Thrombomodulin alfa treatment in patients with acute promyelocytic leukemia and disseminated intravascular coagulation: a retrospective analysis of an open-label, multicenter, post-marketing surveillance study cohort

Thromb Res. 2014 May;133(5):772-81. doi: 10.1016/j.thromres.2014.02.025. Epub 2014 Mar 4.

Abstract

Introduction: Patients with acute promyelocytic leukemia (APL) can develop disseminated intravascular coagulation (DIC) that results in life-threatening hemorrhagic complications. Studies regarding the safety and efficacy of thrombomodulin alfa (TM-α; recombinant human soluble thrombomodulin) in patients with APL and DIC are limited.

Materials and methods: A retrospective evaluation was performed on a cohort of 172 patients with APL from an open-label, multicenter, post-marketing surveillance study of TM-α.

Results: Of the 172 patients, 31 were relapse/refractory APL patients, and 141 were newly diagnosed APL patients. Within the first 30 days, 24 patients (14.0%) died, and six of those deaths (3.5%) were due to hemorrhage. In total, 12 patients (7.0%) had severe hemorrhagic complications. Both the early death rate due to hemorrhage as well as the severe hemorrhage rate did not exceed those in some recent population-based studies of patients with APL. Forty-nine patients received TM-α prior to the initiation of antileukemic treatment, and one patient experienced hemorrhagic early death (ED), suggesting that early TM-α treatment appeared to result in a reduction in the hemorrhagic ED rate. Moreover, TM-α improved coagulopathy regardless of concomitant all-trans retinoic acid treatment.

Conclusions: This study confirmed the safety and efficacy of TM-α in daily clinical practice for patients with APL and DIC. TM-α appeared to reduce hemorrhagic early deaths due to DIC in patients with APL who were receiving antileukemic treatment.

Keywords: Acute promyelocytic leukemia; Disseminated intravascular coagulation; Recombinant human soluble thrombomodulin.

Publication types

  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Aged, 80 and over
  • Child
  • Cohort Studies
  • Disseminated Intravascular Coagulation / drug therapy*
  • Female
  • Humans
  • Leukemia, Promyelocytic, Acute / blood
  • Leukemia, Promyelocytic, Acute / drug therapy*
  • Male
  • Middle Aged
  • Product Surveillance, Postmarketing
  • Recombinant Proteins / adverse effects
  • Recombinant Proteins / therapeutic use
  • Retrospective Studies
  • Thrombomodulin / therapeutic use*
  • Young Adult

Substances

  • Recombinant Proteins
  • THBD protein, human
  • Thrombomodulin