Several drug companies, such as Biogen Idec and Novo Nordisk, are developing new, longer-acting versions of the blood clotting factors used by people with hemophilia. Patients with severe forms of the disease need regular infusions, lasting 30 minutes or more, of relatively short acting and very expensive clotting factors.
The new longer-lasting hemophilia B products can be given every 10 days or two weeks, offering significant advantages for patients, especially young children, who now need infusions every two or three days.
Hemophilia is hereditary, passed from parent to child through genes. People with hemophilia have little or no clotting factor. Hemophilia A and Hemophilia B have different clotting factors that are low or missing, but both can experience spontaneous bleeding, as well as severe bleeding following injuries or surgery.
Worldwide, about one in 5,000 men is born with hemophilia A and one in 25,000 men is born with hemophilia B each year. Since the gene is carried on the X chromosome, hemophilia is almost entirely a disease of men. Women can pass the gene to their offspring. Hemophilia has often been called the “Royal Disease” since it was carried by Britain’s Queen Victoria and affected many of the ruling families of Europe. Blood factor concentrates were not developed until the mid-20th century, and up until that time people with hemophilia had a life expectancy of less than 30 years.
The U.S. Food and Drug Administration is due to decide by mid-year whether to approve a new long-lasting hemophilia B clotting factor from Biogen Idec. Novo Nordisk expects to file next year for regulatory approval of its long-acting hemophilia B drug.
Some industry experts say these and other new treatments could help drive down the price of existing hemophilia products, which can total $300,000 or more a year for a single patient.
Showing posts with label novo nordisk. Show all posts
Showing posts with label novo nordisk. Show all posts
Sunday, March 23, 2014
Thursday, June 16, 2011
Feds, states split $25M in settlement
The Food and Drug Administration approved Novo Nordisk's hemostasis management drug NovoSeven to treat certain bleeding disorders in hemophiliacs. After FDA approval, a manufacturer may not market or promote a drug for uses not specified in its new drug application or for uses not approved by the FDA. Unapproved uses are known as "off-label" uses.
Novo Nordisk's U.S. subsidiary, which is located in Princeton, N.J., allegedly promoted NovoSeven to healthcare professionals for off-label uses, including as a coagulatory agent for trauma patients, general surgery, cardiac surgery, liver surgery, liver transplants and intra-cerebral hemorrhage.
False claims were submitted to government healthcare programs that were not reimbursable because of Novo Nordisk's unlawful promotion, it was alleged. The federal share of the civil settlement is $21,425,790.59. The state Medicaid share of the civil settlement is $3,574,209.41.
"Health care patients should be able to trust that their prescription drugs are safe, effective and prescribed only for FDA approved uses," Maine Attorney General William Schneider said. "These off-label promotions waste Maine taxpayer dollars and we will seek recovery from pharmaceutical companies for this kind of healthcare fraud."
The settlement resolves a a whistleblower lawsuit filed under the qui tam or whistleblower provisions of the False Claims Act that is pending in the District of Maryland. Under terms of the resolution, the whistleblowers are set to receive more than $3.5 million from the federal share of the civil recovery.
Novo Nordisk also agreed to enter into an expansive corporate integrity agreement with the Office of the Inspector General of the Department of Health and Human Services as part of the settlement. That agreement creates procedures and reviews meant to avoid and promptly detect similar conduct in the future.
Thursday, May 5, 2011
Stanford Studies Document Widespread, Risky Use Clotting Drug On Non-Hemophilia Patients
An expensive blood-clotting drug that is intended only for hemophilia patients is being used in hospitals predominantly to treat patients without this disorder, despite evidence suggesting that it could harm them, according to a pair of studies from the Stanford University School of Medicine.
In fact, the studies estimate that only 4 percent of the powerful drug's use in U.S. hospitals from 2000 through 2008 was for treating hemophilia patients, while an enormous 96 percent involved cases of heart surgery, trauma, intracranial hemorrhages (bleeding in or near the brain) and a host of other surgical and medical problems. There are few studies examining these broader uses of the drug, known as recombinant factor 7a, and what little evidence does exist reveals a serious problem: The drug can increase the risk of blood clots, which can lead to heart attacks and strokes.
What's more, RF7a is pricey — it costs an estimated $10,000 for an average dose.
"The stakes are high with this one," said Veronica Yank, MD, an instructor in medicine and the first author of one of the studies. "Because it's such a powerful clotting agent, it has the potential when used off-label to damage the lives of patients without providing any real benefit."
Given the safety issues and the expense of RF7a, Yank and her colleagues are urging physicians to exercise greater caution in using the drug until its safety is effectively evaluated for these broader uses.
The studies will be published in the April 19 issue of Annals of Internal Medicine, along with an accompanying editorial by Harvard Medical School researchers commending the Stanford team for providing "compelling data … about the runaway use, uselessness and risk for this expensive treatment."
The editorial goes on to question whether "improper promotion" of RF7a by its maker, Novo Nordisk, led to the rapid expansion of its use. While the company has denied such practices, it is being investigated by the Defense Department for the use of the drug in overseas combat operations, the editorial reports.
The Stanford authors say RF7a provides a good example of what happens when physicians latch onto a "wonder" drug for uses distinctly different from its original purpose. In this case, the medication was developed to treat a small subset of hemophilia patients. In these genetic disorders, the body is deficient in producing specific proteins, or factors, that aid the blood-clotting process. It primarily affects males, and is estimated to occur in one in 5,000 live male births. There are two main forms of the disorder: hemophilia A (a deficiency in factor 8) and hemophilia B (a deficiency in factor 9).
To curtail bleeding in hemophilia patients, physicians usually administer doses of either factor 8 or factor 9, but this treatment doesn't work for the small group of patients whose antibodies see these factors as foreign substances and attack them. RF7a, approved by the U.S. Food and Drug Administration in 1999, was developed for this group.
Despite the limited range of patients for whom the drug was approved, the Stanford authors noted that physicians and surgeons quickly started using RF7a to either prevent or stop heavy bleeding in patients who didn't have hemophilia. It's a practice known as off-label prescribing, meaning that physicians use medications to treat conditions other than those approved by the FDA. While there is nothing illegal about off-label prescribing — and, in fact, it can be invaluable in treating diseases for which few therapies exist — senior author Randall Stafford, MD, PhD, associate professor of medicine at the Stanford Prevention Research Center, said the danger is that the drug hasn't been rigorously tested for these new uses or with broad ranges of patients.
"Many patients and physicians wrongly assume that the FDA has scrutinized all of the different ways a drug can be used, but they've only examined those uses that have gone through the approval process," Stafford said.
