DC Child Accident & Safety Blog

Drug Labels Still Leave Pediatricians Playing a Guessing Game

Thu, 17 May 2012 13:03:06 -0500

Nearly 50 years ago, Dr. Harry Shirkey coined the phrase "therapeutic orphan" to describe children who took prescription medicine. Because drug studies among a pediatric population were complicated and offered little financial return for pharmaceutical companies, no one really knew how drugs developed for adults would affect children who took them. Providers prescribed them based on their best guess about how to tailor an adult dose to a child.

In 1997, Congress passed the Food and Drug Administration Modernization Act (FDAMA). It offered incentives for pediatric drug development in the form of additional marketing exclusivity. Progress has been made in drug research among children, and drug labeling for these patients has improved.

But not enough.

According to a recent study published in the Journal of the American Medical Association, (JAMA) not even half of drug labels have information on safety and proper dosing for kids. More than 500 drug labels were evaluated.

According a Reuters story about the new study, drugs still pass the FDA approval process and make it to market without much research on how they should be given to children for the best treatment and least harm.

Pediatric research remains difficult—the number of children with a given disease usually is lower than that of adults, and the ethical concerns are thornier because minors can’t provide informed consent.

The JAMA study looked at hundreds of drugs that treated a range of problems from high blood pressure to allergies.

Labels were deemed “adequate” to use in children if they included how effective the drug is, if it’s safe in younger children and teens and enumerated doses of children of different ages.

Two hundred thirty-one drugs were adequately labeled and 29 others carried dosing for some younger age groups, but not all. Still, slightly more than half of all labels lacked useful pediatric information. Even after tossing the results of drugs that were irrelevant to kids (prostate cancer treatments, for example), 44 in 100 labels offered no information for young patients.

Some newer drugs, those approved by the FDA between 2002 and 2008, also lacked safety and dosing information for kids. But the good news is that drugs commonly prescribed for children—asthma meds and vaccines, for example—often undergo rigorous testing.

Still, kids get cancer, arthritis, and they suffer lung and heart disease; studies on how drugs to treat them react in children often fall by the wayside. And the “how much and at what cost” treatment guessing game continues.

Prescribing medications to children that have not been labeled for them is an off-label use (doctors are allowed to prescribe them, but drug companies may not market them for an unapproved use). That might result in benefit, no therapeutic effect or harm (adverse reactions). (To learn more about medication errors, link here.)

Doctors uncertain about a drug’s effect in a kid might be reluctant to prescribe it off-label, and that could deprive the patient of a real benefit.

If a child receiving an unlabeled drug therapy doesn’t respond well, is that because the drug just didn’t work? Was the dosing incorrect? If there are side effects, do they denote a marker of the drug itself, or of an incorrect dose?

The researchers concluded, “Labeling with pediatric information in only 46 percent of products is still insufficient. Legislation to increase pediatric clinical trials and require the resulting information be added to labeling is necessary. The current legislation expires in 2012 without reauthorization.”

That lapse is not acceptable. Contact your congressional representatives to appeal for completion of a process that has shown benefit but is not finished.

And if your child is prescribed a drug lacking information about appropriate doses for certain age groups, and possible side effects, ask your doctor and pharmacist for more information. If they’re uncertain, because there’s no science to inform them, ask if there are alternative therapies.

Children should not be guinea pigs.

Teens Who Develop Diabetes Have a Stark Future

Thu, 10 May 2012 13:50:23 -0500

Youth often confers health benefits not available to adults. One example is metabolism—when you’re young, you burn calories faster than when you’ve stopped growing and age (among other factors) imposes a more sedentary existence. Another example is bone regeneration—younger folks build bone better and faster than their elders, and generally are not at risk of osteoporosis, a degenerative bone disorder.

But a new study signals that one dread disorder—diabetes—is as ominous for youngsters as it is for adults. A study and companion editorial in the New England Journal of Medicine made clear that if a teenager develops Type 2 diabetes, he or she will struggle for life to control it.

The problem is obesity. Thanks to poor diet and insufficient exercise, too many teenagers are overweight, concluded the largest federally funded study ever to examine how to treat diabetes in teens. (Earlier studies primarily focused on adults.)

Most diabetes drugs aren't approved for youths. So the key for kids is to prevent the disease, not to treat it.

As a story in the Associated Press pointed out, in earlier generations, doctors seldom saw children with Type 2 diabetes. (Type 1 diabetes, formerly called “juvenile diabetes,” presents earlier, and is attributed to both genetic risk and external factors, such as diet or an infection.)

One-third of U.S. children and teens are overweight or obese. They are at higher risk of developing Type 2 diabetes, in which the body either fails to make enough insulin or to use it efficiently to metabolize sugar from food.

Treatment for Type 2 diabetes usually is metformin, a drug to lower blood sugar. If that doesn’t control the problem, other drugs and daily insulin shots may be required. The risks of long-term blood sugar problems include blindness, nerve damage, kidney failure, limb amputation, heart attack and stroke.

Read more about diabetes and its standards of care here.

The study tested how to manage blood sugar in teens newly diagnosed with diabetes. Half of the nearly 700 subjects failed within a few years; 1 in 5 suffered serious complications.

The study subjects had their blood sugar normalized with metformin, then were given either metformin alone, metformin plus diet and exercise counseling or metformin plus a second drug, Avandia. After nearly four years, half of the metformin-only group failed to maintain blood sugar control. The group on two drugs did a little better, but not much different from those in the lifestyle group.

