Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study

Emory University

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

SUPPORTIVE_CARE

Masking

NONE

Conditions

Anemia Aplastic Anemia Bone Marrow Failure Hematopoietic and Lymphoid Cell Neoplasm Leukemia Myelodysplastic Syndrome

Interventions

Red Blood Cell Transfusions (RBCT)PROCEDURE

Undergo patient-driven RBCT

Eligibility

Sex: ALLMin age: 19 Years

Medical Language ↔ Plain English

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

Locations (1)

Emory University/Winship Cancer Institute

Atlanta, Georgia, United States

Outcomes

Primary Outcomes

Incidence of laboratory and clinical adverse events (AEs) assessed by the Common Terminology Criteria for Adverse Events version 4.03.

Frequency, duration, and severity of AEs and severe adverse events will be determined. The proportion of subjects with serious side effects will be calculated with 95% confidence interval. Descriptive statistics will be used to characterize subjects enrolled on the trial: age, sex, race and diagnosis.