Moving Forward: The Transition from Pediatric to Adult Care in Sickle Cell Disease

April 11, 2022

ID7692

To CiteDCA Citation Guide

00:00Our last speaker before me
00:03is Doctor Cecilia Calhoun,
00:05who is assistant professor of internal
00:08medicine in the section Pneumatology.
00:10We're talking to us about sickle cell.
00:12Thanks so much for speaking with us.
00:16Fact admit I was kind of nervous
00:18to go after Marcella but I
00:20remember that one of her titles is
00:22guiding light to people like me.
00:23She's the reason I'm here and
00:25how thankful I am for this
00:27opportunity to share my work to her,
00:29to Tisha to the YMCA family for giving
00:31me the space to talk with you all.
00:33A little bit about the work that I do.
00:36OK so I wanna just ground us with the case.
00:39A patient of mine in the because the work
00:42that I do is for my sickle cell patients.
00:44So I want to talk to you about an AG
00:46so he's a 23 year old man that I met.
00:48He has hemoglobin SC disease and I met
00:50him as a young adult in Transition clinic.
00:52Didn't care for him as a kid.
00:54His disease have been complicated
00:56by splenic sequestration and pain
00:58and also some eye challenges and
01:00he was following pretty regularly
01:02when he was a child.
01:03But as he kind of got older
01:05when it's adolescence his care.
01:06Became a bit more intermittent.
01:10I think I want the same dress when
01:12you look at and when you look at AG
01:15you know many of the challenges he
01:17faced went outside of our clinic and
01:19he had academic challenges secondary
01:21to his pain and frequent admissions.
01:23He had lost his insurance coverage.
01:24He wasn't able to take obtained hydroxyurea
01:26or is acute pain medications due to
01:28insurance issues and it had challenges
01:30with the criminal justice system and
01:33subsequent challenges with employment
01:34because of that and because of its pain.
01:37This also precipitated a lot of anxiety.
01:39For him.
01:41And so the reason I excuse me,
01:43the reason I bring up and him as an
01:45example is because he represents
01:46one of many of the patients that we
01:48see who at a very surface level or
01:49one on one interaction and clinical
01:51setting can seem like a series
01:54of pathophysiologic challenges.
01:55But the disease that they experience
01:57is in the context of so much greater,
01:59so much more greater,
02:00and my work and my goal is to help to
02:02understand the full context of what
02:04they are and think about how we can as
02:06physician scientists and researchers
02:08can improve their care overall.
02:10OK,
02:11so let's talk a little bit about
02:12sickle cell disease.
02:13Sickle cell disease is the most
02:15common monogenetic disorder in the
02:17world with approximately 300,000
02:19live births each year in the world.
02:21And that's because of its protective
02:24Ness against malaria.
02:27In the United States,
02:28our most recent data says that
02:30it's about 100,000 Americans,
02:31mostly of African descent.
02:33With increasing incidence and
02:35accounts for about one in every
02:37365 African American births,
02:38mostly concentrated in
02:39the most populous state.
02:41In fact, 91% of sickle cell
02:43patients are in about 23 states.
02:48When we think about sickle cell disease,
02:50the genetic mutation results in
02:52abnormal formation of red blood cells,
02:54which have a negative impact impact
02:57with the microvascular environment
02:58causing many clinical manifestations.
03:00So I know this is a full slide,
03:02but it can range from acute
03:05acute complications like pain,
03:06which I think is most path and
03:08Monica and what most people
03:09associate with sickle cell disease.
03:11But also it's important for us
03:12to think of other complications
03:14like stroke that happen long term.
03:20So the life expectancy of patient with
03:22people with sickle cell disease is
03:25significantly lower than their counterparts.
03:27So with this figure shows us is
03:29probability of survival by age and
03:30you can see that patients with
03:32sickle cell have a decreased life
03:34expectancy of really about 20 years.
03:38Even with the advent of hydroxyurea,
03:40which increases fetal hemoglobin and is one
03:42of our only disease modifying medications,
03:45their life expectancy is
03:47still significantly lowered.
