Rotavirus vaccine effective in reducing pediatric deaths, illness in developing nations

January 28, 2010

Rotavirus vaccine effective in reducing pediatric deaths, illness in developing nations

Rotavirus vaccine use likely contributed to a reduction in pediatric deaths in Mexico and a reduction in severe gastroenteritis in vaccinated African infants, according to two studies published today in The New England Journal of Medicine. However, a brief report in the same journal also cautions against use of rotavirus vaccines in children with severe combined immunodeficiency.

Researchers from the National Center for Child and Adolescent Health, Ministry of Health, Mexico City and the CDC said that a rotavirus vaccination program introduced in Mexico in February 2006 led to a marked reduction in deaths from diarrhea among young children.

The researchers compared annual deaths from diarrhea before and after the immunization program began. The researchers noted that for the three years before the vaccination program began, the median annual number of diarrhea-related deaths among children younger than 5 was 1,793, for a mortality rate of 18.1 deaths per 100,000. That number dropped to 1,118 deaths in 2008, which yielded a mortality rate of 11.8 per 100,000 children. The researchers said diarrhea-related mortality was 29% lower for children between the ages of 12 and 23 months, few of whom were age-eligible for vaccination, but mortality among unvaccinated children between the ages of 24 and 59 months did not change significantly.

The researchers noted that their study findings were limited in that it was not possible to attribute the reduction in deaths to vaccination, because precise vaccine coverage information is lacking and other changes, like improved hand hygiene, may have affected the rates.

In the second study, researchers in South Africa and Malawi enrolled 4,939 infants in clinical trials examining the efficacy of GlaxoSmithKline’s rotavirus vaccine, Rotarix.

Healthy babies were randomly assigned (1:1:1) to receive placebo followed by two or three doses of vaccine, or three doses of placebo, respectively, at 6, 10 and 14 weeks of age with routine childhood vaccines including oral poliovirus vaccine.

The researchers in that study noted rotavirus-related gastroenteritis occurred in 4.9% of the placebo group and in 1.9% of the pooled vaccine group, yielding a statistically significant vaccine efficacy of 61.2%. The researchers said efficacy against all-cause severe gastroenteritis was 30.2%.

In a brief report accompanying the two journal articles, researchers from Baylor College of Medicine noted that it is important to remember that the rotavirus vaccines can actually cause the disease in infants born with severe combined immunodeficiency.

In the report, experts examined three cases in which infants developed rotavirus disease after receiving the live attenuated rotavirus vaccine.

“All three infants (in the study) were vaccinated before they were diagnosed with severe combined immunodeficiency,” Paula Hertel, MD, assistant professor of pediatrics-gastroenterology at BCM and Texas Children’s Hospital, and study researcher said in a press release. “If the children could have been caught in a screening test done within days of birth, they may not have received the vaccine.”

While current routine newborn screening does test for many diseases, severe combined immune deficiency is not one of them, the researchers in the brief report noted.

Usually, experts recommend that children with severe combined immune deficiency not receive live vaccines. However, this vaccine must be given before most children are diagnosed with the immune disorder. The American College of Medical Genetics recently recommended that severe combined immunodeficiency be included as a part of the newborn screen.

Scientists analyzed the viral genetic material in stool specimens from the three children. This enabled them to determine that the rotavirus was of vaccine origin. This study led to a change in the vaccine exclusions listed on the vaccine manufacturer’s label to include a history of severe combined immunodeficiency.

The children did not successfully fight the infection until they underwent bone marrow transplantation or enzyme replacement therapy that gave them a functioning immune system, researchers noted.

The vaccine is very important for healthy infants, and in the United States, rotavirus cases decreased by 50% after the first season of vaccinations against the illness, said Hertel.

linkback url: http://www.pediatricsupersite.com/view.aspx?rid=60461

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Severe Combined Immunodeficiency Added to Newborn Screening Panel

January 28, 2010

Severe Combined Immunodeficiency Added to Newborn Screening Panel

Laurie Barclay, MD

January 28, 2010 — A unanimous vote on January 21 by the Secretary’s Advisory Committee for Heritable Disorders in Newborns and Children (ACHDNC) resulted in the committee’s recommendation to add severe combined immunodeficiency (SCID) to the uniform newborn screening panel. This addition to the mandatory newborn screening panel performed by state public health programs is the first since September 2005.

