Tiniest babies are growing up healthy despite odds

One is a healthy first-grader, the other an honors college student majoring in psychology. Once the tiniest babies ever born, both girls are thriving, despite long odds when they entered the world weighing less than a pound.

A medical report from the doctor who resuscitated the infants at a suburban Chicago hospital is both a success story and a cautionary tale. These two are the exceptions and their remarkable health years later should not raise false hope: Most babies this small do poorly and many do not survive even with advanced medical care.

"These are such extreme cases," said Dr. Jonathan Muraskas of Loyola University Medical Center in Maywood, Ill. They should not be considered "a benchmark" to mean that doctors should try to save all babies so small, he said.

The report involves Madeline Mann, born in 1989 weighing 9.9 ounces, then the world record; and 7-year-old Rumaisa Rahman, whose 9.2-ounce birth weight remains the world's tiniest. Rumaisa's birth weight was initially reported as 8.6 ounces, but that figure was based on a different conversion scale.

Two other babies born since 1989 weighed less than Madeline, and a German girl was born last year at her same birth weight.

The report was released online Monday in Pediatrics.

It addresses a question that was hotly debated when Madeline was born 22 years ago, remains hot now - and still has no answer: "What is the real age of viability? No one knows," said Dr. Stephen Welty, neonatology chief at Baylor College of Medicine and Texas Children's Hospital in Houston.

Muraskas and the report's co-authors say most newborn specialists consider babies born after 25 weeks of pregnancy to be viable - likely to survive - and so they should receive medical intervention if necessary to breathe. Younger babies are generally in a "gray zone," where intervention isn't always so clear cut, the report suggests.

In Japan, doctors have lowered that threshold - the gestational age - to 22 weeks. Normal pregnancies last about 40 weeks.

Some U.S. doctors will attempt to save babies at 22 weeks, but that is not done routinely, said Dr. Edward Bell, a University of Iowa pediatrics professor.

Bell runs an online registry of the world's tiniest babies, born weighing less than about 14 ounces, or slightly less than 1 pound. Since 1936, 124 have been listed. The registry is compiled from doctors' voluntary reports and so does not represent all survivors.

Bell estimates that about 7,500 U.S. babies are born each year weighing less than 1 pound, and that about 10 percent survive.

Sometimes tiny babies with zero chance of surviving show signs of life at birth, and may be able to breathe for a short time if put in an incubator and hooked up to a breathing machine and intravenous treatments. "But even so, if it's a baby that doesn't have a chance, we don't want to put the baby and the family through the discomfort," Bell said.

Muraskas says his report highlights a sometimes overlooked fact: gestational age is even more critical for survival than size.

Rumaisa and Madeline were both palm-sized, weighing less than a can of soda pop - the average size of an 18-week-old fetus but they were several weeks older than that. Their gestational ages - almost 26 weeks for Rumaisa and almost 27 weeks for Madeline - meant their lungs and other organs were mature enough to make survival possible.

But both required intensive medical intervention. They were delivered by cesarean section more than a month early because their mothers had developed severe pre-eclampsia, dangerously high blood pressure linked with pregnancy. Both babies were hooked up immediately to breathing machines with tubes as slender as a spaghetti strand slipped down their tiny airways.

Rumaisa has a twin who was more than twice as big at birth. Few details about her are included in the report.

Before the births, both mothers were given steroid drugs to speed up growth of the babies' immature lungs. Even so, Rumaisa and Madeline were on breathing machines for about two months, and hospitalized for about four months.

Madeline had mild brain bleeding, common in tiny preemies, but with no lasting effects. Severe cases can cause serious mental disabilities. She and Rumaisa got treatment for an eye condition common in preemies called retinopathy, which in severe cases can cause blindness.

Madeline has asthma and remains petite - 4 foot 8 and about 65 pounds at age 20; Rumaisa at age 5 weighed 33 pounds and was 3 1/2 feet tall, smaller than about 90 percent of kids her age. Current information on the girls' size was not in the report; Madeline is now 22 and a senior at Augustana College in Rock Island, Ill.; Rumaisa is 7 and attends first grade in suburban Chicago.

