Doris Quon, MD, PhD, is the medical director of the Orthopaedic Hemophilia Treatment Center at the Orthopaedic Institute for Children in Los Angeles.

Doris Quon, MD, PhD

Advances in prophylaxis have transformed bleeding prevention in 

, but patients with established joint damage may continue experiencing chronic pain even when bleeding is well controlled, according to Doris Quon, MD, PhD, medical director of the Orthopaedic Hemophilia Treatment Center at the Orthopaedic Institute for Children in Los Angeles.

, Quon discussed the importance of distinguishing chronic arthropathy pain from pain caused by ongoing joint bleeding, as well as the growing emphasis on physical function and independence in hemophilia care.

PTHemophilia A Care Beyond Zero Bleeds, With Doris Quon, MD, PhD

Hemophilia A Care Beyond Zero Bleeds, With Doris Quon, MD, PhD

The burden of joint disease remains substantial despite prophylaxis. In a cross-sectional survey of adults with moderate or severe hemophilia A without inhibitors across 5 European countries, nearly two-thirds had hemophilia-affected joints. Patients with affected joints reported greater pain, more frequent pain medication use, and lower health-related quality of life than those without affected joints.¹

Identifying Joint Bleeds With Point-of-Care Ultrasound

“The first thing I would do is to distinguish whether or not this is chronic pain versus acute on chronic pain,” Quon said.

An important part of that assessment is determining whether patients are experiencing subclinical bleeding, or microbleeds, that may be contributing to their symptoms.

At Quon's center, point-of-care ultrasound is incorporated into clinical assessments alongside a detailed patient history to help identify potential sources of joint pain.

Supporting this approach, research involving the Joint tissue Activity and Damage Exam (JADE), a standardized musculoskeletal ultrasound protocol designed for use by nonradiologists, found that ultrasound views helped detect hemarthrosis associated with joint pain. Quon was a coauthor of the study, which evaluated the consistency of serial ultrasound measurements and their relationship to joint health parameters in patients with hemophilia.²

When ultrasound findings indicate an active bleed, Quon described treating with factor replacement. In the absence of evidence suggesting ongoing bleeding, the focus shifts toward managing chronic pain associated with existing joint damage.

A Multimodal Approach to Hemophilic Arthropathy Pain

For patients with chronic joint pain unrelated to an active bleed, Quon described an individualized, stepwise approach combining pharmacologic treatment, physical therapy, and orthopedic evaluation when needed.

Initial medication options include acetaminophen and selective cyclooxygenase-2 (COX-2) inhibitors, such as celecoxib, which Quon's center uses. Depending on the patient's symptoms and response to treatment, additional medications, including gabapentin, may be considered.

Physical therapy is another component of management, helping patients address pain and maintain function without relying exclusively on medication.

For persistent symptoms, Quon may refer patients for orthopedic evaluation to consider additional interventions, including joint or corticosteroid injections.

The approach reflects a challenge in managing established hemophilic arthropathy: Although prophylaxis can reduce the risk of additional joint bleeding, it cannot necessarily reverse existing joint damage or the chronic pain associated with it.

Moving Beyond Zero Bleeds Toward Greater Function

Quon also described how advances in hemophilia A treatment have changed expectations for long-term care.

Historically, treatment focused primarily on preventing life-threatening bleeding events, with annualized bleeding rates serving as a key measure of efficacy. As prophylactic options improved, achieving zero bleeds became an increasingly attainable goal, with Quon noting that more than half of patients in some clinical trials now reach that benchmark.

However, the availability of extended half-life factor products, nonfactor therapies, and gene therapy has also created opportunities to consider outcomes beyond bleeding prevention.

For Quon, the next step is improving physical function, independence, and what she described as a “hemophilia-free mind,” in which patients are less consumed by the daily demands and limitations of their condition.

Reducing treatment burden is part of that shift. With newer therapies, some patients no longer require factor infusions 3 or 4 times weekly, while improved bleeding control can reduce concerns about activities that might previously have been considered too risky.

For patients living with established arthropathy, however, addressing persistent pain remains an important part of helping them benefit from these advances. The continued burden of joint disease despite prophylaxis, as documented in the European survey, underscores why bleeding outcomes alone may not capture patients' overall treatment experience.¹

“We’ve started transitioning away from thinking that it’s just all about bleeds and not bleeding to having more function and more independence,” Quon said, “being able to not limit yourself to just desk work, but be able to enjoy activities, hiking, biking, things like that.”

Editor's Note: Disclosures for Quon include Bayer, BioMarin, and Sanofi.

1. De la Corte-Rodríguez H, et al. Assessment of joint health in patients receiving prophylaxis for haemophilia A: a cross-sectional survey in five European countries. BMJ Open. 2024;14(9):e082204. 

https://bmjopen.bmj.com/content/14/9/e082204

2. Barnes RFW, Aguero P, Hanacek C, et al. Consistency of serial ultrasonographic joint tissue measurements by the Joint tissue Activity and Damage Exam (JADE) protocol in relation to hemophilic joint health parameters. BMC Musculoskelet Disord. 2023. doi:10.1186/s12891-023-06419-5

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A treatment center director explains how point-of-care ultrasound separates subclinical bleeds from chronic joint pain.

Doris Quon, MD, PhD

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