A trial of unrelated donor marrow transplantation for children with severe sickle cell disease

Blood

Volumn 128, Issue 21, 2016, Pages 2561-2567

  Shenoy, Shalini ^a   Eapen, Mary ^b   Panepinto, Julie A ^b,c   Logan, Brent R ^b   Wu, Juan ^d   Abraham, Allistair ^e   Brochstein, Joel ^f   Chaudhury, Sonali ^g   Godder, Kamar ^h   Haight, Ann E ⁱ   Kasow, Kimberly A ^j   Leung, Kathryn ^k   Andreansky, Martin ^l   Bhatia, Monica ^m   Dalal, Jignesh ⁿ   Haines, Hilary ^o   Jaroscak, Jennifer ^p   Lazarus, Hillard M ^q   Levine, John E ^r   Krishnamurti, Lakshmanan ^s   Margolis, David ^c   Megason, Gail C ^t   Yu, Lolie C ^u   Pulsipher, Michael A ^v   Gersten, Iris ^d   DiFronzo, Nancy ^w   Horowitz, Mary M ^b   Walters, Mark C ^x   Kamani, Naynesh ^y   more..

^a ST LOUIS CHILDREN S HOSPITAL   (United States)

^b MEDICAL COLLEGE OF WISCONSIN   (United States)

^c Children's Hospital of Wisconsin   (United States)

^d EMMES CORPORATION   (United States)

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

^f Northwell Health   (United States)

^g CHILDREN'S MEMORIAL HOSPITAL   (United States)

^h VIRGINIA COMMONWEALTH UNIVERSITY SCHOOL OF MEDICINE   (United States)

ⁱ EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

^j UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

^k BAYLOR COLLEGE OF MEDICINE   (United States)

^l UNIVERSITY OF MIAMI MILLER SCHOOL OF MEDICINE   (United States)

^m Department of Medicine   (United States)

ⁿ General Surgery   (United States)

^o University of Alabama at Birmingham   (United States)

^p MEDICAL UNIVERSITY OF SOUTH CAROLINA   (United States)

^q CASE WESTERN RESERVE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^r UNIVERSITY OF MICHIGAN   (United States)

^s CHILDREN S HOSPITAL OF PITTSBURGH   (United States)

^t UNIVERSITY OF MISSISSIPPI MEDICAL CENTER   (United States)

^u LOUISIANA STATE UNIV MEDICAL CENTER   (United States)

^v Utah Blood and Marrow Transplant Program   (United States)

^w NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

^x CHILDREN S HOSPITAL OAKLAND   (United States)

^y AABB   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ALEMTUZUMAB; CYCLOSPORIN; FLUDARABINE; GRANULOCYTE COLONY STIMULATING FACTOR; HLA A ANTIGEN; HLA B ANTIGEN; HLA C ANTIGEN; HLA DRB1 ANTIGEN; MELPHALAN; METHOTREXATE; METHYLPREDNISOLONE; MYCOPHENOLATE MOFETIL; RAPAMYCIN; TACROLIMUS; CALCINEURIN INHIBITOR;

ACUTE CHEST SYNDROME; ACUTE GRAFT VERSUS HOST DISEASE; ADOLESCENT; ADULT; ADULT RESPIRATORY DISTRESS SYNDROME; ARTICLE; BONE MARROW TRANSPLANTATION; CANDIDIASIS; CARDIOPULMONARY INSUFFICIENCY; CEREBROVASCULAR ACCIDENT; CHILD; CHRONIC GRAFT VERSUS HOST DISEASE; CLINICAL ARTICLE; CYTOMEGALOVIRUS INFECTION; DISEASE SEVERITY; DRUG DOSE REDUCTION; ENCEPHALOMYELITIS; ENTEROCOCCAL INFECTION; ERYTHROCYTE TRANSFUSION; EVENT FREE SURVIVAL; FEMALE; FOLLOW UP; GENE LOCUS; GRAFT REJECTION; HERPES SIMPLEX; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; INFECTION PREVENTION; KIDNEY FAILURE; LUNG HEMORRHAGE; MALE; OPPORTUNISTIC INFECTION; OVERALL SURVIVAL; PHASE 2 CLINICAL TRIAL; POSTERIOR REVERSIBLE ENCEPHALOPATHY SYNDROME; PRIORITY JOURNAL; QUALITY OF LIFE; REDUCED INTENSITY CONDITIONING; RESPIRATORY FAILURE; SHORT COURSE THERAPY; SICKLE CELL ANEMIA; STAPHYLOCOCCAL PNEUMONIA; SURGICAL MORTALITY; TIME OF DEATH; TRANSCRANIAL DOPPLER; TREATMENT INDICATION; UNRELATED DONORS; ALLOGRAFT; ANEMIA, SICKLE CELL; CLINICAL TRIAL; DISEASE FREE SURVIVAL; GRAFT VS HOST DISEASE; MORTALITY; PATHOPHYSIOLOGY; SURVIVAL RATE;

