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出境医 / 临床实验 / Impact of the Preparation Method of Red Cell Concentrates on Transfusion Indices in Thalassemic Patients

Impact of the Preparation Method of Red Cell Concentrates on Transfusion Indices in Thalassemic Patients

Study Description
Brief Summary:
This study compares the effects of Packed Red Blood Cells (PRBCs) prepared in two different ways on the transfusion indices in beta(ß)-Thalassemia transfusion-dependent patients. The two blood components types derive from the whole blood. In one case, the whole blood is leukoreduced with subsequent plasma removal. In the other case, plasma, buffy coat, and red blood cells (RBCs) are first separated and subsequently, the RBCs leukoreduced. Each type of blood components will be subsequently given to one-half of the patients for a 6-month period and to the other half for other 6-month at the randomization phase, for a total of 12 months of crossed-treatment per patient.

Condition or disease Intervention/treatment Phase
Thalassemia Major Biological: Blood component A Biological: Blood component B Phase 4

Detailed Description:
At Day Hospital Talassemia ed Emoglobinopatie of Ferrara, two different PRBCs are available. The two types of blood components are obtained from whole blood, pre-storage leukoreduced and suspended in saline-adenine-glucose-mannitol (SAGM). One method of preparation consists of the whole blood leukoreduction with subsequent plasma removal. The other method first separates plasma, buffy coat, and RBCs, and then the RBCs are leukoreduced. The two methods mainly differ in the final haemoglobin (Hb) content: the Hb level is lower (-13%, approximately) in the second method that also shows the advantage to produce platelets from the buffy coat. A PRBCs unit is not as strictly defined as a therapeutic medication dose (pill or vial): individual PRBCs units may substantially differ in their Hb content, much more than the average difference between the two types of preparations. The aim of this study is to document the extent of the average difference between the two types of preparations, and its impacts on the transfusion indices of ß-Thalassaemia transfusion-dependent patients. All patients will receive each blood component for a period of 6 months (crossover design), for a total of 12 months of transfusion treatment per patient.
Study Design
Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 55 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description: Patients will be divided into two groups of approximately equal number. The first group will receive the blood component A for a period of 6 months and then the blood component B for the next 6 months. The second group will receive the blood components in inverted order
Masking: None (Open Label)
Masking Description: The patients will not be informed on the blood components sequence that they will receive. However, the units exterior appearance of the two types of preparations is different. For this reason, patients and care providers will most probably notice it.
Primary Purpose: Treatment
Official Title: Prospective Crossover Study on Beta(ß)-Thalassaemia Transfusion-dependent to Evaluate the Impact on Transfusion Regimen of Two Pre-storage Leukoreduced PRBCs(In-line Filtration + B-C Separation; Whole Blood Filtration + B-C Conservation)
Actual Study Start Date : May 14, 2018
Estimated Primary Completion Date : June 2019
Estimated Study Completion Date : June 2019
Arms and Interventions
Arm Intervention/treatment
Experimental: Sequence A-B
Patients in this arm will receive blood component A for 6 months and blood component B for the next 6 months
 Biological: Blood component A
PRBCs obtained from whole blood after separation of plasma, buffy coat, and RBCs and successive leukoreduction of RBCs

Biological: Blood component B
PRBCs obtained by leukoreduction of whole blood, and successive separation of plasma and RBCs
Experimental: Sequence B-A
Patients in this arm will receive blood component B for 6 months and blood component A for the next 6 months
 Biological: Blood component A
PRBCs obtained from whole blood after separation of plasma, buffy coat, and RBCs and successive leukoreduction of RBCs

Biological: Blood component B
PRBCs obtained by leukoreduction of whole blood, and successive separation of plasma and RBCs
Outcome Measures
Primary Outcome Measures :
  1. Transfusion Power Index [ Time Frame: For each of the two blood components studied, at the end of 6-month period of study ]
    (Average pre-transfusion Hb concentration)/(Unit Index) [for the definition of Unit Index, see the secondary outcomes]


Secondary Outcome Measures :
  1. Average pre-transfusion Hb concentration [ Time Frame: For each of the two blood components studied, at the end of 6-month period of study ]
    Mean pre-transfusion Hb levels, calculated starting from the second transfusion of the period to the first transfusion of the following period

  2. Unit Index [ Time Frame: For each of the two blood components studied, at the end of 6-month period of study ]
    (Total number of PRBCs (A or B) transfused in the period)/(Number of days between the first transfusion of the period and the first transfusion of the following period)

