A pilot study of reduced intensity conditioning and allogeneic stem cell transplantation from unrelated cord blood and matched family donors in children and adolescent recipients

Bone Marrow Transplantation

Volumn 33, Issue 6, 2004, Pages 613-622

  Del Toro, G ^a   Satwani, P ^a   Harrison, L ^a   Cheung, Y K ^a   Brigid Bradley, M ^a   George, D ^a   Yamashiro, D J ^a   Garvin, J ^a   Skerret, D ^a   Bessmertny, O ^a   Wolownik, K ^a   Wischhover, C ^a   van de Ven, C ^a   Cairo, M S ^a  

^a Och Spine at New York Presbyterian Hospitals   (United States)

Author keywords

Allogeneic; Chimerism; Pediatric; Reduced intensity; Umbilical cord blood

Indexed keywords

ACICLOVIR; ALEMTUZUMAB; AMPHOTERICIN B LIPID COMPLEX; BUSULFAN; CD34 ANTIGEN; COTRIMOXAZOLE; CYCLOPHOSPHAMIDE; ETOPOSIDE; FLUDARABINE; FOSCARNET; GANCICLOVIR; GRANULOCYTE COLONY STIMULATING FACTOR; GRANULOCYTE MACROPHAGE COLONY STIMULATING FACTOR; LYMPHOCYTE ANTIBODY; MELPHALAN; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PENTAMIDINE; TACROLIMUS; THYMOCYTE ANTIBODY;

ADOLESCENT; ADULT; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; APLASTIC ANEMIA; ARTICLE; BETA THALASSEMIA; BLOOD DONOR; BONE MARROW TRANSPLANTATION; CANDIDIASIS; CHILD; CHIMERA; CHRONIC MYELOID LEUKEMIA; CLINICAL ARTICLE; COLITIS; CONTROLLED STUDY; DOSE RESPONSE; FAMILY; FEMALE; GASTROENTERITIS; GASTROINTESTINAL INFECTION; GRAFT FAILURE; GRAFT VERSUS HOST REACTION; HEMORRHAGIC CYSTITIS; HERPES SIMPLEX; HERPES ZOSTER; HLA MATCHING; HODGKIN DISEASE; HUMAN; INFANT; INFLUENZA; LEUKOENCEPHALOPATHY; LIVER ABSCESS; LIVER DYSFUNCTION; LIVER TOXICITY; LUNG INFECTION; MALE; MYCOSIS; NAUSEA; NEPHROBLASTOMA; NEUROBLASTOMA; NEUROTOXICITY; NONHODGKIN LYMPHOMA; PANCREATITIS; PERIPHERAL BLOOD STEM CELL TRANSPLANTATION; PILOT STUDY; PNEUMOCYSTOSIS; PNEUMONIA; PRIORITY JOURNAL; RECIPIENT; RNA VIRUS INFECTION; SEPSIS; SEX RATIO; UMBILICAL CORD BLOOD; UPPER RESPIRATORY TRACT INFECTION; VOMITING; WHOLE BODY RADIATION; WISKOTT ALDRICH SYNDROME;

ADOLESCENT; ADULT; ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS; CHILD; CHILD, PRESCHOOL; FAMILY; FEMALE; FETAL BLOOD; GRAFT VS HOST DISEASE; HUMANS; IMMUNOSUPPRESSIVE AGENTS; INFANT; MALE; NEOPLASMS; STEM CELL TRANSPLANTATION; SURVIVAL ANALYSIS; TIME FACTORS; TISSUE DONORS; TRANSPLANTATION CHIMERA; TRANSPLANTATION CONDITIONING; TRANSPLANTATION, HOMOLOGOUS;

EID: 11144355242     PISSN: 02683369     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.bmt.1704399     Document Type: Article

References (54)

