Adolescents and Young Adults with Sickle Cell Disease

Transcript

00:00 --> 00:03Funding for Yale Cancer Answers is provided
00:03 --> 00:07by Smilow Cancer Hospital and AstraZeneca.
00:07 --> 00:09Welcome to Yale Cancer Answers with
00:09 --> 00:12your host doctor Anees Chagpar.
00:12 --> 00:14Yale Cancer Answers features the latest
00:14 --> 00:16information on cancer care by welcoming
00:16 --> 00:19oncologists and specialists who are on the
00:19 --> 00:21forefront of the battle to fight cancer.
00:21 --> 00:24This week it's a conversation about the care
00:24 --> 00:26of adolescents and young adults with sickle
00:26 --> 00:28cell disease with Doctor Cece Calhoun.
00:28 --> 00:31Dr Calhoun is an assistant professor of
00:31 --> 00:33medicine and hematology and assistant
00:33 --> 00:35professor of Pediatrics in hematology
00:35 --> 00:38oncology at the Yale School of Medicine,
00:38 --> 00:41where Doctor Chagpar is a
00:41 --> 00:43professor of surgical oncology.
00:43 --> 00:45Cece, maybe we could start off by
00:45 --> 00:47you telling us a little bit about
00:47 --> 00:49yourself and what you do.
00:49 --> 00:52I like to call myself a lifespan
00:52 --> 00:54hematologist, and both my clinical
00:54 --> 00:57and research interests center around
00:57 --> 00:59the care of young adults with sickle
00:59 --> 01:01cell disease as they transition
01:01 --> 01:04from pediatric to adult care.
01:04 --> 01:05We know it's a really high
01:05 --> 01:06risk time for them.
01:06 --> 01:08And so all the work that I do both in
01:08 --> 01:10the clinic and in the research setting
01:10 --> 01:12is about making that process better.
01:12 --> 01:15Is sickle cell disease a cancer?
01:15 --> 01:16Tell us more about what
01:16 --> 01:18exactly sickle cell disease is
01:18 --> 01:21and why it's being seen
01:21 --> 01:23by an oncologist?
01:23 --> 01:25That's a great question, so actually sickle
01:25 --> 01:28cell disease is an inherited condition
01:28 --> 01:31of the red blood cells and so many
01:31 --> 01:33people are familiar with anemia
01:33 --> 01:36and conditions of that sort,
01:36 --> 01:37which affect red blood
01:37 --> 01:38cells and hemoglobin.
01:38 --> 01:41And that's what sickle cell disease is
01:41 --> 01:43a condition of, and it's genetic.
01:43 --> 01:45So patients are born with it,
01:45 --> 01:47and what it manifests as is a normal
01:47 --> 01:49red blood cells are kind of squishy.
01:49 --> 01:52I like to think of them as Jelly
01:52 --> 01:53doughnuts because I like food.
01:53 --> 01:55But when you have sickle cell disease
01:55 --> 01:56because of a genetic mutation
01:56 --> 01:58your red blood cells
01:58 --> 02:00are not squishy and malleable,
02:00 --> 02:02they can be really stiff
02:02 --> 02:04and misshapen like a sickle.
02:04 --> 02:06They can be shaped like a sickle or a banana,
02:06 --> 02:08and so if you think of
02:08 --> 02:09your blood cells as pipes,
02:09 --> 02:11imagine if you had your Jelly doughnuts
02:11 --> 02:14kind of going through those pipes,
02:14 --> 02:15bouncing off the walls,
02:15 --> 02:17taking oxygen to where it needs to go,
02:17 --> 02:19and you replace those cells
02:19 --> 02:20with sticky stuff,
02:20 --> 02:22fragile misshapen red blood cells like
02:22 --> 02:24sickle cells that are scratching up
02:24 --> 02:26the red blood vessels sticking together,
02:26 --> 02:28causing blockages, impeding flow,
02:28 --> 02:30and then you can imagine all
02:30 --> 02:32the complications that patients
02:32 --> 02:34with sickle cell face.
02:34 --> 02:38Most saliently or what patients have
02:38 --> 02:41to really deal with is a lot of pain.
02:41 --> 02:42That's the thing that brings
02:42 --> 02:43them to the hospital.
02:43 --> 02:46And acute meaning an unplanned basis,
02:46 --> 02:48but any part of our body where
02:48 --> 02:49there are blood vessels,
02:49 --> 02:50those misshapen
02:50 --> 02:52cells can get clogged up in those
02:52 --> 02:55blood vessels and cause problems.
02:55 --> 02:56It's important for patients
02:56 --> 02:58with sickle cell disease to have
02:58 --> 03:00regular care by an oncologist
03:00 --> 03:02who also understands hematology,
03:02 --> 03:04the blood, to make sure that all their
03:04 --> 03:06organs are in tip top condition
03:06 --> 03:08and that we treat anything before
03:08 --> 03:09there's a problem.