Because Avandia has been linked to a higher risk of heart attacks in adults (which became known after the study began), it’s not recommended for teens.

The NEJM editorial, by Dr. David Allen, called the study’s message “stark”: “[T]omorrow and beyond, public-policy approaches — sufficient economic incentives to produce and purchase healthy foods and to build safe environments that require physical movement — and not simply the prescription of more and better pills will be necessary to stem the epidemic of Type 2 diabetes and its associated morbidity.”

Yes, the problem is social. But it’s also individual. Parents must establish healthful eating habits early in life, and ensure that their children exercise their bodies as well as their minds. Those are the earliest, easiest and best interventions to prevent the insidious disease that robs so many people of so much, and many of them too soon.

Less Radiation Is Good Medicine for Diagnosing Appendicitis

Fri, 04 May 2012 06:00:00 -0500

Appendicitis often presents as an emergency, and whether you’re old or young, the belly pain can be extreme. Typically, it’s diagnosed using a CT scan, and a new study indicates that this evaluation should be performed differently for adults and for children.

As published in the New England Journal of Medicine, the research shows that exposure to the radiation in a CT scan is of concern particularly for children and young adults. Such scans can employ different levels of radiation, and it appears that low-dose CT scans are just as effective in diagnosing appendicitis in teens and young adults as standard doses.

Because the effects of radiation exposure can be dire—an increased risk of cancer, primarily—the lower the dose the better. (See our article, “Radiation Overdose Injuries”.) But these lower levels haven’t been accepted widely, according to analysis of the NEJM study by MedPage Today, because of the quality of the images rendered was suspect.

So the researchers conducted a randomized trial of 879 patients ages 15 to 44 suspected of having appendicitis. About half received a low-dose CT scan and half received the standard-dose scan.

About 4 in 10 patients in each group had an appendectomy after the scan. Afterward, it was determined that six patients in each group did not have inflammation, and had undergone the operation unnecessarily.

The difference between the two groups was so negligible that using the low-dose CT instead of the standard dose would have resulted in only one additional unnecessary operation in 330 patients. That risk, researchers noted, is favorably weighed against the potentially higher cancer risk with the standard dose.

"However, it is highly debatable whether the radiation levels used in our two groups can actually induce cancer and whether use of the low dose instead of the standard dose can actually reduce the carcinogenic risk," they added.

The rate of perforated appendix, which indicates that the diagnosis was delayed, also was similar between the two dosage groups.

The welcome performance of the lower dose scan was attributed to superior imaging capability of modern equipment and the fact that it’s fairly simple to interpret CT images of an appendix.

Cells that divide rapidly are more susceptible to cancer. Because the active growth of children signifies rapid cell turnover; because gastrointestinal tract cells continually turn over; and because children have a longer life span and are therefore more vulnerable to all forms of radiation exposure for a longer period, it just makes sense to limit their radiation exposure when you can.

If your child’s doctor prescribes a CT scan to diagnose possible appendicitis, request a low-dose procedure. And if it’s prescribed for other diagnostic reasons, discuss the options. Sometimes, care providers embrace the more-is-more solution out of habit, and that’s not always the best medicine.

Good News for the No. 1 Killer of Children -- Accidental Deaths Are Way Down

Thu, 26 Apr 2012 01:07:25 -0500

The No. 1 killer of children is accidents. More than 9,000 children in the U.S. died in 2009 from what the Centers for Disease Control and Prevention (CDC) call “unintentional injury.”

As grim as is the reality of children who don’t live to see adulthood, the cold statistics are actually good news—death rates from unintentional injuries among people from birth to 19 declined almost 30 percent from 2000 to 2009, according to the CDC.

Despite the overall good news, a couple of causes of death did see an increase—suffocation rose 54 percent among babies younger than 1 year, and poisonings increased a whopping 91 percent among teenagers 15 to 19. The CDC attributes that stark reality to prescription drug overdoses.

The graphically colorful report in the CDC’s April issue of Vital Signs is the first such study to depict fatal unintentional injury trends by cause and by state for this age group.

The most common cause of accidental death is motor vehicle crashes. Other leading causes are:

suffocation;

drowning;

poisoning;

fires;

falls.

Thomas Frieden, director of the CDC, said, “Kids are safer from injuries today than ever before. In fact, the decrease in injury death rates in the past decade has resulted in more than 11,000 children’s lives being saved.”

Significantly, death rates from motor vehicle crashes declined 41 percent during the decade of study. The CDC attributes that improvement to improvements in the use of child-safety and booster seats, and the implementation of graduated licensing systems for teen drivers.

Differences in injury death rates varied enormously from state to state. Massachusetts notched fewer than five deaths per 100,000 children, versus New Jersey, South Dakota and Mississippi, which tallied 23 deaths per 100,000 kids.

Addressing the problem of infant suffocation the CDC says, requires widespread adherence to the American Academy of Pediatrics’ guidelines for safe sleeping environments. Those measures include infants sleeping alone, on their backs in cribs with no loose bedding or soft toys.

