Steroid-refractory acute graft-versus-host disease graded III-IV in pediatric patients. A mono-institutional experience with a long-term follow-up.
Acute Disease
Adolescent
Child
Child, Preschool
Drug Resistance
Female
Follow-Up Studies
Glucocorticoids
/ pharmacology
Graft vs Host Disease
/ diagnosis
Hematologic Neoplasms
/ therapy
Hematopoietic Stem Cell Transplantation
/ adverse effects
Humans
Immunosuppressive Agents
/ therapeutic use
Infant
Italy
/ epidemiology
Male
Prognosis
Retrospective Studies
Severity of Illness Index
Survival Rate
/ trends
Time Factors
Transplantation, Homologous
children
severe acute graft-versus-host disease
survival
Journal
Pediatric transplantation
ISSN: 1399-3046
Titre abrégé: Pediatr Transplant
Pays: Denmark
ID NLM: 9802574
Informations de publication
Date de publication:
11 2020
11 2020
Historique:
received:
27
11
2019
revised:
28
05
2020
accepted:
09
07
2020
pubmed:
28
8
2020
medline:
8
10
2021
entrez:
27
8
2020
Statut:
ppublish
Résumé
aGvHD remains a major obstacle to successful HSCT. We report our experience on steroid-refractory aGvHD III and IV from 1989 to 2017. Ninety patients with aGvHD III or IV were stratified according to the HSCT year: 1989-1998, 1999-2007, and 2008-2017 and to aGvHD extension (GvHD III vs IV) and finally the probability of OS, RI, and TRM was calculated accordingly. aGvHD III patients had a substantial improvement over time: day 100 OS raised from 64% (95% CI 39-89) in the first cohort to 100% in the latest (P = .022), and it was mainly due to a reduction of TRM (it was 28% [95% CI 12-65] in the first cohort to 0% in the latest (P = .01). The aGvHD IV patients did not present a significant improvement. Day 100 OS was 42% (95% CI 16-68) in the first group and 54% (95% CI 25-83) in the year 2008-2017 (P = NS), and the day-100 TRM was very similar (it was 57% [95% CI 36-90] in the first cohort and 45% [95% CI 23-89] in the latest (P = NS). We report significant improvements in OS and TRM in patients diagnosed with grade III aGvHD. Patients with the most severe aGvHD appear to have no or fewer benefits on long-term outcomes.
Substances chimiques
Glucocorticoids
0
Immunosuppressive Agents
0
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
e13806Informations de copyright
© 2020 Wiley Periodicals LLC.
Références
Deeg HJ. How I treat refractory acute GVHD. Blood. 2007;109:4119-4126.
Ferrara JL, Levine JE, Reddy P, Holler E. Graft-versus-host disease. Lancet. 2009;373:1550-1561.
Socié G, Blazar BR. Acute graft-versus-host disease: from the bench to the bedside. Blood. 2009;114:4327-4336.
Hill L, Alousi A, Kebriaei P, Mehta R, Rezvani K, Shpa E. New and emerging therapies for acute and chronic graft versus host disease. Ther Adv Hematol. 2018;9:21-46.
Nassereddine S, Rafei H, Elbahesh E, Tabbara I. Acute graft versus host disease: a comprehensive review. Anticancer Res. 2017;37:1547-1555.
Martin PJ, Rizzo JD, Wingard JR, et al. First- and second-line systemic treatment of acute graft-versus-host disease: recommendations of the American Society of Blood and Marrow Transplantation. Biol Blood Marrow Transplant. 2012;18(8):1150-1163.
Glucksberg H, Storb R, Fefer A, et al. Clinical manifestations of graft-versus-host disease in human recipients of marrow from HL-A-matched sibling donors. Transplantation. 1974;18:295-304.
Shulman HM, Sullivan KM, Weiden PL, et al. Chronic graft-versus-host syndrome in man. A long-term clinicopathologic study of 20 Seattle patients. Am J Med. 1980;69:204-217.
Jagasia MH, Greinix HT, Arora M, et al. National institutes of health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. The 2014 diagnosis and staging working group report. Biol Blood Marrow Transplant. 2015;21:389-401.
Kaplan EL, Meier P. Nonparametric estimation from incomplete observation. J Am Stat Assoc. 1958;53:457-481.
Gooley TA, Leisenring W, Crowley J, Storer BE. Estimation of failure probabilities in the presence of competing risk: new representation of old estimators. Stat Med. 1999;18:695-706.
