Neal S Young

Summary

Affiliation: National Institutes of Health
Country: USA

Publications

  1. pmc Aplastic anemia
    Neal S Young
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland 20892 1202, USA
    Curr Opin Hematol 15:162-8. 2008
  2. ncbi request reprint A mouse model of lymphocyte infusion-induced bone marrow failure
    Michael L Bloom
    Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD 20892 1652, USA
    Exp Hematol 32:1163-72. 2004
  3. ncbi request reprint Brief communication: Successful treatment of pure red-cell aplasia with an anti-interleukin-2 receptor antibody (daclizumab)
    Elaine M Sloand
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland 20892, USA
    Ann Intern Med 144:181-5. 2006
  4. pmc Current concepts in the pathophysiology and treatment of aplastic anemia
    Neal S Young
    Hematology Branch, National Heart, Lung, and Blood Institute NIH, 10 Center Drive, Bldg 10 CRC, Rm 3E 5140, Bethesda, MD 20892 1202, USA
    Blood 108:2509-19. 2006
  5. doi request reprint Telomere biology and telomere diseases: implications for practice and research
    Neal S Young
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Hematology Am Soc Hematol Educ Program 2010:30-5. 2010
  6. ncbi request reprint Mutations in TERT, the gene for telomerase reverse transcriptase, in aplastic anemia
    Hiroki Yamaguchi
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892 1202, USA
    N Engl J Med 352:1413-24. 2005
  7. doi request reprint Establishment of an erythroid cell line from primary CD36+ erythroid progenitor cells
    Susan Wong
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Exp Hematol 38:994-1005.e1-2. 2010
  8. pmc Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug
    Ronan Desmond
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
    Blood 123:1818-25. 2014
  9. ncbi request reprint Minor antigen h60-mediated aplastic anemia is ameliorated by immunosuppression and the infusion of regulatory T cells
    Jichun Chen
    Hematology Branch, National Heart, Lung, and Blood Institute, NIH Building 10, Clinical Research Center, 10 Center Drive, Bethesda, MD 20892, USA
    J Immunol 178:4159-68. 2007
  10. ncbi request reprint Late complications following treatment for severe aplastic anemia (SAA) with high-dose cyclophosphamide (Cy): follow-up of a randomized trial
    John F Tisdale
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Blood 100:4668-70. 2002

Detail Information

Publications108 found, 100 shown here

  1. pmc Aplastic anemia
    Neal S Young
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland 20892 1202, USA
    Curr Opin Hematol 15:162-8. 2008
    ....
  2. ncbi request reprint A mouse model of lymphocyte infusion-induced bone marrow failure
    Michael L Bloom
    Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD 20892 1652, USA
    Exp Hematol 32:1163-72. 2004
    ..To develop a mouse model for the study of the pathophysiologic mechanism and treatment of human bone marrow (BM) failure...
  3. ncbi request reprint Brief communication: Successful treatment of pure red-cell aplasia with an anti-interleukin-2 receptor antibody (daclizumab)
    Elaine M Sloand
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland 20892, USA
    Ann Intern Med 144:181-5. 2006
    ..Most patients respond to some form of immunosuppressive treatment, but few prospective clinical trials have been performed...
  4. pmc Current concepts in the pathophysiology and treatment of aplastic anemia
    Neal S Young
    Hematology Branch, National Heart, Lung, and Blood Institute NIH, 10 Center Drive, Bldg 10 CRC, Rm 3E 5140, Bethesda, MD 20892 1202, USA
    Blood 108:2509-19. 2006
    ..Recent results with alternative sources of stem cells and a variety of conditioning regimens to achieve their engraftment have been promising, with survival in small pediatric case series rivaling conventional transplantation results...
  5. doi request reprint Telomere biology and telomere diseases: implications for practice and research
    Neal S Young
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Hematology Am Soc Hematol Educ Program 2010:30-5. 2010
    ....
  6. ncbi request reprint Mutations in TERT, the gene for telomerase reverse transcriptase, in aplastic anemia
    Hiroki Yamaguchi
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892 1202, USA
    N Engl J Med 352:1413-24. 2005
    ..We investigated whether mutations in genes for other components of telomerase also occur in aplastic anemia...
  7. doi request reprint Establishment of an erythroid cell line from primary CD36+ erythroid progenitor cells
    Susan Wong
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Exp Hematol 38:994-1005.e1-2. 2010
    ..We tried to establish a continuous erythroid cell line from the primary CD36(+) erythroid progenitor cells (EPCs) by the lentivirus-mediated gene transduction system...
  8. pmc Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug
    Ronan Desmond
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
    Blood 123:1818-25. 2014
    ..This study is registered at www.clinicaltrials.gov as #NCT00922883. ..
  9. ncbi request reprint Minor antigen h60-mediated aplastic anemia is ameliorated by immunosuppression and the infusion of regulatory T cells
    Jichun Chen
    Hematology Branch, National Heart, Lung, and Blood Institute, NIH Building 10, Clinical Research Center, 10 Center Drive, Bethesda, MD 20892, USA
    J Immunol 178:4159-68. 2007
    ..Immunosuppressive drug treatment or enhancement of regulatory T cell function abrogated this pathophysiology and protected animals from the development of BM failure...
