Clinical trial NCT03086252: Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study
Official title: Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: NA
Conditions: Anemia
Aplastic Anemia
Bone Marrow Failure
Hematopoietic and Lymphoid Cell Neoplasm
Leukemia
Myelodysplastic Syndrome
Lead sponsor: Emory University
Sponsor class: OTHER
First posted: 2017-03-22
Enrollment: 0
Sex: ALL
Minimum age (years): 19
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

Brief summary: This pilot study evaluates safety of administration of red blood cell transfusions requested by patients based on their symptoms instead of levels of hemoglobin for the treatment of chronic anemia in patients with blood disorders.

Detailed description: PRIMARY OBJECTIVE:

I. To assess feasibility and safety of red blood cell transfusions at the time subjects requested based on symptomatic anemia.

SECONDARY OBJECTIVES:

I. To define a patient-driven red blood cell transfusion (RBCT) threshold and to compare the median hemoglobin (hgb) at the time subjects request RBCT to the standard threshold of hgb \< 8 g/dL.

II. To compare the number of red blood cell (RBC) units transfused and the frequency of transfusions at the time subjects request RBCT during the study period to the number of RBC units received and frequency using the standard threshold hemoglobin \< 8 g/dL during a comparable period prior to enrollment.

III. To compare the quality of life (QoL) scores when subjects request RBCT to the scores obtained at the time hgb is 8 +/- 0.5 g/dL.

OUTLINE:

Patients undergo RBCT based on their perception and/or the presence of anemia symptoms for up to 6 months.

After completion of study treatment, patients are followed up for 30 days.

Eligibility criteria: Inclusion Criteria:

* Diagnosed with hematological malignancy or marrow failure syndrome such as but not limited to: aplastic anemia, myelodysplastic syndrome or leukemia
* Chronic transfusion-dependent anemia with exposure to at least 5 RBCT
* Interested in reducing transfusion exposure
* Willing to sign informed consent

Exclusion Criteria:

* Recent acute bleeding requiring intervention in less than 24 hours
* Hemoglobin levels \< 6 g/dL
* Acute leukemia receiving induction chemotherapy
* Any patient with known ischemic heart disease, history of congestive heart failure, history of stroke, or cardiac arrhythmia for which the patient requires medication or a medical device
* Oxygen dependent
* Oxygen saturation below 92% on room air
* Receiving erythropoietin stimulating agent
* Thalassemia major or sickle cell disease requiring blood transfusion
* Undergoing major surgery
* Hemolytic anemia
* Coagulopathies including disseminated intravascular coagulation (DIC), receiving anticoagulant or antiplatelet agents
* Diagnosed with chronic obstructive pulmonary disease (COPD) oxygen dependent
* Pregnancy
* Participation in a therapeutic clinical trial

Primary outcomes:
measure: Incidence of laboratory and clinical adverse events (AEs) assessed by the Common Terminology Criteria for Adverse Events version 4.03.
timeFrame: Up to 6 months of study participation
Secondary outcomes:
measure: Frequency of RBC units transfused
timeFrame: Up to 6 months of study participation
measure: Hemoglobin concentration at the time subject requests RBCT
timeFrame: Up to 6 months of study participation
measure: Number of RBC units transfused
timeFrame: Up to 6 months of study participation
measure: QoL scores from the Functional Assessment of Cancer Therapy - Anemia/Fatigue (FACT-An) questionnaire at the time hemoglobin is 8 +/- 0.5 g/dL
timeFrame: Up to 6 months of study participation
measure: QoL scores from the Functional Assessment of Cancer Therapy - Anemia/Fatigue (FACT-An) questionnaire at the time subjects request RBCT
timeFrame: Up to 6 months of study participation
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Emory University/Winship Cancer Institute
state: Georgia
Study officials:
affiliation: Emory University
name: Ana Antun, MD
role: PRINCIPAL_INVESTIGATOR
References: