During a recent visit to Uzbekistan with the World Federation of Hemophilia (WFH) as part of their Humanitarian Aid Program, CSL’s Dr. Sujan Sivasubramaniyam witnessed both progress the health care system has made through partnership and ongoing gaps in care for people with inherited bleeding disorders in the central Asian country. Globally, 80–85% of people with hemophilia still lack reliable access to effective treatment, and in Uzbekistan, long travel distances, limited diagnostics, and access to treatment continue to pose significant challenges.
The team met several people with hemophilia whose health has suffered due to the lack of regular treatment with essential medicine that replaces the missing clotting factor in their blood. Families in rural areas often face long journeys to reach care, limited diagnostic testing, and low awareness of bleeding disorders, leading to late diagnoses and premature, irreversible joint damage. One man with severe hemophilia B endures serious joint pain after years without Factor IX, while another person with hemophilia A survived a coma before finally receiving donated medicine. Yet there were hopeful signs: a young person who began low-dose prophylaxis early now moves more easily and dreams of becoming a doctor.
During the trip, WFH leadership and Sivasubramaniyam met with medical staff, patient leaders from the Uzbek Society of Patients with Hemophilia and families to learn how care is changing. The group toured the Tashkent Hemophilia Centre, which treats adults and children and has 50 beds, including 10 for pediatric patients. Sivasubramaniyam saw firsthand how early prophylactic therapy is transforming lives: children gaining mobility and confidence, while hearing stories from older people highlighted the consequences of delayed care. Home visits underscored additional challenges, from long travel times to limited awareness, which often delay diagnosis and increase long-term complications.
Despite these obstacles, the trip revealed progress: The patient advocacy group is growing stronger, local healthcare providers and decision makers are increasingly engaged, and WFH donations are helping more people receive consistent treatment. These experiences underscored how sustainable humanitarian aid, combined with local leadership and long-term collaboration, can improve standards of care.
To better understand the challenges and opportunities observed during this trip, we asked Sivasubramaniyam to reflect on the people he met, the evolving health care landscape, and the WFH Humanitarian Aid Program in action.
How did the visit impact you?
The visit was deeply impactful because it highlighted both the progress and the persistent gaps in hemophilia and bleeding disorders care for people in Uzbekistan as well over 80% of people globally that still have little or no access to effective treatment.
It also reaffirmed CSL’s commitment to the impactful partnership with WFH and the wider patient advocacy community, to address unmet needs, improve awareness and diagnosis rates, support sustainable access to care, and balance inequities so together we can make a tangible difference in people’s lives.
What impressions did you gather about the circumstances there for people with bleeding disorder?
Seeing first-hand the resilience of people living with severe limitations due to inadequate access to treatment was humbling. Meeting individuals, who have endured years of pain, premature joint damage and limited quality of life because of insufficient factor replacement, especially for prophylactic use and hemophilia B reinforced the urgency of equitable access to care. Among them was a 27-year-old man with severe hemophilic arthritis in his left knee, a result of 10 years of recurrent bleeds due to a lack of reliable Factor IX. Knee replacement surgery is now his only option, illustrating the severe consequences of treatment gaps.
Encouragingly, the local patient organization is developing its capacity and capabilities, which is vital for long-term advocacy, meaningful change and improvement in standards of care. In Uzbekistan, being diagnosed with hemophilia once meant living with pain, uncertainty and very few options. Today, that reality is beginning to change. Access to treatment is growing, and the patient advocacy group in the country is becoming stronger and more involved.
What is your view of the WFH program after seeing their work in action?
The WFH Humanitarian Aid Program is transformative as it delivers a sustainable donation model coupled with local education, advocacy and capacity building. Led by Dr. Assad Haffar, their tireless support has dramatically improved hemophilia care in Uzbekistan over the past six years, reaching 112 countries worldwide since its inception. Despite progress, WFH’s estimate that 80–85% of people with bleeding disorders worldwide still lack effective treatment underscores the scale of the challenge and the importance of continued collaboration to close the global treatment gap.
CSL Behring has been donating treatments to the WFH Humanitarian Aid Program for nearly two decades. CSL Behring’s Antti Kourula, who is Vice President for Global Market Access, traveled to Kyrgyzstan with WFH officials in April.
About the WFH Humanitarian Aid Program
The WFH Humanitarian Aid Program improves access to care and treatment by providing much-needed support for people with inherited bleeding disorders in developing countries. By providing patients with a more predictable and sustainable flow of humanitarian aid donations, the WFH Humanitarian Aid Program makes it possible for patients to receive consistent and reliable access to treatment and care. None of this would be possible without the generous support of Sanofi and Sobi, our Founding Visionary Contributors; Bayer, CSL Behring and Roche, our Visionary Contributors; Grifols, our Leadership Contributor; and Takeda, our Contributor. To learn more about the WFH Humanitarian Aid Program, visit www.treatmentforall.org.