  10. ncbi request reprint Late complications following treatment for severe aplastic anemia (SAA) with high-dose cyclophosphamide (Cy): follow-up of a randomized trial
    John F Tisdale
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Blood 100:4668-70. 2002
    ..Bone marrow cytogenetic abnormalities have been observed among surviving patients in both arms (2 of 14 ATG versus 1 of 12 Cy, P =.70). High-dose Cy does not prevent relapse or clonal evolution in SAA...
  11. ncbi request reprint Mitochondrial DNA sequence heterogeneity of single CD34+ cells after nonmyeloablative allogeneic stem cell transplantation
    Yong Gang Yao
    Hematology Branch, National Heart, Lung, and Blood Institute, NIH, Building 10 CRC, Room 3E 5140, 10 Center Drive, Bethesda, Maryland 20892 1202, USA
    Stem Cells 25:2670-6. 2007
    ..Disclosure of potential conflicts of interest is found at the end of this article...
  12. pmc Rabbit ATG but not horse ATG promotes expansion of functional CD4+CD25highFOXP3+ regulatory T cells in vitro
    Xingmin Feng
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Blood 111:3675-83. 2008
    ..The therapeutic effects of rATG may occur not only because of lymphocyte depletion but also enhanced Treg cell number and function...
  13. ncbi request reprint Hematopoietic-specific microRNA expression in human cells
    Shakti H Ramkissoon
    Howard Hughes Medical Institute National Institutes of Health Research Scholars Program, Bethesda, MD 20892, USA
    Leuk Res 30:643-7. 2006
    ..Further our results showed differences in miRNA expression between mouse and human hematopoietic cells, suggesting important regulatory roles of miRNAs in human hematopoiesis and oncogenesis...
  14. pmc Preferential suppression of trisomy 8 compared with normal hematopoietic cell growth by autologous lymphocytes in patients with trisomy 8 myelodysplastic syndrome
    Elaine M Sloand
    Hematology Branch, National Heart, Lung, and Blood Institute, Bldg 10, Rm 7C108, National Institutes of Health, Bethesda, MD 20892, USA
    Blood 106:841-51. 2005
    ..An increased number of T cells with apparent specificity for trisomy 8 cells is consistent with an autoimmune pathophysiology in trisomy 8 MDS...
  15. ncbi request reprint Distinctive gene expression profiles of CD34 cells from patients with myelodysplastic syndrome characterized by specific chromosomal abnormalities
    Guibin Chen
    Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD, USA
    Blood 104:4210-8. 2004
    ..These results imply distinct molecular mechanisms for monosomy 7 and trisomy 8 MDS and implicate specific pathogenic pathways...
  16. doi request reprint Rapid donor T-cell engraftment increases the risk of chronic graft-versus-host disease following salvage allogeneic peripheral blood hematopoietic cell transplantation for bone marrow failure syndromes
    Jeremy Pantin
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Department of Health and Human Services, Bethesda, Maryland Division of Hematology, Medical Oncology and BMT, Department of Medicine, Georgia Regents University, Georgia
    Am J Hematol 88:874-82. 2013
    ..Clinicaltrials.gov identifier: NCT00003838)...
  17. ncbi request reprint Mitochondrial DNA sequence heterogeneity in circulating normal human CD34 cells and granulocytes
    Myung Geun Shin
    Hematology Branch and Flow Cytometry Core Facility, National Heart, Lung and Blood Institute, National Institutes of Health, Bldg 10, Rm 7C103, 9000 Rockville Pike, Bethesda, MD 20892 1652, USA
    Blood 103:4466-77. 2004
    ....
  18. pmc Mutations in the SBDS gene in acquired aplastic anemia
    Rodrigo T Calado
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892 1202, USA
    Blood 110:1141-6. 2007
    ..We propose that heterozygosity for the 258 + 2 T>C SBDS mutation predisposes to AA by accelerating telomere shortening of leukocytes via a telomerase-independent mechanism...
  19. pmc Enrichment of hematopoietic stem cells with SLAM and LSK markers for the detection of hematopoietic stem cell function in normal and Trp53 null mice
    Jichun Chen
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892 1202, USA
    Exp Hematol 36:1236-43. 2008
    ..To test function of hematopoietic stem cells (HSCs) in vivo in C57BL/6 (B6) and Trp53-deficient (Trp53 null) mice by using two HSC enrichment schemes...
  20. pmc Interferon-gamma-induced gene expression in CD34 cells: identification of pathologic cytokine-specific signature profiles
    Weihua Zeng
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Blood 107:167-75. 2006
    ....
  21. pmc Th17 immune responses contribute to the pathophysiology of aplastic anemia
    Regis Peffault De Latour
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Blood 116:4175-84. 2010
    ..01) and more Treg cells (P < .05) at day 10 after lymph node infusion. Th17 immune responses contribute to AA pathophysiology, especially at the early stage during disease progression...