“[The Advisory Committee] has carefully reviewed the detailed, updated evidence review for SCID and related T-lymphocyte deficiencies and found the condition(s) ready to be added to the uniform and secondary panel,” ACHDNC Chairman R. Rodney Howell, MD, said in a news release. “The Advisory Committee, however, understands that there will be additional work from the [National Institutes of Health], [the Health Resources and Services Administration,] and the [Centers for Disease Control and Prevention] to be reported back to the Committee as this newborn screening proceeds.”

SCID, which has an estimated incidence of 1 in 100,000 to 1 in 40,000 newborns, is a group of disorders characterized by the absence of an immune system, resulting in recurrent infections and early childhood mortality unless treated in the first months of life.

Cost-effective methods for universal screening for SCID were developed in Wisconsin and Massachusetts, and several other states have begun training laboratory personnel to start SCID screening.

“This recommendation by the [Advisory] Committee is significant for both newborn screening and for the evidence-based process for decision making,” said Sharon Terry, MA, president and chief executive officer of Genetic Alliance and ACHDNC liaison. “The recommendation includes surveillance, education and quality control, to be contributed by the National Institutes of Health, Health Resources and Services Administration and Centers for Disease Control and Prevention, and thereby supports the development of a systems approach in newborn screening.”

There is currently a formal process for individuals or organizations to nominate a heritable disorder to be considered by the ACHDNC for inclusion in the recommended uniform screening panel. Of 6 conditions (Fabry disease, Krabbe disease, Niemann-Pick disease, Pompe disease, SCID, and spinal muscular atrophy) brought to the advisory committee since it adopted a list of 29 recommended conditions in September 2005, only SCID has been recommended for addition to the uniform screening panel.

“[The] unanimous vote to add SCID to the National Newborn Screening Core Panel was a milestone victory,” said Vicki Modell, cofounder of the Jeffery Modell Foundation. “We thank the Committee for their positive decision on behalf of all the babies with SCID that we lost, the babies today who have hope, and for all the future babies yet to be born, who will have a really good chance at life.”

linkback url: http://www.medscape.com/viewarticle/716046


Enzon Stockholders Approve Sale of Specialty Pharmaceutical Business

January 27, 2010

Enzon Stockholders Approve Sale of Specialty Pharmaceutical Business

BRIDGEWATER, NJ – January 27, 2010 – Enzon Pharmaceuticals, Inc. (Nasdaq: ENZN) (“Enzon”) announced that at a special meeting held this morning, its stockholders approved the sale of Enzon’s specialty pharmaceutical business to the sigma-tau Group (“sigma-tau”).

Enzon’s specialty pharmaceutical business includes four marketed products, Oncaspar®, Adagen®, DepoCyt®, and Abelcet®, as well as a manufacturing facility in Indianapolis, Indiana.

As previously announced, Enzon has entered into a definitive agreement to sell its specialty pharmaceutical business to sigma-tau for $300 million plus an additional amount of up to $27 million based on success milestones. Enzon will also receive royalties of 5 to 10 percent on incremental net sales above a 2009 baseline amount from Enzon’s four marketed specialty pharmaceutical products through 2014.

The asset sale is expected to be completed later this week.

linkback url: http://www.enzon.com/index.php?id=208


Federal Advisory Committee Recommends Severe Combined Immunodeficiency (SCID) for Universal Newborn Screening

January 27, 2010

Untitled DocumentWed Jan 27 08:16:34 2010 Pacific Time

Federal Advisory Committee Recommends Severe Combined Immunodeficiency (SCID) for Universal Newborn Screening

WASHINGTON, Jan. 27 (AScribe Newswire) — In a historic vote on January 21, 2010, the Secretary’s Advisory Committee for Heritable Disorders in Newborns and Children (ACHDNC) unanimously agreed to recommend the addition of Severe Combined Immunodeficiency (SCID) to the uniform newborn screening panel. The ACHDNC adopted a list of 29 recommended conditions in September 2005. Since that time, SCID is the first condition to be added to this list for inclusion into mandatory newborn screening conducted by state public health programs.

SCID is a group of disorders characterized by the absence of an immune system, causing infants with SCID to develop recurrent infections, leading to death in early childhood. SCID affects a minimum of one in 100,000 newborns; however, some studies estimate that the actual number is closer to one in 40,000. Treatment in the first months after birth can prolong life and prevent infections. Wisconsin and Massachusetts both developed cost-effective methods for universal screening for SCID, and a number of other states have started to train their laboratory personnel to start screening for SCID.

