- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02845232
Economic Analysis of Blood Product Transfusions According to the Treatment of Acute Myeloid Leukaemia in the Elderly
Blood transfusion requirement represents one of the most significant cost driver associated with acute myeloid leukemia (AML). In addition to an increase prevalence of co morbidities in older patients, AML in older patients is more often associated with adverse features than in younger adults. Physicians might therefore decide to offer palliative or supportive care rather than intensive chemotherapy. An alternative treatment could be low-intensity therapy, such as LD-AraC or hypomethylating agents, which demonstrated better results than only Best Supportive care (BSC). Blood transfusion requirement represents one of the most significant cost driver associated with AML.
The present study assesses the cost-effectiveness of intensive chemotherapy versus Best Supportive Care (BSC) versus alternative therapies (hypomethylating agents, low-dose cytosine arabinoside (LD-AraC), or other investigational drugs) in elderly patients aged 70 years or older regarding blood product transfusions from a French payer perspective. Intensive chemotherapy and BSC were the comparators in this analysis, since they continue to represent the most commonly used treatment for elderly AML according to the defined status of patients considered as 'fit' or 'unfit' for intensive chemotherapy.
Study Overview
Study Type
Enrollment (Actual)
Contacts and Locations
Study Locations
-
-
-
Pierre-benite, France, 69310
- Hospices Civils de Lyon - Centre Hospitalier Lyon Sud, 165 Chemin du Grand Revoyet
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
Study Population
Three groups of elderly patients aged 70 years or older, with AML. First group: 68 patients receiving a combination of intermediate-dose cytarabine and an anthracycline. One patient with acute promyelocytic leukaemia (APL) also received all-trans retinoic acid (ATRA).
The second study group comprised 70 patients who were treated on frontline by lower-intensity treatments [LD-AraC(39 patients), azacitidine (16 patients), decitabine (11 patients),tipifarnib (3 patients), or ATRA (1 patient)].
The last study group comprises 76 patients: 31 patients received supportive care, while 36 patients also received hydroxyurea and 9 patients received 6-mercaptopurine.
Description
Inclusion Criteria:
- Age ≥ 70 years old
- AML according to the World Health Organization (WHO) criteria (% of blasts ≥ 20% in bone marrow aspiration).
- All FAB subtypes.
- Any type of AML (de novo or secondary)
- All participants to clinical trials gave their written informed consent
Exclusion Criteria:
- Have an Eastern Cooperative Oncology Group (ECOG) score ≥2
- Active uncontrolled infection
Study Plan
How is the study designed?
Design Details
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
---|---|
Intensive chemotherapy
First group: 68 patients receiving a combination of intermediate-dose cytarabine and an anthracycline.
One patient with acute promyelocytic leukaemia (APL) also received all-trans retinoic acid (ATRA).
|
The number and type of blood products administered were registered from the time of diagnosis to the time of death corresponding for all patients to the time of last follow-up.
Transfusion of a single unit of packed red blood cell (PRBC) or one whole blood-derived platelet concentrate (PC) or fresh frozen plasma (FFP) was considered a transfusion event and considered for statistical analysis.
|
Lower-intensity treatments
The second study group comprised 70 patients who were treated on frontline by lower-intensity treatments [LD-AraC(39 patients), azacitidine (16 patients), decitabine (11 patients),tipifarnib (3 patients), or ATRA (1 patient)].
Patients received LD-AraC 20 mg once or twice daily (according to physician'schoice) by subcutaneous injection for 10 consecutive days.
Azacitidine was given at the dose of 75 mg/m2/day for 7 consecutive days by sc injection.
Decitabine was administered by intravenous route once daily at 20 mg/m2 for 5 consecutive days.
Tipifarnib was given at 600 mg administered orally twice daily for 21 consecutive days in 4-week cycles.
ATRA was given at 45 mg/m2until CR achievement followed by maintenance combining 6-mercaptopurine with methotrexate.
|
The number and type of blood products administered were registered from the time of diagnosis to the time of death corresponding for all patients to the time of last follow-up.
Transfusion of a single unit of packed red blood cell (PRBC) or one whole blood-derived platelet concentrate (PC) or fresh frozen plasma (FFP) was considered a transfusion event and considered for statistical analysis.
|
Best Supportive Care
The last study group comprises 76 patients: 31 patients received supportive care, while 36 patients also received hydroxyurea and 9 patients received 6-mercaptopurine.
|
The number and type of blood products administered were registered from the time of diagnosis to the time of death corresponding for all patients to the time of last follow-up.
Transfusion of a single unit of packed red blood cell (PRBC) or one whole blood-derived platelet concentrate (PC) or fresh frozen plasma (FFP) was considered a transfusion event and considered for statistical analysis.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Estimation of mean blood product transfusions costs (in euros) per patient according to overall survival
Time Frame: From starting treatment to death from any cause (up to 21 months)
|
The cost-effectiveness of blood product transfusion was determined among initial treatment subgroups: patients receiving intensive chemotherapy, patients receiving low-intensity treatments, and patients treated only by BSC.
|
From starting treatment to death from any cause (up to 21 months)
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Complete remission (CR) rate
Time Frame: Duration of study (Month 21)
|
Complete remission (CR) was defined by bone marrow aspiration, which was required to consist of more than 50% normal cellularity with evidence of trilineage maturation and less than 5% bone marrow blasts, no evidence of extramedullary disease, and regeneration of the peripheral neutrophil count to 1.0 × 109/L and the platelet count to 100 × 109/L.
