Transplant for Pediatric Patients with Sickle Cell Disease

Transcript

00:00 --> 00:03Funding for Yale Cancer Answers is
00:03 --> 00:06provided by Smilow Cancer Hospital.
00:06 --> 00:07Welcome to Yale Cancer Answers with
00:07 --> 00:09your host, doctor Anees Chagpar.
00:09 --> 00:12Yale Cancer Answers features the
00:12 --> 00:13latest information on cancer
00:13 --> 00:15care by welcoming oncologists and
00:15 --> 00:17specialists who are on the forefront
00:17 --> 00:19of the battle to fight cancer.
00:19 --> 00:22This week, it's a conversation about bone
00:22 --> 00:24marrow transplant with Doctor Niketa Shah.
00:24 --> 00:26Doctor Shah is director of the pediatric
00:26 --> 00:28bone Marrow Transplant program at
00:28 --> 00:30the Yale School of Medicine,
00:30 --> 00:31where Doctor Chgppar is a
00:31 --> 00:33professor of surgical oncology.
00:34 --> 00:36So Niketa, maybe we can start off
00:36 --> 00:38by you telling us a little bit more
00:38 --> 00:40about yourself and what it is you do.
00:41 --> 00:44So I'm a pediatric bone marrow transplant
00:44 --> 00:47physician and I've been here at Yale since
00:47 --> 00:512016 and we do many transplant here.
00:51 --> 00:53We are the only transplant
00:53 --> 00:55program in the state of Connecticut
00:55 --> 00:57and since 2019, we also do CAR
00:58 --> 01:00T-bone marrow transplant which is something
01:00 --> 01:03that people may have heard a
01:03 --> 01:05little bit about but may not be
01:05 --> 01:07really familiar with what it is.
01:07 --> 01:09So can you tell us a little bit
01:09 --> 01:11more about what exactly that
01:11 --> 01:14is and who needs a bone
01:14 --> 01:16marrow transplant and when?
01:17 --> 01:20So bone marrow transplant is a process
01:20 --> 01:23where we remove avpatients own bone marrow and
01:23 --> 01:27replace it with the new bone marrow from
01:27 --> 01:30either the patients own previously collected
01:30 --> 01:33bone marrow or from a different donor.
01:33 --> 01:36So we will focus today on mainly
01:36 --> 01:39allogeneic stem cell transplant or
01:39 --> 01:41allogenic bone marrow transplant
01:41 --> 01:44where we replace a patients own bone marrow
01:44 --> 01:48with a healthy donor.
01:48 --> 01:51Now bone marrow transplant is required
01:51 --> 01:54for those patients whose bone marrow is
01:54 --> 01:57not working properly either from their
01:57 --> 01:59birth or they acquired some disease
01:59 --> 02:02like leukemia down the road which
02:02 --> 02:05is not curable or not treatable with
02:05 --> 02:09the regular chemotherapy and in those
02:09 --> 02:11conditions we replace the diseased
02:12 --> 02:14marrow with the healthy bone marrow
02:14 --> 02:17with the goal that once this healthy
02:17 --> 02:19bone marrow is established in patients
02:19 --> 02:21bone marrow and starts working,
02:21 --> 02:24they produce normal healthy
02:24 --> 02:27blood cells and thus cure the
02:27 --> 02:29patients from their underlying disease.
02:30 --> 02:32So let's dive a little
02:32 --> 02:33bit deeper into that.
02:33 --> 02:36So you mentioned that bone marrow
02:36 --> 02:38transplant is really there for people
02:38 --> 02:40whose bone marrow isn't working properly,
02:40 --> 02:43either because of a condition from birth
02:43 --> 02:46or because of an acquired condition,
02:46 --> 02:47kind of like leukemia.
02:47 --> 02:50Can you talk a little bit about what
02:50 --> 02:52some of the conditions from birth might
02:52 --> 02:54be that people might have bone marrows
02:54 --> 02:57that aren't working properly and might
02:57 --> 02:59require a bone marrow transplant?
03:00 --> 03:02Yes, definitely. So there are
03:02 --> 03:05many patients whose
03:05 --> 03:08bone marrow is not working from
03:08 --> 03:11birth which develops down the road
03:11 --> 03:14either completely aplastic or their
03:14 --> 03:16bone marrow is not at all working.
03:16 --> 03:18So there's some of the elements
03:18 --> 03:21of the bone marrow is not working.
