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A study protocol for a randomised controlled trial evaluating clinical effects of platelet transfusion products: the Pathogen Reduction Evaluation and Predictive Analytical Rating Score (PREPAReS) trial

INTRODUCTION: Patients with chemotherapy-induced thrombocytopaenia frequently experience minor and sometimes severe bleeding complications. Unrestrictive availability of safe and effective blood products is presumed by treating physicians as well as patients. Pathogen reduction technology potentiall...

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Autores principales: Ypma, Paula F, van der Meer, Pieter F, Heddle, Nancy M, van Hilten, Joost A, Stijnen, Theo, Middelburg, Rutger A, Hervig, Tor, van der Bom, Johanna G, Brand, Anneke, Kerkhoffs, Jean-Louis H
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4735127/
https://www.ncbi.nlm.nih.gov/pubmed/26817642
http://dx.doi.org/10.1136/bmjopen-2015-010156
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author Ypma, Paula F
van der Meer, Pieter F
Heddle, Nancy M
van Hilten, Joost A
Stijnen, Theo
Middelburg, Rutger A
Hervig, Tor
van der Bom, Johanna G
Brand, Anneke
Kerkhoffs, Jean-Louis H
author_facet Ypma, Paula F
van der Meer, Pieter F
Heddle, Nancy M
van Hilten, Joost A
Stijnen, Theo
Middelburg, Rutger A
Hervig, Tor
van der Bom, Johanna G
Brand, Anneke
Kerkhoffs, Jean-Louis H
author_sort Ypma, Paula F
collection PubMed
description INTRODUCTION: Patients with chemotherapy-induced thrombocytopaenia frequently experience minor and sometimes severe bleeding complications. Unrestrictive availability of safe and effective blood products is presumed by treating physicians as well as patients. Pathogen reduction technology potentially offers the opportunity to enhance safety by reducing bacterial and viral contamination of platelet products along with a potential reduction of alloimmunisation in patients receiving multiple platelet transfusions. METHODS AND ANALYSIS: To test efficacy, a randomised, single-blinded, multicentre controlled trial was designed to evaluate clinical non-inferiority of pathogen-reduced platelet concentrates treated by the Mirasol system, compared with standard plasma-stored platelet concentrates using the percentage of patients with WHO grade ≥2 bleeding complications as the primary endpoint. The upper limit of the 95% CI of the non-inferiority margin was chosen to be a ≤12.5% increase in this percentage. Bleeding symptoms are actively monitored on a daily basis. The adjudication of the bleeding grade is performed by 3 adjudicators, blinded to the platelet product randomisation as well as by an automated computer algorithm. Interim analyses evaluating bleeding complications as well as serious adverse events are performed after each batch of 60 patients. The study started in 2010 and patients will be enrolled up to a maximum of 618 patients, depending on the results of consecutive interim analyses. A flexible stopping rule was designed allowing stopping for non-inferiority or futility. Besides analysing effects of pathogen reduction on clinical efficacy, the Pathogen Reduction Evaluation and Predictive Analytical Rating Score (PREPAReS) is designed to answer several other pending questions and translational issues related to bleeding and alloimmunisation, formulated as secondary and tertiary endpoints. ETHICS AND DISSEMINATION: Ethics approval was obtained in all 3 participating countries. Results of the main trial and each of the secondary endpoints will be submitted for publication in a peer-reviewed journal. TRIAL REGISTRATION NUMBER: NTR2106; Pre-results.
