脐带血造血干/祖细胞生物技术药物的研究现状
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摘要
脐带血作为造血干/祖细胞的三大来源之一,在造血干/祖细胞移植方面具有来源广泛、配型概率高、移植后排异反应轻和复发率低等独特优势,提高了许多血液及免疫系统疾病治疗的有效率。本文对近几年脐带血造血干/祖细胞生物技术药物的临床研究进行综述。
As one of the three major sources of hematopoie-tic stem/progenitor cells,cord blood has unique advantages in hematopoietic stem/progenitor cell transplantation,such as extensive sources,high probability of matching,light rejection after transplantation,and low recurrence rate,which has improved the treatment outcomes of many blood and immune system diseases. The clinical studies of cord blood hematopoietic stem/progenitor cell biotechnologydrugs in recent years are reviewed in this paper.
As one of the three major sources of hematopoie-tic stem/progenitor cells,cord blood has unique advantages in hematopoietic stem/progenitor cell transplantation,such as extensive sources,high probability of matching,light rejection after transplantation,and low recurrence rate,which has improved the treatment outcomes of many blood and immune system diseases. The clinical studies of cord blood hematopoietic stem/progenitor cell biotechnologydrugs in recent years are reviewed in this paper.
引文
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[2]ALLISON M.Hemacord approval may foreshadow regulatory creep for HSC therapies[J].Nature Biotechnology,2012,30(4):304.
[3]CHEZ M,LEPAGE C,PARISE C,et al.Safety and observations from a placebo-controlled,crossover study to assess use of autologous umbilical cord blood stem cells to improve symptoms in children with autism[J].Stem Cell Transal Med,2018,7(4):333-341.
[4]LU PEZ-GRANADOS L,TORRENT M,SASTRE A,et al.Reduced-intensity conditioning haematopoietic stem cell transplantation in genetic diseases:Experience of the Spanish working group for bone marrow transplantation in children[J].An Pediatr(Barc),2018,88(4):196-203.
[5]ISAEV A A,DEEV R V,KULIEV A,et al.First experience of hematopoietic stem cell transplantation treatment of ShwachmanDiamond syndrome using unaffected HLA matched sibling donor produced through preimplantation HLA typing[J].Bone Marrow Transpl,2017,52(9):1249-1252.
[6]LECCHI L,GIOVANELLI S,GAGLIARDI B,et al.An update on Methods for cryopreservation and thawing of hemopoietic stem cells[J].Transfus Apher Sci,2016,54(3):324-336.
[7]李雯瑛.二甲基亚砜与羟乙基淀粉联合冷冻保护外周造血干细胞的效果[J].中国医药指南,2016,14(33):17.
[8]FRY L J,QUEROL S,GOMEZ S G,et al.Assessing the toxic effects of DMSO on cord blood to determine exposure time limits and the optimum concentration for cryopreservation[J].Vox Sanguinis,2015,109(2):181-190.
[9]郭璐,许丽萍,范晓萍.急性移植物抗宿主病的病理生理学和治疗药物的研究状况[J].中国临床药理学杂志,2015,31(1):73-75.
[10]RANA S,PEARSE W,HAGEN P A,et al.Effect of antithymocyte globulin on outcomes following allogeneic hematopoietic cell transplantation for acute myelogenous leukemia[J].Biol Blood Marrow Transplant,2017,23(3 Supplement 1):294-295.
[11]SHONO Y,DOCAMPO M D,PELED J U,et al.Increased GVHD-related mortality with broad-spectrum antibiotic use after allogeneic hematopoietic stem cell transplantation in human patients and mice[J].Sci Transl Med,2016,8(339):339ra71.
[12]TAUR Y,JENQ R R,PERALES M A,et al.The effects of intestinal tract bacterial diversity on mortality following allogeneic hematopoietic stem cell transplantation[J].Blood,2014,124(7):1174-1182.
[13]MATHEWSON N D,JENQ R,MATHEW A V,et al.Gut microbiome-derived metabolites modulate intestinal epithelial cell damage and mitigate graft-versus-host disease[J].Nat Immunol,2016,17(5):505-513.
[14]DUARTE R,MARTY F,LJUNGMAN P,et al.Letermovir for prevention of cytomegalovirus infection in adult CMV-seropositive recipients of allogeneic hematopoietic cell transplantation[J].Haematologica,2017,102(Supplement 2):331-332.
