Bone marrow transplantation for sickle cell disease.

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 were prepared for transplantation with busulfan, cyclophosphamide, and antithymocyte globulin. Results Twenty of the 22 patients survived, with a median follow-up of 23.9 months (range, 10.1 to 51.0), and 16 patients had stable engraftment of donor hematopoietic cells. In three patients the graft was rejected and sickle cell disease recurred; in a fourth patient graft rejection was accompanied by marrow aplasia. In 1 of the 16 patients with engraftment, there was stable mixed chimerism. Two patients died of central ne...

[1]  K. Sullivan,et al.  Bone marrow transplantation for sickle cell disease. The United States experience. , 1994, The American journal of pediatric hematology/oncology.

[2]  J. Clegg,et al.  The interaction of alpha-thalassemia and homozygous sickle-cell disease. , 1982, The New England journal of medicine.

[3]  H. Deeg,et al.  Marrow transplantation for severe aplastic anemia. Long-term outcome in fifty "untransfused" patients. , 1986, Annals of internal medicine.

[4]  S. Piomelli.  Sickle cell diseases in the 1990s: the need for active and preventive intervention. , 1991, Seminars in hematology.

[5]  A. Look,et al.  Bone-marrow transplantation in a patient with sickle-cell anemia. , 1984, The New England journal of medicine.

[6]  R. Storb,et al.  Aplastic anemia (first of two parts): pathogenesis, diagnosis, treatment, and prognosis. , 1982 .

[7]  C. Vermylen,et al.  Bone marrow transplantation for sickle cell disease. The European experience. , 1994, The American journal of pediatric hematology/oncology.

[8]  N. Flournoy,et al.  Cytomegalovirus immune globulin and seronegative blood products to prevent primary cytomegalovirus infection after marrow transplantation. , 1986, The New England journal of medicine.

[9]  S. Leikin,et al.  Mortality in children and adolescents with sickle cell disease. Cooperative Study of Sickle Cell Disease. , 1989, Pediatrics.

[10]  J. Niland,et al.  Sickle Cell Chronic Lung Disease: Prior Morbidity and the Risk of Pulmonary Failure , 1988, Medicine.

[11]  J. Harousseau,et al.  BONE-MARROW TRANSPLANTATION FOR SICKLE-CELL ANAEMIA , 1988, The Lancet.

[12]  H. Deeg,et al.  Prevention of graft failure in patients with aplastic anemia. , 1993, Bone marrow transplantation.

[13]  G. Koren,et al.  Oral iron chelation with 1,2-dimethyl-3-hydroxypyrid-4-one (L1) in iron loaded thalassemia patients. , 1993, Bone marrow transplantation.

[14]  J. Morgan.  HOSPITAL FOR SICK CHILDREN. , 1884 .

[15]  M. Andreani,et al.  Marrow transplantation in patients with thalassemia responsive to iron chelation therapy. , 1993, The New England journal of medicine.

[16]  Effects of CAMPATH-1 antibodies in vivo in patients with lymphoid malignancies: influence of antibody isotype. , 1989 .

[17]  W. Schroeder,et al.  Is there a threshold level of fetal hemoglobin that ameliorates morbidity in sickle cell anemia? , 1984, Blood.

[18]  K. Sullivan,et al.  Introduction to a symposium on sickle cell anemia: current results of comprehensive care and the evolving role of bone marrow transplantation. , 1991, Seminars in hematology.

[19]  R. Smith.  Using a mock trial to make a difficult clinical decision. , 1992, BMJ.

[20]  E. Kaplan,et al.  Nonparametric Estimation from Incomplete Observations , 1958 .

[21]  JN Weiss,et al.  Is there a threshold level of fetal hemoglobin that ameliorates morbidity in sickle cell anemia , 1984 .

[22]  S. Lemerle,et al.  Bone marrow transplantation (BMT) in 14 children with severe sickle cell disease (SCD): the French experience. GEGMO. , 1993, Bone marrow transplantation.

[23]  R. Mulhern,et al.  High risk of recurrent stroke after discontinuance of five to twelve years of transfusion therapy in patients with sickle cell disease. , 1991, The Journal of pediatrics.

[24]  R. Parkman.  The application of bone marrow transplantation to the treatment of genetic diseases. , 1986, Science.

[25]  J. Kalbfleisch,et al.  The Statistical Analysis of Failure Time Data , 1980 .

[26]  D. Powars.  Natural history of sickle cell disease--the first ten years. , 1975, Seminars in hematology.

[27]  A. Nademanee,et al.  Cyclosporine, methotrexate, and prednisone compared with cyclosporine and prednisone for prophylaxis of acute graft-versus-host disease. , 1993, The New England journal of medicine.

[28]  E. Sariban,et al.  Bone marrow transplantation for severe sickle cell anaemia , 1992, British journal of haematology.

[29]  P. Dr,et al.  Sickle cell anemia: beta s-gene-cluster haplotypes as prognostic indicators of vital organ failure. , 1991, Seminars in hematology.

[30]  D. Powars.  Sickle cell anemia: beta s-gene-cluster haplotypes as prognostic indicators of vital organ failure. , 1991, Seminars in hematology.

[31]  H. Deeg,et al.  Methotrexate and cyclosporine compared with cyclosporine alone for prophylaxis of acute graft versus host disease after marrow transplantation for leukemia. , 1986, The New England journal of medicine.

[32]  C. Stocking,et al.  Bone marrow transplantation for sickle cell disease. A study of parents' decisions. , 1991, The New England journal of medicine.

[33]  G. Lucarelli,et al.  Bone marrow transplantation in patients with thalassemia. , 1990, The New England journal of medicine.

[34]  J. Niland,et al.  Chronic renal failure in sickle cell disease: risk factors, clinical course, and mortality. , 1991, Annals of internal medicine.

[35]  G. Lucarelli.  FOR DEBATE: BONE MARROW TRANSPLANTATION FOR SEVERE THALASSAEMIA (1) THE VIEW FROM PESARO , 1991, British journal of haematology.

[36]  E. Vichinsky,et al.  Effects of a long-term transfusion regimen on sickle cell-related illnesses. , 1994, The Journal of pediatrics.

[37]  C. Vermylen,et al.  BONE MARROW TRANSPLANTATION IN FIVE CHILDREN WITH SICKLE CELL ANAEMIA , 1988, The Lancet.

[38]  K. Sullivan,et al.  Effect of HLA compatibility on engraftment of bone marrow transplants in patients with leukemia or lymphoma. , 1989, The New England journal of medicine.

[39]  E. Vichinsky.  Comprehensive care in sickle cell disease: its impact on morbidity and mortality. , 1991, Seminars in hematology.

[40]  M. Andreani,et al.  Mixed chimerism in thalassemic patients after bone marrow transplantation. , 1992, Bone marrow transplantation.