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

Christopher E. Dandoy, Seth Rotz, Priscila Badia Alonso, Anna Klunk, Catherine Desmond, John Huber, Hannah Ingraham, Christine Higham, Christopher C. Dvorak, Christine Duncan, Michelle Schoettler, Leslie Lehmann, Maria Cancio, James Killinger, Blachy Davila, Rachel Phelan, Kris M. Mahadeo, Sajad Khazal, Nahal Lalefar, Madhav VissaKasiani Myers, Greg Wallace, Adam Nelson, Pooja Khandelwal, Deepika Bhatla, Nicholas Gloude, Eric Anderson, Jeffrey Huo, Philip Roehrs, Jeffery J. Auletta, Ranjit Chima, Adam Lane, Stella M. Davies, Sonata Jodele

Research output: Contribution to journalArticlepeer-review

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.

Original languageEnglish (US)
Pages (from-to)1-11
Number of pages11
JournalBlood Advances
Volume5
Issue number1
DOIs
StatePublished - Jan 12 2021
Externally publishedYes

ASJC Scopus subject areas

  • Hematology

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