  22. pmc Phenotypic and functional characterization of a mouse model of targeted Pig-a deletion in hematopoietic cells
    Valeria Visconte
    Hematology Branch, NHLBI, National Institutes of Health, Bethesda, MD 20892 1202 USA
    Haematologica 95:214-23. 2010
    ..Somatic mutation in the X-linked phosphatidylinositol glycan class A gene (PIG-A) causes glycosyl phosphatidylinositol anchor deficiency in human patients with paroxysmal nocturnal hemoglobinuria...
  23. ncbi request reprint CD34+ cells from paroxysmal nocturnal hemoglobinuria (PNH) patients are deficient in surface expression of cellular prion protein (PrPc)
    Antonio M Risitano
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Exp Hematol 31:65-72. 2003
    ..Conversely, PrP(c) is a good model to elucidate the fate of GPI-AP in PIG-A-deficient cells...
  24. pmc Expansion of haematopoietic stem cells from normal donors and bone marrow failure patients by recombinant hoxb4
    Yong Tang
    Hematology Branch, National Heart, Lung, and Blood Institute, NIH, Bethesda, MD 20892 1202, USA
    Br J Haematol 144:603-12. 2009
    ..Recombinant human hoxb4 could be a promising therapeutic agent for BM failure...
  25. pmc Treatment of severe aplastic anemia with a combination of horse antithymocyte globulin and cyclosporine, with or without sirolimus: a prospective randomized study
    Phillip Scheinberg
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892 1202, USA
    Haematologica 94:348-54. 2009
    ....
  26. pmc Human parvovirus B19 causes cell cycle arrest of human erythroid progenitors via deregulation of the E2F family of transcription factors
    Zhihong Wan
    Hematology Branch, National Heart, Lung, and Blood Institute, NIH, Bethesda, Maryland 20892 1652, USA
    J Clin Invest 120:3530-44. 2010
    ..These findings provide new insight into the molecular pathogenesis of B19V in highly permissive erythroid progenitors...
  27. ncbi request reprint Fas-mediated apoptosis is important in regulating cell replication and death in trisomy 8 hematopoietic cells but not in cells with other cytogenetic abnormalities
    Elaine M Sloand
    Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD 20892 1652, USA
    Blood 100:4427-32. 2002
    ..Clinical data demonstrating the responsiveness of some patients with trisomy 8 to anti-thymocyte globulin (ATG) and cyclosporine (CsA) would favor an active role of the immune system in this syndrome...
  28. pmc Effects of granulocyte-colony-stimulating factor on Monosomy 7 aneuploidy in healthy hematopoietic stem cell and granulocyte donors
    Matthew J Olnes
    Hematology Branch, NHLBI, National Institutes of Health, Bethesda, Maryland 20892, USA
    Transfusion 52:537-41. 2012
    ..However, there are no studies addressing whether repeated administration of G-CSF produces Monosomy 7 aneuploidy in healthy donors...
  29. pmc Granulocyte colony-stimulating factor preferentially stimulates proliferation of monosomy 7 cells bearing the isoform IV receptor
    Elaine M Sloand
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Proc Natl Acad Sci U S A 103:14483-8. 2006
    ..The abnormal response of monosomy 7 cells to GCSF would be explained by the expansion of undifferentiated monosomy 7 clones expressing the class IV GCSFR, which is defective in signaling cell maturation...
  30. ncbi request reprint Transcript profile of CD4+ and CD8+ T cells from the bone marrow of acquired aplastic anemia patients
    Weihua Zeng
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Exp Hematol 32:806-14. 2004
    ..T cells producing IFN-gamma and TNF-alpha have been implicated in hematopoietic destruction in AA. We sought to characterize T cells as immune mediators using the microarray approach...
  31. pmc Distinct EBV and CMV reactivation patterns following antibody-based immunosuppressive regimens in patients with severe aplastic anemia
    Phillip Scheinberg
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, 10 Center Drive, Bethesda, MD 20892, USA
    Blood 109:3219-24. 2007
    ....
  32. pmc The role of the Th1 transcription factor T-bet in a mouse model of immune-mediated bone-marrow failure
    Yong Tang
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Blood 115:541-8. 2010
    ..Our data provided fresh evidence demonstrating a critical role of T-bet in immune-mediated BM failure...
  33. ncbi request reprint Genomic instability in bone marrow failure syndromes
    William G Kearns
    Hematology Branch, National Heart, Lung, and Blood Institute NIH, Building 10, Room 7C103, Bethesda, MD, USA
    Am J Hematol 76:220-4. 2004
    ..We conclude that undetected aneuploidy exists in marrow cells of a significant percentage of patients with bone marrow failure syndromes...
  34. ncbi request reprint Treatment of severe aplastic anaemia with combined immunosuppression: anti-thymocyte globulin, ciclosporin and mycophenolate mofetil
    Phillip Scheinberg
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD 20892 1202, USA
    Br J Haematol 133:606-11. 2006
    ..Over half of the relapses occurred during MMF administration. Despite a strong theoretical rationale for its use, MMF did not result in the improvement of response or relapse rates when compared with historical standard h-ATG/CsA...
  35. pmc Horse versus rabbit antithymocyte globulin in acquired aplastic anemia
    Phillip Scheinberg
    Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD, USA
    N Engl J Med 365:430-8. 2011
    ..Although horse ATG is the standard therapy, rabbit ATG is more potent in depleting peripheral-blood lymphocytes and is preferred in other clinical circumstances...