03:09 --> 03:12Now, I would think that if
03:12 --> 03:14you're a pediatric patient and
03:14 --> 03:17this is an inherited condition,
03:17 --> 03:19you might have a sense of whether or
03:19 --> 03:21not you have sickle cell disease
03:21 --> 03:24based on whether your parents did.
03:24 --> 03:26But somebody had to start with the
03:26 --> 03:29genetic mutation to begin with.
03:29 --> 03:30So how many of your patients
03:30 --> 03:32actually know that they have sickle
03:32 --> 03:34cell disease from the time that
03:34 --> 03:36they were born
03:36 --> 03:39and how many of them present to you acutely?
03:41 --> 03:43In the United
03:43 --> 03:46States we have the benefit of the newborn
03:46 --> 03:49screen that all babies born in hospitals,
03:49 --> 03:50when they
03:50 --> 03:52get their heel poked and get that
03:52 --> 03:54little spot of blood that can test
03:54 --> 03:56for a variety of genetic conditions
03:56 --> 03:58and sickle cell disease is
03:58 --> 03:59included in those conditions.
03:59 --> 04:02So if a child has an abnormal newborn screen,
04:02 --> 04:04oftentimes the pediatrician will
04:04 --> 04:07refer them to a hematologist for
04:07 --> 04:09further evaluation and work up.
04:09 --> 04:11And sometimes, even if it's abnormal
04:11 --> 04:14to show sickle cell trait,
04:14 --> 04:16which means that you don't have the disease,
04:16 --> 04:18but you can be a carrier,
04:18 --> 04:19and if your partner has the disease,
04:19 --> 04:21you can have a child with sickle
04:21 --> 04:21cell disease.
04:21 --> 04:23We can figure that out from
04:23 --> 04:24the newborn screen.
04:24 --> 04:27So these days we know pretty early on
04:27 --> 04:29which is critical to the survival
04:29 --> 04:32of our young children or infants and
04:32 --> 04:34toddlers and in other countries
04:34 --> 04:37the newborn screen isn't quite as universal,
04:37 --> 04:39and so sometimes children could present
04:39 --> 04:41with swelling of the hands and feet.
04:41 --> 04:43That's something called dactylitis,
04:43 --> 04:46which is pretty rare these
04:46 --> 04:48days as a presenting sign.
04:48 --> 04:50And then there's some patients with more
04:50 --> 04:53milder forms of sickle cell disease
04:53 --> 04:55that don't know until they're older
04:55 --> 04:56children or young adults,
04:56 --> 04:59but most of the time we get them in
04:59 --> 05:01our catchment when they are young
05:01 --> 05:02because of their newborn screen and
05:02 --> 05:04can really wrap our arms around them
05:04 --> 05:06and give them the care they need.
05:06 --> 05:08Let's suppose you're a newborn baby and
05:08 --> 05:11you had your heel poked and they tell
05:11 --> 05:13you that you have sickle cell disease.
05:13 --> 05:15Well, presumably they don't tell you
05:15 --> 05:18they tell your parents and you get
05:18 --> 05:19referred to a pediatric oncologist.
05:19 --> 05:22If that means that your red blood
05:22 --> 05:24cells are now more
05:24 --> 05:26like bananas than squishy Jelly
05:26 --> 05:29Donuts, what can you do about that?
05:29 --> 05:30I mean, is it reversible?
05:31 --> 05:33At this time the only cure for sickle
05:33 --> 05:35cell disease or way to reverse
05:35 --> 05:37those cells is by replacing your
05:37 --> 05:39bone marrow with another persons,
05:39 --> 05:41but that's pretty rare.
05:41 --> 05:43Later in the show, you get to talk
05:43 --> 05:45a little bit more about therapies
05:45 --> 05:47coming down the pipeline for patients,
05:47 --> 05:49but right now that's the only
05:49 --> 05:51way to reverse.
05:51 --> 05:53However, if you are a little baby
05:53 --> 05:54and your parents find out that
05:54 --> 05:56you have sickle cell disease
05:56 --> 05:58the benefit of coming and talking
05:58 --> 06:00to a pediatric oncologist and
06:00 --> 06:02hematologist who knows about this
06:02 --> 06:04is that you now have a team member,
06:04 --> 06:05somebody on your team that
06:05 --> 06:06can help your baby,
06:07 --> 06:08or you if you're the baby,
06:08 --> 06:09stay healthy and safe.
06:09 --> 06:12And what that looks like as a
06:12 --> 06:15toddler is getting them started on
06:15 --> 06:16penicillin prophylactically or in
06:16 --> 06:18advance before there's any problems
06:18 --> 06:21because we found that as
06:21 --> 06:25recently as the late 70s,
06:25 --> 06:26there was kind of a peak
06:26 --> 06:29in infancy and toddlerhood of death,
06:29 --> 06:31because patients with sickle cell
06:31 --> 06:32were getting really bad infections,
06:32 --> 06:34but we found that if we vaccinate them
06:34 --> 06:36and give them prophylactic penicillin,
06:36 --> 06:38they live well into adulthood.