[15]GRIGORIADIS N G,GRIGORIADIS I G,MARKOULA S,et al.Pharmacological preconditioning for short-term ex vivo expansion of human umbilical cord blood hematopoietic stem cells by filgrastim[J].Am J Stem Cells,2016,5(1):29-38.
[16]JAROSCAK J,GOLTRY K,SMITH A,et al.Augmentation of umbilical cord blood(UCB)transplantation with ex vivo-expanded UCB cells:results of a phase 1 trial using the Aastrom Replicell System[J].Blood,2003,101(12):5061-5067.
[17]DE LIMA M,MCNIECE I,ROBINSON S N,et al.Cord-blood engraftment with ex vivo mesenchymal-cell coculture[J].N Engl J Med,2012,367(24):2305-2315.
[18]ANAND S,THOMAS S,HYSLOP T,et al.Transplantation of ex vivo expanded umbilical cord blood(Ni Cord)decreases early infection and hospitalization[J].Biol Blood Marrow Tr,2017,23(7):1151-1157.
[19]HORWITZ M E,CILLONI D,VALCARCEL D,et al.Nicord single unit expanded umbilical cord blood transplantation(UCBT):Final results of a multicenter phase I/II trial[J].Biol Blood Marrow Tr,2018,24(3):57.
[20]HORWITZ M E,CHAO N J,RIZZIERI D A,et al.Umbilical cord blood expansion with nicotinamide provides long-term,multilineage engraftment[J].J Clin Invest,2014,124(7):3121-3128.
[21]PARIKH S H,BROCHSTEIN J,MARTIN P L,et al.Successful engraftment of donor umbilical cord blood(UCB)cells after cotransplantation of nicord(ex vivo expanded UCB progenitor cells with nicotinamide)and a second unmanipulated cord blood unit after myeloablative chemotherapy in pediatric patients with severe sickle cell disease[J].Blood,2016,22(128):3651.
[22]WAGNER J E,BRUNSTEIN C G,BOITANO A E,et al.PhaseⅠ/Ⅱtrial of stemregenin-1 expanded umbilical cord blood hematopoietic stem cells supports testing as a stand-alone graft[J].Cell Stem Cell,2016,18(1):144-155.
[23]STIFF P J,MONTESINOS P,PELED T,et al.StemEx(Copper Chelation Based)ex vivo expanded umbilical cord blood stem cell transplantation(UCBT)accelerates engraftment and improves 100day survival in myeloablated patients compared to a registry cohort undergoing double unit UCBT:Results of a multicenter study of 101patients with hematologic malignancies[J].Blood,2013,21(122):295.
[24]SUGIYAMA D,YUMINE A,MATSUMOTO M,et al.Bioactive SL-13R peptide expands human umbilical cord blood hematopoietic stem cells and maintains long-term reconstitution ability[J].Blood,2017,130(Suppl 1):4938.
[2]ALLISON M.Hemacord approval may foreshadow regulatory creep for HSC therapies[J].Nature Biotechnology,2012,30(4):304.
[3]CHEZ M,LEPAGE C,PARISE C,et al.Safety and observations from a placebo-controlled,crossover study to assess use of autologous umbilical cord blood stem cells to improve symptoms in children with autism[J].Stem Cell Transal Med,2018,7(4):333-341.
[4]LU PEZ-GRANADOS L,TORRENT M,SASTRE A,et al.Reduced-intensity conditioning haematopoietic stem cell transplantation in genetic diseases:Experience of the Spanish working group for bone marrow transplantation in children[J].An Pediatr(Barc),2018,88(4):196-203.
[5]ISAEV A A,DEEV R V,KULIEV A,et al.First experience of hematopoietic stem cell transplantation treatment of ShwachmanDiamond syndrome using unaffected HLA matched sibling donor produced through preimplantation HLA typing[J].Bone Marrow Transpl,2017,52(9):1249-1252.
[6]LECCHI L,GIOVANELLI S,GAGLIARDI B,et al.An update on Methods for cryopreservation and thawing of hemopoietic stem cells[J].Transfus Apher Sci,2016,54(3):324-336.
[7]李雯瑛.二甲基亚砜与羟乙基淀粉联合冷冻保护外周造血干细胞的效果[J].中国医药指南,2016,14(33):17.
[8]FRY L J,QUEROL S,GOMEZ S G,et al.Assessing the toxic effects of DMSO on cord blood to determine exposure time limits and the optimum concentration for cryopreservation[J].Vox Sanguinis,2015,109(2):181-190.
