Unrelated donor allogeneic hematopoietic stem cell transplantation for patients with hemoglobinopathies using a reduced-intensity conditioning regimen and third-party mesenchymal stromal cells

Biology of Blood and Marrow Transplantation

Volumn 20, Issue 4, 2014, Pages 581-586

  Kharbanda, Sandhya ^a   Smith, Angela R ^b   Hutchinson, Stephanie K ^a   McKenna, David H ^c   Ball, James B ^d   Lamb, Lawrence S ^e   Agarwal, Rajni ^a   Weinberg, Kenneth I ^a   Wagner, John E ^b  

^a STANFORD UNIVERSITY   (United States)

^b UNIVERSITY OF MINNESOTA   (United States)

^c UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^d Rocky Mountain Hospital for Children   (United States)

^e University of Alabama at Birmingham   (United States)

Author keywords

Bone marrow; Engraftment; Graft versus host disease; Hematopoietic stem cell transplant; Hemoglobinopathies; Mesenchymal stromal cells; Reduced intensity conditioning; Sickle cell disease; Thalassemia; Umbilical cord

Indexed keywords

ALEMTUZUMAB; CYCLOSPORIN A; FLUDARABINE; MELPHALAN; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER;

ADOLESCENT; ADULT; ALLELE; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ARTICLE; BONE MARROW; BRAIN HEMORRHAGE; CHILD; CHIMERA; CLINICAL ARTICLE; DONOR; ENGRAFTMENT; FEMALE; GRAFT FAILURE; GRAFT VERSUS HOST REACTION; HEMOGLOBINOPATHY; HLA SYSTEM; HUMAN; MALE; MESENCHYMAL STROMA CELL; MORTALITY; OPPORTUNISTIC INFECTION; PILOT STUDY; REDUCED INTENSITY CONDITIONING; SCHOOL CHILD; UMBILICAL CORD BLOOD; UNRELATED DONORS; YOUNG ADULT;

BONE MARROW; ENGRAFTMENT; GRAFT-VERSUS-HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANT; HEMOGLOBINOPATHIES; MESENCHYMAL STROMAL CELLS; REDUCED-INTENSITY CONDITIONING; SICKLE CELL DISEASE; THALASSEMIA; UMBILICAL CORD;

ADOLESCENT; ANEMIA, SICKLE CELL; ANTIBODIES, MONOCLONAL, HUMANIZED; BETA-THALASSEMIA; CHILD; CORD BLOOD STEM CELL TRANSPLANTATION; FEMALE; GRAFT VS HOST DISEASE; HISTOCOMPATIBILITY TESTING; HLA ANTIGENS; HUMANS; MALE; MELPHALAN; MESENCHYMAL STEM CELL TRANSPLANTATION; MYELOABLATIVE AGONISTS; SURVIVAL ANALYSIS; TRANSPLANTATION CONDITIONING; TRANSPLANTATION, HOMOLOGOUS; TREATMENT FAILURE; UNRELATED DONORS; VIDARABINE;

EID: 84895812218     PISSN: 10838791     EISSN: 15236536     Source Type: Journal    
DOI: 10.1016/j.bbmt.2013.12.564     Document Type: Article

References (54)

