Sunday, March 23, 2014

Drug companies developing longer-acting clotting agents for hemophiliacs

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.

Saturday, March 22, 2014

Mercy adopts new blood cell transfusion guidelines

SIOUX CITY | Mercy Medical Center -- Sioux City has stepped up efforts to conserve red blood cells.
All hospitals in the Trinity Health network have applied the new national recommendations developed by The Joint Commission and the American Medical Association -- convened Physician Consortium of Performance Improvement that advise adopting a "more restrictive" practice of red blood cell transfusion to produce better patient outcomes.
"We want to give just what is necessary, one unit at a time," said Dr. Gregg Galloway, a pathologist and vice chair of the Infectious Disease Committee at Mercy. "Give him a unit of blood. It will make him feel better -- that's the old paradigm. We don't believe it anymore."
Mercy has always had guidelines in place instructing staff when to give red blood cell transfusions, but Galloway said those rules have significantly changed and are "more restrictive" than they used to be.
"We're doing it because we do believe it is better quality patient care at all health care institutions and because of the cost of the resources," he said.
The new guidelines were released following a September 2012 national summit that convened representatives from 112 professional organizations and associations in effort to curb overuse of five medical treatments. Transfusion of red blood cells in hospitals was one of them.
A decade of studies, according to Galloway, found that when "more restrictive" red blood cell transfusion practices were used, hospitalized patients had lower mortality rates, fewer complications from infectious diseases, less breathing problems and cardiac events, as well as a reduced chance of developing acute respiratory distress syndrome -- a life-threatening lung condition that prevents enough oxygen from getting to the lungs and into the blood.
Galloway said hospital staff give patients red blood cell transfusions based on their hemoglobin (a protein in red blood cells that carries oxygen) and hermatocrit values (percentage of red blood cells found in whole blood). When their hemoglobin value dropped below 10 grams per deciliter (G/dL), the patients received a transfusion. The "more restrictive" guidelines, he said, don't recommend transfusion until hemoglobin values fall between 7 and 8 G/dL.
"Big difference. You're going to use less red cell and units in the hospital," he said. "We predict that overall we'll have a 15 to 20 percent drop over the next year and a half to two years in our red cells that we'll use in our hospital setting."
Once blood is collected at a donation center, it's processed, placed in a refrigerator and stored. Chemical changes, he said, occur in the hemoglobin, and red blood cells lose their malleability.
"Those red blood cells start to change," he said. "They are no longer like the normal red blood cells you and I have floating around in our body. Banked blood is a tissue transplant. Those are somebody else's red cells."
In addition to the new guidelines, Galloway said, improvements in surgical techniques that reduce the need for red blood cell transfusions in the operating room are also contributing to the downward trend.
Surgeons are encouraged to salvage patients' blood in the operating room, a practice that was once reserved for open heart surgery. Now it's being used in trauma, abdominal aneurism and orthopedic surgeries.
"We're trying to save the patient's own blood. We re-process it in the OR and we give it back to them," Galloway said. "We also have new hemostatic medications that we can give patients that keep them from bleeding as much in OR."
Large hospitals, he said, now have blood management programs that staff nurses who review a patient's clinical situation alongside the guidelines. Some patients seeking elective surgery, he said, may have to wait longer to get into the operating room if they lack enough healthy red blood cells.

Adhesion Incidence and Severity Vary by Surgical Procedure

Experts estimate approximately 93% of patients who have undergone laparoscopic surgery develop abnormal fibroid bands that bind organ surfaces to the abdominal wall after a second surgery. Many adhesions are asymptomatic, but in some patients, they can cause pain, small bowel obstruction and other postoperative issues, as well as increase cost and complicate surgical suite workload. As surgeons underestimate the rate of adhesion development given that up to 93% of consent forms don’t address them, the March 2014 issue of Surgery Today contained a systematic review estimating the formation rate, distribution, and severity of postoperative adhesions in abdominal surgery patients.
After reviewing 25 studies published between January 1990 and July 2011, the authors determined the weighted mean formation rate of adhesions after abdominal surgery was 54%. However, different types of surgeries and procedures had different adhesion statistics. Among patients who had gastrointestinal (GI) surgery, 66% developed adhesions. In patients who had gynecologic and urologic surgeries, the rates were 51% and 22%, respectively.
Since those rates were lower than the numbers reported a few decades ago, the authors suggested current practices such as minimizing peritoneal foreign body exposure, careful tissue handling, meticulous hemostasis, specific closure of the peritoneum in caesarean sections, and modern surgical techniques have helped reduce the rate of adhesion.
The authors reported laparoscopic surgery reduced the adhesion formation rate by 25% and also decreased the adhesion severity score for Gl surgery.  They concluded heightening awareness of adhesions is needed to shed light on the complication’s etiology. With better understanding of how and why adhesions form, researchers will be able to develop novel therapeutic approaches, the authors said. They also suggested the potential for adhesions should be addressed clearly in consent forms.