03:21 --> 03:24Now I think most of us know there
03:24 --> 03:26are three types of blood cells,
03:26 --> 03:27one is white blood cells,
03:27 --> 03:29other is red blood cells and
03:29 --> 03:31the third one is platelets.
03:31 --> 03:34So if the patients bone marrow is not
03:34 --> 03:37working from birth they might have
03:37 --> 03:39complete non functioning bone marrow.
03:39 --> 03:42So they don't have any of these three
03:42 --> 03:45types of different types of blood cells.
03:45 --> 03:47Or they may just have their red blood
03:47 --> 03:50cell is not working or white blood
03:50 --> 03:53cells is not working or platelet is not
03:53 --> 03:55working so they either have anemia or
03:55 --> 03:57their white blood cells is not there
03:57 --> 04:00so they have more risk of infection
04:00 --> 04:02or their platelet is not working.
04:02 --> 04:05So they may have a bleeding disorder.
04:05 --> 04:09So one of the most common among this is
04:09 --> 04:11the hemoglobinopathies where
04:11 --> 04:14within their red blood cells
04:14 --> 04:17their hemoglobin is not properly developed.
04:17 --> 04:20And so those patients are called
04:20 --> 04:22having the hemoglobinopathies
04:22 --> 04:25where their hemoglobin is
04:25 --> 04:26not properly developed.
04:26 --> 04:29So they have less red blood cells
04:29 --> 04:32and in turn they have also or their
04:32 --> 04:34red blood cells are destroyed very
04:34 --> 04:37quickly and in turn they have more
04:37 --> 04:41anemia and also other related disorders.
04:44 --> 04:47Maybe give us an example of what some
04:47 --> 04:49of these conditions might be when we
04:49 --> 04:52talk about people not having red blood
04:52 --> 04:54cells or white blood cells or platelets?
04:54 --> 04:56For many people who may be listening,
04:56 --> 04:59that may seem really rather odd because many
04:59 --> 05:03of us are used to having these blood cells.
05:03 --> 05:04We often take our blood cells
05:04 --> 05:06really rather for granted,
05:06 --> 05:09knowing that they're there and working.
05:09 --> 05:12So what conditions might
05:12 --> 05:16lead to these hemoglobinopathies or other
05:16 --> 05:19conditions that the bone marrow is not
05:19 --> 05:22working and how common are they?
05:23 --> 05:25So one of the most common I will
05:25 --> 05:27mention here in the hemoglobinopathy
05:27 --> 05:30where the fact is in their red
05:30 --> 05:32blood cells hemoglobin and that one
05:32 --> 05:35is sickle cell anemia which is most
05:35 --> 05:38common hemoglobinopathy.
05:38 --> 05:40The other hemoglobinopathy is
05:40 --> 05:44thalassemia also then if we go through
05:44 --> 05:47the other red blood cells like
05:47 --> 05:50white blood cells or the platelets.
05:50 --> 05:52These are less common where
05:52 --> 05:54there are congenital neutropenia.
05:54 --> 05:55Or congenital thrombocytopenia,
05:55 --> 05:59or if all cells are not working well.
05:59 --> 06:02There are few common bone marrow failure
06:02 --> 06:04conditions which are present since birth.
06:04 --> 06:07These are called Fanconi anemia
06:07 --> 06:09or dyskeratosis congenita.
06:09 --> 06:11But all these disorders are
06:11 --> 06:14far less common than the most
06:14 --> 06:16common hemoglobinopathy we see,
06:16 --> 06:18which is sickle cell anemia.
06:18 --> 06:21It is almost every year there
06:21 --> 06:24are 300,000 kids
06:24 --> 06:26with sickle cell anemia bone in
06:26 --> 06:29the world, and in the United States,
06:29 --> 06:33one in every 360 African American or one
06:33 --> 06:37in every 16,000 Hispanic patients
06:37 --> 06:40have this hemoglobinopathy,
06:40 --> 06:42which is called sickle cell anemia.
06:42 --> 06:44And in this hemoglobinopathy that
06:44 --> 06:47hemoglobin that is only one of the
06:47 --> 06:50building block of their hemoglobin
06:50 --> 06:53gene is replaced by the different block
06:53 --> 06:55and that caused their sickle cell.
06:55 --> 06:58All of the things that cause them to
06:58 --> 07:01have the sickle cell hemoglobinopathy,
07:01 --> 07:03which is different than what we
07:03 --> 07:06all have as a normal hemoglobin
07:06 --> 07:10in our red blood cells.
