Pilot Optimizing Transfusion Thresholds in Critically-ill Children With Anaemia
Pilot Optimizing Transfusion Thresholds in Critically-ill Children With Anaemia (P-OpTTICCA): a Pilot Trial for a Large Pragmatic International Parallel Open-label Non-inferiority Randomised Controlled Trial.
Lead Sponsor: St. Justine's Hospital
|Source||St. Justine's Hospital|
The Pilot Optimizing Transfusion Thresholds in Critically-ill Children with Anaemia (P-OpTTICCA) study is a pilot trial for a large pragmatic international parallel open-label non-inferiority randomised controlled trial. The primary outcome of the pilot study is feasibility.
Patients in paediatric intensive care units (PICU) are among the main groups of transfusion recipients. Blood transfusions expose recipients to infectious and non-infectious serious hazards. Despite an increasing number of red blood cell (RBC) transfusion threshold randomised controlled trials (RCT) to date, only one large RCT, the Transfusion Requirement In PICU (TRIPICU) study (Lacroix. N Engl J Med 2007;356:1609-19) informs practice in PICU. This dearth of paediatric data was reiterated by a recent National Heart, Lung and Blood Institutes state of the art symposium. Despite being internally robust, the generalizability of TRIPICU has been questioned given that nearly 90% of critically ill children with haemoglobin (Hb) level ≤ 95 g/L were not enrolled for a range of pre-specified reasons including severity of illness, clinical instability and uncertain physician buy-in, thus threatening broader application of this critical knowledge into practice. Recent data and evidence summarised in systematic reviews support the need to explore the benefits and safety of Hb threshold ≤ 70 g/L for almost all critically ill children, not only those who would have been enrolled in TRIPICU.
We propose an international pilot study that will inform the design of a large pragmatic non-inferiority RCT (termed OpTTICCA) conceived to derive generalizable transfusion guidance for physicians.
Objectives of the pilot-RCT. Specific aims are to:
1. Establish the feasibility of enrolling ≥80% of eligible patients.
2. Document adherence to study intervention ≥80% (restrictive transfusion policy).
3. Assess the incidence rate of the primary outcome measure of the full RCT (new and progressive multiple organ dysfunction syndrome, which includes mortality).
4. Establish the feasibility of using routinely collected clinical information from electronic medical data monitoring system (eMDMS) to enhance cost-efficiency.
In the pilot-RCT, we will enrol 120 patients (20 to 40/site) in PICUs equipped with electronic medical data monitoring system (eMDMS). This trial builds on successful prior RCTs that involved international collaboration and funding (Canada, United Kingdom, France): TRIPICU, Age of Blood Evaluation (ABLE) (Lacroix. N Engl J Med 2015) and Age of Blood in Children in PICU (ABC-PICU study: Tucci et al. Trials 2018). The pilot-RCT will be considered as the vanguard phase of the full OpTTICCA RCT if no important design changes are required.
Expected results. This pilot-RCT will focus on feasibility parameters. It will explore whether we can 1) recruit a much broader eligible patient group, 2) assess protocol adherence, 3) assess the primary outcome measure, and 4) abstract most data electronically. A definitive trial will provide transfusion guidance for many important subgroups in whom evidence is presently lacking including children admitted with bone marrow failure, head injury, some cardiac disorders, and sepsis.
|Overall Status||Not yet recruiting|
|Start Date||October 31, 2019|
|Completion Date||September 2020|
|Primary Completion Date||June 2020|
Intervention Type: Procedure
Intervention Name: Less red blood cell transfusions
Description: The instructions given to caregivers will be to consider prescribing a red blood cell transfusion only if the hemoglobin level is ≤ 70 g per L.
Arm Group Label: Restrictive arm (intervention)
Other Name: Restrictive red blood cell transfusion strategy
Intervention Type: Procedure
Intervention Name: More red blood cell transfusion (standard transfusion strategy in PICU)
Description: The instructions given to caregivers will be to prescribe a red blood cell transfusion according to their usual standard practice
Arm Group Label: Standard care arm (comparator)
Inclusion Criteria: - 1. Hemoglobin concentration ≤ 95 g/L, while in PICU - 2. Not already participating to a randomized controlled trial on hemoglobin threshold to guide red blood cell transfusion practice Exclusion Criteria: 1. Post conception age ≤ 36 weeks or > 18 years at PICU entry 2. Refusal of consent by patient and/or parent 3. Uncorrected cyanotic cardiac disease, univentricular physiology 4. Sickle cell disease 5. Brain Death 6. Extracorporeal membrane oxygenation (ECMO)
- 1. Hemoglobin concentration ≤ 95 g/L, while in PICU
- 2. Not already participating to a randomized controlled trial on hemoglobin threshold to guide red blood cell transfusion practice
1. Post conception age ≤ 36 weeks or > 18 years at PICU entry
2. Refusal of consent by patient and/or parent
3. Uncorrected cyanotic cardiac disease, univentricular physiology
4. Sickle cell disease
5. Brain Death
6. Extracorporeal membrane oxygenation (ECMO)
Minimum Age: N/A
Maximum Age: 18 Years
Healthy Volunteers: No
Last Name: Jacques Lacroix
Phone Ext.: 5556
Email: [email protected]
Type: Principal Investigator
Investigator Affiliation: St. Justine's Hospital
Investigator Full Name: Jacques Lacroix, MD, FRCPC, Professor
Investigator Title: Professor
|Has Expanded Access||No|
|Number Of Arms||2|
Label: Restrictive arm (intervention)
Description: Less red blood cell transfusions. The protocol will require that no red blood cell transfusion be given unless the hemoglobin level is below or equal at 70 g per L.
Label: Standard care arm (comparator)
Type: Active Comparator
Description: Clinical teams will follow their usual transfusion practices.
|Study Design Info||
Intervention Model: Parallel Assignment
Intervention Model Description: parallel open-label pragmatic non-inferiority randomised controlled trial (RCT)
Primary Purpose: Treatment
Masking: Double (Investigator, Outcomes Assessor)