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Passengers aboard the South Korean ferry could not board lifeboats because the vessel had already listed too much, a crew member on the ship said, according to a radio transcript released today.
NASA’s UAVSAR airborne radar will create 3-D maps of earthquake faults over wide
swaths of Haiti (red shaded area) and the Dominican Republic (yellow shaded
area). Image credit: NASA
THURSDAY, April 10, 2014 (HealthDay News) — “Researchers report that an experimental drug has cured more than 95 percent of patients infected with hepatitis C, including some who failed other treatments.
If it wins approval from the U.S. Food and Drug Administration, this new drug, called ABT-450, could potentially compete with another innovative hepatitis C medication that costs $1,000 a day.
These newer, advanced treatments are better-tolerated and easier to take than interferon, the traditional standard treatment for hepatitis C, researchers say……”
Notes from the Field: Increase in Vibrio parahaemolyticus Infections Associated with Consumption of Atlantic Coast Shellfish — 2013
April 18, 2014 / 63(15);335-336
Anna E. Newton, MPH1, Nancy Garrett1, Steven G. Stroika1, Jessica L. Halpin, MS1, Maryann Turnsek1, Rajal K. Mody, MD1
Vibrio parahaemolyticus (Vp) is found naturally in coastal saltwater. In the United States, Vp causes an estimated 35,000 domestically acquired foodborne infections annually (1), of which most are attributable to consumption of raw or undercooked shellfish. Illness typically consists of mild to moderate gastroenteritis, although severe infection can occur. Demographic, clinical, and exposure information (including traceback information on implicated seafood) for all laboratory-confirmed illnesses are reported by state health departments to CDC through the Cholera and Other Vibrio Surveillance system. Vp isolates are distinguished by serotyping (>90 serotypes have been described) and by pulsed-field gel electrophoresis (PFGE).
Vp serotypes O4:K12 and O4:K(unknown) comprise the Pacific Northwest (PNW) strain and, within the United States, had not been associated with shellfish outside the Pacific Northwest before 2012. During May–July 2012, Vp of the PNW strain associated with shellfish from Oyster Bay Harbor in New York caused an outbreak of 28 illnesses in nine states. Simultaneously, Vp of the PNW strain caused an outbreak of illnesses on a cruise ship docked on the Atlantic Coast of Spain; illness was associated with cooked seafood cooled with ice made from untreated local seawater. All Vp isolates from ill persons in the U.S. and Spanish outbreaks that were further subtyped were indistinguishable by PFGE (2).
In 2013, this same indistinguishable strain was traced from shellfish consumed by ill persons to a larger area of the U.S. Atlantic Coast, causing illness in 104 persons from 13 states during May–September (Figure).
The median age of patients was 51 years (range = 22–85 years); 62% were male.
Six (6%) patients were hospitalized; none died.
Multiple outbreaks appeared to be occurring, accounting for many of these illnesses. Illness was associated with consumption of raw shellfish and seafood traceback was reported for 59 (57%) illnesses. Of these illnesses, 51 (86%) involved seafood that could be definitively traced to a single harvest area. The implicated harvest areas were located in Connecticut (20 illnesses), Massachusetts (15), New York (10), Virginia (four), Maine (one), and Washington (one). The remaining eight illnesses with traceback information involved seafood that could not be definitively traced to a single harvest area (locations reported included harvest areas of the Atlantic Coast of the United States and Canada). In response to the illnesses, four Atlantic Coast states closed implicated harvest areas; two issued shellfish recalls (3).
The number of foodborne Vp cases in the United States traced to Atlantic Coast shellfish was threefold greater in 2012 and 2013 compared with the annual average number reported during 2007–2011.