07:10 --> 07:13So if we have a normal hemoglobin our red
07:13 --> 07:15blood cell is like a doughnut or soft,
07:15 --> 07:18spongy, while if they have a sickle cell
07:20 --> 07:22in their blood cells,
07:22 --> 07:25their red blood cell looks like sickle.
07:25 --> 07:28Which is as the name
07:28 --> 07:30suggests sickle and it is not soft,
07:30 --> 07:32it's hard, rigid.
07:32 --> 07:35So the red blood cells break
07:35 --> 07:37down easily and that is the main
07:37 --> 07:40cause of sickle cell disease which
07:40 --> 07:43develops in sickle cell anemia patients.
07:45 --> 07:49And so for all of these kids who
07:49 --> 07:51have sickle cell anemia where they
07:51 --> 07:54have these red blood cells that
07:54 --> 07:57form these Crescent like sickles
07:57 --> 07:59that are hard instead of being
07:59 --> 08:01spongy doughnuts like the rest of
08:01 --> 08:03us who have normal red blood cells,
08:05 --> 08:08how does that really affect them
08:08 --> 08:11in terms of their every day health?
08:11 --> 08:15I mean, can you explain to our listeners
08:15 --> 08:17how the shape and consistency,
08:17 --> 08:19for lack of a better word,
08:19 --> 08:22of these red blood cells impacts
08:22 --> 08:23a patients day-to-day life?
08:23 --> 08:25I mean, who really cares about
08:25 --> 08:27the size and shape and consistency
08:27 --> 08:30of a red cell?
08:30 --> 08:32I totally agree that the general population
08:32 --> 08:36might not know how the sickle cell anemia
08:36 --> 08:38effects in each and every person
08:38 --> 08:41who has the sickle cell anemia.
08:41 --> 08:44So as I mentioned earlier that the
08:44 --> 08:47sickle cell anemia patients have
08:47 --> 08:50red blood cells which are sickle cell
08:50 --> 08:53which are rigid and easily breaking down
08:53 --> 08:55and they because of that
08:55 --> 08:58presence in their red blood cells or the
08:58 --> 09:01red blood cells see being sickle cell.
09:01 --> 09:03Their blood can't reach each and
09:03 --> 09:06every organ like the tiny fingers
09:06 --> 09:09or the where the blood has to reach
09:09 --> 09:11through the small blood vessels.
09:11 --> 09:14So particularly when they are experiencing
09:14 --> 09:17some cold weather or they have infection,
09:17 --> 09:19their sickle cell can't reach where
09:19 --> 09:22it needs to go and they break down.
09:22 --> 09:25And when your red blood cells
09:25 --> 09:27are not reaching those area,s
09:27 --> 09:29like even the bone or the kidneys
09:29 --> 09:31or the lungs,
09:31 --> 09:33then you develop all the complications
09:33 --> 09:35and the most common effect initially
09:35 --> 09:38in their lifespan we see is the
09:38 --> 09:41pain crisis because their blood
09:41 --> 09:44is not reaching those required
09:44 --> 09:47areas where it needs to go and they
09:47 --> 09:49experience severe pain crisis.
09:49 --> 09:52They also have an increased rate of
09:52 --> 09:54infection because their immunity also
09:54 --> 09:58down the road goes down and then down the
09:58 --> 10:01road if this continues they may have many
10:01 --> 10:03lung complication called the
10:03 --> 10:05acute Chest syndrome where they
10:05 --> 10:07develop pneumonia like symptoms and
10:07 --> 10:09they may need hospitalization and
10:09 --> 10:12we need to bring down their sickle
10:12 --> 10:15cell number by giving the transfer
10:15 --> 10:16regular blood transfusion.
10:16 --> 10:20And if this issue continues most of the
10:20 --> 10:23other organs also gets affected like kidneys,
10:23 --> 10:24lungs,
10:24 --> 10:27eyes, even risk of stroke
10:28 --> 10:31because the blood flow to the brain also
10:31 --> 10:33is affected and because of this
10:33 --> 10:36chronic changes in the lung they may
10:36 --> 10:38also have pulmonary hypertension.
10:38 --> 10:40They have the eye changes.
10:40 --> 10:43They also have the spleen also down
10:43 --> 10:45the road stops working well.
10:45 --> 10:48So they have also increased
10:48 --> 10:51risk of infection so that
10:51 --> 10:51acute complication,
10:51 --> 10:55if it continues to develop down the road,
10:55 --> 10:57they develop into the chronic
10:57 --> 10:59morbidity and it affects their
10:59 --> 11:02lifestyle and their quality of life.