06:38 --> 06:39The challenge becomes,
06:39 --> 06:41how do we help them when they
06:41 --> 06:42go from infant to adults?
06:42 --> 06:43And that's what I work on in my work.
06:44 --> 06:46So just to back up a little bit when
06:46 --> 06:49you say prophylactic penicillin,
06:49 --> 06:51do you mean like every day for
06:51 --> 06:52the rest of their life?
06:53 --> 06:55So definitely every day for the
06:55 --> 06:58first five years of their life.
06:58 --> 07:00But what it does is it
07:00 --> 07:02protects them against really bad
07:02 --> 07:05infections like pneumococcus you know
07:05 --> 07:07patients with sickle cell disease,
07:07 --> 07:09their spleen doesn't really work
07:09 --> 07:11as well as somebody without sickle
07:11 --> 07:13cell and because of that they are
07:13 --> 07:15susceptible to certain types of
07:15 --> 07:17infections and that penicillin
07:17 --> 07:19every day just like a vitamin helps
07:19 --> 07:21them to stay healthy and safe.
07:21 --> 07:24So why is there this transition then
07:24 --> 07:26from childhood to young adulthood?
07:26 --> 07:28What's the difference in terms of
07:28 --> 07:30the disease and how it's managed
07:30 --> 07:31that requires a specialist like you?
07:33 --> 07:36Well, I think it's a variety of things.
07:36 --> 07:37It's not just the disease,
07:37 --> 07:39but it's becoming a young person
07:39 --> 07:41and learning how to navigate the
07:41 --> 07:43health care system on your own
07:43 --> 07:44and earlier we talked
07:44 --> 07:46about newborns and
07:46 --> 07:48if you were a newborn and found out
07:48 --> 07:51you had sickle cell disease that your parents
07:51 --> 07:53would help you take you to the doctor.
07:53 --> 07:54Manage your care,
07:54 --> 07:56give you that prophylactic penicillin.
07:56 --> 07:57But the beautiful part about being
07:57 --> 07:59a young adult is you can start to
07:59 --> 08:01assume some of that care for yourself,
08:01 --> 08:02so it's pretty
08:02 --> 08:04multi Factorial is a word I always
08:05 --> 08:07like to use and I like to
08:07 --> 08:10think that I was a pretty smart young
08:10 --> 08:12adult like I made some good decisions.
08:12 --> 08:13I'm a doctor now,
08:13 --> 08:15but I still did some foolish things
08:15 --> 08:17as a 16-17 eighteen year old and
08:17 --> 08:19that's without a chronic disease.
08:19 --> 08:21So in sickle cell disease,
08:21 --> 08:23what we can do as lifespan hematologists
08:23 --> 08:25and as health care providers is
08:25 --> 08:27really help our patients as their
08:27 --> 08:29disease complications may become
08:29 --> 08:31a little more severe as they're
08:31 --> 08:33learning to manage themselves.
08:33 --> 08:35As they're learning to navigate a
08:35 --> 08:37pretty complex health care system,
08:37 --> 08:39and as they're just trying to be productive,
08:39 --> 08:40happy young adults.
08:42 --> 08:44What kinds of things do you
08:44 --> 08:46talk about with your patients?
08:46 --> 08:47It sounds like
08:47 --> 08:49after their five years old,
08:49 --> 08:51they're no longer on penicillin,
08:51 --> 08:53but there's still no way to reverse
08:53 --> 08:55the condition, so you're still
08:55 --> 08:58at risk of all of those sticky,
08:58 --> 08:59misshapen blood cells forming
08:59 --> 09:01clots all over your body,
09:01 --> 09:03which presumably can cause
09:03 --> 09:04all kinds of problems.
09:04 --> 09:07Is it just a matter
09:07 --> 09:09of telling your patients what to
09:09 --> 09:12watch for and when to seek help?
09:12 --> 09:15Or are there things that they can do
09:15 --> 09:18to reduce the risk of clots and
09:18 --> 09:21other problems that it can cause?
09:21 --> 09:23Absolutely, so I want to answer
09:23 --> 09:26your question in two parts.
09:26 --> 09:28First, what other parts of the
09:28 --> 09:30body does sickle cell affect?
09:30 --> 09:33How does that show up for
09:33 --> 09:35patients across their lives?
09:35 --> 09:38One of the things that our patients most
09:38 --> 09:41deal with is pain every single day.
09:41 --> 09:43So when those blood vessels get clogged
09:43 --> 09:45up by those sickle cells and those juicy
09:45 --> 09:47Jelly doughnut cells can't get through,
09:47 --> 09:48that means oxygen isn't
09:48 --> 09:50going to where it needs to
09:50 --> 09:51in our bodies.
