The addition of sirolimus to the graft-versus-host disease prophylaxis regimen in reduced intensity allogeneic stem cell transplantation for lymphoma: A multicentre randomized trial

British Journal of Haematology

Volumn 173, Issue 1, 2016, Pages 96-104

  Armand, Philippe ^a   Kim, Haesook T ^a   Sainvil, Marie Michele ^a   Lange, Paulina B ^a   Giardino, Angela A ^b   Bachanova, Veronika ^c   Devine, Steven M ^d   Waller, Edmund K ^e   Jagirdar, Neera ^e   Herrera, Alex F ^f   Cutler, Corey ^a   Ho, Vincent T ^a   Koreth, John ^a   Alyea, Edwin P ^a   Mcafee, Steven L ^g   Soiffer, Robert J ^a   Chen, Yi Bin ^g   Antin, Joseph H ^a  

^a DANA FARBER CANCER INSTITUTE   (United States)

^b BRIGHAM AND WOMEN S HOSPITAL   (United States)

^c UNIVERSITY OF MINNESOTA   (United States)

^d OHIO STATE UNIVERSITY   (United States)

^e EMORY UNIVERSITY   (United States)

^f CITY OF HOPE NATIONAL MEDICAL CENTER   (United States)

^g MASSACHUSETTS GENERAL HOSPITAL   (United States)

Author keywords

GVHD; Clinical trials; Lymphomas; Stem cell transplantation

Indexed keywords

BUSULFAN; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; FLUDARABINE; METHOTREXATE; MYCOPHENOLATE MOFETIL; RAPAMYCIN; TACROLIMUS;

ABDOMINAL PAIN; ACUTE GRAFT VERSUS HOST DISEASE; ADULT; AGED; ALLERGIC REACTION; ALLOGENEIC STEM CELL TRANSPLANTATION; ANOREXIA; ARTICLE; B CELL LYMPHOMA; BRAIN DISEASE; CANCER RECURRENCE; CATARACT; CHRONIC GRAFT VERSUS HOST DISEASE; CONFUSION; CONTROLLED STUDY; DEHYDRATION; DIARRHEA; DRUG SAFETY; EDEMA; HEART DISEASE; HEMATOMA; HEMOLYSIS; HODGKIN DISEASE; HUMAN; HYPERGLYCEMIA; HYPERLIPIDEMIA; HYPONATREMIA; HYPOTENSION; INFECTION; KIDNEY FAILURE; LEUKOPENIA; LIVER DISEASE; MAJOR CLINICAL STUDY; MALE; MUCOSA INFLAMMATION; MULTICENTER STUDY; NAUSEA; NEUROPATHY; NEUTROPENIA; OVERALL SURVIVAL; PAIN; PRIORITY JOURNAL; PROGRESSION FREE SURVIVAL; PSYCHOSIS; RANDOMIZED CONTROLLED TRIAL; RASH; REDUCED INTENSITY CONDITIONING; RESPIRATORY TRACT DISEASE; RETROSPECTIVE STUDY; STOMATITIS; T CELL LYMPHOMA; THROMBOCYTOPENIA; THROMBOSIS; THROMBOTIC THROMBOCYTOPENIC PURPURA; VOMITING; WEAKNESS; WHOLE BODY RADIATION; ADOLESCENT; ALLOGRAFT; CLINICAL TRIAL; FEMALE; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; LYMPHOMA; MIDDLE AGED; PROCEDURES; TRANSPLANTATION CONDITIONING;

ADOLESCENT; ADULT; AGED; ALLOGRAFTS; FEMALE; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; LYMPHOMA; MALE; METHOTREXATE; MIDDLE AGED; SIROLIMUS; TACROLIMUS; TRANSPLANTATION CONDITIONING;

EID: 84953308839     PISSN: 00071048     EISSN: 13652141     Source Type: Journal    
DOI: 10.1111/bjh.13931     Document Type: Article

References (35)

