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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
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Dr. Niketa ShahTo Cite
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