Hematopoietic transplant-associated thrombotic microangiopathy: Case report and review of diagnosis and treatments

Clinical Advances in Hematology and Oncology

Volumn 12, Issue 9, 2014, Pages 565-573

  Chapin, John ^a   Shore, Tsiporah ^a   Forsberg, Peter ^a   Desman, Garrett ^a   Van Besien, Koen ^a   Laurence, Jeffrey ^a  

^a WEILL CORNELL MEDICAL COLLEGE   (United States)

Author keywords

Complement; Eculizumab; Hematopoietic stem cell transplant; Hemolytic uremic syndrome; Thrombotic microangiopathy; Thrombotic thrombocytopenic purpura

Indexed keywords

BORTEZOMIB; BUSULFAN; CALCINEURIN INHIBITOR; COMPLEMENT COMPONENT C5B; COMPLEMENT MEMBRANE ATTACK COMPLEX; CREATININE; CYCLOSPORIN; ECULIZUMAB; FLUDARABINE; HAPTOGLOBIN; HEMOGLOBIN; LACTATE DEHYDROGENASE; MONOCLONAL ANTIBODY; RAPAMYCIN; RITUXIMAB; TACROLIMUS; VON WILLEBRAND FACTOR CLEAVING PROTEINASE;

ADULT; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ARTICLE; CASE REPORT; COMPLEMENT ACTIVATION; DISSEMINATED INTRAVASCULAR CLOTTING; ENGRAFTMENT; GRAFT VERSUS HOST REACTION; HEMOLYTIC ANEMIA; HEMOLYTIC UREMIC SYNDROME; HUMAN; HUMAN TISSUE; HYPERTENSION; IMMUNOHISTOCHEMISTRY; IMMUNOSUPPRESSIVE TREATMENT; MALE; MICROVASCULATURE; PATHOLOGY; PROTEIN EXPRESSION; PROTEINURIA; RISK FACTOR; SKIN BIOPSY; SPLENOMEGALY; THROMBOCYTE COUNT; THROMBOCYTOPENIA; THROMBOTIC THROMBOCYTOPENIC PURPURA; ADVERSE EFFECTS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; THROMBOTIC MICROANGIOPATHIES;

ADULT; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; MALE; THROMBOTIC MICROANGIOPATHIES;

EID: 84907206672     PISSN: 15430790     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (66)

