Ruxolitinib salvage therapy is effective for steroid‐refractory graft‐versus‐host disease in children: A single‐center experience

Background Despite the increasing performance of allogeneic hematopoietic cell transplantation over the last decades, graft‐versus‐host disease (GVHD) remains the main cause of morbidity and mortality. The efficacy of ruxolitinib against GVHD has been demonstrated in adult studies; however, very few...

Full description

Saved in:
Bibliographic Details
Published in:Pediatric blood & cancer 2020-04, Vol.67 (4), p.e28190-n/a
Main Authors: Uygun, Vedat, Karasu, Gülsün, Daloğlu, Hayriye, Öztürkmen, Seda, Kılıç, Suar Çakı, Yalçın, Koray, Çelen, Safiye Suna, Hazar, Volkan, Yeşilipek, Akif
Format: Article
Language:English
Subjects:
Bronchiolitis obliterans
Bronchopneumonia
Children
Graft versus host disease
Graft-versus-host reaction
GVHD
Hematology
Methylprednisolone
Morbidity
Oncology
Patients
Pediatrics
ruxolitinib
Steroid hormones
Targeted cancer therapy
Transplantation
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:Background Despite the increasing performance of allogeneic hematopoietic cell transplantation over the last decades, graft‐versus‐host disease (GVHD) remains the main cause of morbidity and mortality. The efficacy of ruxolitinib against GVHD has been demonstrated in adult studies; however, very few studies have been conducted in children. Procedure This study aimed to evaluate the efficacy of ruxolitinib in 29 children with steroid‐refractory acute or chronic GVHD. Twenty‐five (87%) patients received at least three different immune modulator agents, including methylprednisolone, before initiating ruxolitinib. Results All grade 2 acute GVHD patients completely responded to ruxolitinib treatment; 82% of high‐grade (3‐4) acute GVHD patients and 80% of chronic GVHD (moderate‐severe) patients had at least a partial response. Of seven patients with bronchiolitis obliterans, five had a partial response after ruxolitinib. Of 29 patients, 22 were administered steroids at any time in the first month of acute GVHD or the first three months of chronic GVHD during ruxolitinib usage, which was significantly tapered by the end of the observation period. Conclusion Steroid‐refractory acute and chronic pediatric GVHD patients treated with ruxolitinib had a high overall response rate, with the additional benefit of steroid sparing.
ISSN:1545-5009
1545-5017
DOI:10.1002/pbc.28190