Lewis Hsu, MD, PhD Pediatric Hematologist
Below is a post that answers questions we are frequently asked, so we thought we'd post it here.
Ask the Experts
Question:
My child needs a bone marrow transplant, but there are no matched donors in the family. What can be done?
Answer:
There are experimental protocols offering transplant from alternative donors, which are higher risk than transplant from an HLA-matched sibling. The family needs to be fully aware that the risks of these alternative transplants include death, chronic graft vs host disease, graft rejection (go through all the procedure and end up still with sickle cell disease), and reproductive sterility. Usually the eligibility criteria are: stroke or silent brain infarct and chronic transfusion, recurrent acute chest syndrome (3 episodes) or recurrent hospitalizations for pain (3/yr) despite hydroxyurea and other interventions. Some protocols are only for children under 17yrs.
Different protocols have alternative donors such as HLA-matched unrelated cord blood, partially HLA-matched family member, or HLA-matched unrelated donor. Different preparative regimens are used to combine immunosuppression and bone marrow suppression. Medical compliance is essential and the family needs to set aside all other obligations to follow the pre-transplant evaluation and the post-transplant period can involve very intense medical care for 6 - 24 months... this is a level of intensity more similar to cancer care than to sickle cell care. The child will have to take multiple medications for several months, central line for medications and nutrition, and may have 3 clinic visits a week and frequent hospitalizations. Psychosocial stability and family support are extremely important -- this will be a challenge for a family from Savannah to get a transplant someplace else. However, if they are interested, the family should ask the child's hematologist about making a referral for consultation at a pediatric bone marrow transplant center Emory, Duke, and Jacksonville are relatively close to your patient in Savannah. The transplanters at St. Jude in Memphis, TN, and National Institutes of Health (Dr. John Tisdale) also have sickle cell transplant protocols and often conduct consultations for patients from far away.
Transplantation is the only sickle cell treatment offering the possibility of complete cure from sickle cell disease. However, there are real risks involved, and the alternative donor protocols have higher risks than the HLA-matched-sibling donor transplants. For a patient and family to take on these risks requires considerable discussion to make sure that they understand the risks and their responsibility for medical compliance.
Sincerely,
-Lewis Hsu, MD, PhD
Pediatric Hematologist
Ask the Experts
Question:
My child needs a bone marrow transplant, but there are no matched donors in the family. What can be done?
Answer:
There are experimental protocols offering transplant from alternative donors, which are higher risk than transplant from an HLA-matched sibling. The family needs to be fully aware that the risks of these alternative transplants include death, chronic graft vs host disease, graft rejection (go through all the procedure and end up still with sickle cell disease), and reproductive sterility. Usually the eligibility criteria are: stroke or silent brain infarct and chronic transfusion, recurrent acute chest syndrome (3 episodes) or recurrent hospitalizations for pain (3/yr) despite hydroxyurea and other interventions. Some protocols are only for children under 17yrs.
Different protocols have alternative donors such as HLA-matched unrelated cord blood, partially HLA-matched family member, or HLA-matched unrelated donor. Different preparative regimens are used to combine immunosuppression and bone marrow suppression. Medical compliance is essential and the family needs to set aside all other obligations to follow the pre-transplant evaluation and the post-transplant period can involve very intense medical care for 6 - 24 months... this is a level of intensity more similar to cancer care than to sickle cell care. The child will have to take multiple medications for several months, central line for medications and nutrition, and may have 3 clinic visits a week and frequent hospitalizations. Psychosocial stability and family support are extremely important -- this will be a challenge for a family from Savannah to get a transplant someplace else. However, if they are interested, the family should ask the child's hematologist about making a referral for consultation at a pediatric bone marrow transplant center Emory, Duke, and Jacksonville are relatively close to your patient in Savannah. The transplanters at St. Jude in Memphis, TN, and National Institutes of Health (Dr. John Tisdale) also have sickle cell transplant protocols and often conduct consultations for patients from far away.
Transplantation is the only sickle cell treatment offering the possibility of complete cure from sickle cell disease. However, there are real risks involved, and the alternative donor protocols have higher risks than the HLA-matched-sibling donor transplants. For a patient and family to take on these risks requires considerable discussion to make sure that they understand the risks and their responsibility for medical compliance.
Sincerely,
-Lewis Hsu, MD, PhD
Pediatric Hematologist
posted by MarkAndSusan at 3:22 PM
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