A few previously published studies had raised concerns that RF7a increased the risk of blood clots, and so in 2008 the U.S. Agency for Healthcare Quality and Research, which funded both Stanford studies, asked researchers to probe the issue. The agency specifically wanted to know what kind of clinical evidence existed for five off-label uses of RF7a: heart surgery, intracranial hemorrhage, body and brain trauma, liver transplantation and prostate surgery.
Yank, Stafford and their colleagues searched 10 widely used databases for clinical trials and observational studies of the drug for the five indications. They then rated the available clinical evidence and focused on the randomized clinical trials and observational studies, excluding those that did not directly address clinical outcomes. For the meta-analyses in the first paper, the researchers ended up using data from 16 randomized clinical trials and 10 observational studies.
The researchers found that RF7a didn't reduce mortality rates for any of the five off-label uses, although Yank noted that most of the studies tracked patients for only 30-90 days following the administration of the drug. For the top two uses of the drug, the analyses showed heart-surgery patients who were given RF7a had a 5 percent higher risk of developing blood clots, and intracranial hemorrhage patients had a 3 percent higher risk of developing clots. There was no increased risk of blood clots for body trauma patients, and the evidence was too limited to determine the clotting risks for brain trauma, liver transplants and prostate surgeries.
For the second study, first author Aaron Logan, MD, PhD, a postdoctoral scholar in hematology and bone marrow transplantation, joined Yank and Stafford in analyzing another database to determine how the drug was being used in U.S. hospitals. The data came from Premier Perspectives and includes information from 615 non-federal hospitals throughout the country.
In reviewing records from 2000 to 2008, the researchers found that the use of RF7a grew 140-fold, from 125 cases in 2000 to 17,813 in 2008, primarily for off-label indications. The researchers also noted that of the five off-label uses the AHRQ wanted the team to investigate, three of them — heart surgery, intracranial hemorrhage and trauma — were among the top uses of the drug. "This type of assessment can be quite eye-opening about the real-world use of these medications on the basis of extremely weak evidence," Logan said.
He also noted that the off-label use of RF7a has spread beyond academic medical centers where new or experimental therapies are often tested. The data showed the proportion of RF7a use in non-academic hospitals grew from 11 percent in 2000 to 67 percent in 2008. "This suggests wide adoption of RF7a as a therapy despite concerns about its efficacy and safety," the authors wrote.
Yank said she and her colleagues hope the two studies will prompt physicians and surgeons to be more cautious about the off-label use of RF7a. "Despite the miraculous ability of this drug to stop bleeding, we have an obligation to 'first do no harm,'" she said.
That sentiment was echoed in the accompanying editorial, by Harvard's Jerry Avorn, MD, professor of medicine, and Aaron Kesselheim, MD, JD, research associate in health policy and management, who wrote that, "Allowing physician autonomy to choose medications is appealing, but not when it results in such useless, dangerous and costly decisions."
In fact, the studies estimate that only 4 percent of the powerful drug's use in U.S. hospitals from 2000 through 2008 was for treating hemophilia patients, while an enormous 96 percent involved cases of heart surgery, trauma, intracranial hemorrhages (bleeding in or near the brain) and a host of other surgical and medical problems. There are few studies examining these broader uses of the drug, known as recombinant factor 7a, and what little evidence does exist reveals a serious problem: The drug can increase the risk of blood clots, which can lead to heart attacks and strokes.
What's more, RF7a is pricey — it costs an estimated $10,000 for an average dose.
"The stakes are high with this one," said Veronica Yank, MD, an instructor in medicine and the first author of one of the studies. "Because it's such a powerful clotting agent, it has the potential when used off-label to damage the lives of patients without providing any real benefit."
Given the safety issues and the expense of RF7a, Yank and her colleagues are urging physicians to exercise greater caution in using the drug until its safety is effectively evaluated for these broader uses.
The studies will be published in the April 19 issue of Annals of Internal Medicine, along with an accompanying editorial by Harvard Medical School researchers commending the Stanford team for providing "compelling data … about the runaway use, uselessness and risk for this expensive treatment."
The editorial goes on to question whether "improper promotion" of RF7a by its maker, Novo Nordisk, led to the rapid expansion of its use. While the company has denied such practices, it is being investigated by the Defense Department for the use of the drug in overseas combat operations, the editorial reports.
The Stanford authors say RF7a provides a good example of what happens when physicians latch onto a "wonder" drug for uses distinctly different from its original purpose. In this case, the medication was developed to treat a small subset of hemophilia patients. In these genetic disorders, the body is deficient in producing specific proteins, or factors, that aid the blood-clotting process. It primarily affects males, and is estimated to occur in one in 5,000 live male births. There are two main forms of the disorder: hemophilia A (a deficiency in factor 8) and hemophilia B (a deficiency in factor 9).
To curtail bleeding in hemophilia patients, physicians usually administer doses of either factor 8 or factor 9, but this treatment doesn't work for the small group of patients whose antibodies see these factors as foreign substances and attack them. RF7a, approved by the U.S. Food and Drug Administration in 1999, was developed for this group.
Despite the limited range of patients for whom the drug was approved, the Stanford authors noted that physicians and surgeons quickly started using RF7a to either prevent or stop heavy bleeding in patients who didn't have hemophilia. It's a practice known as off-label prescribing, meaning that physicians use medications to treat conditions other than those approved by the FDA. While there is nothing illegal about off-label prescribing — and, in fact, it can be invaluable in treating diseases for which few therapies exist — senior author Randall Stafford, MD, PhD, associate professor of medicine at the Stanford Prevention Research Center, said the danger is that the drug hasn't been rigorously tested for these new uses or with broad ranges of patients.
"Many patients and physicians wrongly assume that the FDA has scrutinized all of the different ways a drug can be used, but they've only examined those uses that have gone through the approval process," Stafford said.
A few previously published studies had raised concerns that RF7a increased the risk of blood clots, and so in 2008 the U.S. Agency for Healthcare Quality and Research, which funded both Stanford studies, asked researchers to probe the issue. The agency specifically wanted to know what kind of clinical evidence existed for five off-label uses of RF7a: heart surgery, intracranial hemorrhage, body and brain trauma, liver transplantation and prostate surgery.
Yank, Stafford and their colleagues searched 10 widely used databases for clinical trials and observational studies of the drug for the five indications. They then rated the available clinical evidence and focused on the randomized clinical trials and observational studies, excluding those that did not directly address clinical outcomes. For the meta-analyses in the first paper, the researchers ended up using data from 16 randomized clinical trials and 10 observational studies.
