Randomized trial of myeloablative conditioning regimens: Busulfan plus cyclophosphamide versus busulfan plus fludarabine

Journal of Clinical Oncology

Volumn 31, Issue 6, 2013, Pages 701-709

  Lee, Je Hwan ^a,g   Joo, Young Don ^b   Kim, Hawk ^c   Ryoo, Hun Mo ^d   Kim, Min Kyoung ^e   Lee, Gyeong Won ^f   Lee, Jung Hee ^a   Lee, Won Sik ^b   Park, Jae Hoo ^c   Bae, Sung Hwa ^d   Hyun, Myung Soo ^e   Kim, Dae Young ^a   Kim, Sung Doo ^a   Min, Young Joo ^c   Lee, Kyoo Hyung ^a  

^a University of Ulsan College of Medicine   (South Korea)

^b Inje University College of Medicine   (South Korea)

^c University of Ulsan College of Medicine   (South Korea)

^d Catholic University of Daegu   (South Korea)

^e Young Nam University Hospital   (South Korea)

^f Gyeongsang National University   (South Korea)

^g Asan Medical Center   (South Korea)

Author keywords

[No Author keywords available]

Indexed keywords

BUSULFAN; CYCLOPHOSPHAMIDE; CYCLOSPORIN; FLUDARABINE; FLUDARABINE PHOSPHATE; METHOTREXATE; RECOMBINANT GRANULOCYTE COLONY STIMULATING FACTOR;

ACUTE GRAFT VERSUS HOST DISEASE; ACUTE LEUKEMIA; ADOLESCENT; ADULT; AGED; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ARTICLE; CANCER PATIENT; CHIMERA; CHRONIC GRAFT VERSUS HOST DISEASE; CHRONIC MYELOID LEUKEMIA; CONTROLLED STUDY; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; EVENT FREE SURVIVAL; FEMALE; GASTROINTESTINAL SYMPTOM; GRAFT FAILURE; GRAFT VERSUS HOST REACTION; HEART DISEASE; HUMAN; INFECTION; KIDNEY DISEASE; LIVER DISEASE; MAJOR CLINICAL STUDY; MALE; MYELOABLATIVE CONDITIONING; MYELODYSPLASTIC SYNDROME; MYELOPROLIFERATIVE NEOPLASM; NEUROLOGIC DISEASE; ORGAN DONOR; OVERALL SURVIVAL; PHASE 3 CLINICAL TRIAL; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; RECURRENCE FREE SURVIVAL; TREATMENT DURATION;

ADOLESCENT; ADULT; AGED; BUSULFAN; CYCLOPHOSPHAMIDE; DRUG THERAPY, COMBINATION; FEMALE; GASTROENTERITIS; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; LEUKEMIA; MALE; MIDDLE AGED; MULTIVARIATE ANALYSIS; MYELOABLATIVE AGONISTS; MYELODYSPLASTIC SYNDROMES; SURVIVAL ANALYSIS; TRANSPLANTATION CONDITIONING; TRANSPLANTATION, HOMOLOGOUS; TREATMENT OUTCOME; VIDARABINE; YOUNG ADULT;

EID: 84875728832     PISSN: 0732183X     EISSN: 15277755     Source Type: Journal    
DOI: 10.1200/JCO.2011.40.2362     Document Type: Article

References (43)