To reduce prescription drug poisoning, the agency says, providers must prescribe drugs appropriately, and parents must ensure their teens store and dispose of drugs properly, and they should monitor these practices. Also, teens must be discouraged from sharing medications. The CDC also recommends that states establish prescription drug monitoring programs. We recently wrote about prescription drug misadventures being responsible for a disproportionate number of childrens’ emergency room visits.

“Every four seconds,” said Linda C. Degutis, director of the CDC’s National Center for injury Prevention and Control, “a child is treated for an injury in the emergency department, and every hour, a child dies as a result of an injury. Child injury remains a serious problem in which everyone–including parents, state health officials, health care providers, government and community groups–has a critical role to play to protect and save the lives of our young people.”

For more information about preventing injuries to children and a copy of the CDC’s National Action Plan on Child Injury Prevention, compiled in conjunction with 60 partner organizations, link here.

Children Whose Hearts Stop

Thu, 19 Apr 2012 16:07:04 -0500

Happily for most parents, heart trouble is pretty well off their child-care radar. When a child succumbs to cardiac arrest, often it’s a shock and a surprise.

But by some estimates, according to a recent story on MedPage Today, there might be warning signs in as many as half of all cases of sudden cardiac arrest in children. Awareness by clinicians of these signs and a thorough understand of family history by both parents and pediatricians can prevent sudden cardiac arrest in youngsters, says the American Academy of Pediatrics in the journal Pediatrics.

A primary point in the academy’s statement was that some symptoms of cardiac problems in children commonly are associated with other, less dire disorders. Pediatricians must remain not only vigilant in observing them, but open-minded about their cause because pediatric sudden cardiac arrest can be lethal within minutes if it goes unrecognized and untreated.

Approximately 2,000 such deaths are estimated to occur annually in the United States.

Symptoms can include chest pain, dizziness, exercise-induced syncope (temporary loss of consciousness) and dyspnea (difficult or labored breathing). Any or several of these symptoms might have been disregarded by the patient and family, the article notes.

A detailed family history that yields knowledge of a sudden, unexplained death of a young relative also warrants scrutiny of any child exhibiting symptoms of what can easily be ascribed to getting tackled on a Pop Warner football game, or too many rides on the Tilt-a-Whirl.

The most common underlying causes of sudden cardiac arrest among children are:

structural or functional disorders such as hypertrophic cardiomyopathy, a heart defect in which the muscle wall is so strong that it can’t relax enough to allow sufficient blood flow;

coronary artery anomalies, or congenitally malformed heart vessels;

primary cardiac electrical disorders, when the heart’s electrical system malfunctions.

An afflicted child most frequently experiences ventricular tachyarrhythmia, which is an abnormal heart rhythm that is rapid and regular. It originates in the lower chamber, or ventricle, of the heart. Sometimes arrhythmias are of short duration, and resemble seizures.

If a doctor or parent believes a child is having a seizure, he or she probably will be referred to a neurologist. If the real problem, however, is heart-related, the delayed diagnosis can be disastrous.

Similarly, if a child experiences breathing trouble, someone might believe he or she has a respiratory problem, and send to a pulmonary specialist.

If your child has experienced any of the symptoms above, was diagnosed and treated for a problem other than a heart issue and has not responded, see a pediatric cardiologist immediately.

The academy advises use of these risk-assessment tools, which haven’t been validated or assessed for sensitivity or specificity; nevertheless, experts emphasize that if any of the following have occurred, the patient should get a cardiac assessment as soon as possible:

a history of fainting or having a seizure, especially during exercise;

past episodes of chest pain or shortness of breath with exercise; and/or

a family member with unexpected sudden death or a condition such as hypertrophic cardiomyopathy or Brugada syndrome (a certain kind of heart rhythm disorder heart rhythm disorder characterized by a specific abnormal heartbeat and detected by an electrocardiogram test).

Although heart screenings have long been overused, in the case of young athletes, the academy advised against following earlier American Heart Association guidelines that did not endorse widespread use of ECG tests. (The AHA rightfully cited the possibility of false-positive and false-negative results, additional cost and associated problems.) The academy advised screening young athletes before they participate in sports.

The academy also recommended that automated external defibrillators be widely available in schools, as well as cardiopulmonary resuscitation training of staff and others. Parents should determine if these tools are present in their child’s school, and lobby for them if they are not.

Autism Rates Rise

Fri, 13 Apr 2012 05:08:01 -0500

When awareness of a disease or disorder hits critical mass, often its rate of diagnosis increases. That provokes the chicken-and-egg question of which came first, the incidence of disorder or the awareness of it?

In recent years, many people have looked at autism, and the spectrum of autism disorders, through that lens. A recent study boosts the notion that the prevalence of autism in children is increasing. The U.S. Centers for Disease Control and Prevention concluded that 1 in 88 8-year-olds has some form of autism. The previous estimate was 1 in 110.

Based on 2008 data, the updated figure is sure to fuel debate, according to the Los Angeles Times, over whether a growing environmental threat could be responsible. “But autism researchers around the country said the CDC data—including striking geographic and racial variations in the rates and how they have changed—suggest that rising awareness of the disorder, better detection and improved access to services can explain much of the surge, and perhaps all of it.”

Some experts questioned the validity of relying on records to reach the new estimate.
David Mandell, an autism expert at the University of Pennsylvania, told The Times that the CDC's numbers primarily reflect the degree to which the diagnosis and services have become established in different places and among different groups.

