Novel immunosuppression compounds and experimental therapies for chronic graft-versus-host disease

Acta Haematologica

Volumn 130, Issue 1, 2013, Pages 34-43

  Michael, Maria ^a   Shimoni, Avichai ^a   Nagler, Arnon ^a  

^a TEL AVIV UNIVERSITY   (Israel)

Author keywords

Allogeneic stem cell transplantation; Biomarkers; Graft versus host disease

Indexed keywords

BIOLOGICAL MARKER; CORTICOSTEROID; EVEROLIMUS; IMMUNOSUPPRESSIVE AGENT; INTERLEUKIN 2; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PENTOSTATIN; PROTEASOME INHIBITOR; PROTEIN TYROSINE KINASE INHIBITOR; RAPAMYCIN; RITUXIMAB;

ALLOGENEIC STEM CELL TRANSPLANTATION; B LYMPHOCYTE; CHRONIC GRAFT VERSUS HOST DISEASE; CORTICOSTEROID THERAPY; DRUG RESPONSE; EXPERIMENTAL THERAPY; EXTRACORPOREAL PHOTOPHERESIS; FIBROSIS; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; PROSPECTIVE STUDY; PUVA; RANDOMIZED CONTROLLED TRIAL (TOPIC); REGULATORY T LYMPHOCYTE; REVIEW; TARGET CELL; TREATMENT DURATION;

EID: 84873369436     PISSN: 00015792     EISSN: 14219662     Source Type: Journal    
DOI: 10.1159/000345836     Document Type: Review

References (47)

