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

Sponsor

Emory University (Other)

Overall Status

Withdrawn

CT.gov ID

NCT03086252

Collaborator

(none)

Enrollment

Location

Arm

10.2

Actual Duration (Months)

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Anemia Aplastic Anemia Bone Marrow Failure Hematopoietic and Lymphoid Cell Neoplasm Leukemia Myelodysplastic Syndrome	Procedure: Red Blood Cell Transfusions (RBCT)	N/A

Detailed Description

PRIMARY OBJECTIVE:

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

SECONDARY OBJECTIVES:

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.
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.
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 Design

Study Type:

Interventional

Actual Enrollment :

0 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Supportive Care

Official Title:

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

Actual Study Start Date :

Jul 24, 2017

Actual Primary Completion Date :

May 31, 2018

Actual Study Completion Date :

May 31, 2018

Arms and Interventions

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

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

Outcome Measures

Primary Outcome Measures

Incidence of laboratory and clinical adverse events (AEs) assessed by the Common Terminology Criteria for Adverse Events version 4.03. [Up to 6 months of study participation]
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.

Secondary Outcome Measures

Frequency of RBC units transfused [Up to 6 months of study participation]
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.
Hemoglobin concentration at the time subject requests RBCT [Up to 6 months of study participation]
Hemoglobin concentration (Hgb) will be measured in g/dL.
Number of RBC units transfused [Up to 6 months of study participation]
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.
QoL scores from the Functional Assessment of Cancer Therapy - Anemia/Fatigue (FACT-An) questionnaire at the time hemoglobin is 8 +/- 0.5 g/dL [Up to 6 months of study participation]
Will be compared to QoL scores at the time subjects request RBCT by paired t-test and/or Wilcoxon sign rank test.
QoL scores from the Functional Assessment of Cancer Therapy - Anemia/Fatigue (FACT-An) questionnaire at the time subjects request RBCT [Up to 6 months of study participation]
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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

19 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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