scholarly journals Real-World Rates of Bleeding, Factor VIII Use, and Quality of Life in Individuals with Severe Haemophilia A Receiving Prophylaxis in a Prospective, Noninterventional Study

2021 ◽  
Vol 10 (24) ◽  
pp. 5959
Author(s):  
Gili Kenet ◽  
Yeu-Chin Chen ◽  
Gillian Lowe ◽  
Charles Percy ◽  
Huyen Tran ◽  
...  
Keyword(s):  
Factor Viii ◽  
Physical Functioning ◽  
Real World ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Real World Data ◽  
World Data ◽  
Geographic Regions ◽  
Noninterventional Study

Regular prophylaxis with exogenous factor VIII (FVIII) is recommended for individuals with severe haemophilia A (HA), but standardised data are scarce. Here, we report real-world data from a global cohort. Participants were men ≥18 years old with severe HA (FVIII ≤ 1 IU/dL) receiving regular prophylaxis with FVIII. Participants provided 6 months of retrospective data and were prospectively followed for up to 12 months. Annualised bleeding rate (ABR) and FVIII utilisation and infusion rates were calculated. Differences between geographic regions were explored. Of 294 enrolled participants, 225 (76.5%) completed ≥6 months of prospective follow-up. Pre-baseline and on-study, the median (range) ABR values for treated bleeds were 2.00 (0–86.0) and 1.85 (0–37.8), respectively; the median (range) annualised FVIII utilisation rates were 3629.0 (1008.5–13541.7) and 3708.0 (1311.0–14633.4) IU/kg/year, respectively; and the median (range) annualised FVIII infusion rates were 120.0 (52.0–364.0) and 122.4 (38.0–363.8) infusions/year, respectively. The median (range) Haemo-QoL-A Total Score was 76.3 (9.4–100.0) (n = 289), ranging from 85.1 in Australia to 67.7 in South America. Physical Functioning was the most impacted Haemo-QoL-A domain in 4/6 geographic regions. Despite differences among sites, participants reported bleeding requiring treatment and impaired physical functioning. These real-world data illustrate shortcomings associated with FVIII prophylaxis for this global cohort of individuals with severe HA.

A multicentre real-world data study to assess the bleeding rate and management of patients with haemophilia A and factor VIII inhibitors in Argentina

2021 ◽  
Vol Publish Ahead of Print ◽  
Author(s):  
Daniela Neme ◽  
Isabel Ramos Mejía ◽  
Ludmila Elelhou ◽  
Patricia Do Nascimento ◽  
Maria E. Arrieta ◽  
...  
Keyword(s):  
Factor Viii ◽  
Real World ◽  
Haemophilia A ◽  
Real World Data ◽  
Bleeding Rate ◽  
World Data

scholarly journals Dental management of patients with haemophilia in the era of recombinant treatments: increased efficacy and decreased clinical risk

2019 ◽  
Vol 12 (4) ◽  
pp. e227974
Author(s):  
Antonio Liras ◽  
Luis Romeu
Keyword(s):  
Factor Viii ◽  
Conventional Treatment ◽  
Coagulation Factors ◽  
Clotting Factor ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Optimal Result ◽  
Dental Management ◽  
Efficient Access

Haemophilia is a hereditary X-linked recessive disorder caused by a deficiency of either clotting factor VIII (haemophilia A) or IX (haemophilia B). Conventional treatment is currently based on the use of either plasma derived or recombinant coagulation factors. This paper reports on the case of a patient with severe haemophilia who presented with mesial decay and interproximal tartar build-up, for which extraction and scaling to remove tartar deposits were indicated. Following extraction, the usual haemostasis techniques were applied, and postoperative prophylactic antihaemophilic treatment was indicated for 2 or 3 days. The patient presented with moderate bleeding for a few minutes immediately after the procedure. Administration of factor VIII before surgery as well as the patient’s favourable pharmacokinetic response allowed for an optimal result. This treatment has afforded patients with haemophilia a better quality of life, and safe and efficient access to invasive surgical procedures.