03:51So again, this point illustrates
03:52mortality and sickle cell disease,
03:54and what's important to know about this
03:56figure with the age of death by age
03:58group on the X axis and the percentage
04:00of deaths on the Y axis is that in the
04:02late 70s there were two peaks of death.
04:04There was that infancy and toddler peak,
04:05and then it started to rise around
04:07adolescence and young adulthood.
04:09But because of clinical trials
04:11and multi center studies and the
04:13implementation of prophylactic penicillin
04:16and vaccination and newborn screen,
04:18we were really able to decrease.
04:20A peak around infancy and toddlerhood,
04:23but this piece around adolescence and
04:24young adulthood is still persists.
04:29This data was corroborated
04:30by the Dallas newborn cohort,
04:31which was is the largest single center
04:34cohort today with 940 sickle cell patients.
04:37What this cohort showed us is that
04:39the children with sickle cell disease
04:42are really living until adulthood,
04:44but it has, so it's shifted from a
04:47chronic acute disease of childhood
04:49to a chronic disease and the burden
04:51of disease is really on adults.
04:53The interesting thing about this
04:54cohort is that of the patients who
04:56reported deaths in the cohort that
04:58all would die within two years
04:59of transition to adult care.
05:00OK, so what's important to know about
05:02this is that the transition space is
05:04a very vulnerable time for patients
05:06with sickle cell disease persons.
05:08So it's a further characterize this,
05:11and in addition to understanding
05:13the morbidity is high.
05:15Myself and my colleagues at
05:17Washington University and the Center
05:18for Administrative Data Research
05:20took a look at utilization data
05:22for sickle cell disease.
05:23We looked at the healthcare
05:25cost and utilization project,
05:27commonly known as H cup data,
05:29which is a rich source of data for
05:31inpatient and hospitalizations.
05:33Admissions and administrative data.
05:35We looked at the state of New York in 2004,
05:38five to 14.
05:39Been used in a crypted identifier to
05:41understand patient level encounters versus
05:43single center level encounters to get a
05:45better understanding of utilization patterns,
05:47including hospitalizations.
05:48Readmission rates.
05:49We were able to look at over 19,000 patients
05:53with over 140,000 individual encounters.
05:58And what we found corroborated other data.
06:00You know,
06:01we found that adolescents and
06:03adults had the highest rates of
06:05hospitalization amongst the entire cohort.
06:08So we have the percent of total counters
06:10represented by the line in Orange.
06:11And then blue represents per
06:14ten person years.
06:16And then when we looked at readmission rates,
06:18they too were highest among adult that
06:21adolescent and young adult age group.
06:24So and only do we know that the
06:26the adolescent and young adult age
06:27group is a period of high morbidity
06:29and mortality and high utilization.
06:31We can.
06:32We can surmise that young adults
06:34with sickle cell are the most
06:36vulnerable during that transition
06:37from pediatric to adult care.
06:41So transition to sickle cell
06:43disease is a complicated spot.
06:45OK represents this intersection of.
06:47I used to say normal adolescent
06:48development until I got some
06:49pushback like what do you mean?
06:50Normal adolescence like?
06:51You know my my friends with
06:53adolescence tell me it's not normal,
06:55but they're the insidious complications
06:57of their disease coming together.
06:58Changes in resources like our
07:00patient AG and then a big changes
07:03in that social environment.
07:07To better characterize this
07:09specific transition for patients
07:10with sickle cell disease,
07:12we conducted a qualitative
07:14study with 63 participants.
07:16So key stakeholders,
07:18including providers, patients,
07:20and their caregivers about what
07:22are the challenges they face when
07:24they were trying to get essential
07:26healthcare and educational needs.
07:28One thing that I enjoy about
07:29using a qualitative approach
07:30is that it's not prescriptive,
07:32but it allows our patients to tell
07:33us what they need and what's most
07:35important to them so that we as physicians.