  36. pmc Hematopoiesis in 3 dimensions: human and murine bone marrow architecture visualized by confocal microscopy
    Tomoiku Takaku
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Blood 116:e41-55. 2010
    ..The technique should be generally adaptable for basic laboratory investigation and for clinical diagnosis of hematologic diseases...
  37. ncbi request reprint Is there a direct effect of antithymocyte globulin on hematopoiesis?
    Guibin Chen
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Hematol J 5:255-61. 2004
    ..In summary, the spectrum of effects of ATG on hematopoietic progenitors is dependent upon the concentrations of ATG and may not be related to its antigenic specificity...
  38. doi request reprint VP1u phospholipase activity is critical for infectivity of full-length parvovirus B19 genomic clones
    Claudia Filippone
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institute of Health, Bethesda, MD 20892, USA
    Virology 374:444-52. 2008
    ..Investigation of the original serum from which pB19-FL was cloned confirmed that the phospholipase mutation was present in the native B19 virus...
  39. pmc Human telomere disease due to disruption of the CCAAT box of the TERC promoter
    Anna M Aalbers
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Blood 119:3060-3. 2012
    ..We propose that current mutation-screening strategies should include gene promoter regions for the diagnosis of telomere diseases. This clinical trial was registered at www.clinicaltrials.gov as #NCT00071045...
  40. pmc Activity of alemtuzumab monotherapy in treatment-naive, relapsed, and refractory severe acquired aplastic anemia
    Phillip Scheinberg
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892 1202, USA
    Blood 119:345-54. 2012
    ..We conclude that alemtuzumab is effective in SAA, but best results are obtained in the relapsed and refractory settings. The present trials were registered at www.clinicaltrials.gov as NCT00195624, NCT00260689, and NCT00065260...
  41. pmc Association of telomere length of peripheral blood leukocytes with hematopoietic relapse, malignant transformation, and survival in severe aplastic anemia
    Phillip Scheinberg
    Hematology Branch, National Heart, Lung, and Blood Institute, 10 Center Dr, Bldg 10 CRC, Room 3 5140, MSC 1202, Bethesda, MD 20892 1202, USA
    JAMA 304:1358-64. 2010
    ..Variations in telomere length have been reported in severe aplastic anemia but their clinical significance is unknown...
  42. ncbi request reprint Transfer of glycosylphosphatidylinositol-anchored proteins to deficient cells after erythrocyte transfusion in paroxysmal nocturnal hemoglobinuria
    Elaine M Sloand
    Hematology Branch, National Heart, Lung, and Blood Institute, Department of Transfusion Meidcine, Clinical Center, National Instittues of Health, Bethesda, MD 20892, USA
    Blood 104:3782-8. 2004
    ..Increased surface CD59 was measured on recipient red cells and granulocytes 1, 3, and 7 days following transfusion in all 6 patients. Our data suggest a potential therapeutic role for GPI-anchored protein transfer for severe PNH...
  43. pmc Granulocyte transfusions in severe aplastic anemia: an eleven-year experience
    Karen Quillen
    Department of Transfusion Medicine, Warren G Magnuson Clinical Center, National Institutes of Health, Bethesda, MD, USA
    Haematologica 94:1661-8. 2009
    ..Infections, particularly those caused by invasive fungi, are a major cause of death in patients with severe aplastic anemia. The purpose of this study was to analyze our experience with granulocyte transfusions in such patients...
  44. pmc A spectrum of severe familial liver disorders associate with telomerase mutations
    Rodrigo T Calado
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland, United States of America
    PLoS ONE 4:e7926. 2009
    ..Here, we sought to determine the spectrum of clinical manifestations associated with telomerase loss-of-function mutations...
  45. pmc Long-term outcome of pediatric patients with severe aplastic anemia treated with antithymocyte globulin and cyclosporine
    Phillip Scheinberg
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    J Pediatr 153:814-9. 2008
    ....
  46. pmc Lymphocytes with aberrant expression of Fas or Fas ligand attenuate immune bone marrow failure in a mouse model
    Stephanie O Omokaro
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    J Immunol 182:3414-22. 2009
    ....
  47. pmc MHC class II upregulation and colocalization with Fas in experimental models of immune-mediated bone marrow failure
    Andrew J Erie
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892 1202, USA
    Exp Hematol 39:837-49. 2011
    ..To test the hypothesis that γ-interferon (IFN-γ) promotes major histocompatibility complex (MHC) class II expression on bone marrow (BM) cell targets that facilitate T-cell-mediated BM destruction in immune-mediated BM failure...
  48. ncbi request reprint Defective stromal cell function in a mouse model of infusion-induced bone marrow failure
    Jichun Chen
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892 1202, USA
    Exp Hematol 33:901-8. 2005
    ..To study bone marrow (BM) stromal damage in a mouse model of infusion-induced BM failure...
  49. ncbi request reprint Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long-term outcome
    Stephen Rosenfeld
    Hematology Branch, Bldg 10, National Heart, Lung, and Blood Institute, Bethesda, MD, USA
    JAMA 289:1130-5. 2003
    ..In most patients, aplastic anemia results from T-cell-mediated immune destruction of bone marrow. Aplastic anemia can be effectively treated by stem cell transplantation or immunosuppression...
