Comprehensive Care for Sickle Cell Disease
From the Show: Childrens Health - Peer
Summary:
Air Date: 8/26/21
Duration: 10 Minutes
Each year, the Children’s Health sickle cell disease program cares for approximately 900 children with various forms of sickle cell disease, often with the very treatments their researchers developed. Learn more about the state-of-the-art approaches to infection prevention, pain management and treatment of specific organ-related complications.
Transcription:
Scott Webb: Welcome to Pediatric Insights, advances, and innovations with Children's health. I'm Scott Webb. And today, we're discussing the pediatric sickle cell disease program at Children's Health, one of the nation's largest and most distinguished programs for treating children with sickle cell disease. The program focuses on the prevention of disease complications and management using the newest treatment strategies.
And on this episode, we'll learn more about the comprehensive care the team provides from Dr. Lincy Thomas. She's a pediatric hematologist-oncologist and assistant professor at UT Southwestern Medical Center. Doctor, thanks so much for your time today. I want to have you start off by telling us about the sickle cell disease program at Children's Health?
Dr. Lincy Thomas: So we take care of over 900 children with various forms of sickle cell disease. And our team here consists of UT Southwestern physicians, advanced practitioners, nurses, psychologists, and the social worker to help coordinate for the needs of our patients. I truly believe our team works cohesively and as one unit. Because even if the physician is able to medically serve the patient well, if the social worker is not able to meet the social needs of the family, we're not truly able to take care of the whole patient.
Scott Webb: Yeah. And 900 seems like a lot. And I want to ask you, what sets the program apart from others across the nation? And maybe you could start with Dr. Buchanan and his contributions to the program. I know he was actively involved in the diagnosis and management of children and adolescents with blood diseases and was a national leader in investigator-initiated and multicenter collaborative research for sickle cell disease.
Dr. Lincy Thomas: The initiative by Dr. Buchanan was initiated by the Dallas Newborn Cohort and that's what honestly sets this program apart. It's the world's largest group of patients with sickle cell disease that was initially diagnosed by newborn screen. This group of patients has continued to be followed through their admissions for acute chest, for pain crises. Pioneers like Dr. Charles Quinn can extrapolate the data to identify patients, for example, with lower oxygen saturations and lower levels of oxygen in their blood who are more likely to develop stroke. And so this data continues to exist, and I hope to one day use this data because patient information just continues to get collected through time.
Scott Webb: That would be great to be able to do that. And I'm wondering if you can talk about the treatments that researchers developed to treat sickle cell disease.
Dr. Lincy Thomas: There's multiple treatments that researchers use to treat sickle cell disease. Hydroxyurea being the first is the most promising medication use for patients with hemoglobin SS and S beta zero. It works by increasing fetal hemoglobin and making them less likely to sickle. HU also decreases the overall inflammatory state of patients with sickle cell disease and decreases the risk of pain crisis, acute chest, stroke and renal disease by decreasing the sticky nature of sickle cells to platelets and white blood cells.
Hydroxyurea is recommended in children as young as nine months of age, patients are closely monitored with monthly labs to follow up when H two dose is adjusted to monitor for risk of myelosuppression, to make sure that the white blood cell counts are not too low and the platelet counts and hemoglobin are also within inappropriate range.
Voxelotor or Oxbryta is also another agent that researchers use or physicians use to treat sickle cell disease in adults and children older than 12 years of age. Oxbryta interferes with hemoglobin S polymerization and prevents the sickling of red cells and homolysis. And last, Crizanlizumab or Adakveo is indicated to reduce the frequency of vaso-occlusive pain crisis in adults and pediatric patients, 16 years and older with sickle cell disease.
So sickle cell disease is associated with a pro-inflammatory state with overexpression of pieces. Then on activated endothelial cells and platelets, which can lead to visa, occlusion or a pain crisis. So how adapt you works is it's a targeted monoclonal antibody that binds the piece electing on the surface of it.