07:38Scientists can address their needs
07:39and the way that's important to them,
07:42so I want to share with you a little
07:44bit of the themes that emerge and also
07:46a few of what our participants said.
07:50So our patients talk to us about
07:53this change in environment,
07:55about how when they were kids they
07:57got treated one way and when they
07:58went to the healthcare system it was
08:00different and how lead leaving those
08:01that have known him their whole lives
08:03is so intimidating and that they
08:05can see and feel that difference.
08:09Our providers individuals committed
08:10to quality care in these patients told
08:12us we really need to start talking
08:14about this with patients young.
08:16We need to prepare them.
08:17We want to empower them and
08:18give them what they need to
08:20make a successful transition.
08:24So we use the consolidated framework
08:27for implementation research,
08:28which is a commonly used
08:29implementation science framework,
08:30which I'll talk a little bit about
08:33shortly to understand the major themes
08:35in transition and the way that this
08:37study expounded upon those previous
08:39understanding of transition is that
08:41it really started to include the
08:43stigma that patients with sickle cell
08:44disease talked about that they faced.
08:46We highlighted the systemic issues with
08:48challenges with hematologic providers that
08:50want to care for sickle cell patients.
08:51It helped us understand the role of health
08:53literacy and the need and preparing.
08:55Are young adults for transition,
08:56in addition to highlighting
08:58the disease complications and
09:00changing resources that we know.
09:02So we know that transition is not only a
09:05problem for patients with sickle cell,
09:08it's a problem overall.
09:09It's full audience and I'm like how
09:10many of you all were in college.
09:12Seeing your pediatrician.
09:13So the question is where do we start like?
09:16What do we do?
09:17OK, we know that this is a challenge,
09:18but we want to be actual oriented.
09:19So what do we do?
09:21So in 2011,
09:22the American Academy of Pediatrics
09:24published a report for guidelines
09:26for healthcare transition and then
09:28in 2018 they actually included an
09:30update which was a collaboration with
09:32the American Society of Hematology
09:34to outline processes for a success.
09:36What they proposed as a
09:37successful transition,
09:38and this was shown to be effective
09:40in a primary care clinic and
09:42endocrinology clinic as well.
09:46This this guideline centers on
09:486 core elements of transition,
09:50so this includes having a clear
09:52policy tracking appropriately
09:54preparation and readiness planning.
09:56Integrating into adult care,
09:57and then doing iterative feedback to
10:00make sure that this space is completed.
10:02So my question was if we know
10:04that this is a problem and we
10:07have a evidence based solution,
10:09why do we not have structured transition
10:11programs for sickle cell disease and
10:14that is the work that we're doing?
10:16So the way that I do that,
10:18and I believe the first step in
10:20approaching and helping sickle
10:21cell patients to use what we have,
10:23let's apply what we have to a population
10:25that needs it to bring about change.
10:27And so I do this through
10:29implementation science.
10:29So what's implementation science?
10:31It says that exactly,
10:32that it is the study of methods to
10:34promote the increase of evidence
10:36based interventions and to usual
10:38care gives us the space to rigorously
10:40understand what are the context in
10:42which we're applying these evidence
10:44based interventions to change make change.
10:47When we think about the
10:49translational pipeline,
10:49we know that it spans from our
10:51basic scientist colleagues who
10:53are making clinical excuse me
10:54laboratory discoveries all the way
10:56to translation into community,
10:57much like our CSA
11:00dissemination implementation,
11:01science really centers around
11:03that T3T4 pathway,
11:04bringing this knowledge to the community.
11:08Particularly in sickle cell disease,
11:10this is really important,
11:11and So what this slide is showing
11:13is the timeline from understanding
11:15and results in a clinical trial to
11:18stop trial to clinical practice,
11:20and so in 1998 the STOP trial is the
11:21trial that showed us if we're able
11:23in sickle cell to screen patients
11:25using a transcranial Doppler for
11:27abnormal vasculature and we start
11:28them on prophylactic transfusions.