  50. ncbi request reprint Elevated circulating endothelial membrane microparticles in paroxysmal nocturnal haemoglobinuria
    Jan Simak
    Laboratory of Cellular Hematology, Division of Hematology, Center for Biologics Evaluation and Research, Food and Drug Administration, Bethesda, MD, USA
    Br J Haematol 125:804-13. 2004
    ..Analysis of circulating ECMP appears promising to provide useful information on the status of the vascular endothelium in PNH and SCD...
  51. ncbi request reprint In-vivo dominant immune responses in aplastic anaemia: molecular tracking of putatively pathogenetic T-cell clones by TCR beta-CDR3 sequencing
    Antonio M Risitano
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Lancet 364:355-64. 2004
    ..In most cases, no obvious aetiological factor can be identified. However, clinical responses to immunosuppression strongly suggest an immune pathophysiology...
  52. ncbi request reprint Overcoming graft rejection in heavily transfused and allo-immunised patients with bone marrow failure syndromes using fludarabine-based haematopoietic cell transplantation
    Ramaprasad Srinivasan
    Urologic Oncology Branch, National Cancer Institute, Bethesda, MD, USA
    Br J Haematol 133:305-14. 2006
    ..These data show that HCT following fludarabine-based non-myeloablative conditioning results in durable engraftment and excellent survival in SAA and PNH patients at high risk for graft rejection...
  53. pmc Interphase Chromosome Flow-FISH
    Keyvan Keyvanfar
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Blood 120:e54-9. 2012
    ..IC Flow-FISH allows for detecting monosomy 7 without requiring bone marrow procurement or the necessity of metaphase spreads, and wider applications to other chromosomal abnormalities are in development...
  54. pmc Directed therapy for patients with myelodysplastic syndromes (MDS) by suppression of cyclin D1 with ON 01910.Na
    Matthew J Olnes
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Leuk Res 36:982-9. 2012
    ..Knockdown" of cyclin D1 by RNA interference decreased trisomy 8 cell growth, suggesting that this might be a therapeutic target in MDS...
  55. ncbi request reprint Decreased TCR zeta-chain expression in T cells from patients with acquired aplastic anaemia
    Elena E Solomou
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Br J Haematol 138:72-6. 2007
    ..We concluded that TCR zeta-chain expression was decreased in the majority of patients with aplastic anaemia, regardless of disease activity or treatment status...
  56. pmc Alemtuzumab treatment of intermediate-1 myelodysplasia patients is associated with sustained improvement in blood counts and cytogenetic remissions
    Elaine M Sloand
    Hematology Branch, National Heart, Lung, and Blood Institute, Bldg 10 CRC 4 5230, Bethesda, MD 20892, USA
    J Clin Oncol 28:5166-73. 2010
    ..Because of the toxicities associated with h-ATG/CsA, we investigated an alternative regimen with alemtuzumab in MDS...
  57. pmc Deficient CD4+ CD25+ FOXP3+ T regulatory cells in acquired aplastic anemia
    Elena E Solomou
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Blood 110:1603-6. 2007
    ..Our findings indicate that decreased NFAT1 could explain low FOXP3 expression and diminished Treg frequency in aplastic anemia. Treg defects are now implicated in autoimmune marrow failure...
  58. ncbi request reprint Late presentation of dyskeratosis congenita as apparently acquired aplastic anaemia due to mutations in telomerase RNA
    Patrick F Fogarty
    Hematology Branch, National Heart, Lung, and Blood Institute, 10 Center Drive, MSC 1652, Bethesda, MD 20892 1652, USA
    Lancet 362:1628-30. 2003
    ....
  59. pmc Increased soluble urokinase plasminogen activator receptor (suPAR) is associated with thrombosis and inhibition of plasmin generation in paroxysmal nocturnal hemoglobinuria (PNH) patients
    Elaine M Sloand
    National Heart Lung and Blood Institute, Hematology Branch, Office of Biostatistics Research, Division of Prevention and Population Sciences, National Institutes of Health, Bethesda, MD 20892 1260, USA
    Exp Hematol 36:1616-24. 2008
    ..We hypothesized that suPAR, prevents the interaction of urokinase with membrane-anchored uPAR on residual normal cells...
  60. ncbi request reprint Age-dependent accumulation of mtDNA mutations in murine hematopoietic stem cells is modulated by the nuclear genetic background
    Yong Gang Yao
    Hematology Branch, National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD 20892 1202, USA
    Hum Mol Genet 16:286-94. 2007
    ....
  61. pmc Sex hormones, acting on the TERT gene, increase telomerase activity in human primary hematopoietic cells
    Rodrigo T Calado
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892 1202, USA
    Blood 114:2236-43. 2009
    ..These findings have potential implications for the choice of current androgenic compounds and the development of future agents for clinical use...
  62. pmc Cytokine signature profiles in acquired aplastic anemia and myelodysplastic syndromes
    Xingmin Feng
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Haematologica 96:602-6. 2011
    ..Future studies focusing on cytokines that better discriminate these two entities such as thrombopoietin and chemokine (C-C motif) ligand 3 may be useful tools in clinical practice...