1. Wagner JE, Rosenthal J, Sweetman R et al. Successful transplantation of HLA-matched and HLA-mismatched umbilical cord blood from unrelated donors: Analysis of engraftment and acute graft-versus-host disease. Blood 1996; 88: 795-802.
2. Cairo MS, Wagner JE. Placental and/or umbilical cord blood: an alternative source of hematopoietic stem cells for transplantation. Blood 1997; 90: 4665-4678.
3. Laughlin MJ, Barker J, Bambach B et al. Hematopoietic engraftment and survival in adult recipients of umbilical cord blood from unrelated donors. N Engl J Med 2001; 344: 1815-1822.
4. Kurtzberg J, Laughlin M, Graham ML et al. Placental blood as a source of hematopoietic stem cells for transplantation into unrelated recipients. N Engl J Med 1996; 335: 157-166.
5. Rubinstein P, Carrier C, Scaradavou A et al. Outcomes among 562 recipients of placental-blood transplants from unrelated donors. N Engl J Med 1998; 339: 1565-1577.
6. Rocha V, Wagner JE, Sobocinski KA et al. Graft-versus-host disease in children who have received a cord-blood or bone marrow transplant from an HLA-identical sibling. N Engl J Med 2000; 342: 1846-1854.
7. Gluckman E, Rocha V, Boyer-Chammard A et al. Outcome of cord-blood transplantation from related and unrelated donors. Eurocord Transplant Group and the European Blood and Marrow Transplantation Group. N Engl J Med 1997; 337: 373-381.
8. Storb R, Yu C, Wagner JL et al. 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.
9. Georges GE, Storb R, Thompson JD et al. Adoptive immunotherapy in canine mixed chimeras after nonmyeloablative hematopoietic cell transplantation. Blood 2000; 95: 3262-3269.
10. Slavin S, Nagler A, Naparstek E et al. Nonmyeloablative stem cell transplantation and cell therapy as an alternative to conventional bone marrow transplantation with lethal cytoreduction for the treatment of malignant and nonmalignant hematologic diseases. Blood 1998; 91: 756-763.
11. Bornhauser M, Thiede C, Schuler U et al. Dose-reduced conditioning for allogeneic blood stem cell transplantation: Durable engraftment without antithymocyte globulin. Bone Marrow Transplant 2000; 26: 119-125.
12. Giralt S, Thall PF, Khouri I et al. Melphalan and purine analog-containing preparative regimens: Reduced-intensity conditioning for patients with hematologic malignancies undergoing allogeneic progenitor cell transplantation. Blood 2001; 97: 631-637.
13. Spitzer TR, McAfee S, Sackstein R et al. Intentional induction of mixed chimerism, and achievement of antitumor responses after nonmyeloablative conditioning therapy and HLA-matched donor bone marrow transplantation for refractory hematologic malignancies. Biol Blood Marrow Transplant 2000; 6: 309-320.
14. Childs R, Chernoff A, Contentin N et al. Regression of metastatic renal-cell carcinoma after nonmyeloablative allogeneic peripheral-blood stem-cell transplantation. N Engl J Med 2000; 343: 750-758.
15. Nagler A, Slavin S, Varadi G et al. Allogeneic peripheral blood stem cell transplantation using a fludarabine-based low intensity conditioning regimen for malignant lymphoma. Bone Marrow Transplant 2000; 25: 1021-1028.
16. Khouri IF, Saliba RM, Giralt SA et al. Nonablative allogeneic hematopoietic transplantation as adoptive immunotherapy for indolent lymphoma: Low incidence of toxicity, acute graft-versus-host disease, and treatment-related mortality. Blood 2001; 98: 3595-3599.
17. Anderlini P, Giralt S, Andersson B et al. Allogeneic stem cell transplantation with fludarabine-based, less intensive conditioning regimens as adoptive immunotherapy in advanced Hodgkin's disease. Bone Marrow Transplant 2000; 26: 615-620.
