Phase II Study of CD4+-Guided Pentostatin Lymphodepletion and Pharmacokinetically Targeted Busulfan as Conditioning for Hematopoietic Cell Allografting

Biology of Blood and Marrow Transplantation

Volumn 19, Issue 7, 2013, Pages 1087-1093

  Kharfan Dabaja, Mohamed A ^a   Anasetti, Claudio ^a   Fernandez, Hugo F ^a   Perkins, Janelle ^a   Ochoa Bayona, Jose L ^a   Pidala, Joseph ^a   Perez, Lia E ^a   Ayala, Ernesto ^a   Field, Teresa ^a   Alsina, Melissa ^a   Nishihori, Taiga ^a   Locke, Frederick ^a   Pinilla Ibarz, Javier ^a   Tomblyn, Marcie ^a  

^a H LEE MOFFITT CANCER CENTER AND RESEARCH INSTITUTE   (United States)

Author keywords

Allogeneic hematopoietic cell transplantation; CD4 guided lymphodepletion; Intravenous busulfan; Pentostatin

Indexed keywords

BUSULFAN; CD20 ANTIGEN; CD4 ANTIGEN; COTRIMOXAZOLE; LORAZEPAM; METHOTREXATE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PENTAMIDINE; PENTOSTATIN; RAPAMYCIN; RITUXIMAB; TACROLIMUS; URSODEOXYCHOLIC ACID;

ADENOVIRUS INFECTION; ADULT; AGED; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ALLOGRAFT; AREA UNDER THE CURVE; ARTICLE; CAUSE OF DEATH; CD3+ T LYMPHOCYTE; CD4 LYMPHOCYTE COUNT; CD4+ T LYMPHOCYTE; CD8+ T LYMPHOCYTE; CHIMERA; CHRONIC LYMPHATIC LEUKEMIA; CLINICAL ARTICLE; DRUG BLOOD LEVEL; ENGRAFTMENT; ENTERITIS; FEMALE; GRAFT FAILURE; GRAFT VERSUS HOST REACTION; HEART DISEASE; HUMAN; KIDNEY DISEASE; LIVER DISEASE; LOADING DRUG DOSE; LUNG DISEASE; LYMPHOCYTE DEPLETION; LYMPHOPROLIFERATIVE DISEASE; MALE; MORTALITY; NAUSEA; NEPHROTOXICITY; NEUROLOGIC DISEASE; NONHODGKIN LYMPHOMA; ORAL MUCOSITIS; OVERALL SURVIVAL; PATIENT COMPLIANCE; PHASE 2 CLINICAL TRIAL; PNEUMOCYSTIS PNEUMONIA; PROGRESSION FREE SURVIVAL; REMISSION; SEIZURE; TREATMENT OUTCOME; TREATMENT RESPONSE; VASCULAR DISEASE; VOMITING;

ACUTE DISEASE; ADULT; AGED; ANTINEOPLASTIC AGENTS; BUSULFAN; CD4 LYMPHOCYTE COUNT; CD4-POSITIVE T-LYMPHOCYTES; CHRONIC DISEASE; FEMALE; GRAFT VS HOST DISEASE; HEMATOLOGIC NEOPLASMS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; LYMPHOCYTE DEPLETION; MALE; MIDDLE AGED; MOLECULAR TARGETED THERAPY; PENTOSTATIN; SURVIVAL ANALYSIS; TRANSPLANTATION CONDITIONING; TRANSPLANTATION, HOMOLOGOUS;

EID: 84879332093     PISSN: 10838791     EISSN: 15236536     Source Type: Journal    
DOI: 10.1016/j.bbmt.2013.04.020     Document Type: Article

References (43)

