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Transplant-Associated Thrombotic Microangiopathy

MedGen UID:
1758357
•Concept ID:
C5417748
•
Disease or Syndrome

Definition

Thrombotic microangiopathy that presents in some patients undergoing hematopoietic stem cell transplantation (HSCT). Though similar in presentation to thrombotic thrombocytopenic purpura (TTP) and atypical hemolytic uremic syndrome (aHUS), transplant-associated thrombotic microangiopathy (TA-TMA) is a distinct disorder. Several risk factors may be associated with TA-TMA, including high-dose chemotherapy, radiation therapy, HLA mismatch, use of calcineurin inhibitors, acute graft-versus-host disease (GVHD), and infections. [from NCI]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVTransplant-Associated Thrombotic Microangiopathy

Professional guidelines

PubMed

Diagnosis and management of endothelial disorders following haematopoietic stem cell transplantation.
McEwan A, Greenwood M, Ward C, Ritchie D, Szer J, Gardiner E, Colic A, Sipavicius J, Panek-Hudson Y, Kerridge I
Intern Med J 2023 Dec;53(12):2162-2174. Epub 2023 Aug 1 doi: 10.1111/imj.16188. PMID: 37528613
Transplant-associated thrombotic microangiopathy: Diagnostic challenges and management strategies.
Elemary M, Sabry W, Seghatchian J, Goubran H
Transfus Apher Sci 2019 Jun;58(3):347-350. Epub 2019 May 1 doi: 10.1016/j.transci.2019.04.022. PMID: 31097308
Complement in Pathophysiology and Treatment of Transplant-Associated Thrombotic Microangiopathies.
Jodele S
Semin Hematol 2018 Jul;55(3):159-166. Epub 2018 Apr 11 doi: 10.1053/j.seminhematol.2018.04.003. PMID: 30032753

Recent clinical studies

Etiology

Transplant-Associated Thrombotic Microangiopathy in the Context of Allogenic Hematopoietic Stem Cell Transplantation: Where We Stand.
Lazana I
Int J Mol Sci 2023 Jan 6;24(2) doi: 10.3390/ijms24021159. PMID: 36674666Free PMC Article
Emerging therapeutic and preventive approaches to transplant-associated thrombotic microangiopathy.
Schoettler M, Chonat S, Williams K, Lehmann L
Curr Opin Hematol 2021 Nov 1;28(6):408-416. doi: 10.1097/MOH.0000000000000687. PMID: 34534983Free PMC Article
Complement blockade for TA-TMA: lessons learned from a large pediatric cohort treated with eculizumab.
Jodele S, Dandoy CE, Lane A, Laskin BL, Teusink-Cross A, Myers KC, Wallace G, Nelson A, Bleesing J, Chima RS, Hirsch R, Ryan TD, Benoit S, Mizuno K, Warren M, Davies SM
Blood 2020 Mar 26;135(13):1049-1057. doi: 10.1182/blood.2019004218. PMID: 31932840Free PMC Article
Transplant-associated thrombotic microangiopathy: Diagnostic challenges and management strategies.
Elemary M, Sabry W, Seghatchian J, Goubran H
Transfus Apher Sci 2019 Jun;58(3):347-350. Epub 2019 May 1 doi: 10.1016/j.transci.2019.04.022. PMID: 31097308
Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy.
Elsallabi O, Bhatt VR, Dhakal P, Foster KW, Tendulkar KK
Clin Appl Thromb Hemost 2016 Jan;22(1):12-20. Epub 2015 Aug 2 doi: 10.1177/1076029615598221. PMID: 26239316