18. Bornhauser M, Thiede C, Platzbecker U et al. Dose-reduced conditioning and allogeneic hematopoietic stem cell transplantation from unrelated donors in 42 patients. Clin Cancer Res 2001; 7: 2254-2262.
19. Nagler A, Aker M, Or R et al. Low-intensity conditioning is sufficient to ensure engraftment in matched unrelated bone marrow transplantation. Exp Hematol 2001; 29: 362-370.
20. Friedman TM, Varadi G, Hopely DD et al. Nonmyeloablative conditioning allows for more rapid T-cell repertoire reconstitution following allogeneic matched unrelated bone marrow transplantation compared to myeloablative approaches. Biol Blood Marrow Transplant 2001; 7: 656-664.
21. Chakraverty R, Peggs K, Chopra R et al. Limiting transplantation-related mortality following unrelated donor stem cell transplantation by using a nonmyeloablative conditioning regimen. Blood 2002; 99: 1071-1078.
22. Amrolia P, Gaspar HB, Hassan A et al. Nonmyeloablative stem cell transplantation for congenital immunodeficiencies. Blood 2000; 96: 1239-1246.
23. Fraser J, Cairo MS, Wagner EL et al. Cord blood transplantation study (COBLT): Cord Blood Bank Standard Operating Procedures. J Hematother 1998; 7: 521-561.
24. Rubinstein P, Dobrila L, Rosenfield RE et al. Processing and cryopreservation of placental/umbilical cord blood for unrelated bone marrow reconstitution. Proc Natl Acad Sci USA 1995; 92: 10119-10122.
25. Cairo MS, Harrison LA, Ellis H et al. Tacrolimus (FK506) and mycophenolate mofetil (MMF) is a safe and potentially effective alternative GVHD prophylaxis regimen in related (R) and unrelated (UR) donor allogeneic SCT (AlloSCT). Blood 2001, (abstract) 98: 662a.
26. Cairo MS, Szymanski J, Harrrison LA et al. A pilot study of AmBisome® prophylaxis in patients undergoing allogeneic related and unrelated stem cell transplantation (AlloSCT). Blood 2001, (abstract) 98: 354b.
27. Shereck EB, Bessmertny O, Cooney EM et al. Cytomegalovirus (CMB) prophylaxis with alternate day ganciclovir/ foscarnet in at risk pediatric allogeneic stem cell transplant (AlloSCT) recipients is 100% effective in preventing CMV infections and reducing ganciclovir hematopoietic toxicity. Blood 2002, (abstract) 100: 627a.
28. Luhm RA, Bellissimo DB, Uzgiris AJ et al. Quantitative evaluation of post-bone marrow transplant engraftment status using fluorescent-labeled variable number of tandem repeats. Mol Diagn 2000; 5: 129-138.
29. Common Toxicity Criteria Version 20. National Cancer Institute: Bethesda MD, 1999.
30. Glucksberg H, Storb R, Fefer A et al. Clinical manifestations of graft-versus-host disease in human recipients of marrow from HL-A-matched sibling donors. Transplantation 1974; 18: 295-304.
31. Schulman HM, Sullivan KM, Weiden PL et al. Chronic graft-versus-host disease syndrome in man. A long-term clinicopathologic study of 20 Seattle patients. Am J Med 1980; 69: 204-217.
32. Kaplan EL, Meier P. Non-parametric estimation from incomplete observations. J Am Stat Assoc 1958; 53: 457-481.
33. Alter BP, Knobloch ME, Weinberg RS. Erythropoiesis in Fanconi's anemia. Blood 1991; 78: 602-608.
34. Weissinger F, Sandmaier BM, Maloney DG et al. Decreased transfusion requirements for patients receiving nonmyeloablative compared with conventional peripheral blood stem cell transplants from HLA-identical siblings. Blood 2001; 98: 3584-3588.
35. Junghanss C, Marr KA, Carter RA et al. Incidence of bacterial and fungal infections after nonmyeloablative compared to myeloablative allogeneic hematopoietic stem cell transplantation (HSCT). Blood 2001; 98: 479a.
36. Sanders JE, Pritchard S, Mahoney P et al. Growth and development following marrow transplantation for leukemia. Blood 1986; 68: 1129-1135.
