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Open research questions in Hemophilia Treatment and Research

44 unresolved questions extracted from the limitations and future-work sections of 251 Hemophilia Treatment and Research papers in our library. Each links back to the study that raised it.

What the literature leaves open

  • There is a lack of universally accepted definition for standard half-life and extended half-life FVIII replacement products - The half-lives of FVIII replacement products are limited by the chaperone effect of VWF

    Individual Comparative PK Evaluation of Single-dose Octocog Alfa, Rurioctocog Alfa Pegol, and Efanesoctocog Alfa in Adults with Severe Hemophilia A · 2026 · DOI

  • Diagnosing acquired FXIII deficiency in patients with normal routine coagulation screening tests. Managing acquired FXIII deficiency in resource-limited settings. Investigating the relationship between HIV infection and acquired FXIII deficiency.

    Suspected acquired factor XIII deficiency in a man living with HIV: diagnostic and therapeutic challenges in recurrent spontaneous hemorrhage: a case report · 2026 · DOI

  • The diagnosis of acquired FXIII deficiency is challenging in resource-limited settings. The patient's condition was managed with on-demand cryoprecipitate replacement and meticulous perioperative planning, which may not be feasible in all settings. The case report is limited to a single patient and may not be generalizable to all patients with acquired FXIII deficiency.

    Suspected acquired factor XIII deficiency in a man living with HIV: diagnostic and therapeutic challenges in recurrent spontaneous hemorrhage: a case report · 2026 · DOI

  • Hemophilia is a bleeding disorder that affects patients' ability to perform daily activities. Functional assessment is a central component of modern care for hemophilia patients. There is a need for a validated Korean version of the HAL.

    Cross-cultural validation and reliability of the Korean version of the Haemophilia Activities List · 2026 · DOI

  • There is a lack of awareness of hemophilia in developing countries, leading to delayed diagnosis and unnecessary interventions. There is a need for more research on the treatment of hemophilic pseudotumor.

    Case Report: PSEUDOTUMOR OF THE MAXILLA AS INITIAL MANIFESTATION OF HEMOPHILIA IN A 3-YEAR-OLD MALE: A CASE REPORT · 2026 · DOI

  • The challenges of cardiac surgery in patients with haemophilia. The need for a multidisciplinary team approach and strict perioperative monitoring.

    Case Report: Successful concomitant surgical interventions for dilated ascending aorta and aortic valve insufficiency in a severe haemophilia A case · 2026 · DOI

  • Effective pain management in PwH is limited by the lack of valid, reliable pain assessment tools that can be readily incorporated into clinical practice.

    Reliability and validity of patient-reported outcomes measurement information system® (PROMIS®) measures of pain in adult males with hemophilia · 2026 · DOI

  • The lack of accurate data on the total number of patients with haemophilia A and B. The risk of acute hepatitis in patients with haemophilia. The high cost of commercial factor VIII concentrate.

    Treatment of haemophilia and related disorders in Britain and Northern Ireland during 1976-80: report on behalf of the directors of haemophilia centres in the United Kingdom. · 1983 · DOI

  • Further studies are needed to disclose the true incidence of chronic hepatitis in patients with haemophilia. Research is needed to determine the effects of different types of factor VIII and factor IX concentrates on the incidence of acute hepatitis.

    Treatment of haemophilia and related disorders in Britain and Northern Ireland during 1976-80: report on behalf of the directors of haemophilia centres in the United Kingdom. · 1983 · DOI

  • The comparison of VR and GF is limited by the different study designs and populations. The analysis is an unadjusted statistical evaluation rather than an adjusted indirect comparison. Gene therapy for hemophilia A is a complex and rapidly evolving field.

    Indirect comparison of gene therapies in hemophilia A with use of generative artificial intelligence · 2026 · DOI

  • Longer-term follow-up and direct comparative analyses are required to confirm the advantages of each therapy. Further evaluation is needed to determine the relative efficacy and safety of VR and GF. The development of new gene therapies for hemophilia A is an area of ongoing research.

