title: Rationale and design of platelet transfusions in haematopoietic stem cell transplantation: the PATH pilot study.
abstract: In patients with transient thrombocytopenia being treated with high-dose chemotherapy followed by stem cell rescue-haematopoietic stem cell transplantation (HSCT), prophylactic transfusions are standard therapy to prevent bleeding. However, a recent multicentre trial suggests that prophylactic platelet transfusions in HSCT may not be necessary. Additionally, the potential overuse of platelet products places a burden on a scarce healthcare resource. Moreover, the benefit of prophylactic platelet transfusions to prevent clinically relevant haemorrhage is debatable. Current randomised data compare different thresholds for administering prophylactic platelets or prophylactic versus therapeutic platelet transfusions. An alternative strategy involves prescribing prophylactic antifibrinolytic agents such as tranexamic acid to prevent bleeding.
  
  
  
  This report describes the design of an open-labelled randomised pilot study comparing the prophylactic use of oral tranexamic acid with platelet transfusions in the setting of autologous HSCT. In 3-5 centres, 100 patients undergoing autologous HSCT will be randomly assigned to either a prophylactic tranexamic acid or prophylactic platelets bleeding prevention strategy-based daily platelet values up to 30 days post-transplant. The study will be stratified by centre and type of transplant. The primary goal is to demonstrate study feasibility while collecting clinical outcomes on (1) WHO and Bleeding Severity Measurement Scale (BSMS), (2) transplant-related mortality, (3) quality of life, (4) length of hospital stay, (5) intensive care unit admission rates, (6) Bearman toxicity scores, (7) incidence of infections, (8) transfusion requirements, (9) adverse reactions and (10) economic analyses.
  
  
  
  This study is funded by a peer-reviewed grant from the Canadian Institutes of Health Research (201 503) and is registered on Clinicaltrials.gov NCT02650791. It has been approved by the Ottawa Health Science Network Research Ethics Board. Study results will presented at national and international conferences. Importantly, the results of this trial will inform the feasibility and conduct of a larger study.
  
  
  
  NCT02650791; Pre-results.
authors: Department of Medicine, University of Calgary, Calgary, Alberta, Canada.|Department of Medicine, University of Ottawa, Ottawa, Ontario, Canada.|Clinical Epidemiology Program, Ottawa Hospital Research Institute, Ottawa, Ontario, Canada.
authors: Jason
authors: J
authors: Tay
authors: Department of Medicine, University of Ottawa, Ottawa, Ontario, Canada.
authors: David
authors: D
authors: Allan
authors: Department of Psychosocial Oncology, Tom Baker Cancer Centre, Calgary, Alberta, Canada.
authors: Sara
authors: S
authors: Beattie
authors: Department of Medicine, University of Ottawa, Ottawa, Ontario, Canada.|Clinical Epidemiology Program, Ottawa Hospital Research Institute, Ottawa, Ontario, Canada.
authors: Christopher
authors: C
authors: Bredeson
authors: Department of Medicine, University of Ottawa, Ottawa, Ontario, Canada.|Clinical Epidemiology Program, Ottawa Hospital Research Institute, Ottawa, Ontario, Canada.
authors: Dean
authors: D
authors: Fergusson
authors: Department of Medicine, University of Toronto.
authors: Dawn
authors: D
authors: Maze
authors: Department of Medicine, University of Ottawa, Ottawa, Ontario, Canada.
authors: Mitchell
authors: M
authors: Sabloff
authors: Clinical Epidemiology Program, Ottawa Hospital Research Institute, Ottawa, Ontario, Canada.
authors: Kednapa
authors: K
authors: Thavorn
authors: Department of Medicine, University of Ottawa, Ottawa, Ontario, Canada.|Clinical Epidemiology Program, Ottawa Hospital Research Institute, Ottawa, Ontario, Canada.
authors: Alan
authors: A
authors: Tinmouth
mesh_terms: D000293:Adolescent; D000328:Adult; D000933:Antifibrinolytic Agents / Q000627:therapeutic use*; D001792:Blood Platelets; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006470:Hemorrhage / Q000209:etiology / Q000517:prevention & control*; D006801:Humans; D008297:Male; D017063:Outcome Assessment, Health Care; D010865:Pilot Projects; D017713:Platelet Transfusion / Q000009:adverse effects; D012107:Research Design; D013921:Thrombocytopenia / Q000601:surgery / Q000628:therapy*; D014148:Tranexamic Acid / Q000627:therapeutic use*
chemical_list: D000933:Antifibrinolytic Agents; D014148:Tranexamic Acid