Dr. Rebecca Buckley of Duke University Medical Center published the first article showing the effectiveness of early bone marrow transplantation as a treatment for SCID in 1999 in The New England Journal of Medicine. After working for 13 years to get SCID added to the recommended panel, the vote came as “a dream come true.”

The Chairman of the ACHDNC, Dr. R. Rodney Howell, noted that “[the Advisory Committee] has carefully reviewed the detailed, updated evidence review for SCID and related T-lymphocyte deficiencies and found the condition(s) ready to be added to the uniform and secondary panel. The Advisory Committee, however, understands that there will be additional work from the NIH, HRSA and the CDC to be reported back to the Committee as this newborn screening proceeds.”

The ACHDNC was established in February 2003 to advise the Secretary of the U.S. Department of Health and Human Services regarding the most appropriate application of universal newborn screening tests, technologies, policies, guidelines and standards for effectively reducing morbidity and mortality in newborns and children having, or at risk for, heritable disorders. There is currently a formal process for individuals or organizations to nominate a heritable disorder to be considered for inclusion in the recommended uniform screening panel (http://www.hrsa.gov/heritabledisorderscommittee/nominate.htm).

“This recommendation by the [Advisory] Committee is significant for both newborn screening and for the evidence-based process for decision making,” declared Sharon Terry, president and chief executive officer of Genetic Alliance, who serves as a liaison on the ACHDNC. “The recommendation includes surveillance, education and quality control, to be contributed by the National Institutes of Health, Health Resources and Services Administration and Centers for Disease Control and Prevention, and thereby supports the development of a systems approach in newborn screening.”

To date, six conditions have been brought to the Advisory Committee: Fabry Disease, Krabbe Disease, Niemann-Pick Disease, Pompe Disease, Severe Combined Immunodeficiency (SCID), and Spinal Muscular Atrophy (SMA). Thus far, only SCID has been recommended for addition to the uniform screening panel. By law, the Secretary of Health and Human Services must respond to the recommendation within 180 days.

Vicki Modell, co-founder of the Jeffery Modell Foundation, stated that “[the] unanimous vote to add SCID to the National Newborn Screening Core Panel was a milestone victory. We thank the Committee for their positive decision on behalf of all the babies with SCID that we lost, the babies today who have hope, and for all the future babies yet to be born, who will have a really good chance at life.”

ABOUT GENETIC ALLIANCE: Genetic Alliance transforms health through genetics, promoting an environment of openness centered on the health of individuals, families, and communities. Genetic Alliance brings together diverse stakeholders that create novel partnerships in advocacy; integrates individual, family, and community perspectives to improve health systems; and revolutionizes access to information to enable translation of research into services and individualized decision making. For more information about Genetic Alliance, visit http://www.geneticalliance.org .

– – – –

CONTACTS: Natasha Bonhomme, nbonhomme@geneticalliance.org or 202-966-5557 x211

Tiphane Turpin, tturpin@geneticalliance.org or 202-966-5557 x212

Media Contact: See above.

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Senator Skelos and Senator Hannon Introduce Bill to Expand New Yorks Newborn Screening Program

January 27, 2010

Senator Skelos and Senator Hannon Introduce Bill to Expand New Yorks Newborn Screening Program

January 26, 2010

(New York,N.Y.)-Senator Dean Skelos and Senator Kemp Hannon today announced that they have introduced legislation to strengthen New York State’s Newborn Screening Program by adding five rare genetic diseases to the list of disorders that infants are tested for at birth. Early detection and treatment are critical for babies afflicted with these diseases, which can be debilitating and deadly if undiagnosed.

The legislation (S.6656) adds the following diseases to the list of neonatal testing required in New York State: Pompe disease, Fabry disease, Niemann-Pick disease, Gaucher disease, and Severe Combined Immunodeficiency Disease (SCID). Pompe disease is a physically debilitating and often fatal neuromuscular disorder which is a highlighted in the new Harrison Ford film, “Extraordinary Measures.”

Senator Skelos (R-C-I-Rockville Centre) said, “New parents in New York State should have the benefit of knowing that their infants are being tested for as many diseases as possible at birth. A simple blood test can determine if a baby has one of these rare disorders, which can cause abnormal physical development, mental retardation or other problems if untreated. Early diagnosis and medical treatment can often prevent serious illness.”