This PNW strain is possibly becoming endemic in an expanding area of the Atlantic Ocean. The mechanisms for this introduction are not known. During the 2014 Vibrio season, beginning in the spring, clinicians, health departments, and fisheries departments should be prepared for the possibility of shellfish-associated diarrheal illness caused by this strain again. Appropriate actions, such as quick closure of implicated harvest areas, will help prevent additional illnesses. The Interstate Shellfish Sanitation Conference maintains a list of shellfish harvest area closures and recalls.* Clinicians seeking an etiology of diarrhea in a patient who has recently consumed raw or undercooked shellfish should notify the microbiology laboratory that Vp is suspected; the use of special culture media (thiosulfate citrate bile salts sucrose) facilitates identification of Vibrio species. Consumers can reduce their risk for Vp infection by avoiding eating raw or undercooked shellfish, especially oysters and clams.†
* Available at http://www.issc.org/closuresreopenings.aspx.
† Additional information available at http://www.cdc.gov/vibrio/investigations/vibriop-09-13/advice-consumers.html.
Incidence and Trends of Infection with Pathogens Transmitted Commonly Through Food — Foodborne Diseases Active Surveillance Network, 10 U.S. Sites, 2006–2013
April 18, 2014 / 63(15);328-332
Stacy M. Crim, MPH1, Martha Iwamoto, MD1, Jennifer Y. Huang, MPH1, Patricia M. Griffin, MD1, Debra Gilliss, MD2, Alicia B. Cronquist, MPH3, Matthew Cartter, MD4, Melissa Tobin-D’Angelo, MD5, David Blythe, MD6, Kirk Smith, DVM7, Sarah Lathrop, PhD8, Shelley Zansky, PhD9, Paul R. Cieslak, MD10, John Dunn, DVM11, Kristin G. Holt, DVM12, Susan Lance, DVM13, Robert Tauxe, MD1, Olga L. Henao, PhD1 (Author affiliations at end of text)
Foodborne disease continues to be an important problem in the United States. Most illnesses are preventable. To evaluate progress toward prevention, the Foodborne Diseases Active Surveillance Network* (FoodNet) monitors the incidence of laboratory-confirmed infections caused by nine pathogens transmitted commonly through food in 10 U.S. sites, covering approximately 15% of the U.S. population. This report summarizes preliminary 2013 data and describes trends since 2006. In 2013, a total of 19,056 infections, 4,200 hospitalizations, and 80 deaths were reported. For most infections, incidence was well above national Healthy People 2020 incidence targets and highest among children aged <5 years. Compared with 2010–2012, the estimated incidence of infection in 2013 was lower for Salmonella, higher for Vibrio, and unchanged overall.† Since 2006–2008, the overall incidence has not changed significantly. More needs to be done. Reducing these infections requires actions targeted to sources and pathogens, such as continued use of Salmonella poultry performance standards and actions mandated by the Food Safety Modernization Act (FSMA) (1). FoodNet provides federal and state public health and regulatory agencies as well as the food industry with important information needed to determine if regulations, guidelines, and safety practices applied across the farm-to-table continuum are working.
FoodNet conducts active, population-based surveillance for laboratory-confirmed infections caused by Campylobacter, Cryptosporidium, Cyclospora, Listeria, Salmonella, Shiga toxin–producing Escherichia coli (STEC) O157 and non-O157, Shigella, Vibrio, and Yersinia in 10 sites covering approximately 15% of the U.S. population (an estimated 48 million persons in 2012).§ FoodNet is a collaboration among CDC, 10 state health departments, the U.S. Department of Agriculture’s Food Safety and Inspection Service (USDA-FSIS), and the Food and Drug Administration (FDA). Hospitalizations occurring within 7 days of specimen collection are recorded, as is the patient’s vital status at hospital discharge, or at 7 days after specimen collection if the patient was not hospitalized. Hospitalizations and deaths that occur within 7 days are attributed to the infection. Surveillance for physician-diagnosed postdiarrheal hemolytic uremic syndrome (HUS), a complication of STEC infection characterized by renal failure, is conducted through a network of nephrologists and infection preventionists and by hospital discharge data review. This report includes 2012 HUS data for persons aged <18 years.