1. Tutschka PJ, Copelan EA, Klein JP: Bone marrow transplantation for leukemia following a new busulfan and cyclophosphamide regimen. Blood 70:1382-1388, 1987 (Pubitemid 18026142)
2. DeLeve LD: Cellular target of cyclophosphamide toxicity in the murine liver: Role of glutathione and site of metabolic activation. Hepatology 24:830-837, 1996
3. Cleland BD, Pokorny CS: Cyclophosphamide related hepatotoxicity. Aust N Z J Med 23:408, 1993
4. Goldberg JW, Lidsky MD: Cyclophosphamide- associated hepatotoxicity. South Med J 78: 222-223, 1985
5. Modzelewski JR Jr., Daeschner C, Joshi VV, et al: Veno-occlusive disease of the liver induced by low-dose cyclophosphamide. Mod Pathol 7:967-972, 1994
6. McDonald GB, Hinds MS, Fisher LD, et al: Venoocclusive disease of the liver and multiorgan failure after bone marrow transplantation: A cohort study of 355 patients. Ann Intern Med 118:255-267, 1993 (Pubitemid 23046384)
7. McDonald GB, Slattery JT, Bouvier ME, et al: Cyclophosphamide metabolism, liver toxicity, and mortality following hematopoietic stem cell transplantation. Blood 101:2043-2048, 2003 (Pubitemid 36237612)
8. 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 97:631-637, 2001 (Pubitemid 32113394)
9. 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 16:2817-2824, 1998 (Pubitemid 28363047)
10. Laport GG, Sandmaier BM, Storer BE, et al: Reduced-intensity conditioning followed by allogeneic hematopoietic cell transplantation for adult patients with myelodysplastic syndrome and myeloproliferative disorders. Biol Blood Marrow Transplant 14:246-255, 2008
11. 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 91:756- 763, 1998 (Pubitemid 28078275)
12. Bornhauser M, Storer B, Slattery JT, et al: Conditioning with fludarabine and targeted busulfan for transplantation of allogeneic hematopoietic stem cells. Blood 102:820-826, 2003 (Pubitemid 36917770)
13. de Lima M, Couriel D, Thall PF, et al: Oncedaily intravenous busulfan and fludarabine: Clinical and pharmacokinetic results of a myeloablative, reduced-toxicity conditioning regimen for allogeneic stem cell transplantation in AML and MDS. Blood 104:857-864, 2004 (Pubitemid 38970585)
14. Geddes M, Kangarloo SB, Naveed F, et al: High busulfan exposure is associated with worse outcomes in a daily i.v. busulfan and fludarabine allogeneic transplant regimen. Biol Blood Marrow Transplant 14:220-228, 2008
15. Iravani M, Evazi MR, Mousavi SA, et al: Fludarabine and busulfan as a myeloablative conditioning regimen for allogeneic stem cell transplantation in high- and standard-risk leukemic patients. Bone Marrow Transplant 40:105-110, 2007 (Pubitemid 47049482)
16. Russell JA, Duan Q, Chaudhry MA, et al: Transplantation from matched siblings using once-daily intravenous busulfan/fludarabine with thymoglobulin: A myeloablative regimen with low nonrelapse mortality in all but older patients with high-risk disease. Biol Blood Marrow Transplant 14:888-895, 2008 (Pubitemid 351985026)
17. Russell JA, Tran HT, Quinlan D, et al: Once-daily intravenous busulfan given with fludarabine as conditioning for allogeneic stem cell transplantation: Study of pharmacokinetics and early clinical outcomes. Biol Blood Marrow Transplant 8:468-476, 2002 (Pubitemid 35174820)
18. Andersson BS, de Lima M, Thall PF, et al: Once daily i.v. busulfan and fludarabine (i.v. Bu-Flu) compares favorably with i.v. busulfan and cyclophosphamide (i.v BuCy2) as pretransplant conditioning therapy in AML/MDS. Biol Blood Marrow Transplant 14:672-684, 2008 (Pubitemid 351664062)
19. Chae YS, Sohn SK, Kim JG, et al: New myeloablative conditioning regimen with fludarabine and busulfan for allogeneic stem cell transplantation: Comparison with BuCy2. Bone Marrow Transplant 40:541-547, 2007 (Pubitemid 47382124)
20. Shimoni A, Hardan I, Shem-Tov N, et al: Allogeneic hematopoietic stem-cell transplantation in AML and MDS using myeloablative versus reduced-intensity conditioning: The role of dose intensity. Leukemia 20:322-328, 2006 (Pubitemid 43148672)
21. Dohner H, Estey EH, Amadori S, et al: Diagnosis and management of acute myeloid leukemia in adults: Recommendations from an international expert panel, on behalf of the European LeukemiaNet. Blood 115:453-474, 2010
22. Greenberg P, Cox C, LeBeau MM, et al: International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood 89:2079-2088, 1997 (Pubitemid 27132124)
23. Rowe JM: Prognostic factors in adult acute lymphoblastic leukaemia. Br J Haematol 150:389-405, 2010
24. Choi SJ, Lee KH, Lee JH, et al: Prognostic value of hematopoietic chimerism in patients with acute leukemia after allogeneic bone marrow transplantation: A prospective study. Bone Marrow Transplant 26:327-332, 2000