09:51 --> 09:52And because of that,
09:52 --> 09:54that can result in pretty bad bone pain
09:54 --> 09:56for patients with sickle cell disease,
09:56 --> 09:59and this is the thing that really affects
09:59 --> 10:01their quality of life as young students
10:01 --> 10:03trying to learn and keep up in school.
10:03 --> 10:04If you have to be admitted to
10:04 --> 10:05the hospital several times
10:05 --> 10:08a year you can imagine how
10:08 --> 10:11frustrating that can be as a scholar.
10:11 --> 10:13Other parts of the body that are affected
10:13 --> 10:15by sickle cell disease are numerous.
10:15 --> 10:16Though patients with sickle
10:16 --> 10:18cell disease can have something
10:18 --> 10:19called acute chest syndrome,
10:19 --> 10:21which is a really bad infection of the
10:21 --> 10:23lungs that can be very challenging,
10:23 --> 10:27they can even have strokes as young people,
10:27 --> 10:28which is one of the reasons that
10:28 --> 10:30compelled me as a Med student to
10:30 --> 10:32pursue hematology was seeing a sickle
10:32 --> 10:34cell patient eight years old who had
10:34 --> 10:36a stroke in Pediatrics.
10:36 --> 10:38And in order to kind of get a jump on these things,
10:38 --> 10:40we do several things,
10:40 --> 10:41we do screenings.
10:41 --> 10:43Something called a transcranial Doppler,
10:43 --> 10:45which is basically like an ultrasound
10:45 --> 10:47of your head where you can look at the
10:47 --> 10:48blood vessels and make sure you're
10:48 --> 10:50not at risk for having a stroke.
10:50 --> 10:53We always make sure that our patients
10:53 --> 10:56have their eyes checked because
10:56 --> 10:57sometimes in sickle cell disease
10:57 --> 11:00you can have vision changes and a
11:00 --> 11:02regular follow up with a hematologist
11:02 --> 11:04can help you notice any changes
11:04 --> 11:06before they cause problems.
11:06 --> 11:08One of the biggest things and one
11:08 --> 11:10of the things we know works and
11:10 --> 11:11helps prolong life
11:11 --> 11:13in sickle cell patients is a use of
11:13 --> 11:15a medication called Hydroxyurea.
11:15 --> 11:18Now, some of your listeners may be familiar,
11:18 --> 11:21because sometimes this can be used in
11:21 --> 11:24patients who have certain cancer diagnosis,
11:24 --> 11:26but in sickle cell disease,
11:26 --> 11:28the dose that we use is much lower and the
11:28 --> 11:30way that we use it as a bit different.
11:30 --> 11:32And we know that it kind of helps
11:32 --> 11:35you have more juicy fat
11:35 --> 11:37cells then bananas and so your body
11:37 --> 11:39overall does better in the long term.
11:41 --> 11:43So just to follow up on a few
11:43 --> 11:45things that you just said.
11:45 --> 11:47First off taking that last
11:47 --> 11:49comment about Hydroxyurea making
11:49 --> 11:52You have more fat and juicy like
11:52 --> 11:54blood cells rather than sickling bananas,
11:54 --> 11:57is it true that if you
11:57 --> 11:59have sickle cell disease,
11:59 --> 12:01not all of your blood cells are
12:01 --> 12:03bananas and it is possible to
12:03 --> 12:05increase the number of Jelly doughnut
12:05 --> 12:07blood cells that you have instead
12:07 --> 12:09of bananas?
12:10 --> 12:13Absolutely, and that is up until
12:13 --> 12:16recently, the only FDA
12:16 --> 12:18approved medication that we have had
12:18 --> 12:19for our patients is Hydroxyurea
12:19 --> 12:22to increase the amount of non sickle cells,
12:22 --> 12:25Jelly doughnut cells and ensure that
12:25 --> 12:27you're pain complications are lower
12:27 --> 12:30and that your organs can really get
12:30 --> 12:32the oxygen they need to thrive.
12:32 --> 12:34So an obvious question is why
12:34 --> 12:36not use more and make
12:36 --> 12:39all of your blood cells Jelly Donuts?
12:39 --> 12:40But hold that thought.
12:40 --> 12:43Because first we need to take a short
12:43 --> 12:46break for medical minute. Stay tuned
12:46 --> 12:48to learn more about adolescents and
12:48 --> 12:50young adults with sickle cell disease
12:50 --> 12:52with my guest doctor CeCe Calhoun.
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14:15 --> 14:17Welcome back to Yale Cancer Answers.
14:17 --> 14:19This is Doctor Anees Chagpar and I'm
14:19 --> 14:22joined tonight by my guest Dr. Cece Calhoun.