Diagnosis

Transplant-Associated Thrombotic Microangiopathy in the Context of Allogenic Hematopoietic Stem Cell Transplantation: Where We Stand.
Lazana I
Int J Mol Sci 2023 Jan 6;24(2) doi: 10.3390/ijms24021159. PMID: 36674666Free PMC Article
Transplant-associated thrombotic microangiopathy: theoretical considerations and a practical approach to an unrefined diagnosis.
Young JA, Pallas CR, Knovich MA
Bone Marrow Transplant 2021 Aug;56(8):1805-1817. Epub 2021 Apr 19 doi: 10.1038/s41409-021-01283-0. PMID: 33875812Free PMC Article
Complement blockade for TA-TMA: lessons learned from a large pediatric cohort treated with eculizumab.
Jodele S, Dandoy CE, Lane A, Laskin BL, Teusink-Cross A, Myers KC, Wallace G, Nelson A, Bleesing J, Chima RS, Hirsch R, Ryan TD, Benoit S, Mizuno K, Warren M, Davies SM
Blood 2020 Mar 26;135(13):1049-1057. doi: 10.1182/blood.2019004218. PMID: 31932840Free PMC Article
Transplant-associated thrombotic microangiopathy: Diagnostic challenges and management strategies.
Elemary M, Sabry W, Seghatchian J, Goubran H
Transfus Apher Sci 2019 Jun;58(3):347-350. Epub 2019 May 1 doi: 10.1016/j.transci.2019.04.022. PMID: 31097308
Transplant-associated thrombotic microangiopathy: opening Pandora's box.
Gavriilaki E, Sakellari I, Anagnostopoulos A, Brodsky RA
Bone Marrow Transplant 2017 Oct;52(10):1355-1360. Epub 2017 Mar 13 doi: 10.1038/bmt.2017.39. PMID: 28287636

Therapy

Reeling in complement in transplant-associated thrombotic microangiopathy: You're going to need a bigger boat.
Jodele S, Sabulski A
Am J Hematol 2023 May;98 Suppl 4:S57-S73. doi: 10.1002/ajh.26872. PMID: 36746623
Transplant-Associated Thrombotic Microangiopathy in the Context of Allogenic Hematopoietic Stem Cell Transplantation: Where We Stand.
Lazana I
Int J Mol Sci 2023 Jan 6;24(2) doi: 10.3390/ijms24021159. PMID: 36674666Free PMC Article
Emerging therapeutic and preventive approaches to transplant-associated thrombotic microangiopathy.
Schoettler M, Chonat S, Williams K, Lehmann L
Curr Opin Hematol 2021 Nov 1;28(6):408-416. doi: 10.1097/MOH.0000000000000687. PMID: 34534983Free PMC Article
Transplant-associated thrombotic microangiopathy: theoretical considerations and a practical approach to an unrefined diagnosis.
Young JA, Pallas CR, Knovich MA
Bone Marrow Transplant 2021 Aug;56(8):1805-1817. Epub 2021 Apr 19 doi: 10.1038/s41409-021-01283-0. PMID: 33875812Free PMC Article
Complement blockade for TA-TMA: lessons learned from a large pediatric cohort treated with eculizumab.
Jodele S, Dandoy CE, Lane A, Laskin BL, Teusink-Cross A, Myers KC, Wallace G, Nelson A, Bleesing J, Chima RS, Hirsch R, Ryan TD, Benoit S, Mizuno K, Warren M, Davies SM
Blood 2020 Mar 26;135(13):1049-1057. doi: 10.1182/blood.2019004218. PMID: 31932840Free PMC Article

Prognosis

Pathway-driven rare germline variants associated with transplant-associated thrombotic microangiopathy (TA-TMA).
Zhang Z, Hong W, Wu Q, Tsavachidis S, Li JR, Amos CI, Cheng C, Sartain SE, Afshar-Kharghan V, Dong JF, Bhatraju P, Martin PJ, Makar RS, Bendapudi PK, Li A
Thromb Res 2023 May;225:39-46. Epub 2023 Mar 15 doi: 10.1016/j.thromres.2023.03.001. PMID: 36948020Free PMC Article
Transplant-Associated Thrombotic Microangiopathy in the Context of Allogenic Hematopoietic Stem Cell Transplantation: Where We Stand.
Lazana I
Int J Mol Sci 2023 Jan 6;24(2) doi: 10.3390/ijms24021159. PMID: 36674666Free PMC Article
Complement blockade for TA-TMA: lessons learned from a large pediatric cohort treated with eculizumab.
Jodele S, Dandoy CE, Lane A, Laskin BL, Teusink-Cross A, Myers KC, Wallace G, Nelson A, Bleesing J, Chima RS, Hirsch R, Ryan TD, Benoit S, Mizuno K, Warren M, Davies SM
Blood 2020 Mar 26;135(13):1049-1057. doi: 10.1182/blood.2019004218. PMID: 31932840Free PMC Article
Transplant-associated thrombotic microangiopathy: opening Pandora's box.
Gavriilaki E, Sakellari I, Anagnostopoulos A, Brodsky RA
Bone Marrow Transplant 2017 Oct;52(10):1355-1360. Epub 2017 Mar 13 doi: 10.1038/bmt.2017.39. PMID: 28287636
Eculizumab in Transplant-Associated Thrombotic Microangiopathy.
Dhakal P, Giri S, Pathak R, Bhatt VR
Clin Appl Thromb Hemost 2017 Mar;23(2):175-180. Epub 2016 Jul 10 doi: 10.1177/1076029615599439. PMID: 26259912