The researchers found that RF7a didn't reduce mortality rates for any of the five off-label uses, although Yank noted that most of the studies tracked patients for only 30-90 days following the administration of the drug. For the top two uses of the drug, the analyses showed heart-surgery patients who were given RF7a had a 5 percent higher risk of developing blood clots, and intracranial hemorrhage patients had a 3 percent higher risk of developing clots. There was no increased risk of blood clots for body trauma patients, and the evidence was too limited to determine the clotting risks for brain trauma, liver transplants and prostate surgeries.
For the second study, first author Aaron Logan, MD, PhD, a postdoctoral scholar in hematology and bone marrow transplantation, joined Yank and Stafford in analyzing another database to determine how the drug was being used in U.S. hospitals. The data came from Premier Perspectives and includes information from 615 non-federal hospitals throughout the country.
In reviewing records from 2000 to 2008, the researchers found that the use of RF7a grew 140-fold, from 125 cases in 2000 to 17,813 in 2008, primarily for off-label indications. The researchers also noted that of the five off-label uses the AHRQ wanted the team to investigate, three of them — heart surgery, intracranial hemorrhage and trauma — were among the top uses of the drug. "This type of assessment can be quite eye-opening about the real-world use of these medications on the basis of extremely weak evidence," Logan said.
He also noted that the off-label use of RF7a has spread beyond academic medical centers where new or experimental therapies are often tested. The data showed the proportion of RF7a use in non-academic hospitals grew from 11 percent in 2000 to 67 percent in 2008. "This suggests wide adoption of RF7a as a therapy despite concerns about its efficacy and safety," the authors wrote.
Yank said she and her colleagues hope the two studies will prompt physicians and surgeons to be more cautious about the off-label use of RF7a. "Despite the miraculous ability of this drug to stop bleeding, we have an obligation to 'first do no harm,'" she said.
That sentiment was echoed in the accompanying editorial, by Harvard's Jerry Avorn, MD, professor of medicine, and Aaron Kesselheim, MD, JD, research associate in health policy and management, who wrote that, "Allowing physician autonomy to choose medications is appealing, but not when it results in such useless, dangerous and costly decisions."
Tuesday, December 7, 2010
Novo Nordisk presents positive clinical data on two investigational compounds within bleeding disorders
Orlando, US – (7 December 2010) – Novo Nordisk presented data from a phase 2 trial evaluating the safety, pharmacokinetics and efficacy of a recombinant factor VIIa (rFVIIa) analogue, designed to have a faster action profile than NovoSeven® (rFVIIa) in haemophilia patients with inhibitors (antibody formation against factor preparations). The company also presented data from a phase 3 trial investigating a recombinant compound in patients with congenital factor XIII deficiency, a rare, inherited bleeding disorder. The data were presented at the 52nd American Society of Hematology (ASH) Annual Meeting and Exposition.
rFVIIa analogue (NN1731)[1]: phase 2 results
Results were presented from a phase 2 trial, adept™1, in which haemophilia patients aged 12 years or older experiencing a joint bleed were randomised to receive up to three doses of NN1731 or NovoSeven®. The trial showed that NN1731 was safe and no antibody formation against NN1731 was seen in the trial. Evaluation of patients who received NN1731at the two highest dose levels showed that 96% of the joint bleeds were well-controlled with the product; the efficacy of NovoSeven® was similar to that observed in previous clinical studies (efficacy in approximately 90% of bleeds).
In addition, the number of adverse events (AEs), including serious adverse events (SAEs), was lower in patients being treated with NN1731 compared to those in the control group. A total of 12 SAEs were reported and all occurred 16 days or longer after exposure to NN1731.
“There were no safety concerns observed in patients at any dose level of NN1731,” said Dr Erich de Paula, of the State University of Campinas in São Paulo, Brazil, who presented the trial during the meeting. “Additionally, the phase 2 trial results demonstrated the potential of the rFVIIa analogue to stop joint bleeds quickly and effectively. These results further support the distinct fast action profile of the rFVIIa analogue in treating bleeds in haemophilia patients with inhibitors.”
NovoSeven® was used as a control in the trial due to its proven efficacy and safety profile. NovoSeven®was specifically developed to treat people with haemophilia A or B with inhibitors to factor VIII or IX replacement.
rFXIII compound: phase 3 results
Results were presented from mentor™1[2], a phase 3 trial examining the efficacy and safety of a recombinant factor XIII (rFXIII) compound for the prevention of bleeds associated with congenital FXIII deficiency, a rare bleeding disorder with about 600–1,000 diagnosed patients worldwide.
In the trial, 41 patients were treated for one year, with rFXIII administered as a preventative, once-monthly replacement therapy for congenital FXIII deficiency. FXIII-deficient patients with no previous history of FXIII treatment were used as a control. Currently, the only treatment available is derived from human blood plasma, which carries an inherent risk of infections.[3]
The trial results demonstrated that treatment with monthly recombinant FXIII injections significantly decreased the number of bleeding episodes requiring treatment compared to the historic control group. Over the course of the treatment period, a total of five bleeding episodes were observed in four patients. All five events were associated with trauma, and were not related to low FXIII activity levels in patients. Additionally, no thromboembolic events or fatal adverse events were reported.
“These data show the potential for rFXIII to become a safe and effective treatment option for patients who would otherwise use treatments at risk for contamination,” said Prof Aida Inbal of the Hemostasis Unit and Hematology Clinic in the Institute of Hematology at Rabin Medical Center in Tel Aviv, Israel. “We think this is an extremely important milestone in the development of a treatment that is not sourced from human plasma for patients suffering from congenital FXIII deficiency.”
Novo Nordisk plans to file for US Food and Drug Administration approval of the rFXIII in the first half of 2011.
Wednesday, August 11, 2010
FDA APPROVES 8 MG VIAL OF NOVOSEVEN®
Novo Nordisk announced today that the U.S. Food and Drug Administration (FDA) has approved NovoSeven® RT (Coagulation Factor VIIa [Recombinant] Room Temperature Stable) in an 8 mg vial size, making the hemophilia A or B with inhibitors treatment available in 1, 2, 5 and 8 mg vials. The 8 mg vial allows a rapid initiation and administration of this medication for those patients who need a larger dose. In addition, FDA has also approved the extension of shelf life for all vial sizes from 24 months to 36 months at room temperature (at or below 77 degrees Fahrenheit).
NovoSeven® RT is specially formulated to treat people with hemophilia A or B with inhibitors. Hemophilia, which is typically diagnosed in childhood, is a chronic, inherited bleeding disorder that occurs when certain blood clotting factors are missing or do not work properly, resulting in easy bruising and prolonged bleeding from trauma. Spontaneous internal bleeding can occur as well, particularly in the joints and muscles. Inhibitors, a serious complication that can occur after treatment, develop in as many as 30 percent of those with hemophilia. In these cases, antibodies form that neutralize or attack the blood coagulation agents contained in the treatment, resulting in joint disease and making it more difficult to manage bleeds.
