« Previous
Next »
Current Problems in Pediatric and Adolescent Health Care
Volume 38, Issue 1
, Pages 6-18
, January 2008
Hematopoietic Cell Transplantation for Hemoglobinopathies
References
- . Mortality in children and adolescents with sickle cell disease (Cooperative Study of Sickle Cell Disease). Pediatrics. 1989;84:500–508
- Mortality in sickle cell disease (Life expectancy and risk factors for early death). N Engl J Med. 1994;330:1639–1644
- . Natural history and determinants of clinical severity of sickle cell disease. Curr Opin Hematol. 1995;2:103–108
- Pain in sickle cell disease (Rates and risk factors). N Engl J Med. 1991;325:11–16
- Mortality in sickle cell disease (Life expectancy and risk factors for early death [see comments]). N Engl J Med. 1994;330:1639–1644
- . Stroke in sickle cell disease: demographic, clinical, and therapeutic considerations. Semin Hematol. 1991;28:213–219
- 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
- . Cognitive screening for silent cerebral infarction in children with sickle cell disease. J Pediatr Hematol Oncol. 2006;28:166–169
- Silent infarction as a risk factor for overt stroke in children with sickle cell anemia: a report from the Cooperative Study of Sickle Cell Disease. J Pediatr. 2001;139:385–390
- Matched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research. Br J Haematol. 2007;137:479–485
- Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease. Blood. 2007;110:2749–2756
- . Stroke prevention in sickle cell disease. Curr Opin Hematol. 2000;7:101–105
- Impact of bone marrow transplantation for symptomatic sickle cell disease: an interim report. Blood. 2000;95:1918–1924
- Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in Belgium. Bone Marrow Transplant. 1998;22:1–6
- Long-term results of related, myeloablative stem cell transplantation to cure sickle cell disease. Blood. 2007;110:2749–2756
- Impact of bone marrow transplantation for symptomatic sickle cell disease: an interim report (Multicenter investigation of bone marrow transplantation for sickle cell disease). Blood. 2000;95:1918–1924
- . Bone marrow transplantation for sickle cell anemia. Am J Pediatr Hematol Oncol. 1994;16:86–89
- Bone marrow transplantation in sickle-cell anemia in Pesaro. Bone Marrow Transplant. 1993;12:122–123
- . Sickle cell anemia and hematopoietic cell transplantation: when is a pound of cure worth more than an ounce of prevention?. Pediatr Transplant. 2004;8(Suppl 5):33–38
- Bone marrow transplantation corrects the splenic reticuloendothelial dysfunction in sickle cell anemia. Blood. 1993;81:1102–1105
- Effect of myeloablative bone marrow transplantation on growth in children with sickle cell anaemia: results of the multicenter study of haematopoietic cell transplantation for sickle cell anaemia. Br J Haematol. 2007;136:673–676
- Barriers to bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant. 1996;2:100–104
- . A survey on patient perception of reduced-intensity transplantation in adults with sickle cell disease. Bone Marrow Transplant. 2007;39:447–451
- . Bone marrow transplantation in sickle cell disease: the trade-off between early mortality and quality of life. Clin Res. 1990;38:694–700
- . Bone marrow transplantation for sickle cell disease (A study of parents’ decisions). N Engl J Med. 1991;325:1349–1353
- Allogeneic stem cell transplantation for sickle cell disease (A study of patients’ decisions). Bone Marrow Transplant. 2001;28:545–549
- Comprehensive banking of sibling donor cord blood for children with malignant and nonmalignant disease. Blood. 2003;101:351–357
- . Persistence of fetal hemoglobin production after successful transplantation of cord blood stem cells in a patient with sickle cell anemia. J Pediatr. 1996;128:241–243
- . Successful cord blood transplantation for sickle cell anemia from a sibling who is human leukocyte antigen-identical: implications for comprehensive care. J Pediatr Hematol Oncol. 2000;22:437–440
- Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. Blood. 2003;101:2137–2143
- Limiting transplantation-related mortality following unrelated donor stem cell transplantation by using a nonmyeloablative conditioning regimen. Blood. 2002;99:1071–1078
- Unrelated cord blood transplantation in children with sickle cell disease: review of four-center experience. Pediatr Transplant. 2007;11:641–644
- Transplantation of unrelated placental blood cells in children with high-risk sickle cell disease. Bone Marrow Transplant. 2004;34:405–411
- Unrelated cord blood transplantation in children with sickle cell disease: review of four centers experience. Pediatr Transplant. 2007;
- . Unrelated placental cord blood transplantation in a patient with sickle cell disease and acute non-lymphocytic leukemia. Blood. 1997;90:
- Unrelated bone marrow transplantation in thalassemia (The experience of the Italian Bone Marrow Transplant Group (GITMO)). Haematologica. 2002;87:58–61
- . Hematopoietic cell transplantation for benign hematological disorders and solid tumors. Hematology (Am Soc Hematol Educ Program). 2003;372–397