Jim Mann, Madeline's father, said having a baby born so small was "terrifying" at first. But other than asthma, the only lasting effect his daughter has mentioned is having trouble finding age-appropriate clothes because she remains so small, he said.

That she has done so well is a source of pride, and wonder, her dad said.

"I don't know why, we were just extraordinarily lucky," Mann said.

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New method boosts blood-clotting for hemophiliacs

In what's being called a landmark study, researchers used gene therapy to successfully treat six patients with severe hemophilia, a blood-clotting disorder.

The study was preliminary and involved only six patients, and other promising early attempts to use gene therapy against hemophilia ultimately failed. But a single infusion using the new treatment worked in some patients for more than a year, boosting their clotting ability significantly.

"I think this is a terrific advance for the field. It's a good lesson in terms of don't give up on good ideas," said Dr. Ronald Crystal, chairman of genetic medicine at New York City's Weill Cornell Medical College,

It's "truly a landmark study," said Dr. Katherine Ponder, a Washington University School of Medicine physician. She praised the research in an editorial that accompanies the study's publication in the New England Journal of Medicine. The research also was being presented Saturday at an American Society of Hematology conference in San Diego.

Hemophilia is an inherited, potentially life-threatening disorder affecting an estimated 20,000 Americans, almost all of them males. Their blood doesn't clot properly because of a faulty gene.

In severe cases, they can spontaneously start bleeding internally, even in the brain. Internal bleeding in the joints leads to debilitating movement problems and intense pain.

Past gene therapy experiments on hemophiliacs improved blood-clotting for only a few weeks. "We couldn't make it last," said Val Bias, chief executive of the National Hemophilia Foundation.

Experts said the new method needs to be tested on many more patients to confirm it's effective and prove there are no risks. Even if all goes right, it's still several years away from being available to most patients.

Since the late 1960s, doctors have given hemophiliacs infusions of clotting proteins. It's been a success, increasing their average lifespan to 63. But for severe cases, treatment can involve two or three infusions every week and cost more than $250,000 annually.

The new study was led by researchers at the University College London Cancer Institute and St. Jude Children's Research Hospital in Memphis, Tenn. All six of the patients in the study were men seen in London who had severe forms of type B hemophilia.

The six men each got a single, 20-minute infusion of healthy genetic material delivered by a virus found in monkeys. Viruses are often used to transport DNA into cells. Each saw the amount of clotting proteins in their blood increase from less than 1 percent of normal levels to at least 2 percent, and in one case as much as 11 percent.

That may not seem like a lot, but it was enough to allow all the men to ease back on the number of regular treatments they needed, and four stopped conventional treatment altogether.

It's not yet clear how risky the treatment is. In one patient, the level of liver enzymes shots up to five times normal levels. It did not cause symptoms but there are concerns about liver inflammation and he was treated with steroids. Researchers noted it's possible the treatment could trigger hepatitis in some patients.

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FDA panel backs birth control patch despite risks

A panel of federal health advisers said Friday that a birth control patch from Johnson & Johnson probably carries a higher risk of blood clots than older drugs, but should remain available as an option for women who have trouble taking a daily pill.

The Food and Drug Administration's panel of reproductive health experts voted 19-5 that the benefits of the Ortho Evra patch outweigh its risks, specifically a potentially higher risk of dangerous blood clots in the legs and lungs. Panelists said the patch can be especially useful for younger women who have difficulty sticking to a daily pill regimen.

"I have many teenagers and it's the only method they'll use - for them it's the perfect method," said Dr. Melissa Gilliam of the University of Chicago. The FDA sought the experts' advice as it reviews the safety of newer hormone-based contraceptives launched in the past decade. The agency is not required to follow their advice, though it often does.

Johnson & Johnson's weekly Ortho Evra patch was approved in 2001 and has been marketed for its convenience as an "option for busy women who are looking to simplify life." The drug works about as well as other contraceptive medications, allowing about one unplanned pregnancy per year for every 100 women.

Studies assessing Ortho Evra's blood clot risk have reached differing conclusions over the years. At least two studies found that patch users have twice the risk of clots as women taking birth control pills. Even a slightly higher risk can be critical because blood clots can trigger heart attacks, strokes and blockages in lungs or blood vessels, which in rare cases have been fatal, even among young women.