Incidence was calculated by dividing the number of laboratory-confirmed infections in 2013 by U.S. Census estimates of the surveillance area population for 2012.¶ Incidence of culture-confirmed bacterial infections and laboratory-confirmed parasitic infections (e.g., identified by enzyme immunoassay) are reported. A negative binomial model with 95% confidence intervals (CIs) was used to estimate changes in incidence from 2010–2012 to 2013 and from 2006–2008 to 2013 (2). Change in the overall incidence of infection with six key foodborne pathogens was estimated (3). For STEC non-O157, only change since 2010–2012 was assessed because diagnostic practices changed before then; for Cyclospora, change was not assessed because data were sparse. For HUS, incidence was compared with 2006–2008. The number of reports of positive culture-independent diagnostic tests (CIDTs) without corresponding culture confirmation is included for Campylobacter, Listeria, Salmonella, Shigella, STEC, Vibrio, and Yersinia.
Cases of Infection, Incidence, and Trends
In 2013, FoodNet identified 19,056 cases of infection, 4,200 hospitalizations, and 80 deaths (Table). The number and incidence per 100,000 population were Salmonella (7,277 [15.19]), Campylobacter (6,621 [13.82]), Shigella (2,309 [4.82]), Cryptosporidium (1,186 [2.48]), STEC non-O157 (561 [1.17]), STEC O157 (552 [1.15]), Vibrio (242 [0.51]), Yersinia (171 [0.36]), Listeria (123 [0.26]), and Cyclospora (14 [0.03]). Incidence was highest among persons aged ≥65 years for Cyclospora, Listeria, and Vibrio and among children aged <5 years for all the other pathogens.
Among 6,520 (90%) serotyped Salmonella isolates, the top serotypes were Enteritidis, 1,237 (19%); Typhimurium, 917 (14%); and Newport, 674 (10%). Among 231 (95%) speciated Vibrio isolates, 144 (62%) were V. parahaemolyticus, 27 (12%) were V. alginolyticus, and 21 (9%) were V. vulnificus. Among 458 (82%) serogrouped STEC non-O157 isolates, the top serogroups were O26 (34%), O103 (25%), and O111 (14%).
Compared with 2010–2012, the 2013 incidence was significantly lower for Salmonella (9% decrease; CI = 3%–15%), higher for Vibrio (32% increase; CI = 8%–61%) and not significantly changed for other pathogens (Figure 1). Compared with 2006–2008, the 2013 incidence was significantly higher for Campylobacter and Vibrio (Figure 2). The overall incidence of infection with six key foodborne pathogens was not significantly different in 2013 compared with 2010–2012 or 2006–2008.
Compared with 2010–2012, the 2013 incidence of infection with specific Salmonella serotypes was significantly lower for Enteritidis (14% decrease; CI = 0.2%–25%) and Newport (32% decrease; CI = 17%–44%) and not significantly changed for Typhimurium. Compared with 2006–2008, however, the 2013 incidence of infection was significantly changed only for Typhimurium (20% decrease; CI = 10%–28%).
Among 62 cases of postdiarrheal HUS in children aged <18 years (0.56 cases per 100,000) in 2012, 38 (61%) occurred in children aged <5 years (1.27 cases per 100,000). Compared with 2006–2008, the incidence was significantly lower for children aged <5 years (36% decrease; CI = 9%–55%) and for children aged <18 years (31% decrease; CI = 7%–49%).
In addition to culture-confirmed infections (some with a positive CIDT result), there were 1,487 reports of positive CIDTs that were not confirmed by culture, either because the specimen was not cultured at either the clinical or public health laboratory or because a culture did not yield the pathogen. For 1,017 Campylobacter reports in this category, 430 (42%) had no culture, and 587 (58%) were culture-negative. For 247 STEC reports, 59 (24%) had no culture, and 188 (76%) were culture-negative. The Shiga toxin–positive result was confirmed for 65 (34%) of 192 broths sent to a public health laboratory. The other reports of positive CIDT tests not confirmed by culture were of Shigella (147), Salmonella (69), Vibrio (four), Listeria (two), and Yersinia (one).