37. Wingard JR, Plotnick LP, Freemer CS et al. Growth in children after bone marrow transplantation: Busulfan plus cyclophosphamide versus cyclophosphamide plus total body irradiation. Blood 1992; 79: 1068-1073.
38. Huma Z, Boulad F, Black P et al. Growth in children after bone marrow transplantation for acute leukemia. Blood 1995; 86: 819-824.
39. Sanders JE, Hawley J, Levy W et al. Pregnancies following high-dose cyclophosphamide with or without high-dose busulfan or total-body irradiation and bone marrow transplantation. Blood 1996; 87: 3045-3052.
40. Sanders JE, Buckner CD, Amos D et al. Ovarian function following marrow transplantation for aplastic anemia or leukemia. J Clin Oncol 1988; 6: 813-818.
41. Hinterberger-Fischer M, Kier P, Kalhs P et al. Fertility, pregnancies and offspring complications after bone marrow transplantation. Bone Marrow Transplant 1991; 7: 5-9.
42. Witherspoon RP, Fisher LD, Schoch G et al. Secondary cancers after bone marrow transplantation for leukemia or aplastic anemia. N Engl J Med 1989; 321: 784-789.
43. Bhatia S, Ramsay NK, Steinbuch M et al. Malignant neoplasms following bone marrow transplantation. Blood 1996; 87: 3633-3639.
44. Curtis RE, Rowlings PA, Deeg HJ et al. Solid cancers after bone marrow transplantation. N Engl J Med 1997; 336: 897-904.
45. Deeg HJ, Socie G. Malignancies after hematopoietic stem cell transplantation: Many questions, some answers. Blood 1998; 91: 1833-1844.
46. McSweeney PA, Niederwieser D, Shizuru JA et al. Hematopoietic cell transplantation in older patients with hematologic malignancies: replacing high-dose cytotoxic therapy with graft-versus-tumor effects. Blood 2001; 97: 3390-3400.
47. Khouri IF, Keating M, Korbling M et al. Transplant-lite: induction of graft-versus-malignancy using fludarabine-based nonablative chemotherapy and allogeneic blood progenitor-cell transplantation as treatment for lymphoid malignancies. J Clin Oncol 1998; 16: 2817-2824.
48. Maris MB, Niederwieser D, Sandmaier BM et al. Nonmyeloablative hematopoietic stem cell transplants (NM-HSCT) using 10 HLA antigen matched unrelated donors (URDs) for patients (pts) with advanced hematologic malignancies. Blood 2002, (abstract) 100: 76a.
49. Barker JN, Weisdorf DJ, DeFor TE et al. Rapid and complete donor chimerism in adult recipients of unrelated donor umbilical cord blood transplantation after reduced intensity conditioning. Blood 2003; 102: 1915-1919.
50. Rini BI, Zimmerman T, Stadler WM et al. Allogeneic stem-cell transplantation of renal cell cancer after nonmyeloablative chemotherapy: feasibility, engraftment, and clinical results. J Clin Oncol 2002; 20: 2017-2024.
51. Bregni M, Dodero A, Peccatori J et al. Nonmyeloablative conditioning followed by hematopoietic cell allografting and donor lymphocyte infusions for patients with metastatic renal and breast cancer. Blood 2002; 99: 4234-4236.
52. Badros A, Barlogie B, Morris C et al. High response rate in refractory and poor-risk multiple myeloma after allotransplantation using a nonmyeloablative conditioning regimen and donor lymphocyte infusions. Blood 2001; 97: 2574-2579.
53. Carella AM, Cavaliere M, Lerma E et al. Autografting followed by nonmyeloablative immunosuppressive chemotherapy and allogeneic peripheral-blood hematopoietic stem-cell transplantation as treatment of resistant Hodgkin's disease and non-Hodgkin's lymphoma. J Clin Oncol 2000; 18: 3918-3924.
54. Socié G, Stone JV, Wingard JR et al. Long-term survival and late deaths after allogeneic bone marrow transplantation. Late Effects Working Committee of the International Bone Marrow Transplant Registry. N Engl J Med 1999; 341: 14-21.