Science AI Journal
Medicine · Research topic

Open research questions in Hemophilia Treatment and Research

58 gap statements mined from Hemophilia Treatment and Research papers in our 4.5M-paper local library, which holds 528 papers on the topic — drawn mostly from each paper's own stated research gap, future-work, challenge and limitation notes, and its abstract. The ones listed below are a selection still marked open; each names the study that raised it, with a DOI link where the paper has one.

Choosing where to publish on Hemophilia Treatment and Research? See the ranked Medicine journals.

What the literature leaves open

  • 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.

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

  • To our knowledge, the coexistence of HIV infection and acquired FXIII deficiency has not been previously reported, particularly in a virologically suppressed patient with relatively preserved CD4+ T-cell counts.

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

  • 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

  • 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

  • Bridging this access gap is as urgent a priority as developing the next generation of treatments.

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

  • Future multicenter studies with larger cohorts and genetic characterization are warranted to further clarify the role of viscoelastic testing in VWD evaluation.

    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

  • Although Brazil provides universal and free access to clotting factor concentrates (CFC) through the public health system, real‐world adherence remains insufficiently explored.

    Gaps Between Prescription and Practice: Pharmacy‐Based Measurement of Prophylaxis Adherence in Haemophilia · 2026 · DOI

  • Extended half-life (EHL)-FVIII concentrates, developed through Fc-fusion or PEGylation, extend terminal half-life and overall exposure area under the curve (AUC) by ∼30% compared with standard half-life (SHL) products but remain limited by the von Willebrand factor (VWF)-imposed half-life ceiling.

    A revised classification of FVIII concentrates: rationale and novel metrics · 2026 · DOI

  • More recent data pointed to liver sinusoidal endothelial cells (LSECs) as the primary source of FVIII, but conflicting reports and methodological limitations have obscured a clear understanding of hepatic and extrahepatic FVIII expression.

    Factor VIII originates primarily from anatomically distinct subsets of liver sinusoidal endothelial cells · 2026 · DOI

  • The BSH produces Good Practice Papers to recommend good practice in areas where there is a limited evidence base but for which a degree of consensus or uniformity is likely to be beneficial to patient care.

    Implementation and value of providing a multidisciplinary haematology and gynaecology service for individuals with bleeding and thrombotic disorders: A British Society for Haematology & United Kingdom Haemophilia Centre Doctors' Organisation Good Practice Paper · 2026 · DOI

  • Patients with end-stage haemophilic arthritis (HA) are often hesitant about joint replacement surgery, yet little is known about the decision experiences faced by these patients.

    Decision experiences in joint replacement surgery for patients with haemophilic arthritis: A qualitative study · 2025 · DOI

  • While we know that raising factor VIII levels for extended periods of time would reduce the frequency of injections required and provide more consistent bleed protection, the relationship between factor activity levels (FALs), clinical outcomes, and health-related quality of life (HRQoL) for people with haemophilia A (PwHA) has not been well characterised.

    The association of factor VIII activity levels with bleeding and quality of life in haemophilia a: findings from the European CHESS II study · 2025 · DOI

  • Few studies have evaluated direct medical or societal costs of haemophilia in the United Kingdom (UK), and how patient characteristics impact future costs is uncertain.

    Distribution and predictors of haemophilia-related costs in the United Kingdom: analysis of the CHESS I and CHESS II burden of illness studies · 2024 · DOI

  • The proteolytic activation of factor IX to form activated factor IX(a) and subsequent structural rearrangements are insufficient to generate the fully active factor IXa.

    Blood coagulation factor IX: structural insights impacting hemophilia B therapy · 2024 · DOI

  • Due to its known high prevalence, prior HA studies in Colombia have mostly focused on ana-lyzing the role of intron 22 inversion, but have not investigated the prevalence and role of variants located within the exonic region of F8.

