Recombinant Human Soluble Thrombomodulin May Improve Outcomes in Transplant-Associated Thrombotic Microangiopathy

Intravenous administration of recombinant soluble thrombomodulin (rTM) may improve recovery and survival compared to other treatments in patients with transplant-associated thrombotic microangiopathy (TA-TMA) after hematopoietic stem cell transplantation (HSCT), according to a retrospective analysis of 254 consecutive patients at a single Japanese institution. Transplant-associated TA-TMA is a severe complication that is attributed to underlying endothelial cell damage. As rTM has cytoprotective effects against calcineurin inhibitor-induced endothelial cell damage, the investigators hypothesized that patients receiving rTM as a first-line treatment would potentially realize a benefit.

Of 16 post-HSCT patients diagnosed with TA-TMA, nine were treated with rTM and seven received other treatments. Seven of the nine patients (78 percent) in therTM group recovered from TA-TMA without addition of second-line therapy, while none recovered in the control group, who received various therapies including fresh frozen plasma (FFP) and therapeutic plasma exchange (p=0.003). In the entire cohort, three patients (33.3 percent) remained alive in the rTM group, while no patients survived in the control group after their TA-TMA diagnosis.

The investigators concluded that these data suggest that administration of rTM may improve clinical outcomes in patients with TA-TMA.