Family Spotlight

Meet TJ Wilson … A COTA Miracle

Four-year-old TJ Wilson is alive, growing and able to celebrate Valentine’s Day this year because of a donor heart he received nearly three years ago.

TJ’s story is one that strikes terror in a parent’s heart. According to Randy and Lori Wilson, TJ was a happy and healthy toddler until one day in June 2007, while being treated for flu-like symptoms, TJ suffered a cardiac arrest … and the Wilson family’s life changed forever. Doctors quickly went to work on the toddler. Randy and Lori watched while doctors tried unsuccessfully to re-establish a heart beat. TJ was placed on a heart/lung bypass machine and the waiting began. Several weeks later they were told TJ had restrictive cardiomyopathy and his only chance for survival was a heart transplant. The medical team at Children’s Hospital of Pittsburgh moved quickly to equip TJ with a Berlin Heart to buy the time needed to find a donor heart. On July 16, 2007, TJ received his new heart, and his second chance at life.

During these upside-down days, the Wilsons heard about the Children’s Organ Transplant Association (COTA). “COTA gave our family and friends fundraising guidance and structure so they could do something to help us when we were feeling so very helpless,” said Lori. “At a time when we felt hopeless, COTA empowered our family and friends. So many people wanted to help: COTA turned that desire to help into a fundraising effort that generated much-need financial assistance … and a sense of hope.”

TJ was released from the hospital and returned home in mid-August 2007. However the homecoming was short-lived. TJ was readmitted to the hospital in early January 2008 when he was diagnosed with post-transplant lymphoproliferative disorder -- a post-transplant complication caused by the Epstein Barr virus. TJ was very sick again, and his body started to reject his donor heart. After specialized medications and medical intervention, TJ was able to fight off the disease and the rejection. On March 20, 2008, TJ and his Mommy came home for a second time.

“TJ’s entire story is a miracle. On a seemingly routine trip to the ER to address possible dehydration from the flu, our healthy toddler nearly died as we watched. Since that evening, we have witnessed miracle after miracle with TJ. Thanks to COTA and his amazing transplant team, we are home and our boy is thriving,” said Randy.

TJ is now gaining weight, growing taller and takes only two medications each day. TJ has no developmental delays, no learning issues, no speech problems and is able to eat anything he desires.

A true COTA miracle of the heart!

Please visit www.COTAforTJW.com and leave the Wilson family your own
message of encouragement, or make a donation to help with ongoing
transplant-related expenses.

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Transplants Today

Miniaturized Heart Pumps May Be Effective for Children Awaiting Transplant

According to a report in the Cardiovascular Surgery Supplement of Circulation: Journal of the American Heart Association, nine pediatric patients with severe heart failure were successfully kept alive for an average of 35 days with miniaturized heart assist pumps while awaiting a heart transplant.

“It is not unusual for a child at the top of the transplant candidate list to wait several months before an organ becomes available,” said Sanjiv K. Gandhi, MD, Cardiothoracic Surgeon and Surgical Director of the Heart Failure Program at Saint Louis Children’s Hospital in St. Louis, Missouri.

Researchers implanted biventricular assist devices, known as BiVADs, in seven girls and two boys ranging in age from 12 days to 17 years. All had severe heart failure due to cardiomyopathy or complex congenital heart defects and weighed less than 40 kilograms (88 pounds). One child died from kidney failure before receiving a heart transplant. After 19 months of follow up, the other eight were alive with new hearts.

Children who need heart transplants and are very ill can be placed on external circulatory support machines, but their long-term use is associated with significant risks. Patients also must be immobilized, which impairs physical rehabilitation efforts.

The ventricular assist devices allow for physical rehabilitation that improves the patient’s overall condition and likelihood of successful transplantation, researchers said. In this study, complications such as postoperative bleeding and blood clots blocking a blood vessel occurred infrequently, but there was a high incidence of blood clotting in the pumps.

Small heart pump devices have been available in Europe for many years, but they are
not approved for use in North America. However, the miniaturized Berlin Heart EXCOR® ventricular assist device recently became available in North America on a compassionate
use basis, meaning patients can be approved for the pumps if they have no other
treatment options.

The data emphasize the importance of continued development and refinement of mechanical ventricular assist devices in the pediatric population, researchers said.

Source – www.americanheart.org

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‘Needle in Haystack’ Biomarker Validated in Graft-Vs-Host Disease

The protein, elafin, is the first biomarker of graft-vs-host-disease (GVHD) of the skin ever to be identified and could eventually be a boon to physicians treating GVHD, which is potentially life-threatening.

"Elafin is likely to change the way we treat graft-vs-host-disease of the skin," said James F. Ferrara, MD, Director of the Blood and Marrow Transplant Program of the University of Michigan Medical Center in Ann Arbor, Michigan.

Dr. Ferrara is the senior author of a report of preliminary research findings on elafin published in the January 6, 2010, issue of Science Translational Medicine.

GVHD is a potential complication of allogenic bone marrow transplantation (BMT) in patients with leukemia, lymphoma, or sickle-cell anemia, and is the primary cause of nonrelapse mortality in these patients, Dr. Ferrara said.

About 50% of all allogenic BMT patients develop significant GVHD, he added.

