The American Sickle Cell Anemia Association (ASCAA) is an organization that provides quality and comprehensive services through diagnostic testing, evaluation, counseling and supportive services to individuals and families at-risk for Sickle Cell Disease. http://www.ascaa.org/default.asp
Sunday, November 29, 2009
Thursday, September 10, 2009
How do you manage life with sickle cell disease?
Learn what triggers, or sets off, painful events. Triggers often include cold temperatures, wind, dehydration, and too much exercise. Low oxygen caused by cigarette smoke, high altitude, and plane flights is another common trigger.
Make sure that your child takes antibiotics regularly until age 5 to prevent infections. And make sure he or she receives all the usual immunizations on schedule.
Your child can take part in normal school activities. Make sure that teachers understand your child’s special needs, like needing frequent drinks and bathroom trips and avoiding overexertion and cold temperatures.
People with sickle cell disease and their families face ongoing stress. A support network can help ease stress and worry. Ask your doctor if there is a support group in your area. You can also find Web sites on the Internet where you can ask questions, share your feelings, and get advice.
Make sure that your child takes antibiotics regularly until age 5 to prevent infections. And make sure he or she receives all the usual immunizations on schedule.
Your child can take part in normal school activities. Make sure that teachers understand your child’s special needs, like needing frequent drinks and bathroom trips and avoiding overexertion and cold temperatures.
People with sickle cell disease and their families face ongoing stress. A support network can help ease stress and worry. Ask your doctor if there is a support group in your area. You can also find Web sites on the Internet where you can ask questions, share your feelings, and get advice.
Wednesday, September 9, 2009
Ready, Set . . . STOP
Stopping regular blood transfusions in children with sickle cell disease who are at risk for a stroke means their stroke risk likely will return, researchers have found.
A study of children whose stroke risk was reduced by blood transfusions found that within a few months of halting transfusion, 14 of the 41 children resumed at-risk status and two children had strokes, said Dr. Robert J. Adams, neurologist and stroke specialist at the Medical College of Georgia who authored the article in the Dec. 29 edition of the New England Journal of Medicine. None of the 38 children who continued transfusions resumed at-risk status or had a stroke.
“We hoped that maybe we were dealing with something that was relatively short-lived over a few years in a child’s life,” said Dr. Adams. “Most of the time, that doesn’t seem to be the case. Although there were eight children in the halted-transfusion arm that were followed for more than 25 months without any apparent problem, they were the minority and we have no way to predict who they are.”
The study, headquartered at MCG and involving 25 sites in North America, was to enroll 100 patients, but the Data and Safety Monitoring Board appointed by the National Heart, Lung and Blood Institute recommended early closure in late 2004 because so many children resumed their at-risk status. The institute issued a clinical alert in December 2004 to coincide with the closure saying the 10 percent of children with sickle cell disease who have a high stroke risk need ongoing transfusions.
The institute funded the $11 million STOP II study looking at whether children needed to continue transfusions after their stroke risk normalized. That followed another NHLBI-funded study, led by MCG, which showed monthly transfusions cut stroke risk by 90 percent.
STOP II participants included patients identified at risk by abnormal blood flow studies of their brain whose studies had normalized after at least 30 months of transfusion; participants were randomized to either continue or halt transfusion.
The two strokes occurred shortly after a single abnormal transcranial Doppler study, which uses ultrasound to measure blood flow through the brain, and before confirmatory tests could be performed.
Drs. Adams and Virgil C. McKie, MCG Professor Emeritus of Pediatrics, demonstrated the painless, relatively inexpensive transcranial Doppler could identify children at risk in a New England Journal of Medicine article in 1992. Their work to identify and help at-risk children began after Dr. McKie came to Dr. Adams with concerns that some of his young patients with sickle cell disease were experiencing strokes.
End points for the STOP II study included a reversion to an abnormal transcranial Doppler or a stroke. Almost half the children in the halted transfusion arm experienced an end point within 10 months, some in as few as two months.
Dr. Adams noted data on nine patients in the halted transfusion arm were censored because they either resumed transfusion or began taking Hydroxyurea, a drug to treat the pain crises that are a hallmark of sickle cell disease.
One child who had a stroke had his first abnormal Doppler about eight months after halting transfusions and a stroke 14 days later. The second stroke occurred in a child eight days after his first abnormal study.
