Thalassemia Major
Conditions
Keywords
Packed RBCs, leukodepletion, buffy-coat, transfusion
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.
Interventions
BIOLOGICALBlood component A
PRBCs obtained from whole blood after separation of plasma, buffy coat, and RBCs and successive leukoreduction of RBCs
BIOLOGICALBlood component B
PRBCs obtained by leukoreduction of whole blood, and successive separation of plasma and RBCs
Sponsors
Università degli Studi di Ferrara
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
NONE
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.
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
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Transfusion Power Index | 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
| Measure | Time frame | Description |
|---|---|---|
| Average pre-transfusion Hb concentration | 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 | 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 | 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 | 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 | 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) |
Countries
Italy
Outcome results
None listed