"When I get a bleed, I want to infuse my treatment right away. The faster I treat a bleed, the sooner I can continue with my daily routine," said Bob Hoyt, a member of the Novo Nordisk Changing Possibilities Coalition. "Using fewer vials will be a positive change for patients." Hoyt has been living with hemophilia with inhibitors most of his life.
For many people living with hemophilia with inhibitors, the 8 mg vial will allow for faster reconstitution - the time it takes to prepare the injection - than their current NovoSeven® RT regimen. Those who previously had to use three vials to deliver an 8 mg dose will now have to reconstitute only one vial. Though the amount of powder in the 8 mg vial is larger than the 5 mg vial, the vial will be the same size and will have a yellow cap to distinguish it from the smaller dose.
"We have heard from patients and physicians alike that when they are treating a bleed, every second counts. We at Novo Nordisk are committed to improving the lives of people living with hemophilia with inhibitors," said Eddie Williams, Vice President, Biopharmaceuticals, at Novo Nordisk. "The NovoSeven® RT 8 mg vial will allow patients to get the medicine they need much faster when experiencing a bleed, without having to manage multiple vials. We're also pleased that it will have a positive environmental impact as well - fewer vials may mean less waste."
The new vial size is expected to be available by November.
NovoSeven® RT is specially formulated to treat people with hemophilia A or B with inhibitors. Hemophilia, which is typically diagnosed in childhood, is a chronic, inherited bleeding disorder that occurs when certain blood clotting factors are missing or do not work properly, resulting in easy bruising and prolonged bleeding from trauma. Spontaneous internal bleeding can occur as well, particularly in the joints and muscles. Inhibitors, a serious complication that can occur after treatment, develop in as many as 30 percent of those with hemophilia. In these cases, antibodies form that neutralize or attack the blood coagulation agents contained in the treatment, resulting in joint disease and making it more difficult to manage bleeds.
"When I get a bleed, I want to infuse my treatment right away. The faster I treat a bleed, the sooner I can continue with my daily routine," said Bob Hoyt, a member of the Novo Nordisk Changing Possibilities Coalition. "Using fewer vials will be a positive change for patients." Hoyt has been living with hemophilia with inhibitors most of his life.
For many people living with hemophilia with inhibitors, the 8 mg vial will allow for faster reconstitution - the time it takes to prepare the injection - than their current NovoSeven® RT regimen. Those who previously had to use three vials to deliver an 8 mg dose will now have to reconstitute only one vial. Though the amount of powder in the 8 mg vial is larger than the 5 mg vial, the vial will be the same size and will have a yellow cap to distinguish it from the smaller dose.
"We have heard from patients and physicians alike that when they are treating a bleed, every second counts. We at Novo Nordisk are committed to improving the lives of people living with hemophilia with inhibitors," said Eddie Williams, Vice President, Biopharmaceuticals, at Novo Nordisk. "The NovoSeven® RT 8 mg vial will allow patients to get the medicine they need much faster when experiencing a bleed, without having to manage multiple vials. We're also pleased that it will have a positive environmental impact as well - fewer vials may mean less waste."
The new vial size is expected to be available by November.
Sunday, April 18, 2010
World Hemophilia Day
On World Hemophilia Day 2010, April 17, meet the many faces of bleeding disorders - united to achieve Treatment for All. Each year hemophilia organizations around the world celebrate World Hemophilia Day, increasing awareness of hemophilia and other bleeding disorders.
The World Federation of Hemophilia (WFH) will launch “The Many Faces of Bleeding Disorders” video podcast on World Hemophilia Day. This video podcast celebrates the whole bleeding disorders community – people with hemophilia and symptomatic carriers, males and females with von Willebrand disease, as well as rarer factor deficiencies, and inherited platelet disorders. It features the impact of these conditions through global patient perspectives and includes a call-to-action for access and improved patient standard of care.
“The goal of the World Federation of Hemophilia is that, one day, treatment will be available for all those with inherited bleeding disorders, regardless of where they live,” said Mark Skinner, WFH president. “Our vision of Treatment for All means expanding services beyond hemophilia.”
Visit www.wfh.org/whd :
for more information about World Hemophilia Day. The World Hemophilia Day website is supported by Bayer, Baxter, and Novo Nordisk. View “The Many Faces of Bleeding Disorders” video podcast, made possible with funding from Baxter Healthcare Corporation, at www.wfh.org/whd : .
The World Federation of Hemophilia (WFH) will launch “The Many Faces of Bleeding Disorders” video podcast on World Hemophilia Day. This video podcast celebrates the whole bleeding disorders community – people with hemophilia and symptomatic carriers, males and females with von Willebrand disease, as well as rarer factor deficiencies, and inherited platelet disorders. It features the impact of these conditions through global patient perspectives and includes a call-to-action for access and improved patient standard of care.
“The goal of the World Federation of Hemophilia is that, one day, treatment will be available for all those with inherited bleeding disorders, regardless of where they live,” said Mark Skinner, WFH president. “Our vision of Treatment for All means expanding services beyond hemophilia.”
Visit www.wfh.org/whd :
for more information about World Hemophilia Day. The World Hemophilia Day website is supported by Bayer, Baxter, and Novo Nordisk. View “The Many Faces of Bleeding Disorders” video podcast, made possible with funding from Baxter Healthcare Corporation, at www.wfh.org/whd : .Thursday, September 3, 2009
Special Challenges of Hemophilia Patients and Their Families
PRINCETON, N.J., Aug. 27 /PRNewswire-FirstCall/ -- More than 160 families of patients living with a rare form of hemophilia are coming together at educational summits sponsored this fall by the National Hemophilia Foundation (NHF) and the Center for Biomedical Continuing Education (CBCE). For the fifth year, the Inhibitor Education Summits will connect patients and their families with expert healthcare professionals and fellow patients to address pertinent topics such as effective pain management, exercise and nutrition, financial security, and psychological wellbeing. The summits are supported by an educational grant from Novo Nordisk.
Hemophilia is a chronic, inherited bleeding disorder that occurs because certain blood clotting factors are missing or do not work properly. Spontaneous internal bleeding can occur, especially in the joints and muscles.
The 2009 summits will take place in Hollywood, C.A., August 27-30 and in Washington, D.C., September 17-20. These summits are the first and only events of their kind that focus specifically on patients with inhibitors. Inhibitors are rare, affecting approximately 800 to 900 Americans. Inhibitors are a serious complication that can develop after treatment in as many as 30 percent of people with hemophilia, most commonly in young children. These patients develop antibodies (or inhibitors) that circulate in the blood stream and actually neutralize, or attack clotting replacements, resulting in an increased risk of bleeding, arthropathy (or joint disease), physical disability and death.