The most recent study by the FDA found that women using the patch have a 50 percent higher risk of clots than women taking various oral contraceptives. However, agency scientists said the data was not definitive.

Panelists voted 20-3 with one abstention that the drug's current label is inadequate and should be updated with the latest information about the potentially higher risk. In discussion, a majority of panelists said Ortho Evra probably carries a higher risk than older birth control pills, though the risk is less clear when compared with newer birth control pills launched in the last decade.

Despite the safety concerns, the experts stressed that Ortho Evra fills a unique niche among birth control products.

"There is no alternative in this range for women who desire hormonal contraception but can't take the pill, so I think it is important to maintain that option," said Dr. Michele Orza of the George Washington University.

Prescriptions for Ortho Evra have declined steadily over the last five years, from 5 million in 2006 to about 1.3 million last year.

The decline has followed repeated updates by J&J of New Brunswick, N.J., to the product's labeling, including language indicating that patients absorb up to 60 percent more estrogen via the patch than with the pill.

"The company will continue to collaborate with the FDA on a product label that adequately reflects the known risks and benefits of the product, including new information," said Jeff Christensen, communications manager with J&J's Janssen unit.

Ortho Evra is part of a new generation of contraceptives that use recently-developed, laboratory-made forms of the female hormone progesterone. In the past decade the medications have overtaken older medications, though studies have reached conflicting conclusions on whether the drugs carry a higher risk of blood clots. On Thursday, the same panel recommended clearer risk labeling on Yaz, Yasmin and similar birth control pills marketed by Bayer and Teva Pharmaceuticals. As with Ortho Evra, the panel affirmed their overall benefit of the pills for women.

Ortho Evra sales last year totaled $124 million, trailing Merck & Co. Inc.'s NuvaRing, which posted sales of $437 million, according to health care data firm IMS Health. NuvaRing is the most widely used, non-pill contraceptive drug.

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Texas delays action on stem cell therapy rules

The Texas State Medical Board voted Friday to delay until next year final approval of new stem cell therapy rules that could restrict - or even block - procedures such as the one Gov. Rick Perry recently underwent on his aching back.

Its 19 members, including a dozen physicians, voiced support for greater oversight but opted to tweak the proposed rules and take them up again at their next meeting in February. If the reworked rules are satisfactory, formal approval could come as early as April.

The Food and Drug Administration hasn't approved using adult stem cells to help people heal from surgery, but experimentation is common. Some scientists tout the possible benefits, including treatment for heart disease, diabetes, and some cancers. Others argue adult stem cell experimentation actually increases the risk of cancer and can cause blood clots.

Perry, 61, a Republican presidential candidate, had stem cells taken from fat in his own body that were then grown in a lab. They were injected into his back and his bloodstream during an operation in July to fuse part of his spine.

The proposed rules would require an independent panel to conduct a comprehensive safety review of any procedure involving stem cells before it is carried out. Without that review, procedures such as the one Perry underwent would not be allowed.

Some noted that new stem cell regulations could have unintended consequences, however, such as limiting the ability of tissue banks to collect genetic material for transplants. As a result, the board asked its staff to modify the rules.

"My sense is, this 80, 85 percent ready for primetime and they're just adding some material," said Leigh Hopper, a board spokeswoman.

Perry, who appoints the board, sent it a letter in July saying he appreciated the responsibility to protect patients. But he also urged members to "recognize the sound science and good work that is already being done, and will continue to be done in the future, in this field."

"We need to ensure that physicians in this state can continue to pursue new technologies and treatments that will benefit all Texans," Perry wrote. "Texas is a leader in innovation in many fields. It is critical that we continue to foster an environment that encourages technological advancement in the health care arena."

Following his surgery, Perry has worn a back brace but maintained his hectic campaign schedule - though he has begun wearing orthopedic shoes, foregoing his trademark cowboy boots. Adult stem cell therapy is different from using embryonic cells, a controversial technology that the governor opposes.

