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

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.

Study Overview

• Status: Withdrawn
• Conditions: Hematopoietic and Lymphoid Cell Neoplasm; Leukemia; Anemia; Myelodysplastic Syndrome; Aplastic Anemia; Bone Marrow Failure
• Intervention / Treatment: Procedure: Red Blood Cell Transfusions (RBCT)

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.

• Study Type: Interventional
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory University/Winship Cancer Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 19 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Supportive care (patient-driven RBCT); Patients undergo red blood cell transfusions (RBCT) based on their perception and/or the presence of anemia symptoms for up to 6 months.	Procedure: Red Blood Cell Transfusions (RBCT); Undergo patient-driven RBCT; Other Names: Blood Component Transfusion; Blood Transfusion; Transfusion; Transfusion of Blood Products

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Up to 6 months of study participation

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Frequency of RBC units transfused	The frequency of RBC units transfused will be compared between study period and prior to enrollment for the same patient (e.g. each patient will serve his/her own control) by Wilcoxon's sign rank test.	Up to 6 months of study participation
Hemoglobin concentration at the time subject requests RBCT	Hemoglobin concentration (Hgb) will be measured in g/dL.	Up to 6 months of study participation
Number of RBC units transfused	The number of RBC units transfused will be compared between study period and prior to enrollment for the same patient (e.g. each patient will serve his/her own control) by Wilcoxon's sign rank test.	Up to 6 months of study participation
QoL scores from the Functional Assessment of Cancer Therapy - Anemia/Fatigue (FACT-An) questionnaire at the time hemoglobin is 8 +/- 0.5 g/dL	Will be compared to QoL scores at the time subjects request RBCT by paired t-test and/or Wilcoxon sign rank test.	Up to 6 months of study participation
QoL scores from the Functional Assessment of Cancer Therapy - Anemia/Fatigue (FACT-An) questionnaire at the time subjects request RBCT	Will be compared to QoL scores at the time hgb is 8 +/- 0.5 g/dL by paired t-test and/or Wilcoxon sign rank test.	Up to 6 months of study participation

Collaborators and Investigators

• Sponsor: Emory University
• Investigators: Principal Investigator: Ana Antun, MD, Emory University

Study record dates

Study Major Dates

• Study Start (Actual): July 24, 2017
• Primary Completion (Actual): May 31, 2018
• Study Completion (Actual): May 31, 2018

Study Registration Dates

• First Submitted: March 17, 2017
• First Submitted That Met QC Criteria: March 17, 2017
• First Posted (Actual): March 22, 2017

Study Record Updates

• Last Update Posted (Actual): July 26, 2018
• Last Update Submitted That Met QC Criteria: July 25, 2018
• Last Verified: July 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Bone Marrow Diseases; Myelodysplastic Syndromes; Hematologic Diseases; Anemia; Anemia, Aplastic; Bone Marrow Failure Disorders; Pancytopenia
• Other Study ID Numbers: IRB00090306; NCI-2016-01059 (Registry Identifier: CTRP (Clinical Trial Reporting Program)); Winship3145-16 (Other Identifier: Emory University/Winship Cancer Institute)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No