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

NCT07083583 | Recruiting FDA Drug

• 1 other identifier: STUDY25020144
• Study Type: observational
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Bleeding Disorders; Iron; Platelet; Bleeding

Keywords

Iron deficiency; bleeding disorder; platelet function

Outcome Measures

Primary Outcomes (3)

• 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

  Baseline to 3 months post-IV iron replacement

• 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.

  Baseline to 3 months post-IV iron replacement

• 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.

  Baseline to 3 months post-IV iron replacement

Secondary Outcomes (9)

• Change in Fatigue (FACIT-F Score)

  Baseline to 3 months post-IV iron replacement

• Change in Physical Functioning (SF-36)

  Baseline to 3 months

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

  Baseline to 3 months

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

  Baseline to 3 months

• Change in the Vitality domain of the SF-36, measuring energy levels and fatigue.

  Baseline to 3 months

Other Outcomes (2)

• Change in Mitochondrial Reactive Oxygen Species (ROS) in Platelets

  Baseline to 3 months post-IV iron replacement

• Change in Platelet Reactivity by Flow Cytometry

  Baseline to 3 months post-IV iron replacement

Study Arms (1)

Iron Deficient patients with underlying bleeding disorders

Subjects ≥15 years old with a history of an inherited bleeding disorder (including von Willebrand disease, platelet disorders, factor deficiencies, or bleeding disorder of unknown cause) and ferritin \<50 ng/mL identified during routine clinic evaluation.

Drug: IV Iron (standard of care)

Interventions

IV Iron (standard of care) DRUG

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

Iron Deficient patients with underlying bleeding disorders

Eligibility Criteria

• Age: 15 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study 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

You may qualify if:

• 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
• Willingness to have blood drawn
• Willing to return to clinic 3 months post infusion for final blood draw, bleeding and quality of life assessments.

You may not qualify if:

• Previous thrombosis, VTE History.
• Platelet count \< 100,000 \* 109/L
• Concomitant use of antiplatelet drugs, anticoagulants, aspirin, NSAIDs.

Sponsors & Collaborators

• Nicoletta C Machin: lead
• Health Resources and Services Administration (HRSA): collaborator

Study Sites (1)

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, 15232, United States

RECRUITING

Related Publications (11)

• McLaughlin JM, Munn JE, Anderson TL, Lambing A, Tortella B, Witkop ML. Predictors of quality of life among adolescents and young adults with a bleeding disorder. Health Qual Life Outcomes. 2017 Apr 7;15(1):67. doi: 10.1186/s12955-017-0643-7.

  PMID: 28388906 BACKGROUND

• Kassebaum NJ, Jasrasaria R, Naghavi M, Wulf SK, Johns N, Lozano R, Regan M, Weatherall D, Chou DP, Eisele TP, Flaxman SR, Pullan RL, Brooker SJ, Murray CJ. A systematic analysis of global anemia burden from 1990 to 2010. Blood. 2014 Jan 30;123(5):615-24. doi: 10.1182/blood-2013-06-508325. Epub 2013 Dec 2.

  PMID: 24297872 BACKGROUND

• VanderMeulen H, Sholzberg M. Iron deficiency and anemia in patients with inherited bleeding disorders. Transfus Apher Sci. 2018 Dec;57(6):735-738. doi: 10.1016/j.transci.2018.10.015. Epub 2018 Nov 17.

  PMID: 30470664 BACKGROUND

• Lu Z, Machin NC. 2024. Assessment of bleeding severity and prevalence of iron deficiency among hemophilia b carriers by factor ix activity levels. Blood. 144(Supplement 1):2586-2586.

  BACKGROUND

• Chen YC, Chao TY, Cheng SN, Hu SH, Liu JY. Prevalence of von Willebrand disease in women with iron deficiency anaemia and menorrhagia in Taiwan. Haemophilia. 2008 Jul;14(4):768-74. doi: 10.1111/j.1365-2516.2008.01777.x. Epub 2008 May 17.

  PMID: 18498402 BACKGROUND

• Park MJ, Park PW, Seo YH, Kim KH, Park SH, Jeong JH, Ahn JY. The relationship between iron parameters and platelet parameters in women with iron deficiency anemia and thrombocytosis. Platelets. 2013;24(5):348-51. doi: 10.3109/09537104.2012.699641. Epub 2012 Jun 27.

  PMID: 22738419 BACKGROUND

• Liu S, Guo F, Zhang T, Zhu Y, Lu M, Wu X, He F, Yu R, Yan D, Ming Z, Shu D. Iron deficiency anemia and platelet dysfunction: A comprehensive analysis of the underlying mechanisms. Life Sci. 2024 Aug 15;351:122848. doi: 10.1016/j.lfs.2024.122848. Epub 2024 Jun 15.

  PMID: 38885879 BACKGROUND

• Mokhtar GM, Ibrahim WE, Kassim NA, Ragab IA, Saad AA, Abdel Raheem HG. Alterations of platelet functions in children and adolescents with iron-deficiency anemia and response to therapy. Platelets. 2015;26(5):448-52. doi: 10.3109/09537104.2014.931570. Epub 2014 Jul 15.

  PMID: 25026531 BACKGROUND

• Elstrott BK, Lakshmanan HHS, Melrose AR, Jordan KR, Martens KL, Yang CJ, Peterson DF, McMurry HS, Lavasseur C, Lo JO, Olson SR, DeLoughery TG, Aslan JE, Shatzel JJ. Platelet reactivity and platelet count in women with iron deficiency treated with intravenous iron. Res Pract Thromb Haemost. 2022 Mar 23;6(2):e12692. doi: 10.1002/rth2.12692. eCollection 2022 Feb.

  PMID: 35356666 BACKGROUND

• Halimeh S, Rott H, Kappert G, Siebert M. 2013. Establishment of a reference range for the pbac-score. Blood. 122(21):4772-4772.

  BACKGROUND

• Rodeghiero F, Tosetto A, Abshire T, Arnold DM, Coller B, James P, Neunert C, Lillicrap D; ISTH/SSC joint VWF and Perinatal/Pediatric Hemostasis Subcommittees Working Group. ISTH/SSC bleeding assessment tool: a standardized questionnaire and a proposal for a new bleeding score for inherited bleeding disorders. J Thromb Haemost. 2010 Sep;8(9):2063-5. doi: 10.1111/j.1538-7836.2010.03975.x. No abstract available.

  PMID: 20626619 BACKGROUND

MeSH Terms

Conditions

Hemostatic Disorders; Hemorrhage; Iron Deficiencies

Interventions

ferryl iron; Standard of Care

Condition Hierarchy (Ancestors)

Vascular Diseases; Cardiovascular Diseases; Hemorrhagic Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Study Officials

• Nicoletta Machin, DO

  University of Pittburgh

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Nicoletta Machin, DO

CONTACT

(412) 209-7280; MANCHINN@pitt.edu

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: June 30, 2025
• First Posted: July 24, 2025
• Study Start: August 8, 2025
• Primary Completion (Estimated): October 1, 2027
• Study Completion (Estimated): October 1, 2027
• Last Updated: October 3, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Data will be available beginning 6 months after publication and will remain accessible for 5 years.
• Access Criteria: Proposals should be directed to machinnc2@upmc.edu. Access will be granted following approval by our institutional review board or ethics committee and the execution of a data use agreement.

Locations

US (1)