Hemophilia Information Center
One place with in-depth information, up-to-date research, and practical tools
for people living with hemophilia and their families.
Dramatic progress is being made in treatment. Gene therapy, innovative medications, and modern prophylaxis are transforming lives. Here you will find research-based information.
What is Hemophilia?
Basic understanding of the disease
Treatments & Medications
Approved medications and new treatments
Clinical Trials
Active research worldwide
New Research
Breakthroughs and latest discoveries
Children & Hemophilia
Guide for parents and families
Healthy Lifestyle
Nutrition, sport and daily tips
Physiotherapy & Sport
Joint preservation and physical activity
Women & Hemophilia
Carriers, symptoms and pregnancy
Emergency Protocol
What to do in an emergency
Psychological Approach
Mental health and emotional coping
Success Stories
People living full lives
Links & Resources
Websites, organizations and tools
What is Hemophilia?
Basic understanding of the disease, types and symptoms
What happens in the body?
Hemophilia is an inherited bleeding disorder in which the blood does not clot properly due to a deficiency of specific clotting factors. In hemophilia A, factor VIII is missing; in hemophilia B, factor IX is missing. The genes for both factors are located on the X chromosome, so the condition primarily affects males. Women are typically carriers, though some experience symptoms. Hemophilia C (factor XI deficiency) is also known and affects both sexes via autosomal recessive inheritance.
Types of Hemophilia
Hemophilia A (Classic)
Factor VIII deficiency. Accounts for 80–85% of cases. Severity is determined by factor level: mild (5–40%), moderate (1–5%), severe (below 1%).
Hemophilia B (Christmas Disease)
Factor IX deficiency. Accounts for 15–20% of cases. Treatment is similar but uses a different factor. Alprolix and Idelvion allow injections once per week or once every two weeks.
Hemophilia C (Rosenthal Disease)
Factor XI deficiency. Autosomal recessive inheritance, affects both sexes. Generally milder than A and B. More common in certain populations including Ashkenazi Jewish individuals.
Acquired Hemophilia
Not inherited. Caused by autoantibodies against factor VIII. Rare: 1–4 per million per year. More common in older adults. Requires specialized treatment.
Main Symptoms
Joint Bleeds (Hemarthrosis)
Repeated bleeding mainly in the knees, elbows and ankles. Causes pain, swelling, local warmth and cumulative joint damage (hemophilic arthropathy).
Muscle Bleeds
Deep bleeding into muscles that can compress nerves and blood vessels. A bleed in the iliopsoas muscle (abdomen/hip) is a medical emergency.
Excessive Bruising
Large and frequent bruises, sometimes without an obvious cause. One of the first signs in young children.
Prolonged Bleeding
Bleeding that does not stop after cuts, surgeries, dental extractions or injuries. Also spontaneous internal bleeding in severe hemophilia.
The Good News
In recent decades there has been a dramatic revolution in hemophilia treatment. Life expectancy has risen from around 30 years in the 1960s to near-normal today. Preventive therapy (prophylaxis) prevents up to 95% of spontaneous bleeds. In 2022–2024, gene therapies were approved that promise fundamental change: Hemgenix for hemophilia B and Roctavian for hemophilia A.
Blood tests: PT (normal), aPTT (prolonged), specific factor levels (VIII, IX, XI). Genetic testing to identify the exact mutation and for carrier detection. Prenatal diagnosis is possible at week 11–14 (chorionic villus sampling) or week 15–20 (amniocentesis). PGD (pre-implantation genetic diagnosis) is also available. Genetic testing for carriers with a family history is recommended and widely available through specialist centers.
Treatments & Medications
Key medications, innovative therapies, and what to ask your doctor
The Principle of Treatment
The cornerstone of hemophilia management is prevention (prophylaxis), not just treating bleeds. Large studies have proven that regular preventive therapy reduces bleeds by 95%, prevents joint damage, and significantly improves quality of life. The World Health Organization (WHO), World Federation of Hemophilia (WFH) and ISTH all recommend prophylaxis as the standard of care.