11:30We can stop them from having strokes.
11:31OK, so this is an evidence based practice.
11:34This is a multi center institutional study.
11:37OK, but in 2015.
11:39Only 40% of patients were still
11:40getting screened. That's 17 years.
11:42That's a whole lifespan,
11:44and so for those of you all in
11:45the audience who are parents.
11:47If your child had asthma or
11:48a life threatening disease,
11:49how long would you be willing to
11:51wait to understand like a life,
11:52a life saving intervention or a
11:54therapy that could change the
11:55course of their disease.
11:57So for patients with sickle cell disease
11:59who already have a short lifespan,
12:01time is of the absolute essence
12:03and decreasing in this timeline,
12:05that's the role of implementation science.
12:07So that's why it's important
12:08in the work that I do.
12:09Because when we have a population with
12:12limited interventions and a sense of urgency,
12:14having the tools and methodologic
12:16rigor to decrease that timeline
12:18is critically important.
12:19We also know that our CSA works and
12:22collaborates with implementation of
12:23scientists to promote HealthEquity,
12:26so I think it's been a constant theme
12:28running throughout the program today.
12:29But three main ways that they do that
12:31is through community engagement and
12:33partnership through it that workforce
12:35not just myself as a junior faculty,
12:37but we learned a lot about interns
12:38who are being trained up in this
12:40work and then financial support,
12:41which I think we all kind of talked about.
12:43The scientists who are presenting our work.
12:46So implementation science a lot
12:48is executed through the use of
12:51frameworks and models which help
12:53to give structure to to our work.
12:55So for my project,
12:56which I'm going to talk about a bit now,
12:58I used to integrate it promoting
13:00action on healthcare research,
13:02implementation and healthcare
13:03sciences or IPAROS framework.
13:05And So what this framework does is
13:07it looks at the innovation of the
13:09the intervention of the recipients
13:10of the intervention and context
13:12and the role of facilitation and
13:14successful implementation of this work.
13:16So I'm looking at these.
13:17When it's based guidelines,
13:18I want to understand all of these
13:20things and how to implement them
13:22more more expeditiously.
13:23So at first did this by taking a
13:25look at our current transition
13:26processes and understanding of the
13:28guideline So what I did was I did
13:30a qualitative study with pediatric
13:32hematology and adult hematology
13:34providers to get their knowledge
13:37about these AP guidelines had you
13:38heard of them are you using them
13:40what strategies helped you to use
13:42them are they effective do you
13:43agree with them and then we use
13:45the I Paris framework to frame
13:47the role of? What is facilitation
13:48in terms of how we can implement
13:50this more expeditiously.
13:54So what we found are three major
13:56areas in which our guidelines were not
13:58able to be successfully implemented.
14:00So a lot of it had to do with lack of
14:03buy in support and location and Co.
14:06Location of these clinics.
14:07But we also asked them, hey,
14:09what was working well for you and so
14:11that was these readiness assessments
14:12and having these integrated clinics
14:14and ways in which we can improve
14:16like our process standardization
14:17and then identifying, again,
14:19facilitation key personnel involved
14:21in this work that could help on
14:23the uptake of these guidelines.
14:25Interestingly enough,
14:26but not surprisingly,
14:27we also found factors that we weren't
14:29looking for that were relevant to
14:31these guideline implemented guideline
14:33implementation and so now we would.
14:35We would categorize these as a
14:36social determinants of health.
14:38But there are so many factors
14:39outside of our hospital outside of
14:41our clinic where these guidelines
14:42are supposed to be implemented that
14:44really impact the way that we're
14:45able to successfully care for our
14:47patients in the clinic setting.
14:54So now what I'm doing is trying to
14:56figure out optimal implementation
14:57strategies for these guidelines,
14:59so implementation strategies that
15:01are methods and tools with which
15:03we use to increase these guideline
15:05uptake and so there is a cadra of
15:08evidence based recommendations,
15:09but maybe all of them aren't applicable
15:11to this and we use what we found
15:13the determinants of barriers and
15:14facilitators from that prior work to
15:16identify which ones are most important
15:18and that and that the initial pass.