1. Lucarelli G., Galimberti M., Polchi P., et al. Bone marrow transplantation in children and in adults with thalassemia. Bone Marrow Transplant 1991, 7(Suppl. 2):72.
2. Walters M.C., Patience M., Leisenring W., et al. Bone marrow transplantation for sickle cell disease. NEngl J Med 1996, 335:369-376.
3. Parkman R. Bone marrow transplantation for genetic diseases. Pediatr Ann 1991, 20:677-681.
4. Johnson F.L., Mentzer W.C., Kalinyak K.A., et al. Bone marrow transplantation for sickle cell disease. The United States experience. Am J Pediatr Hematol Oncol 1994, 16:22-26.
5. Bernaudin F., Souillet G., Vannier J.P., et al. Bone marrow transplantation (BMT) in 14 children with severe sickle cell disease (SCD): the French experience. GEGMO. Bone Marrow Transplant 1993, 12(Suppl. 1):118-121.
6. Bernaudin F. Results and current indications of bone marrow allograft in sickle cell disease [in French]. Pathol Biol (Paris) 1999, 47:59-64.
7. Walters M.C., Storb R., Patience M., et al. 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.
8. Vermylen C., Cornu G., Ferster A., et al. Bone marrow transplantation in sickle cell disease: the Belgian experience. Bone Marrow Transplant 1993, 12(Suppl. 1):116-117.
9. Vermylen C., Cornu G. Bone marrow transplantation for sickle cell disease. The European experience. Am J Pediatr Hematol Oncol 1994, 16:18-21.
10. Locatelli F., Rocha V., Reed W., et al. Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. Blood 2003, 101:2137-2143.
11. 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.
12. Gore L., Lane P.A., Quinones R.R., Giller R.H. Successful cord blood transplantation for sickle cell anemia from a sibling who is human leukocyte antigen-identical: implications for comprehensive care. JPediatr Hematol Oncol 2000, 22:437-440.
13. Walters M.C., Hardy K., Edwards S., et al. Pulmonary, gonadal, and central nervous system status after bone marrow transplantation for sickle cell disease. Biol Blood Marrow Transplant 2010, 16:263-272.
14. Iannone R., Casella J.F., Fuchs E.J., et al. Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia. Biol Blood Marrow Transplant 2003, 9:519-528.
15. Krishnamurti L., Blazar B.R., Wagner J.E. Bone marrow transplantation without myeloablation for sickle cell disease. NEngl J Med 2001, 344:68.
16. Krishnamurti L., Kharbanda S., Biernacki M.A., et al. Stable long-term donor engraftment following reduced-intensity hematopoietic cell transplantation for sickle cell disease. Biol Blood Marrow Transplant 2008, 14:1270-1278.
17. Shenoy S., Grossman W.J., DiPersio J., et al. Anovel reduced-intensity stem cell transplant regimen for nonmalignant disorders. Bone Marrow Transplant 2005, 35:345-352.
18. Jacobsohn D.A., Duerst R., Tse W., Kletzel M. Reduced intensity haemopoietic stem-cell transplantation for treatment of non-malignant diseases in children. Lancet 2004, 364:156-162.
19. Adamkiewicz T.V., Szabolcs P., Haight A., et al. Unrelated cord blood transplantation in children with sickle cell disease: review of four-center experience. Pediatr Transplant 2007, 11:641-644.
20. Kamani N.R., Walters M.C., Carter S., et al. Unrelated donor cord blood transplantation for children with severe sickle cell disease: results of one cohort from the phase II study from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). Biol Blood Marrow Transplant 2012, 18:1265-1272.
21. Bolanos-Meade J., Fuchs E.J., Luznik L., et al. HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease. Blood 2012, 120:4285-4291.
22. Wang S.B., Hu D.M., Li L., et al. HLA haploidentical peripheral blood stem cells transplantation for beta thalassemia major [in Chinese]. Zhonghua Xue Ye Xue Za Zhi 2011, 32:844-847.
23. Sodani P., Isgro A., Gaziev J., et al. Tcell-depleted HLA-haploidentical stem cell transplantation in thalassemia young patients. Pediatr Rep 2011, 3(Suppl. 2):e13.
24. Caplan A.I. Mesenchymal stem cells. JOrthop Res 1991, 9:641-650.
25. Caplan A.I. The mesengenic process. Clin Plast Surg 1994, 21:429-435.
26. Pittenger M.F., Mackay A.M., Beck S.C., et al. Multilineage potential of adult human mesenchymal stem cells. Science 1999, 284:143-147.
27. Prockop D.J. Marrow stromal cells as stem cells for nonhematopoietic tissues. Science 1997, 276:71-74.
28. Deans R.J., Moseley A.B. Mesenchymal stem cells: biology and potential clinical uses. Exp Hematol 2000, 28:875-884.
29. Koc O.N., Gerson S.L., Cooper B.W., et al. Rapid hematopoietic recovery after coinfusion of autologous-blood stem cells and culture-expanded marrow mesenchymal stem cells in advanced breast cancer patients receiving high-dose chemotherapy. JClin Oncol 2000, 18:307-316.
30. Lazarus H.M., Koc O.N., Devine S.M., et al. Cotransplantation of HLA-identical sibling culture-expanded mesenchymal stem cells and hematopoietic stem cells in hematologic malignancy patients. Biol Blood Marrow Transplant 2005, 11:389-398.