“I think it’s clear that some people are going to look at these findings and suggest if we do Doppler exams often enough, we can get children off transfusions,” said Dr. Adams. “But children in STOP II had frequent Doppler exams, much more frequently than likely would be used in usual clinical practice, and there were still problems.” Participants had Doppler exams at least every 12 weeks and more frequently when any abnormality was found.
Of the 209 children enrolled in the two STOP studies, 20 had strokes and all those had abnormalities on their most recent exam. This confirms the technique’s efficacy as a stroke indicator before transfusions are started and after they are stopped, according to the study.
“These results suggest that if stroke is to be prevented after transfusion is stopped, transcranial Doppler examinations must be performed at frequent intervals and transfusions resumed expeditiously,” Dr. Adams wrote.
Two children in the original STOP study had strokes despite being on transfusion, probably because their disease was so advanced when treatment began, Dr. Adams said. STOP, which followed 130 at-risk children, also was halted early because of the dramatic findings of a 90 percent reduction in stroke risk. A physician advisory issued by the NHLBI in September 1997 recommended regular transcranial Doppler studies in children age 2-16 with sickle cell disease and that transfusion be considered for those at risk.
That’s what Dr. McKie, who now works part time in the MCG Pediatric Sickle Cell Clinic, recommends.
“I’m not surprised at how effective transfusion is,” Dr. Adams said, noting that most children tended to do well on transfusion, with higher hemoglobin levels, fewer pain crises and cases of acute chest syndrome, better liver function and just generally improved quality of life.
However, transfusions are not without problems. No transfusion-related infections were reported, but nine transfusion reactions were reported in STOP II, including one that required hospitalization.
Also, iron overload, which damages vital organs, necessitated chelation therapy five nights a week for most transfusion recipients to remove excess iron. Patients and families don’t like the time-consuming and expensive therapy, Dr. McKie said, noting an oral therapy approved this year by the Food and Drug Administration may make chelation more palatable. (see article on facing page.
Many fundamental questions remain, including why these 10 percent of children have problems with stroke and how transfusion works to stop them. Whether there are better options than transfusion is another.
Dr. Adams’ research team is providing neurological and transcranial Doppler expertise for a study coordinated by St. Jude’s Children’s Research Hospital that should help determine whether Hydroxyurea is one option. The study is comparing the drug to transfusion as a way to prevent second strokes. Dr. Adams doubts Hydroxyurea will be as effective, especially in patients who already have severe abnormalities in their blood vessels. But “if this were to work in secondary prevention, it would be obvious to study it in primary prevention as well. We’ll be watching it,” Dr. Adams said. (Toni Bake http://www.mcg.edu/news/mcgtoday/Spr06/Story1.htm)
A study of children whose stroke risk was reduced by blood transfusions found that within a few months of halting transfusion, 14 of the 41 children resumed at-risk status and two children had strokes, said Dr. Robert J. Adams, neurologist and stroke specialist at the Medical College of Georgia who authored the article in the Dec. 29 edition of the New England Journal of Medicine. None of the 38 children who continued transfusions resumed at-risk status or had a stroke.
“We hoped that maybe we were dealing with something that was relatively short-lived over a few years in a child’s life,” said Dr. Adams. “Most of the time, that doesn’t seem to be the case. Although there were eight children in the halted-transfusion arm that were followed for more than 25 months without any apparent problem, they were the minority and we have no way to predict who they are.”
The study, headquartered at MCG and involving 25 sites in North America, was to enroll 100 patients, but the Data and Safety Monitoring Board appointed by the National Heart, Lung and Blood Institute recommended early closure in late 2004 because so many children resumed their at-risk status. The institute issued a clinical alert in December 2004 to coincide with the closure saying the 10 percent of children with sickle cell disease who have a high stroke risk need ongoing transfusions.
The institute funded the $11 million STOP II study looking at whether children needed to continue transfusions after their stroke risk normalized. That followed another NHLBI-funded study, led by MCG, which showed monthly transfusions cut stroke risk by 90 percent.
STOP II participants included patients identified at risk by abnormal blood flow studies of their brain whose studies had normalized after at least 30 months of transfusion; participants were randomized to either continue or halt transfusion.
The two strokes occurred shortly after a single abnormal transcranial Doppler study, which uses ultrasound to measure blood flow through the brain, and before confirmatory tests could be performed.
