ReviewDeutsches Arzteblatt international2019
Treatment Options in Hemophilia.
Review in Deutsches Arzteblatt international, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
14 citing papers in PubMed, 28 citations in OpenAlex.
- Overview of Patient-Reported Outcomes in Haemophilia Gene Therapy.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2026Review
- Article
- Advances in biopharmaceutical products for hemophilia.iScience · 2024Review
- A time trade-off study in the UK, Canada and the US to estimate utilities associated with the treatment of haemophilia.Health and quality of life outcomes · 2024Article
- Emicizumab as a Promising Form of Therapy for Type A Hemophilia - A Review of Current Knowledge from Clinical Trials.Current protein & peptide science · 2024Review
- Gene Therapy for Hemophilia-Opportunities and Risks.Deutsches Arzteblatt international · 2022Review
- 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 Application of Focused Medium-Energy Extracorporeal Shockwave Therapy in Hemophilic A Arthropathy.Healthcare (Basel, Switzerland) · 2022Article
- [Effect of FVIII doses on joint structure and function in adolescents with severe hemophilia A: mid-term results of a prospective cohort study].Nan fang yi ke da xue xue bao = Journal of Southern Medical University · 2021Article
- [Impact of COVID-19 Pandemic on Medical Care of Patients with Inherited Bleeding Disorders].Gesundheitswesen (Bundesverband der Arzte des Offentlichen Gesundheitsdienstes (Germany)) · 2021Article
- Hsa-miR-5581-3p and Hsa-miR-542-3p Target theMediterranean journal of hematology and infectious diseases · 2021Article
- Gene Therapy for Monogenic Inherited Disorders.Deutsches Arzteblatt international · 2020Article
- The German Hemophilia Registry: Growing with Its Tasks.Journal of clinical medicine · 2020Article
- Patient and Caregiver Preferences for Long-Term Prophylactic Treatment of Bleeds in Haemophilia: A Discrete-Choice Experiment.Haemophilia : the official journal of the World Federation of HemophiliaArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundApproximately 4550 persons were under treatment for hemophilia in Germany in 2017. The condition is currently treated with intravenous supplementa- tion of the missing clotting factor, either prophylactically or as needed. Newer treat- ment options rely on novel mechanisms of action.
methodsThis review is based on pertinent publications retrieved by a selective search in MEDLINE/PubMed, as well as on expert opinions and the recommenda- tions of specialty societies.
resultsRandomized controlled trials have shown that, in children aged 30 months to 6 years, prophylactic clotting-factor supplementation yields a markedly lower an- nual rate of hemorrhage than supplementation as needed: 3.27 (standard deviation [SD] 6.24) for the former vs. 17.69 (SD 9.25) for the latter. A similar large effect was seen in patients aged 12 to 50 years, with hemorrhage rates of 1.9 (SD 4.1) vs. 28.7 (SD 18.8). Clotting-factor preparations with longer half-lives make it possible to lessen the frequency of administration and to prevent subtherapeutic factor levels. A number of alternatives to clotting-factor supplementation have recently been approved or are currently being clinically tested. These new drugs are injected sub- cutaneously and have a longer half-life, possibly enabling better protection against bleeding than the current standard treatment. A further advantage of some of these drugs is that they can be given even in the presence of inhibitors to factor VIII. In addition, initial (phase I) clinical trials of gene therapy have been performed suc- cessfully for both hemophilia A and hemophilia B.
conclusionNow that new alternatives to classic supplementation therapy are be- coming available, pertinent treatment algorithms for patients with hemophilia will have to be developed. It is still unclear to what extent the new drugs might supplant clotting factor supplementation as the first line of treatment.
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What OpenQuestion holds
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.