25. Tutschka PJ, Beschorner WE, Hess AD, et al: Cyclosporin-A to prevent graft-versus-host disease: A pilot study in 22 patients receiving allogeneic marrow transplants. Blood 61:318-325, 1983 (Pubitemid 13137652)
26. Przepiorka D, Khouri I, Ippoliti C, et al: Tacrolimus and minidose methotrexate for prevention of acute graft-versus-host disease after HLAmismatched marrow or blood stem cell transplantation. Bone Marrow Transplant 24:763-768, 1999 (Pubitemid 29469277)
27. Bearman SI: The syndrome of hepatic venoocclusive disease after marrow transplantation. Blood 85:3005-3020, 1995
28. Kim SE, Lee JH, Choi SJ, et al: Morbidity and non-relapse mortality after allogeneic bone marrow transplantation in adult leukemia patients conditioned with busulfan plus cyclophosphamide: A retrospective comparison of oral versus intravenous busulfan. Haematologica 90:285-286, 2005
29. O'Donnell PH, Artz AS, Undevia SD, et al: Phase I study of dose-escalated busulfan with fludarabine and alemtuzumab as conditioning for allogeneic hematopoietic stem cell transplant: Reduced clearance at high doses and occurrence of late sinusoidal obstruction syndrome/veno-occlusive disease. Leuk Lymphoma 51:2240-2249, 2010
30. Kashyap A, Wingard J, Cagnoni P, et al: Intravenous versus oral busulfan as part of a busulfan/cyclophosphamide preparative regimen for allogeneic hematopoietic stem cell transplantation: Decreased incidence of hepatic venoocclusive disease (HVOD), HVODrelated mortality, and overall 100-day mortality. Biol Blood Marrow Transplant 8:493-500, 2002 (Pubitemid 35174823)
31. Lee JH, Choi SJ, Kim SE, et al: Decreased incidence of hepatic veno-occlusive disease and fewer hemostatic derangements associated with intravenous busulfan vs oral busulfan in adults conditioned with busulfan - cyclophosphamide for allogeneic bone marrow transplantation. Ann Hematol 84:321-330, 2005
32. 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 97:3390-3400, 2001
33. Schultz KR, Ratanatharathorn V, Abella E, et al: Graft failure in children receiving HLA-mismatched marrow transplants with busulfan-containing regimens. Bone Marrow Transplant 13:817-822, 1994 (Pubitemid 24195802)
34. Mattsson J, Ringden O, Storb R: Graft failure after allogeneic hematopoietic cell transplantation. Biol Blood Marrow Transplant 14:165-170, 2008 (Pubitemid 350286854)
35. Clift RA, Buckner CD, Thomas ED, et al: Marrow transplantation for chronic myeloid leukemia: A randomized study comparing cyclophosphamide and total body irradiation with busulfan and cyclophosphamide. Blood 84:2036-2043, 1994 (Pubitemid 24286157)
36. Le Blanc K, Remberger M, Uzunel M, et al: A comparison of nonmyeloablative and reducedintensity conditioning for allogeneic stem-cell transplantation. Transplantation 78:1014-1020, 2004 (Pubitemid 39391863)
37. Niederwieser D, Maris M, Shizuru JA, et al: Lowdose total body irradiation (TBI) and fludarabine followed by hematopoietic cell transplantation (HCT) from HLAmatched or mismatched unrelated donors and postgrafting immunosuppression with cyclosporine and mycophenolate mofetil (MMF) can induce durable complete chimerism and sustained remissions in patients with hematological diseases. Blood 101:1620-1629, 2003 (Pubitemid 36182543)
38. Alyea EP, Kim HT, Ho V, et al: Impact of conditioning regimen intensity on outcome of allogeneic hematopoietic cell transplantation for advanced acute myelogenous leukemia and myelodysplastic syndrome. Biol Blood Marrow Transplant 12:1047-1055, 2006 (Pubitemid 44603937)
39. Martino R, Iacobelli S, Brand R, et al: Retrospective comparison of reduced-intensity conditioning and conventional high-dose conditioning for allogeneic hematopoietic stem cell transplantation using HLA-identical sibling donors in myelodysplastic syndromes. Blood 108: 836-846, 2006 (Pubitemid 44154615)
40. de Lima M, Anagnostopoulos A, Munsell M, et al: Nonablative versus reduced-intensity conditioning regimens in the treatment of acute myeloid leukemia and high-risk myelodysplastic syndrome: Dose is relevant for long-term disease control after allogeneic hematopoietic stem cell transplantation. Blood 104:865-872, 2004 (Pubitemid 38970586)
41. Malar R, SjooF, Rentsch K, et al: Therapeutic drug monitoring is essential for intravenous busulfan therapy in pediatric hematopoietic stem cell recipients. Pediatr Transplant 15:580-588, 2011
42. Gaziev J, Nguyen L, Puozzo C, et al: Novel pharmacokinetic behavior of intravenous busulfan in children with thalassemia undergoing hematopoietic stem cell transplantation: A prospective evaluation of pharmacokinetic and pharmacodynamic profile with therapeutic drug monitoring. Blood 115:4597-4604, 2010
43. Ryu SG, Lee JH, Choi SJ, et al: Randomized comparison of four-times-daily versus once-daily intravenous busulfan in conditioning therapy for hematopoietic cell transplantation. Biol Blood Marrow Transplant 13:1095-1105, 2007