Activated endothelial cells and platelets cells. So this blocks the interaction among endothelial cells, platelets, red blood cells and white blood cells. And the was proven to reduce the frequency of VOC, regardless of VOC, baseline frequency or the genotype of sickle cell or hydroxyurea use. So these agents are extremely useful in sickle cell disease.
Scott Webb: Yeah. And I know the team provides comprehensive care, meaning you care for patients with a range of symptoms. Maybe you can talk more about that.
Dr. Lincy Thomas: So patients with sickle cell disease are more prone to a variety of symptoms, such as bedwetting, snoring at night, headaches, renal disease, retinopathy, avascular necrosis of the hip and stroke. So during these comprehensive visits, we actually go through a list of these symptoms to screen for specific disease processes to best care for this population.
For example, we specifically ask patients if they have recurrent symptoms of acute chest, if they're admitted for these symptoms, if they have some symptoms of asthma. And so if they do have these symptoms, we actually try to tailor these patients to come to a combined sickle cell and pulmonology visit. We have a pulmonology in sickle cell clinic where they can come and get better care, because we've got a pulmonologist there as well as a hematology sickle cell provider. These patients can get care there addressing the constant sickling crisis as well as injury to their lungs. This can be a continuous process that can be repetitive. So just tailoring their care this way in a comprehensive setting can be helpful for patients.
Scott Webb: I'm sure it can be. And one of the focuses of the program is preventing disease complications and management, right? So how do you accomplish this for the patients?
Dr. Lincy Thomas: So as providers, it's our duty to educate families regarding disease-related complications and equip them with skills to recognize problems that arise in patients with hemoglobin SS and S-beta 0 thalassemia. One of the best ways to prevent complications should be to give them the option to start hydroxyurea as young as nine months of age to help prevent the complications associated with the disease, given that they understand both the risks and the benefits of this medications. So with decades of research, we now know that hydroxyurea is a disease-modifying agent that decreases the sticky nature of red blood cells to white blood cells and platelets, and overall decreases the incidence of pain crisis, strokes, acute chest, and renal disease in children with SS an S-beta 0 thalassemia. And so really just talking to families and giving them the option of starting this medication so that they can make the best decision for their child is one way of helping prevent the disease-related complications.
Other management options is screening for disease complications, such as renal disease, avascular necrosis for patients who complain of chronic hip pain and retinopathy. But these screenings do not typically start until children are older, around 10 years of age.
Scott Webb: And the flagship hospital for Children's Health is in Dallas, Texas, of course. But you provide care across the Dallas-Fort Worth metroplex as well. I like to have you talk a little bit about how the program has expanded.
Dr. Lincy Thomas: So we've expanded our care to the Plano Campus as well. And currently, I'm seeing sickle cell patients for comprehensive care every two months in Plano. And I hope to continue to do that because I truly believe that I've started to serve more of the patients who live out in the Plano-Frisco area instead of them having to drive all the way out to Dallas. And it's been really convenient for these families because I've noticed that they've become so much more appreciative since I've moved out to that area. And just similar to the care that we provide here in Dallas, it's not just me and the team, but it's the advanced practitioner, the nursing staff and psychology to help provide care for the whole of the patient and the family's needs.
Scott Webb: Yeah. Well, this has been really educational today. Anything else you want to share? What are your takeaways today?
Dr. Lincy Thomas: So as a medical community, we have a lot of work to do to better serve patients with sickle cell disease. And despite being a disease that was first identified more than a hundred years ago, the development of disease-modifying therapies are limited due to the inadequate research funding. I truly believe that it's our time to advocate for our patients raise awareness of biases and make a change for our patients with sickle cell disease and no better time than now.
Scott Webb: Yeah, that's a great way to put that. There is no better time than now. There's still so many advancements, still so much reason for optimism about the future in sickle cell disease treatment and your program there at Children's Health. So thanks so much, doctor, and you stay well.