The incidence of laboratory-confirmed Salmonella infections was lower in 2013 than 2010–2012, whereas the incidence of Vibrio infections increased. No changes were observed for infection with Campylobacter, Listeria, STEC O157, or Yersinia, the other pathogens transmitted commonly through food for which Healthy People 2020 targets exist. The lack of recent progress toward these targets points to gaps in the current food safety system and the need for more food safety interventions.
Although the incidence of Salmonella infection in 2013 was lower than during 2010–2012, it was similar to 2006–2008, well above the national Healthy People target. Salmonella organisms live in the intestines of many animals and can be transmitted to humans through contaminated food or water or through direct contact with animals or their environments; different serotypes can have different reservoirs and sources. Enteritidis, the most commonly isolated serotype, is often associated with eggs and poultry. The incidence of Enteritidis infection was lower in 2013 compared with 2010–2012, but not compared with 2006–2008. This might be partly explained by the large Enteritidis outbreak linked to eggs in 2010.** Ongoing efforts to reduce contamination of eggs include FDA’s Egg Safety Rule, which requires shell egg producers to implement controls to prevent contamination of eggs on the farm and during storage and transportation.†† FDA required compliance by all egg producers with ≥50,000 laying hens by 2010 and by producers with ≥3,000 hens by 2012. Reduction in Enteritidis infection has been one of five high-priority goals for the U.S. Department of Health and Human Services since 2012.§§
In 2013, the incidence of Vibrio infections was the highest observed in FoodNet to date, though still much lower than that of Salmonella or Campylobacter. Vibrio infections are most common during warmer months, when waters contain more Vibrio organisms. Many infections follow contact with seawater (4), but about 50% of domestically acquired infections are transmitted through food, most commonly oysters (5). Foodborne infections can be prevented by postharvest treatment of oysters with heat, freezing, or high pressure, by thorough cooking, or by not eating oysters during warmer months (6). During the summers of 2012 and 2013, many V. parahaemolyticus infections of a strain previously traced only to the Pacific Northwest were associated with consumption of oysters and other shellfish from several Atlantic Coast harvest areas.¶¶ V. alginolyticus, the second most common Vibrio reported to FoodNet in 2013, typically causes wound and soft-tissue infections among persons who have contact with water (7).
The continued decrease in the incidence of postdiarrheal HUS has not been matched by a decline in STEC O157 infections. Possible explanations include unrecognized changes in surveillance, improvements in management of STEC O157 diarrhea, or an actual decrease in infections with the most virulent strains of STEC O157. It is possible that more stool specimens are being tested for STEC, resulting in increased detection of milder infections than in the past. Continued surveillance is needed to determine if this pattern holds.
CIDTs are increasingly used by clinical laboratories to diagnose bacterial enteric infections, a trend that will challenge the ability to identify cases, monitor trends, detect outbreaks, and characterize pathogens (8). Therefore, FoodNet began tracking CIDT-positive reports and surveying clinical laboratories about their diagnostic practices. The adoption of CIDTs has varied by pathogen and has been highest for STEC and Campylobacter. Positive CIDTs frequently cannot be confirmed by culture, and the positive predictive value varies by the CIDT used. For STEC, most specimens identified as Shiga toxin–positive were sent to a public health laboratory for confirmation. However, for other pathogens the fraction of specimens from patients with a positive CIDT sent for confirmation likely is low because no national guidelines regarding confirmation of CIDT results currently exist. As the number of approved CIDTs increases, their use likely will increase rapidly. Clinicians, clinical and public health laboratorians, public health practitioners, regulatory agencies, and industry must work together to maintain strong surveillance to detect dispersed outbreaks, measure the impact of prevention measures, and identify emerging threats.