1. Perez-Simon JA, Encinas C, Silva F, Arcos MJ, Diez-Campelo M, Sanchez-Guijo FM, Colado E, Martin J, Vazquez L, del Canizo C, Caballero D, San Miguel J: Prognostic factors of chronic graft-versus-host disease following allogeneic peripheral blood stem cell transplantation: the National Institute Health scale plus the type of onset can predict survival rates and the duration of immunosuppressive therapy. Biol Blood Marrow Transplant 2008; 14: 1163-1171.
2. Couriel D, Carpenter PA, Cutler C, Bolanos-Meade J, Treister NS, Gea-Banacloche J, Shaughnessy P, Hymes S, Kim S, Wayne AS, Chien JW, Neumann J, Mitchell S, Syrjala K, Moravec CK, Abramovitz L, Liebermann J, Berger A, Gerber L, Schubert M, Filipovich AH, Weisdorf D, Schubert MM, Schulman H, Schultz K, Mittelman B, Pavletic S, Vogelsang GB, Martin PJ, Lee SJ, Flowers ME: Ancillary therapy and supportive care of chronic graftversus-host disease: National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: V. Ancillary Therapy and Supportive Care Working Group report. Biol Blood Marrow Transplant 2006; 12: 375-396.
3. Pavletic SZ, Martin P, Lee SJ, Mitchell S, Jacobsohn D, Cowen EW, Turner ML, Akpek G, Gilman A, McDonald G, Schubert M, Berger A, Bross P, Chien JW, Couriel D, Dunn JP, Fall-Dickson J, Farrell A, Flowers ME, Greinix H, Hirschfeld S, Gerber L, Kim S, Knobler R, Lachenbruch PA, Miller FW, Mittleman B, Papadopoulos E, Parsons SK, Przepiorka D, Robinson M, Ward M, Reeve B, Rider LG, Shulman H, Schultz KR, Weisdorf D, Vogelsang GB: Measuring therapeutic response in chronic graft-versus-host disease: National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: IV. Response Criteria Working Group report. Biol Blood Marrow Transplant 2006; 12: 252-266.
4. Schultz KR, Miklos DB, Fowler D, Cooke K, Shizuru J, Zorn E, Holler E, Ferrara J, Shulman H, Lee SJ, Martin P, Filipovich AH, Flowers ME, Weisdorf D, Couriel D, Lachenbruch PA, Mittleman B, Vogelsang GB, Pavletic SZ: Towards biomarkers for chronic graft-versus-host disease: National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: III. Biomarker Working Group report. Biol Blood Marrow Transplant 2006; 12: 126-137.
5. Schulman HM, Kleiner D, Lee SJ, Morton T, Pavletic SZ, Farmer E, Moresi JM, Greenson J, Janin A, Martin PJ, McDonald G, Flowers ME, Turner M, Atkinson J, Lefkowitch J, Washington MK, Prieto VG, Kim SK, Argenyi Z, Diwan AH, Rashid A, Hiatt K, Couriel D, Schultz K, Hymes S, Vogelsang GB: Histopathologic diagnosis of chronic graft-versus-host disease: National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: II. Pathology Working Group report. Biol Blood Marrow Transplant 2006; 12: 31-47.
6. Fillipovich AH, Weisdorf D, Pavletic S, Socie G, Wingard JR, Lee SJ, Martin P, Chien J, Przepiorka D, Couriel D, Cowen EW, Dinndorf P, Farrell A, Hartzman R, Henslee-Downey J, Jacobsohn D, McDonald G, Mittleman B, Rizzo JD, Robinson M, Schubert M, Schultz K, Shulman H, Turner M, Vogelsang G, Flowers ME: 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.
7. Martin PJ, Weisdorf D, Przepiorka D, Hirschfeld S, Farrell A, Rizzo JD, Foley R, Socie G, Carter S, Couriel D, Schultz KR, Flowers ME, Filipovich AH, Saliba R, Vogelsang GB, Pavletic SZ, Lee SJ: National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: VI. Design of Clinical Trials Working Group report. Biol Blood Marrow Transplant 2006; 12: 491-505.
8. Wolff D, Gerbitz A, Ayuk F, Kiani A, Hildebrandt GC, Vogelsang GB, Elad S, Lawitschka A, Socie G, Pavletic SZ, Holler E, Greinix H: Consensus conference on clinical practice in chronic graft-versus-host disease (GVHD): first-line and topical treatment of chronic GVHD. Biol Blood Marrow Transplant 2010; 16: 1611-1628.
