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)

Biology of Blood and Marrow Transplantation

Volumn 18, Issue 8, 2012, Pages 1265-1272

  Kamani, Naynesh R ^a   Walters, Mark C ^b   Carter, Shelly ^c   Aquino, Victor ^d   Brochstein, Joel A ^e   Chaudhury, Sonali ^f   Eapen, Mary ^g   Freed, Brian M ^h   Grimley, Michael ⁱ   Levine, John E ^j   Logan, Brent ^g   Moore, Theodore ^k   Panepinto, Julie ^g   Parikh, Suhag ^l   Pulsipher, Michael A ^m   Sande, Jane ^a   Schultz, Kirk R ⁿ   Spellman, Stephen ^o   Shenoy, Shalini ^p  

^a CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^b CHILDREN S HOSPITAL OAKLAND   (United States)

^c EMMES CORPORATION   (United States)

^d CHILDREN S MEDICAL CENTER   (United States)

^e Northwell Health   (United States)

^f CHILDREN'S MEMORIAL HOSPITAL   (United States)

^g MEDICAL COLLEGE OF WISCONSIN   (United States)

^h ClinImmune Labs   (United States)

ⁱ TEXAS TRANSPLANT INSTITUTE   (United States)

^j UNIVERSITY OF MICHIGAN   (United States)

^k MATTEL CHILDREN S HOSPITAL   (United States)

^l DUKE UNIVERSITY   (United States)

^m UNIVERSITY OF UTAH SCHOOL OF MEDICINE   (United States)

ⁿ BC CHILDREN S HOSPITAL   (Canada)

^o CENTER FOR INTERNATIONAL BLOOD AND MARROW TRANSPLANT RESEARCH   (United States)

^p WASHINGTON UNIVERSITY   (United States)

more..

Author keywords

Children; Cord blood; Sickle cell disease; Transplantation; Unrelated donor

Indexed keywords

ALEMTUZUMAB; CYCLOSPORIN A; FLUDARABINE; HLA A ANTIGEN; HLA B ANTIGEN; HLA DRB1 ANTIGEN; MELPHALAN; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; RECOMBINANT GRANULOCYTE COLONY STIMULATING FACTOR; TACROLIMUS;

ADOLESCENT; ARTICLE; BLOOD DONOR; BLOOD TRANSFUSION; BODY WEIGHT; BONE MARROW TRANSPLANTATION; CD4+ T LYMPHOCYTE; CHILD; CLINICAL ARTICLE; CRYOPRESERVATION; FEMALE; FOLLOW UP; GRAFT FAILURE; GRAFT RECIPIENT; GRAFT REJECTION; GRAFT SURVIVAL; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HLA TYPING; HUMAN; MALE; MOLECULAR TYPING; NEUTROPHIL COUNT; PHASE 2 CLINICAL TRIAL; RECURRENT DISEASE; SCHOOL CHILD; SICKLE CELL ANEMIA; UMBILICAL CORD BLOOD;

ADOLESCENT; ANEMIA, SICKLE CELL; CHILD; CHILD, PRESCHOOL; COHORT STUDIES; CORD BLOOD STEM CELL TRANSPLANTATION; FEMALE; HUMANS; MALE; RETROSPECTIVE STUDIES; SURVIVAL ANALYSIS; UNRELATED DONORS;

EID: 84864005843     PISSN: 10838791     EISSN: 15236536     Source Type: Journal    
DOI: 10.1016/j.bbmt.2012.01.019     Document Type: Article

References (42)

