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

Study Overview
=================
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.

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

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

Intervention / Treatment
-----------------
* Procedure: Red Blood Cell Transfusions (RBCT)


Participation Criteria
=================
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

Ages Eligible for Study
-----------------
Minimum Age: 19 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Supportive Care
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Supportive care (patient-driven RBCT)<br>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)<br>* Undergo patient-driven RBCT<br>* Other names: 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 |