Stem Cell Transplantation for Sickle Cell Disease: Can We Reduce the Toxicity?

References

• Noguchi C T, Schechter A N. The intercellular polymerization of sickle hemoglobin and its relevance to sickle cell disease. Blood 1981; 58: 1057–1068
   PubMed Web of Science ®Google Scholar
• Reed W, Vichinsky E. Transfusion practice for patients with sickle cell disease. Curr Opin Hematol 1999; 6: 432–436
   Google Scholar
• Steinberg M. Drug therapy: management of sickle cell disease. N Engl J Med 1999; 340: 1021–1030
   PubMed Web of Science ®Google Scholar
• Lane P A. Sickle cell disease. Pediatr Clin North Am 1996; 43: 639–664
   PubMed Web of Science ®Google Scholar
• Adams R J, McKie V C, Hsu L, et al. Prevention of a first stroke by transfusions in children with sickle cell anemia and abnormal results on transcranial doppler ultrasonography. N Engl J Med 1998; 339: 5–11
   PubMed Web of Science ®Google Scholar
• AuBuchon J P, Birkmeyer J D, Busch M P. Safety of the blood supply in the United States: opportunities and controversies. Ann Intern Med 1997; 127: 904–909
   PubMed Web of Science ®Google Scholar
• Pippard M J. Iron overload and iron chelation therapy in thalassaemia and sickle cell haemoglobinopathies. Acta Haematol 1997; 83: 76–78
   Google Scholar
• Charache S, Terrin M L, Moore R D, et al. Effect of hydroxyurea on the frequency of painful crises in sickle cell anemia. Investigators of the multicenter study of hydroxyurea in sickle cell anemia. N Engl J Med 1995; 332: 1317–1322
   PubMed Web of Science ®Google Scholar
• Rogers Z R. Hydroxyurea therapy for diverse pediatric populations with sickle cell disease. Semin Hematol 1997; 34(suppl 3)42–47
   Google Scholar
• Powers D, Weiss J N, Chan L S, Shroeder W A. Is there a threshold level of fetal hemoglobin that ameliorates morbidity in sickle cell anemia?. Blood 1984; 63: 921–926
   Google Scholar
• Scott J P, Hillery C A, Brown E R, Misiewicz V, Labotka R J. Hydroxyurea therapy in children severely affected with sickle cell disease. J Pediatr 1996; 128: 820–828
   PubMed Web of Science ®Google Scholar
• Ferster A, Vermylen C, Cornu G, et al. Hydroxyurea for treatment of severe sickle cell anemia: a pediatric clinical trial. Blood 1996; 88: 1060–1064
   Google Scholar
• Kinney T R, Helms R W, O'Branski E E, et al. Safety of hydroxyurea in children with sickle cell anemia: results of the HUG-KIDS study, a phase I/II trial. Pediatric Hydroxyurea Group. Blood 1999; 94: 1550–1554
   PubMed Web of Science ®Google Scholar
• Johnson F L, Look A T, Gockerman J, et al. Bone-marrow transplantation in a patient with sickle-cell anemia. N Engl J Med 1984; 311: 780–783
   PubMed Web of Science ®Google Scholar
• Vermylen C, Fernandez Robles E, Ninane J, Cornu G. Bone marrow transplantation in five children with sickle cell anemia. Lancet 1988; 1: 1427–1428
   PubMed Web of Science ®Google Scholar
• Platt O S, Guinan E C. Bone marrow transplantation in sickle cell anemia–the dillema of choice. N Engl J Med 1996; 335: 426–428
   Google Scholar
• Walters M C, Storb R, Patience M, et al. Impact of bone marrow transplantation for symptomatic sickle cell disease: long-term follow-up evaluations. Blood 2000; 95: 1918–1924
   PubMed Web of Science ®Google Scholar
• Bernaudin F, Souillet G, Vannier J P, et al. Report of the French experience concerning 26 children transplanted for sever sickle cell disease. Bone Marrow Transplant 1997; 19: 112–115
   Google Scholar
• Vermylen C, Cornu G, Ferster A, et al. Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in Belgium. Bone Marrow Transplant 1998; 22: 1–6
   PubMed Web of Science ®Google Scholar
• Walters M, Patience M, Leisenring W, et al. Bone marrow transplantation for sickle cell disease. N Engl J Med 1996; 335: 369–376
   PubMed Web of Science ®Google Scholar
• Miller S T, Sleeper L A, Pegelow C H, et al. Prediction of adverse outcomes in children with sickle cell disease. N Engl J Med 2000; 342: 83–89
   PubMed Web of Science ®Google Scholar
• Johnson F L, Mentzner W, Kalinyak K, Sullivan K, Abboud M. Bone marrow transplantation for sickle cell disease: the United States experience. Am J Pediatr Hematol Oncol 1994; 16: 22–26
   Google Scholar
• Andreani M, Manna M, Lucarelli G, et al. Persistence of mixed chimerism in patients transplanted for the treatment of thalassemia. Blood 1996; 87: 3494–3499
   PubMed Web of Science ®Google Scholar
• Nesci S, Manna M, Andreani M, Fattorini P, Graziosi G, Lucarelli G. Mixed chimerism in thalassemic patients after bone marrow transplantation. Bone Marrow Tranplant 1992; 10: 143–146
