Bone marrow transplantation for sickle cell disease.

@article{Walters1996BoneMT,
  title={Bone marrow transplantation for sickle cell disease.},
  author={Mark C. Walters and Melinda Patience and Wendy M. Leisenring and James R. Eckman and J. Paul Scott and William C. Mentzer and Sally C. Davies and Kwaku Ohene-Frempong and Françoise Bernaudin and Dana C. Matthews and Rainer F Storb and Keith M. Sullivan},
  journal={The New England journal of medicine},
  year={1996},
  volume={335 6},
  pages={
          369-76
        }
}
BACKGROUND We investigated the risks and benefits of allogeneic bone marrow transplantation in children with complications of sickle cell disease. METHODS Twenty-two children less than 16 years of age who had symptomatic sickle cell disease received marrow allografts from HLA-identical siblings between September 1991 and April 1995. The indications for transplantation included a history of stroke (n = 12), recurrent acute chest syndrome (n = 5), and recurrent painful crises (n = 5). Patients… Expand
Impact of bone marrow transplantation for symptomatic sickle cell disease: an interim report. Multicenter investigation of bone marrow transplantation for sickle cell disease.
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Data confirm that allogenic bone marrow transplantation establishes normal erythropoiesis and is associated with improved growth and stable CNS imaging and pulmonary function in most patients. Expand
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TLDR
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TLDR
The best results are obtained in young children who have HLA-identical sibling donors and are transplanted early in the course of the disease (DFS: 93%). Expand
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TLDR
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TLDR
Allogeneic SCT after RIC is feasible in adult patients with sickle cell disease and mixed chimerism is sufficient to relieve disease-related symptoms and is possibly correlated with less acute GVHD. Expand
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TLDR
Nonmyeloablative conditioning followed by a T-cell-depleted hematopoietic stem-cell allograft is a feasible option for patients with chronic granulomatous disease, recurrent life-threatening infections, and an HLA-identical family donor. Expand
Blood and marrow transplantation for sickle cell disease: is less more?
TLDR
The results obtained when bone marrow transplants are used to treat sickle cell disease are focused on and non-myeloablation could be the preferred type of conditioning and donor availability will not be a barrier anymore to proceed to transplant. Expand
Allogeneic hematopoietic stem cell transplantation for sickle cell disease: the time is now.
TLDR
Results indicate that nonmyeloablative allo-HSCT in adult patients with SCD allows for stable mixed hematopoietic chimerism with associated full-donor erythroid engraftment and normalization of blood counts, and persistence in some without continued immunosuppression suggests immunologic tolerance. Expand
[Bone marrow transplant in patients with sickle cell anaemia. Experience in one centre].
TLDR
There was a transplant related mortality in the study, consistent with multicentre studies, and with aGVHD being the main cause, although the permanent sequelae are mild. Expand
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References

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TLDR
All patients with sickle cell disease treated by matched sibling bone marrow transplantation in the United States are surviving in good to excellent clinical condition and appear to have benefitted from treatment by bone marrow transplants. Expand
Bone marrow transplantation for sickle cell disease. The European experience.
TLDR
Concerning the long-term side effects, six patients had chronic GVHD disease and eight patients returned to Africa where they are continuing to do well. Expand
Bone marrow transplantation for severe sickle cell anaemia
TLDR
It is concluded that BMT or sickle cell anaemia is curative, well tolerated and should be proposed for suitable patients. Expand
Barriers to bone marrow transplantation for sickle cell anemia.
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  • Medicine
  • Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
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TLDR
The major barrier to marrow transplantation for sickle cell anemia is lack of an HLA-identical donor, and for patients reported to meet eligibility criteria, parental refusal and limited financial or psychosocial support were infrequent barriers to transplantation. Expand
Marrow transplantation for severe aplastic anemia. Long-term outcome in fifty "untransfused" patients.
TLDR
It is found that patients with severe aplastic anemia who have transplants before the onset of transfusion-induced sensitization have an excellent probability of long-term survival and a normal life. Expand
Neurologic complications after allogeneic marrow transplantation for sickle cell anemia.
TLDR
Patients with sickle cell anemia are at increased risk for neurologic complications after marrow ablative therapy and patients with prior stroke are at increase risk for intracranial hemorrhage, and transplantation of patients before the onset of overt stroke may reduce this risk. Expand
Bone marrow transplantation in patients with thalassemia.
TLDR
It is concluded that for patients under 16 years of age, transplantation of bone marrow from an HLA-identical donor offers a high probability of complication-free survival, particularly if they do not have hepatomegaly or portal fibrosis. Expand
BONE MARROW TRANSPLANTATION IN FIVE CHILDREN WITH SICKLE CELL ANAEMIA
TLDR
Five children with severe sickle cell anaemia underwent an HLA compatible allogeneic bone marrow transplantation and in four children the engraftment was rapid and sustained and cytogenetic studies in three patients confirmed the donor origin of bone marrow cells. Expand
Bone marrow transplantation for sickle cell disease. A study of parents' decisions.
TLDR
At current rates of mortality and morbidity with bone marrow transplantation, a substantial minority of the parents of children with sickle cell disease may consent to bone marrow transplants for their children, and parental attitudes should be factored into decisions about whether to offer bone marrow graft-versus-host disease. Expand
Effect of HLA compatibility on engraftment of bone marrow transplants in patients with leukemia or lymphoma.
TLDR
It is concluded that donor HLA incompatibility and prior alloimmunization are significant risk factors for graft failure, and that a more effective immunosuppressive regimen than those currently used is needed for consistent achievement of sustained engraftment of marrow transplanted from donors who are not HLA-identical siblings. Expand
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