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spelling pubmed-47351272016-02-09 A study protocol for a randomised controlled trial evaluating clinical effects of platelet transfusion products: the Pathogen Reduction Evaluation and Predictive Analytical Rating Score (PREPAReS) trial Ypma, Paula F van der Meer, Pieter F Heddle, Nancy M van Hilten, Joost A Stijnen, Theo Middelburg, Rutger A Hervig, Tor van der Bom, Johanna G Brand, Anneke Kerkhoffs, Jean-Louis H BMJ Open Haematology (Incl Blood Transfusion) INTRODUCTION: Patients with chemotherapy-induced thrombocytopaenia frequently experience minor and sometimes severe bleeding complications. Unrestrictive availability of safe and effective blood products is presumed by treating physicians as well as patients. Pathogen reduction technology potentially offers the opportunity to enhance safety by reducing bacterial and viral contamination of platelet products along with a potential reduction of alloimmunisation in patients receiving multiple platelet transfusions. METHODS AND ANALYSIS: To test efficacy, a randomised, single-blinded, multicentre controlled trial was designed to evaluate clinical non-inferiority of pathogen-reduced platelet concentrates treated by the Mirasol system, compared with standard plasma-stored platelet concentrates using the percentage of patients with WHO grade ≥2 bleeding complications as the primary endpoint. The upper limit of the 95% CI of the non-inferiority margin was chosen to be a ≤12.5% increase in this percentage. Bleeding symptoms are actively monitored on a daily basis. The adjudication of the bleeding grade is performed by 3 adjudicators, blinded to the platelet product randomisation as well as by an automated computer algorithm. Interim analyses evaluating bleeding complications as well as serious adverse events are performed after each batch of 60 patients. The study started in 2010 and patients will be enrolled up to a maximum of 618 patients, depending on the results of consecutive interim analyses. A flexible stopping rule was designed allowing stopping for non-inferiority or futility. Besides analysing effects of pathogen reduction on clinical efficacy, the Pathogen Reduction Evaluation and Predictive Analytical Rating Score (PREPAReS) is designed to answer several other pending questions and translational issues related to bleeding and alloimmunisation, formulated as secondary and tertiary endpoints. ETHICS AND DISSEMINATION: Ethics approval was obtained in all 3 participating countries. Results of the main trial and each of the secondary endpoints will be submitted for publication in a peer-reviewed journal. TRIAL REGISTRATION NUMBER: NTR2106; Pre-results. BMJ Publishing Group 2016-01-27 /pmc/articles/PMC4735127/ /pubmed/26817642 http://dx.doi.org/10.1136/bmjopen-2015-010156 Text en Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://www.bmj.com/company/products-services/rights-and-licensing/ This is an Open Access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
spellingShingle Haematology (Incl Blood Transfusion)
Ypma, Paula F
van der Meer, Pieter F
Heddle, Nancy M
van Hilten, Joost A
Stijnen, Theo
Middelburg, Rutger A
Hervig, Tor
van der Bom, Johanna G
Brand, Anneke
Kerkhoffs, Jean-Louis H
A study protocol for a randomised controlled trial evaluating clinical effects of platelet transfusion products: the Pathogen Reduction Evaluation and Predictive Analytical Rating Score (PREPAReS) trial
title A study protocol for a randomised controlled trial evaluating clinical effects of platelet transfusion products: the Pathogen Reduction Evaluation and Predictive Analytical Rating Score (PREPAReS) trial
title_full A study protocol for a randomised controlled trial evaluating clinical effects of platelet transfusion products: the Pathogen Reduction Evaluation and Predictive Analytical Rating Score (PREPAReS) trial
title_fullStr A study protocol for a randomised controlled trial evaluating clinical effects of platelet transfusion products: the Pathogen Reduction Evaluation and Predictive Analytical Rating Score (PREPAReS) trial
title_full_unstemmed A study protocol for a randomised controlled trial evaluating clinical effects of platelet transfusion products: the Pathogen Reduction Evaluation and Predictive Analytical Rating Score (PREPAReS) trial
title_short A study protocol for a randomised controlled trial evaluating clinical effects of platelet transfusion products: the Pathogen Reduction Evaluation and Predictive Analytical Rating Score (PREPAReS) trial
title_sort study protocol for a randomised controlled trial evaluating clinical effects of platelet transfusion products: the pathogen reduction evaluation and predictive analytical rating score (prepares) trial
topic Haematology (Incl Blood Transfusion)
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4735127/
https://www.ncbi.nlm.nih.gov/pubmed/26817642
http://dx.doi.org/10.1136/bmjopen-2015-010156
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