A Less Intensive Type Of Bone Marrow Transplant Improves Outcomes For Adults With Severe Sickle Cell Disease

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A Less Intensive Type Of Bone Marrow Transplant Improves Outcomes For Adults With Severe Sickle Cell Disease

INTRO: A standard bone marrow transplant is a potential treatment option for patients with severe sickle cell disease. However, that procedure has only been done in children with sickle cell. A new study examined whether a less intensive and safer type of bone marrow transplant could improve outcomes for adults with sickle cell disease. Catherine Dolf has more in this week’s JAMA Report.

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SOT/FULL Super@:02 Juliana Ejedoghaobi – Sickle Cell Patient Runs:08
“Imagine someone taking a stick and just like stabbing like you know hitting you on your joints, that’s how painful it is.”

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Pictures of Juliana in the hospital, walking down hallway, Graphic: Human body with blood flowing through vessels, normal red blood cells, sickle cells, joints, lungs and brain radiating, normal red blood cells

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THAT PAIN CAUSED JULIANA EJEDOGHAOBI (Edge-ah-DOE-bay) TO SPEND A LOT OF TIME IN THE HOSPITAL. SHE WAS DIAGNOSED WITH SICKLE CELL AT AGE 2. IT’S A GENETIC DISORDER, WHERE NORMAL, DONUT SHAPED RED BLOOD CELLS BECOME SICKLE OR BANANA SHAPED CELLS. THESE “SICKLE” CELLS HAVE A DIFFICULT TIME PASSING THROUGH THE BLOOD VESSELS. THEY CAN CLOG THE JOINTS CAUSING BONE PAIN, IN THE LUNGS, DIFFICULTY BREATHING AND PNEUMONIA, AND IN THE BRAIN, STROKE.  A BONE MARROW TRANSPLANT IS THE ONLY WAY TO CURE THE DISEASE.

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SOT/FULL Super@:34 Matthew M. Hsieh, M.D., – National Institutes of Health Runs:06
“The standard transplant usually involves full doses of chemotherapy and or radiation.”

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SOT/FULL Super@:39 John F. Tisdale, M.D., – National Institutes of Health Runs:09
“Most adults with sickle cell disease have organ damage that would prevent them from being able to get a standard bone marrow transplant.”

(Video covering middle of bite: Dr. Tisdale and Dr. Hsieh talking with Juliana)

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Dr. Tisdale and Dr. Hsieh walking into lab, looking into microscope, sickle cell slide, talking with Juliana

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DOCTORS JOHN TISDALE AND MATTHEW HSIEH (SHAY) FROM THE NATIONAL INSTITUTES OF HEALTH AND CO-AUTHORS TESTED A REGIMEN THAT DIDN’T REQUIRE DESTROYING ALL OF AN ADULT PATIENT’S BONE MARROW. 30 PATIENTS WHO HAD A SIBLING WITH MATCHING IMMUNE CHARACTERISTICS RECEIVED THE LESS INTENSIVE TRANSPLANT.

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SOT/FULL Super@ 1:06 Matthew M. Hsieh, M.D., – National Institutes of Health Runs:16
“Therapies for sickle cell disease are only very few, there is only one drug that’s available to treat. The remainder of the therapy includes blood transfusions in whom these two therapies don’t work other approaches are needed.”

(Video covering 1st and last part of bite: Dr. Hsieh looking in microscope)

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JAMA COVER

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THE STUDY APPEARS IN JAMA, JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION.

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SOT/FULL Super@ 1:23 John F. Tisdale, M.D., – National Institutes of Health Runs:18
“Transplantation was successful in 26 of the 30 patients. Using this less toxic way of preparing a patient for a bone marrow transplant had success rates that are comparable to the more toxic kind of transplant that’s performed in children.”

(Video covering middle of bite: Juliana and doctors talking in exam room)

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Picture of Juliana and her family

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JULIANA RECEIVED BONE MARROW FROM HER OLDER SISTER.

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SOT/FULL Super@ 1:45 Juliana Ejedoghaobi – Sickle Cell Patient Runs:07
“It changed my life. Yeah, I do get pain but it’s not compared to what I used to have before.”

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Pics of her and her parents, her working as a nurse

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FOLLOWING HER PASSION, JULIANA IS NOW A NURSE WITH BIG PLANS FOR HER FUTURE.

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SOT/FULL Super@1:55 Voice of:Juliana Ejedoghaobi – Sickle Cell Patient Runs:06
“I’m just looking forward to just living life, being normal, getting married and just having my own family.”

(Video covering entire bite: Juliana and doctors talking in exam room)

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Juliana and doctors talking in exam room

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CATHERINE DOLF, THE JAMA REPORT.

TAG:RESEARCHERS ALSO REPORT THAT A COMMON TRANSPLANT COMPLICATION KNOWN AS GRAFT VERUS HOST DISEASE, DID NOT HAPPEN IN ANY OF STUDY PARTICIPANTS AND AFTER THE TRANSPLANT 15 PATIENTS STOPPED TAKING THEIR IMMUNOSUPPRESSION MEDICATION.