   Google Scholar
• Kolb H J, Socie G, Duell T, et al. Malignant neoplasms in long-term survivors of bone marrow transplantation. Late effects working party of European Cooperative Group for Blood and Marrow Transplantation and the European Late Effect Project Group. Ann Intern Med 1999; 131: 738–744
   PubMed Web of Science ®Google Scholar
• Michel G, Socie G, Gebhard F, et al. Late effects of allogeneic bone marrow transplantation for children with acute myeloblastic leukemia in first complete remission : the impact of conditioning regimen without total-body irradiation–a report for the Societe Francaise de Greffe de Moelle. J Clin Oncol 1997; 15: 2238–2246
   PubMed Web of Science ®Google Scholar
• Giorgiani G, Bossola M, Locatelli F, et al. Role of busulfan and total body irradiation on growth of prepubertal children receiving bone marrow transplantation and results of treatment with recombinant human growth hormone. Blood 1995; 86: 825–831
   PubMed Web of Science ®Google Scholar
• Sanders J E. Growth and development after hematopoietic cell transplantation. Hematopoietic Cell Transplantation, E D Thomas, S J Forman, J E Sanders. Blackwell Science, Malden, MA 1999; 764–788
   Google Scholar
• Walters M, Sullivan K, Bernaudin F, et al. Neurologic complications after allogenic marrow transplantation for sickle cell anemia. Blood 1995; 86: 879–884
   Google Scholar
• Walters M C, Patience M, Leisenring W, et al. Collaborative multicenter investigation of marrow transplantation for sickle cell disease: current results and future directions. Biol Blood Marrow Transplant 1997; 3: 310–315
   PubMedGoogle Scholar
• Ferster A, Bujan W, Corazza F, et al. Bone marrow transplantation corrects the splenic reticuloendothelial dysfunction in sickle cell anemia. Blood 1993; 81: 1102–1105
   PubMed Web of Science ®Google Scholar
• Giardini C, Galimberti M, Lucarelli G, et al. Bone marrow transplantation in sickle-cell anemia in Pesaro. Bone Marrow Transplant. 1993; 12(Suppl 1)122–123
   Google Scholar
• Swift A, Cohen M, Hynd G, et al. Neuropsychologic impairment in children with sickle cell anemia. Pediatrics 1989; 84: 1077–1085
   PubMed Web of Science ®Google Scholar
• Armstrong F D, Thompson R J, Jr., Wang W, et al. Cognitive functioning and brain magnetic resonance imaging in children with sickle cell disease. Neuropsychology Committee of the Cooperative Study of Sickle Cell Disease. Pediatrics 1996; 97: 864–870
   PubMed Web of Science ®Google Scholar
• Kalinyak K A, Morris C, Ball W S, Ris M D, Harris R, Rucknagel D. Bone marrow transplantation in a young child with sickle cell anemia. Am J Hematol 1995; 48: 256–261
   PubMed Web of Science ®Google Scholar
• Pegelow C, Adams R, McKie V, et al. Risk of recurrent stroke in patients with sickle cell disease treated with erythrocyte transfusions. J Pediatr 1995; 126: 896–899
   PubMed Web of Science ®Google Scholar
• Hernigou P, Bernaudin F, Reinert P, Kuentz M, Vernant J P. Bone-marrow transplantation in sickle-cell disease. Effect on osteonecrosis: a case report with a four-year follow-up. J Bone Joint Surg Am 1997; 79: 1726–1730
   PubMed Web of Science ®Google Scholar
• Sullivan K M, Agura E, Anasetti C, et al. Chronic graft-versus-host disease and other late complications of bone marrow transplantation. Semin Hematol 1991; 28: 250–259
   PubMed Web of Science ®Google Scholar
• Atkinson K, Horowitz M M, Gale R P, et al. Risk factors for chronic graft-versushost disease after HLA-identical sibling bone marrow transplantation. Blood 1990; 75: 2459–2464
   PubMed Web of Science ®Google Scholar
• Sanders J. Bone marrow transplantation for pediatric malignancies. Pediatr Clin North Am 1997; 44: 1005–1016
   Google Scholar
• Ferrara J L, Deeg H J. Graft-versus-host disease. N Engl J Med 1991; 324: 667–674
   PubMed Web of Science ®Google Scholar
• Ferster A, Corraza F, Vertongen F, et al. Transplanted sickle-cell disease patients with autologous bone marrow recovery after graft failure develop increased levels of fetal haemoglobin which corrects disease severity. Br J Haematol 1995; 90: 804–808
   PubMed Web of Science ®Google Scholar
• Walters M C, Storb R, Patience M, et al. Stable mixed chimerism after bone marrow transplantation (BMT) for sickle cell disease (SCD). Blood 1999; 94(Suppl 1)645a, (abstract 2863)
   Google Scholar
