PKD Life - Summer 2020 - 11

DOCTOR Q&A

Children and PKD

Pediatric nephrologist Laurel K. Willig, M.D.,
shares insights and advice. With Debra Gordon

K. Willig, M.D., is a pediatric nephrologist
L aurel
who works in the Center for Pediatric Genomic

Medicine at Children's Mercy in Kansas City, Missouri.
We talked with her about the special issues children
with PKD and their families face.

What are the biggest
issues that arise in
treating children with PKD?
Children with autosomal
recessive polycystic kidney
disease (ARPKD) often have
significant kidney disease,
so they require a lot of
medical management. That
includes blood pressure
management, nutrition,
bone health, and the poor
growth that may accompany
the disease. They may also
have significant liver issues,
which can complicate the
overall management.
Compared with children
who have ARPKD, children
with autosomal dominant
polycystic kidney disease
(ADPKD) often have fewer
medical issues, but some
more common issues seen
in children with ADPKD
include high blood pressure and protein in the
urine. Patients with really
severe disease might benefit
from tolvaptan, but it's only

Q

approved for adults, so it's
pretty much impossible to
get for children right now.
It's very expensive, insurance won't pay for it, and
the kids don't qualify for
drug coupons.
Another issue that's
not very well addressed is
chronic pain. Quite a few of
my teenage patients have
chronic abdominal and
back pain that is associated
with their PKD, but it's often
overlooked in management
strategies for children, which
is a big challenge for me.
What about
screening for PKD
in children?
This is a challenge I face
and struggle with on a daily
basis in children with a family history of PKD who don't
yet exhibit symptoms of
the disease. There are different ethical and financial
complexities to screening,
since it labels children with

Q

a preexisting condition for
which they don't have symptoms, and we don't have any
approved therapies for children. That may become less
of a challenge if we do get
therapies that can prevent
progression in pediatric
patients; then it will make
sense to screen.
What are the key
things that parents
of kids with PKD can do to
help their kids stay as
healthy as possible?
In children under 10,
parents are the primary
caregiver, administering
medications and ensuring the child eats what
they should and stays well
hydrated. It helps to build
those habits early, when
they're less rebellious.
Starting around age 12,
depending on the child's
developmental age, we
encourage them to move
toward independence so
they can care for themselves
and any health problems.
But many patients don't
come until they are older
because they haven't been
diagnosed early. Sometimes
it's hard to get them motivated if they don't have a
lot of signs or symptoms of

Q

the disease. Also, at that age
they are very concrete thinkers; they don't think about
the future. So in these situations, we depend on parents
to help a lot. One thing that
helps is that unlike other diseases, children usually have
relatives with PKD, so they've
seen what the long-term
effects can be. That can be
extra motivating. But it is
a challenge.
What does the future
of PKD treatment
for kids look like?
Studies of tolvaptan and other
drugs in children are underway. Tolvaptan is likely to be
approved soonest because
it's been studied the longest.
That's super exciting.
The pie-in-the-sky treatment is gene therapy. I think
we're a ways off from that
for PKD, but we're learning
more about the genetics of
the disease, and that's the
first step to moving toward
genomic therapies.
I'm also hopeful that we'll
have better options in the
area of transplant medicine
and tissue regeneration. It's
an exciting time in kidney
medicine, and the next 10
years will really transform
what we do.

Q

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11
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PKD Life - Summer 2020

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