Senator Hannon (6th SD-Garden City), Ranking Member of the Senate Health Committee, said “This public health initiative is a crucial step toward identifying and subsequently providing the necessary care for those infants stricken with serious illness, whom might otherwise go untreated. Therefore, I commend Senator Skelos for this important amendment to the current Public Health Law, and provide my full support for the enactment of his proposal.”

New York State’s Newborn Screening Program was established in 1965 to ensure that all newborns are tested for rare disorders. Early diagnosis and medical treatment have been proven to prevent or lessen the severity of many diseases and conditions.

The screening involves a simple heel prick with a small needle to gather blood. Many conditions can be identified with just a tiny sample of blood.

Over the past 45 years, diseases and conditions have been added to the State’s program to keep New York State at the forefront of newborn testing. Several years ago, New York State added Krabbe disease, a lysosomal disorder which killed the son of former Buffalo Bills quarterback Jim Kelly, to the State’s Screening Program. As of 2010, infants born in New York are tested for more than 40 disorders.

SCID, commonly referred to as the “Bubble Boy Disease,” makes sufferers highly susceptible to life-threatening infections by viruses, bacteria, and fungi. SCID can be cured with a bone marrow transplant if diagnosed in the first few weeks or months of life. Children with untreated SCID rarely live past the age of two.

Fabry disease results from a buildup of a particular type of fat in the body’s cells, and causes pain in the hands and feet, hearing loss, dark red spots on the skin and can cause strokes, heart attacks and damage to the kidneys. Niemann-Pick, a condition in which harmful amounts of lipids accumulate in the spleen, liver, lungs, bone marrow and brain, is classified by three different types. Most children affected by type A Niemann-Pick do not survive past early childhood. Gaucher disease, another genetic disorder, affects the body’s organs and tissues.

Linkback url: http://www.newsli.com/2010/01/26/senator-skelos-and-senator-hannon-introduce-bill-to-expand-new-yorks-newborn-screening-program/


Immune Deficiency Foundation Praises ACHDNC Recommendation for SCID Screening in Newborns

January 27, 2010

Immune Deficiency Foundation Praises ACHDNC Recommendation for SCID Screening in Newborns

WASHINGTON, Jan. 25 /PRNewswire/ — On January 21, 2010, the Advisory Committee on Heritable Disorders in Newborns and Children voted unanimously to add screening for Severe Combined Immune Deficiency or SCID — commonly known as bubble boy disease — to the core panel for universal screening of all newborns in the United States.

SCID is a primary immunodeficiency; affected infants lack T lymphocytes, the white blood cells that help resist infections due to a wide array of viruses, bacteria and fungi. Babies with SCID appear healthy at birth, but without early treatment, most often by bone marrow transplant from a healthy donor, these infants cannot survive.

The Advisory Committee’s policy recommendation will now be presented to Kathleen Sebelius, Secretary of Health and Human Services. Ms. Sebelius has 180 days to consider and respond to the committee’s proposal.

Jennifer Puck, Professor of Pediatrics at the University of California, San Francisco and an expert in the field, nominated SCID for consideration by the Committee after pioneering development of a test that can detect SCID in the dried blood spot filter cards that are currently collected from all babies to screen for a variety of inborn disorders.

“The Advisory Committee has taken a momentous step forward for the primary immunodeficiency community,” said Marcia Boyle, President & Founder of the Immune Deficiency Foundation (IDF), the national patient organization for persons with primary immunodeficiency diseases. “The IDF has strongly supported and worked tirelessly toward this goal for many years. But it is imperative that we sustain this momentum by working to establish newborn screening programs in all 50 states.”

The Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) was chartered in February 2003 to perform evidence-based reviews and advise the Secretary regarding application of new screening tests, technologies, policies, guidelines and standards for effectively reducing morbidity and mortality in newborns and children having, or at risk for, heritable disorders. SCID is the first new disease to be recommended for addition to the federal uniform core-screening panel by the evidence-based Committee review process.

“Although this recommendation has been in development for two years,” said Dr. Amy Brower, parent, researcher and former ACHDNC committee member, “and it may take several more years to implement screening in all 50 states and US territories, we must work to reduce the time from this recommendation to the widespread adoption of testing and treatment.”

In 2004, Duke University Professor of Pediatrics and Immunology, Dr. Rebecca H. Buckley — currently the chair of the Immune Deficiency Foundation’s Medical Advisory Committee and an internationally known expert on SCID —  testified before the ACHDNC (to which she was subsequently appointed in 2007).