14:22 --> 14:25We're talking about the care of adolescents
14:25 --> 14:27and young adults with sickle cell
14:27 --> 14:29disease and bright before the break
14:29 --> 14:32CeCe was mentioning that while sickle cell
14:32 --> 14:35disease is completely
14:35 --> 14:37irreversible,
14:37 --> 14:40that actually using a drug called
14:40 --> 14:43Hydroxyurea can help your body to
14:43 --> 14:45create more of these quote juicy
14:45 --> 14:48cells which are normal red blood cells
14:48 --> 14:52and less of these quote banana like
14:52 --> 14:54cells which are the sickle cells.
14:54 --> 14:56So my question to you was.
14:56 --> 15:01before we had the break, is why
15:01 --> 15:03not just give more Hydroxyurea?
15:03 --> 15:06I mean if it helps your body to produce
15:06 --> 15:09more normal cells and less sickle cells,
15:09 --> 15:10wouldn't that be a way to kind
15:10 --> 15:12of reverse it?
15:13 --> 15:16I would love if it could be
15:16 --> 15:17totally reversed by Hydroxyurea
15:17 --> 15:19but we know that when our
15:19 --> 15:21patients are awesome, take their
15:21 --> 15:23medications every day as prescribed,
15:23 --> 15:26there's still an upper limit to
15:26 --> 15:29how many of those juicy fat
15:29 --> 15:31cells they can replace.
15:31 --> 15:33They can produce to replace the banana cells,
15:33 --> 15:36so there's a threshold of how
15:36 --> 15:38effective the drug can be,
15:38 --> 15:39but it can really,
15:39 --> 15:41really help enough to help
15:41 --> 15:43your organs stay healthy.
15:43 --> 15:46So this Hydroxyurea is something
15:46 --> 15:48that you're taking every day?
15:48 --> 15:49For your whole life?
15:49 --> 15:52And the other thing
15:52 --> 15:55that you mentioned before the break
15:55 --> 15:59and I wanted to pick up on as well
15:59 --> 16:02was this concept of pain and the fact
16:02 --> 16:05that many of these patients they present
16:05 --> 16:08with pain and they have pain every day
16:08 --> 16:11which impairs their ability to
16:11 --> 16:14concentrate at school or maybe place
16:14 --> 16:18boards so what do you do about that?
16:18 --> 16:20I mean, are these patients
16:20 --> 16:22treated with daily painkillers?
16:22 --> 16:24Or do you tell them to simply
16:24 --> 16:26wait until they have pain and
16:26 --> 16:28then prescribe pain medication?
16:28 --> 16:31I mean how do they get through their day
16:31 --> 16:33to day life if they're in pain everyday?
16:34 --> 16:37Yeah, so sickle cell patients are warriors
16:37 --> 16:41and you'll often see that described because
16:41 --> 16:44despite having pain of variable severity,
16:44 --> 16:47they managed to live life and be productive.
16:47 --> 16:50That's one of the most awesome things
16:50 --> 16:52about working with sickle cell patients.
16:52 --> 16:55So in terms of pain prevention,
16:55 --> 16:56what can we do?
16:56 --> 16:58Number one Hydroxyurea and get more juicy
16:58 --> 17:00cells around so you have less pain.
17:00 --> 17:03And recently there are a couple
17:03 --> 17:05of medications on the market
17:05 --> 17:07that help with pain prevention.
17:07 --> 17:10Also just keeping yourself well hydrated.
17:10 --> 17:12My patients are so wonderful in
17:12 --> 17:14that they often know their bodies.
17:14 --> 17:15They know their triggers.
17:15 --> 17:18And what situations make their pain worse.
17:18 --> 17:19And what kind of things can
17:19 --> 17:20make their pain better.
17:20 --> 17:22So really being attuned to those things
17:22 --> 17:25in terms of addressing pain acutely
17:25 --> 17:28when it happens and it's not planned,
17:28 --> 17:31we have a couple of things in our toolkit.
17:31 --> 17:33Yes, pain medication is something
17:33 --> 17:36that we give frequently for pain,
17:36 --> 17:38but we can also use red
17:38 --> 17:40blood cell transfusions if we need to.
17:40 --> 17:43If somebody is having pain often,
17:43 --> 17:46but many times we can't predict
17:46 --> 17:47when the pain will come,
17:47 --> 17:49or how severe it will be,
17:49 --> 17:51and so because of that our patients
17:51 --> 17:53have to get care in the ED sometimes
17:53 --> 17:54to get treatment for their pain.
17:55 --> 17:57You mentioned something
17:57 --> 17:59that I found kind of intriguing.
17:59 --> 18:01You said that we have medications
18:01 --> 18:03for pain prevention, like what?
18:04 --> 18:07Hot off the press I know,
18:07 --> 18:09so recently there's been
18:09 --> 18:11an FDA approved medication,
18:11 --> 18:13Adakveo or crizanlizumab
18:15 --> 18:18but I try not to say
18:18 --> 18:20that because crizanlizumab,
18:20 --> 18:23but that can be used to prevent pain
18:23 --> 18:26as an infusion given once monthly.