People living with this disease often navigate tremendous challenges, both physically and emotionally. Sid Ramirez, age 21, has lived with hemophilia his entire life. He developed inhibitors at the age of three. His condition led to significant problems with his knee joints, leaving Sid wheelchair bound by age 11. He underwent lateral knee replacement surgery as a teenager, which enabled him to walk again.
"The summits allow others living with inhibitors and their families to learn from each other's experiences and from experts. No one can understand what you go through as well as those who have experienced it themselves and their families," said Sid. "Hemophilia has significance in the decisions I make, but I will not let it be the deciding factor." Currently enrolled in culinary school to pursue his lifelong passion for cooking, Sid is excited about the future ahead.
NHF Chief Executive Officer Val Bias has a special reason for participating in the summits. He's also a person with hemophilia.
"Education has been a cornerstone of the NHF, and the Inhibitor Education Summit is a prime example of that," said Bias, who will also speak at both summits. "We believe it is critical to be able to provide these kinds of programs that help patients and their families achieve greater success in managing inhibitors."
In addition to education for patients and caregivers, there is a comprehensive Youth Camp for children with inhibitors and their siblings. Children attending the Youth Camp will have the opportunity to show their parents all that they have learned during their time at "camp." There is also ample time for networking with other families and "ask the experts" sessions to interact with physicians and other key opinion leaders in the field. Since the Inhibitor Education Summits began in 2005, hundreds of families have benefited from this unique opportunity to learn and connect with others who share their experience.
"Comprehensive management of hemophilia with inhibitors is vital for maximizing the quality of daily life for patients with this difficult condition," said Dr. Guy Young, Director, Hemostasis and Thrombosis Center, Childrens Hospital Los Angeles and co-chair of the summits. "The 2009 Inhibitor Education Summits provide patients and their families with the opportunity to learn effective management strategies, connect with expert medical professionals, and share experiences with other families." Dr. Leonard Valentino, Director, RUSH Hemophilia and Thrombophilia Center, RUSH University Medical Center, is also co-chair of the summits.
For more information about The Inhibitor Education Summits, log onto www.inhibitorsummits.org.
About the National Hemophilia Foundation (NHF)
The National Hemophilia Foundation is dedicated to finding better treatments and cures for bleeding and clotting disorders and to preventing the complications of these disorders through education, advocacy and research. Established in 1948, the National Hemophilia Foundation has chapters throughout the country. Its programs and initiatives are made possible through the generosity of individuals, corporations and foundations as well as through a cooperative agreement with the Centers for Disease Control and Prevention (CDC).
About CBCE(TM) (The Center for Biomedical Continuing Education)
The CBCE(TM) is a full-service provider of accredited CME/CE with a circular scope of capabilities. Since 1999, the CBCE has partnered with clinical experts in solid tumors and hematologic malignancies to develop and implement local, regional, national and international medical education initiatives designed to accelerate the adoption of best practices and clinical breakthroughs in oncology. The CBCE is dedicated to creating rigorous educational programs at the forefront of science, customized according to the target audience. Our unique experience in oncology and hematology has resulted in an in-depth understanding of the nuanced working lives and learning styles of the specialists, nurses, and pharmacists involved in cancer care, an understanding that informs the development of all interventions.
The CBCE vision is to encourage healthcare professionals to commit to change and to define distinct outcomes in their practices that will improve patient care and quality of life.
About Novo Nordisk
Novo Nordisk is a healthcare company with an 86-year history of innovation and achievement in diabetes care. The company has the broadest diabetes product portfolio in the industry, including the most advanced products within the area of insulin delivery systems. In addition to diabetes care, Novo Nordisk has a leading position within areas such as hemostasis management, growth hormone therapy, and hormone therapy for women. Novo Nordisk's business is driven by the Triple Bottom Line: a commitment to economic success, environmental soundness, and social responsibility to employees and customers. With headquarters in Denmark, Novo Nordisk employs more than 27,000 employees in 81 countries, and markets its products in 179 countries. Novo Nordisk's B shares are listed on the stock exchanges in Copenhagen and London. Its ADRs are listed on the New York Stock Exchange under the symbol 'NVO'. For global information, visit novonordisk.com; for United States information, visit www.novonordisk-us.com.
Hemophilia is a chronic, inherited bleeding disorder that occurs because certain blood clotting factors are missing or do not work properly. Spontaneous internal bleeding can occur, especially in the joints and muscles.
The 2009 summits will take place in Hollywood, C.A., August 27-30 and in Washington, D.C., September 17-20. These summits are the first and only events of their kind that focus specifically on patients with inhibitors. Inhibitors are rare, affecting approximately 800 to 900 Americans. Inhibitors are a serious complication that can develop after treatment in as many as 30 percent of people with hemophilia, most commonly in young children. These patients develop antibodies (or inhibitors) that circulate in the blood stream and actually neutralize, or attack clotting replacements, resulting in an increased risk of bleeding, arthropathy (or joint disease), physical disability and death.
People living with this disease often navigate tremendous challenges, both physically and emotionally. Sid Ramirez, age 21, has lived with hemophilia his entire life. He developed inhibitors at the age of three. His condition led to significant problems with his knee joints, leaving Sid wheelchair bound by age 11. He underwent lateral knee replacement surgery as a teenager, which enabled him to walk again.
"The summits allow others living with inhibitors and their families to learn from each other's experiences and from experts. No one can understand what you go through as well as those who have experienced it themselves and their families," said Sid. "Hemophilia has significance in the decisions I make, but I will not let it be the deciding factor." Currently enrolled in culinary school to pursue his lifelong passion for cooking, Sid is excited about the future ahead.
NHF Chief Executive Officer Val Bias has a special reason for participating in the summits. He's also a person with hemophilia.
"Education has been a cornerstone of the NHF, and the Inhibitor Education Summit is a prime example of that," said Bias, who will also speak at both summits. "We believe it is critical to be able to provide these kinds of programs that help patients and their families achieve greater success in managing inhibitors."
In addition to education for patients and caregivers, there is a comprehensive Youth Camp for children with inhibitors and their siblings. Children attending the Youth Camp will have the opportunity to show their parents all that they have learned during their time at "camp." There is also ample time for networking with other families and "ask the experts" sessions to interact with physicians and other key opinion leaders in the field. Since the Inhibitor Education Summits began in 2005, hundreds of families have benefited from this unique opportunity to learn and connect with others who share their experience.