| Medication | Type | What it does | Notes |
|---|---|---|---|
| Recombinant Factor VIII (Advate, Kovaltry, Eloctate) |
Factor replacement | Replaces the missing factor in hemophilia A | Standard treatment. IV injection 2–3 times per week. Eloctate has an extended half-life. |
| Recombinant Factor IX (BeneFIX, Alprolix, Idelvion) |
Factor replacement | Replaces the missing factor in hemophilia B | Alprolix and Idelvion: injection once per week to once every two weeks only. |
| Emicizumab (Hemlibra) |
Bispecific antibody | Mimics the action of factor VIII. Revolutionary: subcutaneous injection. | Approved in 2017. Reduces bleeds by 87%. Suitable for patients with inhibitors too. |
| DDAVP (Desmopressin) |
Factor releaser | Releases stored factor VIII from natural body reserves | Effective for mild hemophilia A only. Given by injection, infusion or nasal spray. |
| Tranexamic Acid (Cyklokapron) |
Antifibrinolytic | Prevents breakdown of an existing clot | Excellent for oral bleeding and after dental procedures. Available as tablets or mouthwash. |
Gene Therapy: Hemgenix
FDA-approved in November 2022 for hemophilia B. An AAV5 vector delivers a functional copy of the FIX gene to the liver. In the HOPE-B study: 94% of patients stopped routine prophylaxis. FIX levels rose to an average of 36.2% and remained stable even 3 years after a single treatment. Price: approximately $3.5 million for a one-time infusion.
Gene Therapy: Roctavian
Approved by the EMA in 2022 and the FDA in 2023 for severe hemophilia A. AAV5 vector carrying a truncated FVIII gene. 87% of patients stopped prophylaxis in the first year. Note: FVIII levels decline gradually over time. Long-term follow-up is required.
Fitusiran — Alhemo
An antibody against antithrombin. Subcutaneous injection once per month only. Reduces bleeds by 90%+. Suitable for hemophilia A and B, with or without inhibitors. Approved in Europe and Japan; FDA approval process ongoing.
Concizumab
An antibody against TFPI (Tissue Factor Pathway Inhibitor). Daily subcutaneous injection. Bleed reduction of 85%+. In advanced approval stages (Phase 3: explorer7, explorer8).
Mim8 — Novo Nordisk
A next-generation bispecific antibody (next generation after Emicizumab). Promising Phase 3 results (FRONTIER). Subcutaneous injection once per week or once per month. Expected FDA submission in 2025–2026.
The Biggest Challenge
About 30% of patients with severe hemophilia A and 3–5% of those with hemophilia B develop inhibitors (antibodies against the injected factor). This is the greatest challenge in hemophilia treatment today.
Bypassing Agents
FEIBA (aPCC) and NovoSeven (rFVIIa). These bypass the need for factor VIII/IX. Effective in 80–90% of bleeding episodes. Used for acute treatment and prevention.
ITI — Immune Tolerance Induction
Frequent high-dose factor injections over months to years. The goal: to "teach" the immune system to accept the factor. Success rate: 60–80% in hemophilia A. Expensive and complex but worth the investment.
Emicizumab for Inhibitors
Completely changed treatment for patients with inhibitors. Not affected by antibodies because it works through a different pathway. Reduced bleeds by 87% in the HAVEN 1 study (NEJM 2017).
Questions About Prophylaxis
- Am I receiving optimal prophylaxis?
- Is there a treatment with less frequent injections?
- What about Emicizumab?
- Am I a candidate for gene therapy?
Questions About Inhibitors
- What is the likelihood that I will develop inhibitors?
- What tests need to be done?
- When should ITI be considered?
- Is Emicizumab suitable for me?
Questions About Joints
- What is the condition of my joints (MRI/ultrasound)?
- Is there early joint damage starting?
- What physiotherapy is recommended?
- When should orthopaedic surgery be considered?
Clinical Trials
Active research worldwide — participation opportunities
Clinical Trials in Hemophilia
Over 300 active clinical trials in hemophilia are ongoing worldwide. Participating in a trial can offer access to cutting-edge therapies before they are widely available. Every trial is reviewed by an ethics committee, includes close medical monitoring, and contributes to research that benefits everyone.
Hemgenix: Long-Term Follow-Up
The HOPE-B study continues to follow hemophilia B patients who received gene therapy. Three-year data: FIX levels stable, 94% stopped prophylaxis. CSL Behring is continuing long-term monitoring.
