ReviewHematology. American Society of Hematology. Education Program2016
Treatment of rare factor deficiencies in 2016.
Review in Hematology. American Society of Hematology. Education Program, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
29 citing papers in PubMed, 80 citations in OpenAlex.
- Treatment of Deep Vein Thrombosis in Congenital Factor VII Deficiency: A Case Report and Literature Review.Clinical case reports · 2026Article
- Combined deficiency of factor V and factor VIII in a pediatric patient: a case report.Journal of medical case reports · 2025Article
- Severe Congenital Factor X Deficiency as a First Case Report in Cambodia.Case reports in hematology · 2025Article
- Article
- Impact of Replacement Therapy on Pregnancy Outcomes in Hemophilia Carriers: A Historical Cohort Study in Saudi Arabia.Life (Basel, Switzerland) · 2024Article
- A bispecific antibody approach for the potential prophylactic treatment of inherited bleeding disorders.Nature cardiovascular research · 2024Article
- Article
- Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for ultra-rare inherited bleeding disorders.Expert review of hematology · 2023Article
- Utility of ACMG classification to support interpretation of molecular genetic test results in patients with factor VII deficiency.Frontiers in medicine · 2023Article
- Laparoscopic hepatectomy for hepatocellular carcinoma in a patient with congenital factor V deficiency: a case report.Surgical case reports · 2022Article
- Perioperative management of intrahepatic cholangiocarcinoma patients with hereditary coagulation factor V deficiency: a case report and literature review.Translational cancer research · 2022Article
- The Vascular Endothelium and Coagulation: Homeostasis, Disease, and Treatment, with a Focus on the Von Willebrand Factor and Factors VIII and V.International journal of molecular sciences · 2022Review
- The Association between Factor XI Deficiency and the Risk of Bleeding, Cardiovascular, and Venous Thromboembolic Events.Thrombosis and haemostasis · 2022Article
- Standardization of Coagulation Factor V Reference Intervals, Prothrombin Time, and Activated Partial Thromboplastin Time in Mice for Use in Factor V Deficiency Pathological Models.Frontiers in veterinary science · 2022Article
- A case report of congenital factor X deficiency in an adult patient.SAGE open medical case reports · 2022Article
- Analysis of 180 Genetic Variants in a New Interactive FX Variant Database Reveals Novel Insights into FX Deficiency.TH open : companion journal to thrombosis and haemostasis · 2021Article
- Occurrence and management of severe bleeding episodes in patients with hereditary factor X deficiency.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Review
- A Rare Cause of Acquired Factor X Deficiency in an 87-Year-Old Female.Case reports in hematology · 2021Article
- Pharmacotherapeutic Patterns and Patients' Access to Pharmacotherapy for Some Rare Diseases in Bulgaria - A Pilot Comparative Study.Frontiers in pharmacology · 2021Article
- A Real-World Analysis of Commonly Prescribed FVIII Products Based on U.S. Medical Charts: Consumption and Bleeding Outcomes in Hemophilia A Patients.Journal of managed care & specialty pharmacy · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Rare bleeding disorders (RBDs) are a heterogeneous group of coagulation disorders characterized by fibrinogen, prothrombin, factors V, VII, X, XI, or XIII (FV, FVII, FX, FXI, or FXIII, respectively), and the combined factor V + VIII and vitamin K-dependent proteins deficiencies, representing roughly 5% of all bleeding disorders. They are usually transmitted as autosomal, recessive disorders, and the prevalence of the severe forms could range from 1 case in 500 000 for FVII up to 1 in 2-3 million for FXIII in the general population. Patients affected with RBDs may present a wide range of clinical symptoms, varying from mucocutaneous bleeding, common to all types of RBDs to the most life-threatening symptoms such as central nervous system and gastrointestinal bleeding. Treatment of these disorders is mainly based on the replacement of the deficient factor, using specific plasma-derived or recombinant products. In countries where these facilities are not available, bleedings could be managed using cryoprecipitate, fresh frozen plasma (FFP), or virus-inactivated plasma. Minor bleedings could be managed using antifibrinolytic agents. Recently, 2 novel drugs, recombinant FXIIIA and a plasma-derived FX, have been added to the list of available specific hemostatic factors; only prothrombin and FV deficiencies still remain without a specific product. Novel no-replacement therapies, such as monoclonal antibody anti-tissue factor pathway inhibitor, RNA interference, and a bispecific antibody that is an FVIIIa mimetic, enhancing thrombin generation through different mechanisms, were developed for patients with hemophilia and may in the future be a good therapeutic option also in RBDs.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.