11:02 --> 11:05And down the road their lifespan also is
11:05 --> 11:07reduced compared to the normal population.
11:07 --> 11:10And so the size and shape of these blood
11:10 --> 11:13cells really does make a difference in
11:13 --> 11:15terms of where they can go and that
11:15 --> 11:18in turn has an impact on
11:18 --> 11:21the function of various organs.
11:21 --> 11:24Now you mentioned that one of the ways to
11:24 --> 11:28get around this is with blood transfusions.
11:28 --> 11:32So if these patients get blood transfusions
11:32 --> 11:35and are transfused with blood cells
11:35 --> 11:38that are donut shaped and squishy,
11:38 --> 11:41and potentially those blood cells can
11:41 --> 11:45get to places, how about that option.
11:45 --> 11:48I mean does every patient with
11:48 --> 11:52sickle cell anemia need a bone marrow
11:52 --> 11:54transplant or are transfusions good
11:54 --> 11:56enough for some patients?
11:57 --> 11:59Transfusion is definitely good
11:59 --> 12:03enough for the acute condition if
12:03 --> 12:05they develop sickle cell disease.
12:05 --> 12:08However some of the patients
12:08 --> 12:11who are very high risk of developing into
12:11 --> 12:14the chronic conditions like those patients
12:14 --> 12:17who have experienced early stroke or
12:17 --> 12:20their brain blood vessels already have
12:20 --> 12:24started developing the changes because of
12:24 --> 12:27the sickle cell anemia in those patients,
12:27 --> 12:29if you give the chronic blood
12:29 --> 12:30transfusion like every month,
12:30 --> 12:34you can give them the normal healthy
12:34 --> 12:36hemoglobin every month which will
12:36 --> 12:39dilute their underlying sickle cell
12:39 --> 12:44hemoglobin numbers and you can reduce the
12:44 --> 12:45complication.
12:45 --> 12:48However, blood transfusion in a chronic
12:48 --> 12:51stage also has many complications,
12:51 --> 12:53so that you can't continue for lifelong
12:53 --> 12:56because you will be exposed to many,
12:56 --> 12:58many blood transfusion
12:58 --> 13:00products and each blood transfusion
13:00 --> 13:03also carries with it the increased iron
13:03 --> 13:06which comes from our red blood cells.
13:06 --> 13:08So those patients have to go through
13:08 --> 13:10those complications down the road.
13:10 --> 13:12So it is better for them if they
13:12 --> 13:14have an available donor
13:14 --> 13:16for blood bone marrow transplant,
13:16 --> 13:18which is the only curative option right
13:18 --> 13:21now for sickle cell anemia patients.
13:22 --> 13:23Well, we're going to take a
13:23 --> 13:25short break for a medical minute,
13:25 --> 13:26but on the other side of the break,
13:26 --> 13:29we'll learn more about bone marrow
13:29 --> 13:31transplantation and how exactly
13:31 --> 13:34we can help in in the care of
13:34 --> 13:35pediatric patients with my guest,
13:35 --> 13:37doctor Niketa Shah.
13:37 --> 13:39Funding for Yale Cancer Answers
13:39 --> 13:41comes from Smilow Cancer Hospital,
13:41 --> 13:44where you can view videos from their
13:44 --> 13:46survivorship team by searching for the
13:46 --> 13:48smilow survivorship playlist on YouTube.
13:50 --> 13:52Genetic testing can be useful for
13:52 --> 13:54people with certain types of cancer
13:54 --> 13:56that seem to run in their families.
13:56 --> 13:58Genetic counseling is a process that
13:58 --> 14:00includes collecting a detailed personal
14:00 --> 14:03and family history or risk assessment,
14:03 --> 14:06and a discussion of genetic testing options.
14:06 --> 14:08Only about 5 to 10% of all cancers
14:08 --> 14:10are inherited and genetic testing
14:10 --> 14:12is not recommended for everyone.
14:12 --> 14:14Individuals who have a personal
14:14 --> 14:17and or family history that includes
14:17 --> 14:19cancer at unusually early ages,
14:19 --> 14:20multiple relatives
14:20 --> 14:22on the same side of the family
14:22 --> 14:24with the same cancer,
14:24 --> 14:26more than one diagnosis of cancer
14:26 --> 14:28in the same individual rare cancers
14:28 --> 14:31or family history of a known altered
14:31 --> 14:33cancer predisposing gene could be
14:33 --> 14:35candidates for genetic testing.