Roctavian: Follow-Up and Expansion
BioMarin continues follow-up of hemophilia A patients. A key question: how long does the effect last? Data show gradual decline in FVIII levels in some patients.
Pfizer: giroctocogene fitelparvovec
Phase 3 trial for hemophilia A. A different approach: AAV6 vector instead of AAV5. Early results are promising. FDA submission expected in 2025.
Fitusiran: ATLAS Program
Multiple Phase 3 trials (ATLAS-A/B/INH/PPX). Subcutaneous injection once per month. Results: over 90% bleed reduction. Sanofi advancing regulatory approval.
Concizumab: explorer Program
Novo Nordisk: Phase 3 trials (explorer7, explorer8). Anti-TFPI antibody. Bleed reduction 85%+. Daily subcutaneous injection.
Mim8: FRONTIER Program
Novo Nordisk: next-generation bispecific antibody. Phase 3. Weekly or monthly subcutaneous injection. Excellent early results.
Find a Trial Near You
The WFH Clinical Trials Registry and ClinicalTrials.gov list all active hemophilia trials globally. Contact your local hemophilia treatment center to find out which trials you may be eligible for.
New Research
Breakthroughs and latest discoveries in hemophilia
A New Era in Hemophilia (2022–2025)
2022–2025 represent revolutionary years in hemophilia. Gene therapy, new non-factor medications, and advanced monitoring technologies are changing the face of the disease. Here are the most significant breakthroughs.
Gene Therapy: The Next Generation
After the approvals of Hemgenix and Roctavian, researchers are focused on improvements: more efficient vectors, lower doses, longer-lasting effect. Spark Therapeutics is working on SPK-8011 combined with immunosuppression. The potential for a genuine cure for the disease is now real.
The Non-Factor Therapy Revolution
Emicizumab changed the rules in 2017. Now Fitusiran, Concizumab and Mim8 are expanding the options. All are subcutaneous injections; all are suitable for patients with inhibitors as well. The future: personalized therapy tailored to each patient.
Joint Health: MRI Studies
New MRI studies show that early prophylaxis prevents joint damage almost entirely. HEAD2HEAD study (2024): comparison of Emicizumab with factor VIII shows a small advantage for Emicizumab in joint protection.
Personalized Treatment (PK-Guided)
Pharmacokinetic (PK) models allow factor dosing to be calibrated individually for each patient. Apps like myPKFiT predict factor levels at any moment. Result: fewer injections, greater protection.
New Biomarkers
Researchers are developing tools to predict bleeds before they happen. Markers of joint inflammation, new coagulation markers, and digital monitoring enable early intervention and damage prevention.
Children & Hemophilia
A guide for parents and families
Hemophilia in Childhood
Most severe hemophilia cases are diagnosed in the first year of life. Early diagnosis and preventive treatment allow children to grow and develop like any other child. Here is what is important to know.
Early Diagnosis
Most severe cases are diagnosed in the first year: unexplained bruising, prolonged bleeding after circumcision, joint bleeds when crawling begins. A simple blood test (aPTT + factor level) confirms the diagnosis. Newborn testing is recommended for families with a known history.
School and Play
It is important to inform the educational team. Prepare a written emergency action plan. Most physical activities are permitted: swimming, running, cycling, basketball. Avoid full-contact sports (boxing, rugby). Do not wrap the child in cotton wool.
Self-Treatment
From age 8–12, children can begin learning to self-infuse intravenously. A gradual process: watching, assisting, supervised infusion, independent infusion. Improves independence, confidence and quality of life. Hemophilia treatment centers offer professional training.
Emotional Support
Children with hemophilia may experience frustration, fear and isolation. Psychological support is important. Support groups for children and special summer camps (organized by WFH and NHF) are very helpful. Talk to your child openly about the condition.
Dental Care
Aggressive preventive maintenance: brushing with fluoride toothpaste, dental floss, visits every 6 months. Before any invasive procedure: coordinate with the hematologist. Tranexamic acid around procedures. Hospital dental clinics are familiar with hemophilia.
Transition to Adulthood
Transition clinics from age 16–18. Gradual transfer of responsibility from parents to the adolescent. Introduction to the adult hemophilia clinic. Topics include family planning, genetic counseling, and career choice. WFH offers dedicated transition programs.
