Prophylactic recombinant thrombomodulin treatment prevents hepatic sinusoidal obstruction syndrome in high‐risk pediatric patients that undergo hematopoietic stem cell transplants

Abstract

Hepatic SOS is a potentially life-threatening complication of conditioning for allogeneic HSCT. rTM is a new drug for treating DIC. We report our experience of the use of rTM as a prophylaxis against SOS in high-risk pediatric patients that underwent HSCT. We evaluated the cases of 19 pediatric hematology and oncology patients who underwent HSCT at our institution between 2007 and 2016. The patients who received HSCT after 2012 (n = 8) were treated with rTM as a prophylaxis against SOS together with UDCA and LMWH, whereas the others (n = 11) were only treated with UDCA and LMWH. Although SOS occurred by post-HSCT day 35 in 3 (27%) patients in the control group, SOS was not seen in the rTM group. Two of the former three patients suffered severe SOS, and one died of the condition. The mean peak level of PAI-1 (a marker of endothelial damage) was significantly lower in the rTM group. rTM appears to be a safe prophylaxis for SOS. The present findings suggest that prophylactic rTM after HSCT might help to prevent SOS.

Endothelial Dysfunction after Hematopoietic Stem Cell Transplantation: A Review Based on Physiopathology

“…After allogeneic hematopoietic stem cell transplantation, a study reported that TM administration significantly reduced a-GVHD and ameliorated OS [111]. This result has been confirmed in a subsequent study [209]. TM has been found effective, after allogeneic HSCT, in cases of TA-TMA, SOS/VOD, and ES [210][211][212][213][214][215][216].…”

Sinusoidal Obstruction Syndrome/Hepatic Veno-Occlusive Disease

“…Other agents such as low molecular weight heparin, antithrombin III, prostaglandin E1, and pentoxifylline have proven to be ineffective or the study results inconclusive [25]. Recently, in a retrospective study of post-HCT pediatric patients with at least one risk factor for SOS/VOD from 2007 to 2016 at Showa University Fujigaoka Hospital (n = 19), no cases of SOS/VOD developed in 8 patients who received recombinant thrombomodulin with UDCA and lowmolecular-weight heparin (LMWH) as prophylaxis for SOS/VOD, while 3 cases developed in the control group of 11 patients who received only UDCA and LMWH [72].…”