Successful use of defibrotide to treat allogeneic hematopoietic stem cell transplantation associated thrombotic microangiopathy in pediatric patients: report from Chinese single center – Bone Marrow Transplantation

García-Martín P, Alarcón-Payer C, López-Fernández E, Moratalla L, Romero A, Sainz J, et al. Transplantation-Associated Thrombotic Microangiopathy in Patients Treated With Sirolimus and Cyclosporine as Salvage Therapy for Graft-Versus-Host Disease. Ann Pharmacother. 2015;49:986–94.

Article 
PubMed 

Google Scholar 

Pagliuca S, Michonneau D, Sicre de Fontbrune F, Sutra Del Galy A, Xhaard A, Robin M, et al. Allogeneic reactivity-mediated endothelial cell complications after HSCT: a plea for consensual definitions. Blood Adv. 2019;3:2424–35.

Article 
PubMed 
PubMed Central 

Google Scholar 

Jodele S, Laskin BL, Dandoy CE, Myers KC, El-Bietar J, Davies SM, et al. A new paradigm: Diagnosis and management of HSCT-associated thrombotic microangiopathy as multi-system endothelial injury. Blood Rev. 2015;29:191–204.

Article 
PubMed 

Google Scholar 

Elfeky R, Lucchini G, Lum SH, Ottaviano G, Builes N, Nademi Z, et al. New insights into risk factors for transplant-associated thrombotic microangiopathy in pediatric HSCT. Blood Adv. 2020;4:2418–29.

Article 
PubMed 
PubMed Central 

Google Scholar 

Epperla N, Li A, Logan B, Fretham C, Chhabra S, Aljurf M, et al. Incidence, Risk Factors for and Outcomes of Transplant-Associated Thrombotic Microangiopathy. Br J Haematol. 2020;189:1171–81.

Article 
PubMed 
PubMed Central 

Google Scholar 

Jodele S, Davies SM, Lane A, Khoury J, Dandoy C, Goebel J, et al. Diagnostic and risk criteria for HSCT-associated thrombotic microangiopathy: a study in children and young adults. Blood. 2014;124:645–53.

Article 
PubMed 
PubMed Central 

Google Scholar 

Oran B, Donato M, Aleman A, Hosing C, Korbling M, Detry MA, et al. Transplant-associated microangiopathy in patients receiving tacrolimus following allogeneic stem cell transplantation: risk factors and response to treatment. Biol Blood Marrow Transpl. 2007;13:469–77.

Article 

Google Scholar 

Cho BS, Min CK, Eom KS, Kim YJ, Kim HJ, Lee S, et al. Clinical impact of thrombotic microangiopathy on the outcome of patients with acute graft-versus-host disease after allogeneic hematopoietic stem cell transplantation. Bone Marrow Transpl. 2008;41:813–20.

Article 

Google Scholar 

Hematopoietic Stem Cell Application Group of the Hematology Branch of the Chinese Medical Association. Chinese Expert Consensus on the Diagnosis and Treatment of Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy (2021 Edition). Chin J Hematol. 2021;42:177–84.

Google Scholar 

Kennedy GA, Kearey N, Bleakley S, Butler J, Mudie K, Durrant S. Transplantation-associated thrombotic microangiopathy: effect of concomitant GVHD on efficacy of therapeutic plasma exchange. Bone Marrow Transpl. 2010;45:699–704.

Article 

Google Scholar 

Devadas SK, Toshniwal M, Bagal B, Khattry N. Successful Treatment of Transplant Associated Thrombotic Microangiopathy (TA-TMA) with Low Dose Defibrotide. Indian J Hematol Blood Transfus. 2018;34:469–73.

Article 
PubMed 

Google Scholar 

Higham CS, Shimano KA, Melton A, Kharbanda S, Chu J, Dara J, et al. A pilot trial of prophylactic defibrotide to prevent serious thrombotic microangiopathy in high-risk pediatric patients. Pediatr Blood Cancer. 2022;69:e29641.

Article 
PubMed 

Google Scholar