"As the diagnosis is associated with more and more services, this becomes a less and less rigorous way to determine the prevalence of autism," he said, referring to the CDC's methods.

Among the CDC’s results:

Utah, which has widespread screening programs, had the highest rate—1 in 47 children.

New Jersey, which also boasts generous autism services, is next at 1 in 49.

Alabama, one of the poorest states in the country, ranked last. Its autism rate fell between 2006 and 2008 from 1 in 167 to 1 in 208.

The study did have limitations. Researchers looked at tens of thousands of health and special education records in 14 states, looking for an autism diagnosis or symptoms that might indicate one. In some areas, researchers had access only to health records, not school records, and prevalence estimates there generally were lower.

The researchers’ goal was to focus attention on the need for more vigorous screening early in life. Early intervention has been shown to confer the best long-term prospects for autistic children. More than 1 in 5 children deemed autistic by the CDC had no such diagnosis in their records.

A recent series of studies in the journal Nature indicated that the genetic origin of autism is complicated and involves multiple genes. The cause of autism is unknown. There is no blood test or other biological marker—it’s diagnosed by symptoms, which are social and communication difficulties starting in early childhood, and repetitive behaviors or abnormally intense interests. The severity of symptoms can vary widely. Boys are more likely to have the disorder, and whites somewhat more likely than minorities. Ultimately, a diagnosis involves clinical judgment.

Some people, including representatives of Autism Speaks, an advocacy group, believe that the disorder is becoming epidemic in the United States. Others say raised awareness of the disorder enables health-care providers and school authorities to deem a child autistic.

To learn the symptoms of autism, link here. Autism Speaks’ Autism Response Team (ART) members are trained to connect families with information, resources and opportunities. Contact them at 888-288-4762.

Common Products That Can Poison Children

Wed, 04 Apr 2012 15:57:19 -0500

According to the American Association of Poison Control Centers, approximately half of all poison exposures involve children younger than 6.

Most parents are pretty good at identifying and keeping their children safe from obvious toxins, from cleaning fluid to blood thinners. But many common household items, not to mention the contents of mom’s purse, are attractive and potentially lethal.

In his job as director of the Toxics Epidemiology Program for the Los Angeles County Department of Public Health, Dr. Cyrus Rangan tracks and responds to toxic exposures and consults with patients exposed to toxins. Following is his list of common products that pose a poison threat, and why.

Button batteries (for hearing aids, watches, etc.) can get lodged in the airway or the esophagus, causing third degree burns and bleeding. Note that these potentially lethal button batteries can even be found in toys.

Chewing gum is generally safe, but a young child can choke on pieces of gum. Nicotine gum is very poisonous to young children.

Cough drops taste sweet and might seem like candy to young children. But some contain medications like destromethorphan, which can cause gastrointestinal and vision problems, among others. Also, kids can choke on cough drops.

Sanitary gel can be 60 percent alcohol (120 proof). If ingested, a small bottle is like giving a kid a couple small shots of hard liquor.

Cigarettes carry a unique smell and taste that is attractive to some young children. Acute nicotine poisoning can result if they ingest a cigarette.

Nail polish remover can cause gastrointestinal distress, and can be even more harmful if vomited and inhaled into the airways. These products seldom come in child-resistant containers.

Pepper spray can be extremely irritating to the eyes, mouth, throat and lungs of anyone, but it’s worse for children, and such devices are easily deployed by accidental.

"Gummy" vitamins look and taste like candy. Although toxicity is likely to be low, there’s a larger issue here of referring to medicine as candy. Children should learn that medicine is medicine, candy is candy and confusing them is dangerous.

Over-the-counter medications, like cough drops and gummy vitamins, are often colorful, coated with sweetener and mistaken for candy. Some can be just as dangerous to a young child as prescription medications. We’ve tracked the checkered history of one such notable example, acetaminophen.

Prescription medications that aren’t stored safely away from youngsters are hazardous. We’ve addressed this hazard, and the fact that many can kill a 2-year-old in a single dose. Never store them in a container other than what they came in.

If your child has ingested a toxic product or substance, or has a reaction to something he or she has touched, contact the National Capital Poison Center at (800) 222-1222. If you think your child might have swallowed a button battery, go to the nearest emergency room.

Early Sleep Problems Signal Later Emotional Troubles

Mon, 26 Mar 2012 15:49:46 -0500

A milestone in child development, at least for many parents, is when the kid finally sleeps through the night. But a recent study suggests that it’s a good idea for parents to monitor how the wee ones are sleeping as well as how long.

Published in the journal Pediatrics, the study shows that children who have problems breathing while they sleep are more likely to experience behavioral problems such as hyperactivity and aggressiveness when they get older. They’re also more likely to have emotional issues such as difficult peer relationships.

Researchers from the University of Michigan and the Albert Einstein College of Medicine at Yeshiva University followed the sleeping patterns of more than 11,000 children for six years. They found that kids who snored, breathed heavily through their mouths and experienced apnea—long pauses between breaths during sleep—were at risk.

Collectively known as sleep-disordered breathing (SDB), the problem peaks when children are between 2 and 6 years old, but can occur when they are younger. Approximately 1 in 10 children snores regularly and 2 to 4 in 100 have sleep apnea, according to the American Academy of Otolaryngology–Health and Neck Surgery (AAO-HNS). Common causes of SDB are enlarged tonsils or adenoids, but be wary of the “quick-fix” of tonsillectomy—as we have reported, that surgical procedure is often unnecessary, and to conclude that tonsils contribute to sleep disorders requires careful diagnosis.