1. Alyea, E.P., Li, S., Kim, H.T., Cutler, C., Ho, V., Soiffer, R.J. & Antin, J.H. (2008) Sirolimus, tacrolimus, and low-dose methotrexate as graft-versus-host disease prophylaxis in related and unrelated donor reduced-intensity conditioning allogeneic peripheral blood stem cell transplantation. Biology of Blood and Marrow Transplantation, 14, 920-926.
2. Ansell, S.M., Inwards, D.J., Rowland, K.M. Jr, Flynn, P.J., Morton, R.F., Moore, D.F. Jr, Kaufmann, S.H., Ghobrial, I., Kurtin, P.J., Maurer, M., Allmer, C. & Witzig, T.E. (2008) Low-dose, single-agent temsirolimus for relapsed mantle cell lymphoma: a phase 2 trial in the North Central Cancer Treatment Group. Cancer, 113, 508-514.
3. Ansell, S.M., Tang, H., Kurtin, P.J., Koenig, P.A., Inwards, D.J., Shah, K., Ziesmer, S.C., Feldman, A.L., Rao, R., Gupta, M., Erlichman, C. & Witzig, T.E. (2011) Temsirolimus and rituximab in patients with relapsed or refractory mantle cell lymphoma: a phase 2 study. Lancet Oncol, 12, 361-368.
4. Antin, J.H., Kim, H.T., Cutler, C., Ho, V.T., Lee, S.J., Miklos, D.B., Hochberg, E.P., Wu, C.J., Alyea, E.P. & Soiffer, R.J. (2003) Sirolimus, tacrolimus, and low-dose methotrexate for graft-versus-host disease prophylaxis in mismatched related donor or unrelated donor transplantation. Blood, 102, 1601-1605.
5. Armand, P., Gannamaneni, S., Kim, H.T., Cutler, C.S., Ho, V.T., Koreth, J., Alyea, E.P., LaCasce, A.S., Jacobsen, E.D., Fisher, D.C., Brown, J.R., Canellos, G.P., Freedman, A.S., Soiffer, R.J. & Antin, J.H. (2008) Improved survival in lymphoma patients receiving sirolimus for graft-versus-host disease prophylaxis after allogeneic hematopoietic stem-cell transplantation with reduced-intensity conditioning. Journal of Clinical Oncology, 26, 5767-5774.
6. Armand, P., Gibson, C.J., Cutler, C., Ho, V.T., Koreth, J., Alyea, E.P., Ritz, J., Sorror, M.L., Lee, S.J., Deeg, H.J., Storer, B.E., Appelbaum, F.R., Antin, J.H., Soiffer, R.J. & Kim, H.T. (2012) A disease risk index for patients undergoing allogeneic stem cell transplantation. Blood, 120, 905-913.
7. Barnes, J.A., Jacobsen, E., Feng, Y., Freedman, A., Hochberg, E.P., LaCasce, A.S., Armand, P., Joyce, R., Sohani, A.R., Rodig, S.J., Neuberg, D., Fisher, D.C. & Abramson, J.S. (2013) Everolimus in combination with rituximab induces complete responses in heavily pretreated diffuse large B-cell lymphoma. Haematologica, 98, 615-619.
8. Benito, A.I., Furlong, T., Martin, P.J., Anasetti, C., Appelbaum, F.R., Doney, K., Nash, R.A., Papayannopoulou, T., Storb, R., Sullivan, K.M., Witherspoon, R. & Deeg, H.J. (2001) Sirolimus (rapamycin) for the treatment of steroid-refractory acute graft-versus-host disease. Transplantation, 72, 1924-1929.
9. Ceberio, I., Devlin, S.M., Sauter, C., Barker, J.N., Castro-Malaspina, H., Giralt, S., Ponce, D.M., Lechner, L., Maloy, M.A., Goldberg, J.D. & Perales, M.A. (2015) Sirolimus, tacrolimus and low-dose methotrexate based GVHD prophylaxis after non-ablative or reduced intensity conditioning in related and unrelated donor allo-HCT. Leukaemia & Lymphoma, 56, 663-670.