14:35 --> 14:37Resources for genetic counseling and
14:37 --> 14:39testing are available at federally
14:39 --> 14:42designated comprehensive cancer centers,
14:42 --> 14:44such as Yale Cancer Center and
14:44 --> 14:45Smilow Cancer Hospital.
14:45 --> 14:48More information is available
14:48 --> 14:49at yalecancercenter.org. You're
14:49 --> 14:51listening to Connecticut
14:51 --> 14:51Public Radio.
14:52 --> 14:54Welcome back to Yale Cancer Answers.
14:54 --> 14:56This is doctor Anees Chagpar
14:56 --> 14:58and I'm joined tonight by my guest,
14:58 --> 14:59doctor Niketa Shah.
14:59 --> 15:01We're talking about the care
15:01 --> 15:02of pediatric patients in bone
15:02 --> 15:04marrow transplant and
15:04 --> 15:06right before the break,
15:06 --> 15:08we were talking about patients
15:08 --> 15:10with sickle cell anemia.
15:10 --> 15:13And how many of these patients will
15:13 --> 15:15need recurrent blood transfusions
15:15 --> 15:19to try to get over some of these
15:19 --> 15:21acute crises that they have due
15:21 --> 15:24to blood vessels that are sickled
15:24 --> 15:25in shape.
15:25 --> 15:27And you mentioned that while
15:27 --> 15:30blood transfusions are great and
15:30 --> 15:32necessary in the acute setting,
15:32 --> 15:34doing blood transfusions in
15:34 --> 15:37a chronic way has a number
15:37 --> 15:38of potential complications,
15:38 --> 15:40everything from infections to
15:40 --> 15:44iron overload to a number
15:44 --> 15:47of other other issues.
15:47 --> 15:49And one of the things you mentioned
15:49 --> 15:51was that bone marrow transplant is
15:51 --> 15:54at the moment the only curative
15:54 --> 15:56option. Tll us a little bit
15:56 --> 15:57more about how that works.
15:57 --> 16:00Not all patients with sickle cell
16:00 --> 16:03disease should be taken for bone
16:03 --> 16:05marrow transplant right away if we
16:05 --> 16:08know there are many other supportive
16:08 --> 16:11care therapies available for sickle cell
16:11 --> 16:13anemia or sickle cell disease patients
16:13 --> 16:16apart from the blood transfusion.
16:16 --> 16:1950 years back prophylaxis penicillin
16:19 --> 16:22also helped them reduce
16:22 --> 16:24the infection related disease
16:24 --> 16:28which was initially started by one
16:28 --> 16:31of our own mentors here at Yale,
16:31 --> 16:34Doctor Howard Pearson who suggested
16:34 --> 16:36that preventing the pneumococcal
16:36 --> 16:38infection by giving the penicillin
16:38 --> 16:41prophylaxis you can reduce the infection
16:41 --> 16:44related death in first decade of life
16:44 --> 16:46for sickle cell disease later on.
16:46 --> 16:49A vaccine was also added and
16:49 --> 16:51there are also some medicines.
16:51 --> 16:53This approach helped them
16:53 --> 16:55reduce the complications related
16:55 --> 16:58to the sickle cell disease.
16:58 --> 17:01However, it's not curative,
17:01 --> 17:05so those patients need to take some of
17:05 --> 17:08these disease modifying agents lifelong
17:08 --> 17:11to reduce those complications like
17:11 --> 17:15hydroxyurea or a newer newly approved medicine.
17:15 --> 17:17However, to cure the disease we need to
17:17 --> 17:19completely change their bone marrow
17:19 --> 17:21so their bone marrow doesn't
17:21 --> 17:23develop sickle cell disease.
17:24 --> 17:26Which benefits those patients
17:26 --> 17:28who are experiencing more complication,
17:28 --> 17:30who requires hospitalization,
17:30 --> 17:33who develops acute chest syndrome like
17:33 --> 17:35pneumonia every year or
17:35 --> 17:38they have developed some stroke like
17:38 --> 17:40symptoms or have developed stroke.
17:40 --> 17:42So these typse of patients can
17:42 --> 17:44benefit if you do the transplant
17:51 --> 17:54and we reduce
17:54 --> 17:56their chronic morbidity.
17:56 --> 17:59So to do the bone marrow transplant
17:59 --> 18:02we need somebody else's bone marrow
18:02 --> 18:04who is exactly like them.