[9]郭璐,许丽萍,范晓萍.急性移植物抗宿主病的病理生理学和治疗药物的研究状况[J].中国临床药理学杂志,2015,31(1):73-75.
[10]RANA S,PEARSE W,HAGEN P A,et al.Effect of antithymocyte globulin on outcomes following allogeneic hematopoietic cell transplantation for acute myelogenous leukemia[J].Biol Blood Marrow Transplant,2017,23(3 Supplement 1):294-295.
[11]SHONO Y,DOCAMPO M D,PELED J U,et al.Increased GVHD-related mortality with broad-spectrum antibiotic use after allogeneic hematopoietic stem cell transplantation in human patients and mice[J].Sci Transl Med,2016,8(339):339ra71.
[12]TAUR Y,JENQ R R,PERALES M A,et al.The effects of intestinal tract bacterial diversity on mortality following allogeneic hematopoietic stem cell transplantation[J].Blood,2014,124(7):1174-1182.
[13]MATHEWSON N D,JENQ R,MATHEW A V,et al.Gut microbiome-derived metabolites modulate intestinal epithelial cell damage and mitigate graft-versus-host disease[J].Nat Immunol,2016,17(5):505-513.
[14]DUARTE R,MARTY F,LJUNGMAN P,et al.Letermovir for prevention of cytomegalovirus infection in adult CMV-seropositive recipients of allogeneic hematopoietic cell transplantation[J].Haematologica,2017,102(Supplement 2):331-332.
[15]GRIGORIADIS N G,GRIGORIADIS I G,MARKOULA S,et al.Pharmacological preconditioning for short-term ex vivo expansion of human umbilical cord blood hematopoietic stem cells by filgrastim[J].Am J Stem Cells,2016,5(1):29-38.
[16]JAROSCAK J,GOLTRY K,SMITH A,et al.Augmentation of umbilical cord blood(UCB)transplantation with ex vivo-expanded UCB cells:results of a phase 1 trial using the Aastrom Replicell System[J].Blood,2003,101(12):5061-5067.
[17]DE LIMA M,MCNIECE I,ROBINSON S N,et al.Cord-blood engraftment with ex vivo mesenchymal-cell coculture[J].N Engl J Med,2012,367(24):2305-2315.
[18]ANAND S,THOMAS S,HYSLOP T,et al.Transplantation of ex vivo expanded umbilical cord blood(Ni Cord)decreases early infection and hospitalization[J].Biol Blood Marrow Tr,2017,23(7):1151-1157.
[19]HORWITZ M E,CILLONI D,VALCARCEL D,et al.Nicord single unit expanded umbilical cord blood transplantation(UCBT):Final results of a multicenter phase I/II trial[J].Biol Blood Marrow Tr,2018,24(3):57.
[20]HORWITZ M E,CHAO N J,RIZZIERI D A,et al.Umbilical cord blood expansion with nicotinamide provides long-term,multilineage engraftment[J].J Clin Invest,2014,124(7):3121-3128.
[21]PARIKH S H,BROCHSTEIN J,MARTIN P L,et al.Successful engraftment of donor umbilical cord blood(UCB)cells after cotransplantation of nicord(ex vivo expanded UCB progenitor cells with nicotinamide)and a second unmanipulated cord blood unit after myeloablative chemotherapy in pediatric patients with severe sickle cell disease[J].Blood,2016,22(128):3651.
[22]WAGNER J E,BRUNSTEIN C G,BOITANO A E,et al.PhaseⅠ/Ⅱtrial of stemregenin-1 expanded umbilical cord blood hematopoietic stem cells supports testing as a stand-alone graft[J].Cell Stem Cell,2016,18(1):144-155.
[23]STIFF P J,MONTESINOS P,PELED T,et al.StemEx(Copper Chelation Based)ex vivo expanded umbilical cord blood stem cell transplantation(UCBT)accelerates engraftment and improves 100day survival in myeloablated patients compared to a registry cohort undergoing double unit UCBT:Results of a multicenter study of 101patients with hematologic malignancies[J].Blood,2013,21(122):295.
[24]SUGIYAMA D,YUMINE A,MATSUMOTO M,et al.Bioactive SL-13R peptide expands human umbilical cord blood hematopoietic stem cells and maintains long-term reconstitution ability[J].Blood,2017,130(Suppl 1):4938.