Vim & Vigor - Winter 2019 - UVA - 51

of us were with him, we were very careful about washing our hands, and we
kept our homes sterilized and cleaned
all the time."

Bone Marrow Transplant
Offers Promising
Outcome

When Dannen was 2 years old, UVA
pediatric immunologist Monica
Lawrence, MD, referred him to
Children's National Medical Center
(CNMC) in Washington, D.C., for a
bone marrow transplant, a procedure
not currently offered at UVA for patients
with immunodeficiencies. This procedure involves the infusion of healthy
stem cells (the blood-producing cells)
into the body after the destruction of
unhealthy ones via chemotherapy. The
new cells make healthy blood cells and
regenerate the immune system.
"Dannen was a good candidate for
transplant because he was diagnosed
early and maintained infection precautions, antibiotics and IVIG to keep
him as healthy as possible going into
transplant," Dr. Lawrence says. "He
also had a good [unrelated] donor
match identified."
Dannen's transplant procedure
took place in November 2017, and he
remained at CNMC and later at the
nearby Ronald McDonald House until
late April 2018. Dannen is now considered cured, though he occasionally
needs IVIG infusions for low antibody levels.
This summer, Dannen received his
childhood vaccines, and in the fall, he
started preschool, a triumph his family
couldn't have imagined two years ago.
"As scary as this experience has been
for us, I can't say enough about the
doctors, nurses and caregivers at UVA.
They really treated us like family," Sisler
says. "Dannen has received phenomenal
care. We were protective of Dannen for
so long, but now he seems like a typical
4-year-old boy. We feel so blessed." ■

SIGNS OF PRIMARY
IMMUNODEFICIENCY
DISORDER
Primary immunodeficiency disorders can affect almost every organ in the
body, causing recurrent infections and other complications that result in lots of
missed days at school or work and a poor quality of life. Potential warning signs
of PI include:
Frequent ear, sinus or respiratory infections
Prolonged or difficult-to-treat infections
Infections with organisms that usually are harmless
Invasive infections involving internal organs, bones, bloodstream
or central nervous system
Recurrent fungal infections
Family history of primary immunodeficiency disorders
Chronic diarrhea with weight loss
Failure to thrive in childhood

SUPERB CARE FOR PRIMARY IMMUNODEFICIENCIES

UVA recently was designated a Jeffrey Modell Diagnostic and Research Center
for Primary Immunodeficiencies by the Jeffrey Modell Foundation, established
by Fred and Vicki Modell after the loss of their 15-year-old son from primary
immunodeficiency (PI) complications. UVA's dedicated pediatric clinic for
PI patients offers access to a network of specialists in areas such as rheumatology, gastroenterology, dermatology and pulmonology. The clinic's dedicated
nurse coordinator helps patients' families navigate appointments, lab testing
and at-home treatments. There are only 350 such centers worldwide. Learn
more at uvahealth.com/pidd.

Newborn Screenings Save Lives
All newborns in Virginia are screened for severe combined immunodeficiency
(SCID), the most serious primary immunodeficiency disorder, also known as
the "bubble boy disease." It can be fatal if not diagnosed and treated early.
The blood spot test obtained by a needle prick on the baby's heel during
the first 24 to 72 hours of life looks for T-cell receptor excision circles. These
markers signal the immune system's production of T cells, which are critical
for fighting infections.
SCID is just one form of PI, so having a normal newborn screen does not
rule out other immune system disorders. Families can opt out of the screening
if desired.
"Early diagnosis allows for appropriate treatment, which can be curative in
many cases and lead to excellent outcomes if started early," says UVA pediatric
immunologist Monica Lawrence, MD. "Without treatment, children with SCID
die in infancy or early childhood."

WINTE R 2019

http://www.uvahealth.com/pidd

Vim & Vigor - Winter 2019 - UVA

Table of Contents for the Digital Edition of Vim & Vigor - Winter 2019 - UVA