18:04 --> 18:06When we give blood transfusion
18:06 --> 18:09we check the patient and donors blood
18:09 --> 18:11group so in bone marrow transplant
18:11 --> 18:15we do this by doing the HLA typing.
18:15 --> 18:17This is a human leukocyte
18:17 --> 18:19antigen typing which is all the
18:19 --> 18:21blood cells in our body.
18:21 --> 18:24They have some surface markers
18:24 --> 18:27which helps them to identify that
18:27 --> 18:30the given new cells are their own
18:30 --> 18:32or are mimicking like their own
18:32 --> 18:34and they are not different.
18:34 --> 18:37So those cells are accepted by the
18:37 --> 18:40body very easily and that's what we do.
18:40 --> 18:45We type the patient and the
18:45 --> 18:48initially available siblings who are
18:48 --> 18:50biological siblings and if they have a
18:50 --> 18:53match a sibling who doesn't
18:53 --> 18:56have sickle cell that we need to make
18:56 --> 18:59sure we can use that donors
18:59 --> 19:03or the siblings bone marrow to do the
19:03 --> 19:06bone marrow transplant and in the last
19:06 --> 19:09two decades we have done many,
19:09 --> 19:10many sickle cell transplants
19:10 --> 19:13and we have identified that
19:13 --> 19:15if we do the matched sibling,
19:15 --> 19:18from a siblings bone marrow,
19:18 --> 19:21the success rate
19:21 --> 19:24is more than 90% and
19:24 --> 19:27in less than five, six years of age,
19:27 --> 19:29it's up to 99% success rate and
19:29 --> 19:32we can cure
19:32 --> 19:34sickle cell disease and all the
19:34 --> 19:36related complications down the road.
19:37 --> 19:40So a good reason to be kind to
19:40 --> 19:42your siblings because you
19:42 --> 19:44tend to go to the siblings first,
19:44 --> 19:45rather than to the parents,
19:45 --> 19:47grandparents, aunts, and uncles
19:47 --> 19:50who I'm sure are all clamoring
19:50 --> 19:52to try to help their child.
19:52 --> 19:53Is that right?
19:53 --> 19:57Yes. However, the issue with
19:57 --> 20:00sickle cell disease patients there
20:00 --> 20:03are only 20% chance that we find the
20:03 --> 20:07matched sibling who doesn't have
20:07 --> 20:08sickle cell disease as well.
20:10 --> 20:13So in our transplant world we have
20:13 --> 20:15already done the other forms of
20:15 --> 20:17transplant where we can either
20:17 --> 20:19use the half matched mother
20:19 --> 20:22or father or half match sibling
20:22 --> 20:25or as you mentioned another family member.
20:28 --> 20:31We can also use Be the Match which is our
20:31 --> 20:33unknown donor registry where many
20:33 --> 20:36many people have registered themselves
20:36 --> 20:39and they are ready to donate their
20:39 --> 20:42bone marrow if their HLA details
20:42 --> 20:45which are already in the registry are
20:45 --> 20:47matching to the potential patient.
20:47 --> 20:50And we can also do those types of
20:50 --> 20:52transplant using unknown donor
20:52 --> 20:55either 100% match or even 90%
20:55 --> 20:58match transplant using those
20:58 --> 21:00unknown donors bone marrow.
21:00 --> 21:02And so you know,
21:02 --> 21:06one of the questions that comes up then is
21:06 --> 21:08if bone marrow transplant
21:08 --> 21:12has such a high success rate
21:12 --> 21:14in terms of curing sickle cell disease,
21:14 --> 21:18especially if done at a young age,
21:18 --> 21:22why wouldn't you do this in everybody?
21:22 --> 21:25I mean, why wait until people are
21:25 --> 21:26having frequent hospitalizations
21:26 --> 21:28and so on and so forth?
21:28 --> 21:30What's the downside to
21:30 --> 21:32doing bone marrow transplant?
21:32 --> 21:34I'm certain that many parents
21:34 --> 21:36when their child is just
21:36 --> 21:38diagnosed with sickle cell anemia
21:38 --> 21:40if you said, well,
21:40 --> 21:42we have a potentially curative treatment,
21:42 --> 21:44but we're going to hold back on that
21:44 --> 21:47until your
21:47 --> 21:49child requires multiple hospitalizations,
21:49 --> 21:51they might look at you kind of funny.