10. Couriel, D.R., Saliba, R., Escalon, M.P., Hsu, Y., Ghosh, S., Ippoliti, C., Hicks, K., Donato, M., Giralt, S., Khouri, I.F., Hosing, C., de Lima, M.J., Andersson, B., Neumann, J. & Champlin, R. (2005) Sirolimus in combination with tacrolimus and corticosteroids for the treatment of resistant chronic graft-versus-host disease. British Journal of Haematology, 130, 409-417.
11. Cutler, C., Kim, H.T., Hochberg, E., Ho, V., Alyea, E., Lee, S.J., Fisher, D.C., Miklos, D., Levin, J., Sonis, S., Soiffer, R.J. & Antin, J.H. (2004) Sirolimus and tacrolimus without methotrexate as graft-versus-host disease prophylaxis after matched related donor peripheral blood stem cell transplantation. Biology of Blood and Marrow Transplantation, 10, 328-336.
12. Cutler, C., Li, S., Ho, V.T., Koreth, J., Alyea, E., Soiffer, R.J. & Antin, J.H. (2007) Extended follow-up of methotrexate-free immunosuppression using sirolimus and tacrolimus in related and unrelated donor peripheral blood stem cell transplantation. Blood, 109, 3108-3114.
13. Cutler, C., Logan, B.R., Nakamura, R., Johnston, L., Choi, S.W., Porter, D.L., Hogan, W.J., Pasquini, M.C., MacMillan, M.L., Wingard, J.R., Waller, E.K., Grupp, S.A., McCarthy, P.L., Wu, J., Hu, Z., Carter, S.L., Horowitz, M.M. & Antin, J.H. (2012) Tacrolimus/Sirolimus Vs Tacrolimus/Methotrexate for Graft-Vs.-Host Disease Prophylaxis After HLA-Matched, related donor hematopoietic stem cell transplantation: results of blood and Marrow Transplant Clinical Trials Network Trial 0402. Blood (ASH Annual Meeting Abstracts), 120, 739.
14. Filipovich, A.H., Weisdorf, D., Pavletic, S., Socie, G., Wingard, J.R., Lee, S.J., Martin, P., Chien, J., Przepiorka, D., Couriel, D., Cowen, E.W., Dinndorf, P., Farrell, A., Hartzman, R., Henslee-Downey, J., Jacobsohn, D., McDonald, G., Mittleman, B., Rizzo, J.D., Robinson, M., Schubert, M., Schultz, K., Shulman, H., Turner, M., Vogelsang, G. & Flowers, M.E. (2005) 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. Biology of Blood and Marrow Transplantation, 11, 945-956.
15. Freidlin, B., Korn, E.L. & George, S.L. (1999) Data monitoring committees and interim monitoring guidelines. Controlled Clinical Trials, 20, 395-407.
16. Furlong, T., Kiem, H.P., Appelbaum, F.R., Carpenter, P.A., Deeg, H.J., Doney, K., Flowers, M.E., Mielcarek, M., Nash, R.A., Storb, R. & Martin, P.J. (2008) Sirolimus in combination with cyclosporine or tacrolimus plus methotrexate for prevention of graft-versus-host disease following hematopoietic cell transplantation from unrelated donors. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation, 14, 531-537.
17. Ghobrial, I.M., Gertz, M., Laplant, B., Camoriano, J., Hayman, S., Lacy, M., Chuma, S., Harris, B., Leduc, R., Rourke, M., Ansell, S.M., Deangelo, D., Dispenzieri, A., Bergsagel, L., Reeder, C., Anderson, K.C., Richardson, P.G., Treon, S.P. & Witzig, T.E. (2010) Phase II trial of the oral mammalian target of rapamycin inhibitor everolimus in relapsed or refractory Waldenstrom macroglobulinemia. Journal of Clinical Oncology, 28, 1408-1414.
