A pragmatic multi-institutional approach to understanding transplant-associated thrombotic microangiopathy after stem cell transplant Journal Article


Authors: Dandoy, C. E.; Rotz, S.; Alonso, P. B.; Klunk, A.; Desmond, C.; Huber, J.; Ingraham, H.; Higham, C.; Dvorak, C. C.; Duncan, C.; Schoettler, M.; Lehmann, L.; Cancio, M.; Killinger, J.; Davila, B.; Phelan, R.; Mahadeo, K. M.; Khazal, S.; Lalefar, N.; Vissa, M.; Myers, K.; Wallace, G.; Nelson, A.; Khandelwal, P.; Bhatla, D.; Gloude, N.; Anderson, E.; Huo, J.; Roehrs, P.; Auletta, J. J.; Chima, R.; Lane, A.; Davies, S. M.; Jodele, S.
Article Title: A pragmatic multi-institutional approach to understanding transplant-associated thrombotic microangiopathy after stem cell transplant
Abstract: Transplant-associated thrombotic microangiopathy (TA-TMA) is a severe complication of hematopoietic stem cell transplantation (HSCT). A single-center prospective screening study has shown that the incidence of TA-TMA is much higher than prior retrospective studies that did not systematically screen. These data have not been replicated in a multicenter study. Our objective was to determine the incidence and risk factors for TA-TMA and compare outcomes of pediatric HSCT patients with and without TA-TMA. Patients were prospectively screened for TA-TMA at participating centers using a simple to implement and inexpensive strategy from the start of the preparative regimen through day 1100. TA-TMA was diagnosed if $4 of 7 laboratory/clinical markers diagnostic for TA-TMA were present concurrently or if tissue histology showed TA-TMA. A total of 614 patients (359 males; 58%) received prospective TA-TMA screening at 13 pediatric centers. TA-TMA was diagnosed in 98 patients (16%) at a median of 22 days (interquartile range, 14-44) posttransplant. Patients with TA-TMA had significantly increased bloodstream infections (38% [37/98] vs 21% [107/51], P #.001), mean total hospitalization days (68; 95% confidence interval [CI], 63-74 vs 43; 95% CI, 41-45; P #.001), and number of days spent in the intensive care unit (10.1; 95% CI, 6.4-14; vs 1.6; 95% CI, 1.1-2.2; P #.001) in the first 100 days after HSCT compared with patients without TA-TMA. Overall survival was significantly higher in patients without TA-TMA (93%; 490/516) compared with patients with TA-TMA (78%; 76/98) (P #.001). These data support the need for systematic screening for TA-TMA and demonstrate the feasibility and efficacy of an easy to implement strategy to do so. © 2020 by The American Society of Hematology.
Journal Title: Blood Advances
Volume: 5
Issue: 1
ISSN: 2473-9529
Publisher: American Society of Hematology  
Date Published: 2021-01-12
Start Page: 1
End Page: 11
Language: English
DOI: 10.1182/bloodadvances.2020003455
PROVIDER: scopus
PMCID: PMC7805323
PUBMED: 33570619
DOI/URL:
Notes: Article -- Export Date: 1 February 2021 -- Source: Scopus
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  1. Maria   Cancio
    58 Cancio