Drs. Adams and Virgil C. McKie, MCG Professor Emeritus of Pediatrics, demonstrated the painless, relatively inexpensive transcranial Doppler could identify children at risk in a New England Journal of Medicine article in 1992. Their work to identify and help at-risk children began after Dr. McKie came to Dr. Adams with concerns that some of his young patients with sickle cell disease were experiencing strokes.
End points for the STOP II study included a reversion to an abnormal transcranial Doppler or a stroke. Almost half the children in the halted transfusion arm experienced an end point within 10 months, some in as few as two months.
Dr. Adams noted data on nine patients in the halted transfusion arm were censored because they either resumed transfusion or began taking Hydroxyurea, a drug to treat the pain crises that are a hallmark of sickle cell disease.
One child who had a stroke had his first abnormal Doppler about eight months after halting transfusions and a stroke 14 days later. The second stroke occurred in a child eight days after his first abnormal study.
“I think it’s clear that some people are going to look at these findings and suggest if we do Doppler exams often enough, we can get children off transfusions,” said Dr. Adams. “But children in STOP II had frequent Doppler exams, much more frequently than likely would be used in usual clinical practice, and there were still problems.” Participants had Doppler exams at least every 12 weeks and more frequently when any abnormality was found.
Of the 209 children enrolled in the two STOP studies, 20 had strokes and all those had abnormalities on their most recent exam. This confirms the technique’s efficacy as a stroke indicator before transfusions are started and after they are stopped, according to the study.
“These results suggest that if stroke is to be prevented after transfusion is stopped, transcranial Doppler examinations must be performed at frequent intervals and transfusions resumed expeditiously,” Dr. Adams wrote.
Two children in the original STOP study had strokes despite being on transfusion, probably because their disease was so advanced when treatment began, Dr. Adams said. STOP, which followed 130 at-risk children, also was halted early because of the dramatic findings of a 90 percent reduction in stroke risk. A physician advisory issued by the NHLBI in September 1997 recommended regular transcranial Doppler studies in children age 2-16 with sickle cell disease and that transfusion be considered for those at risk.
That’s what Dr. McKie, who now works part time in the MCG Pediatric Sickle Cell Clinic, recommends.
“I’m not surprised at how effective transfusion is,” Dr. Adams said, noting that most children tended to do well on transfusion, with higher hemoglobin levels, fewer pain crises and cases of acute chest syndrome, better liver function and just generally improved quality of life.
However, transfusions are not without problems. No transfusion-related infections were reported, but nine transfusion reactions were reported in STOP II, including one that required hospitalization.
Also, iron overload, which damages vital organs, necessitated chelation therapy five nights a week for most transfusion recipients to remove excess iron. Patients and families don’t like the time-consuming and expensive therapy, Dr. McKie said, noting an oral therapy approved this year by the Food and Drug Administration may make chelation more palatable. (see article on facing page.
Many fundamental questions remain, including why these 10 percent of children have problems with stroke and how transfusion works to stop them. Whether there are better options than transfusion is another.
Dr. Adams’ research team is providing neurological and transcranial Doppler expertise for a study coordinated by St. Jude’s Children’s Research Hospital that should help determine whether Hydroxyurea is one option. The study is comparing the drug to transfusion as a way to prevent second strokes. Dr. Adams doubts Hydroxyurea will be as effective, especially in patients who already have severe abnormalities in their blood vessels. But “if this were to work in secondary prevention, it would be obvious to study it in primary prevention as well. We’ll be watching it,” Dr. Adams said. (Toni Bake http://www.mcg.edu/news/mcgtoday/Spr06/Story1.htm)
Sunday, August 30, 2009
Iron Overload and Sickle Cell
Some people with sickle cell disease have blood transfusions to help reduce complications of the disease. Although transfusions can help improve the quality of life for people with sickle cell disease, they add extra iron to the body. Because the extra iron does not go away by itself, it can build up and overload the body. People with sickle cell disease who've had 10 or more transfusions in a lifetime are at risk for iron overload. And iron overload can damage the heart, liver, and other organs.
The good news is that a simple blood test called a serum ferritin test can help you find out if you have iron overload. Ask your doctor if the serum ferritin test is right for you. Then be sure to talk with your doctor about iron overload, your risk, screening and diagnosis, and how to manage iron overload.
If you continue to have blood transfusions, remember that it's important to stay on track with monitoring to be sure your iron levels are where they should be. Ask your doctor when you should be tested to find out if your iron levels have changed.
