(Press-News.org) Numerous long-term randomized controlled trials (RCTs) have been conducted to investigate the long-term, factor concentrate-based treatment of patients with severe haemophilia A or B, despite the rareness of the diseases and the lack of incentives for pharmaceutical companies. These RCTs include three studies comparing prophylactic versus on-demand treatment (i.e. treatment initiated only in the event of bleeding). For patients with haemophilia A, both hints and an indication of an added benefit of prophylactic versus on-demand treatment can be inferred from these studies, but only for certain outcomes.
No evaluable data were available on the effects of the different treatment strategies on joint function. For children, the results for most outcomes are not interpretable due to inadequate data quality. However, the results support most current (clinical practice) guidelines and treatment algorithms with regard to recommendations on the prevention of severe bleeding.
These are the main conclusions of a rapid report published on 25 June 2015, which the German Institute for Quality and Efficiency in Health Care (IQWiG) prepared on behalf of the German Federal Ministry of Health (BMG).
Three research questions
The report had three aims: IQWiG was to map the available evidence on the long-term, factor concentrate-based treatment of patients with severe haemophilia A or B in respect of different treatment strategies, factor concentrates, dosing regimens, and prophylactic treatment regimens. On this basis, the long-term benefit of prophylactic and on-demand treatment was to be compared with regard to patient-relevant outcomes analysed in controlled prospective studies. In addition, the Institute was to evaluate to what extent current guidelines and treatment algorithms are based on the evidence identified.
16 long-term studies identified
13 completed and three ongoing long-term RCTs were identified for the first research question. Only two studies included patients with haemophilia B. Important clinical questions remained unanswered for children. Likewise, it is still an open question as to whether differences exist between factor concentrates based on blood plasma and those based on biotechnology techniques.
This is partly due to the fact that manufacturers of factor concentrates have no incentive to conduct comparative long-term studies, as these are not required for approval. Even so, the mapping of evidence shows that RCTs are also feasible in relatively rare diseases such as haemophilia A or B.
No conclusion on added benefit possible for haemophilia B
A total of three of the studies were suitable for a comparison of the benefit of prophylactic versus on-demand factor concentrate-based treatment: one in adolescents and adults and two in children, all with severe haemophilia A. No conclusion on a greater or lesser patient-relevant benefit of prophylactic versus on-demand treatment is thus possible for patients with haemophilia B.
For adolescents and adults with severe haemophilia A, the data provide hints of an added benefit of prophylactic treatment for the outcomes of health status and pain, as well as an indication of an added benefit for the outcome of severe bleeding. Both studies in children have such a high risk of bias that a hint of an added benefit of factor concentrate-based prophylactic treatment can only be inferred for severe bleeding. For children in particular, no added benefit of either of the two treatment strategies can therefore be inferred with regard to patient-relevant outcomes such as pain, joint function or quality of life.
Guidelines and treatment algorithms: few contradictions to available evidence
To identify guidelines and treatment algorithms, IQWiG wrote to German institutions specialized in the treatment of patients with haemophilia. Of the 13 potentially relevant guidelines and four treatment algorithms obtained, only three guidelines turned out to be evidence-based. Most of the guidelines and algorithms do not contradict the results of the benefit assessment.
However, for adult patients one of the clinics generally recommends on-demand treatment, which is not supported by the results of the benefit assessment. In addition, several recommendations contain statements on the preservation and functionality of joints that are not supported by the available evidence. This is because no evaluable data on these outcomes were reported in the studies.
Process of report production
The BMG commissioned IQWiG to prepare the report in an accelerated process, known as a "rapid report". In contrast to the usual procedure, no preliminary reports are published here and no hearing takes place at which all interested parties can comment. The present rapid report was sent to the commissioning agency on 28 May 2015.
An overview of the background, methods and further results of the report is provided in the following German executive summary. An English executive summary will be available soon. If you would like to be informed when this document is available, please send an e-mail to » info@iqwig.de.
INFORMATION:
July 13, 2015 - For people who use cocaine, stopping or reducing cocaine use is associated with decreased levels of endothelin-1 (ET-1)--a protein that plays a key role in the development of coronary artery disease, reports a study in the Journal of Addiction Medicine, the official journal of the American Society of Addiction Medicine. The journal is published by Wolters Kluwer.