• Sykes M, Preffer F, McCaffey D, et al. Mixed lymphohaemopoietic chimerism and graft-versus-lymphoma effects after nonmyeloblative chemotherapy and HLA-mismatched bone marrow transplantation. Lancet 1999; 353: 1756–1759
   Google Scholar
• Owen R D. Immunogenetic consequences of vascular anastomoses between bovine twins. Science 1945; 102: 400
   PubMed Web of Science ®Google Scholar
• Wekerle T, Sykes M. Mixed chimerism as an approach for the induction of transplantation tolerance. Transplantation 1999; 68: 459–467
   PubMedGoogle Scholar
• Sharabi Y, Sachs D H. Mixed chimerism and permanent specific transplantation tolerance induced by a nonlethal preparative regimen. J Exp Med 1989; 169: 493–502
   PubMed Web of Science ®Google Scholar
• Tomita Y, Khan A, Sykes M. Role of intrathymic clonal deletion and peripheral anergy in transplantation tolerance induced by bone marrow transplantation in mice conditioned with a nonmyeloablative regimen. J Immunol 1994; 153: 1087–1098
   PubMedGoogle Scholar
• Colson Y L, Wren S M, Schuchert M J, Patrene K D, Johnson P C, Boggs S S, Ildstad S T. A nonlethal conditioning approach to achieve durable multilineage mixed chimerism and tolerance across major, minor, and hematopoietic histocompatibility barriers. J Immunol 1995; 155: 4179–4188
   PubMed Web of Science ®Google Scholar
• Delaney C P, Murase N, Chen-Woan M, Fung J J, Starzl T E, Demetris A J. Allogeneic hematolymphoid microchimerism and prevention of autoimmune disease in the rat: a relationship between allo- and autoimmunity. J Clin Invest 1996; 97: 217–225
   Google Scholar
• Maeda T, Eto M, Nishimura Y, Nomoto K, Kong Y Y. Role of peripheral hemopoietic chimerism in achieving donor-specific tolerance in adult mice. J Immunol 1993; 150: 753–762
   Google Scholar
• Hakim F T, Sharrow S O, Payne S, Shearer G M. Repopulation of host lymphohematopoietic systems by donor cells during graft-versus-host reaction in unirradiated adult F1 mice injected with parental lymphocytes. J Immunol 1991; 146: 2108–2115
   PubMedGoogle Scholar
• Uharek L, Gassmann W, Glass B, Steinmann J, Loeffler H, Mueller-Ruchholtz W. Influence of cell dose and graft-versus-host reactivity on rejection rates after allogeneic bone marrow transplantation. Blood 1992; 79: 1612–1621
   PubMed Web of Science ®Google Scholar
• Li H, Kaufman C L, Boggs S S, Johnson P C, Patrene K D, Ildstad S T. Mixed allogeneic chimerism induced by a sublethal approach prevents autoimmune diabetes and reverses insulitis in nonobese diabetic (NOD) mice. J Immunol 1996; 156: 380–388
   PubMed Web of Science ®Google Scholar
• Mathieu C, Casteels K, Bouillon R, Waer M. Protection against autoimmune diabetes in mixed bone marrow chimeras. J Immunol 1997; 158: 1453–1457
   Google Scholar
• Gammie J S, Li S, Colson Y L, et al. A partial conditioning strategy for achieving mixed chimerism in the rat: tacrolimus and anti-lymphocyte serum substantially reduce the minimum radiation dose for engraftment. Exp Hem 1998; 26: 927–935
   Google Scholar
• Storb R, Weiden P L, Graham T C, Lerner K G, Nelson N, Thomas E D. Hemopoietic grafts between DLA-identical canine littermates following dimethyl myleran. Evidence for resistance to grafts not associated with DLA and abrogated by antithymocyte serum. Transplantation 1977; 24: 349–357
   PubMedGoogle Scholar
• Storb R, Yu C, Wagner J L, Deeg H J, Nash R A, Kiem H-P, Leisenring W, Shulman H. Stable mixed hematopoietic chimerism in DLA-identical littermate dogs given sublethal total body irradiation before and pharmacological immunosuppression after marrow transplantation. Blood 1997; 89: 3048–3054
   PubMed Web of Science ®Google Scholar
• McSweeney P, Rainer S. Establishing mixed chimerism with immunosuppressive, minimally myelosuppressive conditioning: preclinical and clinical studies. The American Society of Hematology Education Program Book (Blood) 1999; 396–405
   Google Scholar
• Battaglia M, Andreani M, Manna M, et al. Coexistence of two functioning T-cell repertoires in healthy Ex-thalassemics bearing a persistent mixed chimerism years after bone marrow transplantation. Blood 1999; 94: 3432–3438
   Google Scholar
• Amrolia P, Kaeda J, Vulliamy T, Roper D, Dokal I, Roberts I. Analysis of chimerism in children with haemoglobinopathies undergoing stem cell transplantation. Br J Haematol 1998; 101(1S)66
   Google Scholar
• Kapelushnik J, Filon D, Nagler G, et al. Analysis of ß-Globin mutations shows stable mixed chimerism in patients with thalassemia after bone marrow transplantation. Blood 1995; 86: 3241–3246
   Google Scholar