The good news is that a simple blood test called a serum ferritin test can help you find out if you have iron overload. Ask your doctor if the serum ferritin test is right for you. Then be sure to talk with your doctor about iron overload, your risk, screening and diagnosis, and how to manage iron overload.
If you continue to have blood transfusions, remember that it's important to stay on track with monitoring to be sure your iron levels are where they should be. Ask your doctor when you should be tested to find out if your iron levels have changed.
Wednesday, August 26, 2009
Bone Marrow Transplant Centers for Sickle Cell Patients
1. Atlanta, GA: Emory University and Grady Memorial Hospital aplatt@emory.edu
2. Augustin, GR: University of Bonn
3. Birmingham, UK: The Birmingham Children's Hospital NHS
4. Boston, MA: Dana Farber Cancer Institute, Children's Hospital, Harvard University, Boston Comprehensive Sickle Center
5. Chapel Hill, NC: University of North Carolina
6. Chicago, IL: University of Illinois
7. Columbia, NC: Richland Memorial Hospital
8. Creteil FR: Hospital Henri Mondor
9. Dallas, TX: University of Texas, Southwestern Medical Center at Dallas
10. Denver, CO: University of Colorado, Sickle Cell Research & Treatment Center
11. Durham, NC: Duke University
12. Gainesville, FL: University of Florida; Shands Teaching Hospital
13. Hackensack, NY: Hackensack Medical Center
14. Houston, TX: MD Anderson Cancer, University of Texas
15. Indianapolis, IN: Indiana University
16. London, UK: Royal Postgraduate Medical School; Hammersmith Hospital
17. Los Angeles, CA: University of Southern California, Children's Hospital of LA
18. Memphis TN - Paul Woodard, MD Hematology/Oncology Division of Stem Cell Transplantation St. Jude Children's Research Hospital Memphis, TN 38105-2794,~ (901) 495-4239 Fax (901) 521-9005
19. Miami, FL: University of Southern Florida
20. Milwaukee, WI: Medical College of Wisconsin, Midwest Children's Cancer Center
21. New Haven, CT: Yale University
22. New Orleans, LA: Tulane University
23. New York, NY: Methodist Hospital
24. Oakland, CA: Children's Hospital of Oakland
25. Philadelphia, PA: Children's Hospital of Philadelphia, University of PA
26. St. Louis, MO: Cardinal Glennon Children's Hospital, University of St. Louis
27. St. Petersburg FL: University of South Florida, All Children's Hospital
28. Sao Paulo, BR: University of Campinas
29. San Francisco, CA: University of California at San Francisco, San Francisco General Hospital, Comprehensive Sickle Center
30. Seattle, WA: University of Washington, Fred Hutchinson Cancer Research Center
31. Stanford, CA: Stanford University
32. Toronto, ON: Hospital for Sick Children
33. Washington, DC: Children's Hospital National Medical Center, George Washington University, Howard University
2. Augustin, GR: University of Bonn
3. Birmingham, UK: The Birmingham Children's Hospital NHS
4. Boston, MA: Dana Farber Cancer Institute, Children's Hospital, Harvard University, Boston Comprehensive Sickle Center
5. Chapel Hill, NC: University of North Carolina
6. Chicago, IL: University of Illinois
7. Columbia, NC: Richland Memorial Hospital
8. Creteil FR: Hospital Henri Mondor
9. Dallas, TX: University of Texas, Southwestern Medical Center at Dallas
10. Denver, CO: University of Colorado, Sickle Cell Research & Treatment Center
11. Durham, NC: Duke University
12. Gainesville, FL: University of Florida; Shands Teaching Hospital
13. Hackensack, NY: Hackensack Medical Center
14. Houston, TX: MD Anderson Cancer, University of Texas
15. Indianapolis, IN: Indiana University
16. London, UK: Royal Postgraduate Medical School; Hammersmith Hospital
17. Los Angeles, CA: University of Southern California, Children's Hospital of LA
18. Memphis TN - Paul Woodard, MD Hematology/Oncology Division of Stem Cell Transplantation St. Jude Children's Research Hospital Memphis, TN 38105-2794,~ (901) 495-4239 Fax (901) 521-9005
19. Miami, FL: University of Southern Florida
20. Milwaukee, WI: Medical College of Wisconsin, Midwest Children's Cancer Center
21. New Haven, CT: Yale University
22. New Orleans, LA: Tulane University
23. New York, NY: Methodist Hospital
24. Oakland, CA: Children's Hospital of Oakland
25. Philadelphia, PA: Children's Hospital of Philadelphia, University of PA
26. St. Louis, MO: Cardinal Glennon Children's Hospital, University of St. Louis
27. St. Petersburg FL: University of South Florida, All Children's Hospital
28. Sao Paulo, BR: University of Campinas
29. San Francisco, CA: University of California at San Francisco, San Francisco General Hospital, Comprehensive Sickle Center
30. Seattle, WA: University of Washington, Fred Hutchinson Cancer Research Center
31. Stanford, CA: Stanford University
32. Toronto, ON: Hospital for Sick Children
33. Washington, DC: Children's Hospital National Medical Center, George Washington University, Howard University
Questions and Answers on Bone Marrow and Cord Blood Stem Cell Transplants
Question: How is the Cord Blood Stem Cell transplant like Bone Marrow transplant?