The findings help in understanding the high rate of atherosclerosis (hardening of the arteries) associated with cocaine use, according to the report by Dr. Shenghan Lai and colleagues of Johns ...
Canadian and British researchers have discovered that chromosomes play an active role in animal cell division. This occurs at a precise stage - cytokinesis - when the cell splits into two new daughter cells. It was observed by a team of researchers including Gilles Hickson, an assistant professor at the University of Montreal's Department of Pathology and Cell Biology and researcher at the CHU Sainte-Justine Research Centre, his assistant Silvana Jananji, in collaboration with Nelio Rodrigues, a PhD student, and Sergey Lekomtsev, a postdoc, working in the group led by Buzz ...
This news release is available in French. Montréal, July 13, 2015 - It's no coincidence that the earthworm's slender shape makes it perfect for weaving through narrow tunnels. Evolution moulds the shapes of living creatures according to the benefits they offer. At the microscopic level, do the various shapes of bacteria also contribute to their survival? Does a spherical bacterium (coccus) have a better chance of infecting its host than its stick-shaped neighbour (bacillus)?
Analysis of the evolution of the pathogenic bacteria that live in the nasopharynx suggests ...
WASHINGTON (July 13,2015) - The American College of Cardiology's National Cardiovascular Data Registry was the source of data for research published in the first half of 2015, including a study that examines public reporting and PCI outcomes as well as research that showed implantable cardioverter-defibrillators are underutilized in older patients.
Is There a Link Between Public Reporting and PCI Outcomes?
Patients who underwent percutaneous coronary intervention (PCI) in states with mandated public reporting of outcomes had similar predicted risks but significantly ...
July 13, 2015 -Children with inflammatory bowel disease (IBD) who are doing well on specific biological medications should not be switched to recently approved "biosimilar" products, concludes an expert consensus statement of the European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN). The statement appears in the Journal of Pediatric Gastroenterology and Nutrition, the official journal of the European Society of Paediatric Gatroenterology, Hepatology and Nutrition and the North American Society for Pediatric Gastroenterology, Hepatology and ...
The world's rarest ape has an increased chance of survival after a team led by international conservation charity the Zoological Society of London (ZSL) found a new family group of Hainan gibbons (Nomascus hainanus).
Until last month, it was thought that there were just 25 Hainan gibbons living in three social groups on an island off the Chinese mainland.
The discovery of a new fourth group, a mating pair with a young baby, sighted within Bawangling National Nature Reserve, Hainan Province, increases the known population by almost 12%.
The discovery of this fourth ...
Numerous long-term randomized controlled trials (RCTs) have been conducted to investigate the long-term, factor concentrate-based treatment of patients with severe haemophilia A or B, despite the rareness of the diseases and the lack of incentives for pharmaceutical companies. These RCTs include three studies comparing prophylactic versus on-demand treatment (i.e. treatment initiated only in the event of bleeding). For patients with haemophilia A, both hints and an indication of an added benefit of prophylactic versus on-demand treatment can be inferred from these studies, ...
Children consume more low-nutrition, high-calorie food such as cookies and candy after observing seemingly overweight cartoon characters, according to a first-of-its-kind study led by the University of Colorado Boulder.
The results of the new study, involving Colorado State University and published online in the Journal of Consumer Psychology, show that kids are responsive to the apparent bodyweight of cartoon characters like the aptly named Grimace, a rotund, milkshake-loving creature created by McDonald's restaurant in the 1970s.
Children tend to perceive ovoid, or ...
Immune cells that creep across blood vessels trigger potentially fatal bleeding in platelet-deficient mice, according to a report published in The Journal of Experimental Medicine. If the same is true in humans, blocking the passage of these cells could prevent dangerous complications in patients undergoing transplants or chemotherapy.
Bone marrow transplantation and chemotherapy are known to deplete blood-clotting cells called platelets, resulting in potentially fatal bleeding (hemorrhage) in some patients. Previous studies showed that inflammation was required for bleeding ...
BOSTON (July 13, 2015)--Physicians in their medical residency training programs often focus on scientific reasoning and research evidence in their efforts to provide medical care. While appropriate, this focus may overshadow subtle and indirect communication that reveals important information about the patient's experience with their illness that will help the physician provide better care. A new study by researchers at Tufts University School of Medicine and Boston College presents the results of a strategy to train medical residents to reflect on interactions with patients ...