    Indirect comparison of gene therapies in hemophilia A with use of generative artificial intelligence · 2026 · DOI

  • Hemophilia is a costly and resource-intensive condition to treat, and people with hemophilia may experience financial distress and delays in treatment. Social vulnerability, including factors such as poverty and lack of access to care, may exacerbate these challenges. The study highlights the need for further research into the development of interventions to address social vulnerability in people with hemophilia.

    Social vulnerability, access to care, and outcomes in hemophilia · 2026 · DOI

  • This study has several limitations. First, this was a cross-sectional study; therefore, we could not measure the temporality between SVI and outcomes, most notably for when/whether a participant relocated during the study. Second, our measure of SVI was based on zip codes and not census tracts, which are smaller units of measure and more precise. Furthermore, there may be other arealevel measures, such as the area deprivation index and social vulnerability metric, which encompass different sociodemographic factors that influence social vulnerability. The area deprivation index is an area-level measure similar to SVI that incorporates poverty level and housing costs at the neighborhood block level, 60-62 whereas the social vulnerability metric is a zip code–level measure using 24 composite factors. 63 However, little data exist comparing these aggregate assessments in the health care field. Third, the participant base was extracted from the Registry and highlights the HTC PP, but it may underrepresent those who did not receive their hemophilia care from an HTC or those who followed at HTC but chose not to participate in the Registry. Literature shows that ~67% to 82% of all persons with hemophilia and 86% to 94% of those with severe hemophilia receive their hemophilia care from an HTC, and 49% of those with hemophilia A and 42% of those with hemophilia B authorize their records to be included in Community Counts. 17,64 The Registry may be more representative of severe disease but may underrepresent those with mild hemophilia, as indicated above, in addition to those who are underinsured, lower income, have distance barriers to care, or live in states with less Medicaid coverage. 65 Racial and ethnic minority groups and MAY 2026 • VOLUME 3, NUMBER 2 100145 SOCIAL VULNERABILITY AND HEMOPHILIA 9 VTHvessels, thrombosis & hemostasis women are also less likely to be captured as well.

    Social vulnerability, access to care, and outcomes in hemophilia · 2026 · DOI

  • The study is limited by the availability of published data. The certainty of the body of evidence for the primary outcome was rated against the GRADE framework. The study did not report thromboembolic-event numerators per agent in their published abstracts or main text for all studies.

    Efficacy and Safety of Recombinant Activated Factor VII versus Activated Prothrombin Complex Concentrate for Bleeding Control in Acquired Haemophilia A: A Systematic Review and Meta-Analysis · 2026 · DOI

  • The comparative efficacy of rFVIIa and aPCC for bleeding control in acquired haemophilia A has not previously been quantified by formal meta-analysis. There is a need for further research on the comparative efficacy of rFVIIa and aPCC.

    Efficacy and Safety of Recombinant Activated Factor VII versus Activated Prothrombin Complex Concentrate for Bleeding Control in Acquired Haemophilia A: A Systematic Review and Meta-Analysis · 2026 · DOI

  • There is a need for an integrated interdisciplinary approach in the prevention of hemophilic arthropathy. There is a lack of understanding of the significance of modern bleeding prophylaxis, physical activity, and physiotherapy in preventing musculoskeletal complications in children with hemophilia.

    Hemophilia A in children: prophylaxis, physical activity and rehabilitation in the prevention of hemophilic arthropathy · 2026 · DOI

  • The small sample size of the available literature limited the ability to conduct robust inferential statistical analyses. The study is based on a systematic review of case reports.

    Intracranial hemorrhage in acquired hemophilia A: illustrative case · 2026 · DOI

  • Further studies are needed to establish the diagnosis and management of AHA-associated intracranial hemorrhage. Research on the use of bypassing agents and immunosuppression in AHA-associated intracranial hemorrhage is needed.

    Intracranial hemorrhage in acquired hemophilia A: illustrative case · 2026 · DOI

  • IN PRESSACCEPTED MANUSCRIPTARTICLE IN PRESS Future longitudinal studies are warranted to determine whether the Korean HAL is sensitive to functional changes associated with clinical interventions and long-term musculoskeletal outcomes. Therefore, additional studies involving contemporary treatment cohorts are warranted.