ADOLESCENT; ALLOGRAFTS; ANEMIA, SICKLE CELL; BONE MARROW TRANSPLANTATION; CALCINEURIN INHIBITORS; CHILD; DISEASE-FREE SURVIVAL; FEMALE; FOLLOW-UP STUDIES; GRAFT VS HOST DISEASE; HUMANS; MALE; SURVIVAL RATE; UNRELATED DONORS;

EID: 85015019083     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2016-05-715870     Document Type: Article

References (57)

1. Owusu-Ansah A, Ihunnah CA, Walker AL, Ofori- Acquah SF. Inflammatory targets of therapy in sickle cell disease. Transl Res. 2016;167(1): 281-297.
2. Walters MC, Patience M, Leisenring W, et al. Bone marrow transplantation for sickle cell disease. N Engl J Med. 1996;335(6):369-376.
3. Panepinto JA, Walters MC, Carreras J, et al; Non- Malignant Marrow Disorders Working Committee, Center for International Blood and Marrow Transplant Research. Matched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research. Br J Haematol. 2007;137(5):479-485.
4. Locatelli F, Kabbara N, Ruggeri A, et al; Eurocord and European Blood and Marrow Transplantation (EBMT) group. Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling. Blood. 2013;122(6):1072-1078.
5. Bernaudin F, Socie G, Kuentz M, et al; SFGM-TC. Long-term results of related myeloablative stemcell transplantation to cure sickle cell disease. Blood. 2007;110(7):2749-2756.
6. Arnold SD, Bhatia M, Horan J, Krishnamurti L. Haematopoietic stem cell transplantation for sickle cell disease - current practice and new approaches. Br J Haematol. 2016;174(4): 515-525.
7. Baronciani D, Angelucci E, Potschger U, et al. Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000-2010. Bone Marrow Transplant. 2016;51(4):536-541.
8. Mentzer WC, Heller S, Pearle PR, Hackney E, Vichinsky E. Availability of related donors for bone marrow transplantation in sickle cell anemia. Am J Pediatr Hematol Oncol. 1994;16(1):27-29.
9. Peters C, Balthazor M, Shapiro EG, et al. Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome. Blood. 1996;87(11):4894-4902.
10. Deeg HJ, O'Donnell M, Tolar J, et al. Optimization of conditioning for marrow transplantation from unrelated donors for patients with aplastic anemia after failure of immunosuppressive therapy. Blood. 2006;108(5):1485-1491.
11. Ruggeri A, Eapen M, Scaravadou A, et al; Eurocord Registry; Center for International Blood and Marrow Transplant Research; New York Blood Center. Umbilical cord blood transplantation for children with thalassemia and sickle cell disease. Biol Blood Marrow Transplant. 2011;17(9):1375-1382.
12. Gragert L, Eapen M, Williams E, et al. HLA match likelihoods for hematopoietic stem-cell grafts in the U.S. registry. N Engl J Med. 2014;371(4): 339-348.
13. Fitzhugh CD, Perl S, Hsieh MM. Late effects of myeloablative bone marrow transplantation (BMT) in sickle cell disease (SCD). Blood. 2008;111(3): 1742-1743, author reply 1744.
14. Walters MC, Hardy K, Edwards S, et al; Multicenter Study of Bone Marrow Transplantation for Sickle Cell Disease. Pulmonary, gonadal, and central nervous system status after bone marrow transplantation for sickle cell disease. Biol Blood Marrow Transplant. 2010; 16(2):263-272.
15. Jagasia M, Arora M, Flowers ME, et al. Risk factors for acute GVHD and survival after hematopoietic cell transplantation. Blood. 2012; 119(1):296-307.
16. Eggleston B, Patience M, Edwards S, et al; Multicenter Study of HCT for SCA. 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(4):673-676.
17. Iannone R, Casella JF, Fuchs EJ, et al. Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia. Biol Blood Marrow Transplant. 2003; 9(8):519-528.
18. Horan JT, Liesveld JL, Fenton P, Blumberg N, Walters MC. Hematopoietic stem cell transplantation for multiply transfused patients with sickle cell disease and thalassemia after lowdose total body irradiation, fludarabine, and rabbit anti-thymocyte globulin. Bone Marrow Transplant. 2005;35(2):171-177.