Quite simply, the study’s authors said, “Parents and pediatricians alike should be paying closer attention to sleep-disordered breathing in young children, perhaps as early as the first year of life.”

Although earlier studies indicated sleep problems could signal later difficulties, they involved only small numbers of patients, short follow-up of a single symptom or limited control of individual traits such as low birth weight that could be responsible for some symptoms.

In the new, more substantial study, children whose symptoms peaked between the ages of six and 18 months were much more likely to experience behavioral problems when they were 7 compared with children who breathe normally during sleep. Children whose SDB symptoms persisted throughout the evaluation period, and were most severe at 30 months, expressed the most severe behavioral problems.

Researchers theorize that SDB might be responsible for behavioral problems because of its effect on the brain. Decreased oxygen levels and increased carbon dioxide interrupts the restorative process of sleep and disrupts various chemical systems. Such malfunctions can impair one’s ability to pay attention, plan ahead and organize. They also impede one’s ability to regulate emotions.

To learn more about SDB and treatment options, consult the AAO-HNS fact sheet.

Is Spinbrush a Toothbrush or a Weapon?

Mon, 19 Mar 2012 14:01:59 -0500

It’s hard enough for some parents to get their kids to brush their teeth regularly. If your little ones use an electric device called the Spinbrush, your job just got a little harder.

According to the FDA, the Spinbrush, whose colorful packaging and design is aimed at children, can cause serious injuries, as reported on WebMD.com.

More than 39 million Spinbrushes have been sold, some models under the Arm & Hammer brand, others under Crest. All are manufactured by Church & Dwight Co. Inc, and are sold as Spinbrush.

According to the FDA’s website, parts can fly off the device and chip teeth, damage eyes, cut the mouth and gums and get stuck in the throat.

The FDA cites the potential for injury while using these Spinbrush models:

Spinbrush ProClean

Spinbrush ProClean Recharge

Spinbrush Pro Whitening

Spinbrush SONIC

Spinbrush SONIC Recharge

Spinbrush Swirl

Spinbrush Classic Clean

Spinbrush For Kids

Spinbrush Replacement Heads

The toothbrushes aren't being recalled, but Church & Dwight is informing consumers how to avoid injury on its website and via television and print advertising.

This isn’t the first time the FDA has come down on Church & Dwight. An inspection last year uncovered evidence that there had been numerous consumer complaints that had not been reported to the agency. In May, the FDA warned the company of its violations of the Federal Food, Drug and Cosmetic Act, including failure to report—within a reasonable period—serious injuries.

“Electric toothbrushes can be very effective in removing dental plaque, and so they can help prevent dental decay and gum disease,” says Susan Runner, D.D.S., chief of FDA’s dental devices branch. “At the same time, it’s important to supervise children when they use these brushes, and to look out for any malfunctions of the toothbrush that might cause an injury.”

If you or your child uses a Spinbrush:

Inspect the Spinbrush for damage or loose brush bristles. If it’s damaged, don’t use the toothbrush.

Check to ensure the headpiece is connected securely to the handle, and test it outside of the mouth. If the connection feels loose or the headpiece easily detaches from the handle, don’t use it.

Do not bite down on the brush head while brushing.

Report damaged toothbrushes to Church & Dwight toll-free at (800) 352-3384 or (800) 561-0752. Report injuries or problems with the Spinbrush to MedWatch, the FDA’s Safety Information and Adverse Event Reporting Program. You can file a report online, by regular mail or by fax or phone.

Good News for Kids About the Orphan Drug Act

Mon, 12 Mar 2012 20:43:39 -0500

Few medical scenarios are more heartbreaking than a child with a dread rare disease who might be able to be treated if only drugs were available.

“Orphan drugs,” as we recently described, treat extremely rare disorders, generally afflicting 6,000 or fewer patients. Because the consumer market for them is so small, there’s little incentive for manufacturers to conduct the R&D necessary to develop them, or to spend the money marketing them.

Legislative efforts are underway to deliver orphan drugs faster to the people who need them. An article published recently in the journal Pediatrics, “What the Orphan Drug Act Has Done Lately for Children With Rare Diseases: A 10-Year Analysis,” reviewed progress for victimized youngsters.

It looked at orphan drug designations and approvals from Jan. 1, 2000, to Dec. 31, 2009, and determined that the Act “led to increased product availability for [rare diseases] overall, with an increasing number of marketing approvals for children this past decade.”

During that time period:

1,138 drugs were designated as “orphan,” of which 148 received marketing approval;
38 (more than 1 in 4) addressed pediatric diseases;
the proportion of approvals for pediatric products increased from fewer than 1 in 5 in the first half of the decade to slightly fewer than 1 in 3 in the second half.

This is good news, as is the fact that approval for “orphan” designation and marketing for pediatric diseases with fewer than 20 000 patients grew more than for any other group.

Anyone interested in knowing more about the FDA’s Office of Orphan Products Development may link here.

How to Help Your Child in the Wake of School Violence

Tue, 06 Mar 2012 13:42:35 -0500

It doesn’t matter that tragedies like the recent school shooting in Ohio are random and nonsensical. If they’re horrifying to an adult, they can be positively terrifying to a school kid.

