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Bone marrow transplants offer cure for sickle cell patients

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Posted: Thursday, July 5, 2012 12:06 am | Updated: 10:17 am, Thu Jul 5, 2012.

Little Gabby Carter of Cape Girardeau, Missouri thought going to the hospital all the time, frequent bouts of pain, staying inside during temperature extremes and fatigue were just a normal part of life. 

That is, until she started kindergarten.

“Kindergarteners have a lot of recesses and they do a lot of things that she couldn’t do,” Debbie Carter, Gabby’s mother, said. “She got tired a lot with the activities that they do; she just couldn’t keep up and so she’d have to take frequent naps.”

Gabby is one of the patients at St. Louis Children’s Hospital with sickle cell disease who are or will become stem cell or the bone marrow transplant recipients this summer.

“We have three that are in right now; we have one that has already had her transplant and is in the early phase of recovery,” Dr. Monica Hulbert, director of the Sickle Cell Disease Program at St. Louis Children’s Hospital and assistant professor of pediatrics Washington University St. Louis.

Sickle cell disease is an inherited blood disorder affecting millions of people of color around the globe. Varying types of sickle cell diseases are commonly found among African, Indian, Mediterranean, Middle-eastern, Caribbean and Latin populations.

With sickle cell disease, red blood cells produce abnormal hemoglobin, a protein that carries oxygen throughout the body and takes carbon dioxide to the lungs to breathe out. Normally rounded red blood cells are crescent or sickle shaped, reducing their ability to transport oxygen throughout the body. The sickled cells can group together and clog pathways in the bloodstream, causing painful attacks, known as episodes or crises. The disease can damage vital organs and cause strokes and premature death.

In Missouri, it is estimated one in 400 African Americans have the disease and one in 12 have the sickle cell trait, which can be passed to their children.  If both parents have the sickle cell trait, there is a one in four chance their child will also have sickle cell anemia.

Hulbert’s colleague, Dr. Shalini Shenoy, associate director of the bone marrow transplant program, has developed and is chair of several national studies to figure out ways to make bone marrow transplants available for more patients and with less side effects.

“It’s really a great opportunity to kind of see that unfold and we have multiple patients coming into the transplant unit this summer for hopefully what will be curative treatment for their sickle cell disease,” Hulbert said.

As exciting as that is for the patients, their families and their health care team, many other children need a bone marrow match.

“We have 15 or 20 kids that are waiting with no donors,” Hulbert said.

To become a bone marrow donor, persons need to sign up through BeTheMatch.org to get tested.

“To register to be a donor it requires just having a condensed swab of your cheek and filling out a bunch of forms and you could be called at any time if there was somebody who matched you – to be willing to donate for any person that you may not know, and you won’t know, most likely,” Hulbert said.

People are more likely to match somebody who is more genetically like them, making it more difficult to find acceptable matches for African Americans and any person of color, she said.

“There are not as many people of color signed up to be donors,” Hulbert said. “And that’s true not just for sickle cell disease but for other diseases that can affect anybody – like leukemia, aplastic anemia or bone marrow failure. We really need people to register to be donors for any patient that may need a bone marrow transplant.”

The hospital is also working with the St. Louis Cord Blood Bank to try and identify ways to improve cord blood donations from women of color after their babies are born.

“The cord blood contains a lot of the elements we need that are called stem cells, which can actually kind of repopulate somebody’s bone marrow and those can be what we call an alternative source instead of a bone marrow donor,” Hulbert said.  “Cord blood can be for somebody to have a transplant for sickle cell or any other similar blood disease.”

After going on the bone marrow transplant list in March 2011, two matching bone marrow donors did not work out - one because of medical reasons and the other donor just backed out. Gabby’s mother said she didn’t have time to become angry or bitter. The medical team pursued and found another option – a cord blood match for a stem cell transplant.

 Gabby underwent two rounds of chemotherapy at Children’s before having a cord blood stem cell transplant on July 3.

The National Institutes of Health researchers have also reported successful results in the U.S. with stem cell transplants to adult sickle cell patients, although risks for rejection are higher with age and after experiencing major sickle cell complications.

Children’s will have an informational booth at the upcoming Sickle Cell Stroll, which takes place Saturday, July 28 at 9 a.m. in the Upper Muny Parking Lot in Forest Park in St. Louis. To register or for more information, call 314-277-3950, email rbritts@sicklecellassociation.org or visit sicklecellstroll.com.

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1 comment:

  • Magalhães posted at 1:39 pm on Mon, Aug 20, 2012.

    Magalhães Posts: 1

    I'm happy when I see the progress and cure for sickle cell anemia. I did a bone marrow transplant in 2005 and was cured. We have much to thank these researchers and physicians who care for us. Elvis Silva Magalhães
    http://www.youtube.com/watch?v=lVSVt9cqiQk&feature=plcp