Next great medical innovations that could save children

What medical advancements could save the lives of our children in the future? That’s the question members of the American Academy of Pediatrics asked themselves. Their answers were published last week in the journal Pediatrics.

“We thought about the next 40 years and what fields of study might lead to great medical achievements,” said co-author Dr. Tina Cheng, Director of the Department of Pediatrics at John Hopkins Hospital.

“We wanted to identify areas that could have the greatest impact on improving children’s lives,” added co-author Dr. Clifford Bogue, a critical care pediatrician at Yale University School of Medicine. “Because we believe child research is a great investment, we thought it would be helpful for advocating for funding in the future.”

What has pediatric research accomplished?

Studies say research into diseases that disable and kill children falls far behind those done on adults in both scope and quality.

“The amount of research being done with children and infants has been declining,” said Cheng, “and funding for pediatric research has been flat or declining as well.”

To draw attention to the disparity, the American Academy of Pediatrics created a campaign: the “7 Great Achievements in Pediatric Research.” By canvassing board members and asking them to rank key advances in childhood health, the AAP created a list of seven of the greatest pediatric research achievements of the last 40 years.

Published in 2015, the historical advances included the famous “Back to Sleep” campaign to prevent sudden infant death syndrome, or SIDS; the all-out push for laws requiring car seats and seat belts for children of all ages; preventing disease with life-saving immunizations and finding a successful treatment for the most common childhood cancer, acute lymphocytic leukemia.

Other great achievements they highlighted were helping premature babies breathe with a surfactant therapy, reducing HIV transmission from mother to baby and increasing the life expectancy for children with sickle cell anemia and cystic fibrosis.

“We wanted to highlight to the public and to legislators just what the value was for the research dollars that were spent, most of which were funded by the National Institutes of Health,” said Bogue, “to show the huge impact the research had on saving and improving the lives of children and their families.”

What could the future hold?

Drawing on that same survey, here are the advancements the AAP’s Committee on Pediatric Research predicts could occur over the next 40 years.

More childhood immunizations

The AAP strongly advocates for routine vaccinations as one of the best ways to protect children from dangerous diseases. Two examples in the survey are rotavirus, a severe gastroenteritis, and Haemophilus influenzae type b (Hib), a leading cause of bacterial meningitis.

Before vaccines were developed, rotavirus killed about 450,000 children worldwide each year. In the United States Haemophilus influenzae type b took the lives of 1,200 children annually.

“We’d had incredible success,” said Cheng. “A lot of residents-in-training have never seen tetanus, influenza or polio. But there are always new and emerging diseases, and even some of the vaccines we have need to be better.”

Some of the vaccines the survey participants hope to see in the future include Zika, Ebola, certain cancers, and the flu.

“As we see from history, there are regular pandemics in our world and we are due for one,” said Cheng. “Many point to the flu as most likely culprit. It’s especially hard on the young and elderly and those with chronic illness.”

Cancer immunotherapy ‘moonshot’

“We need to find an innovation that can really make a difference with childhood cancer, and immunotherapy may be the next great thing, the sort of ‘moonshot’ we need,” said Cheng.

Using one’s own immune system to target cancer cells is cutting-edge research that is showing enormous potential. While most current research focuses on adults, it’s also expected to reap huge dividends in childhood cancer treatment, especially for cancers notoriously difficult to treat.

“Immunotherapy is booming right now and that’s why we picked it,” agreed Bogue. “But children are often left out of that research. One reason is safety issues, but they are also a very small market for pharmaceutical companies.

“Yet it’s very important to do the research in children,” added Bogue. “Because if you can cure a cancer with immunotherapy at age 10 or 15 instead of 65 you have greatly magnified the impact of that therapy.”

Genomic testing

As understanding of our human genome continues to grow, so will the ability of science to discover genetic mutations and test for them “prenatally, at birth, and throughout the life course”, says the committee, which will allow us to better predict, diagnose and prevent disease.

“Right now when you have a baby, she gets a heel stick for a few drops of blood and they test for a host of genetic conditions that if found early, can be much more treatable,” said Cheng.

“30% of disease in young infants and children is due to a genetic disorder and that’s just based on the small number of genes we have identified and confirmed to date,” added Bogue.

He points out that as science works on the human genome, it’s getting cheaper and easier to gather information, take it to the lab and do the work to figure out why a mutation causes a disease, the pathways involved and how science might intervene.