Gray R. A class of K-sample tests for comparing the cumulative incidence of a competing risk. Ann Stat. 1988;16:1141-1154.
El-Jawahri A, Li S, Antin JH, et al. Supportive care improved treatment-related mortality and overall survival of patients with grade IV acute GVHD in the modern years. Biol Blood Marrow Transplant. 2016;22:910-918.
Srinivasan R, Chakrabarti S, Walsh T, et al. Improved survival in steroid-refractory acute graft versus host disease after non-myeloablative allogeneic transplantation using a daclizumab-based strategy with comprehensive infection prophylaxis. Br J Haematol. 2004;124:777-786.
Klein SA, Bug G, Mousset S, et al. Long term outcome of patients with steroid- refractory acute intestinal graft versus host disease after treatment with pentostatin. Br J Haematol. 2011;154:143-146.
Schub N, Günther A, Schrauder A, et al. Therapy of steroid-refractory acute GVHD with CD52 antibody alemtuzumab is effective. Bone Marrow Transplant. 2011;46:143-147.
Bolaños-Meade J, Logan BR, Alousi AM, et al. Phase 3 clinical trial of steroids/mycophenolate mofetil vs steroids/placebo as therapy for acute GVHD: BMT CTN 0802. Blood. 2014;124:3221-3227.
Wolf D, von Lilienfeld-Toal M, Wolf AM, et al. Novel treatment concepts for graft-versus- host disease. Blood. 2012;119(1):16-25.
Reshef R, Luger SM, Hexner EO, et al. Blockade of lymphocyte chemotaxis in visceral graft-versus-host disease. N Engl J Med. 2012;367(2):135-145.
Kharfan-Dabaja MA, Cutler CS. Rituximab for prevention and treatment of graft-versus- host disease. Int J Hematol. 2011;93(5):578-585.
Cahn JY, Bordigoni P, Tiberghien P, et al. Treatment of acute graft-versus-host disease with methylprednisolone and cyclosporine with or without an anti-interleukin-2 receptor monoclonal antibody. A multicenter phase III study. Transplantation. 1995;60(9):939-942.
Lee SJ, Zahrieh D, Agura E, et al. Effect of up-front daclizumab when combined with steroids for the treatment of acute graft-versus-host disease: results of a randomized trial. Blood. 2004;104(5):1559-1564.
Cragg L, Blazar BR, Defor T, et al. A randomized trial comparing prednisone with antithymocyte globulin/prednisone as an initial systemic therapy for moderately severe acute graft-versus-host disease. Biol Blood Marrow Transplant. 2000;6(4A):441-447.
Couriel DR, Saliba R, de Lima M, et al. A phase III study of infliximab and corticosteroids for the initial treatment of acute graft-versus-host disease. Biol Blood Marrow Transplant. 2009;15(12):1555-1562.
Levine JE, Paczesny S, Mineishi S, et al. Etanercept plus methylprednisolone as initial therapy for acute graft-versus host disease. Blood. 2008;111(4):2470-2475.
Alousi AM, Weisdorf DJ, Logan BR, et al. Etanercept, mycophenolate, denileukin, or pentostatin plus corticosteroids for acute graft-versus-host disease: a randomized phase 2 trial from the Blood and Marrow Transplant Clinical Trials Network. Blood. 2009;114(3):511-517.
Pidala J, Kim J, Anasetti C. Sirolimus as primary treatment of acute graft-versus-host disease following allogeneic hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2009;15(7):881-885.
MacMillan ML, Weisdorf DJ, Davies SM, et al. Early antithymocyte globulin therapy improves survival in patients with steroid-resistant acute graft-versus-host disease. Biol Blood Marrow Transplant. 2002;8(1):40-46.
Furlong T, Martin P, Flowers MED, et al. Therapy with mycophenolate mofetil for refractory acute and chronic GVHD. Bone Marrow Transplant. 2009;44(11):739-748.
Jamani K, Russell JA, Daly A, et al. Prognosis of grade 3-4 acute GVHD continues to be dismal. Bone Marrow Transplant. 2013;48:1359-1361.
Horan JT, Logan BR, Agovi-Johnson M-A, et al. Reducing the risk for transplantation- related mortality after allogeneic hematopoietic cell transplantation: how much progress has been made? J Clin Oncol. 2011;29:805-813.
