Haemophilia
Haemophilia and its newer treatments, including gene therapy and non-factor drugs.
Papers per year
Each bar is 12 months; the last one ends 17 Oct 2026. New trials in the same years: 56, 65, 64, 53, 65.
New guidelines and consensus this year
- Recommendations for harmonizing definition, recording and reporting of the annual bleeding rate (ABR) in hemophilia: Communication from the ISTH SSC Subcommittee on Factor VIII, Factor IX and Rare Coagulation Disorders."J Thromb Haemost2026 Sep 9
- Assessment of documentation and reporting methods for annual bleeding rate in hemophilia clinical trials: communication from the ISTH SSC Subcommittee on Factor VIII, Factor IX, and Rare Coagulation DisordersJ Thromb Haemost2026 Sep 9
- The diagnosis and evaluation of women and girls with hemophilia and hemophilia carriers: guidance from the SSC of the ISTHJ Thromb Haemost2026 Sep
- The MAPTO survey: worldwide approaches on unmasking factor VIII inhibitors in children with emicizumab treatment: communication from the ISTH SSC Subcommittee on Factor VIII, Factor IX and Rare Coagulation DisordersJ Thromb Haemost2026 May
- Guideline for the management of hepatitis B and C infection and subsequent liver disease surveillance (where indicated) in people with congenital bleeding disorders: A joint guideline from the British Society of Haematology and the British Viral Hepatitis GroupBr J Haematol2026 Mar
All papers from the last 12 months
1,115 papers (18 Oct 2025 – 17 Oct 2026), newest first. Loading the full list…
- Discovery and optimization of marstacimab, a human monoclonal antibody targeting tissue factor pathway inhibitor for the treatment of hemophilia A and BMAbs2026 Dec 31PMID 42273738
- Care and commitment as catalysts for independence: the impact of hemophilia society support programs through ripple effect mappingInt J Qual Stud Health Well-being2026 Dec 31PMID 41930623
- Gene therapy for hereditary hematological disorders: From clinical breakthroughs to future horizonsMol Ther Nucleic Acids2026 Dec 8PMID 42761173Review
- Cost-effectiveness of etranacogene dezaparvovec compared to eftrenonacog alfa for the treatment of adult patients with severe and moderately severe haemophilia B in SwedenJ Med Econ2026 DecPMID 42829928
- Treatment satisfaction and patient preference for efanesoctocog alfa over previous hemophilia A treatment: Results from the XTEND-1 phase 3 clinical trialCurr Ther Res Clin Exp2026 DecPMID 42598540
- A rare but misleading cause of hematuria in hemophilia A: renal pelvic hemorrhage mimicking tumorPediatr Nephrol2026 DecPMID 42370986
- Precision glycoengineered AAV capsids enhance hepatocyte targeting and attenuated immune activation for liver-directed gene therapyBiomaterials2026 DecPMID 42308604
- Fine-grained evaluation of a domain-specific Q&A dataset to support trustworthy medical language modelsHealth Inf Sci Syst2026 DecPMID 42039929
- Bleeding rates, healthcare utilization, and costs among patients with hemophilia a without inhibitors treated with concomitant octocog alfa or extended half-life factor VIII while on emicizumab prophylaxisJ Med Econ2026 DecPMID 41823988
- Emicizumab enables rapid tolerance, reducing bleeding during low-dose ITI in pediatric hemophilia ABlood Vessel Thromb Hemost2026 NovPMID 42774944
More hematology trends
All specialties →DOACs in cancer-associated thrombosis
Direct oral anticoagulants for venous thromboembolism in people with cancer.
Myelofibrosis treatment
JAK inhibitors and newer drugs in myelofibrosis.
Key point: Ruxolitinib-based combination regimens showed improved spleen and symptom responses with acceptable safety, with efficacy significantly influenced by prior JAK inhibitor exposure.
Post-transplant cyclophosphamide
Cyclophosphamide after stem cell transplant to prevent graft-versus-host disease.
Key point: Post-transplant cyclophosphamide appears to reduce graft-versus-host disease without increasing relapse or mortality in matched related donor transplantation for acute leukemia.
Targeted therapy in CLL
BTK and BCL2 inhibitors in chronic lymphocytic leukaemia.
Key point: In CLL/SLL, treatment selection is tailored based on age, genetic risk and comorbidities, prioritizing zanubrutinib, acalabrutinib and venetoclax-based regimens.
Paroxysmal nocturnal haemoglobinuria
PNH and new oral complement inhibitors.
Menin inhibitors in AML
Revumenib, ziftomenib and other menin inhibitors in acute leukaemia.
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How these numbers are counted
PubMed query, searched in titles and abstracts by publication date:
hemophilia[tiab] OR haemophilia[tiab]Trials: ClinicalTrials.gov studies first submitted in each 12-month window that match the same terms. A query can miss synonyms or catch unrelated papers, so treat counts as approximate.