Answer: The preparation considerations are similar. The Cord Blood Stem Cell donor is unrelated and is an alternative for patient without a brother or sister match. The cost, time, and follow-up are similar. However, the risks of graft versus host disease and graft rejection are higher, because the immunologic matching for an unrelated donor is never quite as perfect as with a matched relative donor.
Question: What are the cost and the steps to consider for bone marrow transplant?
Answer: Evaluation for Bone Marrow Transplantation is a long journey, because the risks and expenses are large. Here is an outline of what we would need to do to evaluate a child for possible bone marrow transplantation. We would certainly plan for many hours of discussion if you are interested in proceeding, but I wanted to make sure that you have a rough idea of the stepwise process:
1. SICKLE CELL ELIGIBILITY -- determine whether your son has had severe enough sickle cell disease to make the risks of bone marrow transplant worthwhile. A summary of his medical history would be very helpful, focusing on whether he has had stroke and chronic transfusion, or very frequent hospital stays for pain or for lung problems, or other major sickle cell problems. This can be done by fax or e-mail.
2. OTHER MEDICAL ISSUES -- You should make your doctor aware that your family is considering the bone marrow transplant (BMT) option. Ask the doctor to let us know whether there are any hidden medical problems that would influence the decision, such as chronic viral or other infections, problems with transfusion reactions, other medical problems unrelated to sickle cell, or anything else unusual. This does not have to be a lengthy or formal statement from the doctor at this point, more like a safety check to make sure that we are not missing any huge medical issues. A more complete medical record would be required later.
3. HLA-TYPING BLOOD TESTS -- Do immunologic typing (HLA typing) of your child and the relative with the highest probability of matching him - only brothers or sisters from the same parents are really potential donors, parents and half-siblings are very unlikely to match unless there was an unusual family tree. The HLA typing will cost several thousand dollars. If the sibling is not a full HLA match, then it is very unlikely that BMT can be done.
4. PRE-BMT EVALUATION -- after all of the above steps, then a formal evaluation by the BMT team can begin. This will include a very detailed look at his medical history and current medical condition from head to toe, plus your family's ability to cope with the BMT process. Parents will need to plan to stay in the Atlanta with the patient for a minimum of 8 months for the pre-transplant evaluation, transplant stay, and post-transplant follow-up. A financial arrangement will need to be made, with an estimated cost for the BMT process of $150,000 to $250,000. An outside expert panel will review the case on ethical grounds.
5. BMT & early follow-up period - This is a risky process, and the statistics are that there is a 5% to 8% chance of death. Death can be caused by infection, bleeding, toxic effects of the treatment, or the new bone marrow engrafting and then attacking the rest of the body. There is also a 10% to 12% chance that the child could go through the BMT process but reject the new bone marrow, ending up still having sickle cell disease. Therefore, the overall success rate of BMT for sickle cell disease is approximately 80% to 85%, of being cured of sickle cell. These require close medical follow-up, often in and out of the hospital and office daily, and many medications daily.
Question: How is the Cord Blood Stem Cell transplant like Bone Marrow Transplant?
Answer: The preparation considerations are similar. The Cord Blood Stem Cell donor is unrelated and is an alternative for patient without a brother or sister match. The cost, time, and follow-up are similar.
Question: I have a daughter that has sickle cell. If she would have a step sibling could she receive a bone narrow transplant if they match? What risk would it be if they do match and her body rejects the transplant?