Clinical prediction guides

Pediatric transplant-associated thrombotic microangiopathy health care utilization and implications of eculizumab therapy.
Schoettler ML, Lehmann L, Kao PC, Chen N, Jodele S, Chonat S, Williams KM, London WB, Duncan C, Dandoy C
Blood Adv 2024 Mar 12;8(5):1220-1233. doi: 10.1182/bloodadvances.2023011078. PMID: 38154068Free PMC Article
Pathway-driven rare germline variants associated with transplant-associated thrombotic microangiopathy (TA-TMA).
Zhang Z, Hong W, Wu Q, Tsavachidis S, Li JR, Amos CI, Cheng C, Sartain SE, Afshar-Kharghan V, Dong JF, Bhatraju P, Martin PJ, Makar RS, Bendapudi PK, Li A
Thromb Res 2023 May;225:39-46. Epub 2023 Mar 15 doi: 10.1016/j.thromres.2023.03.001. PMID: 36948020Free PMC Article
Transplant-Associated Thrombotic Microangiopathy in the Context of Allogenic Hematopoietic Stem Cell Transplantation: Where We Stand.
Lazana I
Int J Mol Sci 2023 Jan 6;24(2) doi: 10.3390/ijms24021159. PMID: 36674666Free PMC Article
Cerebral vascular injury in transplant-associated thrombotic microangiopathy.
Sabulski A, Arcuri G, Szabo S, Care MM, Dandoy CE, Davies SM, Jodele S
Blood Adv 2022 Jul 26;6(14):4310-4319. doi: 10.1182/bloodadvances.2022007453. PMID: 35877136Free PMC Article
Interferon-complement loop in transplant-associated thrombotic microangiopathy.
Jodele S, Medvedovic M, Luebbering N, Chen J, Dandoy CE, Laskin BL, Davies SM
Blood Adv 2020 Mar 24;4(6):1166-1177. doi: 10.1182/bloodadvances.2020001515. PMID: 32208488Free PMC Article

Recent systematic reviews

Risk factors for transplant-associated thrombotic microangiopathy (TA-TMA): a systematic review and meta-analysis.
Guo M, Qi J, Hou Q, Li X, Han Y
Expert Rev Hematol 2023 Mar;16(3):191-203. Epub 2023 Jan 2 doi: 10.1080/17474086.2023.2162501. PMID: 36588482
Efficacy and Safety of Recombinant Thrombomodulin for the Prophylaxis of Veno-Occlusive Complication in Allogeneiccit Hematopoietic Stem Cell Transplantation: A Systematic Review and Meta-Analysis.
Kashyap R, Anwer F, Iqbal MA, Khalid F, Khan A, Ali MA, Anwar MY, Chaudhary A, Jaan A
Hematol Oncol Stem Cell Ther 2023 Jan 17;16(2):93-101. doi: 10.1016/j.hemonc.2021.09.002. PMID: 34655527
A systematic review of diagnostic, prognostic, and risk blood and urine biomarkers of transplant-associated thrombotic microangiopathy.
Schoettler ML, Bhatt H, Vasu S
Front Immunol 2022;13:1064203. Epub 2023 Feb 2 doi: 10.3389/fimmu.2022.1064203. PMID: 36818475Free PMC Article
Efficacy and Safety of Eculizumab in the Treatment of Transplant-Associated Thrombotic Microangiopathy: A Systematic Review and Meta-Analysis.
Zhang R, Zhou M, Qi J, Miao W, Zhang Z, Wu D, Han Y
Front Immunol 2020;11:564647. Epub 2021 Jan 20 doi: 10.3389/fimmu.2020.564647. PMID: 33552043Free PMC Article
A Complete Histologic Approach to Gastrointestinal Biopsy From Hematopoietic Stem Cell Transplant Patients With Evidence of Transplant-Associated Gastrointestinal Thrombotic Microangiopathy.
Warren M, Jodele S, Dandoy C, Myers KC, Wallace G, Nelson A, El-Bietar J
Arch Pathol Lab Med 2017 Nov;141(11):1558-1566. Epub 2017 Aug 10 doi: 10.5858/arpa.2016-0599-RA. PMID: 28795840

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