The findings in this report are subject to at least five limitations. First, health-care–seeking behaviors and other characteristics of the population in the surveillance area might affect the generalizability of the findings. Second, some agents transmitted commonly through food (e.g., norovirus) are not monitored by FoodNet because clinical laboratories do not routinely test for them. Third, the proportion of illnesses transmitted by nonfood routes differs by pathogen; data provided in this report are not limited to infections from food. Fourth, in some fatal cases, infection with the enteric pathogen might not have been the primary cause of death. Finally, changes in incidence between periods can reflect year-to-year variation during those periods rather than sustained trends.
Most foodborne illnesses can be prevented, and progress has been made in decreasing contamination of some foods and reducing illness caused by some pathogens since 1996, when FoodNet began. More can be done; surveillance data provide information on where to target prevention efforts. In 2011, USDA-FSIS tightened its performance standard for Salmonella contamination of whole broiler chickens; in 2013, 3.9% of samples tested positive (Christopher Aston, USDA-FSIS, Office of Data Integration and Food Protection; personal communication; 2014). Because most chicken is purchased as cut-up parts, USDA-FSIS conducted a nationwide survey of raw chicken parts in 2012 and calculated an estimated 24% prevalence of Salmonella (9). In 2013, USDA-FSIS released its Salmonella Action Plan that indicates that USDA-FSIS will conduct a risk assessment and develop performance standards for poultry parts during 2014, among other key activities (10). The Food Safety Modernization Act of 2011 gives FDA additional authority to regulate food facilities, establish standards for safe produce, recall contaminated foods, and oversee imported foods; it also calls on CDC to strengthen surveillance and outbreak response (1). For consumers, advice on safely buying, preparing, and storing foods prone to contamination is available online.
1Division of Foodborne, Waterborne, and Environmental Diseases, National Center for Emerging and Zoonotic Infectious Diseases, CDC; 2California Department of Public Health; 3Colorado Department of Public Health and Environment; 4Connecticut Department of Public Health; 5Georgia Department of Public Health; 6Maryland Department of Health and Mental Hygiene; 7Minnesota Department of Health; 8University of New Mexico; 9New York State Department of Health; 10Oregon Health Authority; 11Tennessee Department of Health; 12Food Safety and Inspection Service, US Department of Agriculture; 13Center for Food Safety and Applied Nutrition, Food and Drug Administration (Corresponding author: Olga L. Henao, email@example.com, 404-639-3393)
Workgroup members, Foodborne Diseases Active Surveillance Network (FoodNet), Emerging Infections Program. Communications team, Division of Foodborne, Waterborne, and Environmental Diseases, National Center for Emerging and Zoonotic Diseases; Enteric Diseases Laboratory Branch, Division of Foodborne, Waterborne, and Environmental Diseases, National Center for Emerging and Zoonotic Diseases, CDC.
* Additional information available at http://www.cdc.gov/foodnet.
† The overall incidence of infection combines data for Campylobacter, Listeria, Salmonella, STEC O157, Vibrio, and Yersinia, six key bacterial pathogens for which >50% of illnesses are estimated to be transmitted by food.
§ FoodNet personnel regularly contact clinical laboratories to ascertain all laboratory-confirmed infections in residents of the surveillance areas.
¶ Final incidence rates will be reported when population estimates for 2013 are available.
** Additional information available at http://www.cdc.gov/salmonella/enteritidis/index.html.
†† Additional information available at http://www.fda.gov/food/guidanceregulation/guidancedocumentsregulatoryinformation/eggs/ucm170615.htm.
§§ Additional information available at http://www.hhs.gov/strategic-plan/appendixb3.html.
¶¶ Additional information available at http://www.cdc.gov/vibrio/investigations/index.html.
What is already known on this topic?
The incidences of infection caused by Campylobacter, Salmonella, Shiga toxin–producing Escherichia coli O157, and Vibrio are well above their respective Healthy People 2020 targets. Foodborne illness continues to be an important public health problem.
What is added by this report?
In 2013, a total of 19,056 infections, 4,200 hospitalizations, and 80 deaths were reported to the Foodborne Diseases Active Surveillance Network (FoodNet). For most infections, incidence was highest among children aged <5 years. In 2013, compared with 2010–2012, the estimated incidence of infection was unchanged overall, lower for Salmonella, and higher for Vibrio infections, which have been increasing in frequency for many years. The number of patients being diagnosed by culture-independent diagnostic tests (CIDT) is increasing.