    Comprehensive Screening of Genetic Variants in the Coding Region of F8 in Severe Hemophilia A Reveals a Relationship with Disease Severity in a Colombian Cohort · 2024 · DOI

  • Healthcare professionals should acknowledge patients’ spiritual beliefs and practices while reinforcing adherence to evidence-based medical treatment.

    Healing through faith: spirituality and shrine visitation as coping mechanisms among haemophilia patients in Kashmir—an interpretative phenomenological study · 2026 · DOI

  • Acquired hemophilia-A (AHA) is grossly underreported in the literature, and in pediatric patients, characterized by the presence of autoantibodies against factor-VIII (F-VIII), which can manifest as a wide spectrum of bleeding events.

    Successful management of an adolescent male with acquired hemophilia‐A with intracranial bleeding using emicizumab · 2024 · DOI

  • 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…

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

  • La falta de escalas específicas para la edad pediátrica puede dificultar el diagnóstico. La necesidad de un tratamiento personalizado para la enfermedad de von Willebrand.

    Trastornos de coagulación · 2026 · DOI

Most-cited papers in Hemophilia Treatment and Research

Most recent work

Find a gap in your own Hemophilia Treatment and Research sub-topic

This page shows what the Hemophilia Treatment and Research literature already flags as unresolved. To narrow it to your specific question, search the Research Gap Finder: the search is free with a free account and lists the papers closest to your topic first. Unlocking that topic (50 credits, charged once) fills the comparison table from our 4.5M-paper local library and writes the gaps from its rows.

Open the Research Gap Finder →

Related topics in Medicine

Artificial Intelligence in Healthcare and Education
2,304 open questions
Substance Abuse Treatment and Outcomes
1,956 open questions
Dementia and Cognitive Impairment Research
1,232 open questions
Ferroptosis and cancer prognosis
1,146 open questions
Liver Disease Diagnosis and Treatment
1,049 open questions
Smoking Behavior and Cessation
1,025 open questions
Physical Activity and Health
1,002 open questions
Attention Deficit Hyperactivity Disorder
982 open questions
Open questions raised by papers listed on this page include: (1) Further evaluation is needed to determine the relative efficacy and safety of VR and GF (“Indirect comparison of gene therapies in hemophilia A with use of generative…”, 2026); (2) Social vulnerability, including factors such as poverty and lack of access to care, may exacerbate these challenges (“Social vulnerability, access to care, and outcomes in hemophilia”, 2026); (3) There is a need for an integrated interdisciplinary approach in the prevention of hemophilic arthropathy (“Hemophilia A in children: prophylaxis, physical activity and rehabilitation in…”, 2026); (4) 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 (“Cross-cultural validation and reliability of the Korean version of the…”, 2026); (5) There is a need for better-designed studies to confirm the evidence (“Prophylaxis for inherited factor X deficiency: a systematic review”, 2026). 58 gap statements have been mined from Hemophilia Treatment and Research papers in our 4.5M-paper local library, which holds 528 papers on the topic; the gaps come from whichever of those papers state one. They are mostly the research gaps the authors state and the papers' abstracts, plus future-work, limitations and challenges passages. The ones listed below are a selection still marked open; each names the study that raised it, with a DOI link where the paper has one, so you can read the original claim in context.

Tools for your next paper

Gaps & Literature SearchFind open questions in any field — 120,000+ open gaps mined from our own library.Pre-CheckIs your paper ready? Acceptance odds for top, mid and open/emerging journals, in seconds.Journal FinderA ranked shortlist of target journals from a 17,500-venue index.Duplicate Publication CheckerCheck for prior publication across 7 sources and our local library.Citation GeneratorFreeA DOI or title → APA, MLA, Vancouver, Chicago, BibTeX, RIS. No signup.Graphical Abstract MakerFreeTurn your findings into a colorblind-safe graphical abstract.

How Pre-Check worksHow AI Review works

Compare the category — Honest roundups of the AI research tools, ours listed alongside the alternatives.

Command palette

Jump anywhere, run any action.