Haemophilia — Research Summary
Printed from RareWays (rareways.com.au) on 5 April 2026
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Care and commitment as catalysts for independence: the impact of hemophilia society support programs through ripple effect mapping.
Shroff Diksha et al. — International journal of qualitative studies on health and well-being (31 December 2026)
https://pubmed.ncbi.nlm.nih.gov/41930623/
- 2.
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 prophylaxis.
Montcrieff Caitlin et al. — Journal of medical economics (1 December 2026)
https://pubmed.ncbi.nlm.nih.gov/41823988/
- 3.
Clinical outcomes and health-system challenges in congenital afibrinogenemia: a single-centre prospective case series.
Radhakrishnan Nita et al. — Blood cells, molecules & diseases (1 May 2026)
https://pubmed.ncbi.nlm.nih.gov/41581368/
- 4.
Levodopa/Carbidopa Intestinal Gel (LCIG) Treatment in a Patient with Parkinson's Disease and Hemophilia A.
Havránková Petra et al. — Movement disorders clinical practice (2 April 2026)
https://pubmed.ncbi.nlm.nih.gov/41927462/
- 5.
Emicizumab prophylaxis in children with hemophilia A with factor VIII inhibitors in China (HAVEN 5).
Wu Runhui et al. — Chinese medical journal (2 April 2026)
https://pubmed.ncbi.nlm.nih.gov/41927303/
Clinical Trials — Currently Recruiting (Australia)
Ask your doctor whether you or your child may be eligible for any of these trials.
- 1.
A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions
Recruiting — Phase 3 — Takeda
https://clinicaltrials.gov/study/NCT06499285
- 2.
Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)
Recruiting — Phase 3 — CSL Behring
https://clinicaltrials.gov/study/NCT06003387
- 3.
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Recruiting — Phase 3 — Pfizer
https://clinicaltrials.gov/study/NCT05145127
- 4.
Study of Individuals Affected With Hypoplasminogenemia
Recruiting — Indiana Hemophilia &Thrombosis Center, Inc.
https://clinicaltrials.gov/study/NCT03797495
- 5.
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
Recruiting — Phase 3 — Pfizer
https://clinicaltrials.gov/study/NCT05611801
- 6.
A Study of Bleeding and Treatment in Participants With Von Willebrand Disease
Recruiting — Hemab ApS
https://clinicaltrials.gov/study/NCT06610201
- 7.
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
Recruiting — Phase 1 — Regeneron Pharmaceuticals
https://clinicaltrials.gov/study/NCT06379789
- 8.
A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease
Recruiting — Phase 3 — Novo Nordisk A/S
https://clinicaltrials.gov/study/NCT06612268
- 9.
Study of VGA039 in Healthy Volunteers and Patients With Von Willebrand Disease (VIVID)
Recruiting — Phase 1 — Vega Therapeutics, Inc
https://clinicaltrials.gov/study/NCT05776069
- 10.
Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
Recruiting — Phase 3 — Pfizer
https://clinicaltrials.gov/study/NCT05568719
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Haemophilia
Haemophilia is a rare inherited bleeding disorder caused by deficiency of clotting factor VIII (Haemophilia A) or factor IX (Haemophilia B), leading to prolonged bleeding after injury or surgery. Extended half-life factor replacement and non-factor therapies such as emicizumab have transformed management. Gene therapy is now approved in several countries.
Most Recent Research
BACKGROUND: Patient organizations play a vital role in supporting individuals with chronic disorders by offering a sense of community and shared understanding to improve patient outcomes. However, systematically evaluating the long-term impact of such organizational activities remains a challenge. Ripple effect mapping (REM), a participatory evaluation tool, offers a structured approach to visually map patient organizations' intended and unintended outcomes. METHODS: This study aimed to understand the impact of a Patient Organization for Hemophiliacs (POH) on people with hemophilia (PwH), who are registered members of the hemophilia patient organization. Data were collected through facilitated REM sessions, where participants collaboratively identified and mapped the direct and indirect impacts of the organization on their life and well-being. Thematic analysis was applied to categorize the emerging patterns and evaluate the long-term influence on the 30 PwH, collectively reflecting on POH's impact and generating consensus themes and a mind map. Data analysis was performed, and the data were entered into XMIND© mapping software and reviewed via group transcription and field notes. RESULTS: Thematic analysis identified five themes, including knowledge and awareness, support systems, care and management, community empowerment, and gratitude. The results of this study support the idea that POH has been a catalyst for transformative change among PwH. CONCLUSIONS: POH actively promoted autonomy, resilience, and meaningful community relationships. Using REM has been proven to show patient organisations' deeper, often subtle, consequences, emphasizing benefits that typical evaluation methods may overlook.
This information is for general awareness only.
For guidance specific to your situation, please speak with your healthcare team.