Dr. Buckley informed the panel that SCID is a pediatric emergency. If a baby with SCID receives a bone marrow transplant in the first 3.5 months of life, the survival rate can be as high as 94 percent. However, the survival rate drops to less than 70 percent for infants who are transplanted after that age. The main causes for the drop in survival rate are serious infections babies with SCID develop prior to transplantation.

“As the parent of a child who was diagnosed with SCID only after he became critically ill,” said Barb Ballard, a member of the IDF Board of Trustees, “I have to say I am immensely pleased with the recommendation of the committee.”

In addition to the IDF, the Jeffrey Modell Foundation and members of the public whose children were affected with SCID argued in favor of SCID screening. One child born in Wisconsin, where pilot SCID screening is ongoing, was diagnosed at 9 days of age and successfully cured by timely bone marrow transplantation, while another child born in Oregon, where screening does not take place, developed failure to thrive and multiple infections and did not survive.

“Our goal is to have Newborn Screening for SCID passed in all 50 states,” said Heather Smith, co-founder of SCID Angels for Life, who lost her six-month-old son, Brandon, to this devastating disease.

“We have already lost way too many children to this disease,” Ms. Smith concluded, “and newborn screening is the only way we can put an end to the pain and suffering these children are forced to endure.”

The Immune Deficiency Foundation is the national patient organization dedicated to improving the diagnosis, treatment and quality of life of persons with primary immunodeficiency diseases through advocacy, education and research. For information on this media release, please contact Marcia Boyle, president, at 410-321-6647 or idf@primaryimmune.org. For further information please visit the IDF website at www.primaryimmune.org, or call IDF at 800-296-4433.

SOURCE Immune Deficiency Foundation

RELATED LINKS
http://www.primaryimmune.org

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Stem cell surgery saved Waynesboro girl’s life

January 22, 2010

Stem cell surgery saved Waynesboro girl’s life

By IRIS HERSH Staff writer

Stem cell transplants have been used since the 1970s to save the lives of cancer victims and children with inherited disorders of the immune systems.

One fortunate 3-year old in Waynesboro is evidence of the procedure’s success.

Within months of her birth, Brianna Berkey, Waynesboro was diagnosed with severe combined immunodeficiency disease. At seven months old in June 2007, she was failing to gain weight normally and was developing severe pneumonia. Her symptoms included a cough, poor appetite and lack of growth. Her immune system simply lacked the ability to fight infections.

“Brianna’s immune disease diagnosis was very hard to deal with,” said her mother, Linda Medina.

For three months, Brianna was a patient at Milton S. Hershey Medical Center Children’s Hospital in Hershey.

“Brianna first received chemotherapy to knock down abnormal cells to make space for the healthy cells, said Brianna’s physician Dr. Ken Lucas, director of the pediatric stem cell transplant program at the children’s hospital and the surgeon who performed the stem cell transplant. “In her situation the immune deficiency was a genetic problem.”

Following her chemotherapy regimen, healthy cells were injected into Brianna’s blood stream through an intravenous line.

“The cells know where to go,” Lucas said.

Stem cell donors are difficult to find, the doctor said. White blood cell proteins found on the surface of cells have to match. If they don’t, rejection of

the transplant can occur. A complication known as graft versus host disease — in which the new cells fight the body — can also occur.

The success rate for transplants in pediatric immune deficiency disease patients is greater than 90 percent, Lucas said, which is a higher percentage than the success rates for pediatric patients with high-risk cancers such as leukemia and in adults with leukemia and lymphoma. In pediatric cancer patients, success can be as high as 70 percent, depending on the status of the disease in a patient, she said.

“Outcomes from stem cell transplants have gotten better since we have an expanded pool of donors,” said Lucas, adding that one in four stem cell transplants are from related donors.

Many other donors are matched through the National Marrow Donor Program, which also coordinates (umbilical) cord blood registries.

Cord blood, he continued, is collected from the placenta of many newborns birthed at medical centers that also serve as collection centers, both in the United States and other countries.

Bone marrow comes from adult donors.

With stem cell transplants, people can expect to live a normal life span, Lucas said. Since the transplant, Brianna has become a healthy girl who is right on track developmentally, said her mother.

“Now Brianna can lead a normal life and be in a daycare center with other children while I am at work.”

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Iris Hersh may be reached at 262-4757 or ihersh@publicopinionnews.com.

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