1. A pragmatic multi-institutional approach to understanding transplant-associated thrombotic microangiopathy after stem cell transplant
- Author
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Dandoy, Christopher E, Rotz, Seth, Alonso, Priscila Badia, Klunk, Anna, Desmond, Catherine, Huber, John, Ingraham, Hannah, Higham, Christine, Dvorak, Christopher C, Duncan, Christine, Schoettler, Michelle, Lehmann, Leslie, Cancio, Maria, Killinger, James, Davila, Blachy, Phelan, Rachel, Mahadeo, Kris M, Khazal, Sajad, Lalefar, Nahal, Vissa, Madhav, Myers, Kasiani, Wallace, Greg, Nelson, Adam, Khandelwal, Pooja, Bhatla, Deepika, Gloude, Nicholas, Anderson, Eric, Huo, Jeffrey, Roehrs, Philip, Auletta, Jeffery J, Chima, Ranjit, Lane, Adam, Davies, Stella M, and Jodele, Sonata
- Subjects
Prevention ,Clinical Research ,Pediatric ,Regenerative Medicine ,Clinical Trials and Supportive Activities ,Transplantation ,Cancer ,Child ,Hematopoietic Stem Cell Transplantation ,Humans ,Incidence ,Male ,Prospective Studies ,Retrospective Studies ,Thrombotic Microangiopathies - 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 +100. 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.
- Published
- 2021