1. Ricklin D, Cines DB. TMA: beware of complements. Blood. 2013;122(12):1997-1999.
2. George JN, Selby GB. Thrombotic microangiopathy after allogeneic bone marrow transplantation: a pathologic abnormality associated with diverse clinical syndromes. Bone Marrow Transplant. 2004;33(11):1073-1074.
3. Changsirikulchai S, Myerson D, Guthrie KA, McDonald GB, Alpers CE, Hingorani SR. Renal thrombotic microangiopathy after hematopoietic cell transplant: role of GVHD in pathogenesis. Clin J Am Soc Nephrol. 2009;4(2):345-353.
4. Nakamae H, Yamane T, Hasegawa T, et al. Risk factor analysis for thrombotic microangiopathy after reduced-intensity or myeloablative allogeneic hematopoietic stem cell transplantation. Am J Hematol. 2006;81(7):525-531.
5. Willems E, Baron F, Seidel L, Frère P, Fillet G, Beguin Y. Comparison of thrombotic microangiopathy after allogeneic hematopoietic cell transplantation with high-dose or nonmyeloablative conditioning. Bone Marrow Transplant. 2010;45(4):689-693.
6. Uderzo C, Bonanomi S, Busca A, et al. Risk factors and severe outcome in thrombotic microangiopathy after allogeneic hematopoietic stem cell transplantation. Transplantation. 2006;82(5):638-644.
7. van der Plas RM, Schiphorst ME, Huizinga EG, et al. von Willebrand factor proteolysis is deficient in classic, but not in bone marrow transplantation-associated, thrombotic thrombocytopenic purpura. Blood. 1999;93(11):3798-3802.
8. Peyvandi F, Siboni SM, Lambertenghi Deliliers D, et al. Prospective study on the behaviour of the metalloprotease ADAMTS13 and of von Willebrand factor after bone marrow transplantation. Br J Haematol. 2006;134(2):187-195.
9. Elliott MA, Nichols WL Jr, Plumhoff EA, et al. Posttransplantation thrombotic thrombocytopenic purpura: a single-center experience and a contemporary review. Mayo Clin Proc. 2003;78(4):421-430.
10. Arends MJ, Harrison DJ. Novel histopathologic findings in a surviving case of hemolytic uremic syndrome after bone marrow transplantation. Hum Pathol. 1989;20(1):89-91.
11. Laurence J. Atypical hemolytic uremic syndrome (aHUS): making the diagnosis. Clin Adv Hematol Oncol. 2012;10(10)(suppl 17):1-12.
12. Tsai HM. Pathophysiology of thrombotic thrombocytopenic purpura. Int J Hematol. 2010;91(1):1-19.
13. Hosler GA, Cusumano AM, Hutchins GM. Thrombotic thrombocytopenic purpura and hemolytic uremic syndrome are distinct pathologic entities. A review of 56 autopsy cases. Arch Pathol Lab Med. 2003;127(7):834-839.
14. Jodele S, Licht C, Goebel J, et al. Abnormalities in the alternative pathway of complement in children with hematopoietic stem cell transplant-associated thrombotic microangiopathy. Blood. 2013;122(12):2003-2007.
15. Siami K, Kojouri K, Swisher KK, Selby GB, George JN, Laszik ZG. Thrombotic microangiopathy after allogeneic hematopoietic stem cell transplantation: an autopsy study. Transplantation. 2008;85(1):22-28.
16. Cutler C, Henry NL, Magee C, et al. Sirolimus and thrombotic microangiopathy after allogeneic hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2005;11(7):551-557.
17. Hershko K, Simhadri VL, Blaisdell A, et al. Cyclosporin A impairs the secretion and activity of ADAMTS13 (a disintegrin and metalloprotease with thrombospondin type 1 repeat). J Biol Chem. 2012;287(53):44361-44371.
18. Fuge R, Bird JM, Fraser A, et al. The clinical features, risk factors and outcome of thrombotic thrombocytopenic purpura occurring after bone marrow transplantation. Br J Haematol. 2001;113(1):58-64.
19. Daly AS, Hasegawa WS, Lipton JH, Messner HA, Kiss TL. Transplantationassociated thrombotic microangiopathy is associated with transplantation from unrelated donors, acute graft-versus-host disease and venoocclusive disease of the liver. Transfus Apheresis Sci. 2002;27(1):3-12.
20. Inamoto Y, Ito M, Suzuki R, et al; Nagoya Blood and Marrow Transplantation Group. Clinicopathological manifestations and treatment of intestinal transplantassociated microangiopathy. Bone Marrow Transplant. 2009;44(1):43-49.
21. Laskin BL, Goebel J, Davies SM, et al. Early clinical indicators of transplantassociated thrombotic microangiopathy in pediatric neuroblastoma patients undergoing auto-SCT. Bone Marrow Transplant. 2011;46(5):682-689.
22. Iacopino P, Pucci G, Arcese W, et al; Gruppo Italiano Trapianto Midollo Osseo (GITMO). Severe thrombotic microangiopathy: an infrequent complication of bone marrow transplantation. Bone Marrow Transplant. 1999;24(1):47-51.