9. Wolff D, Schleuning M, von Harsdorf S, Bacher U, Gerbitz A, Stadler M, Ayuk F, Kiani A, Schwerdtfeger R, Vogelsang GB, Kobbe G, Gramatzki M, Lawitschka A, Mohty M, Pavletic SZ, Greinix H, Holler E: Consensus conference on clinical practice in chronic GVHD: second-line treatment of chronic graft-versus-host disease. Biol Blood Marrow Transplant 2011; 17: 1-17.
10. Koreth J, Antin JH: Current and future approaches for control of graft-versus-host disease. Expert Rev Hematol 2008; 1: 111.
11. Lopez F, Parker P, Nademanee A, Rogriguez R, Al-Kadhimi Z, Bhatia R, Cohen S, Falk P, Fung H, Kirschbaum M, Krishman A, Kogut N, Molina A, Nakamura R, O'Donnel M, Popplewell L, Pullarkat V, Rosenthal J, Sahebi F, Smith E, Snyder D, Somlo G, Spielberger R, Stein A, Sweetman R, Zain J, Forman S: Efficacy of mycophenolate mofetil in the treatment of chronic graft-versus-host disease. Biol Blood Marrow Transplant 2005; 11: 307-313.
12. Martin PJ, Storer BE, Rowley SD, Flowers ME, Lee SJ, Carpenter PA, Wingard JR, Shaughnessy PJ, DeVetten MP, Jasagia M, Fay JW, van Besien K, Gupta V, Kitko C, Johnston LJ, Maziarz RT, Arora M, Jacobsohn PA, Weisdorf D: Evaluation of mycophenolate mofetil for initial treatment of chronic graft-versushost disease. Blood 2009; 113: 5074-5082.
13. Jacobsohn DA, Chen AR, Zahurak M, Piantadosi S, Anders V, Bolanos-Meade J, Higman M, Margolis J, Kaup M, Vogelsang GB: Phase II study of pentostatin in patients with corticosteroid-refractory chronic graft-versushost disease. J Clin Oncol 2007; 25: 4255-4261.
14. Wolf D, Lilienfeld-Toal M, Wolf AM, Schleuning M, Von Bergwelt-Baildon M, Held SAE, Brossart P: Novel treatment concepts for graft-versus-host disease. Blood 2012; 119: 16-25.
15. Zeiser R, Nguyen VH, Beilhack A, Buess M, Schulz S, Baker J, Contag CH, Negrin RS: Inhibition of CD4+CD25+ regulatory T-cell function by calcineurin-dependent interleukin-2 production. Blood 2006; 108: 390-399.
16. Couriel DR, Saliba R, Escalon MP, Hsu Y, Ghosh S, Ippoliti C, Hicks K, Donato M, Giralt S, Khouri IF, Hosing C, de Lima MJ, Andersson B, Neumann J, Champlin R: Sirolimus in combination with tacrolimus and corticosteroids for the treatment of resistant chronic graft-versus-host disease. Br J Haematol 2005; 130: 409-417.
17. Jedlickova Z, Burlakova I, Bug G, Baurmann H, Schwerdtfeger R, Schleuning M: Therapy of sclerodermatous chronic graft-versus-host disease with mammalian target of rapamycin inhibitors. Biol Blood Marrow Transplant 2011; 17: 657-663.
18. Hoffmann P, Ermann J, Edinger M, Fathman CG, Strober S: Donor-type CD4+CD25+ regulatory T cells suppress lethal acute graft-versus-host disease after allogeneic bone marrow transplantation. J Exp Med 2002; 196: 389-399.
19. Di Ianni M, Falzetti F, Carotti A, Terenzi A, Castellino F, Bonifacio E, Del Papa B, Zei T, Ostini RI, Cecchini D, Aloisi T, Perruccio K, Ruggeri L, Balucani C, Pierini A, Sportoletti P, Fallini B, Reisner Y, Velardi A, Aversa F, Martelli MF: Tregs prevent GVHD and promote immune reconstitution in HLA-haploidentical transplantation. Blood 2011; 117: 3921-3928.
20. Koreth J, Matsuoka K, Kim HT, McDonough SM, Bindra B, Alyea EP 3rd, Armand P, Cutler C, Ho VT, Treister NS, Bienfang DC, Prasad S, Tzachanis D, Joyce RM, Avigan DE, Antin JH, Ritz J, Soiffer RJ: Interleukin-2 and regulatory T cells in graft-versus-host disease. N Engl J Med 2011; 365: 2055-2066.
21. Shin HJ, Baker J, Leveson-Gower DB, Smith AT, Sega EI, Negrin RS: Rapamycin and IL-2 reduce lethal acute graft-versus-host disease associated with increased expansion of donor type CD4+CD25+Foxp3+ regulatory T cells. Blood 2011; 118: 2342-2350.
