TY - JOUR
T1 - A pragmatic multi-institutional approach to understanding transplant-associated thrombotic microangiopathy after stem cell transplant
AU - Dandoy, Christopher E.
AU - Rotz, Seth
AU - Alonso, Priscila Badia
AU - Klunk, Anna
AU - Desmond, Catherine
AU - Huber, John
AU - Ingraham, Hannah
AU - Higham, Christine
AU - Dvorak, Christopher C.
AU - Duncan, Christine
AU - Schoettler, Michelle
AU - Lehmann, Leslie
AU - Cancio, Maria
AU - Killinger, James
AU - Davila, Blachy
AU - Phelan, Rachel
AU - Mahadeo, Kris M.
AU - Khazal, Sajad
AU - Lalefar, Nahal
AU - Vissa, Madhav
AU - Myers, Kasiani
AU - Wallace, Greg
AU - Nelson, Adam
AU - Khandelwal, Pooja
AU - Bhatla, Deepika
AU - Gloude, Nicholas
AU - Anderson, Eric
AU - Huo, Jeffrey
AU - Roehrs, Philip
AU - Auletta, Jeffery J.
AU - Chima, Ranjit
AU - Lane, Adam
AU - Davies, Stella M.
AU - Jodele, Sonata
N1 - Publisher Copyright:
© 2020 by The American Society of Hematology.
PY - 2021/1/12
Y1 - 2021/1/12
N2 - 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.
AB - 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.
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U2 - 10.1182/bloodadvances.2020003455
DO - 10.1182/bloodadvances.2020003455
M3 - Article
C2 - 33570619
AN - SCOPUS:85099262070
SN - 2473-9529
VL - 5
SP - 1
EP - 11
JO - Blood Advances
JF - Blood Advances
IS - 1
ER -