21:51 --> 21:53That's a very interesting
21:53 --> 21:55question and very good
21:55 --> 21:57debate going on between
21:57 --> 21:59us as a transplanter and the
21:59 --> 22:01hematologist who take care of
22:01 --> 22:03their sickle cell disease patient.
22:03 --> 22:06Because in sickle cell anemia,
22:06 --> 22:07not every patient with sickle
22:07 --> 22:10cell anemia has a severe disease.
22:10 --> 22:12Some may have just the milder
22:12 --> 22:14disease in the initial lifespan,
22:14 --> 22:17and they may develop more complex
22:17 --> 22:20or severe disease down the road.
22:20 --> 22:23And particularly by
22:23 --> 22:25using this penicillin prophylaxis
22:25 --> 22:28and regular follow up with a
22:28 --> 22:30multidisciplinary team
22:30 --> 22:34we pick them up early and support them.
22:34 --> 22:36So they are not having this severe disease.
22:36 --> 22:39However, as I mentioned, as a
22:39 --> 22:42transplanter we do have a good
22:42 --> 22:45success rate with them if we do
22:45 --> 22:46the HLA match sibling transplant.
22:52 --> 22:55We recommend that they have
22:55 --> 22:58their HLA typing and if they have
22:58 --> 23:01a biological sibling we do
23:01 --> 23:03their actual typing and find out
23:03 --> 23:06if they have a matched sibling in the
23:06 --> 23:08family and we keep that in mind.
23:08 --> 23:10And if they start experiencing
23:10 --> 23:13this complication related to the
23:13 --> 23:15sickle cell disease it is better
23:15 --> 23:18we consider them for a mathed sibling
23:18 --> 23:20donor transplant early in life.
23:20 --> 23:23So that is one of the recommendations
23:23 --> 23:25we try to follow.
23:25 --> 23:25However,
23:25 --> 23:28I need to also as a transplanter
23:28 --> 23:30mention that transplant itself,
23:30 --> 23:32bone marrow transplant is not
23:32 --> 23:34a simple process.
23:34 --> 23:36You remove a patients own bone marrow
23:36 --> 23:38and give them the new bone marrow
23:38 --> 23:40and it's
23:40 --> 23:41not a one day surgery.
23:41 --> 23:46It is a complex process that takes time.
23:46 --> 23:49So first we need to remove a patients own
23:49 --> 23:52bone marrow by giving little chemotherapy
23:52 --> 23:54to remove their underlying bone
23:54 --> 23:57marrow which is diseased then we
23:57 --> 23:59give new bone marrow from the donor
23:59 --> 24:01which takes time, maybe two weeks
24:01 --> 24:04to settle down in the patients
24:04 --> 24:07body and then they start working.
24:07 --> 24:09So that requires a complex
24:09 --> 24:11process and hospitalization and
24:11 --> 24:14afterwards also we need to closely
24:14 --> 24:16monitor for the initial few months with
24:16 --> 24:19some medicines and
24:19 --> 24:21regular follow up so
24:21 --> 24:24bone marrow transplant is not a
24:24 --> 24:27simple one day surgery type process.
24:27 --> 24:30However, over the last 10 years we
24:30 --> 24:33have learned how to do it better with
24:33 --> 24:36less side effects and less toxicity.
24:36 --> 24:39I mentioned earlier that to remove
24:39 --> 24:41patients own bone marrow we may we
24:41 --> 24:44have to use some little chemotherapy.
24:44 --> 24:46However because initially we were
24:46 --> 24:48doing this type of transplant
24:49 --> 24:51similarly what we do for leukemia.
24:51 --> 24:54But in last 10 years we learned
24:54 --> 24:56that for sickle cell transplant
24:56 --> 24:59we don't need to use that high
24:59 --> 25:02dose of chemotherapy which we use
25:02 --> 25:03for leukemia patients.
25:03 --> 25:06So nowadays we do sickle cell
25:06 --> 25:08transplant with
25:11 --> 25:14or even some of the protocol
25:14 --> 25:16or in the study we identified
25:16 --> 25:18that we may do transplant without
25:18 --> 25:20using any chemotherapy,
25:20 --> 25:23just one dose of radiation.
25:26 --> 25:29So over the last 10 years there
25:29 --> 25:32has been a great success in
25:32 --> 25:34doing sickle cell transplant
25:34 --> 25:36better with less side effects.