18. Gooley, T.A., Chien, J.W., Pergam, S.A., Hingorani, S., Sorror, M.L., Boeckh, M., Martin, P.J., Sandmaier, B.M., Marr, K.A., Appelbaum, F.R., Storb, R. & McDonald, G.B. (2010) Reduced mortality after allogeneic hematopoietic-cell transplantation. New England Journal of Medicine, 363, 2091-2101.
19. Gray, R. (1988) A class of K-sample tests for comparing the cumulative incidence of a competing risk. The Annals of Statistics, 16, 1140-1154.
20. Hess, G., Herbrecht, R., Romaguera, J., Verhoef, G., Crump, M., Gisselbrecht, C., Laurell, A., Offner, F., Strahs, A., Berkenblit, A., Hanushevsky, O., Clancy, J., Hewes, B., Moore, L. & Coiffier, B. (2009) Phase III study to evaluate temsirolimus compared with investigator's choice therapy for the treatment of relapsed or refractory mantle cell lymphoma. Journal of Clinical Oncology, 27, 3822-3829.
21. Ho, V.T., Aldridge, J., Kim, H.T., Cutler, C., Koreth, J., Armand, P., Antin, J.H., Soiffer, R.J. & Alyea, E.P. (2009) Comparison of Tacrolimus and Sirolimus (Tac/Sir) versus Tacrolimus, Sirolimus, and mini-methotrexate (Tac/Sir/MTX) as acute graft-versus-host disease prophylaxis after reduced-intensity conditioning allogeneic peripheral blood stem cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation, 15, 844-850.
22. Jennison, C. & Turnbull, B.W. (1984) Repeated confidence intervals for group sequential clinical trials. Controlled Clinical Trials, 5, 33-45.
23. Johnston, L.J., Brown, J., Shizuru, J.A., Stockerl-Goldstein, K.E., Stuart, M.J., Blume, K.G., Negrin, R.S. & Chao, N.J. (2005) Rapamycin (sirolimus) for treatment of chronic graft-versus-host disease. Biology of Blood and Marrow Transplantation, 11, 47-55.
24. Jurado, M., Vallejo, C., Perez-Simon, J.A., Brunet, S., Ferra, C., Balsalobre, P., Perez-Oteyza, J., Espigado, I., Romero, A., Caballero, D., Sierra, J., Ribera, J.M. & Diez, J.L. (2007) Sirolimus as part of immunosuppressive therapy for refractory chronic graft-versus-host disease. Biology of Blood and Marrow Transplantation, 13, 701-706.
25. Kornblit, B., Maloney, D.G., Storer, B.E., Maris, M.B., Vindelov, L., Hari, P., Langston, A.A., Pulsipher, M.A., Bethge, W.A., Chauncey, T.R., Lange, T., Petersen, F.B., Hubel, K., Woolfrey, A.E., Flowers, M.E., Storb, R. & Sandmaier, B.M. (2014) A randomized phase II trial of tacrolimus, mycophenolate mofetil and sirolimus after non-myeloablative unrelated donor transplantation. Haematologica, 99, 1624-1631.
26. Perez-Simon, J.A., Martino, R., Parody, R., Cabrero, M., Lopez-Corral, L., Valcarcel, D., Martinez, C., Solano, C., Vazquez, L., Marquez-Malaver, F.J., Sierra, J. & Caballero, D. (2013) The combination of sirolimus plus tacrolimus improves outcome after reduced-intensity conditioning, unrelated donor hematopoietic stem cell transplantation compared with cyclosporine plus mycofenolate. Haematologica, 98, 526-532.
27. Pidala, J., Kim, J., Jim, H., Kharfan-Dabaja, M.A., Nishihori, T., Fernandez, H.F., Tomblyn, M., Perez, L., Perkins, J., Xu, M., Janssen, W.E., Veerapathran, A., Betts, B.C., Locke, F.L., Ayala, E., Field, T., Ochoa, L., Alsina, M. & Anasetti, C. (2012) A randomized phase II study to evaluate tacrolimus in combination with sirolimus or methotrexate after allogeneic hematopoietic cell transplantation. Haematologica, 97, 1882-1889.