31. Macmillan M.L., Blazar B.R., DeFor T.E., Wagner J.E. Transplantation of ex-vivo culture-expanded parental haploidentical mesenchymal stem cells to promote engraftment in pediatric recipients of unrelated donor umbilical cord blood: results of a phase I-II clinical trial. Bone Marrow Transplant 2009, 43:447-454.
32. Dominici M., Le Blanc K., Mueller I., et al. Minimal criteria for defining multipotent mesenchymal stromal cells. The International Society for Cellular Therapy position statement. Cytotherapy 2006, 8:315-317.
33. Simon R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials 1989, 10:1-10.
34. Horwitz E.M., Prockop D.J., Fitzpatrick L.A., et al. Transplantability and therapeutic effects of bone marrow-derived mesenchymal cells in children with osteogenesis imperfecta. Nat Med 1999, 5:309-313.
35. Koc O.N., Day J., Nieder M., et al. Allogeneic mesenchymal stem cell infusion for treatment of metachromatic leukodystrophy (MLD) and Hurler syndrome (MPS-IH). Bone Marrow Transplant 2002, 30:215-222.
36. Lee S.T., Jang J.H., Cheong J.W., et al. Treatment of high-risk acute myelogenous leukaemia by myeloablative chemoradiotherapy followed by co-infusion of T cell-depleted haematopoietic stem cells and culture-expanded marrow mesenchymal stem cells from a related donor with one fully mismatched human leucocyte antigen haplotype. Br J Haematol 2002, 118:1128-1131.
37. Gonzalo-Daganzo R., Regidor C., Martin-Donaire T., et al. Results of a pilot study on the use of third-party donor mesenchymal stromal cells in cord blood transplantation in adults. Cytotherapy 2009, 11:278-288.
38. Le Blanc K., Samuelsson H., Gustafsson B., et al. Transplantation of mesenchymal stem cells to enhance engraftment of hematopoietic stem cells. Leukemia 2007, 21:1733-1738.
39. Horwitz E.M., Gordon P.L., Koo W.K., et al. Isolated allogeneic bone marrow-derived mesenchymal cells engraft and stimulate growth in children with osteogenesis imperfecta: implications for cell therapy of bone. Proc Natl Acad Sci USA 2002, 99:8932-8937.
40. Fouillard L., Bensidhoum M., Bories D., et al. Engraftment of allogeneic mesenchymal stem cells in the bone marrow of a patient with severe idiopathic aplastic anemia improves stroma. Leukemia 2003, 17:474-476.
41. Koc O.N., Peters C., Aubourg P., et al. Bone marrow-derived mesenchymal stem cells remain host-derived despite successful hematopoietic engraftment after allogeneic transplantation in patients with lysosomal and peroxisomal storage diseases. Exp Hematol 1999, 27:1675-1681.
42. Glennie S., Soeiro I., Dyson P.J., et al. Bone marrow mesenchymal stem cells induce division arrest anergy of activated T cells. Blood 2005, 105:2821-2827.
43. Aggarwal S., Pittenger M.F. Human mesenchymal stem cells modulate allogeneic immune cell responses. Blood 2005, 105:1815-1822.
44. Beyth S., Borovsky Z., Mevorach D., et al. Human mesenchymal stem cells alter antigen-presenting cell maturation and induce T-cell unresponsiveness. Blood 2005, 105:2214-2219.
45. Krampera M., Glennie S., Dyson J., et al. Bone marrow mesenchymal stem cells inhibit the response of naive and memory antigen-specific T cells to their cognate peptide. Blood 2003, 101:3722-3729.
46. Maccario R., Moretta A., Cometa A., et al. Human mesenchymal stem cells and cyclosporin a exert a synergistic suppressive effect on invitro activation of alloantigen-specific cytotoxic lymphocytes. Biol Blood Marrow Transplant 2005, 11:1031-1032.
47. Ringden O., Uzunel M., Rasmusson I., et al. Mesenchymal stem cells for treatment of therapy-resistant graft-versus-host disease. Transplantation 2006, 81:1390-1397.
48. Le Blanc K., Frassoni F., Ball L., et al. Mesenchymal stem cells for treatment of steroid-resistant, severe, acute graft-versus-host disease: a phase II study. Lancet 2008, 371:1579-1586.
49. von Bonin M., Stolzel F., Goedecke A., et al. Treatment of refractory acute GVHD with third-party MSC expanded in platelet lysate-containing medium. Bone Marrow Transplant 2009, 43:245-251.
50. von Bahr L., Sundberg B., Lonnies L., et al. Long-term complications, immunologic effects, and role of passage for outcome in mesenchymal stromal cell therapy. Biol Blood Marrow Transplant 2012, 18:557-564.
51. Milano F., Pergam S.A., Xie H., et al. Intensive strategy to prevent CMV disease in seropositive umbilical cord blood transplant recipients. Blood 2011, 118:5689-5696.
52. Leen A.M., Christin A., Myers G.D., et al. Cytotoxic T lymphocyte therapy with donor T cells prevents and treats adenovirus and Epstein-Barr virus infections after haploidentical and matched unrelated stem cell transplantation. Blood 2009, 114:4283-4292.
53. Blyth E., Clancy L., Simms R., et al. Donor-derived CMV-specific T cells reduce the requirement for CMV-directed pharmacotherapy after allogeneic stem cell transplantation. Blood 2013, 121:3745-3758.
54. Gerdemann U., Keirnan J.M., Katari U.L., et al. Rapidly generated multivirus-specific cytotoxic T lymphocytes for the prophylaxis and treatment of viral infections. Mol Ther 2012, 20:1622-1632.