15:20We identified 52 right?
15:22So the work I'm doing?
15:24Now is to narrow it down amongst
15:26pediatric and adult hematologists.
15:27Which one of these have which
15:28of these have been most useful?
15:30Which one of these have you tried?
15:31Which ones have you not tried at
15:33all and the goal of the next step in
15:35this work is to put those together
15:37with these guidelines to create
15:38an implementation facilitation
15:39guide which I plan on planning,
15:41piloting here at Yale.
15:42So the idea is to say what are your
15:45specific needs as a sickle cell community?
15:47How do your guidelines fit with these?
15:49What are the strategies we
15:51can use to optimize success?
15:54My work now works on developing the processes
15:57to improve guideline implementation,
16:00but the goal is to develop structure
16:03transition programs that will overall
16:05ultimately improve outcomes in our
16:07young adults with sickle cell disease.
16:09And we know, and I'm I'm biased.
16:11I know that that truly
16:13adolescents and young adults.
16:14I believe they're at a critical juncture
16:16in which we can optimize equity and
16:18support them and the decisions we
16:19make and how the support that we show
16:21them at this very pivotal time in
16:23their life can have long term positive
16:25implications for how they move forward.
16:29And so with the hindsight of knowledge,
16:30when you think about a patient
16:31that I mentioned earlier, like AG,
16:33how would you treat him if you
16:34were his clinician,
16:35how would you work with him?
16:36Because despite all of those challenges,
16:38I'm that I described he managed
16:40to establish a regular healthcare
16:42with our clinic he got.
16:44He was in a behavioral health care
16:46program to navigate his own trauma.
16:47We worked,
16:48he worked with our social worker to find
16:50stable housing and employment and before
16:51his untimely death due to gun violence,
16:54he was on a pathway to success.
16:55So he's very, very important.
16:57He's a great example for me because.
16:59Shows us if we really wrap around
17:00in a rigorous way our young people,
17:02they can really achieve success.
17:05And I asked that because I truly
17:06believe that our adolescents and
17:08young adults with sickle cell disease
17:09are a microcosm of how we treat
17:11sickle cell patients as a whole,
17:12but also the most vulnerable
17:14populations among us.
17:16And so the strides that we make with them,
17:18the care and investment to rigorous
17:20research that we that we make
17:22regarding this population has
17:24greater implications for us all.
17:25And I'll end with this slide because.
17:28One of the things about the work that
17:30I do helps me to understand a bit more
17:33about how and and the patient that I
17:36mentioned help me to understand how
17:37the work that I do in treatment with
17:39patients really fits into a greater context.
17:41We know that no matter where
17:43we are along this pipeline, OK,
17:45whether we are creating the laws,
17:46regulations,
17:47and policies,
17:47whether we are affecting the living
17:50environment or whether we are
17:52understanding the risk factors and
17:54diagnosis and treatment ultimate
17:55outcomes in a specific disease.
17:57Equity is along that whole piece.
17:59So I wanted to end with this just
18:01to implore you all in the audience
18:02to think carefully about the work
18:04you do and where that fits on
18:06this HealthEquity pipeline.
18:07I think we want to be intentional
18:09on the work that we do,
18:11because if we're not making
18:12a conscious investment,
18:13we're choosing to divest.
18:14And so I just wanted to take a moment
18:16to end with that and encourage you
18:18all wherever you are to leverage the
18:20power you have for the importance of equity,
18:23and so that's all I have.
18:24I want to thank Marcella,
18:26but I'm very much for this opportunity
18:27and for her continuous mentorship.
18:29Mentor Allison Kinga watched you,
18:31my peers, and adult college.
18:32Obviously the Yale Center for
18:34Clinical Investigation was
18:35supporting me and doing this work,
18:36and more than anybody in
18:38my patients and families.