What are the implications for public health practice?
Reducing the incidence of foodborne infections requires greater commitment and more action to implement measures to reduce contamination of food. Monitoring the incidence of these infections is becoming more difficult because some laboratories are now using CIDTs, and some do not follow up a positive CIDT result with a culture.
|TABLE. Number of cases of culture-confirmed bacterial and laboratory-confirmed parasitic infection, hospitalizations, and deaths, by pathogen — Foodborne Diseases Active Surveillance Network, United States, 2013*|
|Abbreviations: N/A = not available; STEC = Shiga toxin–producing Escherichia coli.* Data for 2013 are preliminary.
† Per 100,000 population.
§ Healthy People 2020 objective targets for incidence of Campylobacter, Listeria, Salmonella, STEC O157, Vibrio, and Yersinia infections per 100,000 population.
¶ No national health objective exists for these pathogens.
* No significant change = 95% CI is both above and below the no change line; significant increase = estimate and entire CI are above the no change line; significant decrease = estimate and entire CI are below the no change line.
Alternate Text: The figure above shows estimated percentage change in incidence of culture-confirmed bacterial and laboratory-confirmed parasitic infections in 2013 compared with average annual incidence during 2010–2012, by pathogen, in the United States. Compared with 2010–2012, the 2013 incidence was significantly lower for Salmonella (9% decrease; 95% confidence interval = 3%–15%), higher for Vibrio (32% increase; 95% confidence interval = 8%–61%) and not sig¬nificantly changed for other pathogens.
† The position of each line indicates the relative change in the incidence of that pathogen compared with 2006–2008. The actual incidences of these infections cannot be determined from this figure.
Alternate Text: The figure above shows relative rates of culture-confirmed infections with Campylobacter, Shiga toxin–producing Escherichia coli O157, Listeria, Salmonella, and Vibrio compared with 2006–2008 rates, by year, in the United States during 2006–2013. Compared with 2006–2008, the 2013 incidence was significantly higher for Campylobacter and Vibrio.
Cost of Treatment May Influence Doctors
By ANDREW POLLACK
“Saying they can no longer ignore the rising prices of health care, some of the most influential medical groups in the nation are recommending that doctors weigh the costs, not just the effectiveness of treatments, as they make decisions about patient care.
The shift, little noticed outside the medical establishment but already controversial inside it, suggests that doctors are starting to redefine their roles, from being concerned exclusively about individual patients to exerting influence on how health care dollars are spent.
“We understand that we doctors should be and are stewards of the larger society as well as of the patient in our examination room,” said Dr. Lowell E. Schnipper, the chairman of a task force on value in cancer care at the American Society of Clinical Oncology.
In practical terms, new guidelines being developed by the medical groups could result in doctors choosing one drug over another for cost reasons or even deciding that a particular treatment — at the end of life, for example — is too expensive. In the extreme, some critics have said that making treatment decisions based on cost is a form of rationing…….”
Hemophilia Treatments Have Come a Long Way
Gone are the days when a hemophilia diagnosis meant you could not live a normal life. Now more treatments are approved by the Food and Drug Administration (FDA), and people with the condition can better manage bleeding. That’s good news as thousands observe World Hemophilia Day on April 17, 2014.
Hemophilia is a rare bleeding disorder. It is usually hereditary, but it can be acquired in rare cases if a person’s body develops antibodies that attack clotting factors in the bloodstream. Hereditary hemophilia usually occurs in males and currently affects about 23,500 Americans.
People with hemophilia are lacking one or more important clotting factors, which are proteins needed for blood clotting. Several types of clotting factors exist and there are two main types of hemophilia, says Nisha Jain, M.D., chief of the Clinical Review Branch in FDA’s Office of Blood Research and Review. Hemophilia A occurs when people have low levels, or missing, clotting factor VIII (8). Hemophilia B occurs when people have low, or missing, clotting factor IX (9).