  3. Average Transfusion Interval [ Time Frame: For each of the two blood components studied, at the end of 6-month period of study ]
    (Number of days between the first transfusion of the period and the first transfusion of the following period)/(Number of transfusions in the period)

  4. Number of Transfusion Reactions [ Time Frame: Study periods (2 periods of 6 months each) ]
    Number of transfusion reactions to the two blood components that may occur in the study periods (2 periods of 6 months each)

  5. Transfusion Reaction Rate [ Time Frame: Study periods (2 periods of 6 months each) ]
    (Number of transfusion reactions to the two blood components occurring in the study periods)/(Total number of PRBCs (A or B) transfused in the period)


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patient with beta(ß)-Thalassaemia transfusion-dependent (ß-Thalassemia Major or ß-Thalassemia Intermedia transfusion-dependent, regularly transfused since at least 5 years

Exclusion Criteria:

  • Patient not exclusively transfused at Day Hospital Thalassaemia and Haemoglobinopathies of Ferrara
  • Patient with haemolytic auto-antibodies
  • Patient transfused with washed Packet RBCs units
  • Severe splenomegaly (>18 cm on echography)
  • Elevated blood consumption (>200 mL/kg of pure RBCs in the last year)
  • Patient receiving haemoglobin inducers in the last 6 months
  • Any significant clinical pulmonary, cardiovascular, endocrine, hepatic, gastrointestinal, renal, infectious, immunological including significant allo- or auto-immunisation disease, considered not adequately controlled prior to the study
  • Patient treated with erythrocyte exchange
  • Pregnant females
Contacts and Locations

