Tadashi Matsushita1, Jyunichi Watanabe2, Goichi Honda2, Jun Mimuro3, Hoyu Takahashi4, Hajime Tsuji5, Yutaka Eguchi6, Isao Kitajima7, Yoichi Sakata3. 1. Department of Transfusion Medicine, Nagoya University Hospital, Aichi, Japan; The Japanese Society on Thrombosis and Hemostasis Post-Marketing Surveillance Committee for Recomodulin(®) Injection, Japan. Electronic address: tmatsu@med.nagoya-u.ac.jp. 2. ART Project, Asahi Kasei Pharma Corporation, Tokyo, Japan. 3. Division of Cell and Molecular Medicine, Center for Molecular Medicine, Jichi Medical University, School of Medicine, Tochigi, Japan; The Japanese Society on Thrombosis and Hemostasis Post-Marketing Surveillance Committee for Recomodulin(®) Injection, Japan. 4. Department of Internal Medicine, Niigata Prefectural Kamo Hospital, Niigata, Japan; The Japanese Society on Thrombosis and Hemostasis Post-Marketing Surveillance Committee for Recomodulin(®) Injection, Japan. 5. Department of Blood Transfusion, Kyoto Prefectural University of Medicine, Kyoto, Japan; The Japanese Society on Thrombosis and Hemostasis Post-Marketing Surveillance Committee for Recomodulin(®) Injection, Japan. 6. Critical and Intensive Care Medicine, Shiga University of Medical Science, Shiga, Japan; The Japanese Society on Thrombosis and Hemostasis Post-Marketing Surveillance Committee for Recomodulin(®) Injection, Japan. 7. Department of Clinical Laboratory and Molecular Pathology, Graduate School of Medical and Pharmaceutical Science, University of Toyama, Toyama, Japan; The Japanese Society on Thrombosis and Hemostasis Post-Marketing Surveillance Committee for Recomodulin(®) Injection, Japan.
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.
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 APLpatients, and 141 were newly diagnosed APLpatients. 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.
Authors: Miguel A Sanz; Pierre Fenaux; Martin S Tallman; Elihu H Estey; Bob Löwenberg; Tomoki Naoe; Eva Lengfelder; Hartmut Döhner; Alan K Burnett; Sai-Juan Chen; Vikram Mathews; Harry Iland; Eduardo Rego; Hagop Kantarjian; Lionel Adès; Giuseppe Avvisati; Pau Montesinos; Uwe Platzbecker; Farhad Ravandi; Nigel H Russell; Francesco Lo-Coco Journal: Blood Date: 2019-02-25 Impact factor: 22.113
Authors: Madhvi Rajpurkar; Todd A Alonzo; Yi-Cheng Wang; Robert B Gerbing; Alan S Gamis; James H Feusner; John Gregory; Matthew A Kutny Journal: J Pediatr Hematol Oncol Date: 2019-01 Impact factor: 1.289