Answer: The main concerns with sickle cell bone marrow transplantation are
a. the immunologic match between donor and recipient (HLA types) and
b. the health status of your daughter.
1. A full HLA match between brothers or sisters will have the very best chances for successful bone marrow transplant (BMT). Lesser degrees of match means greater chances of two bad outcomes:
a. Graft Rejection (your child goes through the BMT process but at the end her own bone marrow grows back and she still has sickle cell disease) or
b. Graft Versus Host Disease (GVHD - the transplanted marrow attacks the rest of your child's body as foreign tissue and can cause great damage.
c. very seldom will a person have a full HLA match with half-siblings or parents, unless the family tree is very inbred (for example, everyone is from the same isolated village or clan and all are related to each other's cousins. Therefore, your child's step-sister would not have a high chance of being an HLA-matched donor for BMT.
2. In addition to the chances of the two types of problems listed above (Graft Rejection and GVHD), there is a third set of bad problems that are side effects of the harsh BMT treatment process. Death may occur due to overwhelming infection, uncontrollable bleeding, and failure of organs such as liver or kidney or lungs. The chances that these bad side effects will occur are probably greater if your child is in worse health going into transplant. Therefore, the general feeling among sickle cell doctors in North and South America is that the only patients with severe sickle cell complications should be offered BMT (because only then are the high risks worthwhile), but that they be in relatively good physical condition. Your child may or may not meet these eligibility criteria.
Answer: The preparation considerations are similar. The Cord Blood Stem Cell donor is unrelated and is an alternative for patient without a brother or sister match. The cost, time, and follow-up are similar. However, the risks of graft versus host disease and graft rejection are higher, because the immunologic matching for an unrelated donor is never quite as perfect as with a matched relative donor.
Question: What are the cost and the steps to consider for bone marrow transplant?
Answer: Evaluation for Bone Marrow Transplantation is a long journey, because the risks and expenses are large. Here is an outline of what we would need to do to evaluate a child for possible bone marrow transplantation. We would certainly plan for many hours of discussion if you are interested in proceeding, but I wanted to make sure that you have a rough idea of the stepwise process:
1. SICKLE CELL ELIGIBILITY -- determine whether your son has had severe enough sickle cell disease to make the risks of bone marrow transplant worthwhile. A summary of his medical history would be very helpful, focusing on whether he has had stroke and chronic transfusion, or very frequent hospital stays for pain or for lung problems, or other major sickle cell problems. This can be done by fax or e-mail.
2. OTHER MEDICAL ISSUES -- You should make your doctor aware that your family is considering the bone marrow transplant (BMT) option. Ask the doctor to let us know whether there are any hidden medical problems that would influence the decision, such as chronic viral or other infections, problems with transfusion reactions, other medical problems unrelated to sickle cell, or anything else unusual. This does not have to be a lengthy or formal statement from the doctor at this point, more like a safety check to make sure that we are not missing any huge medical issues. A more complete medical record would be required later.
3. HLA-TYPING BLOOD TESTS -- Do immunologic typing (HLA typing) of your child and the relative with the highest probability of matching him - only brothers or sisters from the same parents are really potential donors, parents and half-siblings are very unlikely to match unless there was an unusual family tree. The HLA typing will cost several thousand dollars. If the sibling is not a full HLA match, then it is very unlikely that BMT can be done.
4. PRE-BMT EVALUATION -- after all of the above steps, then a formal evaluation by the BMT team can begin. This will include a very detailed look at his medical history and current medical condition from head to toe, plus your family's ability to cope with the BMT process. Parents will need to plan to stay in the Atlanta with the patient for a minimum of 8 months for the pre-transplant evaluation, transplant stay, and post-transplant follow-up. A financial arrangement will need to be made, with an estimated cost for the BMT process of $150,000 to $250,000. An outside expert panel will review the case on ethical grounds.
5. BMT & early follow-up period - This is a risky process, and the statistics are that there is a 5% to 8% chance of death. Death can be caused by infection, bleeding, toxic effects of the treatment, or the new bone marrow engrafting and then attacking the rest of the body. There is also a 10% to 12% chance that the child could go through the BMT process but reject the new bone marrow, ending up still having sickle cell disease. Therefore, the overall success rate of BMT for sickle cell disease is approximately 80% to 85%, of being cured of sickle cell. These require close medical follow-up, often in and out of the hospital and office daily, and many medications daily.