“That’s the big promise in genomic information,” Bogue said. “We can not only tell parents what their child has, but also why and even come up with new treatments.”

Early interventions

Wouldn’t you like to know as a child if you might get type 2 diabetes, heart disease or cancer later in life?

“Many adult diseases are present in childhood,” said Bogue, “and follow a pathway that may take years to develop.”

As research on the human genome progresses, it is beginning to pinpoint the environmental, behavioral and genetic triggers that might flip a disease switch into action.

“For example, science is beginning to show that young adults who develop schizophrenia may have had brain abnormalities as infants,” explained Bogue. “So we can now begin to predict and intervene before the disease becomes severe.

“In the future, if we knew someone was going to become a diabetic at 25, we could start intervening at age 5. Just think of the impact on that child’s life, as well as society.”

Impact of social, behavior and environmental factors

Exposure to toxic chemicals in our environment has long been known to impact a child’s development. For example, a mother or father’s exposure to heavy metals, such as arsenic, mercury and lead, can lead to lower IQ and other neurologic and psychological disorders in their children.

But according to the AAP committee, there are social and behavioral ways that can limit a child’s potential. They point to maternal malnutrition and other stresses, as well as childhood exposure to violence as stress factors that can “clearly re-program a variety of gene interactions.”

“Even interactions that parents have with their child, such as reading to them versus propping them in front of a TV,” said Bogue,” can have a huge impact on their child’s development. We need to focus on those factors as well.”

Improving our systems of care

What’s the third most common cause of death in the United States? It’s not a disease. According to researchers at Johns Hopkins, medical errors by health care providers lead to approximately 251,000 deaths each year. The AAP committee sees advances in service among health care providers to be a key area of growth that will have an impact on children’s health in the future.

“We’re not talking on the individual level, but improving health care itself,” explained Cheng. “If we decrease medical errors and provide the same highest quality of care every time we see a patient, we will save lives.”

“It’s about systems of care,” agreed Bogue. “How do we engineer the care we deliver so that it gives us better outcomes? And oddly it often ends up being cheaper.”

Increasing global access to care

Today, many children around the world are without access to even the basics of proper health care, such as sanitary water, medical supplies, and food. Nearly one in five children have not received routine immunizations, much less the many advances that have occurred in the last 40 years.

Knowing that intervention A reduces disease B by 90% is great medicine, said Bogue, but how do you actually implement that science so providers and even whole countries are doing it?

“It can take years for those breakthroughs to become standard practice,” he explained. “We need to markedly shorten time frame from knowing when it works to putting it into practice so that people can benefit from it.”

Are these seven medical advancements a definite for the future? No, and they aren’t meant to be, said the authors.

“It’s true we can’t really predict what comes next, but we need to be thinking about it and where are we going,” said Cheng. “The take home message here is that pediatric research has led to improved life expectancy and there are emerging diseases and issues we need to combat. We just want to start the conversation.”

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Doctors charged in first genital mutilation case in US

In the first federal case involving female genital mutilation filed in the United States, two Michigan doctors and the wife of one of the doctors have been charged with performing the banned procedure on two 7-year-old girls.

Dr. Fakhruddin Attar, 53, and his wife, Farida Attar, 50, were arrested Friday at their medical office in Livonia, Michigan, west of Detroit.

They were charged with three federal criminal counts including conspiracy, female genital mutilation and aiding and abetting.

Detroit emergency room physician Jumana Nagarwala, 44, was arrested April 12 and is currently in jail awaiting trial after a federal judge deemed her a flight risk and a threat to the community.

The three defendants belong to a “religious and cultural community” that investigators allege practices female genital mutilation on young girls — a painful surgical procedure to remove part of the clitoris or clitoral hood to suppress female sexuality.

During a court hearing April 17, Nagarwala’s defense attorney, Shannon Smith, told a judge the procedure did not involve cutting and was religious in nature, CNN affiliate WXYZ reported.

Smith argued the procedure is practiced by the Dawoodi Bohra, an Islamic sect based in India, and that the clinic was used to keep procedures sterile, WXYZ reported.

The Detroit Free Press reported from the hearing that Smith said her client removed membrane from the girls’ genital area using a “scraper” as part of a religious practice. The girls’ parents would then bury the membrane in the ground in accordance with their religious custom, Smith said, according to the Free Press account.