Rashes, which are common in patients after BMT, can signal the onset of acute GVHD. However, rashes are also a sign of numerous other problems, such as allergic reactions to transplant-related antibiotics. Currently, there is no highly accurate and speedy way to confirm that a rash is related to GVHD, explained Dr. Ferrara.

Because of the life-threatening nature of acute GVHD, clinicians typically treat rash in the absence of a confirmed diagnosis. According to Dr. Ferrara and his colleagues, treating all rashes as if they are related to GVHD is problematic. The treatment, which consists of high-dose systemic steroids, weakens the immune system in patients, including those who do not have a rash related to GVHD but who are being treated as if they do. Also, the treatment is not especially well-tailored to the varying severity of the GVHD.

In the new study, in plasma samples taken from 492 patients who received allogenic BMT at the University of Michigan Medical Center, concentrations of elafin were significantly higher at the onset of skin GVHD, correlated with the eventual maximum grade of GVHD, and were associated with a greater risk for death than other known risk factors (hazard ratio, 1.78), reported Dr. Ferrara and his colleagues.

"We got relatively lucky inasmuch as elafin helped diagnose the disease but also predicted the maximum severity," said Dr. Ferrara. A marker (especially one found in a large-scale quantitative proteomic discovery procedure) that addresses both of these clinical issues is rare.

"It's like finding a needle in a haystack," he said.

The researchers chose elafin as the lead candidate biomarker for GVHD, and bypassed many others, because "suitable antibodies were available for high-throughput screening of plasma samples by enzyme-linked immunosorbent assay (ELISA)," according to the team.

Work to Be Done

Acute GVHD after allogeneic BMT can present in the gastrointestinal tract or more commonly as a skin rash, said Dr. Ferrara. The diagnosis of acute skin GVHD is based on clinical criteria (rash, elevated bilirubin concentration, nausea, and diarrhea) and can be confirmed by biopsy.

There are three main problems with skin biopsy, Dr. Ferrara said. "It's a piece of flesh and there's morbidity associated with the procedure. It takes two to three days for results. And it's tricky to evaluate under the microscope," he explained.

Hence, a plasma-based biomarker, the levels of which can be evaluated quantitatively in a blood test, is very desirable. Working with Samir Hanash, MD, PhD, from the Fred Hutchinson Cancer Research Institute in Seattle, Washington, the Michigan investigators used mass spectrometry to screen a large number of proteins in the blood and the skin of BMT patients to search for biomarkers involved in GVHD of the skin.

It is not the first time that mass spectrometry has been used to evaluate GVHD, but it is the first time specific proteins were identified, according to the study authors.

Elafin, which was first identified in the medical literature as an elastase inhibitor over expressed in inflamed epidermis, stood out from the other proteins in the screening. "None of the other candidates had readily available antibodies for the ELISA test," Dr. Ferrara said.

Before a blood test for elafin reaches the clinic, much work needs to be done, explained Dr. Ferrara, adding that the Michigan findings first need to be confirmed at other centers.

Also, he and his team want to evaluate elafin levels before the time of rash in allogenic BMT patients to see if the test "could predict future occurrence." This could allow "pre-emptive therapy" akin to the way cytomegalovirus (CMV) is treated with antivirals, Dr. Ferrara said. The number of virus copies increases before clinical symptoms with CMV. Dr. Ferrara said that elafin and GVHD could possibly work in a similar manner.

Finally, a clinical trial of the test is needed in patients with GVHD to help investigators "tailor therapy," he said.

Source – www.medscape.com

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COTA News

Referring Transplant Families to COTA

The Children's Organ Transplant Association (COTA) wants to say ‘Thank You' for referring transplant families to our organization. In fact, patient/family referrals from the nation's transplant professionals account for the majority of COTA's patients/families.

The most common way for transplant patients and families to learn about COTA is through you, a transplant professional. Here are some helpful tips for referring patients to COTA. Feel free to share this information with transplant families during your pre-transplant evaluations.

• Families who do organized fundraising typically navigate the transplant process with less stress.
• Families should consider the vast array of transplant-related expenses they will have to cover including: loss of income, meals, lodging, travel, prescription medications, etc.
• COTA’s key features include services provided free-of-charge, comprehensive orientation and training for campaign volunteers and COTA's no-cost customizable patient campaign websites.
• COTA’s marketing materials are available free of charge to transplant professionals and to transplant families. Please feel free to request materials by visiting www.cota.org, or by sending an email to

More than 80 patients were successfully transplanted during COTA's 2009 fiscal year that ended June 30, 2009. Since its founding in 1986, COTA has served more than 1,400 transplant patients and their families.

“Giving Hope ... Making Miracles is more than a slogan for the Children's Organ Transplant Association -- it is a guiding vision,” said Rick Lofgren, COTA’s President. “We are extremely thankful for the transplant professionals nationwide who continue to share COTA as a fundraising option with their transplant families and patients.”

We look forward to hearing from you, and we look forward to helping make your families’ road to transplant a little easier. COTA’s staff is just a phone call or an
email away.

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COTA Fast Fact

The Children's Organ Transplant Association (COTA) provides:
Stewardship of the Funds and Non-Profit Status
The Children’s Organ Transplant Association is a national, non-profit organization. COTA is responsible for the oversight of the activities of community fundraising campaigns, and serves as steward of funds raised. Funds donated to COTA are deductible to the fullest extent allowable by law.

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