- . Bone marrow transplantation without myeloablation for sickle cell disease. N Engl J Med. 2001;344:68
- . Matched sibling donor hematopoietic cell transplantation for sickle cell disease using a reduced intensity conditioning regimen can lead to stable long term engraftment. Blood. 2005;106(11):6569;(Abstract #3172)
- . Hematopoietic stem cell transplantation after reduced-intensity conditioning as treatment of sickle cell disease. Exp Hematol. 2002;30:7–10
- A novel reduced-intensity stem cell transplant regimen for nonmalignant disorders. Bone Marrow Transplant. 2005;35:345–352
- Fludarabine-based conditioning for allogeneic transplantation in adults with sickle cell disease. Bone Marrow Transplant. 2000;26:445–449
- . Transplantation of a child with sickle cell anemia with an unrelated cord blood unit after reduced intensity conditioning. J Pediatr Hematol Oncol. 2006;28:840–844
- . Hematopoietic stem cell transplantation for multiply transfused patients with sickle cell disease and thalassemia after low-dose total body irradiation, fludarabine, and rabbit anti-thymocyte globulin. Bone Marrow Transplant. 2005;35:171–177
- Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia. Biol Blood Marrow Transplant. 2003;9:519–528
- . Non-myeloablative hematopoietic cell transplant for treatment of nonmalignant disorders in children. Int J Hematol. 2002;76(Suppl 2):271–277
- Survival in medically treated patients with homozygous beta-thalassemia. N Engl J Med. 1994;331:574–578
- . Stem cell transplantation and gene therapy for hemoglobinopathies. Curr Hematol Rep. 2005;4:126–131
- . Bone marrow transplantation in adults with thalassemia: treatment and long-term follow-up. Ann NY Acad Sci. 2005;1054:196–205
- Long-term safety and effectiveness of iron-chelation therapy with deferiprone for thalassemia major. N Engl J Med. 1998;339:417–423
- . Long term deferiprone chelation therapy. Adv Exp Med Biol. 2002;509:127–139
- A randomized, placebo-controlled, double-blind trial of the effect of combined therapy with deferoxamine and deferiprone on myocardial iron in thalassemia major using cardiovascular magnetic resonance. Circulation. 2007;115:1876–1884
- A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia. Blood. 2006;107:3455–3462
- . Oral chelators deferasirox and deferiprone for transfusional iron overload in thalassemia major: new data, new questions. Blood. 2006;107:3436–3441
- . The hypercoagulable state in thalassemia. Blood. 2002;99:36–43
- . Outcome of sickle cell anemia: a 4-decade observational study of 1056 patients. Medicine (Baltimore). 2005;84:363–376
- Pulmonary hypertension as a risk factor for death in patients with sickle cell disease. N Engl J Med. 2004;350:886–895
- Sildenafil therapy in patients with sickle cell disease and pulmonary hypertension. Br J Haematol. 2005;130:445–453
- Pain in sickle cell disease: rates and risk factors. N Engl J Med. 1991;325:11–16
- Hydroxyurea and sickle cell anemia (Clinical utility of a myelosuppressive “switching” agent. The Multicenter Study of Hydroxyurea in Sickle Cell Anemia). Medicine (Baltimore). 1996;75:300–326
- . Pharmacologic modulation of fetal hemoglobin. Medicine (Baltimore). 2001;80:328–344
- . The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin: a novel mechanism of human disease. JAMA. 2005;293:1653–1662
- Lactate dehydrogenase as a biomarker of hemolysis-associated nitric oxide resistance, priapism, leg ulceration, pulmonary hypertension, and death in patients with sickle cell disease. Blood. 2006;107:2279–2285
- Sickle cell leg ulcers: associations with haemolysis and SNPs in Klotho (TEK and genes of the TGF-beta/BMP pathway). Br J Haematol. 2006;133:570–578
- Stroke risk in siblings with sickle cell anemia. Blood. 2003;101:2401–2404
- Gene interactions and stroke risk in children with sickle cell anemia. Blood. 2004;103:2391–2396
- . Genetic dissection and prognostic modeling of overt stroke in sickle cell anemia. Nat Genet. 2005;37:435–440
- Association of klotho, bone morphogenic protein 6, and annexin A2 polymorphisms with sickle cell osteonecrosis. Blood. 2005;106:372–375
- UGT1A1 variation and gallstone formation in sickle cell disease. Blood. 2005;105:968–972
- . Phenotype/genotype relationships in sickle cell disease: a pilot twin study. Clin Lab Haematol. 2005;27:384–390
- A network model to predict the risk of death in sickle cell disease. Blood. 2007;110:2727–2735
- Effect of hydroxyurea on mortality and morbidity in adult sickle cell anemia: risks and benefits up to 9 years of treatment. JAMA. 2003;289:1645–1651
- Morbidity and mortality in chronically transfused subjects with thalassemia and sickle cell disease: a report from the multi-center study of iron overload. Am J Hematol. 2007;82:255–265
- A randomised comparison of deferasirox versus deferoxamine for the treatment of transfusional iron overload in sickle cell disease. Br J Haematol. 2007;136:501–508