18:32 --> 18:33And another medication that's recently been
18:33 --> 18:36approved is something called Oxbryta
18:36 --> 18:38and really, what that does is increase
18:38 --> 18:40patients with sickle cell disease,
18:40 --> 18:41their hemoglobin,
18:41 --> 18:44and so the thought is if their
18:44 --> 18:46hemoglobin is better they
18:46 --> 18:49may in turn have less pain,
18:49 --> 18:51but the primary medication that
18:51 --> 18:54is out there for pain
18:54 --> 18:56prevention is Adakveo.
18:57 --> 18:59That sounds like a
18:59 --> 19:00pretty good deal, right?
19:00 --> 19:02If instead of having pain everyday,
19:02 --> 19:05if you had an infusion once a month,
19:05 --> 19:07does that infusion kind of really get
19:07 --> 19:09rid of the chances of having pain?
19:09 --> 19:10Or not really?
19:11 --> 19:14I think that the medication is pretty
19:14 --> 19:17new and patients themselves are
19:17 --> 19:19are individuals,
19:19 --> 19:20and so I've had some patients
19:20 --> 19:21who it's worked great for.
19:21 --> 19:23I've had some patients that we
19:23 --> 19:25just have to try other things.
19:25 --> 19:27I think the wonderful thing
19:27 --> 19:28about being a physician
19:28 --> 19:30scientist and sickle cell,
19:30 --> 19:32or even being a patient right now
19:32 --> 19:33who has sickle cell is that it is
19:33 --> 19:35such a fertile time for discovery.
19:35 --> 19:37In terms of sickle cell disease,
19:37 --> 19:38how to prevent complications
19:38 --> 19:40and how to cure it.
19:40 --> 19:42So you just have to work with
19:42 --> 19:43your hematologist to find
19:43 --> 19:45the right regimen for you.
19:46 --> 19:48So I want to pick up on
19:48 --> 19:50that discovery and some of the
19:50 --> 19:52new advances that are going on
19:52 --> 19:54in terms of sickle cell research.
19:54 --> 19:56But before that I had one other question
19:56 --> 19:58about the complications
19:58 --> 20:01you had mentioned before the break.
20:01 --> 20:04One of the impetuses for you to
20:04 --> 20:05become a pediatric climatologist
20:05 --> 20:08was an 8 year old who had a stroke,
20:08 --> 20:12which just I mean is heartbreaking to me.
20:12 --> 20:14But clearly if you think about these
20:15 --> 20:17sickle cells, it makes sense, right?
20:17 --> 20:18These sickle cells kind of glom
20:18 --> 20:20together and they cut off blood
20:20 --> 20:21supply to a part of your brain
20:21 --> 20:22that's called the stroke.
20:22 --> 20:25Now when we think about
20:25 --> 20:26older patients who
20:26 --> 20:29may be at risk of stroke or who may
20:29 --> 20:31be at risk of heart attack or who
20:31 --> 20:33may be at risk of other clotting,
20:33 --> 20:35whether it's in their lungs or
20:35 --> 20:37in their legs or whatever,
20:37 --> 20:39we often use blood thinners,
20:39 --> 20:41so are sickle cell patients put
20:41 --> 20:44on blood thinners to
20:44 --> 20:45prevent these complications?
20:45 --> 20:46Since we know that they're
20:46 --> 20:48at risk of getting clots.
20:48 --> 20:51So the blockages that occur in sickle
20:51 --> 20:54cell disease are a little bit different
20:54 --> 20:56than your normal blood clot, which is
20:56 --> 20:59caused by a different series of events,
20:59 --> 21:02and so for patients with sickle cell disease,
21:02 --> 21:03though they are at an increased
21:03 --> 21:04risk to have those
21:04 --> 21:07traditionally, what we think of blood clots,
21:07 --> 21:09we don't put them on blood thinners
21:09 --> 21:11to try to prevent complications
21:11 --> 21:12with sickle cell disease.
21:12 --> 21:14We know those blockages can be stuck
21:14 --> 21:16like a clot, or they can be transient,
21:16 --> 21:18they come and go because of the
21:18 --> 21:19cells sticking together.
21:19 --> 21:20It's not like the other proteins in
21:20 --> 21:22your body are swimming over there,
21:22 --> 21:23making a huge clot.
21:23 --> 21:26What we do in our young people
21:26 --> 21:28to maximize stroke prevention
21:28 --> 21:30is we do screenings like the
21:30 --> 21:31Transcranial Doppler I mentioned.
21:31 --> 21:34And if we notice any kind
21:34 --> 21:35of abnormality at all,
21:35 --> 21:37we have a couple of options.
21:37 --> 21:40One we can start them on chronic
21:40 --> 21:43transfusion to decrease the
21:43 --> 21:45amount of sickle cells circulating in their
21:45 --> 21:48blood and give them more normal cells.