Healthy Lifestyle
Nutrition, sport and daily tips
Living Fully with Hemophilia
Living with hemophilia does not mean giving things up. With proper preventive treatment and planning, you can travel, work, play sport and live a full life. Here is what is worth knowing.
Nutrition
Maintaining a healthy weight is important for joint protection. Vitamin D and calcium to prevent osteoporosis (common in hemophilia). Iron if bleeds are frequent. Avoid high-dose omega-3 supplements (may affect clotting). A Mediterranean diet is recommended.
Physical Activity
Swimming, walking, cycling, Pilates and yoga are excellent. Strengthen muscles around joints. Avoid full-contact sport (boxing, rugby, ice hockey). Always coordinate with your hematologist and physiotherapist. Regular physical activity actually reduces bleeds.
Dental Care
Thorough daily dental cleaning. Visits every 6 months. Coordinate with the hematologist before any invasive procedure. Tranexamic acid before and after procedures. Prefer dental clinics at hospitals familiar with hemophilia.
Travel
Carry double the usual medication supply. A medical letter in English from the hematologist. List of hemophilia centers at your destination (WFH Global Treatment Centre Directory). Travel insurance covering pre-existing conditions. Keep medications in carry-on luggage (not checked baggage).
Emergency Preparedness
Always wear a medical identification bracelet or necklace. An emergency kit at home and in the car (medication, emergency numbers, medical letter). Treatment center emergency numbers saved on your phone. The HemMobile app for tracking bleeds and infusions.
Medications to Avoid
Aspirin and NSAIDs (such as ibuprofen, diclofenac) increase bleeding. Paracetamol (acetaminophen) is safe. Always inform every doctor and pharmacist that you have hemophilia. Be careful with herbal products that affect clotting (ginkgo, garlic, ginseng).
Physiotherapy & Sport
Joint preservation and physical activity
Physical Activity as Part of Treatment
Regular physical activity is a critical part of hemophilia management. Strong muscles protect joints, reduce bleeds, and improve quality of life. Studies from 2024 showed that a tailored physiotherapy program reduces joint bleeds by 40%.
Swimming and Hydrotherapy
The most recommended activity for hemophilia. Zero load on joints. Strengthens muscles. Suitable even after a joint bleed and during rehabilitation. Souza et al., 2024: swimming improved range of motion by 30% in hemophilia patients.
Cycling
Low joint load. Strengthens the legs. A stationary bike is preferable (safer than outdoor cycling). 30 minutes, 3 times per week. Strengthens thigh muscles that protect the knees.
Yoga and Pilates
Improves flexibility, strength and coordination. Reduces chronic pain. Strengthens the core. Cakmak et al., 2022: yoga improved quality of life by 40% in hemophilia patients. Choose an instructor who knows how to make adaptations.
Strength Training
Strengthening muscles around vulnerable joints is critical. Light to moderate weights, 2–3 sets of 12–15 repetitions. Physiotherapist guidance is essential at the start. Avoid very heavy weights and explosive movements.
Sports to Avoid
Full-contact sports (boxing, karate, rugby, ice hockey), sports with a high fall risk (skiing, skateboarding, horse riding), very heavy weightlifting. Always consult with your hematologist and physiotherapist before starting a new activity.
Tip: WFH Sport Risk Categories
The WFH recommends Risk Categories for sport. Category 1 (safe): swimming, walking, cycling. Category 2 (moderate): basketball, football, tennis. Category 3 (high risk): boxing, rugby, ice hockey. Choose from Category 1–2 and ensure adequate prophylaxis.
Women & Hemophilia
Carriers, symptoms, pregnancy and childbirth
Not Only Men
Hemophilia does not only affect men. Female carriers can suffer from significant symptoms, and approximately 30% of carriers experience increased bleeding. It is important for carrier women to have their factor levels checked.
Carriers
Women with a mutation in the F8 or F9 gene on one X chromosome. Factor levels can range from 10–100% (due to random X-inactivation). Genetic testing is recommended for families with a known history. Specialist hemophilia centers provide carrier testing and counseling.