Lucy Nashed, a spokeswoman for the governor, said Friday that he "expects the Texas Medical Board to review all the facts and make the appropriate decision regarding the use of this promising technology in Texas."

At least 10 states - including California, Illinois and New York - have enacted rules governing stem cell research, according to the Interstate Alliance on Stem Cell Research.

The impetus for Texas' proposed rules did not grow out of Perry's procedure but did involve the physician who performed it, Dr. Stanley Jones. The Houston-based orthopedist, who is Perry's doctor and friend, touted the benefits of stem cell therapies at the medical board's June meeting. He said many Americans pay thousands of dollars to undergo treatment abroad and that it is a shame that Texans can't access the treatment in their home state.

Perry also helped push an amendment to a larger health care bill through the Texas Legislature in June creating a state bank to store and cultivate adult stem cells for treatment purposes.

NBC reported in September that the first bank approved by the state - Celltex Therapeutics Corp. of Houston - is co-owned by Jones and David G. Eller, the former chairman of the board of Texas A&M University, and a top Perry donor.

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Too posh to push? More C-sections on demand in UK

Pregnant women in Britain, where the government provides free health care, may soon be able to get a cesarean section on demand thanks to a rule change that critics describe as the health system caving into the "too posh to push" crowd.

Currently, British women who can't afford to pay private doctors for their baby's delivery have been allowed to have planned C-sections only if there are health concerns for mother or baby. Emergency C-sections are done when the situation demands it.

But new guidelines set to take effect later this month say pregnant women "with no identifiable reason" should be allowed a cesarean if they still want it following a discussion with mental health experts.

"It's about time women who have no desire to view labor as a rite of passage into motherhood be able to choose how they want to have their baby," said Pauline Hull, who has had two children by cesarean because of medical reasons. "The important thing to me was meeting my baby, not the experience of labor."

Hull runs the website, Elective Cesarean, from her home in Surrey, south of London. She said midwives tend to overexaggerate the risks of C-sections and underestimate those of vaginal births.

The new draft guidelines come from the National Institute for Health and Clinical Excellence, or NICE. The agency's guidelines are usually accepted by the government and determine what will be paid for by its health system.

"In general, a C-section is a safe operation, especially when performed as a planned procedure," the new guidance says.

The agency says it routinely updates guidance every few years and denies there was any pressure to change its more restrictive C-section advice. But in recent years, advocates and some doctors have slammed the U.K. health system for not giving women a greater say in childbirth.

The change comes at a price for Britain's cash-strapped health system. NICE estimates C-sections cost about 800 pounds ($1,280) more than a vaginal birth, although that doesn't include the price of treating possible long-term complications like urinary incontinence from vaginal births.

The report notes that for every percentage point the C-section rate falls, the health system could save 5.6 billion pounds ($8.9 billion).

In the U.K., about 25 percent of women have C-sections, versus about 30 percent in the U.S. In both countries, rates have doubled in recent years, though doctors say that's not just due to demand, but because pregnant women increasingly have other problems like obesity and diabetes.

About 10 percent of all U.K. births are planned C-sections while about 15 percent are emergency procedures, according to NHS figures.

The World Health Organization has previously said wealthy countries should aim for a C-section rate of about 15 percent, though it also says there isn't enough evidence to know what the ideal rate is.

The National Health Service estimates that about 15 percent of British births take place in private hospitals, which tend to have higher cesarean rates. At Portland Hospital in London, where many celebrities check in, the C-section rate ranges from 35 to 40 percent.

The issue of women having C-sections on demand has long been a hot button issue in the U.K., with celebrities like Madonna and former Spice Girl Victoria Beckham having scheduled procedures at upscale hospitals. Criticism for the wealthy getting these elective surgeries led to the phrase "too posh to push."

But some experts say the new British guidance won't dramatically change how pregnant women are treated.

"It's only a small percentage of women who ask for a C-section," said Cathy Warwick, chief executive of the Royal College of Midwives.

Warwick said doctors and midwives regularly talk to women who have concerns about childbirth and after addressing their fears, most women agree to skip a planned C-section.