Even if your child isn’t within three area codes of this or any other school shooting, he or she can be traumatized. Several child violence experts on Psychcentral.com suggest ways to help a child overcome an irrational fear in the wake of such incidents.

Q. What can parents tell their children if they are afraid to go to school after a school shooting?
A. Let children voice their fears and concerns. Open a conversation by saying, “When we hear about something as sad and scary as a school shooting, it makes mommies, daddies and children worry about our children being safe at school.”

If your child expresses concern about safety or violence at his or her school, talk about it and offer to join the child in discussions with the appropriate school personnel.

Shootings can remind children of a previous experience with danger. If it does, discuss these prior experiences and differentiate them from this recent shooting.

For a limited time, accompany your child to school or home. Spend a little extra time with them at bedtime. But make sure your child understands this is temporary help to assist in returning to a normal routine.

Give your child realistic assurances — that while these events can and do happen, they are rare.

Q. What can you do to protect your children?
A. If violence occurs in your home between adults or between adults and children, get help immediately. Violence at home is the primary exposure for children to violence and violent injury.

All parents experience constant tension between allowing children to be independent and setting limitations for their own protection. Most important is to continually educate your children and openly discuss the safety strategies that accompany increased independence. You can renegotiate this balance with your children on a temporary basis because of immediate safety issues within their own communities and schools.

If you must restrict your child from activities because of safety concerns, explain that the restrictions are temporary and that you are looking forward to the time when the child can enjoy more independence. Know their friends and communicate with their friends’ parents and other parents who might have information about your child’s friends and activities.

Q. How can you recognize the potential for a child to be violent?
A. The American Academy of Child and Adolescent Psychiatry says the presence of one or more of the following increases the risk of violent or dangerous behavior:

past violent or aggressive behavior (including uncontrollable angry outbursts)

access to guns or other weapons

bringing a weapon to school

past suicide attempts or threats

family history of violent behavior or suicide attempts

recent experience of humiliation, shame, loss or rejection

bullying or intimidating peers or younger children

a pattern of threats

being a victim of abuse or neglect (physical, sexual or emotional)

witnessing abuse or violence in the home

themes of death or depression evident in conversation, written expressions, reading selections or artwork

preoccupation with themes and acts of violence in TV shows, movies, music, magazines, comics, books, video games and Internet sites

mental illness, such as depression, mania, psychosis or biopolar disorder

use of alcohol or illicit drugs

disciplinary problems at school or in the community (delinquent behavior)

past destruction of property or vandalism

cruelty to animals

fire-setting behavior

poor peer relationships and/or social isolation

involvement with cults or gangs

little or no supervision or support from parents or other caring adult

Communication is key. Encourage your child to express any concerns he or she has about the behavior of others. Be prepared to speak to other parents if your child observes something concerning them about a peer’s behavior. Make sure that your child’s school authorities address any concerns that you bring to their attention.

Schools must have an appropriate procedure to evaluate children of concern to others. Find out if your child’s school has a procedure, and what it is. Find out how it monitors a child’s progress. Parents must impress upon their schools the need to adopt a comprehensive, team approach to ensure one person does not miss the warning signs someone else might see.

Q. What can schools do to prevent violent incidents?
A. After the recent school shootings, the U.S. Department of Education issued school safety guidelines to every school in the country.

Parents can ask school personnel if they have reviewed and implemented any of the Department of Education recommendations. In addition, parents and schools can refer to the recommendations of the National School Safety Center and the material provided by the National Education Association on its website. In coordination with parents, teachers, community agencies, community law enforcement and mental health professionals, the school should develop a plan for violence prevention and intervention.

Q. What can parents do to ease their own anxiety about sending their children to school?
A. School shootings challenge our belief that parents or schools can guarantee total protection and make us keenly aware of our children’s vulnerability to harm or injury.

These concerns are appropriate if they prompt parents to constructively review issues of safety within the family, neighborhood and school community. Taking constructive actions is an important way to alleviate anxieties that result from real-life events. If an act of violence exposes parents to a realistic concern, they must take practical steps to address this concern.

A parent might be experiencing severe anxiety if the shooting brings back previous memories of danger or loss. It can be helpful for parents to talk over these issues with other parents, school personnel and community or religious leaders.

CT Scans for All Kids with Head Injuries?

Tue, 28 Feb 2012 04:35:53 -0500

From high-profile former NFL players to soldiers returning from Afghanistan, the emerging picture of head trauma is not pretty. And of course the lingering, often-delayed effects of traumatic brain injury (TBI) aren’t limited to adults.

We’ve talked about children at risk of concussion when they play sports, and how manufacturers are developing “anti-concussion” athletic equipment.

Writing on KevinMD.com, malpractice attorney Maxwell S. Kennerly suggested that some practitioners still aren’t taking head injuries to children seriously enough.

Last year, he noted, the Centers for Disease Control and Prevention (CDC) reported “a growing awareness among parents and coaches, and the public as a whole, about the need for individuals with a suspected TBI to be seen by a health-care professional.” He pointed to the CDC’s Heads Up initiative as exemplary of efforts to raise consciousness that seeing stars isn’t a badge of athletic honor, it’s a reason to seek medical care.

What remains questionable, Kennerly wrote, is how prepared emergency and primary care physicians are to handle the increased number of head trauma cases they’ll see as a result of greater awareness of the problem.