Clinic under surveillance

A 2012 US Centers for Disease Control and Prevention report found that roughly 513,000 women and girls in the United States were at risk of undergoing female genital mutilation, which was more than twice an earlier estimate based on 1990 data. The World Health Organization considers the procedure a violation of human rights of girls and women.

According to the complaint, the case against Nagarwala and the Attars dates to at least February, after the FBI had received information that the procedure was being performed at the clinic. Court documents do not indicate the source of the information.

Investigators were watching the clinic and saw the two girls and their mothers arrive at the Burhani Medical Clinic, which Attar owns.

Attar, his wife and Nagarwala were already inside.

The girls, both from Minnesota, later told investigators their mothers said they were going to Detroit for “a special girls’ trip,” according to court documents.

According to those court documents:

One girl said they had gone to the doctor’s office because their “tummies hurt” and the doctor had to “get the germs out.” The second girl said that after the procedure, “she could barely walk, and that she felt pain all the way down to her ankle.”

Both girls said their parents told them not to talk about the procedure. When investigators questioned the parents, one couple described the procedure as a “cleansing” of extra skin.

Investigators believe there are more young victims in Michigan.

Nagarwala is accused of performing the mutilation while the Attars allegedly assisted.

Attorney: Attar ‘will be vindicated’

“Dr. Attar is not aware of any crimes that occurred at his clinic,” Attar’s defense attorney, Mary Chartier, told CNN. “He has confidence that he will be vindicated through the justice system because he has done nothing to violate the law.”

Attar told investigators that Nagarwala used his clinic to treat girls between ages 6 and 9 for genital problems and that Nagarwala didn’t charge for her services. Attar said his wife was present during the examinations to “comfort” the girls and “hold their hands,” according to court documents.

Attar said Nagarwala saw patients in his clinic five to six times a year.

“Dr. Attar voluntarily spoke with the FBI without an attorney,” said Chartier. “He has no criminal history. He knew his colleague had been arrested, yet he continued to live in his home and work at his clinic serving his patients. He certainly did not try to flee. He is a respected and trusted community member.”

Farida Attar’s attorney, Matt Newburg, told CNN he did not wish to “comment on the charges.”

Mosque says it’s assisting investigators

The Detroit News reported that members of the Dawoodi Bohra sect in the area where the defendants live and work belong to the Anjuman-e-Najmi mosque, the only Dawoodi Bohra mosque in Michigan.

Leaders of the mosque released a statement Friday saying they are offering assistance to investigators, according to the News.

“Any violation of US law is counter to instructions to our community members,” the statement said.

“It is an important rule of the Dawoodi Bohras that we respect the laws of the land, wherever we live,” the statement continued. “This is precisely what we have done for several generations in America. We remind our members regularly of their obligations.”

CNN’s calls to Nagarwala’s attorney and the mosque were not returned.

Nagarwala, who has four children of her own, has no prior criminal history, according to court documents.

She has been charged with female genital mutilation, transportation with intent to engage in criminal sexual activity and conspiracy, and making a false statement to a federal officer.

Nagarwala’s employer, the Henry Ford Health System, told CNN in a statement, “the alleged criminal activity did not occur at any Henry Ford facility. We would never support or condone anything related to this practice. The doctor was immediately placed on administrative leave and her clinical privileges have been suspended.”

A detention hearing for the Attars is scheduled for April 26 at 1 p.m. ET. It’s not clear whether any of the defendants have entered a plea.

No charges have been filed against the parents of the girls.

The FBI has a tip line for victims of female genital mutilation, or anyone who might suspect such activity. They can call 800-CALL-FBI (225-5984) or submit a tip via FBI.GOV/FGM.

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Possible shutdown, health care quagmire awaits U.S. Congress

Lawmakers returning to Washington this coming week will find a familiar quagmire on health care legislation and a budget deadline dramatized by the prospect of a protracted battle between President Donald Trump and Democrats over his border wall.

Trump’s GOP allies control Congress, but they’ve been unable to send him a single major bill as his presidency faces the symbolic 100-day mark on April 29 — the very day when the government, in a worst-case scenario, could shut down.

Feeling pressure to deliver results, Trump wants to revive a troubled health care measure from House Republicans to repeal and replace President Barack Obama’s Affordable Care Act. Trump also hopes to use a $1 trillion catchall spending bill to salvage victories on his promised U.S.-Mexico border wall, a multibillion-dollar down payment on a Pentagon buildup, and perhaps a crackdown on cities that refuse to cooperate with immigration enforcement by federal authorities.