"Comprehensive management of hemophilia with inhibitors is vital for maximizing the quality of daily life for patients with this difficult condition," said Dr. Guy Young, Director, Hemostasis and Thrombosis Center, Childrens Hospital Los Angeles and co-chair of the summits. "The 2009 Inhibitor Education Summits provide patients and their families with the opportunity to learn effective management strategies, connect with expert medical professionals, and share experiences with other families." Dr. Leonard Valentino, Director, RUSH Hemophilia and Thrombophilia Center, RUSH University Medical Center, is also co-chair of the summits.
For more information about The Inhibitor Education Summits, log onto www.inhibitorsummits.org.
About the National Hemophilia Foundation (NHF)
The National Hemophilia Foundation is dedicated to finding better treatments and cures for bleeding and clotting disorders and to preventing the complications of these disorders through education, advocacy and research. Established in 1948, the National Hemophilia Foundation has chapters throughout the country. Its programs and initiatives are made possible through the generosity of individuals, corporations and foundations as well as through a cooperative agreement with the Centers for Disease Control and Prevention (CDC).
About CBCE(TM) (The Center for Biomedical Continuing Education)
The CBCE(TM) is a full-service provider of accredited CME/CE with a circular scope of capabilities. Since 1999, the CBCE has partnered with clinical experts in solid tumors and hematologic malignancies to develop and implement local, regional, national and international medical education initiatives designed to accelerate the adoption of best practices and clinical breakthroughs in oncology. The CBCE is dedicated to creating rigorous educational programs at the forefront of science, customized according to the target audience. Our unique experience in oncology and hematology has resulted in an in-depth understanding of the nuanced working lives and learning styles of the specialists, nurses, and pharmacists involved in cancer care, an understanding that informs the development of all interventions.
The CBCE vision is to encourage healthcare professionals to commit to change and to define distinct outcomes in their practices that will improve patient care and quality of life.
About Novo Nordisk
Novo Nordisk is a healthcare company with an 86-year history of innovation and achievement in diabetes care. The company has the broadest diabetes product portfolio in the industry, including the most advanced products within the area of insulin delivery systems. In addition to diabetes care, Novo Nordisk has a leading position within areas such as hemostasis management, growth hormone therapy, and hormone therapy for women. Novo Nordisk's business is driven by the Triple Bottom Line: a commitment to economic success, environmental soundness, and social responsibility to employees and customers. With headquarters in Denmark, Novo Nordisk employs more than 27,000 employees in 81 countries, and markets its products in 179 countries. Novo Nordisk's B shares are listed on the stock exchanges in Copenhagen and London. Its ADRs are listed on the New York Stock Exchange under the symbol 'NVO'. For global information, visit novonordisk.com; for United States information, visit www.novonordisk-us.com.
Friday, August 7, 2009
Novo Nordisk expects to start a phase 1 trial with a recombinant long-acting factor IX compound
Within the area of haemostasis, Novo Nordisk has initiated a randomised, double-blinded, placebo-controlled, phase 2 trial with rFXIII in cardiac surgery. The aim of the trial is to investigate the safety and efficacy of rFXIII on transfusion needs in patients undergoing heart surgery. The trial is expected to enrol around 400 patients and results are expected early 2011.
The phase 1 trial with subcutaneous injection of rFVIIa has now been completed. While the study showed that subcutaneous dosing is possible, the bioavailability was lower than expected. Hence, Novo Nordisk has decided not to continue into phase 2 clinical development with this mode of administration for this compound and will instead focus on subcutaneous administration of a long-acting rFVIIa expected to enter clinical development this year.
In addition, Novo Nordisk expects to start phase 2 clinical development in the third quarter this year with the long-acting recombinant FVIIa derivative, NN7128, intended for prophylactic treatment of haemophilia patients with inhibitors. The phase 2 trial will involve around 24 patients and results are expected in 2011.
Furthermore, Novo Nordisk expects to start a phase 1 trial with a recombinant long-acting factor IX compound in the third quarter of 2009. The trial is expected to enrol around 20 patients in a dose-finding trial and the study is expected to be completed in mid-2010.
The phase 1 trial with subcutaneous injection of rFVIIa has now been completed. While the study showed that subcutaneous dosing is possible, the bioavailability was lower than expected. Hence, Novo Nordisk has decided not to continue into phase 2 clinical development with this mode of administration for this compound and will instead focus on subcutaneous administration of a long-acting rFVIIa expected to enter clinical development this year.
In addition, Novo Nordisk expects to start phase 2 clinical development in the third quarter this year with the long-acting recombinant FVIIa derivative, NN7128, intended for prophylactic treatment of haemophilia patients with inhibitors. The phase 2 trial will involve around 24 patients and results are expected in 2011.
Furthermore, Novo Nordisk expects to start a phase 1 trial with a recombinant long-acting factor IX compound in the third quarter of 2009. The trial is expected to enrol around 20 patients in a dose-finding trial and the study is expected to be completed in mid-2010.
Thursday, September 18, 2008
Neose selling out to Novo Nordisk, BioGenerix
Neose Technologies Inc. reached agreements Thursday to sell its assets to two foreign drug company collaboration partners for about $43 million.
The struggling Horsham, Pa., biopharmaceutical company stock was trading at 23 cents a share when the deals with Novo Nordisk of Denmark and BioGenerix of Germany were signed.
The asset sales are the initial step in a contemplated liquidation of Neose.
Neose (NASDAQ:NTEC), which specializies in using its enzyme pegylation technology to remodel molecules and develop next-generation therapeutic proteins, has been working with BioGenerix on a treatment for chemotherapy-induced neutropenia (which causes a drop in infection-fighting white blood cells), and with Novo Nordisk on an improved hemostasis compound to inhibit bleeding.
Under the terms of the deal, Neose is retaining certain intellectual property rights, including those related to producing glycolipids, for “future disposition.”
The struggling Horsham, Pa., biopharmaceutical company stock was trading at 23 cents a share when the deals with Novo Nordisk of Denmark and BioGenerix of Germany were signed.
The asset sales are the initial step in a contemplated liquidation of Neose.
Neose (NASDAQ:NTEC), which specializies in using its enzyme pegylation technology to remodel molecules and develop next-generation therapeutic proteins, has been working with BioGenerix on a treatment for chemotherapy-induced neutropenia (which causes a drop in infection-fighting white blood cells), and with Novo Nordisk on an improved hemostasis compound to inhibit bleeding.
Under the terms of the deal, Neose is retaining certain intellectual property rights, including those related to producing glycolipids, for “future disposition.”
Thursday, June 12, 2008
Novo stops NovoSeven trauma Phase III trial
COPENHAGEN, June 11 (Reuters) - Denmark's Novo Nordisk (NOVOb.CO: Quote, Profile, Research) said on Wednesday it stopped a Phase III trial with its NovoSeven drug for treatment of bleeding in severe trauma, but added the decision was not due to safety concerns.