23. Holler E, Kolb HJ, Hiller E, et al. Microangiopathy in patients on cyclosporine prophylaxis who developed acute graft-versus-host disease after HLA-identical bone marrow transplantation. Blood. 1989;73(7):2018-2024.
24. Wolff D, Wilhelm S, Hahn J, et al. Replacement of calcineurin inhibitors with daclizumab in patients with transplantation-associated microangiopathy or renal insufficiency associated with graft-versus-host disease. Bone Marrow Transplant. 2006;38(6):445-451.
25. Kersting S, Koomans HA, Hené RJ, Verdonck LF. Acute renal failure after allogeneic myeloablative stem cell transplantation: retrospective analysis of incidence, risk factors and survival. Bone Marrow Transplant. 2007;39(6):359-365.
26. Roy V, Rizvi MA, Vesely SK, George JN. Thrombotic thrombocytopenic purpuralike syndromes following bone marrow transplantation: an analysis of associated conditions and clinical outcomes. Bone Marrow Transplant. 2001;27(6):641-646.
27. Ruutu T, Hermans J, Niederwieser D, et al; EBMT Chronic Leukaemia Working Party. Thrombotic thrombocytopenic purpura after allogeneic stem cell transplantation: a survey of the European Group for Blood and Marrow Transplantation (EBMT). Br J Haematol. 2002;118(4):1112-1119.
28. Ho VT, Cutler C, Carter S, et al. Blood and marrow transplant clinical trials network toxicity committee consensus summary: thrombotic microangiopathy after hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2005;11(8):571-575.
29. Le Quintrec M, Roumenina L, Noris M, Frémeaux-Bacchi V. Atypical hemolytic uremic syndrome associated with mutations in complement regulator genes. Semin Thromb Hemost. 2010;36(6):641-652.
30. Roumenina LT, Jablonski M, Hue C, et al. Hyperfunctional C3 convertase leads to complement deposition on endothelial cells and contributes to atypical hemolytic uremic syndrome. Blood. 2009;114(13):2837-2845.
31. Roumenina LT, Loirat C, Dragon-Durey MA, Halbwachs-Mecarelli L, Sautes-Fridman C, Fremeaux-Bacchi V. Alternative complement pathway assessment in patients with atypical HUS. J Immunol Methods. 2011;365(1-2):8-26.
32. Roumenina LT, Roquigny R, Blanc C, et al. Functional evaluation of factor H genetic and acquired abnormalities: application for atypical hemolytic uremic syndrome (aHUS). Methods Mol Biol. 2014;1100:237-247.
33. Cohen JA, Brecher ME, Bandarenko N. Cellular source of serum lactate dehydrogenase elevation in patients with thrombotic thrombocytopenic purpura. J Clin Apher. 1998;13(1):16-19.
34. Cho BS, Yahng SA, Lee SE, et al. Validation of recently proposed consensus criteria for thrombotic microangiopathy after allogeneic hematopoietic stem-cell transplantation. Transplantation. 2010;90(8):918-926.
35. Copelovitch L, Kaplan BS. The thrombotic microangiopathies. Pediatr Nephrol. 2008;23(10):1761-1767.
36. Ruutu T, Barosi G, Benjamin RJ, et al; European Group for Blood and Marrow Transplantation; European LeukemiaNet. Diagnostic criteria for hematopoietic stem cell transplant-associated microangiopathy: results of a consensus process by an International Working Group. Haematologica. 2007;92(1):95-100.
37. Kanamori H, Takaishi Y, Takabayashi M, et al. Clinical significance of fragmented red cells after allogeneic bone marrow transplantation. Int J Hematol. 2003;77(2):180-184.
38. Jodele S, Davies SM, Lane A, et al. Diagnostic and risk criteria for HSCTassociated thrombotic microangiopathy: a study in children and young adults. Blood. 2014;124(4):645-653.
39. Chapin J, Weksler B, Magro C, Laurence J. Eculizumab in the treatment of refractory idiopathic thrombotic thrombocytopenic purpura. Br J Haematol. 2012;157(6):772-774.
40. Tsai E, Chapin J, Laurence J., Tsai HM. Use of eculizumab in the treatment of a case of refractory, ADAMTS13-deficient thrombotic thrombocytopenic purpura: additional data and clinical follow-up. Br J Haematol. 2013;162(4):558-559.
41. Chapin J, Eyler S, Smith R, Tsai H-M, Laurence J. Complement factor H mutations are present in ADAMTS13-deficient, ticlopidine-associated thrombotic microangiopathies. Blood. 2013;121(19):4012-4013.
42. Rock GA, Shumak KH, Buskard NA, et al; Canadian Apheresis Study Group. Comparison of plasma exchange with plasma infusion in the treatment of thrombotic thrombocytopenic purpura. N Engl J Med. 1991;325(6):393-397.
43. Laurence J. Atypical hemolytic uremic syndrome (aHUS): treating the patient. Clin Adv Hematol Oncol. 2013;11(10)(suppl 15):4-15.