Congress faces a midnight Friday deadline to avert a government shutdown. But negotiations on the spending measure, a huge pile of leftover business from last year that includes the budgets of almost every federal agency, have hit a rough patch.

Rank-and-file Republicans received few answers on a Saturday conference call by top House GOP leaders, who offered little detail and said deals remained elusive on both health care and the catchall spending measure, with no votes scheduled yet.

It’s looking like a one- or two-week temporary measure will be needed to prevent a shutdown and buy time for more talks. Negotiations have faltered because of disputes over the border wall and health law subsidies to help low-income people afford health insurance.

Trump’s Capitol Hill allies had been tempering expectations that the president will win much in the budget talks. Democratic support will be needed to pass the spending measure and Republicans fear taking the blame if the government shuts down on their watch.

“We have the leverage and they have the exposure,” House Democratic leader Nancy Pelosi of California told fellow Democrats on a conference call Thursday, according to a senior Democratic aide. Pelosi wants the spending bill to give the cash-strapped government of Puerto Rico help with its Medicaid obligations, and Democrats are pressing for money for overseas famine relief, treatment for opioid abuse, and the extension of health benefits for 22,000 retired Appalachian coal miners and their families.

An additional Democratic demand is for cost-sharing payments to insurance companies that help low-income people afford health policies under Obama’s health law. The payments are a critical subsidy and the subject of a lawsuit by House Republicans. Trump has threatened to withhold the money to force Democrats to negotiate on health legislation.

Trump’s presidential victory makes it “completely reasonable to ask and to insist that some of his priorities are funded,” White House budget director Mick Mulvaney said in an interview. “We are more than happy to talk to the Democrats about some of their priorities but we encourage them to recognize that they are a minority party.”

Both the White House and Democrats have adopted hard-line positions on Trump’s $1 billion request for a down payment on construction of the border wall, a central plank of last year’s campaign. Talk of forcing Mexico to pay for it has largely been abandoned. But in an interview with The Associated Press on Friday, Trump stopped short of demanding that money for the project be included in the must-pass spending bill.

Health care is on a separate track and facing trouble, too. The White House is pressing House Republicans to rally behind a revised bill so GOP leaders can schedule a vote this coming week that could let Trump fulfill a 100-days promise.

A quick vote, let alone approval, seems unlikely.

GOP leaders have shown no desire to revisit the issue until they’re assured there’ll be no replay of the legislative train wreck from March. The failure of that earlier attempt stung Trump and House Speaker Paul Ryan, R-Wis. The measure would have repealed much of Obama’s 2010 overhaul and replaced it with fewer coverage requirements and less generous federal subsidies for many people.

As part of the White House drive to resuscitate the bill, members of Trump’s team including Vice President Mike Pence and chief of staff Reince Priebus have made multiple calls to Republicans.

Two leaders of the House GOP’s warring moderate and conservative factions devised a compromise during Congress’ recess to let states get federal waivers to ignore some requirements of the health law. Those include one that now obligates insurers to cover specified services such as for mental health, and one that bars them from raising premiums on seriously ill patients.

But there are widespread doubts that the new attempt has achieved the support it needs.

Rep. Dan Donovan, R-N.Y., an opponent of the bill, said last week that “it doesn’t cure the issues that I had concerns” about the bill. The moderate said his objections included changes to Obama’s law that would still leave people with excessive out-of-pocket costs.

The potential amendment was brokered by Rep. Mark Meadows, R-N.C., who heads the conservative House Freedom Caucus and Rep. Tom MacArthur, R-N.J., a leader of the moderate House Tuesday Group.

Ryan called off a March 24 House vote on the measure after realizing that objections by conservative and moderate Republicans would have assured its defeat. Democrats were uniformly against the legislation.

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Clamp down on visas for doctors could hurt these parts of the U.S.

Limiting the number of foreign doctors who can get visas to practice in the United States could have a significant impact on certain hospitals and states that rely on them, according to a new study.

The research, published online in the Journal of the American Medical Association this week, found that more than 2,100 U.S. employers were certified to fill nearly 10,500 physician jobs through H-1B visas nationwide, in 2016. That represents 1.4% of the physician workforce overall.

There were wide variations by state and employer, however.

Employers in New York, Michigan and Illinois accounted for the most H-1B visa applications for foreign physicians, nearly a third of the total. North Dakota, however, had the most applicants as a percentage of its physician workforce: 4.7%.