It said the study population had a mortality rate of 10 percent compared to more than 25 percent in the preceding Phase II trial. The study, it said, was unlikely to determine whether NovoSeven would be successful in this treatment area.
"Of course it's not positive but I had not factored in an additional (indication) for NovoSeven," said Sydbank analyst Rune Dahl. "The way to view NovoSeven currently is as (a drug with) stable sales in its existing area, and nothing more." NovoSeven is a haemophilia drug with total sales of 5.9 billion Danish crowns ($1.23 billion) last year.
By 1302 GMT, Novo shares were down 1 percent at 310 crowns on the Copenhagen exchange after briefly falling as much as 4 percent.
A Novo Nordisk spokesman said that the company had not yet decided whether to design a new Phase III study with NovoSeven for bleeding in severe trauma.
In February 2007, Novo Nordisk halted regulatory filing for the drug for bleeding in the brain after initial results from a Phase III clinical trial showed NovoSeven reduces bleeding in the brain but does not improve long-term clinical outcomes.
Monday, June 2, 2008
Novo Nordisk and Neose Announce Completion of Initial Phase 1 Clinical Trial
COPENHAGEN, Denmark & HORSHAM, Pa.--(BUSINESS WIRE)--Novo Nordisk A/S (NYSE:NVO) and Neose Technologies, Inc. (Nasdaq GM:NTEC) today announced that Novo Nordisk has completed the initial Phase 1 clinical trial with NN7128 (GlycoPEGylated Factor VIIa), a long-acting version of NovoSeven® Coagulation Factor FVIIa (Recombinant) administered intravenously. The trial assessed the safety and pharmacokinetics of NN7128 in 30 healthy subjects.
In the trial a significant prolongation of the half-life of NN7128 was observed. Furthermore, single doses of NN7128 were well tolerated with no serious adverse events.
“We are pleased to report the successful completion of this Phase 1 study and to have demonstrated a prolonged half-life of NN7128,” said Søren Bjørn, Corporate Vice President, Biopharm Research at Novo Nordisk. “As we continue our analysis of the data collected in this study, we look forward to presenting the full results at upcoming scientific and medical meetings.”
“We are encouraged by the safety and pharmacokinetic profile that NN7128 has demonstrated in this Phase 1 study,” said George J. Vergis, Ph.D., Neose President and Chief Executive Officer. “We look forward to continued progress in the clinical development of this compound.”
In the trial a significant prolongation of the half-life of NN7128 was observed. Furthermore, single doses of NN7128 were well tolerated with no serious adverse events.
“We are pleased to report the successful completion of this Phase 1 study and to have demonstrated a prolonged half-life of NN7128,” said Søren Bjørn, Corporate Vice President, Biopharm Research at Novo Nordisk. “As we continue our analysis of the data collected in this study, we look forward to presenting the full results at upcoming scientific and medical meetings.”
“We are encouraged by the safety and pharmacokinetic profile that NN7128 has demonstrated in this Phase 1 study,” said George J. Vergis, Ph.D., Neose President and Chief Executive Officer. “We look forward to continued progress in the clinical development of this compound.”
Friday, May 9, 2008
FDA Approves New Formulation of Coagulation Therapy
WASHINGTON (Reuters) - Denmark's Novo Nordisk won U.S. approval to sell a new formulation of a genetically engineered protein therapy that helps the blood clot, regulators said on Friday.
The product, NovoSeven RT, can be stored at room temperature for up to two years, the Food and Drug Administration said. An older formulation had to be refrigerated.
NovoSeven RT is a genetically engineered version of Factor VIIa, a protein found in plasma that is essential for the clotting of blood.
Approved uses of NovoSeven RT include treatment of bleeding, and prevention of surgical bleeding, in certain patients with hemophilia.
Read FDA News HERE
Tuesday, March 18, 2008
Novoseven
Novo Nordisk has the following interesting details plus another video.
Blood use
- Some statistics
Approximately 10,000 units of blood are required per day to meet the demands of hospitals in England and North Wales. Thirty-two thousand units of red blood cells are used per day in the USA and 26.5 million units of blood are transfused per year. Similar requirements exist throughout Europe.
To meet these demands, 2.5 million blood donations are made in the UK every year, and almost 14 million in the USA. At each donation, approximately 475 ml (1 unit) of blood are collected. 1.9 million volunteer donors in the UK and 8 million in the USA (approximately 5% of the population of each country) donate this blood. With 10% of the adult population (260,000) donating blood Denmark has the highest participation rate as well as the highest consumption of blood products per capita in Europe and is primarily due to the high acceptance of blood donation in Denmark.
Donation by apheresis is also becoming increasingly common and essential for the supply of specific components. Apheresis describes any procedure in which blood is drawn from a donor and a component (platelets, plasma or white blood cells) is separated out, with the remaining blood being returned to the body. Apheresis allows the donor's blood volume to replenish itself much more quickly than whole blood donation. One type of apheresis, plasmapheresis, is commonly used in commercial blood banks. In plasmapheresis the plasma is separated from donated blood, the red blood cells being returned to the donor.
How many components can be made from one unit of blood?
Once blood has been tested for infectious agents and blood groups have been determined, it is separated into its useful components, allowing several patients to benefit from one unit of whole blood.
Red cells are used for the treatment of certain types of anaemia, such as bone marrow failure, sickle cell disease and anaemia associated with chronic renal failure. Transfusion is also used to replace lost red cells after accidents, surgery and childbirth. Red cells have a shelf life of up to 42 days, but if fresh-frozen can be stored for up to 10 years.
Platelets are mainly used in the treatment of patients with bone marrow failure or those undergoing chemotherapy. The shelf life of platelets is up to five days.
Fresh frozen plasma (FFP) is normally used after substantial blood loss, for example following childbirth or during cardiac surgery. FFP is also used for the reversal of anticoagulant treatments, burn/shock cases and to replace clotting factors after blood transfusion. Its shelf life is one year.
Processed plasma is also fractionated for the production of factor VIII and factor IX, immunoglobulins and antibodies. Very large 'pools' of donated units of plasma are used in the production of such products.
The main uses of whole blood are: general surgery (23%), general medicine (15%), cardiothoracic surgery (13%), orthopaedics (11%), haematology (9%), accident and emergency (8%).