19. Kamani NR, Walters MC, 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(8):1265-1272.
20. Hsieh MM, Fitzhugh CD, Weitzel RP, et al. Nonmyeloablative HLA-matched sibling allogeneic hematopoietic stem cell transplantation for severe sickle cell phenotype. JAMA. 2014; 312(1):48-56.
21. King AA, Kamani N, Bunin N, et al. Successful matched sibling donor marrow transplantation following reduced intensity conditioning in children with hemoglobinopathies. Am J Hematol. 2015; 90(12):1093-1098.
22. Matthes-Martin S, Lawitschka A, Fritsch G, et al. Stem cell transplantation after reduced-intensity conditioning for sickle cell disease. Eur J Haematol. 2013;90(4):308-312.
23. Nichols FT, Jones AM, Adams RJ. Stroke prevention in sickle cell disease (STOP) study guidelines for transcranial Doppler testing. J Neuroimaging. 2001;11(4):354-362.
24. Walters MC, Sullivan KM, Bernaudin F, et al. Neurologic complications after allogeneic marrow transplantation for sickle cell anemia. Blood. 1995;85(4):879-884.
25. Walters MC, Patience M, Leisenring W, et al; Multicenter Investigation of Bone Marrow Transplantation for Sickle Cell Disease. Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant. 2001;7(12):665-673.
26. https://web.emmes.com/study/bmt2/public/MOP/BMT%20CTN%20Technical%20MOP%20v3.pdf.
27. Bearman SI, Appelbaum FR, Buckner CD, et al. Regimen-related toxicity in patients undergoing bone marrow transplantation. J Clin Oncol. 1988; 6(10):1562-1568.
28. Palermo TM, Schwartz L, Drotar D, McGowan K. Parental report of health-related quality of life in children with sickle cell disease. J Behav Med. 2002;25(3):269-283.
29. Panepinto JA, O'Mahar KM, DeBaun MR, Rennie KM, Scott JP. Validity of the child health questionnaire for use in children with sickle cell disease. J Pediatr Hematol Oncol. 2004;26(9): 574-578.
30. Kaplan EL. Meier P. Nonparametric estimation for incomplete observations. J Am Stat Assoc. 1958;53(282):457-481.
31. Gooley TA, Leisenring W, Crowley J, Storer BE. Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Stat Med. 1999;18(6):695-706.
32. Landgraf JM, Abetz L, Ware JE. The CHQ User's Manual. Boston, MA: Health Act; 1999.
33. Hays RD, Farivar SS, Liu H. Approaches and recommendations for estimating minimally important differences for health-related quality of life measures. COPD. 2005;2(1):63-67.
34. Beverung LM, Strouse JJ, Hulbert ML, et al; SIT trial investigators. Health-related quality of life in children with sickle cell anemia: impact of blood transfusion therapy. Am J Hematol. 2015;90(2): 139-143.
35. Bernardo ME, Piras E, Vacca A, et al. Allogeneic hematopoietic stem cell transplantation in thalassemia major: results of a reduced-toxicity conditioning regimen based on the use of treosulfan. Blood. 2012;120(2):473-476.
36. Panepinto JA, O'Mahar KM, DeBaun MR, Loberiza FR, Scott JP. Health-related quality of life in children with sickle cell disease: child and parent perception. Br J Haematol. 2005;130(3): 437-444.
37. Walters MC, Patience M, Leisenring W, et al. Barriers to bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant. 1996; 2(2):100-104.
38. Besse K, Maiers M, Confer D, Albrecht M. On Modeling Human Leukocyte Antigen-Identical Sibling Match Probability for Allogeneic Hematopoietic Cell Transplantation: Estimating the Need for an Unrelated Donor Source. Biol Blood Marrow Transplant. 2016;22(3):410-417.
39. Hingorani SR, Seidel K, Lindner A, Aneja T, Schoch G, McDonald G. Albuminuria in hematopoietic cell transplantation patients: prevalence, clinical associations, and impact on survival. Biol Blood Marrow Transplant. 2008;14(12):1365-1372.