25:36 --> 25:39So that needs to be considered in
25:39 --> 25:42overall care or while we manage
25:42 --> 25:44sickle cell patients to find out
25:44 --> 25:47if they have a HLA matcedh sibling
25:47 --> 25:49and if they have
25:49 --> 25:51started developing some complications
25:51 --> 25:53bone marrow transplant should be
25:53 --> 25:54offered to them.
25:55 --> 25:57And when we think about transplants,
25:57 --> 26:00I mean, I think many people know a
26:00 --> 26:02lot more about organ transplants,
26:02 --> 26:04for example, than maybe they know
26:04 --> 26:05about bone marrow transplants.
26:05 --> 26:07But certainly when we think about
26:07 --> 26:10people who have had transplants,
26:10 --> 26:12one of the things that we often worry about
26:12 --> 26:15is something like graft versus host disease,
26:15 --> 26:19where you can actually reject
26:19 --> 26:22due to a mismatch
26:22 --> 26:25or at least a partial mismatch.
26:25 --> 26:30That either the graft orthe new organ,
26:30 --> 26:33the new bone marrow might start
26:33 --> 26:35reacting to your native cells,
26:35 --> 26:38or vice versa, your immune system
26:38 --> 26:41starts attacking the new
26:41 --> 26:44bone marrow,
26:44 --> 26:47does that happen in bone marrow transplant?
26:47 --> 26:50And do patients who have a bone
26:50 --> 26:52marrow transplant need to be
26:52 --> 26:54on lifelong immunosuppressants?
26:54 --> 26:55I mean,
26:55 --> 26:56is that something that people
26:56 --> 26:58consider in the decision of whether
26:58 --> 27:00to undergo a bone marrow transplant?
27:02 --> 27:05A very good point and I would like to explain
27:05 --> 27:08that yes similar to organ transplant,
27:08 --> 27:10bone marrow transplant patients
27:10 --> 27:13also experience some
27:13 --> 27:16either graft
27:16 --> 27:18failure where a patients own immunity,
27:18 --> 27:19particularly those patients
27:19 --> 27:21who have received many,
27:21 --> 27:23many blood transfusion before
27:23 --> 27:25they go for transplant,
27:25 --> 27:28they experience complications and they
27:28 --> 27:32reject the donor cells or the donor cells
27:32 --> 27:35fight with the patients own cells and
27:35 --> 27:38that is called graft vs host disease.
27:38 --> 27:41So the main point here if you
27:41 --> 27:44do transplant early in the life,
27:44 --> 27:47particularly less than 10 years of age,
27:47 --> 27:49this type of complication with matched
27:49 --> 27:52sibling donor transplant is less.
27:52 --> 27:54So that's why we recommend early
27:54 --> 27:55bone marrow transplant.
27:55 --> 27:57And then just the last query
27:57 --> 28:00which you asked is do they need
28:00 --> 28:01the lifelong immunosuppression?
28:01 --> 28:03Not in bone marrow.
28:03 --> 28:05We are completely
28:05 --> 28:07changing their immunity so once the
28:07 --> 28:10new bone marrow has settled down
28:10 --> 28:12they don't need this
28:12 --> 28:13lifelong immunosuppression.
28:13 --> 28:15That is the main difference
28:15 --> 28:16between the solid organ transplant
28:16 --> 28:18and our bone marrow transplant.
28:18 --> 28:21Bone marrow transplant patients are
28:21 --> 28:23mainly on immunosuppression for maybe
28:23 --> 28:26six months or a year or little longer,
28:26 --> 28:27but afterwards no medicine.
28:28 --> 28:30Doctor Niketa Shah is associate
28:30 --> 28:33professor of Pediatrics and hematology
28:33 --> 28:35oncology and director of the pediatric
28:35 --> 28:37bone Marrow Transplant program
28:37 --> 28:39at the Yale School of Medicine.
28:39 --> 28:41If you have questions,
28:41 --> 28:43the address is canceranswers@yale.edu,
28:43 --> 28:46and past editions of the program
28:46 --> 28:48are available in audio and written
28:48 --> 28:49form at yalecancercenter.org.
28:49 --> 28:51We hope you'll join us next week to
28:51 --> 28:53learn more about the fight against
28:53 --> 28:55cancer here on Connecticut Public Radio.
28:55 --> 28:56Funding for Yale Cancer Answers
28:56 --> 28:58is provided by Smilow Cancer
28:58 --> 29:00Hospital.

Information

Transplant for Pediatric Patients with Sickle Cell Disease August 28, 2022 Yale Cancer Center visit: http://www.yalecancercenter.org email: canceranswers@yale.edu call: 203-785-4095