Heavy Menstrual Bleeding
About 60% of carriers suffer from heavy menstrual bleeding (menorrhagia). Treatments: tranexamic acid, hormonal contraceptives, levonorgestrel intrauterine device (IUD), DDAVP. Anyone with heavy periods and a family history of hemophilia should be evaluated.
Pregnancy and Childbirth
Pregnancy in a hemophilia carrier requires close monitoring by a hematologist, obstetrician and anesthesiologist. Factor VIII levels naturally rise during pregnancy (partial protection), but FIX levels do not. Epidural analgesia is possible only if factor levels are above 50%. Advance planning of delivery is essential.
Prenatal Diagnosis
Chorionic villus sampling (CVS) at weeks 11–14, amniocentesis at weeks 15–20. PGD (pre-implantation genetic diagnosis) allows selection of embryos without the mutation. NIPT (non-invasive prenatal testing) can determine fetal sex. Genetic testing options are available through specialist centers worldwide.
Emergency Protocol
What to do in a bleeding emergency
The Rule: Treat First, Ask Questions Later
Active bleeding in hemophilia requires prompt treatment. When in doubt, give factor. An intracranial bleed is the number one cause of death in hemophilia and requires immediate factor replacement plus emergency care.
Intracranial Bleed (Absolute Emergency)
Signs: sudden severe headache, vomiting, confusion, loss of consciousness, one-sided weakness. Action: give factor immediately — do not wait for tests. Go to the emergency room immediately. Even a head injury without symptoms requires immediate factor replacement and 24-hour monitoring.
Joint Bleed
Signs: pain, swelling, local warmth, difficulty moving. Action: RICE (Rest, Ice 15 minutes, Compression with an elastic bandage, Elevation) + factor replacement. If this is a first bleed in a new joint: see a doctor within 24 hours.
Muscle Bleed
Signs: deep pain, swelling, difficulty moving. Iliopsoas bleed (abdominal/hip muscle): abdominal or hip pain, difficulty straightening the leg. This is an emergency. Action: factor replacement + rest. Physiotherapy only after the bleed has stopped.
External Bleeding That Won't Stop
Direct continuous pressure on the wound for 15–20 minutes (do not lift the dressing to check). If it does not stop: factor replacement + emergency room. Tranexamic acid for oral bleeding.
After Dental Treatment
Tranexamic acid 3 times daily for 5–7 days. Soft, cold food for the first 48 hours. Do not rinse the mouth forcefully. If bleeding recurs: tranexamic acid + factor replacement.
When to Go to the Emergency Room
| Situation | Action |
|---|---|
| Sudden severe headache + vomiting | Immediate emergency room + factor |
| Bleeding that does not stop after 20 minutes of pressure | Emergency room |
| Neck/throat bleed (risk of airway obstruction) | Immediate emergency room + factor |
| Abdominal bleed (pain + swelling) | Emergency room + factor |
| Head injury (even without symptoms) | Immediate factor + 24-hour monitoring |
| Eye bleed (pain + vision change) | Emergency room + factor |
| Blood in urine or stool | Contact doctor the same day |
Psychological Approach
Mental health, emotional coping and living with hemophilia
The Mind is Part of Treatment
Current research shows that more than 40% of people with hemophilia experience symptoms of depression, and 55% report anxiety (Frontiers in Public Health, 2024). World Federation of Hemophilia (WFH) guidelines establish that psychological support is an inseparable part of comprehensive multidisciplinary care. Mental health is not a "bonus" — it is an essential component of disease management.
Key Psychological Challenges
Fear of Bleeding and Activity Avoidance
84% of patients report avoiding physical activity, and 73.7% have difficulty participating in sport and hobbies. Fear of bleeding creates a negative cycle: avoidance of activity leads to muscle weakness, which increases vulnerability to bleeds. This phenomenon is called kinesiophobia (fear of movement) and is documented in many studies. Breaking the cycle requires combined professional support from a psychologist and physiotherapist.
Chronic Pain and Psychological Impact
40–70% of people with hemophilia suffer from chronic pain resulting from hemophilic arthropathy (cumulative joint damage). The American PFiQ study found that 62% reported current pain and 85% had experienced pain in the previous four weeks. Pain, depression and anxiety form a self-reinforcing cycle: pain intensifies depression, depression reduces coping with pain, and anxiety amplifies the perception of pain.