- . Cardiopulmonary complications of sickle cell disease: role of nitric oxide and hemolytic anemia. Hematology Am Soc Hematol Educ Program. 2005;51–57
- . Overt and incomplete (silent) cerebral infarction in sickle cell anemia: diagnosis and management. Hematol Oncol Clin North Am. 2005;19:839–855vi
- Restoration of ovarian function after orthotopic (intraovarian and periovarian) transplantation of cryopreserved ovarian tissue in a woman treated by bone marrow transplantation for sickle cell anaemia: case report. Hum Reprod. 2006;21:183–188
- . The genetic engineering of hematopoietic stem cells: the rise of lentiviral vectors, the conundrum of the LTR, and the promise of lineage-restricted vectors. Mol Ther. 2007;15:445–456
- Therapeutic options for patients with severe beta-thalassemia: the need for globin gene therapy. Hum Gene Ther. 2007;18:1–9
- . Correction of sickle cell disease by homologous recombination in embryonic stem cells. Blood. 2006;108:1183–1188
- . Correction of the sickle cell mutation in embryonic stem cells. Proc Natl Acad Sci USA. 2006;103:1036–1040
- Long-term expression of gamma-globin mRNA in mouse erythrocytes from retrovirus vectors containing the human gamma-globin gene fused to the ankyrin-1 promoter. Proc Natl Acad Sci USA. 2000;97:13294–13299
- A minimal ankyrin promoter linked to a human gamma-globin gene demonstrates erythroid specific copy number dependent expression with minimal position or enhancer dependence in transgenic mice. J Biol Chem. 2000;275:28549–28554
- Variegated expression from the murine band 3 (AE1) promoter in transgenic mice is associated with mRNA transcript initiation at upstream start sites and can be suppressed by the addition of the chicken beta-globin 5′ HS4 insulator element. Mol Cell Biol. 2003;23:4753–4763
- . Fetal gene therapy of alpha-thalassemia in a mouse model. Proc Natl Acad Sci USA. 2007;104:9007–9011
- . Successful treatment of murine beta-thalassemia intermedia by transfer of the human beta-globin gene. Blood. 2002;99:1902–1908
- . A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human beta-globin gene transfer. Blood. 2003;101:2932–2939
- . Functional requirements for phenotypic correction of murine beta-thalassemia: implications for human gene therapy. Blood. 2001;97:3275–3282
- . Extended beta-globin locus control region elements promote consistent therapeutic expression of a gamma-globin lentiviral vector in murine beta-thalassemia. Blood. 2004;104:2281–2290
- . Regulated expression of a complete human beta-globin gene encoded by a transmissible retrovirus vector. Mol Cell Biol. 1987;7:887–897
- . Lineage-specific expression of a human beta-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells. Nature. 1988;331:35–41
- . A human beta-globin gene fused to the human beta-globin locus control region is expressed at high levels in erythroid cells of mice engrafted with retrovirus-transduced hematopoietic stem cells. Blood. 1993;81:1384–1392
- . Retroviral vectors for the beta-globin gene that demonstrate improved titer and expression. Ann NY Acad Sci. 1990;612:427–441
- . High-level beta-globin expression after retroviral transfer of locus activation region-containing human beta-globin gene derivatives into murine erythroleukemia cells. Proc Natl Acad Sci USA. 1990;87:3386–3390
- Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin. Nature. 2000;406:82–86
- . The cHS4 insulator increases the probability of retroviral expression at random chromosomal integration sites. J Virol. 2000;74:4679–4687
- Permanent and panerythroid correction of murine beta thalassemia by multiple lentiviral integration in hematopoietic stem cells. Proc Natl Acad Sci USA. 2002;99:14380–14385
- LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science. 2003;302:415–419
- . Repair of a splicing defect in erythroid cells from patients with beta-thalassemia/HbE disorder. Mol Ther. 2002;6:718–726
- Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides. Mol Pharmacol. 2002;62:545–553
- . Repair of thalassemic human beta-globin mRNA in mammalian cells by antisense oligonucleotides. Proc Natl Acad Sci USA. 1996;93:12840–12844
- A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference. Nat Biotechnol. 2006;24:89–94
- . Stem cell-derived erythroid cells mediate long-term systemic protein delivery. Nat Biotechnol. 2006;24:1017–1021
- Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells. Blood. 2003;102:506–513
- Transient in vivo selection of transduced peripheral blood cells using antifolate drug selection in rhesus macaques that received transplants with hematopoietic stem cells expressing dihydrofolate reductase vectors. Blood. 2004;103:796–803
This review is the result of a symposium at the meeting of American Society of Pediatric Hematology and Oncology (ASPHO) in May, 2007.
Supported by: Children’s Cancer Research Fund, Minneapolis, MN.
PII: S1538-5442(07)00087-9
doi: 10.1016/j.cppeds.2007.10.002
© 2008 Mosby, Inc. All rights reserved.
« Previous
Next »
Current Problems in Pediatric and Adolescent Health Care
Volume 38, Issue 1
, Pages 6-18
, January 2008