  63. pmc Long-term follow-up of patients with moderate aplastic anemia and pure red cell aplasia treated with daclizumab
    Elaine M Sloand
    National Heart Lung and Blood Instititute, NIH, Bethesda, MD 20892, USA
    Haematologica 95:382-7. 2010
    ....
  64. pmc Telomere length is inherited with resetting of the telomere set-point
    Y Jeffrey Chiang
    Experimental Immunology Branch, National Cancer Institute, Hematology Branch, National Heart, Lung and Blood Institute, and National Institute on Aging, National Institutes of Health, Bethesda, MD 20892, USA
    Proc Natl Acad Sci U S A 107:10148-53. 2010
    ..These findings suggest that the set point of telomere lengths of offspring is determined by the telomere lengths of their parents in the presence of normal expression of telomerase...
  65. pmc Paroxysmal nocturnal hemoglobinuria clones in severe aplastic anemia patients treated with horse anti-thymocyte globulin plus cyclosporine
    Phillip Scheinberg
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, 20892 1202, USA
    Haematologica 95:1075-80. 2010
    ..Flow cytometry has allowed for sensitive and precise measurement of glycosylphosphatidylinositol-anchor protein-deficient red blood cells and neutrophils in severe aplastic anemia...
  66. pmc T-cell immune responses to Wilms tumor 1 protein in myelodysplasia responsive to immunosuppressive therapy
    Elaine M Sloand
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Blood 117:2691-9. 2011
    ..Thus, our results suggest that WT1 is one of the antigens that triggers T cell-mediated myelosuppression in MDS...
  67. pmc Role of perforin-mediated cell apoptosis in murine models of infusion-induced bone marrow failure
    Annahita K Sarcon
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892 1202, USA
    Exp Hematol 37:477-86. 2009
    ..To investigate the role of perforin-mediated cell apoptosis in murine models of immune-mediated bone marrow (BM) failure...
  68. ncbi request reprint Oligoclonal and polyclonal CD4 and CD8 lymphocytes in aplastic anemia and paroxysmal nocturnal hemoglobinuria measured by V beta CDR3 spectratyping and flow cytometry
    Antonio M Risitano
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Blood 100:178-83. 2002
    ..As V beta skewing may correlate with relative V beta size, oligoclonality in combination with numerical V beta expansion can be applied to recognition of disease-specific T-cell receptors...
  69. pmc CD34 cells from patients with trisomy 8 myelodysplastic syndrome (MDS) express early apoptotic markers but avoid programmed cell death by up-regulation of antiapoptotic proteins
    Elaine M Sloand
    National Heart Lung and Blood Institute, Bethesda, MD 20892, USA
    Blood 109:2399-405. 2007
    ..Knock-down of survivin by siRNA resulted in preferential loss of trisomy 8 cells. These results suggest that trisomy 8 cells undergo incomplete apoptosis and are nonetheless capable of colony formation and growth...
  70. ncbi request reprint Recombinant viral-like particles of parvovirus B19 as antigen carriers of anthrax protective antigen
    Yoji Ogasawara
    Hematology Branch, National Heart, Lung and Blood Institute, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland 20892, USA
    In Vivo 20:319-24. 2006
    ..These findings suggest that the recombinant VLPs of parvovirus B19 have potential as an additional tool in the development of sub-unit vaccines...
  71. ncbi request reprint Mutations of the human telomerase RNA gene (TERC) in aplastic anemia and myelodysplastic syndrome
    Hiroki Yamaguchi
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, 10 Center Dr, MSC 1652, Bethesda, MD 20892 1652, USA
    Blood 102:916-8. 2003
    ..Another 21 patients with short telomeres did not show TERC mutations. Our results suggest that cryptic DKC, at least secondary to mutations in the TERC gene, is an improbable diagnosis in patients with otherwise typical AA, PNH, and MDS...
  72. ncbi request reprint Antithymocyte globulin for treatment of the bone marrow failure associated with myelodysplastic syndromes
    Jeffrey J Molldrem
    National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland 70892, USA
    Ann Intern Med 137:156-63. 2002
    ..Almost half of the deaths that result from myelodysplastic syndromes are due to cytopenia associated with bone marrow failure. Treatment is mostly supportive care...
  73. ncbi request reprint Superior growth of glycophosphatidy linositol-anchored protein-deficient progenitor cells in vitro is due to the higher apoptotic rate of progenitors with normal phenotype in vivo
    Guibin Chen
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Exp Hematol 30:774-82. 2002
    ..These results are consistent with the theory that PNH cells have an intrinsic growth advantage, but their superior expansion in vitro could also be the outcome of selective extrinsic pressure in vivo...
  74. ncbi request reprint Distinct clinical outcomes for cytogenetic abnormalities evolving from aplastic anemia
    Jaroslaw P Maciejewski
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Blood 99:3129-35. 2002
    ..Although AA patients with monosomy 7 showed a similar prognosis to those with primary MDS, trisomy 8 in AA appears to have a more favorable prognosis than in MDS...