Dr. Lincy Thomas: Thank you.
Scott Webb: And thank you for listening to Pediatric Insights. For more information, go to childrens.com/ccbd.
Scott Webb: Welcome to Pediatric Insights, advances, and innovations with Children's health. I'm Scott Webb. And today, we're discussing the pediatric sickle cell disease program at Children's Health, one of the nation's largest and most distinguished programs for treating children with sickle cell disease. The program focuses on the prevention of disease complications and management using the newest treatment strategies.
And on this episode, we'll learn more about the comprehensive care the team provides from Dr. Lincy Thomas. She's a pediatric hematologist-oncologist and assistant professor at UT Southwestern Medical Center. Doctor, thanks so much for your time today. I want to have you start off by telling us about the sickle cell disease program at Children's Health?
Dr. Lincy Thomas: So we take care of over 900 children with various forms of sickle cell disease. And our team here consists of UT Southwestern physicians, advanced practitioners, nurses, psychologists, and the social worker to help coordinate for the needs of our patients. I truly believe our team works cohesively and as one unit. Because even if the physician is able to medically serve the patient well, if the social worker is not able to meet the social needs of the family, we're not truly able to take care of the whole patient.
Scott Webb: Yeah. And 900 seems like a lot. And I want to ask you, what sets the program apart from others across the nation? And maybe you could start with Dr. Buchanan and his contributions to the program. I know he was actively involved in the diagnosis and management of children and adolescents with blood diseases and was a national leader in investigator-initiated and multicenter collaborative research for sickle cell disease.
Dr. Lincy Thomas: The initiative by Dr. Buchanan was initiated by the Dallas Newborn Cohort and that's what honestly sets this program apart. It's the world's largest group of patients with sickle cell disease that was initially diagnosed by newborn screen. This group of patients has continued to be followed through their admissions for acute chest, for pain crises. Pioneers like Dr. Charles Quinn can extrapolate the data to identify patients, for example, with lower oxygen saturations and lower levels of oxygen in their blood who are more likely to develop stroke. And so this data continues to exist, and I hope to one day use this data because patient information just continues to get collected through time.
Scott Webb: That would be great to be able to do that. And I'm wondering if you can talk about the treatments that researchers developed to treat sickle cell disease.
Dr. Lincy Thomas: There's multiple treatments that researchers use to treat sickle cell disease. Hydroxyurea being the first is the most promising medication use for patients with hemoglobin SS and S beta zero. It works by increasing fetal hemoglobin and making them less likely to sickle. HU also decreases the overall inflammatory state of patients with sickle cell disease and decreases the risk of pain crisis, acute chest, stroke and renal disease by decreasing the sticky nature of sickle cells to platelets and white blood cells.
Hydroxyurea is recommended in children as young as nine months of age, patients are closely monitored with monthly labs to follow up when H two dose is adjusted to monitor for risk of myelosuppression, to make sure that the white blood cell counts are not too low and the platelet counts and hemoglobin are also within inappropriate range.
Voxelotor or Oxbryta is also another agent that researchers use or physicians use to treat sickle cell disease in adults and children older than 12 years of age. Oxbryta interferes with hemoglobin S polymerization and prevents the sickling of red cells and homolysis. And last, Crizanlizumab or Adakveo is indicated to reduce the frequency of vaso-occlusive pain crisis in adults and pediatric patients, 16 years and older with sickle cell disease.
So sickle cell disease is associated with a pro-inflammatory state with overexpression of pieces. Then on activated endothelial cells and platelets, which can lead to visa, occlusion or a pain crisis. So how adapt you works is it's a targeted monoclonal antibody that binds the piece electing on the surface of it.
Activated endothelial cells and platelets cells. So this blocks the interaction among endothelial cells, platelets, red blood cells and white blood cells. And the was proven to reduce the frequency of VOC, regardless of VOC, baseline frequency or the genotype of sickle cell or hydroxyurea use. So these agents are extremely useful in sickle cell disease.
