factor regimen (p=0.013), and serological tests (p=0.006). All deaths occurred in those who did not comply with the protocol, and the majority were due to bleeding. Patients who were noncompliant with all protocol items showed the highest mortality (50.0%, 5/10). Treatment at the hemophilia treatment center (OR: 2.388; 95% CI: 1.052-5.418, p=0.037) was positively and independently associated with compliance with the protocol in multivariate analysis. Conclusion: Noncompliance with the treatment protocol was high. Treatment at a hemophilia treatment center was positively and independently associated with compliance with the protocol, which reinforces the importance of comprehensive care by a multidisciplinary team.

show abstract

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

<p>Factors Associated with Compliance with the Treatment Protocol and Mortality in Adults with Hemophilia</p>

Soares

Simeoni

Almeida

et al. 2020

PPA

View full text Add to dashboard Cite

show abstract

“…Brazil's HTCs have been very involved in advancing care and establishing clinical research to improve haemophilia care (eg the MSK Made in Brazil project) and make new products available. This process was fuelled by participation in clinical trials and successfully supported by the Ministry of Health 52,64 . With accumulating experience in clinical trials, multiple Brazilian centres are now participating in a phase 3 AAV‐FVIII gene therapy trial (NCT03370913) and a lead‐in study for alternative FVIII or FIX gene therapy trials (NCT03587116).…”

Section: Haemophilia Care and Gene Therapy Development In Latin Amerimentioning

confidence: 99%

Hemophilia gene therapy—New country initiatives

2020

View full text Add to dashboard Cite

Gene therapy is an opportunity for haemophilia patients to receive a one‐time treatment and have lasting factor levels for years or decades instead of dependence on repeated administration within short intervals and on sustained supply of drug. Great strides have been made in the development of gene therapy for haemophilia in the last decade. Adeno‐associated virus (AAV) vector–mediated gene transfer in haemophilia A and B has entered the phase III trial stage. Gene transfer by lentiviral vector or gene editing technologies using factor VIII (FVIII) or IX (FIX) genes are now entering clinical evaluation. It is expected that the first FVIII and FIX gene therapy products will soon be approved and distributed in major markets. Global access to gene therapy is a critical goal. This review presents new and ongoing efforts towards this goal in countries other than North America and Europe. In Japan, researchers, regulators and funders have established a promising gene therapy development platform for multiple diseases including haemophilia. Decades of scientific and clinical research in haemophilia gene therapy in China have led to a recently registered clinical trial of AAV‐mediated gene therapy for haemophilia B. Other countries are in earlier phases of building gene therapy programmes or participate in international trials. A phase 2 feasibility trial of AAV‐mediated FIX gene therapy in low‐ and middle‐income countries aims to demonstrate that gene therapy could become available in resource‐constrained socio‐economic settings. The different strategies for establishing gene therapy provide opportunities for closing the global gap in haemophilia care.

show abstract

Functional and joint evaluation in a prospective cohort of patients with severe haemophilia

et al. 2021

View full text Add to dashboard Cite

Introduction Arthropathy is the main morbidity of haemophilia. Periodic joint health assessment enables a better understanding of the limitations of these patients. Objective To evaluate the functional and joint performance in patients with haemophilia at one‐year follow‐up, as well as its association with prophylactic treatment and attendance at scheduled consultations at a haemophilia treatment centre. Methods This prospective cohort study included patients with severe haemophilia aged 7 years or more and treated at Fundação Hemocentro de Brasília, Brazil, from January 2014 to December 2018. The Hemophilia Joint Health Score and Functional Independence Score in Hemophilia were assessed at the first consultation and after a one‐year follow‐up. Results The study included 69 patients. The mean age at study recruitment was 22.5 ± 4.5 years, 62.3% of patients aged 18 years or older, and 29 patients were receiving primary prophylaxis (38.0%). There was a positive correlation between HJHS and age and a negative correlation between FISH and age. The worsening HJHS was associated with non‐primary prophylaxis and non‐attendance at scheduled multidisciplinary consultations. The worsening FISH was associated with non‐primary prophylaxis. The correlation between FISH and treatment adherence was significant for the delta. Conclusion The older the patient with haemophilia, the higher the probability of a worsening of the HJHS. In the presence of more arthropathies, the older the patient, the worse the FISH. Patients receiving primary prophylaxis show better results in the HJHS and FISH when compared to patients receiving secondary prophylaxis and/or on‐demand treatment.

show abstract

Challenges and perspectives in the treatment of patients with haemophilia in Brasil

Cited by 6 publications

References 5 publications

<p>Factors Associated with Compliance with the Treatment Protocol and Mortality in Adults with Hemophilia</p>

<p>Factors Associated with Compliance with the Treatment Protocol and Mortality in Adults with Hemophilia</p>

Hemophilia gene therapy—New country initiatives

Functional and joint evaluation in a prospective cohort of patients with severe haemophilia

Contact Info

Product

Resources

About