scholarly journals Use of Extracorporeal Photopheresis for the Treatment of Steroid Refractory Acute Graft-Versus-Host Disease in Pediatric Patients: Experience at a Private Institution in Brazil

2020 ◽  
Vol 26 (3) ◽  
pp. S300-S301
Author(s):  
Raquel Neves ◽  
Alessandra Gomes ◽  
Ana Beatriz Mafra ◽  
Giancarlo Fatobene ◽  
Vanderson Rocha
2018 ◽  
Vol 24 (3) ◽  
pp. S194 ◽  
Author(s):  
Marietta Nygaard ◽  
Tonny Karlsmark ◽  
Niels Smedegaard ◽  
Ida Schjødt ◽  
Lone S. Friis ◽  
...  

2013 ◽  
Vol 18 (1) ◽  
pp. 94-102 ◽  
Author(s):  
Pooja Khandelwal ◽  
Julia Lawrence ◽  
Alexandra H. Filipovich ◽  
Stella M. Davies ◽  
Jacob J. Bleesing ◽  
...  

2021 ◽  
Vol 8 (1) ◽  
pp. 10-17
Author(s):  
Gabriel Tremblay ◽  
Dimitrios Tomaras ◽  
Eric Strati ◽  
Anna Forsythe

Background: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) can be a lifesaving treatment for hematologic malignancies, but acute graft-versus-host-disease (aGVHD) is a potentially deadly adverse effect experienced by up to half of allo-HSCT recipients. Inadequate response to steroid therapy for aGVHD is associated with poor prognosis and high mortality, including among pediatric patients, who are the focus of this study. Ruxolitinib and remestemcel-L-rknd were evaluated for the treatment of steroid-refractory (SR) aGVHD in two separate single-arm trials. To effectively compare the safety and efficacy of these treatments without a head-to-head trial, a simulated treatment comparison (STC) was conducted. Methods: Regression techniques were used to adjust individual patient-level data from the remestemcel-L-rknd trial to mutually reported baseline characteristics from the ruxolitinib trial. Outcomes of interest included a 28-day overall response rate (ORR), a 28-day ORR in the grade III-IV aGVHD population, and adverse events (AEs). Results: In the full populations, the STC of risk ratios (RRs) found treatment with remestemcel-L-rknd to be associated with a numerical but not statistically significant improvement in the 28-day ORR versus ruxolitinib. In the grade III-IV aGVHD sub-group, the STC showed significantly improved 28-day ORR for remestemcel-L-rknd versus ruxolitinib (P=0.04). Remestemcel-L-rknd was also associated with improved safety outcomes (P<0.05) in 17 out of 30 AEs, including hematologic events, peripheral edema, muscular weakness, nausea, back pain, and fatigue. Conclusion: Remestemcel-L-rknd was associated with significant improvements in day 28 ORR compared with ruxolitinib in patients with severe (grade III-IV) SR aGVHD. Across all grades of SR aGVHD, remestemcel-L-rknd was associated with fewer all-grade treatment-emergent adverse events (TEAEs) (27/30) available for comparison, including the majority reaching statistical significance.


2019 ◽  
Vol 25 (4) ◽  
pp. 743-748 ◽  
Author(s):  
Maura Faraci ◽  
Maria Grazia Calevo ◽  
Stefano Giardino ◽  
Massimiliano Leoni ◽  
Erica Ricci ◽  
...  

2016 ◽  
Vol 7 (1) ◽  
pp. 58-64
Author(s):  
Andrey V Kozlov ◽  
Julia G Fedukova ◽  
Tatzana A Bykova ◽  
Ivan S Moiseev ◽  
Marya A Estrina ◽  
...  

Allogeneic hematopoietic stem cell transplantation (allo-SCT) is used widely in the management of children with hematological, oncological and inherited diseases. Study to compare efficiency of steroid-refractory acute graft versus host disease treatment (extracorporeal photopheresis (ECP) vs anticytokine therapy) was conducted in Raisa Gorbacheva Memorial Institute of Children Oncology, Hematology and Transplantation, First Pavlov State Medical University of Saint Petersburg. Sixty four children were included in the analysis. Patients were divided into two groups: first “group with ECP” (n = 31; 50,5 %) ("ECP group") and second - "group without ECP" (n = 33; 49,5 %). Treatment in the second group consisted of anticytokine therapy (etanercept (n = 12), infliximab (n = 9), daclizumab (n = 8)) and alemtuzumab (n = 4). Ten-year overall survival (OS) of children with steroid-refractory acute graft versus host disease was 39 % without difference in OS between ECP and anticytokine therapy (5-year OS 40 % vs 35 %, p = 0,34). Response rate to the therapy was also the same in both groups (68 % after ECP and 70 % after anticytokine therapy, p = 0,77). Difference in cumulative incidence of relapse in "ECP group" and "group without ECP" was not statistically significant (18 % and 7 %, respectively, p = 0,2). In conclusion, ECP and anticytokine therapy are equally effective in the treatment of children with steroid-refractory acute graft versus host disease and are associated with the same cumulative incidence of relapse.


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