Scott Webb: Yeah. And I know the team provides comprehensive care, meaning you care for patients with a range of symptoms. Maybe you can talk more about that.
Dr. Lincy Thomas: So patients with sickle cell disease are more prone to a variety of symptoms, such as bedwetting, snoring at night, headaches, renal disease, retinopathy, avascular necrosis of the hip and stroke. So during these comprehensive visits, we actually go through a list of these symptoms to screen for specific disease processes to best care for this population.
For example, we specifically ask patients if they have recurrent symptoms of acute chest, if they're admitted for these symptoms, if they have some symptoms of asthma. And so if they do have these symptoms, we actually try to tailor these patients to come to a combined sickle cell and pulmonology visit. We have a pulmonology in sickle cell clinic where they can come and get better care, because we've got a pulmonologist there as well as a hematology sickle cell provider. These patients can get care there addressing the constant sickling crisis as well as injury to their lungs. This can be a continuous process that can be repetitive. So just tailoring their care this way in a comprehensive setting can be helpful for patients.
Scott Webb: I'm sure it can be. And one of the focuses of the program is preventing disease complications and management, right? So how do you accomplish this for the patients?
Dr. Lincy Thomas: So as providers, it's our duty to educate families regarding disease-related complications and equip them with skills to recognize problems that arise in patients with hemoglobin SS and S-beta 0 thalassemia. One of the best ways to prevent complications should be to give them the option to start hydroxyurea as young as nine months of age to help prevent the complications associated with the disease, given that they understand both the risks and the benefits of this medications. So with decades of research, we now know that hydroxyurea is a disease-modifying agent that decreases the sticky nature of red blood cells to white blood cells and platelets, and overall decreases the incidence of pain crisis, strokes, acute chest, and renal disease in children with SS an S-beta 0 thalassemia. And so really just talking to families and giving them the option of starting this medication so that they can make the best decision for their child is one way of helping prevent the disease-related complications.
Other management options is screening for disease complications, such as renal disease, avascular necrosis for patients who complain of chronic hip pain and retinopathy. But these screenings do not typically start until children are older, around 10 years of age.
Scott Webb: And the flagship hospital for Children's Health is in Dallas, Texas, of course. But you provide care across the Dallas-Fort Worth metroplex as well. I like to have you talk a little bit about how the program has expanded.
Dr. Lincy Thomas: So we've expanded our care to the Plano Campus as well. And currently, I'm seeing sickle cell patients for comprehensive care every two months in Plano. And I hope to continue to do that because I truly believe that I've started to serve more of the patients who live out in the Plano-Frisco area instead of them having to drive all the way out to Dallas. And it's been really convenient for these families because I've noticed that they've become so much more appreciative since I've moved out to that area. And just similar to the care that we provide here in Dallas, it's not just me and the team, but it's the advanced practitioner, the nursing staff and psychology to help provide care for the whole of the patient and the family's needs.
Scott Webb: Yeah. Well, this has been really educational today. Anything else you want to share? What are your takeaways today?
Dr. Lincy Thomas: So as a medical community, we have a lot of work to do to better serve patients with sickle cell disease. And despite being a disease that was first identified more than a hundred years ago, the development of disease-modifying therapies are limited due to the inadequate research funding. I truly believe that it's our time to advocate for our patients raise awareness of biases and make a change for our patients with sickle cell disease and no better time than now.
Scott Webb: Yeah, that's a great way to put that. There is no better time than now. There's still so many advancements, still so much reason for optimism about the future in sickle cell disease treatment and your program there at Children's Health. So thanks so much, doctor, and you stay well.
Dr. Lincy Thomas: Thank you.
Scott Webb: And thank you for listening to Pediatric Insights. For more information, go to childrens.com/ccbd.