1. Santos G.W., Tutschka P.J., Brookmeyer R., et al. Marrow transplantation for acute nonlymphocytic leukemia after treatment with busulfan and cyclophosphamide. NEngl J Med 1983, 309:1347-1353.
2. Anderson J.E., Appelbaum F.R., Schoch G., et al. Allogeneic marrow transplantation for myelodysplastic syndrome with advanced disease morphology: a phase II study of busulfan, cyclophosphamide, and total body irradiation and analysis of prognostic factors. JClin Oncol 1996, 14:220-226.
3. Ringdén O., Ruutu T., Remberger M., et al. Arandomized trial comparing busulfan with total body irradiation as conditioning in allogeneic marrow transplant recipients with leukemia: a report from the Nordic Bone Marrow Transplantation Group. Blood 1994, 83:2723-2730.
4. Brodsky I., Biggs J.C., Szer J., et al. Treatment of chronic myelogenous leukemia with allogeneic bone marrow transplantation after preparation with busulfan and cyclophosphamide (BuCy2): an update. Semin Oncol 1993, 20:27-31.
5. Ringdén O., Labopin M., Tura S., et al. Acomparison of busulphan versus total body irradiation combined with cyclophosphamide as conditioning for autograft or allograft bone marrow transplantation in patients with acute leukaemia. Acute Leukaemia Working Party of the European Group for Blood and Marrow Transplantation (EBMT). Br J Haematol 1996, 93:637-645.
6. Blume K.G., Kopecky K.J., Henslee-Downey J.P., et al. Aprospective randomized comparison of total body irradiation-etoposide versus busulfan-cyclophosphamide as preparatory regimens for bone marrow transplantation in patients with leukemia who were not in first remission: a Southwest Oncology Group study. Blood 1993, 81:2187-2193.
7. Przepiorka D., Dimopoulos M., Smith T., et al. Thiotepa, busulfan, and cyclophosphamide as a preparative regimen for marrow transplantation: risk factors for early regimen-related toxicity. Ann Hematol 1994, 68:183-188.
8. Kolb H.J., Mittermüller J., Clemm C., et al. Donor leukocyte transfusions for treatment of recurrent chronic myelogenous leukemia in marrow transplant patients. Blood 1990, 76:2462-2465.
9. 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.
10. Champlin R., Khouri I., Shimoni A., et al. Harnessing graft-versus-malignancy: non-myeloablative preparative regimens for allogeneic haematopoietic transplantation, an evolving strategy for adoptive immunotherapy. Br J Haematol 2000, 111:18-29.
11. Kharfan-Dabaja M.A., Bazarbachi A. Hematopoietic stem cell allografting for chronic lymphocytic leukemia: a focus on reduced-intensity conditioning regimens. Cancer Control 2012, 19:68-75.
12. Niederwieser D., Maris M., Shizuru J.A., et al. Low-dose total body irradiation (TBI) and fludarabine followed by hematopoietic cell transplantation (HCT) from HLA-matched 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 2003, 101:1620-1629.
13. Russell J., Tran H., 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 2002, 8:468-476.
14. de Lima M., Couriel D., Thall P.F., et al. Once-daily 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 2004, 104:857-864.
15. Miller K.B., Roberts T.F., Chan G., et al. Anovel reduced-intensity regimen for allogeneic hematopoietic stem cell transplantation associated with a reduced incidence of graft-versus-host disease. Bone Marrow Transplant 2004, 33:881-889.
16. Pavletic S.Z., Bociek R.G., Foran J.M., et al. Lymphodepleting effects and safety of pentostatin for nonmyeloablative allogeneic stem-cell transplantation. Transplantation 2003, 76:877-881.
17. Spiers A.S., Moore D., Cassileth P.A., et al. Remissions in hairy-cell leukemia with pentostatin (2'-deoxycoformycin). NEngl J Med 1987, 316:825-830.
18. Ho A.D., Hensel M. Pentostatin for the treatment of indolent lymphoproliferative disorders. Semin Hematol 2006, 43:S2-S10.
19. Mariotti J., Taylor J., Massey P.R., et al. The pentostatin plus cyclophosphamide nonmyeloablative regimen induces durable host T cell functional deficits and prevents murine marrow allograft rejection. Biol Blood Marrow Transplant 2011, 17:620-631.
20. Fernandez H.F., Tran H.T., Albrecht F., et al. Evaluation of safety and pharmacokinetics of administering intravenous busulfan in a twice-daily or daily schedule to patients with advanced hematologic malignant disease undergoing stem cell transplantation. Biol Blood Marrow Transplant 2002, 8:486-492.
21. Pidala J., Kim J., Anasetti C., et al. Pharmacokinetic targeting of intravenous busulfan reduces conditioning regimen related toxicity following allogeneic hematopoietic cell transplantation for acute myelogenous leukemia. JHematol Oncol 2010, 3:36.