  75. ncbi request reprint Immunosuppressive treatment of acquired aplastic anemia and immune-mediated bone marrow failure syndromes
    Neal S Young
    Hematology Branch, National Heart, Lung, and Blood Institute, NIH, Bethesda MD 20892 1652, USA
    Int J Hematol 75:129-40. 2002
    ..are under study. Effective therapies for aplastic anemia might also be applied to other T-cell mediated, organ-specific human diseases...
  76. ncbi request reprint Recombinant humanized anti-IL-2 receptor antibody (daclizumab) produces responses in patients with moderate aplastic anemia
    Jaroslaw P Maciejewski
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, 9000 Rockville Pike, Bethesda, MD 20892, USA
    Blood 102:3584-6. 2003
    ..Daclizumab appears safe; its efficacy in this pilot protocol suggests that expanded study of this monoclonal antibody in immune-mediated bone marrow failure syndrome is warranted...
  77. ncbi request reprint A pilot study of the recombinant soluble human tumour necrosis factor receptor (p75)-Fc fusion protein in patients with myelodysplastic syndrome
    Jaroslaw P Maciejewski
    Hematology Branch and Office of Biostatistics Research, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Br J Haematol 117:119-26. 2002
    ..Although anti-TNF therapy with Enbrel was well tolerated at the dosages used in MDS, its efficacy as a single agent appears low...
  78. ncbi request reprint Marked mitochondrial DNA sequence heterogeneity in single CD34+ cell clones from normal adult bone marrow
    Myung Geun Shin
    NIH 9000 Rockville Pike, Bethesda, MD 20892 1652, USA
    Blood 103:553-61. 2004
    ....
  79. ncbi request reprint Changes in T-cell receptor VB repertoire in aplastic anemia: effects of different immunosuppressive regimens
    Hoon Kook
    Hematology Branch of the National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Blood 99:3668-75. 2002
    ..Our data indicate that multiple specific clones mediate the immune process in AA...
  80. ncbi request reprint Acquired aplastic anemia
    Neal S Young
    National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland 20892 1652, USA
    Ann Intern Med 136:534-46. 2002
    ..However, recovery of blood cell count is often incomplete, recurrent pancytopenia requires retreatment, and some patients develop late complications (especially myelodysplasia)...
  81. ncbi request reprint Increased risk for aplastic anemia and myelodysplastic syndrome in individuals lacking glutathione S-transferase genes
    Joanne F Sutton
    Hematology Branch, NHLBI, NIH, Bethesda, MD, USA
    Pediatr Blood Cancer 42:122-6. 2004
    ..An absence of the glutathione S-transferase (GST) enzyme may genetically predispose individuals to AA or MDS...
  82. ncbi request reprint Prevalence of parvovirus B19 in liver tissue: no association with fulminant hepatitis or hepatitis-associated aplastic anemia
    Susan Wong
    Virus Discovery Group, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland 20892, USA
    J Infect Dis 187:1581-6. 2003
    ..There was a significant increase (P<.1) in the prevalence of variant erythrovirus sequences in livers of patients with HBV or HCV hepatitis, the reason for which is currently unknown...
  83. ncbi request reprint In vitro and in vivo evidence of PNH cell sensitivity to immune attack after nonmyeloablative allogeneic hematopoietic cell transplantation
    Yoshiyuki Takahashi
    Hematology Branch and Flow Cytometry Core Facility, National Heart, Lung, and Blood Institute, National Institutes of Health, 9000 Rockville Pike, Bethesda, MD 20892 1652, USA
    Blood 103:1383-90. 2004
    ..These in vitro and in vivo studies show PNH cells can be immunologically eradicated following nonmyeloablative HCT. Relative to normal cells, no evidence for a decreased sensitivity of PNH cells to T-cell-mediated immunity was observed...
  84. ncbi request reprint Mitochondrial DNA mutations in patients with myelodysplastic syndromes
    Myung Geun Shin
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Blood 101:3118-25. 2003
    ....
  85. pmc Dyskeratosis congenita: the first NIH clinical research workshop
    Sharon A Savage
    Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, National Institutes of Health, Rockville, Maryland 20892, USA
    Pediatr Blood Cancer 53:520-3. 2009
    ..dcoutreach.com/). Ongoing, open collaboration between the clinical, scientific, and family communities is required for continued improvement in our understanding of DC and the clinical consequences of telomeric defects...
  86. pmc Perforin gene mutations in patients with acquired aplastic anemia
    Elena E Solomou
    Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD 20892, USA
    Blood 109:5234-7. 2007
    ..Our data suggest that PRF1 genetic alterations help explain the aberrant proliferation and activation of cytotoxic T cells and may represent genetic risk factors for bone marrow failure...
  87. ncbi request reprint Paroxysmal nocturnal hemoglobinuria: current issues in pathophysiology and treatment
    Neal S Young
    National Heart, Lung, and Blood Institute, Building 10 CRC, 3E 5140 10 Center Drive, Bethesda, MD 20892 1202, USA
    Curr Hematol Rep 4:103-9. 2005
    ..The mechanism of thrombosis in PNH is also obscure, but frequently fatal clotting episodes may be prevented by Coumadin (Bristol-Myers Squibb Pharma Co., Wilmington, DE) prophylaxis...