1. Walters M., Patience M., Leisenring W., et al. Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant 2001, 7:665-673.
2. Bernaudin F., Socie G., Kuentz M., et al. Long-term results of myeloablative stem-cell transplantation to cure sickle cell disease. Blood 2007, 110:2749-2756.
3. Vermylen C., Cornu G., Ferster A., et al. Haematopoietic stem cell transplantation for sickle cell anemia: the first 50 patients transplanted in Belgium. Bone Marrow Transplant 1998, 22:1-6.
4. Walters M., Patience M., Leisenring W., et al. Bone marrow transplantation for sickle cell disease. N Engl J Med 1996, 335:369-376.
5. Eggleston B., Patience M., Edwards S., et al. 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.
6. Shenoy S., Grossman W., DiPersio J., et al. A novel reduced-intensity stem cell transplant regimen for nonmalignant disorders. Bone Marrow Transplant 2005, 35:345-352.
7. Rao A., Kamani N., Filopovich A., et al. Successful bone marrow transplantation for IPEX syndrome after reduced intensity conditioning. Blood 2007, 109:383-385.
8. Iannone R., Casella J., Fuchs E., 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.
9. Horan J., Liesveld J., Fenton P., Blumberg N., Walters M. 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.
10. Hsieh M., Kang E., Fitzhugh C., et al. Allogeneic hematopoietic stem-cell transplantation for sickle cell disease. N Engl J Med 2009, 361:2309-2317.
11. Weisdorf D., Hurd D., Carter S., Howe C., Jensen L., Wagner J. Prospective grading of graft-versus-host disease after unrelated donor marrow transplantation: a grading algorithm versus blinded expert panel review. Biol Blood Marrow Transplant 2003, 9:512-518.
12. Gooley T., Leisenring W., Crowley J., Storer B. Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Stat Med 1999, 18:695-706.
13. Walters M., Patience M., Leisenring W., et al. Barriers to bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant 1996, 2:100-104.
14. Kelly P., Kurtzberg J., Vichinsky E., Lubin B. Umbilical cord blood stem cells: application for the treatment of patients with hemoglobinopathies. J Pediatr 1997, 130:695-703.
15. Laughlin M. UCB allogeneic transplantation for hemoglobinopathies. Blood 2003, 101:2077.
16. Adamkiewicz T., Boyer M., Bray R., Haight A., Yeager A. Identification of unrelated cord blood units for hematopoietic stem cell transplantation in children with sickle cell disease. J Pediatr Hematol Oncol 2006, 28:29-32.
17. Krishnamurti L., Abel S., Maiers M., Flesch S. Availability of unrelated donors for hematopoietic stem cell transplantation for hemoglobinopathies. Bone Marrow Transplant 2003, 31:547-550.
18. 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.
19. Adamkiewicz T., Mehta P., Boyer M., et al. Transplantation of unrelated placental blood cells in children with high-risk sickle cell disease. Bone Marrow Transplant 2004, 34:405-411.
20. Adamkiewicz T., 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.
21. Ruggeri A., Eapen M., Scaravadou A., et al. Umbilical cord blood transplantation for children with thalassemia and sickle cell disease. Biol Blood Marrow Transplant 2011, 17:1375-1382.
22. Jaing T., Sun C., Lee W., Wen Y., Yang C., Hung I. Second transplant with two unrelated cord blood units for early graft failure after cord blood transplantation for thalassemia. Pediatr Transplant 2009, 13:766-768.
23. Anasetti C., Amos D., Beatty P., et al. Effect of HLA compatibility on engraftment of bone marrow transplants in patients with leukemia or lymphoma. N Engl J Med 1989, 320:197-204.
24. Kurtzberg J., Prasad V., Carter S., et al. Results of the Cord Blood Transplantation Study (COBLT): clinical outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with hematologic malignancies. Blood 2008, 112:4318-4327.
25. Gluckman E., Rocha V., Arcese W., et al. Factors associated with outcomes of unrelated cord blood transplant: guidelines for donor choice. Exp Hematol 2004, 32:397-407.
26. Kamani N., Spellman S., Hurley C., et al. State of the art review: HLA matching and outcome of unrelated donor umbilical cord blood transplants. Biol Blood Marrow Transplant 2008, 14:1-6.
27. Rocha V., Gluckman E. Improving outcomes of cord blood transplantation: HLA matching, cell dose and other graft- and transplantation-related factors. Br J Haematol 2009, 147:262-274. Eurocord-Netcord registry and European Blood and Marrow Transplant group.
28. Barker J., Scaradavou A., Stevens C. Combined effect of total nucleated cell dose and HLA match on transplantation outcome in 1061 cord blood recipients with hematologic malignancies. Blood 2010, 115:1843-1849.
29. Fleischhauer K., Locatelli F., Zecca M., et al. Graft rejection after unrelated donor hematopoietic stem cell transplantation for thalassemia is associated with nonpermissive HLA-DPB1 disparity in host-versus-graft direction. Blood 2006, 107:2984-2992.
30. Gale R., Ho W., Feig S., et al. Prevention of graft rejection following bone marrow transplantation. Blood 1981, 57:9-12.
31. Camitta B., Storb R., Thomas E. Aplastic anemia (first of two parts): pathogenesis, diagnosis, treatment, and prognosis. N Engl J Med 1982, 306:645-652.
32. Camitta B., Storb R., Thomas E. Aplastic anemia (second of two parts): pathogenesis, diagnosis, treatment, and prognosis. N Engl J Med 1982, 306:712-718.
33. Kanda J., Ichinohe T., Matsuo K., et al. Impact of ABO mismatching on the outcomes of allogeneic related and unrelated blood and marrow stem cell transplantations for hematologic malignancies: IPD-based meta-analysis of cohort studies. Transfusion 2009, 49:624-635.
34. McPherson M., Anderson A., Castillejo M., et al. HLA alloimmunization is associated with RBC antibodies in multiply transfused patients with sickle cell disease. Pediatr Blood Cancer 2010, 54:552-558.
35. Spellman S., Bray R., Rosen-Bronson S., et al. The detection of donor-directed, HLA-specific alloantibodies in recipients of unrelated hematopoietic cell transplantation is predictive of graft failure. Blood 2010, 115:2704-2708.
36. Takanashi M., Atsuta Y., Fujiwara K., et al. The impact of anti-HLA antibodies on unrelated cord blood transplantations. Blood 2010, 116:2839-2846.
37. Cutler C., Kim H.T., Sun L., et al. Donor-specific anti-HLA antibodies predict outcome in double umbilical cord blood transplantation. Blood 2011 Sep 22, [Epub ahead of print].
38. van Rood J., Stevens C., Smits J., Carrier C., Carpenter C., Scaradavou A. Reexposure of cord blood to noninherited maternal HLA antigens improves transplant outcome in hematological malignancies. Proc Natl Acad Sci USA 2009, 106:19952-19957.
39. Sodani P., Gaziev D., Polchi P., et al. New approach for bone marrow transplantation in patients with class 3 thalassemia aged younger than 17 years. Blood 2004, 104:1201-1203.
40. Hongeng S., Pakakasama S., Chuansumrit A., et al. Reduced intensity stem cell transplantation for treatment of class 3 Lucarelli severe thalassemia patients. Am J Hematol 2007, 82:1095-1098.
41. Barker J., Weisdorf D., DeFor T., et al. Transplantation of 2 partially HLA-matched umbilical cord blood units to enhance engraftment in adults with hematologic malignancy. Blood 2005, 105:1343-1347.
42. Brunstein C., Barker J., Weisdorf D., et al. Umbilical cord blood transplantation after nonmyeloablative conditioning: impact on transplantation outcomes in 110 adults with hematologic disease. Blood 2007, 110:3064-3070.