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

Not Applicable

Withdrawn

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

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.

Recruitment & Eligibility

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

Arm && Interventions

Group	Intervention	Description
Supportive care (patient-driven RBCT)	Red Blood Cell Transfusions (RBCT)	Patients undergo red blood cell transfusions (RBCT) based on their perception and/or the presence of anemia symptoms for up to 6 months.

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
Hemoglobin concentration at the time subject requests RBCT	Up to 6 months of study participation	Hemoglobin concentration (Hgb) will be measured in g/dL.
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.
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.
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.

Locations (1): Emory University/Winship Cancer Institute
🇺🇸
Atlanta, Georgia, United States