Stigma and Social Isolation
Many patients conceal their diagnosis out of fear of rejection and stigma. Misconceptions persist, including confusion that hemophilia is contagious. Children are particularly affected and struggle to build close friendships. A 2025 study (Frontiers in Psychiatry) documents that stigma damages interpersonal relationships and leads to social isolation. Recommended interventions include structured family education, peer-led support groups, and awareness-raising among medical teams.
Psychological Barriers to Treatment Adherence
57% of patients aged 25–40 struggle with adherence to prophylaxis. Psychological barriers include: denial (especially common in adolescence), fear of needles, inaccurate health beliefs, depression that reduces motivation, and lifestyle conflicts. Depression, anxiety and pain each independently predict lower treatment adherence.
By Age: Unique Challenges
Children: Fear of Needles and Restrictions
Fear of needles is a major source of distress in children with hemophilia. Anxiety causes vein constriction, making infusion harder and creating a negative cycle. Evidence-based approaches: medical play with real equipment on dolls, topical anesthetic cream (EMLA), the Buzzy device (vibration and cooling), distraction techniques (bubbles, screens, games), gradual exposure from watching to self-injection, and giving the child a sense of control (choice of arm, position, timing). Beyond injections, children also cope with activity restrictions, feeling different from peers, and school difficulties.
Adolescents: Transition, Identity and Self-Management
Adolescence is particularly challenging. Self-infusion begins on average at age 12.3, but full self-management is achieved only at an average age of 22.6. Treatment adherence during adolescence is at its lowest, leading to repeated bleeds and cumulative damage. Disease denial is a common mechanism at this age. Adolescents also navigate decisions about disclosure (whom to tell), building a social identity, peer pressure, and the transition from pediatric to adult care. Telehealth education has shown significant improvement in transition readiness and self-efficacy.
Parents and Caregivers: Emotional Burden
65.4% of parents report an emotional impact from hemophilia, and 92.3% feel financial strain. Mothers sometimes experience guilt (because the condition is transmitted through the X chromosome). The central conflict: wanting the child to grow up normally versus the need to protect against bleeds. "Healthy" siblings may be pushed to the margins when care demands dominate family life. Parents lose an average of 8.35 work days per year. It is important for parents to also receive psychological support, not only the child.
The Good News: There Are Tools That Work
Studies show that psychological interventions in hemophilia are effective. Cognitive Behavioral Therapy (CBT), mindfulness, support groups and empowerment programs have been proven to improve quality of life, emotional regulation and coping with pain. You do not have to cope alone.
Evidence-Based Tools and Interventions
Cognitive Behavioral Therapy (CBT)
CBT has been specifically proven effective in hemophilia for improving emotional regulation and quality of life (Cochrane, 2020). Combining CBT with physiotherapy simultaneously reduced chronic pain and kinesiophobia. Therapy helps halt negative thoughts ("the bleed will never stop"), manage anger, and cope better in social situations. Recommended for any patient dealing with chronic pain, anxiety or depression.
Mindfulness and Pain Management
A multimodal approach to pain management is the professional standard (expert recommendations, PMC 2021). Beyond medication, recommendations include: Mindfulness-Based Stress Reduction (MBSR), guided imagery, biofeedback, hypnosis, hydrotherapy, and electrical stimulation (TENS). The combined approach includes: clotting factor treatment, analgesic medication, physiotherapy, psychological therapy, and complementary treatments such as acupuncture.
Support Groups and Community
Meeting others living with the same condition reduces isolation and provides a sense of belonging. Organizations such as the WFH and NHF run support groups for patients and families, summer camps for children, events and meetups, and assistance with accessing equipment and services. Online communities and telehealth programs offer additional access. The WFH also facilitates global peer support networks.
International Resources
Major hemophilia treatment centers worldwide operate multidisciplinary teams including psychologists and social workers. Contact your nearest World Federation of Hemophilia (WFH) member organization for local support resources. The NHF (National Hemophilia Foundation) and European Haemophilia Consortium (EHC) also maintain directories of patient support services. Do not hesitate to ask for help — seeking support is a sign of strength, not weakness.
