Table 3 Select studies on intended or incidental prophylaxis with endothelium-protective drugs in allo-SCT.
Drug | Study type and objective (application) | Number of patients | Target population | Outcomes | References |
Miscellaneous statins | Retrospective; post-transplant hyperlipidaemia(incidental) | No statins: 541, statins: 220 | recipients RD, UD | Grade II-IV aGVHD significantly increased in patients with hiperlipidaemia; statins reduced hyperlidpidaemia without significant side effects | Blaser et al. 2012 [81] |
Miscellaneous statins | Retrospective; incidence and severity of aGVHD (incidental) | No statins: 57, statins: 10 | Recipients (AML, ALL) | Trend to less aGVHD (II-IV) in the statin group (p = 0.08), no effect on cGVHD, no effect on GVL | Hamadani et al. 2008 [83] |
Atorvastatin | Prospective single arm; safety, grade II-IV aGVHD (intended) | Statins: 69; 30 (MRD) 39 (MUD) | Recipients | No negative safety signals; preliminary positive efficacy signals. | Kanate et al. 2017 [85] |
Miscellaneous statins | Retrospective; GVHD risk(incidental) | No statins: 464, statins: 75 | Donors and/or recipients, RD | Grade III-IV aGVHD significantly reduced with donor statin treatment; trend for less NRM with recipient statin treatment; effects seen only with CSA | Rotta et al. 2010 [87] |
Miscellaneous statins | Retrospective; GVHD risk, NRM, Relapse, mortality(incidental) | No statins: 1130; statins: 76 | Recipients, RD and UD | Chronic GVHD significantly reduced but relapse risk increased with statins;effects seen only with CSA;no statin effect on any other endpoint | Rotta et al. 2010 [88] |
Atorvastatin | Prospective single arm; safety, grade II-IV aGVHD (prophylactic use) | Statins: 30 | Donors and recipients, RD | No negative safety signals; preliminary positive efficacy signals. | Hamadani et al. 2013 [84] |
UDA | Prospective randomized; chronic GVHD and survival outcomes (intended) | No UDA: 119 UDA: 123 | Recipients, RD and UD | Grade III-IV aGVHD significantly reduced and NRM and OS significantly improved with UDA; no significant effects on chronic GVHD and relapse risk | Ruutu et al. 2014 [89] |
Pravastatin ± UDA | Retrospective cohort comparison; TA-TMA, refractory aGVHD (intended) | No statins/UDA: 356 statins/UDA: 415 | Recipients, RD and UD | TA-TMA, refractory aGVHD significantly reduced with statins/UDA | Zeisbrich et al. 2017 [27] |
Pravastatin  ± UDA | Retrospective cohort comparison; SOS/VOD (intended) | No statins/UDA: 826, statins/UDA: 359 | Recipients, RD and UD | SOS/VOD significantly reduced with statins/UDA; effect most pronounced in the highest EASIX quartile | Jiang et al.2020 [69] |
Pravastatin ± UDA | Retrospective cohort comparison; survival outcomes (intended) | No statins/UDA: 576 statins/UDA: 344 | Recipients, RD and UD | NRM reduced with statins/UDA | Rachakonda et al. 2018 [4] |
Defibrotide ± UDA | Prospective randomized; SOS/VOD (intended) | Defibrotide: 180, No defibrotide: 176 | Recipients, autologous and allogeneic, pediatric only, high risk | SOS/VOD reduced with defibrotide; grade I-IV aGVHD significantly reduced with defibrotide; no negative safety signals including bleeding events; no effect on TA-TMA, NRM, and overall mortality | Corbacioglu et al. 2012 [82] |
Defibrotide + UDA | Retrospective; SOS/VOD (intended) | Defibrotide: 63 | Recipients(adult, high risk) | No negative safety signals except for bleeding events in 22%; preliminary positive efficacy signals | Picod et al. 2018 [86] |
