One of Congress's most powerful committees is calling for a government investigation into whether experimental surgeries were carried out without the consent of patients, including heart valves that were implanted for years at UPMC Children’s Hospital of Pittsburgh, the Pittsburgh Post-Gazette has learned.
The House Committee on Energy and Commerce wants the Food and Drug Administration to investigate whether patients who received rare implants were properly informed, including the case of a Pennsylvania mother whose 14-year-old son received an artificial valve from UPMC that later failed and had to be removed.
In a letter signed by three majority members last month, the panel asked the agency to determine whether doctors at UPMC had the necessary permission to insert a device used to regulate blood flow between the heart and lungs on vulnerable children.
Investigators for the panel have been reviewing published reports about UPMC doctors who implanted what’s known as a MASA valve in young patients, some struggling to stay alive, records and interviews show.
"They want to know what the heck is going on," said Dr. Nalini Rajamannan, a Chicago cardiologist and a consultant in the case of the 14-year-old boy. "It's a big problem. Children are a vulnerable population and we need to know what procedures were performed."
As part of the inquiry, House members asked the FDA to turn over records of any other investigations by the agency into experimental medical devices used on patients since 2019 without their consent.
One of the main reasons for the probe is to determine whether the FDA is using its authority to ensure that patients are informed of the risks before undergoing life-altering procedures.
In emails to the Post-Gazette, UPMC said the hospital provides necessary information to patients to obtain their consent and the valves implanted in children were carried out under an established medical practice.
"This technique of building and implanting specific valved-conduits for pediatric patients is a longstanding standard of clinical care," the hospital system said, adding that the fabric used in the device is approved by the FDA.
A review by the UPMC Children’s compliance office of the 14-year-old’s case said that "no experimental valves" were placed in the teenager and that the graft inserted was “appropriate,” records show.
But one of the key materials used to construct the valve was not approved for use in a heart implant, according to records turned over to the committee, and that’s expected to be part of the panel's inquiry into whether families should have been notified, the letter said.
Critical implant
The procedure under scrutiny was developed by cardiothoracic surgeon Masahiro Yoshida, who began to implant the custom-made device in the hearts of young patients with defective valves.
In the six years after he joined UPMC in 2008, Dr. Yoshida carried out nearly 90 of the implants on children with congenital valve defects, according to a 2014 article in the hospital system's Pediatric Insights magazine.
A subsequent version of the valve, said to be “equivalent” to what Dr. Yoshida designed, was approval for a clinical trial by the FDA in 2022 – years after UPMC started using the device in surgeries. Dr. Yoshida is now practicing medicine in Japan.
Dr. Rajamannan said the device implanted in the teenager required FDA approval before any of the surgeries could be conducted because it's a high-risk device that needed to be screened for safety under a clinical trial.
"They were putting these [devices] in patients without any approval," said Dr. Rajamannan, a former chair of a National Institute of Health working group on heart valve disease. “These were experimental.”
The inquiry by the House panel comes months after the mother of the teenager filed a complaint with the Pennsylvania attorney general's office, alleging she wasn’t told before her son's surgery in 2017 that he would be receiving an "experimental" valve that was not FDA approved.
Ann-Marie Swanson, a registered nurse, said her son, Michael Carlo, now 21, was born with a septal defect — a hole in the wall between the right and left ventricle — and underwent his first open heart surgery when he was nearly 2 years old.
By the time he was 14, the left ventricle was enlarged and, due to other complications of the heart muscle, the normal flow of blood was not reaching his body.
Ms. Swanson said she agreed to allow Dr. Victor Morell, chief of the hospital’s pediatric cardiothoracic surgery division, to proceed with what he referred to as the MASA valve. "I had never heard of it," she said, adding that she was told there was less chance of rejection.
Valves can fail for a variety of reasons, necessitating follow up surgeries to replace them.
‘Running out of breath’
Four years later, her son began to experience symptoms of congestive heart failure, including chest pains and shortness of breath. "He was constantly fatigued, running out of breath," she said.
By early 2022, tests at UPMC showed the implant had failed. Blood that was supposed to flow to the lungs was leaking back into the right ventricle, records show.
"I started searching for info on the MASA valve to see if this is normal, [and] come to find out, it doesn't appear to have any FDA approval," she said. “I wanted to scream.”
In late 2022, her son underwent his fourth open heart surgery, at the Mayo Clinic in Rochester, Minn., where the MASA valve was removed and a bioprosthetic valve was successfully implanted.
In the ensuing years, Ms. Swanson began to file complaints with the state attorney general's office, the state Department of Health, the state medical board and eventually to U.S. Rep. Mike Kelly, R-Pa., alleging the procedure was experimental and that it had not been FDA approved.