22. Khouri I.F., McLaughlin P., Saliba R.M., et al. Eight-year experience with allogeneic stem cell transplantation for relapsed follicular lymphoma after nonmyeloablative conditioning with fludarabine, cyclophosphamide, and rituximab. Blood 2008, 111:5530-5536.
23. + lymphoid malignancies without increased risk of infectious complications. Int J Hematol 2011, 93:206-212.
24. Kharfan-Dabaja M.A., Bazarbachi A. Emerging role of CD20 blockade in allogeneic hematopoietic cell transplantation. Biol Blood Marrow Transplant 2010, 16:1347-1354.
25. Giralt S., Estey E., Albitar M., et al. Engraftment of allogeneic hematopoietic progenitor cells with purine analog-containing chemotherapy: harnessing graft-versus-leukemia without myeloablative therapy. Blood 1997, 89:4531-4536.
26. + dose impact donor engraftment following nonmyeloablative allogeneic stem cell transplantation in patients with solid tumors. Blood 2004, 103:1560-1563.
27. Perkins J., Field T., Kim J., et al. Arandomized phase II trial comparing tacrolimus and mycophenolate mofetil to tacrolimus and methotrexate for acute graft-versus-host disease prophylaxis. Biol Blood Marrow Transplant 2010, 16:937-947.
28. Pidala J., Kim J., Jim H., et al. Arandomized phase II study to evaluate tacrolimus in combination with sirolimus or methotrexate after allogeneic hematopoietic cell transplantation. Haematologica 2012, 97(12):1882-1889.
29. Cheson B.D., Bennett J.M., Kopecky K.J., et al. Revised recommendations of the International Working Group for Diagnosis, Standardization of Response Criteria, Treatment Outcomes, and Reporting Standards for Therapeutic Trials in Acute Myeloid Leukemia. JClin Oncol 2003, 21:4642-4649.
30. Cheson B.D., Greenberg P.L., Bennett J.M., et al. Clinical application and proposal for modification of the International Working Group (IWG) response criteria in myelodysplasia. Blood 2006, 108:419-425.
31. Cheson B.D., Pfistner B., Juweid M.E., et al. Revised response criteria for malignant lymphoma. JClin Oncol 2007, 25:579-586.
32. Hallek M., Cheson B.D., Catovsky D., et al. Guidelines for the diagnosis and treatment of chronic lymphocytic leukemia: a report from the International Workshop on Chronic Lymphocytic Leukemia updating the National Cancer Institute Working Group 1996 guidelines. Blood 2008, 111:5446-5456.
33. Weber D., Treon S.P., Emmanouilides C., et al. Uniform response criteria in Waldenstrom's macroglobulinemia: consensus panel recommendations from the Second International Workshop on Waldenstrom's Macroglobulinemia. Semin Oncol 2003, 30:127-131.
34. Przepiorka D., Weisdorf D., Martin P., et al. 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplant 1995, 15:825-828.
35. Filipovich A.H., Weisdorf D., Pavletic S., et al. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease, I: Diagnosis and Staging Working Group report. Biol Blood Marrow Transplant 2005, 11:945-956.
36. Baron F., Baker J.E., Storb R., et al. Kinetics of engraftment in patients with hematologic malignancies given allogeneic hematopoietic cell transplantation after nonmyeloablative conditioning. Blood 2004, 104:2254-2262.
37. Lin D.Y. Non-parametric inference for cumulative incidence functions in competing risks studies. Stat Med 1997, 16:901-910.
38. Kaplan E., Meier P. Nonparametric estimation from incomplete observations. JAm Stat Assoc 1958, 53:457-481.
39. Bishop M.R., Hou J.W., Wilson W.H., et al. Establishment of early donor engraftment after reduced-intensity allogeneic hematopoietic stem cell transplantation to potentiate the graft-versus-lymphoma effect against refractory lymphomas. Biol Blood Marrow Transplant 2003, 9:162-169.
40. Bethge W.A., Kerbauy F.R., Santos E., et al. Extracorporeal photopheresis in addition to pentostatin in conditioning for canine hematopoietic cell transplantation: role in engraftment. Bone Marrow Transplant 2011, 46:1382-1388.
41. 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 2004, 104:865-872.
42. Shimoni A., Hardan I., Avigdor A., et al. Rituximab reduces relapse risk after allogeneic and autologous stem cell transplantation in patients with high-risk aggressive non-Hodgkin's lymphoma. Br J Haematol 2003, 122:457-464.
43. Sorror M., Maris M., Storb R., et al. Hematopoietic cell transplantation (HCT)-specific comorbidity index: a new tool for risk assessment before allogeneic HCT. Blood 2005, 106:2912-2919.