The persistence of myelodysplastic features did not exclude the diagnosis of CR.
|
Duration of study (Month 21)
|
Number of blood product transfusions per patient
Time Frame: Duration of study (Month 21)
|
Decisions concerning transfusion indications were based on institutional policy and the clinical judgment of treating physicians.
Transfusion practice was consistent during the period of study.
Prophylactic transfusions were consistently given at morning platelet counts of < 20 ×109/L and haemoglobin level < 80 g/L.
Protocol Blood products were leukoreduced through discarding the buffy coat and administered through a standard 140 to 170 µm blood filter but were not irradiated
|
Duration of study (Month 21)
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 69HCL16_0461
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Acute Myeloid Leukemia
-
University of PennsylvaniaActive, not recruitingAcute Myeloid Leukemia, in Relapse | Acute Myeloid Leukemia, Refractory | Acute Myeloid Leukemia, PediatricUnited States
-
Terrence J Bradley, MDImago BioSciences, Inc., a subsidiary of Merck & Co., Inc., (Rahway, New...RecruitingAcute Myeloid Leukemia | Refractory Acute Myeloid Leukemia | Acute Myeloid Leukemia, in RelapseUnited States
-
National Cancer Institute (NCI)RecruitingAcute Myeloid Leukemia | Secondary Acute Myeloid Leukemia | Recurrent Acute Myeloid Leukemia | Refractory Acute Myeloid LeukemiaUnited States
-
Bhavana BhatnagarCTI BioPharmaCompletedRecurrent Adult Acute Myeloid Leukemia | Secondary Acute Myeloid Leukemia | Untreated Adult Acute Myeloid Leukemia | Therapy-Related Acute Myeloid LeukemiaUnited States
-
Washington University School of MedicineWithdrawnRefractory Acute Myeloid Leukemia | Relapsed Acute Myeloid LeukemiaUnited States
-
C. Babis AndreadisGateway for Cancer Research; AVEO Pharmaceuticals, Inc.TerminatedAcute Myeloid Leukemia | Refractory Acute Myeloid Leukemia | Relapsed Acute Myeloid LeukemiaUnited States
-
Jacqueline Garcia, MDEli Lilly and CompanyCompletedCombination Merestinib and LY2874455 for Patients With Relapsed or Refractory Acute Myeloid LeukemiaRelapsed Adult Acute Myeloid Leukemia | Refractory Adult Acute Myeloid LeukemiaUnited States
-
Kronos BioActive, not recruitingAcute Myeloid Leukemia | Refractory Acute Myeloid Leukemia | Relapsed Acute Myeloid LeukemiaUnited States, Spain
-
Massachusetts General HospitalExelixisCompletedRefractory Acute Myeloid Leukemia | Relapsed Acute Myeloid LeukemiaUnited States
-
City of Hope Medical CenterNational Cancer Institute (NCI)RecruitingAcute Myeloid Leukemia | Recurrent Adult Acute Myeloid Leukemia | Secondary Acute Myeloid Leukemia | Refractory Acute Myeloid LeukemiaUnited States
Clinical Trials on Transfusion
-
National Institutes of Health Clinical Center (CC)Recruiting
-
Assistance Publique - Hôpitaux de ParisCompletedMyocardial Infarction | Anemia | Blood TransfusionSpain, France
-
Hamad Medical CorporationSidra Medical and Research Center; World Anti-Doping Agency; Anti-Doping Lab... and other collaboratorsUnknownBlood Disease | Blood Transfusion, Autologous | Blood Doping | Blood Transfusion, HomologousQatar
-
Daping Hospital and the Research Institute of Surgery...Children's Hospital of Chongqing Medical UniversityWithdrawnAnemia | Necrotizing Enterocolitis | Red Blood Cell (RBC) TransfusionChina
-
Fred Hutchinson Cancer CenterNational Cancer Institute (NCI)TerminatedAcute Myeloid Leukemia | Primary Myelofibrosis | Thrombocytopenia | Acute Lymphoblastic Leukemia | Chronic Lymphocytic Leukemia | Venous Thromboembolism | Myelodysplastic Syndrome | B-Cell Non-Hodgkin Lymphoma | Hematopoietic Cell Transplantation Recipient | Chronic Myelogenous Leukemia, BCR-ABL1 Positive and other conditionsUnited States
-
Chang Gung Memorial HospitalCompletedTrauma Patients Received Blood Transfusion ≥ 10 U | Trauma Patients Had Not Received Blood Transfusion ≥ 10 UTaiwan
-
Assistance Publique - Hôpitaux de ParisInstitut National de la Santé Et de la Recherche Médicale, France; Etablissement... and other collaboratorsRecruitingThe Focus is on the Detection of the Consequences of Autologous Blood Transfusion in Healthy VolunteersFrance
-
Hospital PitangueirasUnknown
-
Unity Health TorontoRecruitingCardiac Surgery | Heart | Postoperative | DisorderCanada, Russian Federation
-
Haukeland University HospitalDentsply Sirona Implants and ConsumablesCompleted