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 John P. 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… 
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TLDR
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.
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TLDR
Although the 1-year EFS met the prespecified target of ≥75%, this regimen cannot be considered sufficiently safe for widespread adoption without modifications to achieve more effective GVHD prophylaxis.
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TLDR
SCT from a suitable HLA-matched, related donor should become the primary option for curing children with sickle cell anemia, as there is an excellent survival rate and a return to normal life, free of SCA-related events.
Treatment of chronic granulomatous disease with nonmyeloablative conditioning and a T-cell-depleted hematopoietic allograft.
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.
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.
Stem Cell Transplantation for Hemoglobinopathies
TLDR
This chapter reviews the results and controversies surrounding myeloablative allogeneic stem cell transplantation for the hemoglobinopathies and newer promising developments in the field will be discussed, including the use of lower intensity preparative regimens and alternative sources of donor hematopoietic stem cells that have been generated in the hope of establishing curative therapy for these diseases under conditions of acceptable toxicity.
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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.
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.
Barriers to bone marrow transplantation for sickle cell anemia.
  • M. Walters, M. Patience, K. Sullivan
  • Medicine
    Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
  • 1996
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.
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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.
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.
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.
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.
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TLDR
Fourteen S/S children with severe SCD were transplanted with marrow from HLA identical siblings with a stable total chimerism in 10/14 patients who are cured, with a high stable 22% Hb F level.
Analysis of beta-globin mutations shows stable mixed chimerism in patients with thalassemia after bone marrow transplantation.
TLDR
The detection of beta-globin gene point mutation, as used here, is a highly specific and sensitive marker for engraftment and MC in patients with thalassemia and the high incidence of MC found in patients may be a consequence of the pre-BMT T-cell depletion.
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