40. Maradei SC, Maiolino A, de Azevedo AM, Colares M, Bouzas LF, Nucci M. Serum ferritin as risk factor for sinusoidal obstruction syndrome of the liver in patients undergoing hematopoietic stem cell transplantation. Blood. 2009;114(6): 1270-1275.
41. Eckrich MJ, Ahn KW, Champlin RE, et al. Effect of race on outcomes after allogeneic hematopoietic cell transplantation for severe aplastic anemia. Am J Hematol. 2014;89(2):125-129.
42. Pidala J, Lee SJ, Ahn KW, et al. Nonpermissive HLA-DPB1 mismatch increases mortality after myeloablative unrelated allogeneic hematopoietic cell transplantation. Blood. 2014;124(16): 2596-2606.
43. Morishima Y, Kashiwase K, Matsuo K, et al; Japan Marrow Donor Program. Biological significance of HLA locus matching in unrelated donor bone marrow transplantation. Blood. 2015; 125(7):1189-1197.
44. 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(7):2984-2992.
45. Morishima S, Kashiwase K, Matsuo K, et al; Japan Marrow Donor Program. High-risk HLA alleles for severe acute graft-versus-host disease and mortality in unrelated donor bone marrow transplantation. Haematologica. 2016;101(4): 491-498.
46. Zhang MJ, Davies SM, Camitta BM, et al. Comparison of outcomes after HLA-matched sibling and unrelated donor transplantation for children with high-risk acute lymphoblastic leukemia. Biol Blood Marrow Transplant. 2012; 18(8):1204-1210.
47. Weisdorf DJ, Nelson G, Lee SJ, et al; Chronic Leukemia Working Committee. Sibling versus unrelated donor allogeneic hematopoietic cell transplantation for chronic myelogenous leukemia: refined HLA matching reveals more graft-versus-host disease but not less relapse. Biol Blood Marrow Transplant. 2009;15(11): 1475-1478.
48. Marsh JCGV, Gupta V, Lim Z, et al. Alemtuzumab with fludarabine and cyclophosphamide reduces chronic graft-versus-host disease after allogeneic stem cell transplantation for acquired aplastic anemia. Blood. 2011;118(8):2351-2357.
49. Kröger N, Solano C, Wolschke C, et al. Antilymphocyte globulin for prevention of chronic graft-versus-host disease. N Engl J Med. 2016; 374(1):43-53.
50. Lindemans CA, Chiesa R, Amrolia PJ, et al. Impact of thymoglobulin prior to pediatric unrelated umbilical cord blood transplantation on immune reconstitution and clinical outcome. Blood. 2014;123(1):126-132.
51. Bolaños-Meade J, Fuchs EJ, 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(22):4285-4291.
52. Strocchio L, Zecca M, Comoli P, et al. Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in children with sickle cell disease. Br J Haematol. 2015;169(5):726-736.
53. Erer B, Polchi P, Lucarelli G, et al. CsA-associated neurotoxicity and ineffective prophylaxis with clonazepam in patients transplanted for thalassemia major: analysis of risk factors. Bone Marrow Transplant. 1996; 18(1):157-162.
54. Khademian Z, Speller-Brown B, Nouraie SM, Minniti CP. Reversible posterior leukoencephalopathy in children with sickle cell disease. Pediatr Blood Cancer. 2009;52(3): 373-375.
55. Pegelow CH, Colangelo L, Steinberg M, et al. Natural history of blood pressure in sickle cell disease: risks for stroke and death associated with relative hypertension in sickle cell anemia. Am J Med. 1997;102(2):171-177.
56. Noè A, Cappelli B, Biffi A, et al. High incidence of severe cyclosporine neurotoxicity in children affected by haemoglobinopaties undergoing myeloablative haematopoietic stem cell transplantation: early diagnosis and prompt intervention ameliorates neurological outcome. Ital J Pediatr. 2010;36:14.
57. Hammerstrom AE, Howell J, Gulbis A, Rondon G, Champlin RE, Popat U. Tacrolimus-associated posterior reversible encephalopathy syndrome in hematopoietic allogeneic stem cell transplantation. Am J Hematol. 2013;88(4): 301-305.