“It was very frustrating,” she said. “My son went through congestive heart failure for a year.”
Records show a UPMC Children's Hospital compliance officer reviewed her complaint with the attorney general's office in 2023 and challenged several allegations, saying the "standard of care procedure” was "appropriate."
The officer noted the term "MASA valve” described in the UPMC medical record was "used colloquially" and did not represent the type of valve that her son had received: a tri-leaflet PTFE valve conduit.
However, a review of medical records by the Post-Gazette shows the term, "MASA” valve was noted 44 times in her son's UPMC physician, nursing, and post-operative reports in 2017. And the Mayo Clinic — where the device was removed — referred multiple times to the implant as a "MASA valve," which was kept by the clinic after the surgery.
The UPMC compliance officer also noted that the material used in the device — Gore-Tex, according to the hospital's own records — was approved by the FDA.
Records from Gore & Associates show the material is not approved for use in artificial heart valves, and that in one case in 2019, the fabric was used in a heart valve procedure on a 10-year-old who was injured, according to a company report to the FDA.
“This device does not have an FDA approved indication for use in pulmonary valved conduits,” Gore said in an email to the Post-Gazette.
What's not clear is how many surgeries took place at UPMC that used the material during the valve implants.
Last week, Ms. Swanson filed a complaint with the FDA, describing the use of the Gore material in her son’s failed device, records show.
"It was off-label," she said in an interview.
In a letter to the state attorney general's office last year, UPMC's compliance office cited a federal regulation that discusses the use of the material in a vascular graft prosthesis.
However, that provision says the material should be used to repair or replace sections of blood vessels — not a heart valve.
A spokesman for the House committee declined comment, saying the letter “speaks for itself.”
A representative for Mr. Kelly, who referred the case to the House committee, also declined to comment.
Audra Harrison, a spokeswoman for the FDA, said the agency will respond directly to the committee, but does not have oversight of consent outside of a clinical trial.
Records show the FDA initially turned down a request by Mr. Kelly in August to investigate the case, saying the agency did not have the authority to look into issues of patient consent.
But the House pushed back in its letter last month, saying that when it comes to experimental medical devices, the FDA does have direct responsibility.
Peter Pitts, a former FDA associate commissioner, told the Post-Gazette that “it’s very clearly within the area of FDA authority. And, in fact, the agency just recently, like within the last month, has spoken about new types of guidance on ways that patients can be asked to provide consent.”
Mr. Pitts said the House’s inquiry is “completely legitimate” in light of the questions being raised about the procedures carried out for years at UPMC.
“When it comes to these types of medical devices, they’re always on the cutting edge of advancing the technology. That doesn’t mean they can be cavalier about consent,” said Mr. Pitts, who now leads the Center for Medicine in the Public Interest.
FDA responsibility
UPMC and the FDA both said doctors can resort to off-label uses of devices to treat patients.
Citing the American Academy of Pediatrics, UPMC said the off-label use of medical and surgical devices in children can be “necessary and appropriate when there is no device available that has been approved or cleared by the FDA for the specific pediatric indication.’’’
In this case, however, there were several approved implants, including bioprosthetic and mechanical valves, that had been cleared by the FDA dating back more than a decade, records show.
The congressional inquiry comes as a startup with close ties to UPMC has launched its own clinical trial for the MASA valve, which it describes as “equivalent” to what UPMC has been installing for years.
UPMC said it’s not involved in the ongoing clinical trial and has never implanted one of the MASA valves made by Peca Labs.
The lab, which worked closely with Dr. Yoshida during his years at UPMC, launched the clinical trial with other hospitals last year after obtaining an Investigational Device Exemption from the FDA.
Peca Labs has been promoting the MASA valve for years, including a 2013 Facebook post that said the valve was “fully developed” and had been implanted in 49 patients.
By 2020, in documents posted by its investors, Peca Labs had described positive results from “70 human implantations of the intra-operatively constructed version.”
Michael Carlo, the 21-year-old son of Ms. Swanson, said after recovering from the failed implant, he’s hoping the House investigation can resolve his own questions about the implant surgeries.
“It took a year out of my life," said Mr. Carlo, who now works for a landscape company in North East, Erie County. “I’m just one person. I couldn’t imagine how many other people this happened to. It’s another step forward in hopefully informing other families.”
Post-Gazette reporters Michael Korsh and Jonathan D. Salant contributed to this report.
Evan Robinson-Johnson: ejohnson@post-gazette.com; Michael Sallah: msallah@post-gazette.com; @MikeSallah7
First Published: March 24, 2024, 9:30 a.m.
Updated: March 25, 2024, 7:12 p.m.