44. Sarode R, McFarland JG, Flomenberg N, et al. Therapeutic plasma exchange does not appear to be effective in the management of thrombotic thrombocytopenic purpura/hemolytic uremic syndrome following bone marrow transplantation. Bone Marrow Transplant. 1995;16(2):271-275.
45. Riedl M, Fakhouri F, Le Quintrec M, et al. Spectrum of complement-mediated thrombotic microangiopathies: pathogenic insights identifying novel treatment approaches. Semin Thromb Hemost. 2014;40(4):444-464.
46. Jodele S, Laskin BL, Goebel J, et al. Does early initiation of therapeutic plasma exchange improve outcome in pediatric stem cell transplant-associated thrombotic microangiopathy? Transfusion. 2013;53(3):661-667.
47. Howard MA, Williams LA, Terrell DR, Duvall D, Vesely SK, George JN. Complications of plasma exchange in patients treated for clinically suspected thrombotic thrombocytopenic purpura-hemolytic uremic syndrome. Transfusion. 2006;46(1):154-156.
48. Takatsuka H, Takemoto Y, Yamada S, et al. Complications after bone marrow transplantation are manifestations of systemic inflammatory response syndrome. Bone Marrow Transplant. 2000;26(4):419-426.
49. Worel N, Greinix HT, Leitner G, et al. ABO-incompatible allogeneic hematopoietic stem cell transplantation following reduced-intensity conditioning: close association with transplant-associated microangiopathy. Transfus Apheresis Sci. 2007;36(3):297-304.
50. Scully M, McDonald V, Cavenagh J, et al. A phase 2 study of the safety and efficacy of rituximab with plasma exchange in acute acquired thrombotic thrombocytopenic purpura. Blood. 2011;118(7):1746-1753.
51. Scully M, Hunt BJ, Benjamin S, et al; British Committee for Standards in Haematology. Guidelines on the diagnosis and management of thrombotic thrombocytopenic purpura and other thrombotic microangiopathies. Br J Haematol. 2012;158(3):323-335.
52. Scully M. Rituximab in the treatment of TTP. Hematology. 2012;17(S1)(suppl 1):S22-S24.
53. Au WY, Ma ES, Lee TL, et al. Successful treatment of thrombotic microangiopathy after haematopoietic stem cell transplantation with rituximab. Br J Haematol. 2007;137(5):475-478.
54. Laskin BL, Goebel J, Davies SM, Jodele S. Small vessels, big trouble in the kidneys and beyond: hematopoietic stem cell transplantation-associated thrombotic microangiopathy. Blood. 2011;118(6):1452-1462.
55. Sana G, Dragon-Durey M-A, Charbit M, et al. Long-term remission of atypical HUS with anti-factor H antibodies after cyclophosphamide pulses. Pediatr Nephrol. 2014;29(1):75-83.
56. Corti P, Uderzo C, Tagliabue A, et al. Defibrotide as a promising treatment for thrombotic thrombocytopenic purpura in patients undergoing bone marrow transplantation. Bone Marrow Transplant. 2002;29(6):542-543.
57. Legendre CM, Licht C, Loirat C. Eculizumab in atypical hemolytic-uremic syndrome. N Engl J Med. 2013;369(14):1379-1380.
58. Réti M, Farkas P, Csuka D, et al. Complement activation in thrombotic thrombocytopenic purpura. J Thromb Haemost. 2012;10(5):791-798.
59. Wu TC, Yang S, Haven S, et al. Complement activation and mortality during an acute episode of thrombotic thrombocytopenic purpura. J Thromb Haemost. 2013;11(10):1925-1927.
60. Cataland SR, Holers VM, Geyer S, Yang S, Wu HM. Biomarkers of the alternative pathway and terminal complement activity at presentation confirms the clinical diagnosis of aHUS and differentiates aHUS from TTP. Blood. 2014;123(24):3733-3738.
61. Marshall RJ, Sweny P. Haemolytic-uraemic syndrome in recipients of bone marrow transplants not treated with cyclosporin A. Histopathology. 1986;10(9):953-962.
62. Laskin BL, Maisel J, Goebel J, et al. Renal arteriolar C4d deposition: a novel characteristic of hematopoietic stem cell transplantation-associated thrombotic microangiopathy. Transplantation. 2013;96(2):217-223.
63. Jodele S, Fukuda T, Vinks A, et al. Eculizumab therapy in children with severe hematopoietic stem cell transplantation-associated thrombotic microangiopathy. Biol Blood Marrow Transplant. 2014;20(4):518-525.
64. Skerka C, Zipfel PF, Müller D, et al. The autoimmune disease DEAP-hemolytic uremic syndrome. Semin Thromb Hemost. 2010;36(6):625-632.
65. Kim CH, Wu W, Wysoczynski M, et al. Conditioning for hematopoietic transplantation activates the complement cascade and induces a proteolytic environment in bone marrow: a novel role for bioactive lipids and soluble C5b-C9 as homing factors. Leukemia. 2012;26(1):106-116.
66. Legendre CM, Licht C, Muus P, et al. Terminal complement inhibitor eculizumab in atypical hemolytic-uremic syndrome. N Engl J Med. 2013;368(23):2169-2181.