Key points from today’s guests:
Dr. Joseph McGuirk, D.O., hematologist & oncologist, division director, Hematological Malignancies and Cellular Therapeutics, The University of Kansas Health System
- Normally, blood cell are nice and smooth and malleable, and they make their way through blood vessels carrying oxygen and carrying waste products away.
- But when they sickle like that, because of the genetic abnormality, they stack up and obstruct the blood vessels. They cause serious damage to organs because organs can't get an appropriate blood supply and tissues become damaged and die.
- Sickle cell disease an extremely serious disease that has a shortened life expectancy of only 42 years. And for many, many patients it can be a life of great suffering.
- Most people affected in the world are in Sub Saharan Africa, the Mediterranean basin, and India. In the United States, sickle cell disease affects about 100,000 people a year.
- Stem cell donors are not easy to find. If you're Caucasian of Western European descent, chances of having a match is 75-80 percent. If you're of African American descent, it's about a 29 percent chance.
- But we have fantastic news about newer options. This past December at the largest blood cancer and blood disorders meeting in the world – the American Society of Hematology meeting – we showed that 90 percent of patients after allogeneic stem cell transplant at two years follow up, the overall survival exceeds 95 percent.
- So it's really a stunning time and mismatched transplants are safe and highly effective in these patients. And that opens the door for everybody with sickle cell disease.
- There is also recently approved gene therapies, which are also seeing incredibly positive outcomes.
- Many of the affected communities – the African American community and the Hispanic American community – need additional resources to help those affected by sickle cell disease.
Dr. Zahra Mahmoudjafari, Pharm.D., clinical pharmacy manager, hematology/BMT/cellular therapies, The University of Kansas Health System
- These new therapies are incredibly exciting and are recently approved for patients who do not have transplant as an option.
- They're also extremely complex. It's not it is a one-time infusion but it does take several months of treatment, mobilization, chemotherapy, and long hospital stays.
- And so with that comes an extremely large price tag. From what we understand the lifetime cost of sickle cell disease can be anywhere between $4-$6 million for patients who do have a prolonged survival.
- It is important to work with insurance companies so patients do not have to carry such financial burdens.
- The biggest thing is hope and potential for patients who've been suffering for an incredibly long time. The CRISPR technology is itself incredibly unique. It's the first of its kind to be FDA approved. And really, it's the potential for other genetic disorders to utilize the same technology.
- The researchers who looked into CRISPR technology recently won a Nobel Prize, so you know that they're doing really great and remarkable work.
Desiree Ramirez, sickle cell disease survivor
- Growing up with sickle cell disease was very difficult because she was regularly in pain, spent holidays in the hospital and battled jaundiced skin.
- In 2016, when she was 23, she received a stem cell transplant, which changed her life.
- She reassures young people going through this that there's hope. For one, there is a cure for sickle cell disease. You can get a bone marrow transplant if you have a donor, and she would tell them to remain positive and know that everything will be okay.
- Her donor wishes to remain anonymous, but she has sent letters to her through the donor program thanking her for how her donation changed her life.
Dr. Dana Hawkinson, medical director of Infection Prevention and Control, The University of Kansas Health System
- This week, the COVID count is at 23 active inpatients, up from 16 active inpatients last week.
- There's also an estimated 16 million Americans living with long COVID.
- Just last week Congress had its first ever hearing to discuss long COVID. We know it involves many different symptoms. There's a lot of different research going on right now and we're still figuring out how this condition really works.
Wednesday, January 31 at 8 a.m. is the next Open Mics with Dr. Stites. After nearly four years of the COVID pandemic, where do we stand now? The virus is still out there and vaccination rates are lagging. Hear what leading doctors across the metro are seeing inside their hospitals.
ATTENTION MEDIA: Please note access is with Microsoft Teams:
Join on your computer or mobile app
Click here to join the meeting
Meeting ID: 235 659 792 451
Passcode: 6CSfGE
Download Teams | Join on the web
Or call in (audio only)
+1 913-318-8863,566341546# United States, Kansas City
TVU Grid link: UoK_Health_SDI
Restream links: Facebook.com/kuhospital
YouTube.com/kuhospital
Send advance questions to medicalnewsnetwork@kumc.edu.