When to Seek Professional Help
If you are experiencing persistent sadness, changes in sleep or appetite, withdrawal from social activities, avoidance of treatment, or feelings of hopelessness, contact your hemophilia treatment center team. Teams include psychologists and social workers familiar with the unique challenges of living with hemophilia. You are not alone.
Success Stories
People living full lives with hemophilia
Full Lives with Hemophilia
Today, a child born with hemophilia in a country with access to modern treatment can expect a long, active and full life. Modern preventive therapy enables sport, work, travel and family life. Here are some inspiring examples.
Alex Dowsett
British professional cyclist with severe hemophilia A. He broke the world hour record in 2015 (52.937 km). He proved that even with hemophilia it is possible to reach the pinnacle of sport. Founder of the Little Bleeders charity for children with hemophilia.
Ryan White
Born with hemophilia in 1971. He contracted HIV from contaminated blood products. He became a symbol of the fight against stigma and changed US legislation (Ryan White CARE Act 1990). His story transformed blood product safety forever.
The Global Community
The World Federation of Hemophilia and national member organizations run activities, summer camps and family meetups worldwide. Over 1.2 million people with hemophilia globally benefit from advancing science and an expanding network of hemophilia treatment centers.
The Disease That Changed Blood Medicine
Thanks to the hemophilia community, enormous advances have been made in blood medicine: recombinant technology, blood product safety, gene therapy. Achievements that are transforming the lives of millions of people with various bleeding disorders around the world.
Links & Resources
Websites, organizations, tools and useful apps
Leading Organizations
World Federation of Hemophilia (WFH)
The leading international organization. Information, guidelines, global treatment centre directory.
wfh.orgNational Hemophilia Foundation (NHF)
The US organization. Comprehensive information, research, community and advocacy.
hemophilia.orgEuropean Haemophilia Consortium (EHC)
The European organization. Guidelines, advocacy and research.
ehc.euHemophilia Federation of America
Patient advocacy, education, and community resources for people with bleeding disorders in the United States.
hemophiliafed.orgMajor Treatment Centers Worldwide
WFH Global Treatment Centre Directory
The WFH maintains a comprehensive directory of hemophilia treatment centers in over 130 countries. Use it to find an accredited center near you or when traveling.
Find a CenterUS Hemophilia Treatment Centers
The US has over 140 federally funded hemophilia treatment centers providing comprehensive multidisciplinary care. Managed by the American Thrombosis and Hemostasis Network (ATHN).
ATHN HTC DirectoryEuropean Centers of Expertise
The European Reference Network on Rare Haematological Diseases (ERN-EuroBloodNet) coordinates hemophilia care across Europe.
ERN-EuroBloodNetPediatric Hemophilia Centers
Many comprehensive hemophilia treatment centers have dedicated pediatric programs. Ask your national hemophilia organization for the nearest center with a specialist pediatric team.
Recommended Apps
| App | What it does | Cost |
|---|---|---|
| HemMobile | Track bleeds, infusions and medications | Free |
| myPKFiT | Personal factor level prediction | Free |
| Haemophilia Physio | Physiotherapy exercises tailored to hemophilia | Free |
| HemDiary | Bleed diary and joint monitoring | Free |
Key Research Articles (Citations)
Gene Therapy
- Pipe et al. (2023) NEJM: Hemgenix 3-year follow-up
- Mahlangu et al. (2023) NEJM: Roctavian Phase 3
- Ozelo et al. (2022) NEJM: Hemgenix HOPE-B results
Non-Factor Therapies
- Oldenburg et al. (2017) NEJM: Emicizumab HAVEN 1
- Young et al. (2023): Fitusiran ATLAS results
- Shapiro et al. (2023): Concizumab explorer5
Guidelines and Reviews
- Srivastava et al. (2020): WFH Guidelines 3rd edition
- Mannucci (2020) Haematologica: 50 years of treatment
- Manco-Johnson et al. (2007) NEJM: Joint Outcome Study
Recommended Books
Raising a Child with Hemophilia
Laurie Kelley. A comprehensive guide for parents. Covers all aspects: treatment, school, sport, emotion.
My Blood Doesn't Clot
A children's book that explains hemophilia in simple language. Excellent for children aged 4–8.
Guidelines for the Management of Hemophilia
WFH, 2020. The official guidelines. Free to download from the WFH website.
Download