  88. ncbi request reprint Bystander destruction of hematopoietic progenitor and stem cells in a mouse model of infusion-induced bone marrow failure
    Jichun Chen
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bldg 10, Rm 7C118, 9000 Rockville Pike, Bethesda, MD 20892 1652, USA
    Blood 104:1671-8. 2004
    ..This effect can be partially abrogated by anti-IFN-gamma antibody...
  89. doi request reprint Predicting response to immunosuppressive therapy and survival in severe aplastic anaemia
    Phillip Scheinberg
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD 20892 1202, USA
    Br J Haematol 144:206-16. 2009
    ..In the era of IST, the baseline ARC and ALC together serve as a simple predictor of response following IST, which should guide in risk stratification among patients with SAA...
  90. ncbi request reprint Parvovirus B19
    Neal S Young
    Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD 20892 1652, USA
    N Engl J Med 350:586-97. 2004
  91. ncbi request reprint Intracellular interferon-gamma in circulating and marrow T cells detected by flow cytometry and the response to immunosuppressive therapy in patients with aplastic anemia
    Elaine Sloand
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892 1652, USA
    Blood 100:1185-91. 2002
    ..IFN-gamma is increased in the PB lymphocytes of many patients with AA, and these cells decline with therapy. The presence of intracellular IFN-gamma may predict response to immunosuppressive treatment and also the onset of relapse...
  92. ncbi request reprint Selective reduction of natural killer T cells in the bone marrow of aplastic anaemia
    Weihua Zeng
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Br J Haematol 119:803-9. 2002
    ..These results show that NKT cells are profoundly decreased in AA and MDS, and their deficiency may, as in other human autoimmune diseases, play a role in the local immune dysregulation in AA and MDS...
  93. pmc Mitochondrial DNA sequence variation in single cells from leukemia patients
    Yong Gang Yao
    Hematology Branch and Flow Cytometry Core Facility, National Heart, Lung, and Blood Institute, NIH Bldg 10 CRC, Rm 3E 5216, 10 Center Dr Bethesda, MD 20892, USA
    Blood 109:756-62. 2007
    ..Our results suggest that the somatic mutation process in leukemia is complex, leading to diverse levels of genetic alterations due to either intrinsic aspects of leukemia pathophysiology or chemotherapy effects...
  94. pmc External contamination in single cell mtDNA analysis
    Yong Gang Yao
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland, United States of America
    PLoS ONE 2:e681. 2007
    ..External DNA contamination, notoriously difficult to avoid, threatens the integrity of such studies...
  95. ncbi request reprint The relationship of aplastic anemia and PNH
    Neal S Young
    Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD, USA
    Int J Hematol 76:168-72. 2002
    ....
  96. pmc Natural history of pulmonary fibrosis in two subjects with the same telomerase mutation
    Souheil El-Chemaly
    Cardiovascular and Pulmonary Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Chest 139:1203-9. 2011
    ..In familial pulmonary fibrosis, asymptomatic preclinical alveolar inflammation associated with mutation in TERT and telomerase insufficiency can progress to fibrotic lung disease over 2 to 3 decades...
  97. ncbi request reprint Decreased susceptibility of leukemic cells with PIG-A mutation to natural killer cells in vitro
    Shoichi Nagakura
    Second Department of Internal Medicine, Kumamoto University School of Medicine, Japan
    Blood 100:1031-7. 2002
    ..Our experiments suggest that PIG-A mutations confer a relative survival advantage to a PNH clone, contributing to selective expansion of these cells in the setting of marrow injury by cytotoxic lymphocytes...
  98. ncbi request reprint HLA-DR15 (DR2) is overrepresented in myelodysplastic syndrome and aplastic anemia and predicts a response to immunosuppression in myelodysplastic syndrome
    Yogen Saunthararajah
    University of Illinois at Chicago, USA
    Blood 100:1570-4. 2002
    ..008). In MDS with RA, DR15 may be useful as a guide to pathophysiology, prognosis, and treatment...
  99. ncbi request reprint Gene expression profiling in CD34 cells to identify differences between aplastic anemia patients and healthy volunteers
    Weihua Zeng
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Blood 103:325-32. 2004
    ..Many of the genes showing differential expression in AA deserve further detailed analysis, including comparison with other marrow failure states and autoimmune disease...
  100. ncbi request reprint Cytogenetic abnormalities in paroxysmal nocturnal haemoglobinuria usually occur in haematopoietic cells that are glycosylphosphatidylinositol-anchored protein (GPI-AP) positive
    Elaine M Sloand
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892 1652, USA
    Br J Haematol 123:173-6. 2003
    ..These findings suggest that environmental factors, but not genetic instability of the GPI-AP-deficient clone, foster development or survival of haematopoietic cells with chromosomal abnormalities...
  101. ncbi request reprint Molecular analysis of TCR clonotypes in LGL: a clonal model for polyclonal responses
    Christine L O'Keefe
    Experimental Hematology and Hematopoiesis Section, and Hematopathology Section, Cleveland Clinic Foundation, Cleveland, OH 44195, USA
    J Immunol 172:1960-9. 2004
    ....