22. Zorn E, Kim HT, Lee SJ, Floyd BH, Litsa D, Arumugarajah S, Bellucci R, Alyea EP, Antin JH, Soiffer RJ, Ritz J: Reduced frequency of FOXP3+CD4+CD25+ regulatory T cells in patients with chronic graft-versus-host disease. Blood 2005; 106: 2903-2911.
23. Cutler C, Miklos D, Kim HT, Treister N, Woo SB, Bienfang D, Klickstein LB, Levin J, Miller K, Reynolds C, Macdonnel R, Pasek M, Lee SJ, Ho V, Soiffer R, Antin JH, Ritz J, Alyea E: Rituximab for steroid-refractory chronic graft-versus-host disease. Blood 2006; 108: 756-762.
24. Kharfan-Dabaja MA, Mhaskar AR, Djulbegovic B, Cutler C, Mohty M, Kumar A: Efficacy of rituximab in the setting of steroid-refractory chronic graft-versus-host disease: a systematic review and meta-analysis. Biol Blood Marrow Transplant 2009; 15: 1005-1013.
25. Gutierrez-Aguirre CH, Cantu-Rodriguez OG, Borjas-Almaguer OD, Gonzalez-Llano O, Jaime-Perez JC, Solano-Genesta M, Gomez-Guijosa M, Mancias-Guerra C, Tarin L, Gomez-Almaguer D: Effectiveness of subcutaneous low-dose alemtuzumab and rituximab combination therapy for steroid-resistant chronic graft-versus-host disease. Haematologica 2012; 97: 717-722.
26. Van Dorp S, Pietersma F, Wolfl M, Verdonck LF, Petersen EJ, Lokhorst HM, Martens E, Theobald M, van Baarle D, Meijer E, Kuball J: Rituximab treatment before reduced-intensity conditioning transplantation associates with a decreased incidence of extensive chronic GVHD. Biol Blood Marrow Transplant 2009; 15: 671-678.
27. Arai S, Sahaf B, Narasimhan B, Chen GL, Jones CD, Lowsky R, Shizuru JA, Johnston LJ, Laport GG, Weng WK, Benjamin JE, Schaenman J, Brown J, Ramirez J, Zehnder JL, Negrin RS, Miklos DB: Prophylactic rituximab after allogeneic transplantation decreases B cell alloimmunity with low chronic GVHD incidence. Blood 2012; 119: 6145-6154.
28. Svegliati S, Olivieri A, Campelli N, Luchetti M, Poloni A, Trappolini S, Moroncini G, Bacigalupo A, Leoni P, Avvedimento EV, Gabrielli A: Stimulatory autoantibodies to PDGF receptor in patients with extensive chronic graft-versus-host disease. Blood 2007; 110: 237-241.
29. Olivieri A, Locatelli F, Zecca M, Sanna A, Cimminiello M, Raimondi R, Gini G, Mordini N, Balduzzi A, Leoni P, Gabrielli A, Bacigalupo A: Imatinib for refractory chronic graft-versus-host disease with fibrotic features. Blood 2009; 114: 709-718.
30. Stadler M, Ahlborn R, Kamal H, Diedrich H, Buchholz S, Eder M, Ganser A: Limited efficacy of imatinib in severe pulmonary chronic graft-versus-host disease. Blood 2009; 114: 3718-3719.
31. Magro L, Mohty M, Catteau B, Coiteux V, Chevallier P, Terriou L, Jouet JP, Yakoub-Agha I: Imatinib mesylate as salvage therapy for refractory sclerotic chronic graft-versushost disease. Blood 2009; 114: 719-722.
32. Sanchez-Ortega I, Servitje O, Arnan M, Orti G, Peralta T, Manresa F, Duarte RF: Dasatinib as salvage therapy for steroid refractory and imatinib resistant or intolerant sclerotic chronic graft-versus-host disease. Biol Blood Marrow Transplant 2012; 18: 318-323.
33. Blanco B, Perez-Simon JA, Sanchez-Abarca LI, Carvajal-Vergara X, Mateos J, Vidriales B, Lopez-Holgado N, Maiso P, Alberca M, Villaron E, Schenkein D, Pandiella A, San Miguel J: Bortezomib induces selective depletion of alloreactive T lymphocytes and decreases the production of Th1 cytokines. Blood 2006; 107: 3575-3583.
34. Sun K, Welniak LA, Panoskaltsis-Mortari A, O'Shaughnessy MJ, Lin H, Barao I, Riordan W, Sitcheran R, Wysocki C, Serody JS, Blazar BR, Sayers TJ, Murphy WJ: Inhibition of acute graft-versus-host disease with retention of graft-versus-tumor effects by the proteasome inhibitor bortezomib. Proc Natl Acad Sci USA 2004; 101: 8120-8125.
