Health TrialsbyHealthPanel

Impact of IV Iron on Bleeding Symptoms in Iron Deficient Patients With Inherited Bleeding Disorders

ConditionsBleeding DisordersIronPlateletBleeding
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age15+
SponsorNicoletta C Machin

About this trial

This is a prospective, single-arm, single-center observational study evaluating the impact of intravenous (IV) iron replacement in patients with inherited bleeding disorders and iron deficiency (ferritin \<50ng/dL). Subjects will undergo baseline bleeding assessments, quality-of-life measures, and laboratory tests before receiving standard-of-care IV iron. Follow-up blood work and questionnaires will be conducted post-replacement to assess for changes

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Males and Females > 15 years of age

Diagnosed with an Inherited Bleeding Disorder (Von Willebrand disease, platelet disorders, factor deficiencies, or bleeding disorder of unknown cause)

Evidence of Iron Deficiency (Ferritin < 50 ng/mL)

Receiving IV iron at Hemophilia Center of Western Pennsylvania

Disqualifiers

Previous thrombosis, VTE History.

Platelet count < 100,000 * 109/L

Concomitant use of antiplatelet drugs, anticoagulants, aspirin, NSAIDs.

Trial population

Patient in the outpatient setting with a bleeding disorder and Iron Deficiency planned for IV Iron as per Standard of care followed at the Hemophilia Center of Western Pennsylvania

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • IV Iron (standard of care)

    Drug

    Participants will receive IV iron therapy as part of standard management for iron deficiency

Treatment groups

40 Participants
are divided into 1 treatment group
Group A: Iron Deficient patients with underlying bleeding disorders1 intervention

Trial outcomes

Primary outcomes

1

Change in bleeding assessment (ISTH-BAT score)

Change in bleeding severity 3 months after IV iron replacement, assessed using the ISTH-BAT (International Society on Thrombosis and Haemostasis Bleeding Assessment Tool). ISTH-BAT: Score range 0-56; higher scores indicate greater bleeding severity. Safety Issue: No

Time frame
Baseline to 3 months post-IV iron replacement
2

Change in Menstrual Blood Loss (PBAC Score)

Change in menstrual bleeding 3 months after IV iron replacement, measured using the Pictorial Blood Loss Assessment Chart (PBAC). Assessed only in menstruating female participants. PBAC: Score \<100 indicates normal menstrual blood loss; scores \>100 suggest heavy menstrual bleeding.

Time frame
Baseline to 3 months post-IV iron replacement
3

Change in Epistaxis Severity Score (ESS)

Change in epistaxis severity from baseline to 3 months post-IV iron replacement, measured using the Epistaxis Severity Score (ESS), a validated clinical tool for quantifying frequency, duration, and impact of nosebleeds. Score Range: 0 to 10 Scoring Interpretation: Higher scores indicate more severe or frequent nosebleeds. A reduction in score reflects clinical improvement.

Time frame
Baseline to 3 months post-IV iron replacement

Secondary outcomes

1

Change in Fatigue (FACIT-F Score)

Change in fatigue levels from baseline to 3 months after IV iron replacement, as measured by the Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-F). Score Range: 0 to 160 Scoring Interpretation: Lower scores indicate greater fatigue; higher scores indicate improved energy and well-being.

Time frame
Baseline to 3 months post-IV iron replacement
2

Change in Physical Functioning (SF-36)

Change in physical functioning score on the SF-36 from baseline to 3 months post-IV iron. Score Range: 0 to 100 Scoring Interpretation: Higher scores indicate better physical functioning.

Time frame
Baseline to 3 months
3

Change in Role Limitations Due to Physical Health (SF-36)

Change in score from the Role Physical domain of the SF-36, which evaluates problems with work or daily activities due to physical health. Score Range: 0 to 100 Scoring Interpretation: Higher scores reflect fewer role limitations.

Time frame
Baseline to 3 months
4

Change in Role Limitations Due to Emotional Problems (SF-36)

Change in the Role Emotional domain score of the SF-36, which assesses limitations in daily activities due to emotional difficulties. Score Range: 0 to 100 Scoring Interpretation: Higher scores indicate better emotional role functioning.

Time frame
Baseline to 3 months

Other outcomes

1

Change in Mitochondrial Reactive Oxygen Species (ROS) in Platelets

Evaluate changes in mitochondrial reactive oxygen species (ROS) levels in platelet samples from baseline to 3 months post-IV iron replacement. This exploratory analysis will assess whether iron repletion alters platelet oxidative stress, potentially contributing to changes in platelet function and bleeding phenotype.

Time frame
Baseline to 3 months post-IV iron replacement
2

Change in Platelet Reactivity by Flow Cytometry

Evaluate changes in platelet reactivity before and after IV iron replacement, using flow cytometry to assess surface expression of activation markers (e.g., P-selectin, activated αIIbβ3 integrin) in response to standard agonists (e.g., ADP, CRP-XL). This exploratory analysis will examine whether iron repletion restores platelet responsiveness in iron-deficient individuals with inherited bleeding disorders.

Time frame
Baseline to 3 months post-IV iron replacement

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Nicoletta C Machin

Lead sponsor

University of Pittsburgh

Sponsor institution

Health Resources and Services Administration (HRSA)

Collaborator

Source ClinicalTrials.gov
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