"As long as it's safe for both mother and baby, a vaginal birth is absolutely the best way for anyone to deliver," said Dr. Daghni Rajasingham, an obstetrician and spokeswoman for the Royal College of Obstetricians and Gynaecologists. She said the physical stress put on a baby's lungs during labor helps them adapt to breathing after being born.

Rajasingham also said while C-sections are safe, the operation comes with risks including infections, bleeding, and the potential for problems with future pregnancies.

"As an obstetrician working with limited resources, I want to make sure we have safe and high-quality health care for all women and not be skewed by a few women who want something specifically," Rajasingham said.

For women like Hull, however, the policy shift is long overdue.

"Women shouldn't think a C-section is going to be a walk in the park, but they should have all the information they need to help them make an informed choice," she said.

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FDA approves innovative, non-invasive heart valve

Federal health officials have approved a first-of-a-kind artificial heart valve that can be implanted without major surgery, offering a new treatment option for patients who are too old or frail for the chest-cracking procedure currently used.

The Food and Drug Administration said late Wednesday it approved Edwards Lifesciences' Sapien heart valve, which can be threaded into place through a major artery that runs from the leg up to the heart. Cardiologists say the highly anticipated new approach will help old, sickly patients who cannot undergo the more invasive open heart surgery, which has been used to replace valves for decades.

Other companies have won approval for less-invasive heart valves before, but Edwards' implant is the first replacement for the aortic valve, the heart's main doorway.

About 300,000 U.S. patients suffer from deterioration of the valve, which forces the heart to work harder to pump blood, often leading to heart failure, blood clots and sudden death. More than half of patients diagnosed with the condition, called aortic stenosis, die within two years, according to the FDA.

Every year about 50,000 people in the U.S. undergo open-heart surgery to replace the valve, which involves sawing the breastbone in half, stopping the heart, cutting out the old valve and sewing a new one into place. Thousands of other patients are turned away, deemed too old or ill to survive the operation.

The Mayo Clinic's Dr. David Holmes said the Sapien valve is a "game changer" for those inoperable patients, many of whom are in their 80s with medical conditions like diabetes, emphysema and liver disease.

"We don't have very good therapy for them at this time - some of them receive palliative care and some receive medication," said Holmes, who is president of the American College of Cardiology. "But this is really a mechanical problem, and for mechanical problems medications don't work very well."

Edwards' transcatheter valve is threaded through the femoral artery via a small incision in the leg, and then guided up to the heart via catheter. The valve is then wedged into the aortic opening by an inflatable balloon, replacing the natural heart valve. The device is made from cow tissue and polyester supported by a steel frame.

FDA based its approval on a 365-patient study that compared outcomes for patients with the valve and those who received basic comfort care and other non-surgical treatment. After one year, 70 percent of patients with the valve were still alive, compared with only 50 percent of those who received alternatives. However, the device was associated with serious complications, including stroke and internal bleeding. Under the conditions of FDA approval, Edwards will track the medical history of all patients who receive the valve.

The device is only approved for patients who cannot undergo open-heart surgery.

About 20,000 new U.S. patients will be eligible to receive a heart valve each year based on Wednesday's approval, according to Morgan Keegan analyst Jan Wald.

The larger opportunity for the new valve is in patients who are healthy enough to undergo surgery, but are considered high-risk and could benefit from a less invasive procedure. The FDA is expected to clear the device for those patients next year, and analysts estimate that group could eventually number between 50,000 and 80,000 annually as the U.S. population ages.

Edwards is expected to charge about $30,000 for the valve, though hospital fees could bring the total cost of surgery closer to $70,000. Standard heart valve replacement costs upward of $50,000, mostly from surgical and hospitalization fees.

The approval represents a dramatic business opportunity for Irvine, Calif.-based Edwards Lifesciences Corp., which had total sales of $1.5 billion last year. Analysts estimate that sales of the Sapien valve could help double the company's revenue to $3 billion within a decade. Company shares rose $3.11, or 4.2 percent, to $77.48 in after-hours trading.

The company expects to train surgeons at 150 to 250 sites across the U.S. to implant the Sapien in the coming year.

The valve has already been approved for four years in 40 countries around the world, including most of Europe. In most of those countries Edwards already sells a next-generation version of the device.

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