People with hemophilia may bleed for a longer time than others after injury or surgery. They may also have internal bleeding—especially in knees, ankles, and elbows—that can damage organs and tissues and even be life-threatening.
“We have seen shifting toward the prevention of bleeds,” says Nisha Jain, M.D., chief of the Clinical Review Branch in FDA’s Office of Blood Research and Review.
At present hemophilia is not curable, but treatments have come a long way, Jain says.
Hemophilia is sometimes called the “Royal Disease” because it affected the royal family of England during the 1800s. By the 1960s, hemophilia was treated with whole blood or fresh plasma. But these treatments didn’t have enough factor VIII or IX proteins to stop serious internal bleeding.
Now the primary type of hemophilia treatment is replacement therapy: Concentrates of clotting factor VIII (for hemophilia A) or clotting factor IX (for hemophilia B) are injected into a patient’s vein to replace low or missing factor.
These concentrates have traditionally been made from human blood. Today, an increasing number are made using recombinant DNA technology (a form of artificial DNA), with some made without any material sourced from humans or animals. The final product is a powder that is mixed with sterile water before use.
Some people have regular preventive or “prophylactic” therapy to prevent bleeding. This can be done in less than five minutes, excluding prep time, Jain explains. Others have therapy only as needed.
The type and frequency of treatment varies depending on the severity of hemophilia, which can be classified as mild, moderate or severe.
People with mild hemophilia have 6% to 49% of the normal levels of clotting factors in their blood. They generally only have bleeding problems after serious injury or surgery.
Those with moderate hemophilia—about 15 percent of the hemophilia population, according to the National Hemophilia Foundation—have 1% to 5% of normal clotting factor levels. They can have bleeding problems after injury and spontaneously.
Those with severe hemophilia—about 60% of the hemophilia population, per NHF—have less than 1% of normal clotting factor levels. They bleed after injury and may have frequent spontaneous bleeding, including bleeds into joints and muscles. In addition to factor replacement, pain medication and physical therapy are also used to lessen pain and swelling if joint bleeds occur.
“The mild patients rarely need treatment. And moderate patients are the same,” explains Jain. In certain situations, such as before dental work, patients with mild hemophilia receive Desmopressin (DDAVP) a man-made hormone that increases the level of factor in the blood. “Patients with severe hemophilia are those who really need treatment to prevent or resolve bleeds.”
Doctors, particularly hematologists who specialize in the study of blood, tend to identify people with severe hemophilia early. “Patients can be diagnosed as infants during circumcision,” says Jain. Then families can work with hematologists and hematology treatment centers.
“In recent times, we have seen shifting toward the prevention of bleeds,” Jain says. “You want to prevent bleeding that causes joint damage. Once joint damage starts, it is difficult to stop progression unless bleeding into joints is reduced.”
The treatments for hemophilia continue to improve, and FDA has approved many replacement factors in recent years. For instance, in March 2014 it approved Alprolix, the first Hemophilia B treatment designed to require less frequent injections when used to prevent or reduce the frequency of bleeding. It also recently approved Rixubis—a factor IX product—to control, prevent and reduce bleeding associated with hemophilia B. And the agency approved Novoeight, a factor VIII product, for control of bleeding and a routine prophylaxis treatment for adults, adolescents and children with hemophilia A.
Of course, the agency continues to carry out its broad responsibility to regulate medicines made from blood and blood components, including clotting factors. Today, due to strengthened FDA safeguards and oversight, these products are safer than they have ever been.
“Patients should stay informed about various treatment options,” Jain adds, “and should consult with their health care providers to obtain, and follow, a comprehensive management plan.”
She says those with hemophilia tend to be well-educated about their health and notes, “With appropriate treatment and care, people with hemophilia can, and do, live normal lives.”
This article appears on FDA’s Consumer Updates page, which features the latest on all FDA-regulated products.
April 16, 2014