Clinical effects of recombinant thrombomodulin and defibrotide on sinusoidal obstruction syndrome after allogeneic hematopoietic stem cell transplantation.
Adolescent
Adult
Aged
Allografts
Child
Child, Preschool
Disease-Free Survival
Female
Hematopoietic Stem Cell Transplantation
Hepatic Veno-Occlusive Disease
/ drug therapy
Humans
Infant
Infant, Newborn
Japan
/ epidemiology
Male
Middle Aged
Polydeoxyribonucleotides
/ administration & dosage
Recombinant Proteins
/ administration & dosage
Survival Rate
Thrombomodulin
/ administration & dosage
Journal
Bone marrow transplantation
ISSN: 1476-5365
Titre abrégé: Bone Marrow Transplant
Pays: England
ID NLM: 8702459
Informations de publication
Date de publication:
05 2019
05 2019
Historique:
received:
19
05
2018
accepted:
30
07
2018
revised:
28
07
2018
pubmed:
22
8
2018
medline:
2
7
2020
entrez:
22
8
2018
Statut:
ppublish
Résumé
Sinusoidal obstruction syndrome (SOS) is a lethal complication after hematopoietic stem cell transplantation (HSCT). Defibrotide (DF) is the only drug internationally recommended for SOS treatment in Western countries. Recombinant human soluble thrombomodulin (rhTM), which is promising for the treatment of patients with disseminated intravascular coagulation, is also reported to be potentially effective for SOS. To clarify the safety and efficacy of DF and rhTM, we conducted a retrospective survey of these agents in Japan. Data from 65 patients who underwent allogeneic HSCT and received DF (n = 24) or rhTM (n = 41) for SOS treatment were collected. The complete response rates for SOS on day 100 were 50% and 54% in the DF and rhTM groups, respectively. The 100-day overall survival rates were 50% in the DF group, and 48% in the rhTM group. Several severe hemorrhagic adverse events were observed in one patient in the DF group and five patients in the rhTM group. The main causes of death were SOS-related death, and no patient died of direct adverse events of DF or rhTM. Our results suggest that rhTM, as well as DF, can be effective as a novel treatment option for SOS.
Identifiants
pubmed: 30127466
doi: 10.1038/s41409-018-0304-4
pii: 10.1038/s41409-018-0304-4
doi:
Substances chimiques
Polydeoxyribonucleotides
0
Recombinant Proteins
0
THBD protein, human
0
Thrombomodulin
0
defibrotide
438HCF2X0M
Types de publication
Clinical Trial
Journal Article
Multicenter Study
Langues
eng
Sous-ensembles de citation
IM
Pagination
674-680Références
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