The top three employers that submitted applications for the most doctors through the visa program were William Beaumont Hospital in southeastern Michigan, with 470 physician applications, Bronx-Lebanon Hospital Center in New York City, with 213, and Cleveland Clinic foundation in Ohio, with 180.

“People underestimate the fragility of certain hospitals and their reliance on certain physicians for their functioning,” said study co-author Peter Kahn, who’s graduating from Albert Einstein College of Medicine in the Bronx this spring.

The H-1B visa program allows employers to hire highly skilled professionals from abroad to fill employment gaps in the U.S., typically in high-tech, science, engineering and math jobs. But hospitals use the program as well, often to recruit doctors to serve in rural or underserved urban areas. The number of visas is capped at 85,000 annually.

That could change.

On Tuesday, President Donald Trump signed an executive order reiterating his administration’s priority to buy American goods and hire American workers. Among other things, it requires federal agencies to suggest reforms to the H-1B visa program to ensure the visas are awarded appropriately.

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Miami Gardens family wins $33.8 million medical malpractice award against federal government

The federal government will have to pay a Miami Gardens family a $33.8 million award in a medical malpractice lawsuit after a judge ruled on Monday that a federally employed doctor’s decisions caused a teen’s baby boy to suffer irreversible brain damage.

U.S. District Judge Robert N. Scola awarded the baby’s mother, Marla Dixon, $3.3 million, and the father, Earl Reese-Thornton, $1.1 million for their pain and suffering. Scola awarded $21.7 million for the boy’s economic damages and $7.6 million for his pain and suffering. 

Dixon was a patient at Jessie Trice Community Health Center in Miami Gardens. Dr. Ata Atogho was the on-call physician at the community center, which provides care to the uninsured and undocumented with the help of federal funding. 

While she was at North Shore Medical Center, Dixon was also under the care of Yolande McCray, an experienced labor and delivery nurse. McCray testified that Atogho’s claims that he offered Dixon a cesarean section and that she declined were false. 

Both McCray and Reese-Thornton testified that the 19-year-old mother was yelling, “Just cut me!” 

McCray said Atogho ignored her request despite the baby’s slowed heart rate and left Dixon for another delivery. He was also reportedly on the phone with his financial adviser. When the baby was born on Dec. 2, 2013, he wasn’t breathing.

“At the anticipated, joyful moment of birth of a crying, bouncing baby, they are instead presented with the dreadful specter of a blue, floppy, lifeless child,” Scola wrote in his order.

It was not a high-risk pregnancy. After the baby was revived, the parents learned that he had suffered brain damage because of a lack of oxygen. Medical experts from both the defense and the plaintiffs agreed that a C-section could have prevented her baby’s brain damage.

“The court has considered all the evidence in the case, including the testimony of the defendant’s life expectancy expert, who has opined [the 3-year-old boy] has a life expectancy of an additional 9 to 12 years,” Scola wrote.

Florida’s statutory caps did not limit the award, which the government will have to pay in periodic payments. 

Charles White of the U.S. Attorney’s Office in Miami led the federal government’s defense team. The family’s attorneys were from two Miami law firms: Vidian Mallard and Richard Sharp of Mallard & Sharp and Lauri Ross of Ross & Girten. 

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New labels to remind parents codeine, tramadol can kill child

The Food & Drug Administration announced on Thursday that they will be strengthening the requirements for drug labels for codeine and tramadol to protect children. 

Both opioid medicines are in some cough and cold remedies, and were commonly prescribed by doctors and dentists despite the risk of life-threatening breathing problems in children.

The FDA also warned nursing mothers who are taking the medications can pass unsafe levels of opioids to their babies through breast milk. 

Children and teens ages 12 to 18 shouldn’t take them if they are obese, have obstructive sleep apnea or a weakened respiratory system. The risks factors can increase their chances of serious breathing problems, according to the FDA. 

The American Academy of Pediatrics  strengthened its warnings about prescribing codeine for children late last year, because of reports of deaths.

Dr. Charles Cote, a Boston anesthesiologist and co-author of the report, told the Associated Press that considering other remedies such as acetaminophen and ibuprofen for pain and simple remedies such as ice or popsicles after tonsillectomies are better options. 

“Maybe a little pain is better than the alternative,” Cote said.

 

 

 

 

 

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