Locations
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Italy
Day Hospital Thalassaemia and Haemoglobinopathies (DHTE)
Ferrara, Italy, 44134
Sponsors and Collaborators
Università degli Studi di Ferrara
Investigators
Layout table for investigator information
Study Chair: Maria Rita Gamberini, MD D.H. Thalassaemia-Haemoglobinopathies (DHTE) - A.O.U. S. Anna of Ferrara
Tracking Information
First Submitted Date  ICMJE November 15, 2018
First Posted Date  ICMJE June 19, 2019
Last Update Posted Date June 19, 2019
Actual Study Start Date  ICMJE May 14, 2018
Estimated Primary Completion Date June 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 18, 2019) 		Transfusion Power Index [ Time Frame: For each of the two blood components studied, at the end of 6-month period of study ]
(Average pre-transfusion Hb concentration)/(Unit Index) [for the definition of Unit Index, see the secondary outcomes]
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: June 18, 2019)
  • Average pre-transfusion Hb concentration [ Time Frame: For each of the two blood components studied, at the end of 6-month period of study ]
    Mean pre-transfusion Hb levels, calculated starting from the second transfusion of the period to the first transfusion of the following period
  • Unit Index [ Time Frame: For each of the two blood components studied, at the end of 6-month period of study ]
    (Total number of PRBCs (A or B) transfused in the period)/(Number of days between the first transfusion of the period and the first transfusion of the following period)
  • Average Transfusion Interval [ Time Frame: For each of the two blood components studied, at the end of 6-month period of study ]
    (Number of days between the first transfusion of the period and the first transfusion of the following period)/(Number of transfusions in the period)
  • Number of Transfusion Reactions [ Time Frame: Study periods (2 periods of 6 months each) ]
    Number of transfusion reactions to the two blood components that may occur in the study periods (2 periods of 6 months each)
  • Transfusion Reaction Rate [ Time Frame: Study periods (2 periods of 6 months each) ]
    (Number of transfusion reactions to the two blood components occurring in the study periods)/(Total number of PRBCs (A or B) transfused in the period)
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Impact of the Preparation Method of Red Cell Concentrates on Transfusion Indices in Thalassemic Patients
Official Title  ICMJE Prospective Crossover Study on Beta(ß)-Thalassaemia Transfusion-dependent to Evaluate the Impact on Transfusion Regimen of Two Pre-storage Leukoreduced PRBCs(In-line Filtration + B-C Separation; Whole Blood Filtration + B-C Conservation)
Brief Summary This study compares the effects of Packed Red Blood Cells (PRBCs) prepared in two different ways on the transfusion indices in beta(ß)-Thalassemia transfusion-dependent patients. The two blood components types derive from the whole blood. In one case, the whole blood is leukoreduced with subsequent plasma removal. In the other case, plasma, buffy coat, and red blood cells (RBCs) are first separated and subsequently, the RBCs leukoreduced. Each type of blood components will be subsequently given to one-half of the patients for a 6-month period and to the other half for other 6-month at the randomization phase, for a total of 12 months of crossed-treatment per patient.
Detailed Description At Day Hospital Talassemia ed Emoglobinopatie of Ferrara, two different PRBCs are available. The two types of blood components are obtained from whole blood, pre-storage leukoreduced and suspended in saline-adenine-glucose-mannitol (SAGM). One method of preparation consists of the whole blood leukoreduction with subsequent plasma removal. The other method first separates plasma, buffy coat, and RBCs, and then the RBCs are leukoreduced. The two methods mainly differ in the final haemoglobin (Hb) content: the Hb level is lower (-13%, approximately) in the second method that also shows the advantage to produce platelets from the buffy coat. A PRBCs unit is not as strictly defined as a therapeutic medication dose (pill or vial): individual PRBCs units may substantially differ in their Hb content, much more than the average difference between the two types of preparations. The aim of this study is to document the extent of the average difference between the two types of preparations, and its impacts on the transfusion indices of ß-Thalassaemia transfusion-dependent patients. All patients will receive each blood component for a period of 6 months (crossover design), for a total of 12 months of transfusion treatment per patient.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description:
Patients will be divided into two groups of approximately equal number. The first group will receive the blood component A for a period of 6 months and then the blood component B for the next 6 months. The second group will receive the blood components in inverted order
Masking: None (Open Label)
Masking Description:
The patients will not be informed on the blood components sequence that they will receive. However, the units exterior appearance of the two types of preparations is different. For this reason, patients and care providers will most probably notice it.
Primary Purpose: Treatment
Condition  ICMJE Thalassemia Major
Intervention  ICMJE
  • Biological: Blood component A
    PRBCs obtained from whole blood after separation of plasma, buffy coat, and RBCs and successive leukoreduction of RBCs
  • Biological: Blood component B
    PRBCs obtained by leukoreduction of whole blood, and successive separation of plasma and RBCs
Study Arms  ICMJE
  • Experimental: Sequence A-B
    Patients in this arm will receive blood component A for 6 months and blood component B for the next 6 months
    Interventions:
    • Biological: Blood component A
    • Biological: Blood component B
  • Experimental: Sequence B-A
    Patients in this arm will receive blood component B for 6 months and blood component A for the next 6 months
    Interventions:
    • Biological: Blood component A
    • Biological: Blood component B
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Actual Enrollment  ICMJE
 (submitted: June 18, 2019) 		55
Original Actual Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2019
Estimated Primary Completion Date June 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Patient with beta(ß)-Thalassaemia transfusion-dependent (ß-Thalassemia Major or ß-Thalassemia Intermedia transfusion-dependent, regularly transfused since at least 5 years

Exclusion Criteria:

  • Patient not exclusively transfused at Day Hospital Thalassaemia and Haemoglobinopathies of Ferrara
  • Patient with haemolytic auto-antibodies
  • Patient transfused with washed Packet RBCs units
  • Severe splenomegaly (>18 cm on echography)
  • Elevated blood consumption (>200 mL/kg of pure RBCs in the last year)
  • Patient receiving haemoglobin inducers in the last 6 months
  • Any significant clinical pulmonary, cardiovascular, endocrine, hepatic, gastrointestinal, renal, infectious, immunological including significant allo- or auto-immunisation disease, considered not adequately controlled prior to the study
  • Patient treated with erythrocyte exchange
  • Pregnant females
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Italy
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03992001
Other Study ID Numbers  ICMJE CrossoverFE2018
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description: There is a plan to make IPD (deidentified) available to other researchers and support already published results
Supporting Materials: Study Protocol
Supporting Materials: Statistical Analysis Plan (SAP)
Supporting Materials: Informed Consent Form (ICF)
Time Frame: Beginning 3 months and ending 3 years following article publication
Access Criteria: Please contact the Central Contact Person
Responsible Party Maria Rita Gamberini, Università degli Studi di Ferrara
Study Sponsor  ICMJE Università degli Studi di Ferrara
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Chair: Maria Rita Gamberini, MD D.H. Thalassaemia-Haemoglobinopathies (DHTE) - A.O.U. S. Anna of Ferrara
PRS Account Università degli Studi di Ferrara
Verification Date June 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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