“Viewed through a narrow lens, the solution to a suspected brain injury is obvious,” he wrote. “[I] f a kid complains about anything relating to their head, give them a CT scan. But CT scans come with their own costs and risks, not least exposing a developing brain to a year’s worth of background radiation.”

Too often medicine overtreats patients because technology enables it, insurance pays for it and/or doctors are worried about being challenged if they fail to perform every test within reach. Often a more conservative approach is better for the patient.

But when it comes to kids and head injuries, in deciding whether to order a CT scan for a kid with new head trauma, Kennerly says the conservative treatment approach is wrong.

“I’m not here to tell you where the CT / no-CT line should be drawn,” Kennerly claimed. “I can tell you, however, how I would draw that line as a medical malpractice lawyer when a parent comes in and tells me their doctor didn’t order a CT scan after a minor head trauma and their child later developed serious sequelae [i.e., a brain injury]."

In the world of medical malpractice, such a circumstance is known as “failure to diagnose.” As Kennerly explained, laypeople—like jurors—might well respond to the concept of incremental risk a witness might offer to defend against the charge of failure to diagnose. “One head CT scan,” the witness might say, “has more radiation than 20,000 trips through the TSA scanner at the airport.”

That sounds scary. But is it scarier than risking chronic, lifelong problems with the ability to think, loss of memory, headaches, attention deficit, mood swings and the other markers of brain injury?

Kennerly cited an article published in the January issue of Critical Decisions in Emergency Medicine, “Evaluation of Minor Head Trauma in Pediatric Patients," that argues for CT scans for children who present with any evidence of brain trauma.

If you’re the parent of a child who has fallen from a great height, who has emerged woozy from a collision in soccer, who claims his or her vision is blurred from a bang on the noggin, take him or her to your physician or emergency room immediately. And if the child doesn't show full recovery of normal consciousness very quickly, without any symptoms of brain trouble, a CT scan could be in order.

A British website offers guidelines for CT scans for anyone younger than 16 with a head injury, and the CDC's Heads-Up site helps observers spot the signs of concussion. They are:

loss of consciousness;

loss of memory;

abnormal drowsiness or sluggishness;

nausea or vomiting;

bruise, swelling or laceration on the head, behind the ears;

bruising around the eyes ("panda" eyes);

fluid leakage from ears or nose;

confused, dazed or stunned appearance;

personality changes;

headache;

problems balancing or abnormal clumsiness;

double or blurry vision; or

abnormal sensitivity to light or noise.

Cancer Drug's Short Supply Moves to Critical Stage

Sun, 19 Feb 2012 20:01:49 -0500

After it failed an inspection in November by the FDA, Ben Venue Laboratories in Ohio was shut down. No one in his or her right mind would want to take drugs manufactured in a place where mold was growing on the walls and machinery rust was falling into vials.

Now, the toll of the lab’s decrepit state might have to be paid by children with acute lymphoblastic leukemia (ALL) and a type of bone cancer, osteogenic sarcoma. They rely on a longtime cancer drug, methotrexate, that, as NPR reported, can mean the difference between death and cure.

Ben Venue was a principal supplier of injectable methotrexate. Now, its shortage is acute, the latest in long line of critical drug shortages. We’ve written often on the topic, and what is being done to address it.

According to NPR, hospitals throughout the country are “perilously close” to exhausting their supplies of the kind of methotrexate that treats ALL and osteogenic sarcoma. In a few weeks, substituting or delaying therapy might be forced on some patients. Their doctors aren’t certain how that will affect their chances of a cure, which are 9 in 10 with the right treatment.

One expert on drug shortages told NPR that most people don’t realize how many U.S. drug plants are in extreme disrepair, especially those that manufacture generic drugs. Ben Venue claims to have invested $250 million recently to upgrade its facilities, but it’s unclear when it will reopen. That lab also was the sole manufacturer of Doxil, an ovarian and breast cancer drug that’s now unavailable.

But there’s cause for hope. The FDA said the methotrexate and Doxil crises soon may be over. Three other manufacturers are increasing production of the specific kind of methotrexate at issue, and they told the feds that additional supplies should be available within weeks.

The FDA is considering licensing a foreign company to make Doxil, a measure it has taken eight times in the last year to address other drugs in critical shortage.

And last week Ben Venue posted a message on its website that it was working with the FDA to expedite the availability of methotrexate and immediately would begin releasing a limited supply to oncology clinics, hospitals and pediatric facilities. "We hope this supply will help address near-term patient needs while other companies licensed to manufacture methotrexate increase production," the statement read.

Doctors were optimistic about restocking hospital supplies of methotrexate, but, as one told NPR, “[T]his is not the last shortage we’ll have to cope with.”

Panel Recommends Suicide Warning Be Added to ADHD Drug Label

Mon, 13 Feb 2012 00:38:55 -0500

A panel of pediatric experts has recommended that the FDA change the label for Focalin to address the risk of suicidal thoughts by children, according to Reuters.

The drug is prescribed for attention deficit disorder and is manufactured by Novartis AG. It was approved for children 6 and older in 2001.

Children with ADHD are excessively restless, impulsive, easily distracted and often have behavioral issues. Symptoms generally are relieved with behavioral therapy and medication (at least short term; the long term benefits of medication are less clear).