Expense associated with blood collection
Collecting blood is expensive. The main costs are for staff salaries, transport, storage, and for the screening tests required by individual nations in addition to blood aquisition cost. In Europe, blood is usually screened for:
Type (ABO/Rh)
Antibodies to red cell antigens
Antibodies to HIV-1/2 and HIV antigen
Antibody to HTLV I/II
Hepatitis B surface antigen and antibody to the core antigen
Antibody to hepatitis C
Syphilis
Liver function (alanine aminotransferase)
Blood transfusion
The majority of blood transfusions are heterologous (also referred to as allogenic), which means that the patient receives donated blood from another person (usually an unnamed volunteer donor via a blood bank or transfusion service such as 'The National Blood Service' in the UK or the 'American Association of Blood Banks' (AABB).
Alternatively patients can donate and later, that is during elective surgery, receive back their own blood (autologous transfusion). Autologous blood donors can give blood twice weekly for up to five weeks prior to elective surgery. After donating, the patient is immediately given IV fluids to compensate for the decrease in blood volume. The advantage of this type of donation is that disease transmission and allergic reactions are eliminated - the patient's own blood is the safest blood for their transfusion needs. Autologous transfusion is also useful for patients with rare blood types who might have difficulty in finding a compatible donor. In addition, autologously collected blood can be frozen and stored for up to 10 years. Disadvantages of autologous transfusions include the fact that the donation must be planned in advance, that it may delay surgery, and that certain medical conditions (eg cardiac conditions under which patients cannot tolerate sudden blood loss) disqualify individuals from this type of donation. Units infected with HBV or HIV from autologous donations, are not stored in the blood bank because of the potential risk of clerical error.
Blood that would otherwise be lost during surgery can also be collected and returned to the patient. This is a different kind of autologous donation called perioperative autologous transfusion or PAT and reduces the need for allogenic blood transfusion. Anticoagulant is added to this salvaged blood prior to centrifugation and washing of the resulting packed red cells. These are pumped into a transfusion bag and re-infused into the patient during or after some types of surgery, such as cardiac, gynaecologic and orthopaedic procedures.
Autologous transfusions are generally more costly than traditional heterologous transfusions and are only appropriate in certain types of treatment. Patients must be stable enough to donate their own blood and to cope with the potential anaemia that may be suffered after surgery.
Blood use
- Some statistics
Approximately 10,000 units of blood are required per day to meet the demands of hospitals in England and North Wales. Thirty-two thousand units of red blood cells are used per day in the USA and 26.5 million units of blood are transfused per year. Similar requirements exist throughout Europe.
To meet these demands, 2.5 million blood donations are made in the UK every year, and almost 14 million in the USA. At each donation, approximately 475 ml (1 unit) of blood are collected. 1.9 million volunteer donors in the UK and 8 million in the USA (approximately 5% of the population of each country) donate this blood. With 10% of the adult population (260,000) donating blood Denmark has the highest participation rate as well as the highest consumption of blood products per capita in Europe and is primarily due to the high acceptance of blood donation in Denmark.
Donation by apheresis is also becoming increasingly common and essential for the supply of specific components. Apheresis describes any procedure in which blood is drawn from a donor and a component (platelets, plasma or white blood cells) is separated out, with the remaining blood being returned to the body. Apheresis allows the donor's blood volume to replenish itself much more quickly than whole blood donation. One type of apheresis, plasmapheresis, is commonly used in commercial blood banks. In plasmapheresis the plasma is separated from donated blood, the red blood cells being returned to the donor.
How many components can be made from one unit of blood?
Once blood has been tested for infectious agents and blood groups have been determined, it is separated into its useful components, allowing several patients to benefit from one unit of whole blood.
Red cells are used for the treatment of certain types of anaemia, such as bone marrow failure, sickle cell disease and anaemia associated with chronic renal failure. Transfusion is also used to replace lost red cells after accidents, surgery and childbirth. Red cells have a shelf life of up to 42 days, but if fresh-frozen can be stored for up to 10 years.
Platelets are mainly used in the treatment of patients with bone marrow failure or those undergoing chemotherapy. The shelf life of platelets is up to five days.
Fresh frozen plasma (FFP) is normally used after substantial blood loss, for example following childbirth or during cardiac surgery. FFP is also used for the reversal of anticoagulant treatments, burn/shock cases and to replace clotting factors after blood transfusion. Its shelf life is one year.
Processed plasma is also fractionated for the production of factor VIII and factor IX, immunoglobulins and antibodies. Very large 'pools' of donated units of plasma are used in the production of such products.The main uses of whole blood are: general surgery (23%), general medicine (15%), cardiothoracic surgery (13%), orthopaedics (11%), haematology (9%), accident and emergency (8%).
Expense associated with blood collection
Collecting blood is expensive. The main costs are for staff salaries, transport, storage, and for the screening tests required by individual nations in addition to blood aquisition cost. In Europe, blood is usually screened for:
Type (ABO/Rh)
Antibodies to red cell antigens
Antibodies to HIV-1/2 and HIV antigen
Antibody to HTLV I/II
Hepatitis B surface antigen and antibody to the core antigen
Antibody to hepatitis C
Syphilis
Liver function (alanine aminotransferase)
Blood transfusion
The majority of blood transfusions are heterologous (also referred to as allogenic), which means that the patient receives donated blood from another person (usually an unnamed volunteer donor via a blood bank or transfusion service such as 'The National Blood Service' in the UK or the 'American Association of Blood Banks' (AABB).
Alternatively patients can donate and later, that is during elective surgery, receive back their own blood (autologous transfusion). Autologous blood donors can give blood twice weekly for up to five weeks prior to elective surgery. After donating, the patient is immediately given IV fluids to compensate for the decrease in blood volume. The advantage of this type of donation is that disease transmission and allergic reactions are eliminated - the patient's own blood is the safest blood for their transfusion needs. Autologous transfusion is also useful for patients with rare blood types who might have difficulty in finding a compatible donor. In addition, autologously collected blood can be frozen and stored for up to 10 years. Disadvantages of autologous transfusions include the fact that the donation must be planned in advance, that it may delay surgery, and that certain medical conditions (eg cardiac conditions under which patients cannot tolerate sudden blood loss) disqualify individuals from this type of donation. Units infected with HBV or HIV from autologous donations, are not stored in the blood bank because of the potential risk of clerical error.
Blood that would otherwise be lost during surgery can also be collected and returned to the patient. This is a different kind of autologous donation called perioperative autologous transfusion or PAT and reduces the need for allogenic blood transfusion. Anticoagulant is added to this salvaged blood prior to centrifugation and washing of the resulting packed red cells. These are pumped into a transfusion bag and re-infused into the patient during or after some types of surgery, such as cardiac, gynaecologic and orthopaedic procedures.Autologous transfusions are generally more costly than traditional heterologous transfusions and are only appropriate in certain types of treatment. Patients must be stable enough to donate their own blood and to cope with the potential anaemia that may be suffered after surgery.
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