28. Przepiorka, D., Weisdorf, D., Martin, P., Klingemann, H.G., Beatty, P., Hows, J. & Thomas, E.D. (1995) 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplantation, 15, 825-828.
29. Pulsipher, M.A., Langholz, B., Wall, D.A., Schultz, K.R., Bunin, N., Carroll, W.L., Raetz, E., Gardner, S., Gastier-Foster, J.M., Howrie, D., Goyal, R.K., Douglas, J.G., Borowitz, M., Barnes, Y., Teachey, D.T., Taylor, C. & Grupp, S.A. (2014) The addition of sirolimus to tacrolimus/methotrexate GVHD prophylaxis in children with ALL: a phase III COG/PBMTC trial. Blood, 123, 2017-2025.
30. Renner, C., Zinzani, P.L., Gressin, R., Klingbiel, D., Dietrich, P.Y., Hitz, F., Bargetzi, M., Mingrone, W., Martinelli, G., Trojan, A., Bouabdallah, K., Lohri, A., Gyan, E., Biaggi, C., Cogliatti, S., Bertoni, F., Ghielmini, M., Brauchli, P. & Ketterer, N. (2012) A multicenter phase II trial (SAKK 36/06) of single-agent everolimus (RAD001) in patients with relapsed or refractory mantle cell lymphoma. Haematologica, 97, 1085-1091.
31. Rodriguez, R., Nakamura, R., Palmer, J.M., Parker, P., Shayani, S., Nademanee, A., Snyder, D., Pullarkat, V., Kogut, N., Rosenthal, J., Smith, E., Karanes, C., O'Donnell, M., Krishnan, A.Y., Senitzer, D. & Forman, S.J. (2010) A phase II pilot study of tacrolimus/sirolimus GVHD prophylaxis for sibling donor hematopoietic stem cell transplantation using 3 conditioning regimens. Blood, 115, 1098-1105.
32. Rosenbeck, L.L., Kiel, P.J., Kalsekar, I., Vargo, C., Baute, J., Sullivan, C.K., Wood, L., Abdelqader, S., Schwartz, J., Srivastava, S., Abonour, R., Robertson, M.J., Nelson, R.P. Jr, Cornetta, K., Fausel, C.A. & Farag, S.S. (2011) Prophylaxis with sirolimus and tacrolimus +/- antithymocyte globulin reduces the risk of acute graft-versus-host disease without an overall survival benefit following allogeneic stem cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation, 17, 916-922.
33. Rowlings, P.A., Przepiorka, D., Klein, J.P., Gale, R.P., Passweg, J.R., Henslee-Downey, P.J., Cahn, J.Y., Calderwood, S., Gratwohl, A., Socie, G., Abecasis, M.M., Sobocinski, K.A., Zhang, M.J. & Horowitz, M.M. (1997) IBMTR Severity Index for grading acute graft-versus-host disease: retrospective comparison with Glucksberg grade. British Journal of Haematology, 97, 855-864.
34. Witzig, T.E., Geyer, S.M., Ghobrial, I., Inwards, D.J., Fonseca, R., Kurtin, P., Ansell, S.M., Luyun, R., Flynn, P.J., Morton, R.F., Dakhil, S.R., Gross, H. & Kaufmann, S.H. (2005) Phase II trial of single-agent temsirolimus (CCI-779) for relapsed mantle cell lymphoma. Journal of Clinical Oncology, 23, 5347-5356.
35. Witzig, T.E., Reeder, C.B., LaPlant, B.R., Gupta, M., Johnston, P.B., Micallef, I.N., Porrata, L.F., Ansell, S.M., Colgan, J.P., Jacobsen, E.D., Ghobrial, I.M. & Habermann, T.M. (2011) A phase II trial of the oral mTOR inhibitor everolimus in relapsed aggressive lymphoma. Leukemia, 25, 341-347.