    Cross-cultural validation and reliability of the Korean version of the Haemophilia Activities List · 2026 · DOI

  • Study quality was mostly low (31%) to intermediate (56%). Better-designed studies are needed to confirm the evidence.

    Prophylaxis for inherited factor X deficiency: a systematic review · 2026 · DOI

  • There is a need for better-designed studies to confirm the evidence. The study aims to evaluate the efficacy and safety of prophylaxis in people with hereditary factor X deficiency.

    Prophylaxis for inherited factor X deficiency: a systematic review · 2026 · DOI

  • The etiopathogenesis of missed abortion is not fully understood, with no underlying cause identified in nearly 50% of cases. There is a need for new biomarkers and diagnostic tools for missed abortion. The relationship between hematologic inflammatory markers and missed abortion is not well understood.

    Evaluation of Hematologic Inflammatory Markers in Missed Abortus: A Retrospective Case-Control Study · 2026 · DOI

  • There is a need to assess the effectiveness of rFVIIIFc prophylaxis in people with haemophilia A across different age groups, body mass indexes, disease severities, and inhibitor histories.

    Post-Hoc Analyses of A-SURE and PREVENT Confirm the Effectiveness of rFVIIIFc Prophylaxis Across All Ages, BMIs, Severities and Inhibitor Histories · 2026 · DOI

  • Taken together, the contributions to this Research Topic illuminate three converging themes that are likely to define hemophilia care in the coming decade. First, precision medicine is now inescapable in hemophilia. Population pharmacokinetic modeling (Terasaka and McKeand), mutation-guided diagnosis (Peng et al.), and genotype-phenotype correlations (Dushimova et al.) all point toward an era in which treatment decisions—factor product choice, dosing interval, prophylaxis intensity—are individually tailored to each patient’s molecular and pharmacological profile. This shift demands investment in laboratory infrastructure, genomic testing, and clinical expertise, particularly in resource-limited settings. Second, the therapeutic frontier continues to advance rapidly but unevenly. Non-viral gene therapy (Kao et al.) and emicizumab for neonatal prophylaxis (Peng et al.) represent genuine innovations that may reshape the disease course for future patients. Yet as Dushimova et al. powerfully remind us, approximately 70% of patients worldwide today do not have access to the therapies that are already available. Bridging this access gap is as urgent a priority as developing the next generation of treatments. rare and severe complications of hemophilia demand clinical vigilance and institutional expertise. The cases of intracranial pseudotumor (Regmi et al.) and cardiac Third, surgery (Ursu et al.) underscore that hemophilia can produce life-threatening emergencies that tax even the most experienced multidisciplinary teams. Both cases demonstrate that with meticulous hemostatic management, previously considered unsurvivable or inoperable scenarios can be successfully navigated. Disseminating such institutional experience through case reports and guidelines is essential for raising standards globally. The editors express sincere gratitude to all contributing authors, reviewers, and editorial board members whose expertise and commitment made this Research Topic possible. We hope that these manuscripts stimulate further research, foster interdisciplinary collaboration, and ultimately contribute to a world in which every person with hemophilia—regardless of geography or socioeconomic circumstance—has access to safe, effective, and personalized therapy.

    Editorial: Hemophilia advances: from genetic insights to optimal therapy in 2025 · 2026 · DOI

  • Accurate assessment of bleeding severity and disease classification remains clinically challenging. There is a need for a reliable screening tool for VWD. The role of INTEM-ROTEM parameters in VWD identification and classification is not well understood.

    International Society on Thrombosis and Hemostasis Bleeding Assessment Tool (ISTH-BAT) and Intrinsic Rotational Thromboelastometry (INTEM-ROTEM) in the Evaluation and Classification of von Willebrand Disease (VWD): An Egyptian Center Cross-Sectional Observational Study. · 2026 · DOI

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44 open questions have been extracted from the limitations and future-work passages of 251 Hemophilia Treatment and Research papers in our library. Each one below links back to the study that raised it, so you can read the original claim in context.

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