Pavletic SZ, Fowler DH. Are we making progress in GVHD prophylaxis and treatment? Hematology Am Soc Hematol Educ Program. 2012;2012:251-264.
Drobyski WR, Pasquini M, Kovatovic K, et al. Tocilizumab for the treatment of steroid refractory graft-versus-host disease. Biol Blood Marrow Transplant. 2011;17:1862-1868.
Jaiswal SR, Zaman S, Chakrabarti A, et al. T cell costimulation blockade for hyperacute steroid refractory graft versus-host disease in children undergoing haploidentical transplantation. Transpl Immunol. 2016;39:46-51.
Kanate AS, Hari PN, Pasquini MC, et al. Recipient immune modulation with atorvastatin for acute graft-versus-host disease prophylaxis after allogeneic transplantation. Biol Blood Marrow Transplant. 2017;23:1295-1302.
Holtan SG, Weisdorf DJ. Vorinostat is victorious in GVHD prevention. Blood. 2017;12(130):1690-1691.
González Vicent M, Molina B, et al. Ruxolitinib treatment for steroid refractory acute and chronic graft vs host disease in children: clinical and immunological results. Am J Hematol. 2019;94:319-326.
Wu J, Gu J, Zhou S, et al. Anti-IL-22 antibody attenuates acute graft-versus-host disease via increasing Foxp3(+) T cell through modulation of CD11b(+) cell function. J Immunol Res. 2018;2018(7):1605341.
Danylesko I, Bukauskas A, Paulson M, et al. Anti-α4β7 integrin monoclonal antibody (vedolizumab) for the treatment of steroid-resistant severe intestinal acute graft-versus-host disease. Bone Marrow Transplant. 2019;54:987-993.
Gao F, Chiu SM, Motan DAL, et al. Mesenchymal stem cells and immunomodulation: current status and future prospects. Cell Death Dis. 2016;7(1):e2062.
Qi X, Li X, Zhao YE, et al. Treating steroid refractory intestinal acute graft-vs.-host disease with Fecal Microbiota transplantation: a pilot study. Front Immunol. 2018;25(9):2195.
Koreth J, Ritz J. Tregs, HSCT, and acute GVHD: up close and personal. Blood. 2013;5(122):1690-1691.
Hill GR, Crawford JM, Cooke KR, Brinson YS, Pan L, Ferrara JL. Total body irradiation and acute graft-versus-host disease: the role of gastrointestinal damage and inflammatory cytokines. Blood. 1997;90(8):3204-3213.
Hill GR, Ferrara JL. The primacy of the gastrointestinal tract as a target organ of acute graft- versus-host disease: rationale for the use of cytokine shields in allogeneic bone marrow transplantation. Blood. 2000;95(9):2754-2759.
Burman AC, Banovic T, Kuns RD, et al. IFNgamma differentially controls the development of idiopathic pneumonia syndrome and GVHD of the gastrointestinal tract. Blood. 2007;110(3):1064-1072.
Robb RJ, Hill GR. The interferon-dependent orchestration of innate and adaptive immunity after transplantation. Blood. 2012;119(23):5351-5358.
Yi T, Chen Y, Wang L, et al. Reciprocal differentiation and tissue-specific pathogenesis of Th1, Th2, and Th17 cells in graft-versus-host disease. Blood. 2009;114(14):3101-3112.
Serody JS, Hill GR. The IL-17 differentiation pathway and its role in transplant outcome. Biol Blood Marrow Transplant. 2012;18(1 suppl):S56-S61.
Couturier M, Lamarthée B, Arbez J, et al. IL-22 deficiency in donor T cells attenuates murine acute graft-versus-host disease mortality while sparing the graft-versus-leukemia effect. Leukemia. 2013;27(7):1527-1537.
Bucher C, Koch L, Vogtenhuber C, et al. IL-21 blockade reduces graft-versus-host disease mortality by supporting inducible T regulatory cell generation. Blood. 2009;114(26):5375-5384.
Nikolic B, Lee S, Bronson RT, Grusby MJ, Sykes M. Th1 and Th2 mediate acute graft- versus-host disease, each with distinct end-organ targets. J Clin Invest. 2000;105(9):1289-1298.
Bacigalupo A, Lamparelli T, Bruzzi P, et al. Antithymocyte globulin for graft-versus-host disease prophylaxis in transplants from unrelated donors: 2 randomized studies from Gruppo Italiano Trapianti Midollo Osseo (GITMO). Blood. 2001;98(10):2942-2947.