21:48 --> 21:50Or if somebody has been
21:50 --> 21:52on transfusions,
21:52 --> 21:54their transcranial dopplers looks fine,
21:54 --> 21:56we can switch them to again Hydroxyurea
21:56 --> 21:59put more Jelly Donuts around,
21:59 --> 22:00have less sickle cells,
22:00 --> 22:02decrease the risk of complications,
22:02 --> 22:04and that's again why it's important
22:04 --> 22:05to connect with your
22:05 --> 22:07friendly hematologist so we
22:07 --> 22:09can help you on that journey.
22:09 --> 22:11Yeah, but presumably you would have
22:11 --> 22:13already been on the Hydroxyurea
22:13 --> 22:16so if that transcranial Doppler finds
22:16 --> 22:19that you're at increased risk
22:19 --> 22:21I guess the transfusion
22:21 --> 22:23is your only alternative,
22:23 --> 22:26but the issue there is if you keep
22:26 --> 22:28getting transfusions on a regular basis,
22:28 --> 22:31doesn't that increase your risk of
22:31 --> 22:33transfusion reactions and potentially
22:33 --> 22:34ultimately developing antibodies such
22:34 --> 22:37that there are fewer and fewer blood
22:37 --> 22:39types that you can actually take?
22:42 --> 22:43Absolutely.
22:43 --> 22:46For patients who have chronic transfusions,
22:46 --> 22:48they're a variety of risks that
22:48 --> 22:50come along with that.
22:50 --> 22:51There's obviously a clear benefit
22:51 --> 22:53in that it keeps you safe
22:53 --> 22:54and protects you against stroke
22:54 --> 22:56and may decrease your pain.
22:56 --> 22:57But you're absolutely right,
22:57 --> 22:58our bodies recognize
22:58 --> 23:00things that aren't foreign,
23:00 --> 23:03and that's why we really work in tandem
23:03 --> 23:05and together with our transfusion
23:05 --> 23:06medicine colleagues to do extended
23:06 --> 23:08typing in patients with sickle cell
23:08 --> 23:10disease to prevent that risk of
23:10 --> 23:12developing antibodies.
23:14 --> 23:16Another big risk is something
23:16 --> 23:17called iron overload,
23:17 --> 23:19where excess iron from the blood deposits
23:19 --> 23:22in different organs like your liver,
23:22 --> 23:23your heart, or your eyes.
23:23 --> 23:26So we measure that regularly and again,
23:26 --> 23:28medicine is so cool because we're
23:28 --> 23:30always ideally moving forward and
23:30 --> 23:33there's also a procedure
23:33 --> 23:35called Erythrocytosis
23:35 --> 23:36which I don't too much mind
23:36 --> 23:37saying five times fast,
23:37 --> 23:40but I like it, which can help
23:40 --> 23:41decrease that risk of iron overload.
23:49 --> 23:50Let's talk a little bit
23:50 --> 23:52about some of the exciting advances
23:52 --> 23:55in terms of sickle cell disease.
23:55 --> 23:56Tell us about what you think are the
23:56 --> 23:58most exciting things that are on the
23:58 --> 24:00forefront that you think are really going
24:00 --> 24:01to make a difference for your patients.
24:03 --> 24:06I think there are a lot of
24:06 --> 24:09medications in the works to address pain
24:09 --> 24:12and complications of sickle cell disease.
24:12 --> 24:15But one of the things I think that is
24:15 --> 24:19most exciting is the idea of a cure
24:19 --> 24:22through gene therapy,
24:22 --> 24:23and that's pretty awesome.
24:23 --> 24:27There's been some media, the
24:29 --> 24:32New York Times has published about it
24:32 --> 24:34and the Washington Post as
24:34 --> 24:37well about how we can use different
24:37 --> 24:38scientific technologies like CRISPR
24:38 --> 24:41technology or use different vectors
24:41 --> 24:43like viral vectors to take somebody's
24:43 --> 24:46stem cells and correct that defect
24:46 --> 24:49in their DNA that caused them to
24:49 --> 24:51be making sickle cells and then
24:51 --> 24:54give it back to them in a safe way,
24:54 --> 24:56and then when those new and
24:56 --> 24:57improved cells from their bodies
24:57 --> 24:58replicate
24:58 --> 25:00they are no longer affected
25:00 --> 25:02by sickle cell disease.
25:02 --> 25:04They may still make some sickle cells,
25:04 --> 25:06but will effectively be cured or
25:06 --> 25:08be like somebody who just has the
25:08 --> 25:10trait and that's one of the things
25:10 --> 25:11I think that's most exciting.
25:11 --> 25:13The possibility of a cure
25:13 --> 25:15in our future.
25:18 --> 25:21And is that 10-15, 30-50 years from now?