The FDA is not required to follow the advice of its panels, but usually does. It is required to hold regular advisory meetings to review the safety of drugs used by children. The panel also recommended that Focalin's label acknowledge the risk of anaphylaxis, an allergic reaction, and angioedema, a type of swelling beneath the skin.

The current label for Focalin advises patients about psychotic or manic side effects, but does not mention suicidal thoughts.

The FDA received eight reports of suicidal thoughts by children or adolescents who took the drug over the last six years. This risk did not present during the clinical trials of the drug, and the number of such reports is tiny in comparison to the number of patients taking it. If your child is taking Focalin, he or she shouldn’t stop taking it. But do consult your pediatrician.

Diagnoses of ADHD (attention deficit hyperactivity disorder) have boomed in recent years; an estimated 3 to 5 kids in 100 are affected. Some experts question whether these diagnoses are made too quickly and drugs prescribed too easily. We’ve addressed the suitability of prescription drugs for ADHD.

According to Reuters, approximately 2.7 million people in the U.S. have prescriptions for ADHD drugs. Approximately 1.8 children received prescriptions for Focalin or its generic versions from 2005 to 2011.

A Doctor's Advice to Parents About Vaccinations

Tue, 07 Feb 2012 12:09:49 -0500

There’s been a lot of chatter in recent years about childhood vaccinations and their alleged potential to do more harm than good. Most of the cautionary tales shed more heat than light, and endanger the children they purport to protect.

Writing in the New England Journal of Medicine last week, Dr. Douglas S. Diekema published a thoughtful commentary about the struggle some parents have with the idea of vaccinations and how the medical community can encourage people to get their kids appropriately protected with vaccines.

He recounted the story of a mother who wanted to follow an “alternative” schedule versus that driven by best medical practice. The pediatrician went along, prompting the mother to believe that the recommended schedule was no better than her alternative.

Thousands of parents are like her. Some choose not to vaccinate their children at all. Some have religious or philosophical reasons, some want to spare their children from uncomfortable procedures, some just don’t believe the benefit outweighs the risk. Many have no experience with or knowledge of serious vaccine-preventable diseases like polio or measles.

But that doesn’t mean there aren’t contemporary health threats that vaccines neutralize. In 2010, California reported more than 9,000 cases of pertussis (whooping cough) — more than the state had seen since 1947. Nearly 9 in 10 involved infants younger than 6 months, a group too young to be adequately immunized and largely dependent on “herd immunity” (when vaccinating a significant portion of a population imparts protection for all members who have not developed immunity). Ten of the California infants died from the infection.

Because some parents—those with religious or strong personal beliefs—probably aren’t capable of being persuaded to vaccinate their children, whether for their own good or the good of society—Diekema focuses on the parents who question the safety of vaccines or have logistic or financial concerns.

1. Eliminate money barriers and disincentives to vaccination. Even small co-payments or fees pose substantial barriers for some families. Public health clinics are one option, but can require travel and time away from work — all disincentives to following through. “Incentives can take several forms, including reduced insurance rates, tax rebates or direct payments,” he writes.

2. Strengthen and enforce school-entry requirements. Such requirements effectively boost immunization rates for school-age children, but vary widely by state in terms of exemptions allowed. “All states allow exemptions for medical reasons, 48 for religious reasons and 20 for philosophical reasons.”

Acknowledging that eliminating exemptions for religious and personal beliefs would encounter substantial resistance, Diekema remains resolute. “The exemption process should not be easier or less costly than the vaccination process. Obtaining a religious or personal-belief exemption should at least require a visit to the physician's office, including counseling on the risks posed by remaining unvaccinated; insurance should pay for such visits.” States could require exemption requests to be signed by both parents if both are legally authorized. “Although such measures wouldn't change the stance of the most resistant parents, they would eliminate many exemptions sought because of convenience rather than conviction.”

Diekema also points out that lax enforcement of school-entry requirements communicates that vaccination is merely bureaucratic rather than a way to ensure students' safety.

3. Address misinformation about vaccines promptly and aggressively. “False or misleading information about vaccination is widely dispersed by a few influential individuals, self-described vaccine-safety advocates and some clinicians. Public health officials and professional organizations should respond swiftly to dishonest or unbalanced portrayals of vaccination.”

4. Clinicians, health-care organizations and public health departments must be persuasive. “Data and facts,” Diekema states, “no matter how strongly supportive of vaccination, will not be sufficient to compete with the opposition's emotional appeals. The use of a compelling story about a single victim of vaccine-preventable illness is far more likely than data to move an audience to action.”

This outreach, he says, is the responsibility of primary care providers. “Parents will be most receptive to considering vaccination if they believe their provider is primarily motivated by the welfare of the individual child rather than an abstract public health goal.” As we’ve said over and over, a doctor’s willingness to listen respectfully, encourage questions and respect parental concerns are essential for any professional health-care provider. Accurate information about both risks and benefits is crucial to maintaining trust, and must include a discussion of risks associated with both remaining unvaccinated and delaying certain vaccines.

5. Set an example. “We're unlikely to achieve optimal vaccination rates until health-care professionals comply with vaccine recommendations for themselves and their children. The unwillingness of many clinicians to submit to influenza vaccination each year is disgraceful, sets a poor example and gives patients reason to question the safety and efficacy of vaccines.”

We couldn’t agree more: Doctor, heal thyself.

First published on Technorati.