scholarly journals Real-world data: how they can help to improve quality of care

2021 ◽  
Vol 8 ◽  
pp. 134-139
Author(s):  
Giovanni Corrao ◽  
Giovanni Alquati ◽  
Giovanni Apolone ◽  
Andrea Ardizzoni ◽  
Giuliano Buzzetti ◽  
...  
Keyword(s):  
Public Health ◽  
Quality Of Care ◽  
Real World ◽  
Ethical Issues ◽  
Data Availability ◽  
Quality Data ◽  
Real World Data ◽  
World Data ◽  
Critical Issues

The current COVID pandemic crisis made it even clearer that the solutions to several questions that public health must face require the access to good quality data. Several issues of the value and potential of health data and the current critical issues that hinder access are discussed in this paper. In particular, the paper (i) focuses on “real-world data” definition; (ii) proposes a review of the real-world data availability in our country; (iii) discusses its potential, with particular focus on the possibility of improving knowledge on the quality of care provided by the health system; (iv) emphasizes that the availability of data alone is not sufficient to increase our knowledge, underlining the need that innovative analysis methods (e.g., artificial intelligence techniques) must be framed in the paradigm of clinical research; and (v) addresses some ethical issues related to their use. The proposal is to realize an alliance between organizations interested in promoting research aimed at collecting scientifically solid evidence to support the clinical governance of public health.

scholarly journals Breast cancer patients’ quality of life: Real-world data

2019 ◽  
Vol 30 ◽  
pp. v744-v745
Author(s):  
T. Kosmidis ◽  
B. Athanasakou ◽  
P.A. Kosmidis
Keyword(s):  
Breast Cancer ◽  
Quality Of Life ◽  
Cancer Patients ◽  
Real World ◽  
Breast Cancer Patients ◽  
Real World Data ◽  
World Data

scholarly journals Health Related Quality of Life-Outcome in National Care - Real World Data from the German Psoriasis-Registry “Psobest”

2013 ◽  
Vol 16 (7) ◽  
pp. A511
Author(s):  
S. Purwins ◽  
C. Spehr ◽  
M. Augustin ◽  
M.A. Radtke ◽  
K. Reich ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Real World ◽  
Health Related Quality ◽  
Real World Data ◽  
World Data ◽  
Related Quality ◽  
Health Related

Contributions of Real-World Evidence and Real-World Data to Decision-Making in the Management of Soft Tissue Sarcomas

Oncology ◽  
2021 ◽  
Vol 99 (Suppl. 1) ◽  
pp. 3-7
Author(s):  
George D. Demetri ◽  
Silvia Stacchiotti
Keyword(s):  
Decision Making ◽  
Soft Tissue ◽  
Real World ◽  
Soft Tissue Sarcomas ◽  
Clinical Settings ◽  
Disease Entity ◽  
Real World Data ◽  
World Data ◽  
Real World Evidence

Real-world data are defined as data relating to any aspect of a patient’s health status collected in the context of routine health surveillance and medical care delivery. Sources range from insurance billing claims through to electronic surveillance data (e.g., activity trackers). Real-world data derive from large populations in diverse clinical settings and thus can be extrapolated more readily than clinical trial data to patients in different clinical settings or with a variety of comorbidities. Real-world data are used to generate real-world evidence, which might be regarded as a “meta-analysis” of accumulated real-world data. Increasingly, regulatory authorities are recognizing the value of real-world data and real-world evidence, especially for rare diseases where it may be practically unfeasible to conduct randomized controlled trials. However, the quality of real-world evidence depends on the quality of the data collected which, in turn, depends on a correct pathological diagnosis and the homogeneous behaviour of a reliably defined and consistent disease entity. As each of the more than 80 varieties of soft tissue sarcoma (STS) types represents a distinct disease entity, the situation is exceedingly complicated. Discordant diagnoses, which affect data quality, present a major challenge for use of real-world data. As real-world data are difficult to collect, collaboration across sarcoma reference institutions and sophisticated information technology solutions are required before the potential of real-world evidence to inform decision-making in the management of STS can be fully exploited.

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