25:21 --> 25:23No, the time is totally now,
25:23 --> 25:25so there are clinical,
25:25 --> 25:27active clinical trials going on
25:27 --> 25:29to better understand the safety
25:29 --> 25:32and efficacy of this process
25:32 --> 25:34for patients and so
25:34 --> 25:36that's happening now.
25:36 --> 25:38Wow, that's
25:38 --> 25:41super exciting. What else is going on?
25:42 --> 25:45So I think the other main things are
25:45 --> 25:47the development of oral medications
25:47 --> 25:49to improve pain and to decrease
25:49 --> 25:51complications from sickle cell disease.
25:51 --> 25:53That one medication,
25:53 --> 25:56Adakveo, the way that it works,
25:56 --> 25:57it's something called
25:57 --> 25:58a B selection inhibitor.
25:58 --> 26:00And so they're more medications
26:00 --> 26:03coming around that look at that.
26:03 --> 26:06And there's some additional
26:06 --> 26:09oral medications coming that target
26:09 --> 26:12different mechanisms and other blood
26:12 --> 26:14problems like thalassemia and
26:14 --> 26:16they want to see if those medications
26:16 --> 26:18can work well in
26:18 --> 26:20patients with sickle cell disease.
26:20 --> 26:22So I think that fact that we are shining
26:22 --> 26:24a light on this community of people
26:24 --> 26:27with sickle cell disease and that we as
26:27 --> 26:28a scientific community have committed
26:28 --> 26:31to making their quality of life better,
26:31 --> 26:33that's the thing that's most exciting to me,
26:33 --> 26:36because oftentimes I think my
26:36 --> 26:38patients feel unseen and unheard,
26:38 --> 26:42and so it's great to see so many people,
26:42 --> 26:44brilliant people standing up for them
26:44 --> 26:45and helping to make their lives better.
26:46 --> 26:46That's awesome.
26:46 --> 26:50I guess the last question that I have is
26:50 --> 26:52really with regards to clinical trials.
26:52 --> 26:55I mean, it sounds like there's so
26:55 --> 26:58many great things on the horizon.
26:58 --> 27:01Do you find that young people
27:01 --> 27:04adolescents are interested in clinical
27:04 --> 27:07trials and willing to participate?
27:07 --> 27:10Are there barriers to participation?
27:10 --> 27:11How has that been going along?
27:12 --> 27:14Yeah, so anybody who has lived
27:14 --> 27:16with sickle cell or chronic pain,
27:16 --> 27:18I think is enthusiastic about
27:18 --> 27:21finding a way to have a better
27:21 --> 27:23life and to come have a better
27:23 --> 27:25quality of life and to find a cure.
27:25 --> 27:27When it comes to clinical trials,
27:28 --> 27:31there's a careful balance
27:31 --> 27:33between understanding clinical
27:33 --> 27:36studies and not wanting to feel like
27:36 --> 27:37an experiment and understanding
27:37 --> 27:39how the medical system can wrap
27:39 --> 27:41around you to keep you safe.
27:41 --> 27:43As we understand more about
27:43 --> 27:46how to help you have a cure.
27:46 --> 27:49And so when I think about
27:49 --> 27:50my young people,
27:50 --> 27:52are they interested in clinical trials?
27:52 --> 27:55I think that they have a lot of excellent
27:55 --> 27:57questions about the benefits and
27:57 --> 27:59risks of participating in clinical trials.
27:59 --> 28:01But many of them ultimately,
28:01 --> 28:04when we sit and talk and take the time,
28:04 --> 28:05they understand that it is their
28:05 --> 28:07contribution to not only their health,
28:07 --> 28:09but the community of sickle cell patients.
28:09 --> 28:11And that's the beauty of having providers
28:11 --> 28:13that have known you through the lifespan.
28:13 --> 28:14You have a relationship.
28:14 --> 28:17They know that I care for them.
28:17 --> 28:18They can trust me.
28:18 --> 28:20And so when I offer them this option,
28:23 --> 28:24then there's a little bit
28:24 --> 28:25more willingness to enroll.
28:26 --> 28:29Dr Cece Calhoun is an assistant
28:29 --> 28:31professor of medicine in hematology
28:31 --> 28:32and assistant professor of
28:32 --> 28:34Pediatrics in hematology,
28:34 --> 28:37oncology at the Yale School of Medicine.
28:37 --> 28:38If you have questions,
28:38 --> 28:39the address is canceranswers@yale.edu
28:39 --> 28:42and past editions of
28:42 --> 28:45the program are available in audio and
28:45 --> 28:48written form at yalecancercenter.org.
28:48 --> 28:50We hope you'll join us next week to
28:50 --> 28:52learn more about the fight against
28:52 --> 28:53cancer here on Connecticut Public
28:53 --> 28:55radio funding for Yale Cancer
28:55 --> 28:57Answers is provided by Smilow
28:57 --> 28:59Cancer Hospital and Astra Zeneca.

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August 29, 2021

Yale Cancer Center

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