Question: How is the Cord Blood Stem Cell transplant like Bone Marrow Transplant?
Answer: The preparation considerations are similar. The Cord Blood Stem Cell donor is unrelated and is an alternative for patient without a brother or sister match. The cost, time, and follow-up are similar.
Question: I have a daughter that has sickle cell. If she would have a step sibling could she receive a bone narrow transplant if they match? What risk would it be if they do match and her body rejects the transplant?
Answer: The main concerns with sickle cell bone marrow transplantation are
a. the immunologic match between donor and recipient (HLA types) and
b. the health status of your daughter.
1. A full HLA match between brothers or sisters will have the very best chances for successful bone marrow transplant (BMT). Lesser degrees of match means greater chances of two bad outcomes:
a. Graft Rejection (your child goes through the BMT process but at the end her own bone marrow grows back and she still has sickle cell disease) or
b. Graft Versus Host Disease (GVHD - the transplanted marrow attacks the rest of your child's body as foreign tissue and can cause great damage.
c. very seldom will a person have a full HLA match with half-siblings or parents, unless the family tree is very inbred (for example, everyone is from the same isolated village or clan and all are related to each other's cousins. Therefore, your child's step-sister would not have a high chance of being an HLA-matched donor for BMT.
2. In addition to the chances of the two types of problems listed above (Graft Rejection and GVHD), there is a third set of bad problems that are side effects of the harsh BMT treatment process. Death may occur due to overwhelming infection, uncontrollable bleeding, and failure of organs such as liver or kidney or lungs. The chances that these bad side effects will occur are probably greater if your child is in worse health going into transplant. Therefore, the general feeling among sickle cell doctors in North and South America is that the only patients with severe sickle cell complications should be offered BMT (because only then are the high risks worthwhile), but that they be in relatively good physical condition. Your child may or may not meet these eligibility criteria.
Monday, August 24, 2009
Understanding Sickle Cell Anemia
Sickle cell disease is a group of disorders that affects hemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. People with this disorder have atypical hemoglobin molecules called hemoglobin S, which can change the shape of normal red blood cells into a sickle, or crescent, shape.
Signs and symptoms of sickle cell disease usually begin in early childhood. Characteristics of this disorder include a low number of red blood cells (anemia), repeated infections, and recurring episodes of pain. The severity of symptoms varies from person to person. Some people have mild symptoms, while others are frequently hospitalized for more serious complications.
The signs and symptoms of sickle cell disease are caused by the sickling of red blood cells. When red blood cells sickle, they break down prematurely, which can lead to anemia. Anemia can cause shortness of breath, fatigue, and delayed growth and development in children. The rapid breakdown of red blood cells may also cause yellowing of the eyes and skin, which are signs of jaundice.
Painful episodes can occur when sickled red blood cells, which are stiff and inflexible, get stuck in small blood vessels. These episodes deprive tissues and organs of oxygen-rich blood and can lead to organ damage, especially in the lungs, kidneys, spleen, and brain. A particularly serious complication of sickle cell disease is high blood pressure in the blood vessels that supply the lungs (pulmonary hypertension).
Pulmonary hypertension occurs in about one-third of adults with sickle cell disease and can lead to heart failure.
Signs and symptoms of sickle cell disease usually begin in early childhood. Characteristics of this disorder include a low number of red blood cells (anemia), repeated infections, and recurring episodes of pain. The severity of symptoms varies from person to person. Some people have mild symptoms, while others are frequently hospitalized for more serious complications.
The signs and symptoms of sickle cell disease are caused by the sickling of red blood cells. When red blood cells sickle, they break down prematurely, which can lead to anemia. Anemia can cause shortness of breath, fatigue, and delayed growth and development in children. The rapid breakdown of red blood cells may also cause yellowing of the eyes and skin, which are signs of jaundice.
Painful episodes can occur when sickled red blood cells, which are stiff and inflexible, get stuck in small blood vessels. These episodes deprive tissues and organs of oxygen-rich blood and can lead to organ damage, especially in the lungs, kidneys, spleen, and brain. A particularly serious complication of sickle cell disease is high blood pressure in the blood vessels that supply the lungs (pulmonary hypertension).
Pulmonary hypertension occurs in about one-third of adults with sickle cell disease and can lead to heart failure.
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