35. Jean El-Cheikh, Mauricette Michallet, Arnon Nagler, de Lavallade H, Nicolini FE, Shimoni A, Faucher C, Sobh M, Revesz D, Hardan I, Furst S, Blaise D, Mohty M: High response rate and improved graft-versus-host disease following bortezomib as salvage therapy after reduced intensity conditioning allogeneic stem cell transplantation for multiple myeloma. Haematologica 2008; 93: 455-458.
36. Koreth J, Stevenson KE, Kim HT, Garcia M, Ho VT, Armand P, Cutler C, Ritz J, Antin JH, Soiffer RJ, Alyea EP 3rd: Bortezomib, tacrolimus and methotrexate for prophylaxis of graft-versus-host disease after reduced-intensity conditioning allogeneic stem cell transplantation from HLA-mismatched unrelated donors. Blood 2009; 114: 3956-3959.
37. Couriel DR, Hosing C, Saliba R, Shpall EJ, Anderlini P, Rhodes B, Smith V, Khouri I, Giralt S, de Lima M, Hsu Y, Ghosh S, Neumann J, Andersson B, Qazilbash M, Hymes S, Kim S, Champlin R, Donato M: Extracorporeal photochemotherapy for the treatment of steroidresistant chronic GVHD. Blood 2006; 107: 3074-3080.
38. Flowers ME, Apperley JF, van Besien K, Elmaagacli A, Grigg A, Reddy V, Bacigalupo A, Kolb HJ, Bouzas L, Michallet M, Prince HM, Knobler R, Parenti D, Gallo J, Greinix HT: A multicenter prospective phase 2 randomized study of extracorporeal photopheresis for treatment of chronic graft-versus-host disease. Blood 2008; 112: 2667-2674.
39. Dignan FL, Greenblatt D, Cox M, Cavenagh J, Oakervee H, Apperley JF, Fielding AK, Pagliuca A, Mufti G, Raj K, Marks DI, Amrolia P, Peniket A, Medd P, Potter MN, Shaw BE, Scarisbrick JJ: Efficacy of bimonthly extracorporeal photopheresis in refractory mucocutaneous GVHD. Bone Marrow Transplant 2012; 47: 824-830.
40. Kuzmina Z, Greinix HT, Knobler R, Worel N, Kouba M, Weigl R, Kormoczi U, Rottal A, Pohlreich D, Zielinski C, Pickl WF: Proportions of immature CD19+CD21-B lymphocytes predict the response to extracorporeal photopheresis in patients with chronic graftversus-host disease. Blood 2009; 114: 744-746.
41. Whittle R, Taylor PC: Circulating B-cell activating factor level predicts clinical response of chronic graft-versus-host disease to extracorporeal photopheresis. Blood 2011; 118: 6446-6449.
42. Zhou H, Guo M, Bian C, Sun Z, Yang Z, Zeng Y, Ai H, Zhao RC: Efficacy of bone marrowderived mesenchymal stem cells in the treatment of sclerodermatous chronic graft-versus-host disease: clinical report. Biol Blood Marrow Transplant 2010; 16: 403-412.
43. Weng JY, Du X, Geng SX, Peng YW, Wang Z, Lu ZS, Wu SJ, Luo CW, Guo R, Ling W, Deng CX, Liao PJ, Xiang AP: Mesenchymal stem cell as salvage treatment for refractory chronic GVHD. Bone Marrow Transplant 2010; 45: 1732-1740.
44. Ning H, Yang F, Jiang M, Hu L, Feng K, Zhang J, Yu Z, Li B, Xu C, Li Y, Wang J, Hu J, Lou X, Chen H: The correlation between cotransplantation of mesenchymal stem cells and higher recurrence rate in hematologic malignancy patients: outcome of a pilot clinical study. Leukemia 2008; 22: 593-599.
45. Le Blanc K, Frassoni F, Ball L, Locatelli F, Roelofs H, Lewis I, Lanino E, Sundberg B, Bernardo ME, Remberger M, Dini G, Egeler RM, Bacigalupo A, Fibbe W, Ringden O, Developmental Committee of the European Group for Blood and Marrow Transplantation: Mesenchymal stem cells for treatment of steroid-resistant, severe, acute graft-versus-host disease: a phase II study. Lancet 2008; 371: 1579-1586.
46. Kebriaei P, Robinson S: Treatment of graftversus-host disease with mesenchymal stromal cells. Cytotherapy 2011; 13: 262-268.
47. Levine JE, Paczesny S, Sarantopoulos S: Clinical applications for biomarkers of acute and chronic graft-versus-host disease. Biol Blood Marrow Transplant 2012; 18(1 suppl):S116-S124.